Paulness
1 week ago
NEWS -- Theriva™ Biologics Announces Positive Topline Data from Investigator Sponsored Phase 1 Trial of Intravitreal VCN-01 in Pediatric Patients with Refractory Retinoblastoma
Phase 1 trial in collaboration with Sant Joan de Déu-Barcelona Children’s Hospital (SJD) determined to have a positive outcome by the study Monitoring Committee
Safety and clinical outcomes support the therapeutic potential of VCN-01 in retinoblastoma and emphasize VCN-01’s potential for use in diverse cancer indications
ROCKVILLE, Md., April 23, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced positive topline data from the investigator sponsored Phase 1 trial conducted by collaborators at Sant Joan de Déu-Barcelona Children’s Hospital (SJD). The Phase 1 trial was designed to evaluate the safety and tolerability of two intravitreal injections of Theriva’s investigational oncolytic adenovirus VCN-01 in patients (n=9) with intraocular retinoblastoma that is refractory to chemotherapy or radiotherapy, and for whom enucleation was the only recommended treatment.
“Results from the investigator sponsored trial further validate VCN-01’s unique mechanism of action and therapeutic potential to improve patient outcomes in otherwise refractory cancers,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “We look forward to building on the encouraging safety profile and antitumor activity, which further supports and informs the design of our proposed Phase 2 clinical trial. The Monitoring Committee determined that the trial results were positive, and therefore, Theriva will receive an exclusive, worldwide license, and related patents from SJD for the treatment of pediatric patients with advanced retinoblastoma. The positive completion of this trial is an important step in refining our clinical strategy for VCN-01 as an adjunct to chemotherapy to address the high unmet need in this underserved indication.”
Key Takeaways: Patients received two intravitreal injections of VCN-01, 14 days apart, at a dose of either 2 x 109 vp/eye (n=1) or 2 x 1010 vp/eye (n=8). The data for 9 evaluable patients were reviewed by the study Monitoring Committee who agreed that the trial had a positive outcome:
Safety: VCN-01 was well tolerated after intravitreal administration at the 2 doses and the most frequently reported treatment-related adverse events were Grade 1 or 2. There were no dose limiting toxicities and no ocular or systemic toxicities equal to or greater than Grade 3 during the evaluation period.Some degree of ocular inflammation and associated turbidity was observed after VCN-01 injection. Inflammation was managed, and vitreous haze improved in some cases, by local and systemic administration of anti-inflammatory drugs.Antitumor effects: intravitreal VCN-01 demonstrated promising antitumor activity and did not appear to change the retinal function.Four patients presented a response characterized by unequivocal improvement in vitreous seed density.Eye enucleation was avoided in 3 patients to date, one of whom has retained their eye after 4 years of follow-up.About Retinoblastoma
Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 - 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In the U.S., retinoblastoma shows an incidence rate of 3.3 per 1,000,000 with only about 200 to 300 children diagnosed per year according to the American Cancer Society. Preserving life and preventing the loss of an eye, blindness and other serious effects of treatment that reduce the patient’s life span or the quality of life, remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries.
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company is advancing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. The Company’s lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients; and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at https://www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding regarding the therapeutic potential of VCN-01 to improve patient outcomes in otherwise refractory cancers, building on the encouraging safety profile and antitumor activity, Theriva receiving an exclusive, worldwide license, and related patents from SJD for the treatment of pediatric patients with advanced retinoblastoma and VCN-01 demonstrating promising antitumor activity Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s and VCN’s ability to reach clinical milestones when anticipated, generating clinical data that establishes VCN-01 may lead to improved patient outcomes in otherwise refractory cancers; the Company’s and VCN’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s and VCN’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s and VCN’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s and VCN’s ability to maintain license agreements, the continued maintenance and growth of the Company’s and VCN’s patent estate, the ability to continue to remain well financed, and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Source: Theriva Biologics, Inc.
Paulness
1 week ago
NEWS -- Theriva™ Biologics to Present Preclinical Data Supporting the Potential Synergy of VCN-01 and First-Line Pancreatic Cancer Chemotherapy Regimens at the American Society for Cell and Gene Therapy 27th Annual Meeting
Lead product candidate, VCN-01 in combination with liposomal irinotecan demonstrated enhanced anti-tumor effects in a human pancreatic mouse xenograft
The observed synergy emphasizes VCN-01’s potential in diverse chemotherapy combinations for improved efficacy in the treatment of pancreatic cancer
ROCKVILLE, Md., April 22, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced the presentation of preclinical data demonstrating enhanced anti-tumor effects in human pancreatic cancer xenograft-bearing mice treated with lead product candidate VCN-01 and liposomal irinotecan. These data support the potential synergy of VCN-01 and first-line pancreatic cancer chemotherapy regimens, and will be featured in a poster presentation at the American Society for Cell and Gene Therapy (ASGCT) 27th Annual Meeting, being held both virtually and in Baltimore from May 7-11, 2024.
“The data featured at the upcoming ASGCT meeting build on recent findings that suggest the combination of VCN-01 and topoisomerase I inhibitors, such as liposomal irinotecan, may provide a synergistic antitumor effect to improve therapeutic outcomes across indications,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “We look forward to leveraging these findings and evaluating the combination of VCN-01 with additional first-line pancreatic cancer chemotherapy regimens, including NALIRIFOX and FOLFIRINOX. In parallel, we continue to progress our on-going VIRAGE Phase 2b trial evaluating VCN-01 in combination with gemcitabine/nab-paclitaxel to treat metastatic pancreatic ductal adenocarcinoma (PDAC). Together, these important steps bring us one step closer to building a portfolio of potentially improved therapeutic combinations for PDAC patients with high unmet medical needs.”
Key takeaways include:
Overview: The combination of VCN-01 + topoisomerase I (topo1) inhibitors, such as liposomal irinotecan, has a tolerable toxicity profile and may improve efficacy in the treatment of human pancreatic cancer.
In vitro: Viral protein expression was increased in human pancreatic cancer cell lines when they were exposed to topo1 inhibiting chemotherapeutics, irinotecan, its active metabolite, SN-38, and topotecan.In vivo: Synergy of VCN-01 plus liposomal irinotecan was observed in animals bearing subcutaneous human pancreatic tumors.In human pancreatic mouse xenograft models, treatment with VCN-01 at a dose of 4x1010 vp or liposomal irinotecan alone (at both the 10 mg/kg and 5 mg/kg doses) resulted in significant tumor growth inhibition compared to saline.Combination therapy with VCN-01 + liposomal irinotecan at either dose displayed significantly reduced tumor growth compared to each treatment alone.qPCR analyses performed on tumors collected at end of study confirmed the presence of viral genomes, indicating ongoing transcriptional activity of VCN-01, which is consistent with viral replication for several days after administration.The full abstract (1760) for the poster presentation is accessible on the ASGCT Congress portal and the poster will be available starting, Friday, May 10, 2024. Additional details on the poster are provided below:
Title: Enhanced Anti-Tumor Efficacy of Combination Therapy with the Oncolytic Adenovirus, VCN-01, and Liposomal Irinotecan in a Human Pancreatic Mouse Xenograft
Session Title: Cancer - Oncolytic Viruses
Presenting Author: Dr. Sheila Connelly, Vice President of Research, Theriva Biologics, Inc.
Poster Session Date and Time: Friday, May 10, 2024 at 12:00 p.m. ET
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company is advancing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. The Company’s lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients; and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at https://www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding the potential synergy between lead product candidate, VCN-01 and first-line pancreatic cancer chemotherapy regimens; the suggestion that the combination of VCN-01 and topoisomerase I inhibitors, such as liposomal irinotecan, may provide a synergistic antitumor effect to improve therapeutic outcomes across indications; leveraging these findings and evaluating the combination of VCN-01 with the additional first-line pancreatic cancer chemotherapy regimens, including NALIRIFOX and FOLFIRINOX; and continuing to progress our on-going VIRAGE Phase 2b trial evaluating VCN-01 in combination with gemcitabine/nab-paclitaxel to treat metastatic pancreatic ductal adenocarcinoma (PDAC). Important factors that could cause actual results to differ materially from current expectations include, among others, the ability to generate clinical data that establishes a synergy between lead product candidate, VCN-01 and first-line pancreatic cancer chemotherapy regimen and provides an antitumor effect; the Company’s and VCN’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s and VCN’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s and VCN’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s and VCN’s ability to maintain license agreements, the continued maintenance and growth of the Company’s and VCN’s patent estate, the ability to continue to remain well financed, and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Source: Theriva Biologics, Inc.
Paulness
3 weeks ago
NEWS -- Theriva™ Biologics Announces Presentation at the American Society for Cell and Gene Therapy 27th Annual Meeting
ROCKVILLE, Md., April 08, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced the presentation of preclinical data demonstrating the potential synergy between lead product candidate, VCN-01 and liposomal irinotecan in a human pancreatic mouse xenograft. Data will be featured in a poster presentation at the American Society for Cell and Gene Therapy (ASGCT) 27th Annual Meeting, being held both virtually and in Baltimore from May 7-11, 2024.
“We look forward to presenting data at the upcoming ASGCT meeting that further highlight the potential of our systemically administered oncolytic adenovirus, VCN-01, to synergistically combine with an expanding range of standard-of-care chemotherapies for difficult-to-treat cancers,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “ Our on-going VIRAGE Phase 2b trial is evaluating VCN-01 in combination with gemcitabine/nab-paclitaxel to treat metastatic pancreatic ductal adenocarcinoma (PDAC). The enhanced anti-tumor effects observed in the human pancreatic mouse xenograft model with VCN-01 and liposomal irinotecan provides compelling support for evaluating the combination of VCN-01 with the additional first-line pancreatic cancer chemotherapy regimens NALIRIFOX and FOLFIRINOX to provide a portfolio of improved therapeutic combinations for patients with this deadly disease.”
ASGCT Presentation Details
Title: Enhanced Anti-Tumor Efficacy of Combination Therapy with the Oncolytic Adenovirus, VCN-01, and Liposomal Irinotecan in a Human Pancreatic Mouse XenograftAbstract: 1760Session Title: Cancer - Oncolytic VirusesPresenting Author: Dr. Sheila Connelly, Vice President of Research, Theriva Biologics, Inc.Poster Session Date and Time: Friday, May 10, 2024 at 12:00 p.m. ETAbout Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company is advancing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. The Company’s lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients; and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at https://www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding the potential synergy between lead product candidate, VCN-01 and liposomal irinotecan in a human pancreatic mouse xenograft and the potential of the Company’s systemically administered oncolytic adenovirus, VCN-01, to synergistically combine with an expanding range of standard-of-care chemotherapies for difficult-to-treat cancers. Important factors that could cause actual results to differ materially from current expectations include, among others, the ability to generate clinical data that establishes VCN-01 may expand the range of standard-of-care chemotherapies for difficult-to-treat cancers; the Company’s and VCN’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s and VCN’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s and VCN’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s and VCN’s ability to maintain license agreements, the continued maintenance and growth of the Company’s and VCN’s patent estate, the cash runway, and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Source: Theriva Biologics, Inc.
Paulness
1 month ago
NEWS -- Q4 2023 Theriva Biologics Inc Earnings Call
Participants
Steven A. Shallcross; CEO, CFO, Treasurer, Corporate Secretary & Director; Theriva Biologics, Inc.
Presentation
Operator
And greetings, and welcome to the Theriva Biologics full year 2023 investor conference call.
At this time, all participants are in a listen only mode. As a reminder, this conference is being recorded. I would now like to turn the call over to your host Chris Calabrese with LifeSci Advisors. Thank you. You may begin.
Thank you, operator, and good morning, everyone. Welcome to the Theriva Biologics full year 2023 investor conference call. Leading the call today will be Steven Shallcross, Chief Executive and Chief Financial Officer of Theriva Biologics; Dr. Medel, Qwest guy of General Director of the RevA biologics, European subsidiary, and Dr. Vince wager, Head of Corporate and Product Development of Spiriva biologics are also on the call and will be available to answer questions during the Q&A session.
The review Biologics issued a press release this morning, which provided operational highlights and included the financial results for the full year ending December 31, 2023. The press release can be found in the Investors section of the Company's website at w. w. w. dot Areva bio.com, together with the annual report on Form 10-K for full year ended December 31, 2023, which we plan to file today with the Securities and Exchange Commission. In addition to the phone line, this call is being streamed live via webcast, which will be archived on the Company website, w. w. w. dot Areva bio.com for 90 days.
During this call, certain forward-looking statements regarding Theriva Biologics and DCM Biosciences, current expectations and projections about future events will be made. Generally, the forward-looking statements can be identified by terminologies such as may, should, expects, anticipates, intends, plans, believes, estimates and similar expressions. These statements are based upon current beliefs, expectations and assumptions and are subject to a number of risks and uncertainties, including those set forth in three-V Biologics' filings with the SEC, many of which are difficult to predict.
No forward-looking statements can be guaranteed, and actual results may differ materially from such statements. The information on this call is provided only as of the date of this call and to review. Biologics undertakes no obligation to update any forward-looking statements contained on this conference call on account of new information, future events or otherwise, except as required by law.
With that, I'd like to turn the call over to Steve.
Steven A. Shallcross
Thank you, Chris, and good morning. I appreciate everyone for taking the time to join us today. In 2023, we continued to make steady progress to drive forward our oncology forward focused portfolio designed to address unmet needs for difficult to treat cancers. Our primary efforts and resources are focused on pursuing multiple therapeutic opportunities for our lead clinical candidate, VCN-01.
As a reminder, VCN-01 is a systemically administered oncolytic adeno virus designed to selectively replicate within the tumor, the grade the tumor matrix increase tumor immunogenicity. We believe these multiple modes of action position, VCN-01 for optimized tumor killing in combination with chemotherapy and immuno-oncology products and otherwise refractory solid tumors.
We have shown that repeated systemic dosing of VCN-01 is feasible from a safety perspective, and we can now focus on whether the repeated dose VCN, Reg VCN-01 regimen may lead to improved clinical outcomes for patients beyond VCN-01, we are pursuing new oncolytic virus candidates to leverage our novel human shield technology, which is designed to protect systemically administered oncolytic viruses from the host immune system and may facilitate more frequent repeated administration of oncolytic virus therapies. This movement may enable our pipeline of products to be used in standardized treatment cycles that are well established in cancer, chemotherapy and immunotherapy.
Additionally, as part of our oncology focus portfolio. We continue to screen and enroll patients in the second cohort of the Phase Ib IIa clinical trial of sin for design to prevent potentially fatal adverse outcomes in patients who undergo alginate committed periodic cell transplant ACT. to treat hematologic cancers. With our cash runway into the first quarter of 2025, we believe we're well positioned to execute on our corporate objectives and remain on track to achieving multiple value-enhancing milestones.
With this brief introduction, I'd like to expand on key pipeline updates. Starting with our lead program. VCN-01 BCL. one has been administered to more than 100 patients across diverse indications, which speaks to the broad therapeutic potential, including pancreatic ductal adenocarcinoma or PDX retinol BLISS, stoma, head and neck squamous cell carcinoma, colorectal cancer and ovarian cancer.
VCN-01 has been granted orphan drug designation in the US and Europe for the treatment of pancreatic cancer and in the US for retinol, but Soma, providing additional opportunities for regulatory engagement and if approved market exclusivity, our most advanced program for VCN-01 is in PDX for which incidence continues to rise in an indication that has one of the lowest survival rates among all cancers.
It is well established that the pediatric tumor matrix is one of the key reasons for the overall poor therapeutic outcomes for these patients. We believe the CNO one's differentiated mechanism of action has the potential to address the urgent need for new treatment options for patients with PTAC. by degrading the twofer matrix and increasing tumor access by co administering cancer therapies. We are pleased to report that dosing is well underway for Viraj, our Phase 2b trial of VCN-01 in combination with standard of care chemotherapy, gemcitabine and nab-paclitaxel, which is being evaluated as a first-line therapy for patients with PDX with six sites open in the US and nine sites opened in Spain. Faraj remains on track to complete enrollment in the first half of 2024.
In the first quarter of 2024, we completed the first safety review with the Independent Data Monitoring Committee or IDMC with a positive recommendation from the IDMC. Faraj will continue to enroll patients without any changes to the protocol. Notably, intravenous VCN-01 has been well tolerated and demonstrated a safety profile consistent with prior clinical trials.
Importantly, no additional toxicities were observed in patients receiving a second dose of VCN-01, providing the first clinical evidence of the feasibility of repeated systemic dosing. As a reminder, primary endpoints for the Virage trial include overall survival and BCNO. one safety and tolerability. Additional endpoints include progression-free survival objective response rate measures of VCN-01 biodistribution replication, immune response and measures of the quality of life of treated patients. Since this is an open-label trial, progress will be monitored very closely in steps to accelerate the clinical program may be implemented if supported by emerging data.
More broadly, the Virage trial will enable us to determine the feasibility of repeated dosing of VCN-01 This could shift the approach to standardize treatment cycles that are well established in cancer chemotherapy and immunotherapy and may lead to improved clinical outcomes for patients with PDX and other difficult to treat solid tumors.
In addition to advancing the Virage PDN trial, we continue to work closely with key opinion leaders in the US, Europe and Central and South America, as well as with regulatory agencies to refine our clinical strategy in Renovo stoma. We believe intravitreal VCN-01 has the potential to treat vitreous seeds in children with retinol Bluestone.
Since current clinical practice varies in, there's no regulatory guidance specific to retinol plus stoma drug development. We held a pre IND meeting with the FDA in the fourth quarter of 2023 to discuss the development pathway for VCN-01 is an adjunct to chemotherapy in pediatric patients with advanced renal milestones. During our meeting with the FDA, we were provided some guidance on the potential endpoints in patient population for an advanced clinical trial and encouraged to submit a formal protocol under a US IND.
In order to provide a more detailed commentary for this program, we are encouraged by interactions with the FDA and look forward to driving this program forward.
In parallel with company-sponsored studies, the potential utility of VCN-01 is being explored and a number of investigator-sponsored studies that are underway at leading oncology research institutions around the world, notably collaborators at St. Johns and Dale have completed patient treatment in the Phase one investigator sponsored trial evaluating the safety and activity of intravitreal BC and one in pediatric patients with refractory retinol Bluestone.
The trial evaluated escalating doses of VCN-01 administered by two intravitreal injections separated by 14 days and remains on track to complete patient follow-up in the first half of 2024, which will help to inform the planned Phase 2 trial and design and the protocol. As a reminder, preclinical data has shown that topotecan treatment enhanced BCNO. one oncolytic activity against rentable stoma and more broadly reinforced VCN-01 possibility as an adjunct to intravitreal chemotherapy in patients who fail currently available treatments. We remain encouraged by the potential of this novel combination approach to provide superior clinical benefits for children with this devastating cancer.
Additionally, the University of Pennsylvania continues to enroll and treat patients in their Phase one investigator sponsored trial, administering VCN-01 with your CAR T muscle cells to patients with ovarian or pancreatic cancers. VCN-01 designed to increase tumor immunogenicity and improve access by additional therapies such as UCargo muscle cells while cell-based immunotherapies have had limited efficacy against solid tumors to date, we are encouraged by initial results highlighting the feasibility of administering VCN-01 with this type of CAR T therapy. These preliminary results were presented last year at the Society for Immunotherapy of Cancer Annual Meeting or cities. European investigators are continuing to explore the optimal dosing regimen for VCN-01 co-administered with your card muscle cells, and we look forward to further data from the study in 2024.
Turning to our ongoing Phase 2b 2a clinical trial of Washington University evaluating same for or write-backs. The trials designed to evaluate the therapeutic potential of sin for two fatal to reduce fatal adverse events related to IV beta-lactam antibiotic use and allogeneic HCT recipients, including acute graft versus host disease or AGVHD. in overgrowth and infection by pathological organisms such as C. difficile and Vanco myosin resistant and tear Koksay. The Phase 1b 2a study is designed to assess the feasibility of using sense for and consist of three sequential cohorts comparing different IV beta-lactam antibiotics following conditioning therapy in each cohort eight patients will receive and four and four receive placebo. While the data remain blinded, interim analysis suggests it's in four is well tolerated and was not observed in the blood samples of a majority of development patients.
Our second cohort is underway and is designed to evaluate SIN for combination with piperacillin tazobactam. The trial is on track to complete enrollment in the second cohort in the second quarter of 2024. This cohort will provide important additional safety information. In particular, whether oral sema four has the potential to alter IV antibiotic levels in this patient population. We look forward to sharing this data in the second half of 2024.
Overall, we are encouraged by the progress across our pipeline in the growing clinical data that underscore the promise of our systemically administered oncolytic virus in key indications and combinations. We remain focused on driving our clinical programs forward in exploring opportunities to leverage our novel albumin shield technology and exciting additional technologies from Rovi discovery platform. I'm confident that the Company's upcoming catalysts will provide a solid foundation for execution and value creation.
Specifically we remain on track to complete enrollment for the Virage study in the first half of 2024, complete follow-up in the Phase 1 investigator sponsored trial evaluating the safety and activity of intravitreal, the VCN-01 in pediatric patients with refractory reasonable stoma and the first half of 2024 and complete enrollment in the second cohort of our Phase 1b 2a clinical study of seven four for the prevention of AGVHD. in bone marrow transplant patients in the second quarter of 2024.
Now I briefly turn to our financial results for the first full year ended at December 31, 2023, general and administrative expenses decreased to $7.1 million for the year ended December 31, 2023 from $9.9 million for the year ended December 31, 2022. This decrease of 28% is primarily comprised of the decrease in the fair value of the contingent consideration of $2.8 million, along with lower salary, investor relations, legal costs, consulting fees related to the VCN. acquisition and director and officer insurance, offset by higher audit fees and other consulting fees.
The charge related to stock-based compensation expense was $0.4 million for the year ended December 31, 2023 compared to $0.4 million for the year ended December 31, 2022. Research and development expenses increased to $14.3 million for the year ended December 31, 2023 from $11.7 million for the year ended December 31, 2022.
This increase of 22% is primarily the result of higher clinical trial expenses related to our Virage Phase 2 clinical trial of VCN-01 and PDx offset by lower expenses related to our Phase 1b 2a clinical trial of sin for an allogeneic HCT recipients. The completed phase one, a clinical trial have seen 20 decreased manufacturing expenses related to our Phase 1a clinical trials in 20 and lower other indirect costs.
We anticipate research and development expense to increase as we continue enrollment in our Virage Phase two clinical trial of VCN-01 and P. deck in our ongoing Phase 1 clinical trial and renewable stoma, expand GMP manufacturing activities for VCN-01 and continue supporting RBC and 11 on other preclinical and discovery initiatives.
Research and development expense also includes a charge related to noncash stock-based compensation expense of $165,000 for the year ended December 31, 2023, compared to $112,000 for the year ended December 31, 2022. Other income was $1,442,000 for the year ended December 31st, 2023, compared to other income of $471,000 for the year ended December 31, 2022. Other income for the year ended December 31, 2024, was primarily comprised of interest income of $1,439,000 and an exchange gain of $3,000. Other income for the year ended December 31, 2022 is primarily comprised of interest income of $512,000, offset by an exchange loss of $41,000.
Cash and cash equivalents totaled $23.2 million as of December 31, 2023, compared to $41.8 million as of December 31, 2022. We remain deeply committed to improving patient outcomes for these very hard-to-treat cancers.
And before we conclude today's call, I want to extend my sincere appreciation and gratitude for the foundational work that has brought us closer to delivering on our mission.
I'd like to thank the entire Theriva team, our investors and the many people who have been supportive along the way, including our patients and their families.
With that, we're happy to take questions.
Question and Answer Session
Operator
Thank you. And at this time, we'll be conducting a question and answer session. (Operator Instructions)
James Molloy, Alliance Global Partners.
Yes, good morning. Thank you very much for taking my questions. Pillow had a question looking below Stephen, Alex, Christopher, just even your mental on looking across the multiple IST.s, which are very cost efficient way of getting trials done excellent. Excellent use of capital, which you guys see as sort of most promising of like all the volatile and of course, of which seems like it might be maybe growing the task. We look across the thrust of the plethora of IST as you guys have got underway.
Steven A. Shallcross
So let me let me just highlight where what we've got going on once again. And then Manav, you could talk specifically about some of the exciting findings that we found of had disclosed at various scientific conferences. So as you're aware, we put out some incredible head and neck cancer data at asthma last October. This is using BC and one in combination with checkpoint inhibitors in patients that were refractory to previous rounds of checkpoint inhibitor therapy on someone else could talk a little bit about that.
We've got the retinol Bluestone, the program on which we've just concluded, and now we're in the middle of follow-up. And then we've got the ongoing New Penn study, which is down to of their organization, trying to isolate the right dose take forward. So we would expect to see some more of data sometime this year related to the work they're doing there.
So now maybe you could just highlight the important findings on those three studies that are that are ongoing or recently completed?
Yes, sure, sure. So obviously, the muscle complete data, let's say, from the trial in head and neck had a good percentage of asthma in October as you know, this was a trial combining VISION one with the development and efficient refractory to the action of immune checkpoint inhibitors in head and neck carcinoma.
Okay. And we have previously presented safety data that demonstrated that additional one has an acceptable safety profile when administered prior to durvalumab. And in this new presentation that we conducted in October, we presented data on efficacy.
And we have seen that the patients treated with Vision Show with an increase of restaurants, two, a subset of chemotherapy treatments after progressions and style. But specifically, we have also seen that the survival of patients has been large for a number of patients. And in fact, some patients are still alive more than four years after we didn't participate in the trial, which in my view, it's quite remarkable because those patients were entering the trial had some effect on shelf life of between three or four months. And what we are seeing obviously, it's really interesting.
And moreover, it's not just a question of efficacy that does a very good correlation between the restaurants and the biological data we are getting from the tumor biopsies of these patients. And in fact, we have seen down-regulation of the more massive genes. We have been observing increasing levels of immune markers in the tumor biopsies. And interestingly, there was a correlation between the survival and with the CPS score and a day after vision one for investigator scoring, it's a kind of marker about immunological status and what looks in our biological, the that you said that vision one treatment change in this environment and definite correlation between the magnitude of the change that we use and the survival of patients, which is I think exciting.
Obviously, it's use a proportion, but it's exciting in regional customer. We have been treating more patients. We have seen some patients with reductions that we do see it. But specifically, we have been also collecting data of combination with the chemotherapy, the chemotherapy that is using rational estimate, it's topotecan. And in this trial, we have seen not derailing the clinical patients. But in preclinical work that we have done with biopsies and from a human cells, we have been observing that there is synergy between the action of this in one and a couple of Beacon, which is very encouraging and opens the door for a combination approach in our Phase two program that is massively with cash and with FDA in our meeting.
Okay.
So basically, the data we are collecting, it's very exciting. And in my view, it's very exciting to see that in different indications, we can confirm the mechanism of action. And we can see initial evidence of activity in happening in RightNow customer, which is also the same thing that we have observed in our Phase one program in pancreatic cancer so that the data is quite consistently that way.
Thank you very much. Taking my quick follow-up on the on the retina. Last on the ISTI. when you anticipate potentially filing an IND and starting the Phase two three again, presuming the Phase one wraps up as you hope.
And then a follow-up to the Phase IIb Virage, I know the enrollment completed in first half 2024, which we just data final data top line data for us.
So Manoj, why don't you take the first one and then Vince, you could take the second guess our four original restaurant model right now we are just following the a lot of patients.
Okay. As you know, our clinical trial to treat the patient that part, it's already finished that we need to follow the patients for six months after the last dosing. So we are expecting to finish our database with data of the trial in mid-2024, probably. And after that, we need to rise to interest a rep or and start that discussion with FDA. The final design of the and the so I don't expect to go for an IND until 2024, 2025, sorry by shore because obviously we need to finish some activities. We got before submitting any AMD for RightNow customer base. We want to give some color on the pancreatic program.
Thanks, Menno. So I'll as Steve indicated, a Virage study is enrolling and we anticipate completing enrollment in the first half of this year with the with the patients only. And then we will be very closely monitoring the emergence of our data. And this there's two key outcomes one. Obviously, our primary endpoint of survival will be following those patients and the longer the better from our perspective because we want to have a good effect.
So the primary endpoint data. The survival won't read out until next year as I kind of peak when we would like to say that to be potentially in the second half, the longer as long as these patients keep going. We want patients on our drug to do well.
In the interim, we will be looking at our data to see if there's something that we can around which we can build a formal interim analysis to review with the FDA and discuss next steps how we can potentially advance our program quickly into a pivotal trial. And as we know, as you know, we've got the orphan drug designation, so we'll leverage that strategy. But again, we can't necessarily predict the timing other than later in the year for a potential interim analysis if we choose to do one. But the overall endpoints for the primary endpoint of survival will be next year
Thank you very much. And maybe a follow-up questions from me on Cyno forking giving you actually sort of the endgame for CNO for where a so what though which we anticipate sort of coming to a conclusion on that on the data, what sort of timing on that? And then can you talk a little bit about how the partnership and characterize the partnership environment currently for Finjan licensing OTOR. for linear compounds?
Steven A. Shallcross
Sure. So on, as you know, the same for trials, a single single-site study at Wash U and those partners have been outstanding on fortunately or unfortunately, on the time it takes to enroll a trial like that is subject to the number of patients that actually meet the screening criteria so they can be brought into the trial and we're going to complete that trial, as I stated earlier on in relative short order.
And then we'll obviously have some disclosure around that. This cohort is pretty important because a sin for does degrade the combination of pepper so on and take it back to him. And obviously, we're monitoring the data in this trial and we want to make sure that the antibiotic is not and interfered with in this more fragile patient population.
So once we have that data in hand, we'll make a decision about whether or not we advance into the third cohort or whether or not we have enough and to answer our questions that were brought to us by the FDA on then this asset ties in more broadly to the initiatives we have underway to identify potential partners across our entire pipeline. So we've hired some outside advisors on one group specific to help us finding a home for person for and we've had engagement.
And again, a lot of this has to do about when we have the data on. We've also have a group we're working with and outreach to potential partners for the VCNO. one platform. And once again, we've had multiple engagements and interest and we will continue those discussions. And once again, data is key and we've also have some folks that we're working with trying to find a home for the Cine 20 program.
And again, those discussions are ongoing. I think the environment has gotten a bit better recently on, I think over the last couple of years, given sort of the bear market that biotech and small and micro-cap biotech has been and has hindered a lot of discussion on, but I am more and more optimistic things seem to be picking up a little bit. And in keeping with our strategy and how we view these ongoing discussions we're not going to talk about specific interactions and when we have something, obviously we'll make a disclosure about that, of course.
Thank you very much taking questions.
Operator
Thank you. And as a reminder, if you'd like to ask a question, please press star one on your telephone keypad or pause a moment to allow for any other question. Mr. Shah cross.
I'm not showing any other questions at this time. I'll turn the floor back to you for any final.
Steven A. Shallcross
Okay. Thanks, Melissa. Well, thanks again, everyone, for taking the time to join us today. I hope you sense that we're incredibly focused on driving all of our programs forward.
We're doing this in a way which I think we're very good stewards with our cash and making and stretching that dollar the best we can to get as much data and as many clinical outcomes and results as possible.
Will continue is we just ended with Jim here to evaluate our strategic options, and we'll continue to look for ways to drive additional value for our shareholders and for the long-term success of what we're trying to do namely delivering, you know, promising treatments for very, very difficult-to-treat cancers.
Thanks again for joining us, and we look forward to keeping you updated on our progress.
Operator
Thank you. This concludes today's conference. You may disconnect your lines at this. Thank you for your participation.
Paulness
3 months ago
NEWS -- Theriva Biologics Announces Positive Recommendation from the Independent Data Monitoring Committee of VIRAGE, the Phase 2b Clinical Trial of VCN-01 in Combination with Chemotherapy for Metastatic Pancreatic Ductal Adenocarcinoma
The independent data monitoring committee (IDMC) recommended the continuation of VIRAGE with no safety concerns raised; VIRAGE remains on track to complete enrollment in the first half of 2024
ROCKVILLE, Md., Feb. 07, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced that the Independent Data Monitoring Committee (IDMC) recommended the continuation of enrollment as planned into VIRAGE, a multinational, Phase 2b, randomized, open-label, controlled clinical trial evaluating VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first-line therapy for patients with metastatic pancreatic ductal adenocarcinoma (PDAC).
According to the IDMC's comprehensive assessment of clinical data from patients enrolled across 6 sites open in the U.S. and 9 sites open in Spain, the ongoing Phase 2b trial will continue without any changes to the protocol. No safety concerns were raised based on the evaluation of data presented at the IDMC meeting. Intravenous VCN-01 has been well tolerated and demonstrated a safety profile consistent with prior clinical trials. Importantly, no additional toxicities were observed in patients receiving a second dose of VCN-01, providing the first clinical evidence of the feasibility of repeated systemic dosing. VIRAGE remains on track to complete enrollment in the first half of 2024.
"The positive IDMC review of VCN-01 safety following repeated systemic dosing marks a significant step forward for our lead program. VCN-01 is a highly differentiated, systemic, selective, stroma-degrading oncolytic adenovirus," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “With the IDMC’s recommendation, we will continue to drive forward the VIRAGE study and explore the potential of VCN-01 to improve outcomes in first-line metastatic PDAC patients treated with standard-of-care chemotherapy. We have shown that repeated systemic dosing of VCN-01 is feasible from a safety perspective, and can now focus on whether the repeated-dose VCN-01 regimen may lead to improved clinical outcomes for patients with PDAC and other solid cancers.”
About VIRAGE
VIRAGE is a two-arm Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. VIRAGE is expected to enroll up to 92 adult participants at up to 25 sites across the US and Spain. In both the control and treatment arms, patients will receive gemcitabine/nab-paclitaxel standard-of-care chemotherapy over 28-day cycles. In the treatment arm only, patients will also receive systemically administered VCN-01 seven-days prior to the first and fourth cycles of gemcitabine/nab-paclitaxel treatment. Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, objective response rate, and measures of biodistribution, VCN-01 replication, and immune response. Since this is an open-label trial, progress will be monitored very closely and steps to accelerate the clinical program may be implemented if supported by the emerging data. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection).
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company is advancing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. The Company’s lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients; and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at https://www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding the ongoing Phase 2b trial continuing without any changes to the protocol, VIRAGE remaining on track to complete enrollment in the first half of 2024, VCN-01 continuing to be well tolerated with a safety profile consistent with prior clinical trials, exploring the potential of VCN-01 to improve outcomes in first-line metastatic PDAC patients treated with standard-of-care chemotherapy, and the VIRAGE trial enrolling 92 patients. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s and VCN’s ability to reach clinical milestones when anticipated, including completion of enrollment in Virage in the first half of 2024, generating clinical data that establishes VCN-01 being an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma and combining with immunotherapy products to treat solid tumors, the Company’s ability to successfully combine and operate the business of the Theriva Biologics and VCN, the Company’s and VCN’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s and VCN’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s and VCN’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s and VCN’s ability to maintain license agreements, the continued maintenance and growth of the Company’s and VCN’s patent estate, the ability to continue to remain well financed , and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Source: Theriva Biologics, Inc.
Paulness
5 months ago
NEWS -- Theriva Biologics, Inc. (NYSE:TOVX) Q3 2023 Earnings Call Transcript
Operator: Greetings, and welcome to the Theriva Biologics, Inc. 2023 Third Quarter Operational Highlights and Financial Results. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Steve Shallcross. Thank you. You may begin.
Steve Shallcross: Thank you, Irene, and good morning, everyone, and thank you for joining our call today. Welcome to Theriva Biologics Third Quarter 2023 Investor Conference Call. Joining me on today's call will be Dr. Manel Cascallo, Director General of Theriva Biologics' European Subsidiary; and Dr. Vince Wacher, Head of Corporate and Product Development of Theriva Biologics. Theriva Biologics issued a press release this morning, which provided operational highlights and included the financial results for the third quarter ended September 30, 2023. The press release can be found in the Investors section of the company website at www.therivabio.com together with the quarterly report on Form 10-Q for the quarter ended September 30, 2023, which we plan to file today with the Securities and Exchange Commission.
A doctor consulting with a patient, discussing treatment options for breast cancer.
In addition to the phone line, this call is being streamed live via webcast, which will be archived on the company's website, https://www.therivabio.com for 90 days. During this call, certain forward-looking statements regarding Theriva Biologics and VCN Biosciences' current expectations and projections about future events will be made. Generally, the forward-looking statements can be identified by terminologies such as may, should, expects, anticipates, intends, plans, believes, estimates and similar expressions. These statements are based upon current beliefs, expectations and assumptions and are subject to a number of risks and uncertainties, including those set forth in Theriva Biologics filings with the SEC, many of which are difficult to predict.
No forward-looking statements can be guaranteed, and actual results may differ materially from such statements. The information on this call is provided only as of the date of this call, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained on this conference call on account of new information, future events or otherwise, except as required by law. With that, I'd like to start by discussing our progress during the quarter. In the third quarter of 2023, we continue to make steady progress to drive forward our oncology-focused portfolio designed to address unmet needs for difficult-to-treat cancers. With our extended cash runway into the first quarter of 2025, we believe we're well positioned to execute on our corporate objectives and remain on track to achieving multiple value-enhancing milestones.
Our primary efforts and resources are focused on pursuing multiple therapeutic opportunities for our lead clinical candidate, VCN-01. As a reminder, VCN-01 is a systemically administered oncolytic adenovirus designed to selectively replicate within the tumor, degreed to tumor matrix and increase tumor immunogenicity. We believe these multiple modes of action position VCN-01 for optimized tumor killing across several indications and in combination with different types of therapies. The potential use of VCN-01 to enable and enhance the use of chemotherapy and immuno-oncology products and otherwise refractory solid tumors is a strategic focus for Theriva that may provide multiple opportunities in areas of high therapeutic need. Today, I'm pleased to report recent highlights from our ongoing programs, evaluating VCN-01 in different indications in combination with chemotherapy, immune checkpoint inhibitors and CAR-T cells.
Building on our exploration of the potentially broad synergistic clinical benefit of VCN-01, we are pursuing new oncolytic virus candidates to leverage our novel Albumin Shield Technology, which is designed to protect systemically administered oncolytic viruses from the host immune system and may facilitate repeated administration of oncolytic virus therapies. This may enable our pipeline programs to be used in standardized treatment cycles that are well established in cancer chemotherapy and immunotherapy. Additionally, as part of our oncology focused portfolio, we continue to screen and enroll patients in the second cohort of the Phase Ib/IIa clinical trial of SYN-004 designed to prevent potentially fatal adverse outcomes in patients who undergo allogeneic hematopoietic cell transplant, or HCT, to treat hematologic cancers.
With this brief introduction, I will now provide further details on how these programs continue to position Theriva the forefront of oncolytic virus development, starting with our lead program, VCN-01. Our confidence in VCN-01 is built on a strong clinical foundation as VCN-01 has been administered to more than 100 patients across diverse indications, including pancreatic ductal adenocarcinoma or PDAC, head and neck squamous cell carcinoma, colorectal cancer, ovarian cancer and retinoblastoma. VCN-01 has been granted orphan drug designation in the U.S. and Europe for the treatment of pancreatic cancer and in the U.S. for retinoblastoma, providing additional opportunities for regulatory engagement and if approved, market exclusivity. Our most advanced program for VCN-01 is in PDAC, which has one of the lowest survival rates among all cancers and is an indication that is ripe for innovation.
It is well established that the PDAC tumor matrix is one of the key reasons for the overall poor therapeutic outcomes for these patients. We believe VCN-01 has the potential to address the urgent need for new treatment options for patients with PDAC by degrading the tumor matrix and increasing tumor access by co-administered cancer therapies. VIRAGE, our Phase IIb trial of VCN-01 in combination with standard of care chemotherapy, gemcitabine and nab-paclitaxel as a first-line therapy for patients with PDAC, continues to advance with dosing well underway across sites in the U.S. and Spain. VCN-01 has been well-tolerated with a safety profile consistent with prior clinical trials. We remain on track to complete enrollment with 92 available patients in the first half of 2024.
As a reminder, the primary endpoint for the trial include overall survival and VCN-01 safety and tolerability. Additional endpoints include progression-free survival, objective response rate and measures of VCN-01 biodistribution replication and immune response. Since this is an open-label trial, progress will be monitored very closely and steps to accelerate the clinical program may be implemented and supported by emerging data. More broadly, the VIRAGE trial will enable us to determine the feasibility of repeated dosing of VCN-01, which could shift the paradigm to standardized treatment cycles that are well established in cancer chemotherapy and immunotherapy and may lead to improved clinical outcomes for patients with PDAC and other solid tumors.
In addition to advancing the VIRAGE PDAC trial, we continue to work closely with key opinion leaders in the U.S., Europe, Central and South America to refine our clinical strategy in retinal blastoma. Since current clinical practice varies and there's no regulatory guidance specific to retinoblastoma drug development, we have submitted our meeting request with regulatory agencies and look forward to discussing the development pathway for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma. We believe intravitreal VCN-01 has the potential to treat Vitreous seeding in children with retinoblastoma, and we look forward to leveraging our orphan drug designation in this indication to facilitate protocol discussions with the FDA and other regulatory agencies to enable the development of new potential treatment options for this difficult-to-treat cancer.
In parallel with company-sponsored studies, the potential utility of VCN-01 is being explored in a number of investigator-sponsored studies that are underway at leading oncology research institutions around the world. Today, I'll focus on recent updates from our collaboration with the Catalan Institute of Oncology, or ICO, for patients with head and neck cancer and the University of Pennsylvania for patients with pancreatic and ovarian cancer. Data from the ongoing study of VCN-01 in combination with durvalumab in patients with recurrent metastatic head and neck cancer were recently presented at the European Society for Medical Oncology Annual Congress, or ESMO. Results showed enhanced patient survival up to almost 4 years in one patient, which correlated with VCN-01 mediated increases in CPS score, a key determinant of outcomes with anti-PD-L1 checkpoint inhibitor therapies.
These data are remarkable, given these patients had all failed prior lines of anti-PD-L1 treatment. In addition to the presentation at ESMO, we hosted a virtual KOL event featuring Dr. Ricard Mesia of the ICO. In addition to reviewing key takeaways from the ESMO poster presentation, Dr. Mesia discussed the unmet medical needs in head and neck cancer, current treatment limitations and the therapeutic potential of VCN-01. Dr. Mesia also highlighted data from the ICO Phase I study showing that VCN-01-treated patients had improved responses to later lines of therapy. This is consistent with VCN-01's matrix degrading effect, which enables better access by the co-administered cancer therapies and the potential to elicit an extended antitumor immune response.
Consistent with these clinical data, a significant increase in the infiltration of tumors with anti-PD-L1 positive immune cells was observed, which statistically correlated with patient survival. Additionally, the University of Pennsylvania continues to enroll and treat patients in their Phase I investigator-sponsored study, administering VCN-01 with huCART-meso cells to patients with ovarian and pancreatic cancers. VCN-01 is designed to increase tumor immunogenicity and improve access by additional therapies such as huCART-meso cells. While cell-based immunotherapies have had limited efficacy against solid tumors to date, we are encouraged by the initial results, highlighting the feasibility of administering VCN-01 with huCART-meso cells. These preliminary results were recently presented at the Society for Immunotherapy of Cancer Annual Meeting or SITC.
With no dose-limiting toxicities observed to date, the study will continue to explore higher doses of VCN-01 co-administered with huCART-meso cells. We look forward to further data from the study to determine if VCN-01 can improve patient outcomes with these powerful immunotherapies to treat solid tumors. Turning to our ongoing Phase Ib/IIa clinical trial, Washington University evaluating SYN-004 ribaxamase to reduce potentially fatal adverse events related to IV beta-lactam antibiotic use in allogeneic HCT recipients, including acute graft versus host disease, or aGVHD, and overgrowth in infection by pathological organisms such as C. difficile and vancomycin-resistant enterococci. The Phase Ib/IIa study is designed to assess the feasibility of using VCN-04 and consists of 3 sequential cohorts comparing different IV beta-lactam antibiotics following conditioning therapy.
In each cohort, patients will receive SYN-004 and 4 will receive placebo. While the data remain blinded, interim analysis suggests that SYN-004 is well tolerated and was not observed in the blood samples of a majority of the available patients. Our second cohort is underway and is designed to evaluate SYN-004 in combination with piperacillin and tazobactam. This cohort will provide important additional safety information, in particular, whether oral SYN-004 has the potential to alter IV antibiotic levels in this patient population. Overall, we're encouraged by the progress across our pipeline and the growing clinical data that underscore the promise of our systemically administered oncolytic adenovirus in key indications and combinations. We remain focused on driving our clinical programs forward and exploring opportunities to leverage our novel Albumin Shield Technology and exciting additional technologies from our OV discovery platform.
I'm confident that the company's strong cash position and upcoming catalysts provide a solid foundation for execution and value creation. We remain on track to complete enrollment for VIRAGE in the first half of 2024, meet with the FDA to discuss the clinical program and potential registration pathway for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma before the end of the year and complete enrollment in the second cohort of our Phase Ib/IIa clinical study of SYN-004 for the prevention of aGVHD and bone marrow transplant patients in the first half of 2024. Now I'd like to briefly turn to our financial results for the third quarter ended September 30, 2023. General and administrative expenses decreased to $212,000 for the 3 months ended September 30, 2023, from $2.4 million for the 3 months ended September 30, 2022.
This decrease of 91% is primarily comprised of the decrease in the fair value of contingent consideration of $1.6 million, along with lower salary and bonus costs investor relation fees, audit fees, travel and VCN administrative expenses not included in the prior year, offset by an increase in consulting fees. The charge related to stock-based compensation expense was $95,000 for the 3 months ended September 30, 2023, compared to $93,000 for the 3 months ended September 30, 2022. Research and development expenses increased to $4 million for the 3 months ended September 30, 2023, from approximately $2.6 million for the 3 months ended September 30, 2022. This increase of 56% is primarily the result of higher clinical trial expenses related to our VIRAGE Phase II clinical trial of VCN-01 in PDAC offset by decreased expenses related to our Phase Ib/IIa clinical trial of SYN-004 in allogeneic HCT recipients, Phase Ia clinical trial of SYN-020 and decreased manufacturing expenses related to our Phase Ia clinical trial of SYN-020.
We anticipate research and development expense to increase as we continue enrollment in our VIRAGE Phase II clinical trial of VCN-01 in PDAC and our ongoing Phase I clinical trial in retinal blastoma, expand GMP manufacturing activities for VCN-01 and continue supporting our VCN-11 and other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $40,000 for the 3 months ended September 30, 2023, compared to $28,000 related to stock-based compensation expense for the 3 months ended September 30, 2022. Other income was $388,000 for the 3 months ended September 30, 2023, compared to other income of $161,000 for the 3 months ended September 30, 2022. Other income for the 3 months ended September 30, 2023, is primarily comprised of interest income of $382,000 and an exchange gain of $6,000.
Other income for the 3 months ended September 30, 2022, is primarily comprised of interest income of $170,000, offset by an exchange loss of $9,000. And a further strengthening of our balance sheet during the quarter ended September 30, 2023, we recognized a $1.4 million tax credit receivable and offsetting deferred R&D tax credit is a result of our participation in a research and development program sponsored by the Spanish government. The program provides for reimbursement of certain expenses incurred in research and development efforts that we incurred in Spain. As a condition for participation in the program, we will be required to maintain certain workforce levels in research and develop expenditures over the next 24-month period. Beginning in Q1 2024, the deferred R&D credit will be amortized monthly as a contra expense during 2024 and 2025.
We expect to receive the full cash payment under this program by the end of 2024. Cash and cash equivalents totaled $31.2 million as of September 30, 2023, compared to $41 million -- $41.8 million as of December 30, 2022. We remain deeply committed to improving patient outcomes through these very hard to treat cancers. And before we conclude today's call, I want to extend my sincere appreciation and gratitude for the foundational work that has brought us closer to developing and delivering on our mission. I'd like to thank the entire Theriva team, our investors and the many people who have been supportable along the way, including our patients and their families. With that, we're happy to take a few questions.
To continue reading the Q&A session, please click here.
Paulness
6 months ago
NEWS -- Theriva™ Biologics Reports Third Quarter 2023 Operational Highlights and Financial Results
VIRAGE, the Phase 2b clinical trial of VCN-01 in combination with chemotherapy for metastatic Pancreatic Ductal Adenocarcinoma remains on track to complete enrollment in the first half of 2024; multiple patients have received second doses of VCN-01, which continues to be well tolerated with a safety profile consistent with prior clinical trialsPresented survival outcomes data from the Phase 1 investigator-sponsored study evaluating VCN-01 in combination with durvalumab in patients with recurrent/metastatic squamous cell carcinoma of the head and neck at the European Society for Medical Oncology (ESMO) Congress 2023Data from the Phase 1 investigator-sponsored study evaluating VCN-01 in combination with CAR-T cell immunotherapy in patients with pancreatic and serous epithelial ovarian cancer was presented at the Society for Immunotherapy of Cancer’s (SITC) 2023 Annual MeetingAs of September 30, 2023, Theriva Biologics reports $31.2 million in cash, which is expected to provide runway into the first quarter of 2025Conference call and webcast to be held on Monday, November 13th at 8:30 a.m. ETROCKVILLE, Md., Nov. 13, 2023 (GLOBE NEWSWIRE) — Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today reported financial results for the third quarter ended September 30, 2023, and provided a corporate update.
“We are encouraged by the growing clinical data that underscores the promise of our systemically administered oncolytic adenovirus and lead program, VCN-01, in key indications and combinations,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “Data from the ongoing study of VCN-01 in combination with durvalumab in patients with recurrent/metastatic squamous cell carcinoma of the head and neck (R/M HNSCC) were recently presented at the annual ESMO Congress. Results showed enhanced patient survival, which correlated with VCN-01 mediated increases in the CPS (combined positive score for PD-L1 staining), a key determinant of outcomes with anti-PD-(L)1 checkpoint inhibitor therapies. Together with data presented at this year’s SITC meeting, these data further validate the feasibility of combining VCN-01 with immunotherapies.”
Mr. Shallcross continued, “We continue to advance VIRAGE, our Phase 2b trial of VCN-01 in newly-diagnosed metastatic pancreatic ductal adenocarcinoma (PDAC), with patients dosed across sites in the U.S. and Spain. We have observed a consistent safety and tolerability profile and remain on track to complete enrollment for VIRAGE in the first half of 2024. As part of our commitment to transforming therapeutic approaches for devastating cancers, we will meet with the FDA before year-end to discuss the development pathway for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma. While our key area of focus is on advancing and maximizing the therapeutic potential of VCN-01, we continue to explore opportunities to bolster our pipeline with new oncolytic virus candidates from utilizing our Albumin Shield technology.”
Recent Program Highlights and Anticipated Milestones:
VCN-01:
Dosing is underway and enrollment continues to progress for VIRAGE, the randomized, controlled, multicenter, open-label Phase 2b trial of VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first line therapy in newly diagnosed metastatic PDAC patients. Dosing at sites across the U.S. and Spain continues and VCN-01 has been well tolerated with a safety profile consistent with prior clinical trials. The trial is expected to enroll 92 patients and remains on track to complete enrollment in H1 2024.The Institut Catala d’Oncologia (ICO) presented Phase 1 data from the investigator-sponsored study evaluating VCN-01 in combination with durvalumab for patients with R/M HNSCC. Encouraging survival was observed in patients progressing to anti-PD(L)-1 agents after systemic VCN-01 in combination with durvalumab. These data were featured in a poster presentation at the ESMO Congress, held both virtually and in Madrid, Spain from October 20-24, 2023.Theriva hosted a virtual KOL event featuring expert oncologist Ricard Mesia, M.D. (Institut Català d’Oncologia / Catalan Institute of Oncology). In addition to reviewing key takeaways from the ESMO poster presentation, Dr. Mesia discussed the unmet medical needs in head and neck cancer, current treatment limitations, and the therapeutic potential of VCN-01 to elicit an extended anti-tumor immune response.The University of Pennsylvania presented initial data from a Phase 1 study evaluating VCN-01 in combination with mesothelin-directed lentiviral transduced human chimeric antigen receptor modified T cells (huCART-meso) in patients with pancreatic and serous epithelial ovarian cancer. Initial results highlight the feasibility of administering VCN-01 with huCART-meso cells to treat solid tumors. These data were featured in a poster presentation at the SITC Annual Meeting, held both virtually and in San Diego, November 1-5, 2023.Additional anticipated milestones:The Company will meet with the FDA before year-end to discuss the clinical development and potential registration pathway for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma.SYN-004 (ribaxamase):
Dosing is underway for the ongoing Phase 1b/2a randomized, double-blinded, placebo-controlled clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant (HCT) recipients for the prevention of acute graft-versus-host-disease (aGVHD). SYN-004 appeared to be well tolerated in HCT patients treated with IV meropenem and SYN-004 was not detected in blood samples from the majority of the evaluable patients. The trial is on track to complete enrollment in the second cohort in H1 2024.Third Quarter Ended September 30, 2023 Financial Results
General and administrative expenses decreased to $212,000 for the three months ended September 30, 2023, from $2.4 million for the three months ended September 30, 2022. This decrease of 91% is primarily comprised of the decrease in the fair value of the contingent consideration of $1.6 million, along with lower salary and bonus costs, investor relations fees, audit fees, travel, and VCN administrative expenses not included in the prior year, offset by an increase in consulting fees. The charge related to stock-based compensation expense was $95,000 for the three months ended September 30, 2023, compared to $93,000 for the three months ended September 30, 2022.
Research and development expenses increased to $4.0 million for the three months ended September 30, 2023, from approximately $2.6 million for the three months ended September 30, 2022. This increase of 56% is primarily the result of higher clinical trial expenses related to our VIRAGE Phase 2 clinical trial of VCN-01 in PDAC, offset by decreased expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, Phase 1a clinical trial of SYN-020, and decreased manufacturing expenses related to our Phase 1a clinical trial of SYN-020. We anticipate research and development expenses to increase as we continue enrollment in our VIRAGE Phase 2 clinical trial of VCN-01 in PDAC and our ongoing Phase 1 clinical trial in retinoblastoma, expand GMP manufacturing activities for VCN-01, and continue supporting our VCN-11 and other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $40,000 for the three months ended September 30, 2023, compared to $28,000 related to stock-based compensation expense for the three months ended September 30, 2022.
Other income was $388,000 for the three months ended September 30, 2023 compared to other income of $161,000 for the three months ended September 30, 2022. Other income for the three months ended September 30, 2023 is primarily comprised of interest income of $382,000 and an exchange gain of $6,000. Other income for the three months ended September 30, 2022 is primarily comprised of interest income of $170,000 offset by an exchange loss of $9,000.
Cash and cash equivalents totaled $31.2 million as of September 30, 2023, compared to $41.8 million as of December 31, 2022.
Conference Call
Theriva Biologics will host a conference call on Monday, November 13, 2023, at 8:30 a.m. ET to discuss its financial results for the quarter ended September 30, 2023 and provide a corporate update. Individuals may participate in the live call via telephone by dialing 1-877-451-6152 (domestic) or 1-201-389-0879 (international) and using the conference ID: 13741546. Participants are asked to dial in 15 minutes before the start of the call to register. Investors and the public can access the live and archived webcast of this call via the “News & Media” section of the company’s website, https://www.therivabio.com, under “Events” or by clicking here, up to 90 days after the call.
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company is advancing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. The Company’s lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients; and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding VIRAGE remaining on track to complete enrollment in the first half of 2024, VCN-01 continuing to be well tolerated with a safety profile consistent with prior clinical trials, cash providing a runway into the first quarter of 2025, continuing to advance VIRAGE in newly-diagnosed metastatic PDAC with patients dosed across sites in the U.S. and Spain, the therapeutic potential of VCN-01 to elicit an extended anti-tumor immune response, plans to meet with regulatory agencies before year-end to discuss the development pathway for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma, continuing to explore opportunities to bolster the Company’s pipeline with new oncolytic virus candidates from utilizing its Albumin Shield technology, the VIRAGE trial enrolling 92 patients, the SYN-004 trial being on track to complete the second cohort in H1 2024 and research and development expense increasing as the Company continues enrollment in the VIRAGE Phase 2 clinical trial of VCN-01 in PDAC and its ongoing Phase 1 clinical trial in retinoblastoma, expanding GMP manufacturing activities for VCN-01, and continuing supporting our VCN-11 and other preclinical and discovery initiatives. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s and VCN’s ability to reach clinical milestones when anticipated, including completion of enrollment in Virage in the first half of 2024 and completing the SYN-004 second cohort in the first quarter of 2024, generating clinical data that establishes VCN-01 being an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma and combining with immunotherapy products to treat solid tumors, the Company’s ability to successfully combine and operate the business of the Theriva Biologics and VCN, the Company’s and VCN’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s and VCN’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s and VCN’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s and VCN’s ability to maintain license agreements, the continued maintenance and growth of the Company’s and VCN’s patent estate, the ability to continue to remain well financed and the cash providing a runway into the first quarter of 2025, and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Paulness
6 months ago
NEWS -- Theriva™ Biologics and Sant Joan de Déu-Barcelona Children’s Hospital Advance Strategic Collaboration to Explore the Combination of VCN-01 with Topoisomerase Inhibitors to Treat Cancer
- Theriva signs exclusive option to license intellectual property from Sant Joan de Déu-Barcelona Children’s Hospital (SJD) to explore the therapeutic potential of VCN-01 in combination with topoisomerase I inhibitors -
- Strengthens long-term research collaboration with SJD and builds on ongoing trial evaluating VCN-01 in pediatric cancers -
- Emphasizes VCN-01’s potential for use in diverse chemotherapy combinations and cancer indications -
ROCKVILLE, Md. and BARCELONA, Spain, Nov. 02, 2023 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, and Sant Joan de Déu-Barcelona Children’s Hospital (“SJD”), a university hospital specializing in maternal, child and adolescent health care, today announced that Theriva has executed an exclusive worldwide option to negotiate an exclusive license certain SJD intellectual property rights related to the use of VCN-01 in combination with topoisomerase I inhibitor chemotherapies for the treatment of cancer. VCN-01 is Theriva’s systemic, selective, stroma-degrading oncolytic adenovirus.
The collaboration builds on growing data that suggests coadministration of VCN-01 with topoisomerase I inhibitors such as topotecan can enhance VCN-01 replication and antitumor activity in preclinical cancer models. Combination of VCN-01 with a topoisomerase I inhibitor is expected to provide a synergistic antitumor effect wherein a chemotherapy-mediated increase in tumor VCN-01 levels may enable greater degradation of the tumor stroma, significantly increasing chemotherapy access and tumor destruction.
“This option with SJD reinforces our strategy of employing VCN-01’s unique modes-of-action to improve patient outcomes by enabling effective use of VCN-01 with different classes of chemotherapies and immunotherapies in otherwise refractory cancers,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “Topoisomerase I inhibitors are used to treat a range of challenging tumors, including lung, cervical, colorectal, and pancreatic cancers as well as retinoblastoma. Recent findings suggest that the combination of VCN-01 and topoisomerase I inhibitors may provide a synergistic antitumor effect to improve therapeutic outcomes in these indications. SJD has been a valuable, long-term, research partner with Theriva, and we are very pleased to advance our collaboration with SJD to refine the technology and potentially advance a VCN-01 and topoisomerase I inhibitor combination into advanced clinical trials.”
Angel Carcaboso, Ph.D., senior group leader at SJD, commented, “The new treatment combination shows a powerful synergy in preclinical human cancer models, including intraocular and leptomeningeal retinoblastoma, Ewing sarcoma and neuroblastoma. Synergy happens through the effect of topotecan increasing the susceptibility of cancer cells to VCN-01 infection. The main goal of our work is to bring new treatments to children with catastrophic diseases, and our smooth partnership with Theriva facilitates the translation of our laboratory results to phase 1 trials.”
Theriva will pay SJD an option fee of twenty-five thousand Euros (€25,000). Final license terms will be negotiated during a 12-month option period.
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells, and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 90 patients to date in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.
About Theriva™ Biologics, Inc.
Theriva Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include: (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at https://www.therivabio.com.
About Sant Joan de Déu – Barcelona Children’s Hospital
Sant Joan de Déu-Barcelona Children’s Hospital (SJD) is a university hospital specializing in maternal, child and adolescent health care. Founded in 1867, SJD belongs to the Hospitaller Order of Saint John of God and is a private, non-profit institution that has been integrated in the public network of the Spanish national health system since 1973. SJD is the largest children’s hospital in Spain and is one of the most recognized of its category across Europe. The hospital is accredited by the Spanish Ministry of Health as a reference centre for all Spain in a wide range of specialties such as Onco-haematology, Cardiology and Cardiac Surgery, Orthopedics, Neurosciences, Genetics, Rare Diseases, Ophthalmology and Reconstructive Surgery to name a few. The hospital is also accredited by the European Commission in 14 European Reference Networks for specialized care in rare diseases.
Associated with the University of Barcelona since 1994 and with the Hospital Clínic of Barcelona, a university adult hospital, SJD has a specialized pediatric research program managed by the Sant Joan de Déu Research Foundation. SJD together with the Hospital Clínic is one of the most well-known hospital alliances in Spain and an international reference for highly specialized hospital care, teaching and research. Thanks to numerous donors, on October 18, 2018 the hospital had the stone laying ceremony for the future SJD Pediatric Cancer Center Barcelona, which will be one of the largest pediatric oncology centers in Europe.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding data suggesting coadministration of VCN-01 with topoisomerase I inhibitors such as topotecan can enhance VCN-01 replication and antitumor activity in preclinical cancer models; the combination of VCN-01 with a topoisomerase I inhibitor providing a synergistic antitumor effect to improve therapeutic outcomes in these indications; a chemotherapy-mediated increase in tumor VCN-01 levels enabling greater degradation of the tumor stroma, significantly increasing chemotherapy access and tumor destruction; the Company’s work bringing new treatments to children with catastrophic diseases and SJD’s smooth partnership with the Company facilitating the translation of laboratory results to phase 1 trials. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, results of the research collaboration enhancing VCN-01 replication and antitumor activity in preclinical cancer models; the Company’s ability to complete enrollment in on-going and planned clinical trials when anticipated and anticipated results, the Company’s ability to address the unmet medical needs for treatment of PDAC and other refractory cancers, the Company’s ability to take advantage of the potential benefits of orphan drug designation, the Company’s ability to reach clinical milestones when anticipated, the Company’s ability to successfully operate the combined US and Spanish business entities, the Company’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s ability to maintain license agreements, the continued maintenance and growth of the Company’s patent estate, the ability to continue to remain well financed and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Paulness
6 months ago
NEWS -- Theriva™ Biologics Presents Survival Outcomes Data from Phase 1 Study Evaluating VCN-01 in Combination with Durvalumab in Patients with Recurrent/Metastatic Squamous Cell Carcinoma of the Head and Neck at ESMO Congress 2023
-Results show enhanced patient survival, correlating with VCN-01 mediated increases in the CPS score, a key determinant of outcomes with anti-PD-(L)1 checkpoint inhibitor therapies-
-Key Opinion Leader (KOL) webinar featuring expert oncologist Ricard Mesia M.D., Ph.D., to be held today, Monday, October 23, 2023 at 8:00 a.m. ET-
ROCKVILLE, Md., Oct. 23, 2023 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced new clinical data from the Phase 1 investigator-sponsored study with the Institut Catala d’Oncologia (ICO) evaluating VCN-01 in combination with durvalumab for patients with recurrent/metastatic squamous cell carcinoma of the head and neck (R/M HNSCC). These data were presented at the European Society for Medical Oncology (ESMO) Congress, held both virtually and in Madrid, Spain from October 20-24, 2023.
“Results presented at ESMO further validate VCN-01’s unique mechanism of action for devastating cancers with high unmet need,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “We are encouraged by the data generated to date, highlighted by the enhanced patient survival, correlating with VCN-01 mediated increases in the CPS score, a key determinant of outcomes with anti-PD-(L)1 checkpoint inhibitor therapies. These results build on the previously reported acceptable safety profile seen with sequential dosing of VCN-01 and durvalumab. Taken together, we believe VCN-01 based combinations may address the need for improved treatments with the potential to overcome previous resistance to anti-PD-(L)1 therapies in patients with R/M HNSCC.”
The poster (#937P) titled “Survival Outcomes in Phase I Trial Combining VCN-01 and Durvalumab (MEDI4736) in Subjects with Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma Refractory to Previous Immunotherapy Treatment,” was presented by Maria Jové (Hospitalet de Llobregat, Spain).
Presentation Highlights:
Key Takeaway: VCN-01 combined with durvalumab showed encouraging overall survival (OS) in patients who previously progressed on anti-PD(L)-1 therapy.Survival: VCN-01 induced upregulation of PD-L1, which correlated with enhanced patient survival.In the concomitant (CS) cohort at the 3.3×1012 viral particles (vp) dose, overall survival (OS) was 10.4 months and progression free survival (PFS) was 1.7 months.In the sequential (SS) cohort at the 3.3×1012vp dose OS was 15.5 months and PFS was 3.7, whereas in the SS cohort at the 1×1013 vp dose OS was 17.3 months and PFS was 2.1 months.VCN-01 induces changes in the immune status of tumorsVCN-01 combined with durvalumab increased CD8 T cells, a marker of tumor inflammation and the expression of PD(L)-1 in tumors. An increase of PD(L)-1 CPS (8/11 at day 8; 8/10 at day 28) and CD8 T cells (7/11 at day 8; 5/10 at day 28) from baseline were found in tumor biopsies.VCN-01 alone increased the CPS score of tumor biopsies at day 8 after administration by 62.5% in the sequential arm.VCN-01 induced PD(L)-1 upregulation with enhanced patient survival. A statistical correlation was observed between CPS on day 8 and patient OS (p=0.005).Pharmacodynamics and shedding of VCN-01PH20 expression from VCN-01 peaked on day 3-8 and remained elevated in some patients up to day 42. Quantification of VCN-01 genomes in stool demonstrated viral shedding that peaked at day 8.KOL Webinar on Monday, October 23, 2023 at 8:00 a.m. ET (2:00 p.m. CEST)
The webinar will feature KOL, Ricard Mesia, M.D., Ph.D., head of Medical Oncology Department at Catalan Institut of Oncology in Barcelona. Dr. Mesia will discuss the unmet medical need in the head and neck cancer treatment landscape, the current limitations, and the need for new approaches, along with the key takeaways from Theriva’s ESMO poster presentation. A live Q&A session will follow the formal discussion. To register for the event, please click here. An archived webcast will also be accessible in the “Events” section of the company’s website at https://www.therivabio.com.
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection).
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include: (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at https://www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding VCN-01 based combinations addressing the need for improved treatments with the potential to overcome previous resistance to anti-PD-(L)1 therapies in patients with R/M HNSCC. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s ability to complete enrollment in its trials when anticipated, the Company’s ability to address the unmet medical needs for treatment of cancer and related diseases, the Company’s ability to take advantage of the potential benefits of orphan drug designation, the Company’s ability to reach clinical milestones when anticipated, the Company’s ability to successfully operate the combined US and Spanish business entities, the Company’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s ability to maintain license agreements, the continued maintenance and growth of the Company’s patent estate, the ability to continue to remain well financed and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Paulness
7 months ago
NEWS -- Theriva™ Biologics Announces Presentation at ESMO Congress 2023 Featuring Survival Outcomes in Phase 1 Study Evaluating VCN-01 in Combination with Durvalumab in Patients with Recurrent/Metastatic Squamous Cell Carcinoma of the Head and Neck
Results from investigator-sponsored study in collaboration with the Institut Catala d’Oncologia (ICO) show enhanced patient survival, correlating with VCN-01 induced upregulation of PD(L)-1Key Opinion Leader (KOL) webinar featuring expert oncologist Ricard Mesia M.D., Ph.D., to be held Monday, October 23, 2023 at 8:00 a.m. ETROCKVILLE, Md., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced a presentation of Phase 1 data from the investigator-sponsored study evaluating VCN-01 in combination with durvalumab for patients with recurrent/metastatic squamous cell carcinoma of the head and neck (R/M HNSCC). Encouraging survival was observed in patients progressing to anti-PD(L)-1 agents after systemic VCN-01 in combination with durvalumab. Data will be featured in a poster presentation at the European Society for Medical Oncology (ESMO) Congress, being held both virtually and in Madrid, Spain from October 20-24, 2023.
“We are encouraged by the biological activity observed in R/M HNSCC patients previously treated with anti-PD(L)-1 agents, where new options are urgently needed to offer patients the best chance of long-term survival,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “Results show enhanced patient survival, which correlated with VCN-01 induced upregulation of PD(L)-1 and underscores the promise of VCN-01-based combination approaches that may transform treatment for devastating cancers with high unmet needs. We look forward to leveraging our findings as we advance VCN-01 through clinical development.”
Key data and conclusions featured in the ESMO presentation include:
20 patients were enrolled with a median of 4 prior lines of therapy, from which six in the concomitant (CS) (single dose of VCN-01 in combination with durvalumab on day 1) and 12 in the sequential (SS) (single dose of VCN-01 on day -14 and durvalumab on day 1) were evaluable for response.In the CS cohort at the 3.3×1012 viral particles (vp) dose, overall survival (OS) was 10.4 months.In the SS cohort at the 3.3×1012vp dose OS was 15.5 months, whereas in the SS cohort at the 1×1013 vp dose OS was 17.3 months.11 patients (61.1%) were alive >12 months (2 in CS; 5 in SS at 3.3×1012vp, 4 in SS at 1×1013 vp).In spite of the advanced stage of the disease and objective response rate of 0%, most of the patients appeared to benefit from subsequent treatment.Biological activity: Patients showed VCN-01 replication and increased serum hyaluronidase levels were maintained for over six weeks.Observed an increase in CD8 T cells, a marker of tumor inflammation and an upregulation of PD-L1 in tumors.Increase of PDL1-CPS (16/21; p=0.013) and CD8 T-cells (12/21; p=0.007) from baseline were found in tumor biopsies.CPS score of tumor biopsies was increased by administration of VCN-01 at day 8 after administration in the sequential group.A statistical correlation was observed between CPS on day 8 and patient OS (p=0.005).The full abstract for the presentation (#937P) is accessible on the ESMO Congress portal and the poster will be available starting Sunday, October 22, 2023 at 9:00 a.m. CEST. Additional details of the poster are provided below.
Title: Survival Outcomes in Phase I Trial Combining VCN-01 and Durvalumab (MEDI4736) in Subjects with Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma Refractory to Previous Immunotherapy TreatmentPresenting Author: Maria Jové (Hospitalet de Llobregat, Spain)Poster Session Date and Time: Sunday, October 22 from 12:00-1:00 p.m. CESTLocation: Hall 8 of the IFEMA Madrid, SpainKOL Webinar on Monday, October 23, 2023 at 8:00 a.m. ET (2:00 p.m. CEST)
The webinar will feature KOL, Ricard Mesia, M.D., Ph.D., head of Medical Oncology Department at Catalan Institut of Oncology in Barcelona. Dr. Mesia will discuss the unmet medical need in the head and neck cancer treatment landscape, the current limitations, and the need for new approaches, along with the key takeaways from Theriva’s ESMO poster presentation. A live Q&A session will follow the formal discussion. To register for the event, please click here. An archived webcast will also be accessible in the “Events” section of the company’s website at https://www.therivabio.com.
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection).
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include: (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding the VCN-01-based combination approaches transforming treatment for devastating cancers with high unmet needs and leveraging the findings as VCN-01 advances through clinical development. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s ability to complete enrollment in its trials when anticipated and anticipated results, the Company’s ability to address the unmet medical needs for treatment of cancer and related diseases, the Company’s ability to take advantage of the potential benefits of orphan drug designation, the Company’s ability to reach clinical milestones when anticipated, the Company’s ability to successfully operate the combined US and Spanish business entities , the Company’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s ability to maintain license agreements, the continued maintenance and growth of the Company’s patent estate, the ability to continue to remain well financed and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
Paulness
9 months ago
NEWS -- Theriva™ Biologics Announces Key Progress in VIRAGE, an Ongoing, Multinational Phase 2b Trial of VCN-01 in Combination with Chemotherapy in Pancreatic Ductal Adenocarcinoma
– Initiated dosing at U.S. sites for VIRAGE –
– Second doses of intravenous VCN-01 administered to patients in Spain and were well tolerated –
– VIRAGE remains on track to complete enrollment in the first quarter of 2024 –
ROCKVILLE, Md., Aug. 02, 2023 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced key progress in VIRAGE, a multinational, Phase 2b, randomized, open-label, controlled clinical trial evaluating VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first-line therapy for patients with metastatic pancreatic ductal adenocarcinoma (PDAC). Patient dosing has initiated in the U.S. and with four sites open in the U.S. and eight sites open in Spain the trial remains on track to be fully-enrolled in the first quarter of 2024. Dosing in Spain initiated in January 2023 and the first patients have now received their second doses of intravenous VCN-01, which were well tolerated with a safety profile consistent with prior clinical trials.
VCN-01 is Theriva’s systemic, selective, stroma-degrading oncolytic adenovirus. VCN-01 has been granted Orphan Drug designations from the US Food and Drug Administration and the European Medicines Agency for the treatment of pancreatic cancer.
“Initiating dosing in the U.S., and the completion of the second VCN-01 doses for the first patients in Spain, are important accomplishments that add to the strong momentum for VIRAGE, which remains on track to complete enrollment in the first quarter of 2024,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “With a dearth of novel therapies available and a five-year survival rate for metastatic PDAC of only 3%, pancreatic cancer is an indication that is ripe for innovation. Through VIRAGE’s advancement, we aim to demonstrate VCN-01’s ability to address the unmet needs of pancreatic cancer patients by synergistically combining with standard-of-care chemotherapy. We are extremely encouraged by the favorable safety profile following the advancement to the second dose that further differentiates and positions VCN-01 as a leading oncolytic adenovirus. More broadly, this trial will enable us to determine the feasibility of repeated dosing of VCN-01, which could shift the paradigm for standardized treatment cycles that are well established in cancer chemotherapy and immunotherapy, and thereby lead to improved clinical outcomes for patients with PDAC and other solid cancers.”
About VIRAGE
VIRAGE is a two-arm Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. VIRAGE is expected to enroll up to 92 adult participants at up to 25 sites across the US and Spain. In both the control and treatment arms, patients will receive gemcitabine/nab-paclitaxel standard-of-care chemotherapy over 28-day cycles. In the treatment arm only, patients will also receive systemically administered VCN-01 seven-days prior to the first and fourth cycles of gemcitabine/nab-paclitaxel treatment. Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, objective response rate, and measures of biodistribution, VCN-01 replication, and immune response. Since this is an open-label trial, progress will be monitored very closely and steps to accelerate the clinical program may be implemented if supported by the emerging data. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection).
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include: (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at https://www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding the Virage trial remaining on track to be fully-enrolled in the first quarter of 2024, the trial enabling the Company to determine the feasibility of repeated dosing of VCN-01, the feasibility of repeated doses shifting the paradigm for standardized treatment cycles that are well established in cancer chemotherapy and immunotherapy, and the trial leading to improved clinical outcomes for patients with PDAC and other solid cancers. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s ability to complete enrollment in the Virage trial when anticipated and anticipated results, the Company’s ability to address the unmet medical needs for treatment of PDAC, the Company’s ability to take advantage of the potential benefits of orphan drug designation, the Company’s ability to reach clinical milestones when anticipated, the Company’s ability to successfully operate the combined US and Spanish business entities , the Company’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s ability to maintain license agreements, the continued maintenance and growth of the Company’s patent estate, the ability to continue to remain well financed and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
mailto://ccalabrese@lifesciadvisors.com
917-680-5608
subslover
10 months ago
NEWS
Theriva Biologics Announces Orphan Drug Designation Granted by the U.S. FDA for VCN-01 for the Treatment of Pancreatic Cancer
ROCKVILLE, Md., June 27, 2023 (GLOBE NEWSWIRE) -- Theriva Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to lead clinical candidate VCN-01, Theriva’s systemic, selective, stroma-degrading oncolytic adenovirus for the treatment of pancreatic cancer. In VIRAGE, the ongoing multinational Phase 2b clinical study, intravenous VCN-01 is being evaluated in combination with standard-of-care (SoC) chemotherapy (gemcitabine/nab-paclitaxel) as a first line therapy for patients with pancreatic ductal adenocarcinoma (PDAC). Previously, the FDA granted orphan drug designation to VCN-01 for treatment of retinoblastoma.
“The FDA’s decision to grant orphan drug designation to VCN-01 highlights the urgent need for new treatment options for patients with PDAC, which has one of the lowest survival rates among all cancers,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “Efforts to improve upon the standard of care treatment have largely stalled, despite the growing incidence of PDAC, and the need for novel therapies in this indication is acute. The growing clinical data that underscore VCN-01’s multiple modes of action and the compelling clinical outcomes observed in Phase 1 studies of VCN-01 in combination with chemotherapy or immunotherapy in patients with PDAC and other solid tumors, give us confidence that VCN-01 has the potential to address this unmet medical need.”
The FDA’s Office of Orphan Products Development grants orphan status to drugs being developed to treat, diagnose, or prevent a rare disease or condition affecting fewer than 200,000 people in the United States. Orphan Drug Designation is designed to provide drug developers with various benefits to support the development of novel drugs, including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance and potential qualification for expedited development programs.
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.
About VIRAGE
VIRAGE is a two-arm Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. VIRAGE is expected to enroll up to 92 adult participants at up to 25 sites across the US and Europe. In both the control and treatment arms, patients will receive gemcitabine/nab-paclitaxel standard of care chemotherapy over 28-day cycles. In the treatment arm only, patients will also receive systemically administered VCN-01 seven-days prior to the first and fourth cycles of gemcitabine/nab-paclitaxel treatment. Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, objective response rate, and measures of biodistribution, VCN-01 replication, and immune response. Since this is an open label trial, progress will be monitored very closely and steps to accelerate the clinical program may be implemented if supported by the emerging data. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).
About Theriva Biologics, Inc.
Theriva Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding VCN-01’s potential to address the unmet medical need for treatments for PDAC, the potential benefits of orphan drug designation including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance and potential qualification for expedited development, expected to enrollment of the Virage of up to 92 adult participants at up to 25 sites across the US and Europe. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s ability to address the unmet medical needs for treatment of PDAC, the Company’s ability to take advantage of the potential benefits of orphan drug designation, the Company’s ability to reach clinical milestones when anticipated, the Company’s ability to successfully operate the combined US and Spanish business entities , the Company’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s ability to maintain license agreements, the continued maintenance and growth of the Company’s patent estate, the ability to continue to remain well financed and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
917-680-5608
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Source: Theriva Biologics, Inc.
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