FDA confirms Prometic's PBI-4050 IPF clinical trial design

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Apr 18, 2017, 07:00 ET

FDA concurs with add-on placebo controlled phase 2/3 clinical trial for PBI-4050 in combination with nintedanib
Prometic to also conduct placebo controlled phase 2/3 PBI-4050 Monotherapy clinical trial

LAVAL, QC, April 18, 2017 /PRNewswire/ - Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) ("Prometic" or the "Corporation") announced today that it has received concurrence from the US Food and Drug Administration ("FDA") on the design of the first of its PBI-4050's planned phase 2/3 clinical trials for IPF based on the efficacy data generated in the recently completed 40 patient Phase 2 open-label study.

In that phase 2 study, PBI-4050 was given for 12 weeks to patients who were receiving pirfenidone, nintedanib, or neither agent. The results of the study showed that the Forced Vital Capacity (FVC) remained stable in patients on PBI-4050 alone (n=9, FVC -12 ml) or in patients on PBI-4050 in combination with nintedanib (n=15, FVC +2 ml). In contrast, the results of said study showed that the FVC declined significantly in patients on 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 that received the PBI-4050 and pirfenidone combination, suggesting a drug-drug interaction.

Dr. John Moran, Chief Medical Officer of Prometic commented: "This early evidence of efficacy observed with PBI-4050 alone or in combination with nintedanib points toward very promising treatment options that will be further tested in two separate phase 2/3 clinical trials. We are very pleased that the FDA concurs with our decision to exclude a combined pirfenidone and PBI-4050 treatment arm in the upcoming phase 2/3 add-on study".

The design for the add-on placebo-controlled phase 2/3 pivotal trial will therefore be an amended version of the protocol the FDA originally requested during the pre-IND meeting: IPF patients currently treated with nintedanib would be randomized to receive in addition either PBI-4050 or a placebo.

Prometic also intends to initiate a second phase 2/3 placebo-controlled trial which would enroll IPF patients who have failed to tolerate nintedanib or pirfenidone and would be randomized to receive either PBI-4050 or placebo, ("PBI-4050 Monotherapy").

"To date, PBI-4050 has presented a remarkable safety and tolerability profile throughout all clinical trials", said Mr. Pierre Laurin, President and Chief Executive Officer of Prometic. "We are also very pleased that our phase 2 open label study in IPF patients has served the very important strategic purpose of allowing us to optimize the design of our pivotal clinical program for this devastating medical condition".

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 die each year with IPF, a similar number of deaths to those due to breast cancer. The 5-year mortality rate for subjects 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 our lead drug candidates' anti-fibrotic activity in several key organs including the kidneys, the heart, the lungs and the liver. Twenty six million subjects in the U.S. alone are believed to suffer from chronic kidney diseases ("CKD"). Subjects with severe CKD stages (3 and 4) suffer from a progressive loss of their renal function leading to end-stage renal disease and the need for dialysis or kidney transplant. Cardiovascular complications are the most common cause of death in dialysis subjects.

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 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. Prometic is also active in developing its own novel small-molecule therapeutic products targeting unmet medical needs in the field of fibrosis, anemia, neutropenia, cancer and autoimmune diseases/inflammation as well as certain nephropathies. 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, Russia, Asia and Australia.

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.