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From Zack's:
CVM: Confirmatory Study Clarity
05/21/2024
By John Vandermosten, CFA
NYSE:CVM
READ THE FULL CVM RESEARCH REPORT
First Quarter Fiscal Year 2024 Update
CEL-SCI Corporation (NYSE:CVM) reported its fiscal second quarter on May 15, 2024 with the submission of its Form 10-Q with the SEC and a press release on the following day. Since our previous update in mid-April, CEL-SCI reported that it had received a nod from the FDA on the design of a confirmatory study allowing it to proceed and announced a new director. Other highlights during the period include the grant of a waiver for pediatric requirements by the European Medicines Agency (EMA) and an article featuring CEL-SCI’s Ligand Epitope Antigen Presentation System (LEAPS) technology in Frontiers in Immunology.
Financial Review
CEL-SCI recognized no revenues for its fiscal second quarter ending March 31, 2023 and incurred operating expenses totaling $7.1 million during the three-month period. This resulted in a net loss available to common shareholders of ($7.2) million, or ($0.14) per share.
For the quarter ending March 31, 2023 versus the same prior year period:
? Expenses for research and development fell 24% to $4.6 million from $6.1 million. Lower Phase III study, employee stock compensation and miscellaneous costs contributed to the decrease;
? General and administrative expenses increased 17% to $2.4 million from $2.1 million on higher consulting fees, employee stock compensation and other miscellaneous expenses;
? Other non-operating items were $14,000 compared to ($8,000) in the prior year;
? Net interest expense of ($0.2) million compared with ($0.2) million was related to lease liabilities and was relatively constant;
? Net loss totaled ($6.7) million versus ($8.0) million or ($0.14) and ($0.18) per share, respectively.
As of March 31, 2024, cash and equivalents totaled $5.3 million. Cash burn for the three-month period amounted to approximately ($9.5) million, up from 2Q:23’s ($7.6) million. During the quarter, CEL-SCI closed on a gross $7.8 million in a common stock offering. CEL-SCI holds no debt on its balance sheet.
CEL-SCI Milestones
? EMA waiver for pediatric requirements – January 2024
? Commissioning of Multikine manufacturing facility – 2024
? Appointment of Mario Gobbo to Board of Directors – April 2024
? Feedback from various regulatory agencies - 2024
? Submission of license application to various agencies – 2024+
? Preparation for Multikine confirmatory trial - 2024
LEAPS Article Published in Frontiers in Immunology
Scientific journal Frontiers in Immunology published an article in its March 2024 issue on CEL-SCI’s LEAPS technology as reported in a March 19th press release. While there are approved treatments for rheumatoid arthritis (RA) including adalimumab and other TNF blockers and JAK inhibitors they do not balance adaptive immune homeostasis. These classes of drugs also present the risk of stimulating other disease. CEL-SCI’s CEL-4000 therapeutic vaccine may be able to treat RA while limiting negative side effects by promoting antigen-specific regulatory rather than inflammatory responses, thereby modulating the immunopathological course of RA. In CEL-SCI’s article entitled Current status of immunological therapies for rheumatoid arthritis with a focus on antigen-specific therapeutic vaccines, the authors discuss in-development and approved products for RA and preclinical data for CEL-4000. CEL-SCI’s candidate is able to redirect autoreactive pro-inflammatory memory T cells towards rebalancing the runaway immune/inflammatory responses that characterize it. The drug’s features require the use of more advanced trial design such as adaptive trials and the use of biomarkers for endpoints. The article concludes by asserting that an antigen-specific immunomodulating vaccine, such as CEL-4000, may deliver therapy for RA without weakening important immune defense mechanisms against microbial infections or cancer.
FDA Confirmatory Study Acceptance
On May 8th, CEL-SCI announced that its selection criteria for populating a confirmatory study and study design was cleared by the FDA. In a recent meeting, the agency indicated that the company may move forward with a confirmatory registration study of Multikine. These patients will be newly diagnosed with advanced primary head and neck cancer with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy). Statistical analysis indicates that 212 subjects will be required to generate the confirmatory data and the FDA has given its blessing to this design. It will be a randomized controlled trial with two arms. The FDA has cleared the trial within the context of the unmet need for squamous cell head and neck cancer (SCCHN).
Based on a discussion of the path forward with the FDA which was included in a 29 page report, the agency responded in written notes accepting the eligibility criteria. It further acknowledged that the nonclinical data appears sufficient, the stratification factors appear reasonable and CEL-SCI’s approach for product specifications is acceptable. Furthermore, the FDA had no safety objections to further treatment of patients with Multikine.
Similar to the IT-MATTERS study, we anticipate that eligible individuals will have a diagnosis of untreated SCCHN, measurable tumor, normal immune function and no use of immunosuppressives in the previous year among other criteria. Based on the performance in the Phase III trial, further population characteristics for the confirmatory study will include patients with no lymph node involvement of the disease and low PD-L1 tumor expression.
The study design calls for a two arm, two stage, 1:1 randomized controlled trial. The first stage will examine pre-surgical response rates, which can be determined shortly after completion of enrollment. Stage two will assess overall survival (OS) as the primary endpoint. Histopathology biomarkers will also be measured.
In its confirmatory trial, CEL-SCI anticipates that it will need to surpass a hurdle of a 10% improvement in overall survival (OS) and produce a hazard ratio of 0.72 or less to merit approval. In the IT-MATTERS study, the target population achieved a 28.5% improvement in OS and produced a hazard ratio of 0.35.
Later sections in the report provided a summary of Multikine’s mechanism of action, emphasizing its ability to enable a local antitumor response to occur. A review of Multikine’s tumor histopathology was provided along with graphics showing pre-surgical response rates and overall survival for the various groupings of patients.
CEL-SCI’s report concluded with a management summary asserting that the FDA recognized an unmet neet for better therapies and is supportive of the confirmatory trial. It also noted the lack of other approved therapies for SCCHN and the lack of success for other immunotherapies in this indication. Details on timing and cost were not provided; however, we anticipate that the management team is in the planning stages for a new trial along with efforts in the other geographies where CEL-SCI seeks to obtain approval.
imanjen13
4 days ago
By now you should have figured out that the daily short sale volume as reported by Finra is not a very good indicator of present or future stock action. A better investment criteria is looking at actual results and future possibilities based on R&D.
There is only one bet to make on this stock: Does Multikine help people with head and neck cancer survive or live longer than those who do not take this treatment. If the results are positive, this company and its shareholders will be a successful.
As for shorting of stocks, with a price this low few investors, other than Market Makers, are participating. If you think about it, sometimes shorts are positive for investors, since, except for bankruptcy, they eventually have to cover and buy to close the position. Ever hear of a short squeeze.
He who sells what isnt' hisn, mst buy it back or go to end prisn- Daniel Drew.
rdneum
3 weeks ago
CEL-SCI Receives FDA Go-Ahead for Its Confirmatory Study of Multikine in the Treatment of Head & Neck Cancer
Critical milestone achieved—the FDA accepted CEL-SCI’s selection criteria defining the Multikine target population before surgery
Selected patient population had 73% survival with Multikine vs. 45% without Multikine based on analysis of completed 928-patient Phase 3 randomized controlled trial
212-patient confirmatory study is de-risked, backed by robust efficacy and safety data from completed Phase 3 trial, the largest in advanced primary head and neck cancer
View CEL-SCI’s comprehensive report regarding the FDA’s go-ahead and the Company’s value proposition for investors here: https://cel-sci.com/wp-content/uploads/2024/05/CEL_SCI-Report-on-FDA-Path-Forward-May-2024-FINAL.pdf
CEL-SCI Corporation (NYSE American: CVM) today announced a significantly positive outcome from its recent meeting with the U.S. Food and Drug Administration (FDA) regarding the path to approval for its first-line investigational cancer immunotherapy Multikine* (Leukocyte Interleukin, Injection). Based on strong safety and efficacy data from CEL-SCI’s completed Phase 3 head and neck cancer study, the FDA indicated CEL-SCI may move forward with a confirmatory Registration Study of Multikine in newly diagnosed advanced primary head and neck cancer patients with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy).
“Through this discussion and agreement with the FDA, we have achieved a tremendous milestone for people who are newly diagnosed with head and neck cancer. The survival benefit was so strong and clear in the target patient population that our confirmatory study needs to enroll only 212 people to confirm what was already achieved in the Phase 3 study. This gives us a clear path forward,” stated CEL-SCI CEO, Geert Kersten. “We are eager to begin the study as soon as possible.”
CEL-SCI published a report on the FDA’s agreement and Multikine’s path forward. Please click on this link https://cel-sci.com/wp-content/uploads/2024/05/CEL_SCI-Report-on-FDA-Path-Forward-May-2024-FINAL.pdf to read the full report. Highlights include:
The FDA agreed to a 212-person confirmatory Registration Study based on the strength of the safety and survival benefit data in the selected target population from the prior 928-person Phase 3 study. The confirmatory study will be a randomized controlled trial with two arms: Multikine treatment plus standard of care versus standard of care alone. As presented at the ESMO cancer conference in October 2023, Multikine-treated patients in the selected group had a 73% 5-year survival vs a 45% 5-year survival in the control group who did not receive Multikine.
Generally, patient selection for different treatments in newly diagnosed head and neck cancer is done only after surgery. That presented CEL-SCI with a challenge, because Multikine has to be given before surgery. By analyzing Multikine's biological mechanism of action, as supported by the completed Phase 3 study, CEL-SCI developed criteria for selecting, before surgery, those patients who would have the best survival from Multikine. The FDA accepted the selection criteria and the proposed study design, which now permits CEL-SCI to enroll patients in the confirmatory study.
CEL-SCI met a very high bar set by the FDA, which requires more stringent analysis for newly-diagnosed patients than for terminal cancer patients. One regulator called these newly-diagnosed cancer patients “much more delicate” and explained that the standard for permitting a new study with these patients has to be more stringent, since they are not all expected to die.
CEL-SCI has been advised by statisticians and physicians that the confirmatory study has a high likelihood of success because a large survival benefit has already been demonstrated in the target population in the completed Phase 3 study. The much smaller confirmatory study—less than a quarter the size of the prior study—will focus on the patients who saw the greatest survival benefit when treated with Multikine.
If approved as a pre-surgical treatment, Multikine should be added to the standard of care for the target population.
The FDA also acknowledged in the meeting that there is a great unmet need in the target population for improved therapies. This is an important factor that weighs in favor of approval for Multikine.
CEL-SCI believes that its de-risked value proposition for investors presents a unique opportunity to invest in a Phase 3 oncology company with a large body of data demonstrating not only tumor responses, but also long-term survival, in the target patient population. The goal of our smaller confirmatory study is to confirm these positive results in a prospectively defined target population.
About CEL-SCI Corporation
CEL-SCI believes that boosting a patient’s immune system while it is still intact should provide the greatest possible impact on survival. Multikine is designed to help the immune system "target" the tumor at a time when the immune system is still relatively intact and thereby thought to be better able to mount an attack on the tumor. In the completed Phase 3 study, CEL-SCI studied patients who were newly diagnosed with locally advanced primary squamous cell carcinoma of the head and neck with the investigational product Multikine administered first, before they received the standard of care, which involved surgery followed by either radiation or chemoradiation. The Phase 3 study enrolled 928 patients. Our approach is unique because most other cancer immunotherapies are administered only after conventional therapies have been tried and/or failed.
After analyzing data from the Phase 3 study, we have better defined the target population for Multikine, which is locally advanced primary head and neck cancer patients with no lymph node involvement and with low PD-L1 tumor expression. In the Phase 3 study, we observed statistically significant survival in this target population, showing that Multikine cut the risk of death in half at five years vs control.
Multikine (Leukocyte Interleukin, Injection) received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck.
The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. When used in this press release, the words "intends," "believes," "anticipated," "plans" and "expects," and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Such statements include, but are not limited to, statements about the terms, expected proceeds, use of proceeds and closing of the offering. Factors that could cause or contribute to such differences include an inability to duplicate the clinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company's potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI's filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K for the year ended September 30, 2023. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.
* Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy. This proprietary name is subject to FDA review in connection with the Company's future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240508140076/en/
Gavin de Windt
CEL-SCI Corporation
(703) 506-9460
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