PBI-4050 is an oral compound designed to prevent inflammation and tissue scarring in several organs, including the lungs, kidneys, liver, heart and pancreas. Prometic is developing it not only as a potential treatment for IPF, but also for Alström syndrome, metabolic syndrome and chronic kidney disease associated with type 2 diabetes. A pivotal trial is one intended to provide evidence for a drug’s regulatory approval.
A 20-week Phase 2 clinical trial (NCT02538536) showed that PBI-4050, alone or in combination with Ofev (nintedanib; Boehringer Ingelheim), slowed IPF patients’ lung function decline and stabilized their breathing capacity.
The Phase 3 trial will cover people with mild to moderate IPF, including some on Ofev and some who are not. Prometic will not enroll patients on Genentech’s Esbriet (pirfenidone) because a drug-drug interaction between Esbriet and PBI-4050 was reported in previous trials.
Using both patients who are on Ofev and patients who are not in the trial will show whether PBI-4050 is effective alone and in combination with Ofev.
Two groups of patients in the year-long trial will receive one of two doses of PBI-4050 — either 800 mg or 1,200 mg. A third group will receive a placebo.
The trial’s primary objective will be to assess PBI-4050’s ability alone, or in combination with Ofev, to slow patients’ annual rate of decline in a measure of lung function known as forced vital capacity. That is the amount of air a person can force from their lungs after taking the deepest breath possible.
Researchers will do an interim analysis of results after six months.
Prometic expects to begin recruiting patients for the trial in the middle of 2018. They will come from the United States, Canada, Australia, Britain and other places in Europe.
“The recommendations provided by the FDA will allow us to conduct a clinical trial that is much more reflective of current treatment of IPF patients,” Joseph Parker, Prometic’s senior director of clinical affairs, who will supervise the trial, said in a press release.
“Prometic is now exceptionally well positioned to address the significant unmet medical needs in IPF. We have two late-stage clinical candidates tackling different aspects of this devastating disease which affects more than 130,000 patients in the US alone,” added Pierre Laurin, Prometic’s president and chief executive officer.
Prometic is also developing a therapy called Ryplazim to manage acute IPF flare-ups.