LAVAL, CANADA, and CAMBRIDGE, ENGLAND – November 5, 2020 – Liminal BioSciences Inc. (Nasdaq: LMNL) (“Liminal BioSciences” or the “Company”), a clinical-stage biopharmaceutical company, today announced the acceptance of two abstracts related to the Company’s clinical development activities of Ryplazim® (plasminogen) for the treatment of Congenital Plasminogen Deficiency (“C-PLGD”) at the 62nd American Society of Hematology Annual Meeting and Exposition scheduled to take place in a virtual format on 5th-8th Dec 2020.
Both poster presentations will also be published in the November supplemental issue of Blood along with a Comprehensive Case Review entitled ‘Painting the Clinical Picture Of Congenital Plasminogen Deficiency (C-PLGD) through A Comprehensive Case Study Review’.
“These abstracts further highlight the potential for Ryplazim® and the impact it could have on patients, if approved, having submitted our Biologic License Application ("BLA") for Ryplazim® in September for the treatment of clinical signs and symptoms associated with C-PLGD in pediatric and adult patients,” stated Kenneth Galbraith, Chief Executive Officer
Abstracts selected for poster presentations are summarized below and are available on the conference website at https://ash.confex.com/ash/2020/webprogram/start.html.
Successful Pregnancy in A Patient with Infertility Due to Congenital Plasminogen Deficiency Treated with Intravenous Plasminogen (Human) Replacement Therapy
Abstract Number: 2703
Session: 322 Disorders of Coagulation: Poster III
Date & Time: Monday 7 December 2020
Acute Airway Obstruction in 4 Pediatric Patients with Congenital Plasminogen Deficiency (C-PLGD) Treated with Intravenous Plasminogen (Human) Replacement Therapy Under an Expanded Access Protocol
Abstract Number: 1799
Session: 322 Disorders of Coagulation: Poster II
Date & Time: Sunday 6 December 2020
The Virtual Poster Hall will be open for conference attendees to browse a different set of posters each day. The Poster Hall hours are as follows:
Saturday, December 5: 7:00 a.m. - 3:30 p.m. (Pacific Time)
Sunday, December 6: 7:00 a.m. - 3:30 p.m. (Pacific Time)
Monday, December 7: 7:00 a.m. - 3:00 p.m. (Pacific Time)
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 congenital 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, 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. Liminal BioSciences’ lead small molecule product candidate, fezagepras (PBI-4050), is expected to enter a Phase 1 clinical trial in Q4-2020 in the UK to evaluate multiple ascending doses in healthy volunteers, at daily dose exposures higher than those evaluated in Liminal BioSciences’ previously completed Phase 2 clinical trials. Fezagepras is expected to be further evaluated in a global Phase 2b clinical trial in patients with idiopathic pulmonary fibrosis (IPF) to be initiated in H2-2021. In addition, Liminal BioSciences expects to initiate a Phase 1b/2a clinical trial in the United States of fezagepras for patients with high triglyceride levels (hypertriglyceridemia) in H2-2021.
Fezagepras has previously been granted Orphan Drug Designation by the FDA and the European Medical Agency for the treatment of IPF. The treatment has also received a Promising Innovative Medicine (PIM) designation by the Medicines and Healthcare products Regulatory Agency (MHRA) for IPF.
Liminal BioSciences has also leveraged its experience in bioseparation technologies through its subsidiary Prometic Bioproduction Inc. to isolate and purify biopharmaceuticals from human plasma. Liminal BioSciences’ lead plasma-derived product candidate is Ryplazim® for which the Company, through its US subsidiary, Prometic Biotherapeutics Inc., resubmitted a BLA in September 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. Liminal BioSciences’ 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). Liminal BioSciences’ 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. 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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