RARE Daily

FDA Grants Promedior Breakthrough Therapy Designation for Idiopathic Pulmonary Fibrosis Drug

March 26, 2019

Rare Daily Staff

Photo: Jason Lettmann

The U.S. Food and Drug Administration granted Promedior Breakthrough Therapy designation for its experimental therapy PRM-151, an anti-fibrotic immunomodulator for idiopathic pulmonary fibrosis.

Idiopathic pulmonary fibrosis (IPF) is a serious, life-limiting lung disease characterized by fibrosis and scarring of lung tissue with a median survival of 3 to 5 years after diagnosis. Replacement of normal lung tissue by fibrosis results in restriction in the ability to fill the lungs with air and decreased transfer of oxygen from inhaled air into the bloodstream resulting in lower oxygen delivery to the brain and other organs. Patients with IPF most often suffer from progressive shortness of breath, particularly with exertion; chronic cough; fatigue and weakness; and chest discomfort.

Currently approved drugs slow down but do not halt disease progression and the only curative therapy is lung transplant, an option available for a small group of patients. While estimates vary, it is believed that IPF could affect approximately 130,000 patients in the United States and approximately 76,000 patients in Europe.

PRM-151, Promedior’s lead product candidate, is a recombinant form of the endogenous human innate immunity protein pentraxin-2, which is specifically active at the site of tissue damage. PRM-151 is an agonist that potentially reverses fibrosis. PRM-151 has shown broad anti-fibrotic activity in multiple preclinical models of fibrotic disease, including pulmonary fibrosis, myelofibrosis, acute and chronic nephropathy, liver fibrosis, and age-related macular degeneration.

In addition to the randomized phase 2 study in IPF, phase 1a and 1b clinical studies in healthy subjects and IPF patients have demonstrated that PRM-151 was well-tolerated. Additionally, the phase 1b study in patients with IPF showed encouraging results in exploratory efficacy endpoints.  

The company recently announced that it had reached an agreement with the FDA on the design of a phase 3 registrational study for PRM-151 in IPF using forced vital capacity as a primary endpoint and six-minute walk distance as a key secondary endpoint.

Breakthrough Therapy designation is intended to expedite the development and regulatory review of drugs intended to address a significant unmet need in serious or life-threatening conditions. To qualify for the designation, preliminary clinical evidence must demonstrate that the candidate offers the potential for substantial improvement over existing therapies.  The designation offers a sponsor more intensive guidance from the FDA, access to a scientific liaison to help accelerate review time, and eligibility for Accelerated Approval and Priority Review designations.  

“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,” said Jason Lettmann, CEO of Promedior. “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.”

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