- FDA assigns PDUFA target action date of March 5, 2021
LAVAL, CANADA, and CAMBRIDGE, ENGLAND – September 21, 2020 – Liminal BioSciences Inc. (Nasdaq: LMNL) (“Liminal BioSciences” or the “Company”), a clinical-stage biopharmaceutical company, announced today that the U.S. Food and Drug Administration ("FDA") acknowledged receipt of the resubmission, by its US subsidiary, Prometic Biotherapeutics Inc., of the Biologic License Application ("BLA") for Ryplazim® (plasminogen) for the treatment of clinical signs and symptoms associated with congenital plasminogen deficiency (“C-PLGD”) in pediatrics and adults. The FDA confirmed that the resubmission is a complete, Class 2 response and has provided a Prescription Drug User Fee Act ("PDUFA") target action date of March 5, 2021.
“We look forward to continuing to work closely with FDA during the BLA review process for Ryplazim as we move one step closer to potentially bringing the first FDA-approved treatment to patients with this devastating disease,” said Kenneth Galbraith, Chief Executive Officer of Liminal BioSciences.
The BLA resubmission for Ryplazim® is supported by data from a Phase 2/3 clinical study which evaluated 15 patients, both pediatric and adults, with congenital plasminogen deficiency over 48 weeks of therapy with Ryplazim®. In the Phase 2/3 clinical study, 100% of the patients enrolled met both primary efficacy endpoints of the clinical study. Adverse events reported in the clinical study were characterized as mild with no patient deaths, serious adverse events or adverse events that caused study discontinuation reported.
“In parallel to the FDA review process, we will build our commercial readiness for a potential approval and potential commercial launch in the US in 2021,” stated Patrick Sartore, Chief Operating Officer – Plasma-Derived Therapeutics. “We are continuing our work to build awareness of congenital plasminogen deficiency with patient advocacy groups, physicians and payors to enable patient access in the US if Ryplazim® is approved by the FDA. In addition, we are exploring development and regulatory pathways in countries outside the US to seek marketing approval for Ryplazim® with potential third-party collaboration partners.”
Under the FDA’s rare pediatric disease designation program, the FDA may grant a priority review voucher (PRV) to a sponsor who receives a product approval for a “rare pediatric disease” which is defined as a serious life-threatening disease in which the serious or life-threatening manifestations primarily affects fewer than 200,000 people in the U.S. Subject to FDA approval of Ryplazim® for the treatment of C-PLGD, the Company may be eligible to receive one priority review voucher, which then could be redeemed to receive priority review for any subsequent marketing application, or sold or transferred to other companies for their programs.
About Congenital Plasminogen Deficiency
Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system and is the main enzyme involved in the lysis of blood clots and clearance of extravasated fibrin. Plasminogen is therefore vital in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis. Patients may be born with the inability to produce sufficient plasminogen naturally, a condition referred to as congenital plasminogen deficiency, or suffer an acute or acquired deficiency following a trauma or an illness. Patients with congenital plasminogen deficiency experience an accumulation of fibrin growths or lesions on mucosal surfaces throughout the body. Many cases are first diagnosed in the pediatric population, and if left untreated, disease manifestations may be organ-compromising. Peer-reviewed publications report that the condition may have a prevalence of 1.6 cases per million globally. Proprietary data sources and analyses involving the U.S. population suggest that the number of people potentially affected by plasminogen deficiency in the United States may be greater than these early epidemiological estimates. Congenital plasminogen deficiency may require lifelong therapy to avoid recurrence of lesions. There are currently no approved therapies for the treatment of congenital plasminogen deficiency.
About Ryplazim® (plasminogen)
Ryplazim® is an investigational plasma-derived protein currently being developed at Prometic Bioproduction Inc., the plasma-derived therapeutics segment of Liminal BioSciences, Inc., for the treatment of congenital plasminogen deficiency (C-PLGD). This highly purified glu-pasminogen acts as a replacement therapy for activated plasminogen, a fundamental component of the fibrinolytic system and the main enzyme involved in the lysis of blood clots, clearance of extravasated fibrin and other vital physical processes. Liminal Biosciences has received Rare Pediatric Disease and Orphan Drug Designations from the FDA for Ryplazim®.
About Liminal BioSciences Inc.
Liminal BioSciences is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing novel treatments for patients suffering from diseases of high unmet medical need, primarily related to fibrosis, including respiratory, liver and kidney diseases. Liminal BioSciences has a deep understanding of certain biological targets and pathways that have been implicated in the fibrotic process, including fatty acid receptors such as FFAR1, or GPR40, G-protein-coupled receptor 84, or GPR84, and peroxisome proliferator-activated receptors, or PPARs. Our lead small molecule product candidate, fezagepras (PBI-4050), is expected to enter an additional Phase 1 clinical trial in Q4-2020 to evaluate multiple ascending doses in both healthy volunteers and patients, at daily dose exposures higher than those evaluated in our previously completed Phase 2 clinical trials. The optimal dose and dosing regimen of fezagepras is expected to be further evaluated in Phase 2 clinical trials in selected fibrosis indications to be initiated in 2021.
Liminal BioSciences has also leveraged its experience in bioseparation technologies through its subsidiary Prometic Bioproduction Inc. to isolate and purify biopharmaceuticals from human plasma. Our lead plasma-derived product candidate is Ryplazim® for which the Company, through its US subsidiary, Prometic Biotherapeutics Inc., resubmitted a BLA on September 4, 2020 with the FDA seeking approval to treat patients with congenital plasminogen deficiency. The PDUFA target action date for this BLA filing is March 5, 2021. Ryplazim® has previously been granted Orphan Drug and Rare Pediatric Disease Designations by the FDA for the treatment of congenital plasminogen deficiency.
Prometic Plasma Resources, a subsidiary of Liminal BioSciences Inc., has joined the CoVIg-19 Plasma Alliance to contribute to the acceleration of the development of a potential new therapy for COVID-19. Our Canadian center located in Winnipeg, Manitoba is licensed by the FDA and Health Canada, and is certified by the European Union and the Plasma Protein Therapeutics Association (PPTA). Our American center located in Amherst, New York is licensed by the State of New York and its BLA submission is currently under review by the FDA.
Liminal BioSciences has active business operations in Canada, the United Kingdom and the United States.
Forward Looking Statement
This press release contains forward-looking statements about Liminal BioSciences’ objectives, strategies and businesses that involve risks and uncertainties. Forward‐looking information includes statements concerning, among other things, statements with respect to the timing for FDA review of the BLA for Ryplazim®, our plans for commercial launch of Ryplazim® in the United States if approved, our regulatory and commercial plans for Ryplazim® outside the United States, the potential of our product candidates and development of R&D programs and the timing of initiation of clinical trials.
These statements are "forward-looking" because they are based on our current expectations about the markets we operate in and on various estimates and assumptions. Actual events or results may differ materially from those anticipated in these forward-looking statements if known or unknown risks affect our business, or if our estimates or assumptions turn out to be inaccurate. At this stage, the product candidates of the Company have not been authorized for sale in any country. Among the factors that could cause actual results to differ materially from those described or projected herein include, but are not limited to, risks associated with FDA review, our ability to effectively establish a commercial organization, Liminal BioSciences’ ability to develop, manufacture, and successfully commercialize product candidates, if ever, the impact of the COVID-19 pandemic on its business operations, clinical development, regulatory activities and financial and other corporate impacts, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical trials, the ability of Liminal BioSciences to take advantage of business opportunities in the pharmaceutical industry, uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals and general changes in economic conditions. You will find a more detailed assessment of these risks, uncertainties and other risks that could cause actual events or results to materially differ from our current expectations in the filings the Company makes with the U.S. Securities and Exchange Commission and Canadian Securities Commissions filings and reports filings and reports, including in the Annual Report on Form 20-F, as amended, for the year ended December 31, 2019 and future filings and reports by the Company, from time to time. Such risks may be amplified by the COVID-19 pandemic and its potential impact on Liminal BioSciences’ business and the global economy. As a result, we cannot guarantee that any forward-looking statement will materialize. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements and estimates, which speak only as of the date hereof. We assume no obligation to update any forward-looking statement contained in this Press Release even if new information becomes available, as a result of future events or for any other reason, unless required by applicable securities laws and regulations.
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