Biopharmaceutical company Prometic Life Sciences Inc (TSX:PLI) (OTCQX:PFSCF) said on Monday that Alström syndrome (AS) could be the first approved clinical indication for its lead anti-fibrotic drug candidate, PBI-4050.

Alström syndrome is a rare inherited autosomal recessive syndrome characterised 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.

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, stated the company.

Following the positive feedback and clear clinical and regulatory guidance from its meetings with regulatory authorities, the company has agreed to the finalisation of the design of a pivotal placebo-controlled Phase 3 clinical trial, including agreement on multiple endpoints including liver and cardiac fibrosis.

The company added the protocol for the Phase 3 clinical trial will be designed to meet expectations from both the US and EU regulatory agencies.

In the the second quarter 2019, the company said it expects to enroll approximately 40 AS subjects in several countries to be randomised into three study arms, one receiving placebo and two receiving PBI-4050 (800 mg and 1200 mg) for 36 weeks. 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.