logo
  

Fulcrum : Phase 2b Trial On Facioscapulohumeral Muscular Dystrophy Fails To Meet Main Goal

Fulcrum Therapeutics Inc. (FULC) said Thursday that its phase 2b trial for people with facioscapulohumeral muscular dystrophy or FSHD did not meet its primary endpoint.

Changes in DUX4-driven gene expression, which were included in the trial as an experimental biomarker endpoint, could not be demonstrated, the company believes due to several technical and biologic variables with the endpoint.

The company plans to meet with health authorities, including the U.S. Food and Drug Administration (FDA), in the second half of 2021 to determine the regulatory path for losmapimod in FSHD.

The trial results with losmapimod showed clinically relevant and statistically significant benefits versus placebo on multiple measures of structural and functional FSHD disease progression and patient reported outcomes at 48 weeks.

Losmapimod was generally well-tolerated, with no drug-related serious adverse events reported.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
Japanese entrepreneur Yusaku Maezawa has selected ten crewmembers, comprising artists, content creators, and athletes, to fly with him on SpaceX's Starship around the Moon on the dearMoon mission. SpaceX's first private lunar flight with dearMoon crew, including two backups, is planned to take place in 2023. Maezawa announced the crew for the mission in a YouTube video and a tweet. SpaceX, owned by Elon Musk, has launched 40 satellites of British communications network OneWeb into orbit. From NASA's Kennedy Space Center or KSC in Florida, Falcon 9 rocket on Thursday evening carried the satellites. The Falcon 9's first stage separated, and it then returned to Earth to make a landing on a SpaceX pad at the nearby Cape Canaveral Space Force Station. Google, owned by Alphabet Inc., plans to merge its teams working on Maps products and the mapping service Waze with effect from December 9, reports said. The decision to consolidate processes comes as the search giant is facing pressure to streamline operations and cut costs. The restructuring is expected to reduce overlapping work across the Waze and Maps products.
Follow RTT