The Fast Track designation follows the FDA’s approval of an Investigational New Drug application for PBI-4050 and its sign-off on Prometic’s plan to conduct a pivotal Phase 2/3 clinical trial of the therapy.
A Fast Track designation will accelerate PBI-4050’s development and regulatory approval process. The designation is aimed at making medicines with considerable potential available to patients as soon as possible. To obtain the designation, a pharmaceutical company must show that a therapy is better or safer than available treatments.
Preclinical-trial studies have shown that PBI-4050 can slow scarring in the lungs, kidneys, heart, and liver.
“The treatment options for patients with fibrotic diseases like IPF are limited in scope and effectiveness,” Pierre Laurin, president and chief executive officer of Prometic, said in a press release.
“Fast Track designation will allow us to advance PBI-4050 and seek to potentially gain approval in a more efficient process,” he said. “PBI-4050 has the potential to provide patients with IPF a more effective treatment option. We look forward to demonstrating PBI-4050’s clinical efficacy and strong safety and tolerability profiles in a longer-term, placebo-controlled trial.”
A 20-week Phase 2 clinical trial (NCT02538536) showed that PBI-4050, both alone or combined with Ofev (nintedanib), slowed patients’ decline in lung function and stabilized their ability to breathe. Ofev is an approved IPF therapy developed by Boehringer Ingelheim.
The Phase 2 study, which involved 40 patients, also demonstrated that a daily dose of PBI-4050 was safe, and that patients could tolerate it well.
Researchers also tested a combination of PBI-4050 and another approved IPF therapy, Genentech’s Esbriet (pirfenidone). The combo led to a significant decline in lung function, probably because of unforeseen drug-drug interactions.
The pivotal Phase 2/3 clinical trial will have two stages. In the first stage, patients will receive a combination of PBI-4050 and Ofev or Ofev alone for 26 weeks. The combo will be tested in two doses, with some patients receiving the lower dose and others the higher one.
In the second stage of the trial, patients will be eligible to continue treatment at what a trial review board considers an optimal dose of PBI-4050.