CAMBRIDGE, Mass., Jan. 29, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum also announced the issuance of U.S. patent 10,537,560 with claims covering the use of other p38 kinase inhibitors for the treatment of FSHD.

“We are pleased to have been granted Orphan Drug Designation for losmapimod in FSHD as it underscores the critical need for treating this rare muscular dystrophy that has no approved therapies,” said Robert J. Gould, Ph.D., Fulcrum’s president and chief executive officer. “We believe losmapimod represents a promising, novel approach to treat the known root cause of FSHD and remain on-track to announce data from the Phase 2b ReDUX4 clinical trial in the third quarter of 2020. Our recently issued patent also expands our intellectual property protection relating to the use of other clinical-stage p38 inhibitors for the treatment of FSHD, strengthening our position as a leader in the treatment of genetically defined diseases.”

In October 2019, the Company announced preliminary results from a Phase 1 clinical trial of losmapimod in patients with FSHD, which indicated that losmapimod was generally well tolerated and achieved dose-dependent concentrations in plasma and muscle believed to be adequate for efficacy based on preclinical pharmacology studies. Additionally, losmapimod has shown adequate safety and tolerability in over 3,500 patients and healthy volunteers across multiple indications, with no safety signals attributed to the drug in those trials.

The patent announced today is in addition to U.S. patent 10,342,786, which covers the method of using losmapimod for the treatment of FSHD. These two patents each provide protection through 2038.

About Orphan Drug Designation
The FDA Office of Orphan Products Development grants orphan status to support development of medicines that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval if received, exemption of FDA application fees and tax credits for qualified clinical trials. For more information about orphan designation, please visit the FDA website at www.fda.gov.

About Losmapimod

Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed by Fulcrum Therapeutics following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD. (Article from : www.drugs.com)