Rare Disease Report

Ongoing Study of PBI-4050 in Alstrom Syndrome

SEPTEMBER 19, 2017
Mathew Shanley
Prometic Life Sciences has released data from an ongoing Phase 2 open-label study, highlighting the safety and efficacy of PBI-4050 in patients with Alström Syndrome.

Alström Syndrome is a rare genetic autosomal recessive disorder characterized by dysfunction in several organs. It is the result of a mutation in the ALMS1 gene, and symptoms often begin in childhood and commonly include a progressive loss of vision and hearing, obesity and heart disease. The only current treatment options include management of the symptoms.

Following 24 weeks of treatment with PBI-450, data for 9 is now available showing the drug can reduce kidney damage.

Table 1

Additionally, the company has noted that biomarkers for liver function has also been observed in all 8 patients that data is available.

Based in the positive efficacy responses, the Data Safety Monitoring Board (DSMB) and Medicines and Healthcare Products Regulatory Agency (MHRA) have twice extended the duration of treatment – first from the original 24 weeks to 60 weeks, and then again for an additional 12 weeks (72 total weeks).

The most recent extension was a way to ensure that subjects could remain on treatment while the regulatory authorities review a rollover protocol.

"We believe we now have enough convincing clinical data to trigger meetings with both the European and the US regulatory authorities to determine the clinical-regulatory pathway for these patients, who are in dire need,” Pierre Laurin, Prometic's President and Chief Executive Officer, stated in a press release. “We intend to hold such meetings in the fall of this year to determine whether this ultra-rare pediatric unmet medical need could formally become an indication granted priority review".

The positive results from the study support Prometic’s requests for meeting with both the U.S. Food and Drug Administration (FDA) and European Medicines Association (EMA) to define the clinical-regulatory pathway for approval of PBI-4050 for the treatment of Alstrom Syndrome.

Clinical trials are also ongoing for the treatment of idiopathic pulmonary fibrosis (IPF), chronic kidney disease (CKD), and metabolic syndrome and associated type 2 diabetes with PBI-4050.

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