The investigational anti-fibrotic immunomodulator is being investigated for respiratory, oncology, hepatology, and nephrology indications.
The US Food and Drug Administration (FDA) granted Breakthrough Therapy designation for PRM-151, an investigational anti-fibrotic immunomodulator being investigated by Promedior, Inc. for idiopathic pulmonary fibrosis (IPF).
The Breakthrough Therapy designation (BTD) is intended to support the development and review of treatments being developed for significant unmet needs in serious or life-threatening conditions.
"BTD underscores the disease-modifying potential of PRM-151 in IPF, a serious, life-limiting lung disease for which despite existing therapies, patient prognosis remains poor with a median survival of 3-5 years," said Jason Lettmann, Chief Executive Officer of Promedior. "Our pivotal program in IPF is designed to show outcomes that could provide meaningful benefit to patients and differentiation in the market over the standard of care.”
The FDA’s decision to grant this designation was supported by data from a phase 2, randomized, double-blind clinical trial evaluating the safety and efficacy of PRM-151. The trial included 117 patients, aged 40 to 80 years, with idiopathic pulmonary fibrosis who were randomized to either PRM-151 10 mg/kg or placebo. The study drug or placebo was given via intravenous infusion over 60 minutes on days 1, 3, 5, and then once every 4 weeks. Many patients (79%) also received standard of care treatment with pirfenidone or nintedanib.
At week 28, efficacy was evaluated through the primary endpoint of change from baseline in forced vital capacity. Patients in the active treatment arm demonstrated a change in forced vital capacity percentage of predicted value of −2.5% compared with −4.8% with placebo (P = .001).
A secondary efficacy endpoint was the change from baseline in 6-minute walk distance, which was -0.5 meters for participants receiving PRM-151 compared to -31.8 meters for those receiving placebo (P <.001).
“Outside of IPF, PRM-151 can address additional fibrotic diseases, and the team remains committed to evaluating strategic opportunities for advancing this promising candidate in other respiratory, oncology, hepatology, and nephrology indications," added Lettmann.