Prometic receives FDA clearance of its IND to initiate pivotal PBI-4050 Phase 2/3 trial in patients with idiopathic pulmonary fibrosis

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Sep 25, 2017, 06:30 ET

Prometic moving immediately to formalize enrollment of specialist clinical sites across the United States
U.S. IND to be followed by clinical trial applications in Canada, Europe, Australia and Japan throughout Q4 2017
Prometic plans to supplement the IND with a protocol for a study of PBI-4050 monotherapy in IPF patients in October 2017

LAVAL, QC, Sept. 25, 2017 /PRNewswire/ - Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic) today announced that its oral anti-fibrotic lead drug candidate, PBI-4050, has received U.S. Food and Drug Administration Investigational New Drug (IND) approval to commence its pivotal Phase 2/3 clinical trial in patients suffering from idiopathic pulmonary fibrosis (IPF).

The pivotal Phase 2/3 clinical trial is a two-stage adaptive, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of PBI-4050 when combined with nintedanib (OFEV™, Boehringer Ingelheim) in subjects with IPF. The number of subjects required to be enrolled has been based on the results seen in the recently-completed open label study of PBI-4050 in IPF. The Phase 2 stage will enroll 375 subjects with IPF, who will be randomly assigned to one of three groups: 1) 125 subjects who will receive placebo + nintedanib, 2) 125 subjects who will receive PBI-4050 800 mg + nintedanib or 3) 125 subjects with PBI-4050 1200 mg + nintedanib. An independent Data and Safety Monitoring Board (DSMB) will conduct an interim 26-week analysis, and based on the safety and efficacy results, will recommend whether the study should continue into Phase 3 stage and which dose of PBI-4050 should be continued. This Phase 3 stage would randomize an additional up to 450 subjects to receive nintedanib plus either placebo or the chosen PBI-4050 dose.

Prometic has already completed an open label Phase 2 study in patients who received PBI-4050 for 12 weeks in addition to either pirfenidone, nintedanib, or placebo. The study showed the Forced Vital Capacity (FVC) remained stable in patients on PBI-4050 alone (n=9, FVC -12 ml) and in patients on PBI-4050 in combination with nintedanib (n=15, FVC +2 ml). In contrast, the FVC declined significantly in patients receiving PBI-4050 in combination with pirfenidone (n=16, FVC -105 ml). PBI-4050's plasma concentration was sub-therapeutic at 50% of the expected level in patients receiving PBI-4050 in combination with pirfenidone, suggesting a drug-drug interaction.

"We are very encouraged by the results of our open-label trial of PBI-4050 in combination with nintedanib, and are pleased to advance the clinical development plan with this pivotal study", said Dr. John Moran, Prometic's Chief Medical Officer. "IPF is a very serious condition and we believe that patients may benefit from this novel therapeutic approach. We have multiple key opinion leaders who have expressed a wish to participate in the study, and now that the IND has been cleared we can begin a formal study startup".

Pierre Laurin, Prometic's President and Chief Executive Officer, stated, "We are very excited to be entering the pivotal stages of our IPF clinical program, which is another important milestone towards bringing this therapy to patients who suffer from IPF. We believe that PBI-4050 is an important advancement in this indication and represents a potential significant step forward for helping patients".

More about Idiopathic Pulmonary Fibrosis ("IPF")

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 about 130,000 people in the United States, with about 48,000 new cases diagnosed annually. Approximately 40,000 people with IPF die each year, a similar number of deaths to those due to breast cancer. The 5-year mortality rate for patients with IPF is estimated to range from 50% to 70%.

More about PBI-4050

PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles confirmed in several in vivo experiments targeting fibrosis. Fibrosis is a very complex process by which continuing inflammation causes vital organs to lose their function as normal tissue is replaced by fibrotic scar tissue. The proof-of-concept data generated to date confirms the anti-fibrotic activity of PBI-4050 in several key organs including the kidneys, the heart, the lungs and the liver. It is also effective in improving glucose control in animal models of diabetes. Open label Phase 2 studies in idiopathic pulmonary fibrosis and in Type 2 diabetes have provided preliminary evidence that the results seen in preclinical models translate into the corresponding human diseases.

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.

SOURCE ProMetic Life Sciences Inc.