A pivotal Phase 3 trial evaluating PBI-4050, a potential treatment of idiopathic pulmonary fibrosis, will be open to all patients with mild-to-moderate IPF regardless of whether they are also taking Ofev (nintedanib) or not, the therapy’s developer, Prometic Life Sciences, announced.
The trial, in other words, will evaluate the efficacy and safety of PBI-4050 as a standalone therapy and as an add-on treatment to Ofev. IPF patients using Esbriet (pirfenidone), however, will be excluded from enrolling, as previous studies have reported a drug-drug interaction between Esbriet and PBI-4050.
Design of this Phase 3 trial came through an agreement between Prometic and the U.S. Food and Drug Administration (FDA), the company announced in its press release.
“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,” said Joseph Parker, senior director, Prometic clinical affairs, who is overseeing the study.
“This is going to be a multinational study, involving an experienced CRO [contract research organization] in the field to help manage multiple sites across the United States, Canada, Australia, the U.K. and Europe,” Parker said.
Prometic is hoping to begin enrolling eligible patients by mid-2018.
Participants will be randomized to receive orally 800 mg or 1200 mg of PBI-4050 or placebo for up to 52 weeks. The trial will evaluate its potential to slow lung function decline, determined as the annual rate of decline in forced vital capacity (FVC; the total amount of air exhaled during a forced breath). An interim analysis will be conducted after 26 weeks of treatment.
A Phase 2 study (NCT02538536) in 40 IPF patients reported a stabilization in lung function among those given PBI-4050 as a stand-alone therapy or in combination with Ofev after 12 weeks of treatment, while a significant further decline was seen in those taking PBI-4050 plus Esbriet.
Prometic is also developing Ryplazim (plasminogen) to manage acute exacerbation episodes in the lungs of IPF patients.
PBI-4050 has been designated an orphan drug, a status that works to advance its development, as a potential IPF treatment by both the FDA and the European Medicines Agency. Ryplazim received orphan drug designation for IPF by the FDA in December 2017.