Policy & Regulation
Prometic Life Sciences Inc to proceed with lead anti-fibrotic drug candidate PBI-4050 for Alström syndrome
6 December 2018 -

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

According to the company, 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.

The positive feedback from the recent meetings with regulatory authorities have provided the company with clear clinical and regulatory guidance, enabling the finalisation of the design of a pivotal placebo-controlled Phase 3 clinical trial, including agreement on multiple endpoints including liver and cardiac fibrosis.

Additionally, the company expects to start dosing AS subjects in the second quarter of 2019. The trial will enrol approximately 40 AS subjects in several countries to be randomised 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.

Under the company's Phase 3 clinical trial, 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.

In conjunction, the company has received the US FDA's Rare Pediatric Disease Designation as well as the US FDA's and the EMA's Orphan Drug Designation for PBI-4050 for the treatment of Alström syndrome, 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.