- Clinical trial design confirmed during FDA Type C meeting held on January 3, 2018
- Simplified patient entry criteria to include IPF patients whether on background standard of care (i.e. nintedanib) or not
- Primary endpoint analysis to be stratified on background therapy (+/- nintedanib)
- Planned Interim analysis at 26 weeks
- Company to host IPF KOL luncheon presentation and live webcast for the investment community on January 31 at 12 noon EST
LAVAL, QUEBEC, CANADA, – January 29, 2018 – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“Prometic”) announces today the outcome of a successful clinical development Type C meeting with the US Food and Drug Administration (FDA) for its orally active anti-fibrotic lead drug candidate, PBI-4050. The purpose of the meeting was to reach final agreement on the design of the Phase 3 pivotal clinical trial for PBI-4050 in patients with idiopathic pulmonary fibrosis (IPF).
Based on recommendations from the FDA, Prometic now will undertake an “all comers study”. The enrollment criteria will be greatly simplified such that the study will enroll patients with mild-to-moderate IPF, regardless of whether they are on background standard of care with nintedanib (OFEV®) or not. Therefore, the study will provide efficacy data on both PBI-4050 as a stand-alone agent, and as an add-on to nintedanib, and will be part of the dataset to support a simple, all-inclusive indication “for the treatment of IPF”. Patients will be randomized to receive placebo, or one of two doses of PBI-4050 (800 mg or 1,200 mg) for a total of 52 weeks. An interim analysis will be conducted at 26 weeks. The primary endpoint is the annual rate of decline in forced vital capacity (FVC), the total amount of air exhaled during a forced breath, expressed in mL and measured over 52 weeks (mL/year). Patients taking pirfenidone will be excluded because of a known drug-drug interaction between pirfenidone and PBI-4050.
“We are very pleased by the outcome of the meeting with the FDA. The recommendations provided by the FDA will allow us to conduct a clinical trial that is much more reflective of current treatment of IPF patients,” commented Dr. Joseph Parker, Senior Director, Prometic Clinical Affairs, who is overseeing the study. “This is going to be a multinational study, involving an experienced CRO in the field to help manage multiple sites across the United States, Canada, Australia, the UK and Europe. Our goal is to start patient enrollment mid-year.”
Pierre Laurin, President and Chief Executive Officer commented, “Prometic is now exceptionally well positioned to address the significant unmet medical needs in IPF. We have two late stage clinical candidates tackling different aspects of this devastating disease which affects more than 130,000 patients in the US alone. In addition to PBI-4050, we are pleased that Ryplazim™ was recently granted Orphan Drug Designation for IPF by the FDA. We are developing RyplazimTM to help manage the acute exacerbation episodes during which IPF patients accumulate hyaline membrane in the lungs, contributing to further significant loss of lung function.”
Key Opinion Leader (KOL) Luncheon:
Prometic will be hosting a KOL luncheon event entitled, Novel Treatments for Idiopathic Pulmonary Fibrosis (IPF), intended for institutional investors, sell-side analysts, investment bankers, and business development professionals only. The event will feature presentations by Martin Kolb, MD, PhD (McMaster University), and Gerard Criner, MD (Temple University), who will discuss the current treatment landscape, as well as the unmet medical need for treating patients with IPF. The event is scheduled to take place on Wednesday, Jan. 31, 2018, 12:00-1:30 pm EST in New York City.
This event is intended for institutional investors, sell-side analysts, investment bankers, and business development professionals only. Please RSVP in advance if you plan to attend, as space is limited. For those who are unable to attend in person, a live webcast and replay will be accessible here (http://lifesci.rampard.com/20180131/reg.jsp).
More about Idiopathic Pulmonary Fibrosis (IPF) and Acute Exacerbation
Idiopathic pulmonary fibrosis (IPF) is a chronic, devastating, and ultimately fatal disease characterized by a progressive decline in lung function. It is a specific type of interstitial lung disease in which the small air sacs of the lung, the “alveoli,” gradually become replaced by fibrotic (scar) tissue and is the cause of worsening dyspnea (shortness of breath). IPF is usually associated with a poor prognosis. The term “idiopathic” is used because the cause of pulmonary fibrosis is still unknown. IPF usually occurs in adult individuals of between 50 and 70 years of age, particularly those with a history of cigarette smoking, and affects men more often than women. IPF affects approximately 130,000 people in the United States, with about 48,000 new cases diagnosed annually. Nearly 40,000 people with IPF die each year, a mortality rate similar to breast cancer. The 5-year mortality rate for patients with IPF is estimated to range from 50% to 70%. Acute exacerbation of IPF (AE-IPF) is defined as a sudden acceleration of the disease or an idiopathic acute injury superimposed on diseased lung tissue that leads to a significant decline in lung function. An AE-IPF is associated with a mortality rate as high as 85%, with mean survival periods between 3 to 13 days.
More About PBI-4050
PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles in a large number of animal models of fibrosis affecting different organs, including the lung, liver, heart, kidney, and pancreas. Fibrosis is a very complex process by which continuing inflammation leads to progressive loss of organ function, as normal tissue is replaced by fibrotic scar tissue. The effects of PBI-4050 demonstrated in animal models have been replicated in Phase 2 studies in IPF, in Type 2 diabetes with metabolic syndrome, and in Alström syndrome. Placebo-controlled studies are in progress in Type 2 diabetes, and are planned in IPF and in chronic kidney disease associated with Type 2 diabetes.
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 therefore is vital in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis.
About Prometic Life Sciences Inc.
Prometic Life Sciences Inc. (www.prometic.com) is a long-established biopharmaceutical company with globally recognized expertise in bioseparations, plasma-derived therapeutics and small-molecule drug development. Prometic is active in developing its own novel small molecule therapeutic products targeting unmet medical needs in the field of fibrosis, cancer and autoimmune diseases/inflammation. A number of plasma-derived and small molecule products are under development for orphan drug indications. Prometic also offers its state-of-the-art technologies for large-scale purification of biologics, drug development, proteomics and the elimination of pathogens to a growing base of industry leaders and uses its own affinity technology that provides for highly efficient extraction and purification of therapeutic proteins from human plasma in order to develop best-in-class therapeutics and orphan drugs. Headquartered in Laval (Canada), Prometic has R&D facilities in the UK, the U.S. and Canada, manufacturing facilities in the UK and commercial activities in the U.S., Canada, Europe and Asia.
Forward Looking Statements
This press release contains forward-looking statements about Prometic’s objectives, strategies and businesses that involve risks and uncertainties. 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. Such risks and assumptions include, but are not limited to, Prometic’s ability to develop, manufacture, and successfully commercialize value-added pharmaceutical products, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical studies, the ability of Prometic to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process and general changes in economic conditions. You will find a more detailed assessment of the risks that could cause actual events or results to materially differ from our current expectations in Prometic’s Annual Information Form for the year ended December 31, 2016, under the heading “Risk and Uncertainties related to Prometic’s business”. As a result, we cannot guarantee that any forward-looking statement will materialize. We assume no obligation to update any forward-looking statement 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. All amounts are in Canadian dollars unless indicated otherwise.