DewDiligence
4 years ago
PTLA -40% on soft 4Q19 Andexxa sales—consistent with yesterday’s AH session (#msg-153217853).
Why such a steep selloff? 4Q19 Andexxa sales of $28M were down 15% from 3Q19, and investors evidently aren’t buying these excuses in the PR: Fourth quarter Andexxa net sales in the U.S. were impacted primarily by two factors:
• A $5 million gross to net adjustment due to a return reserve for short-dated product. The Company expects this to be mitigated going forward by its current longer-dated, 36-month product, which began shipping in November 2019.
• Flat quarter over quarter demand due to a decrease in utilization, primarily in tier 1 accounts. While physician demand remains strong, the Company believes that in certain of these accounts, hospital pharmacies curtailed use of Andexxa following drug utilization reviews in an effort to manage pharmacy budgets. Following this reduction, re-ordering patterns are stabilizing in many of these accounts. Even without the adjustment in the first bullet item, 4Q19 sales would’ve been only flat vs 3Q19—and well short of prior guidance.
whytestocks
4 years ago
News: $PTLA Portola Pharmaceuticals Presents New Interim Data from Ongoing Phase 2a Study of its Oral SYK/JAK Inhibitor Cerdulatinib in Heavily Pre-Treated T-Cell Malignancies
SOUTH SAN FRANCISCO, Calif. , Dec. 8, 2019 /PRNewswire/ -- Portola Pharmaceuticals, Inc. ® (Nasdaq: PTLA) today announced new interim results from the Company's ongoing Phase 2a study of cerdulatinib, an investigational, oral SYK/JAK inhibitor, in patients with specific subtype...
Got this from PTLA - Portola Pharmaceuticals Presents New Interim Data from Ongoing Phase 2a Study of its Oral SYK/JAK Inhibitor Cerdulatinib in Heavily Pre-Treated T-Cell Malignancies
H2R
5 years ago
Europe conditional Marketing Authorization for Ondexxya™
https://seekingalpha.com/pr/17490076-european-commission-grants-conditional-marketing-authorization-portola-pharmaceuticals
– Major Advance in the Treatment of European Factor Xa Inhibitor Patients Hospitalized with Life-Threatening Bleeding –
– Initial Launch in Select Countries to Begin 2H 2019 –
SOUTH SAN FRANCISCO, Calif., April 26, 2019 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals, Inc. (PTLA)® (Nasdaq: PTLA) today announced that the European Commission (EC) has granted conditional Marketing Authorization for Ondexxya™ (andexanet alfa). Ondexxya is the first and only antidote approved in Europe for adult patients treated with the Factor Xa inhibitor apixaban or rivaroxaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
“The medical community has been eagerly anticipating the approval of Ondexxya and the ability to rapidly reverse the anticoagulating effects of rivaroxaban or apixaban,” said Jan Beyer-Westendorf, M.D., Head of the Thrombosis Research Unit, Department of Medicine I; Division Hematology, University Hospital Dresden, Germany. “As the number of European patients prescribed these Factor Xa inhibitors continues to increase, so too does the incidence of hospital admissions related to bleeding. The approval of Ondexxya represents a significant step forward in the treatment of these patients, who previously had no approved treatment option.”
The conditional approval of Ondexxya was based on data from two Phase 3 ANNEXA studies (ANNEXA-R and ANNEXA-A) that evaluated the safety and efficacy of Ondexxya in reversing the anticoagulant activity of the Factor Xa inhibitors rivaroxaban or apixaban in healthy subjects, and data from the Phase 3b/4 ANNEXA-4 study that evaluated efficacy and safety data from 352 bleeding patients. Results from all three studies were published in The New England Journal of Medicine – ANNEXA-R and ANNEXA-A in 2015, and ANNEXA-4 in February 2019.
“The unmet need for a reversal agent in Europe is significant, as the number of patients taking a Factor Xa inhibitor is nearly double that of the U.S. and continuing to grow at a significant rate. To help ensure we reach as many patients as quickly as possible, our initial launch efforts will be focused on a select group of countries where Factor Xa use is among the highest,” said Scott Garland, Portola’s president and chief executive officer. “We are very proud that, in just one year, we have achieved both U.S. and European approval of this potentially life-saving medicine and look forward to continuing to expand patient access in these countries and beyond.”
The EC grants conditional authorizations to medicines that fulfill a medical need and show a potential benefit to public health, and for which additional data are required by the EC. As part of the conditional approval, Portola will provide the final study reports for both the ANNEXA-4 trial and the randomized controlled clinical trial requested by the U.S. Food and Drug Administration (FDA), as well as additional pharmacokinetic data.
Ondexxya was approved by the FDA in May 2018 and is marketed by Portola in the U.S. under the trade name Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo].
H2R
5 years ago
Thanks ACTcrazy :)
For now, a positive opinion from the CHMP!
https://seekingalpha.com/pr/17429923-european-chmp-adopts-positive-opinion-ondexxya-andexanet-alfa-portola-pharmaceuticals-factor
SOUTH SAN FRANCISCO, Calif., March 01, 2019 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on the marketing authorization application (MAA) for Ondexxya™ (andexanet alfa). The Committee has recommended that the European Commission (EC) grant Ondexxya conditional approval for the reversal of the anticoagulant effects of the Factor Xa inhibitors apixaban or rivaroxaban in patients experiencing uncontrolled or life-threatening bleeding.
The MAA will now be reviewed by the EC, which has the authority to approve medicines for use in the 28 countries of the European Union, Norway, Liechtenstein and Iceland. If approved, Ondexxya would be the first and only specific reversal agent available in Europe to address life-threatening bleeding associated with the use of apixaban or rivaroxaban. The EC is expected to issue a decision in early May 2019.
Best of luck with your investments!
H2R
5 years ago
Feb 28 coming up!
investors.portola.com/phoenix.zhtml?c=198136&p=irol-newsroomArticle&ID=2380212
CHMP Extends Review Period for Portola Pharmaceuticals’ Ondexxya™ (andexanet alfa)
SOUTH SAN FRANCISCO, Calif., Dec. 11, 2018 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has extended the review period for the Company’s marketing authorization application (MAA) for Ondexxya™ (andexanet alfa), and cancelled the Oral Explanation scheduled for Wednesday, December 12, 2018. An opinion is now expected by February 28, 2019.
Best of luck with your investments!
H2R
5 years ago
Positive Hemostasis in 82 Percent of Evaluable Patients
Portola Pharmaceuticals Announces Full Results from the ANNEXA-4 Study of the Factor Xa Inhibitor Reversal Agent Andexxa® in Patients with Life-Threatening Bleeding
Thu February 7, 2019 4:03 PM|GlobeNewswire|About: PTLA
– Excellent or Good Hemostasis Achieved in 82 Percent of Evaluable Patients –
–Thrombotic Event Rate of 9.7 Percent Across All Patients, No Thrombotic Events Observed Among Patients Who Re-started Oral Anticoagulation Therapy –
– Data Presented as a Late-Breaking Oral Presentation at the International Stroke Conference 2019 and Published in The New England Journal of Medicine –
SOUTH SAN FRANCISCO, Calif., Feb. 07, 2019 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced full results from ANNEXA-4, the Company’s Phase 3b/4 trial of its Factor Xa inhibitor antidote Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo] in patients experiencing acute major bleeding while taking a Factor Xa inhibitor. Data are being presented as a late-breaking oral presentation today at the International Stroke Conference 2019 and published simultaneously online by The New England Journal of Medicine (NEJM).
Full data from 352 patients (249 of which were evaluable for hemostatic efficacy; all 352 were evaluable for safety) showed that Andexxa rapidly and significantly reversed anti-Factor Xa activity when administered as a bolus, and sustained this reversal when followed by a 120-minute infusion. Anti-Factor Xa activity is a measure of the anticoagulant activity of apixaban, rivaroxaban, edoxaban and enoxaparin, the anticoagulants studied in ANNEXA-4. Among all 352 patients, 64 percent (n=227) were treated for intracranial hemorrhage (ICH) and 26 percent (n=90) were treated for a gastrointestinal bleed. Of those evaluated for efficacy 82 percent (n=204) achieved excellent or good hemostasis (stoppage of bleeding) over the 12-hour period following treatment with Andexxa, as determined by an independent adjudication committee.
Within 30 days of enrollment, thrombotic events occurred in 34 patients (9.7 percent) and death occurred in 49 patients (13.9 percent), consistent with previous ANNEXA-4 trial results and with the high background thrombotic risk of the enrolled patient population. The majority of thrombotic events occurred in patients who delayed or did not re-start anticoagulation therapy with a Factor Xa inhibitor during the follow-up period. Among the 100 patients who re-started oral anticoagulation therapy, no thrombotic events were observed. Two patients experienced an infusion reaction and none developed antibodies to Factor Xa or Factor X or neutralizing antibodies to Andexxa.
“The consistency of the hemostatic efficacy and safety – regardless of age, dose, type of Factor Xa inhibitor and type of bleed – confirms the potential of Andexxa to address life-threatening bleeding associated with the use of the Factor Xa inhibitors apixaban or rivaroxaban,” said Truman J. Milling Jr., M.D., FACEP, Seton Dell Medical School Stroke Institute and ANNEXA-4 Executive Committee member. “We now also have new data demonstrating no observed thrombotic events after resumption of oral anticoagulation. This is a particularly significant finding given the clinical importance of re-initiating therapy as soon as possible, even in the wake of a major bleeding event.”
The worldwide use of Factor Xa inhibitors is rapidly growing because of their efficacy and safety profile compared to warfarin and enoxaparin in preventing and treating thromboembolic conditions such as stroke, pulmonary embolism and venous thromboembolism (VTE). This growth has come with a proportional increase in the incidence of hospital admissions and deaths related to bleeding, the major complication of anticoagulation. In the U.S. alone in 2017, there were approximately 140,000 hospital admissions attributable to Factor Xa inhibitor-related bleeding.
“The need for a specific Factor Xa inhibitor reversal agent is clear. As Factor Xa inhibitor use continues to grow, so does the prevalence of life-threatening bleeds such as an ICH, which carries a mortality rate of nearly 50 percent,” said John Curnutte, M.D., Ph.D., head of research and development at Portola. “The consistency of the ANNEXA-4 data, the strong response from the international medical societies that have already added Andexxa to their guidelines and the initial demand following the approval of Andexxa in May 2018 highlight its potential to benefit thousands of patients facing life-threatening bleeding associated with the use of rivaroxaban or apixaban.”
Andexxa received both U.S. Orphan Drug and U.S. Food and Drug Administration (FDA) Breakthrough Therapy designations, and was approved on May 3, 2018 under the FDA's Accelerated Approval pathway. Andexxa is the first and only antidote indicated for patients treated with rivaroxaban or apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding, and it was recently added to the 2019 AHA/ACC/HRS guidelines.
ANNEXA-4 Study Design
ANNEXA-4 is a global, single-arm, open-label clinical trial designed to evaluate andexanet alfa in patients who present with an acute major bleed while receiving apixaban, rivaroxaban, edoxaban or enoxaparin. This multi-center, prospective cohort study was not randomized and all participants received andexanet alfa given as a bolus dose over 20-30 minutes followed by a two-hour (120 minute) infusion. Patients received a low or high dose infusion depending on which Factor Xa inhibitor they received and the time since they received the last dose. Patients were evaluated for 30 days following andexanet alfa administration. The co-primary efficacy endpoints are the maximum percent reduction in anti-Factor Xa activity and assessment of hemostasis over 12 hours following the infusion. Hemostatic efficacy was assessed by an independent endpoint adjudication committee using predetermined criteria as either excellent, good or poor/none. The primary safety endpoints were death, thrombotic events and development of antibodies to Andexxa or to native Factor X and Factor Xa.
H2R
5 years ago
PTLA will target a large number of hospitals
Now that the PAS is approved, the CC comments can be taken more seriously.
So we expect that to continue next year as we target more accounts. So going into the second part of your question, in terms of the Gen 2 rollout, we're - we have multiple tiers. And we've I think discussed this a little bit previously. The tier 1 or our first set of targets are about 600 U.S. hospitals are Level I or Level II trauma centers or have Comprehensive Stroke designation. So those are the biggest institutions across the country. The next tier of about 900 additional accounts, additional hospitals were the Advanced Stroke Center designation.
So those are all pretty important accounts for us. And then finally, from what we've seen in the Early Supply Program, we also want to be able to stock affiliates of these accounts too, because oftentimes patients might present to an affiliate account. That's where we want the drug to get started, if they're showing up there. And then they're transferred to the larger account. So we'll be going on next year to target as many as we can.
Scott Garland, CEO
https://seekingalpha.com/article/4219652-portola-pharmaceuticals-ptla-ceo-scott-garland-q3-2018-results-earnings-call-transcript?part=single
This is to pair up with today's PR:
Full Commercial Launch to Begin January 2019
http://investors.portola.com/phoenix.zhtml?c=198136&p=irol-newsArticle&ID=2381753
I do expect a very good stock performance in 2019
H2R
5 years ago
"PAS" APPROVED !
http://investors.portola.com/phoenix.zhtml?c=198136&p=irol-newsArticle&ID=2381753
U.S. Food and Drug Administration Approves Portola Pharmaceuticals’ Prior Approval Supplement for Andexxa® Generation 2 Manufacturing Process
– Expands Patient Access to Andexxa, the First and Only Antidote for Reversal of
the Factor Xa Inhibitors Rivaroxaban or Apixaban –
– Full Commercial Launch to Begin January 2019 –
SOUTH SAN FRANCISCO, Calif., Dec. 31, 2018 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced that the U.S. Food and Drug Administration (FDA) has approved the Company’s Prior Approval Supplement (PAS) for its large-scale, second generation Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo], allowing for broad commercial launch in the United States.
Andexxa received both U.S. Orphan Drug and FDA Breakthrough Therapy designations and was initially approved on May 3, 2018 under the FDA's Accelerated Approval pathway. It is the first and only antidote indicated for patients treated with rivaroxaban or apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
“It is clear from the response to the Andexxa Early Supply Program that there is significant need for a specific reversal agent that can address life-threatening bleeding associated with the use of the Factor Xa inhibitors apixaban and rivaroxaban,” said Scott Garland, Portola’s president and chief executive officer. "We are pleased to now be able to stock hospitals nationwide and serve all patients in the U.S. who could benefit from the potential life-saving impact of Andexxa.”
The use of Factor Xa inhibitors is rapidly growing because of their efficacy and safety profile compared to enoxaparin and warfarin in preventing and treating thromboembolic conditions such as stroke, pulmonary embolism and venous thromboembolism (VTE). This growth has come with a related increase in the incidence of hospital admissions and deaths related to bleeding, the major complication of anticoagulation. In the U.S. alone in 2017, there were approximately 140,000 hospital admissions attributable to Factor Xa inhibitor-related bleeding.
The Company will provide additional details on the commercial launch plans for Andexxa during its annual corporate webcast scheduled for Tuesday, January 8, at 7:00 a.m. PT (10:00 a.m. ET). The live webcast will be available on the Company's website at www.portola.com.
Nice!!!
Best of luck to Long investors ! 2019 should be a good year!
H2R
5 years ago
PTLA getting ready for the PDUFA - December 31, 2018 for Prior Approval Supplement (PAS)-:
Portola Pharmaceuticals to Webcast Corporate Update on January 8, 2019
SOUTH SAN FRANCISCO, Calif., Dec. 18, 2018 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced that Scott Garland, Portola’s president and chief executive officer, will provide a corporate update on Tuesday, January 8, at 7 a.m. PT (10 a.m. ET). The live webcast will be available on the Company's website at www.portola.com.
http://investors.portola.com/phoenix.zhtml?c=198136&p=irol-newsArticle&ID=2381016
H2R
5 years ago
PR from PTLA:
http://investors.portola.com/phoenix.zhtml?c=198136&p=irol-newsroomArticle&ID=2380212
SOUTH SAN FRANCISCO, Calif., Dec. 11, 2018 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has extended the review period for the Company’s marketing authorization application (MAA) for Ondexxya™ (andexanet alfa), and cancelled the Oral Explanation scheduled for Wednesday, December 12, 2018. An opinion is now expected by February 28, 2019.
The CHMP informed Portola yesterday that it will provide a list of outstanding questions related to the data package the Company submitted for Ondexxya last quarter, which will require additional responses from the Company. The preliminary timetable provided to the Company by the CHMP sets a deadline of January 29, 2019 for responses to the questions followed by a 30-day assessment period for the CHMP to review the Company’s responses.
Ondexxya is under review for the reversal of the anticoagulant effects of the Factor Xa inhibitors apixaban and rivaroxaban in patients experiencing uncontrolled or life-threatening bleeding.
“We will work diligently to address the Committee’s questions as they review and further consider the data package supporting the MAA,” said Scott Garland, Portola’s president and chief executive officer. “We look forward to continuing the dialogue as we progress toward a formal opinion and potential European approval early next year.”
Andexanet alfa was approved by the U.S. Food and Drug Administration in May 2018 and is marketed by Portola in the U.S. under the trade name Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo].
PTLA PR'ed one day after being informed on the CHMP questions. I don't know what else they can do but respond ASAP. Kind of too bad, but does not look like a big deal. At least, the CHMP is examining the submission.A delay.
One of the major catalyst is coming up, the PAS ... this month.
I just checked, it's still on for "Dec. 31st" :
https://www.biopharmcatalyst.com/calendars/pdufa-calendar
H2R
5 years ago
Interesting comment on SA's
https://seekingalpha.com/news/3414289-portolas-cerdulatinib-continues-show-positive-action-mid-stage-lymphoma-study
Fightdoctor
This was a positive study in a drug that may produce something for Portola investors. However, the main reason to be in this stock is for the drug Andexxa, the reversal agent for Eliquis and Xarelto. The drug is approved but the method of producing it, referred to as Gen 1 is inefficient. Portola is only offering this life saving drug to about 160 US hospitals at this time. Investors are awaiting two decisions. The CHMP decision for the European Union is due next week and the FDA approval decision of the Gen 2 method of producing is due by Dec. 31. Portola has stock piled over $300,000,000 of the Gen. 2 drug in anticipation of that approval. The drug is being manufactured by a very reliable Swiss company, Lonza. They have an excellent record of bringing drugs through regulatory processes.
The stock as been beaten down with a very large short interest. That has presented an uncommon opportunity to get in on a company at a low price.
I am a long term believer in Portola and Andexxa. There are lots of opinions as to when this company share price will take off and how high it will go. But, based on the action at the time of previous, less momentous, announcements I am expecting two large pops soon.
H2R
5 years ago
A couple of notes from the CC:
On Finances:
"Importantly, as a result of our change in focus to prioritize Andexxa, we've been able to extend our cash runway into the first half of 2020."
On Andexxa adoption:
" We estimate that more than 4 million people in the United States are currently taking a Factor Xa inhibitor, and we expect that number to continue to grow at strong double-digit rates."
"We believe the success we are having in getting formulary approvals, orders and reorders is setting us up nicely for the anticipated launch of Gen 2 in early 2019."
"While our focus is on the Andexxa U.S. market, we continue to assess our European launch strategy as we approach the CHMP opinion date."
On PAS:
"we are on track for the December 31, 2018 PDUFA data for the anticipated approval of our Andexxa Gen 2 manufacturing process. In fact, we just received notification from the FDA that the PAS was deemed okay to file."
From the Q&A on targets:
" The tier 1 or our first set of targets are about 600 U.S. hospitals are Level I or Level II trauma centers or have Comprehensive Stroke designation. So those are the biggest institutions across the country. The next tier of about 900 additional accounts, additional hospitals were the Advanced Stroke Center designation."
Overall a very good CC in my opinion.
It will take time for the PPS to move, but it will move once revenue start pouring in.
H2R
5 years ago
From the PR
The key for me is the end of year PAS Date: December 31, 2018 and getting set up for the launch in 2019.
So far, it looks like the target is still relevant (and of course I'm hoping for a positive outcome). Financials are OK in view of that launch.
Third Quarter 2018 Financial Results
Total revenue for the third quarter of 2018 was $14.2 million, compared with $3.8 million for the third quarter of 2017. This includes $7.7 million in product revenue for our first full quarter of Andexxa sales and $7.0 million in collaboration and license revenues. Bevyxxa product orders remained relatively flat in the third quarter, during which we recognized a net product loss for Bevyxxa of $552 thousand. This adjustment was primarily related to potential returns of initial launch quantities provided to wholesalers.
Total operating expenses for the third quarter of 2018 were $83.3 million, compared with $84.3 million for the same period in 2017. Total operating expenses for the third quarter of 2018 included $11.4 million in stock-based compensation expense, compared with $10.1 million for the same period in 2017.
Research and development expenses were $40.2 million for the third quarter of 2018, compared with $55.3 million for the third quarter of 2017. The decrease is driven primarily by the timing of manufacturing costs for Andexxa Generation 2 campaigns. Approximately $12 million, or 30 percent, of R&D expense for the quarter and $75 million, or 45 percent, for nine months year-to-date of total R&D expense was related to Andexxa manufacturing.
Selling, general and administrative expenses for the third quarter of 2018 were $38.8 million, compared with $28.9 million for the same period in 2017, reflecting the investment in our field force and marketing for our product launches.
For the third quarter of 2018, Portola reported a net loss of $71.3 million, or $1.08 net loss per share, compared with a net loss of $82.9 million, or $1.41 net loss per share, for the same period in 2017.
Cash, cash equivalents and investments at September 30, 2018 totaled $380.9 million, compared with $534.2 million as of December 31, 2017.
2018 Annual Financial Guidance
For the fiscal year 2018, Portola is updating its guidance for GAAP operating expenses, which are now expected to be between $355 million and $365 million, a decrease from the prior guidance range of between $390 million and $430 million, both including stock-based compensation. The updated guidance reflects the Company’s narrowed focus on a group of key hospitals for the Bevyxxa launch, as well as additional cost savings for the year.
Recent Achievements and Events
Appointed industry veteran Scott Garland as president and chief executive officer.
Submitted Prior Approval Supplement (PAS) to U.S. FDA for large-scale Generation 2 Andexxa manufacturing process; assigned a PDUFA date of December 31, 2018.
Transitioned commercial focus to the ongoing Andexxa U.S. launch under the Early Supply Program.
Secured New Technology Add-on Payment (NTAP) for Andexxa, which became effective as of October 1, 2018.
Implemented new strategy for Bevyxxa U.S. launch focused on establishing 10 Centers of Excellence to provide a model for driving broader adoption.
Received Orphan Drug Designation from the U.S. FDA for cerdulatinib for the treatment of peripheral T-cell lymphoma.
Eight abstracts presented at the European Society of Cardiology (ESC) meeting.
Upcoming Milestones
Anticipated broad launch of Andexxa upon U.S. FDA approval of Generation 2 supply.
Committee for Medicinal Products for Human Use (CHMP) opinion on andexanet alfa, with potential for European approval of andexanet alfa in the first half of 2019.
End-of-Phase 2 meeting and determination of regulatory path forward for cerdulatinib in Q1 2019.
New interim Phase 2a study results for cerdulatinib to be presented in an oral session at the 60th American Society of Hematology (ASH) Annual Meeting. Also accepted for presentation: outcomes-based research on the burden of hospital readmissions for venous thromboembolism among patients with cancer (oral) and two poster presentations on andexanet alfa.
H2R
6 years ago
Cerdulatinib Orphan Drug Designation
http://investors.portola.com/phoenix.zhtml?c=198136&p=irol-newsArticle&ID=2368929
Portola Pharmaceuticals Receives FDA Orphan Drug Designation for Cerdulatinib, an Oral Syk/JAK Inhibitor for the Treatment of Peripheral T-Cell Lymphoma
SOUTH SAN FRANCISCO, Calif., Sept. 25, 2018 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals (Nasdaq: PTLA) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to cerdulatinib, an investigational, oral Syk/JAK inhibitor for the treatment of peripheral T-cell lymphoma (PTCL).
“We are pleased that the FDA has granted cerdulatinib Orphan Drug Designation, as it recognizes its potential to provide a significant clinical benefit to a group of patients with limited treatment options,” said John Curnutte, M.D., Ph.D., Portola’s interim co-president and head of research and development. “We look forward to presenting additional data from the Phase 2a trial at a scientific congress early next year and to continuing discussions with the FDA regarding next steps for the development of cerdulatinib, including the potential for an accelerated approval pathway.”
The FDA's Office of Orphan Products Development grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA market application fees.
About PTCL
PTCL is a group of rare and often fast-growing lymphomas classified as a subtype of non-Hodgkin’s lymphoma. There are approximately 6,000 to 10,000 cases of PTCL annually in the United States. The three most common types of PTCL are anaplastic large cell lymphoma (ALCL), angioimmunoblastic T-cell lymphoma (AITL), and PTCL not otherwise specified (PTCL-NOS), but many other rarer types exist. PTCL is most often treated with a combination of chemotherapies and the options for patients that fail front-line therapy are limited.
About Cerdulatinib
Cerdulatinib is an investigational oral, dual spleen tyrosine kinase (Syk) and janus kinase (JAK) inhibitor that uniquely inhibits two key cell signaling pathways implicated in certain hematologic malignancies and autoimmune diseases. There is a strong rationale for inhibiting both Syk (B-cell receptor pathway) and JAK (cytokine receptors) in B-cell malignancies where both targets have been shown to promote cancer cell growth and survival. In addition, pre-clinical data suggest an important role for Syk and JAK in peripheral T-cell lymphoma (PTCL) tumor survival.
In addition to PTCL, cerdulatinib is being evaluated in an ongoing Phase 2a study among patients with other specific subtypes of B-cell and T-cell Non-Hodgkin Lymphoma (NHL), including relapsed/refractory follicular lymphoma (FL) and chronic lymphocytic lymphoma/small lymphocytic lymphoma (CLL/SLL). The Company reported new data from this study in June at both the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting and at the 23rd Congress of the European Hematology Association (EHA). Cerdulatinib demonstrated broad clinical activity including an objective response rate of 47 percent in all patients, and was generally well tolerated. Additionally, seven of the 20 patients in the PTCL cohort achieved a complete response at the time of presentation.
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