- Clinical-regulatory pathway defined following recent meetings with regulatory authorities
- Pivotal Phase 3 clinical trial design finalized to support regulatory submissions in the U.S. and Europe
- Provides potential for faster route to regulatory approval with Alström syndrome
- Opens an efficient route to market for PBI-4050 and potential to monetize Prometic's second PRV
LAVAL, QC /PRNewswire/ - Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) ("Prometic" or the "Corporation") today confirmed its decision to formally pursue Alström syndrome (AS) as a clinical indication for PBI-4050 following positive feedback from its recent meetings with regulatory authorities. These meetings have provided Prometic with clear clinical and regulatory guidance, enabling the finalization of the design of a pivotal placebo-controlled Phase 3 clinical trial, including agreement on multiple endpoints including liver and cardiac fibrosis.
"We are very excited that Alström syndrome could be the first approved clinical indication for our lead anti-fibrotic drug candidate, PBI-4050," commented John Moran, M.D., Prometic's Chief Medical Officer. "Our ongoing U.K. Phase 2 trial continues to generate encouraging safety and clinical activity data with patients averaging more than one year of drug exposure. We are confident in PBI-4050's ability to reproduce similar positive clinical activity on specific key fibrotic clinical endpoints in the pivotal placebo-controlled Phase 3 study."
The protocol for the Phase 3 clinical trial will be designed to meet expectations from both the U.S and EU regulatory agencies. The Corporation expects to start dosing AS subjects in the second quarter of 2019. The trial will enroll approximately 40 AS subjects in several countries to be randomized into 3 study arms, one receiving placebo and two receiving PBI-4050 (800 mg and 1200 mg) for 36 weeks. The placebo subjects would then be crossed over to one of the PBI-4050 arms for an additional 16 weeks of treatment. The endpoints will include liver steatosis measured by magnetic resonance imaging Fat Fraction assessment (MRI-PDFF), liver fibrosis measured by magnetic resonance elastography (MRE) and cardiac fibrosis measured by MRI.
Commenting on the cost of the trial, Pierre Laurin, Prometic's President and Chief Executive Officer noted, "This will be a relatively small and quick trial, which can be undertaken within our financial means, and more importantly within the guidance on R&D spend given during our Q3 earnings call". He added, "Alström Syndrome remains a very efficient route for us to potentially bring PBI-4050 quickly to approval, opening up both interesting commercial opportunities and the potential to monetize another PRV."
The FDA has granted a Rare Pediatric Disease Designation to PBI-4050 for the treatment of AS, thus making it potentially eligible to receive a priority review voucher (PRV) upon regulatory approval. In addition to the Rare Pediatric Disease Designation, PBI-4050 has also been granted Orphan Drug Designation by both the FDA and the EMA for the treatment of AS and of idiopathic pulmonary fibrosis (IPF) as well as PIM (Promising Innovative Medicine) designation by the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of IPF and AS.
More about Alström syndrome:
Alström syndrome is a rare inherited autosomal recessive syndrome characterized by the onset of obesity in childhood or adolescence, Type 2 diabetes, often with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis involving the liver, kidney and heart. Alström syndrome is also characterized by a progressive loss of vision and hearing, a form of heart disease that weakens the heart muscle (dilated cardiomyopathy), and short stature. This disorder can also cause serious or life-threatening medical problems involving the liver, kidneys, bladder, and lungs. The clinical manifestations of Alström syndrome vary in severity, and not all affected individuals have all of the features associated with the disorder.
More about PBI-4050
PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles demonstrated in a large number of animal models of fibrosis affecting different organs, including the lung, liver, heart, kidney, and pancreas. The effects of PBI-4050 demonstrated in animal models have been replicated in Phase 2 studies in idiopathic pulmonary fibrosis (IPF), in metabolic syndrome with type 2 diabetes and in Alström syndrome. PBI-4050's IND has already been cleared by the FDA for IPF pivotal placebo-controlled Phase 3 clinical trial.
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, 2017 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.