Promedior Gets FDA Orphan Status For Myelofibrosis Drug
Clinical stage biotech firm Promedior announced that its drug PRM-151 has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) for the treatment of myelofibrosis.
The company’s lead candidate PRM-151 is a recombinant form of an endogenous human protein, Pentraxin-2 (PTX-2), which becomes active specifically at the site of tissue damage. The drug is an agonist that works as a monocyte/macrophage differentiation factor that prevents and may possibly reverse fibrosis. According to the company, PRM-151 has demonstrated broad anti-fibrotic activity in several preclinical models of fibrotic disease. The drug previously received orphan status in the U.S. and EU as treatment for idiopathic pulmonary fibrosis (IPF).
Dr. Suzanne L. Bruhn, President and CEO of Promedior, said that the new orphan drug designation propels the drug’s development forward, specifically for patients with myelofibrosis. “Based on the encouraging clinical results reported to date in myelofibrosis patients we believe that PRM-151’s novel mechanism of action is compelling with its potential to target and reverse the fundamental bone marrow fibrosis that underlies patients’ disease.”
Myelofibrosis is a serious and debilitating cancer characterized by fibrosis of the bone marrow. Symptoms include anemia, fatigue, and increased risk of infection and bleeding. Symptomatic myelofibrosis impacts around 18,000 patients with a median age of 61 to 66 every year in the U.S. The single potentially curative treatment for this disease is allogeneic bone marrow transplant, which, unfortunately, is applicable only to a few patients. Other available treatments only target the symptoms of myelofibrosis.
The company said results from a Phase II study of PRM-151 indicate the drug’s treatment improved hemoglobin and platelets levels, decreased bone marrow fibrosis, and reduced spleen size, among others. These results were presented by Promedior in June at the American Society for Clinical Oncology (ASCO) 2014 Annual Meeting.