Fulcrum Therapeutics Presents Data Highlighting Reachable Workspace as Functional Endpoint in FSHD at MDA Conference

Fulcrum Therapeutics Inc, a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announces data demonstrating that Reachable Workspace (RWS) is a quantitative and relevant measurement of function that can be used to assess disease progression as well as response to treatment in people with facioscapulohumeral muscular dystrophy (FSHD).

Data from two Phase 2 clinical trials also suggest that losmapimod preserved or improved function in people with FSHD, as measured by RWS. These data are being presented this week at the 2022 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.

“FSHD typically moves from the upper body down, with almost universal involvement of the shoulder girdle, leading to profound decreases in the ability to perform activities of daily living,” says Judith Dunn, PhD, Fulcrum’s president of research and development. “RWS is a quantitative measurement of function that assesses shoulder and arm mobility that has been used in clinical research in other muscular dystrophies and diseases affecting the upper extremities. Growing evidence now supports its use in FSHD as a relevant and reliable endpoint that has been shown to correlate with how people function and feel, making it an ideal primary endpoint for our Phase 3 REACH trial.”

“There is a need for objective, sensitive, and clinically meaningful outcome measures in neuromuscular diseases, including FSHD, to support clinical development of new therapies and to monitor disease severity and progression,” says Jay J. Han, MD, professor and vice chair of Physical Medicine & Rehabilitation at University of California, Irvine, and one of the developers of RWS. “RWS uses 3D motion sensor technology to reliably and reproducibly measure upper extremity range of motion and has been shown across numerous studies to quantify and track when people are having difficulties performing activities of daily living and maintaining independence.”

Reachable Workspace Evaluates Shoulder and Arm Mobility

People with FSHD consistently rank difficulty with use of their shoulders and proximal arms as the most prevalent and severe impairment impacting their quality of life. RWS evaluates shoulder and proximal arm mobility by utilizing 3D motion sensor technology to track upper limb trajectory across five regions. Fulcrum compared the effect of losmapimod to placebo using RWS in its ReDUX4 Phase 2b study and in an open-label Phase 2 study. In both studies, losmapimod was shown to preserve function and, in some instances, improve function, as measured by RWS.

“The progressive loss of range of motion in my shoulders and arms has had a tremendous impact on my life,” says Carden Wyckoff, a member of Fulcrum’s FSHD patient advisory board and the FSHD Society board of directors. “Reaching out to open a door, putting a pot of water on the stove, dressing myself, or picking up my cat has all become harder. As I continue to decline, I am looking ahead to needing caretaking, and that is scary. Reachable Workspace is a clinical endpoint that recognizes the impact that this loss of range of motion in our shoulders and arms has on the lives of people with FSHD, and critical for developing a drug that slows the progression of disease and allows me to keep my independence longer.”

Phase 3 REACH Trial

Fulcrum plans to begin enrolling patients in its Phase 3 REACH clinical trial in the second quarter of 2022. REACH will be a randomized, double-blind, placebo-controlled, multi-national trial to evaluate the efficacy and safety of losmapimod for the treatment of FSHD. The trial is expected to enroll approximately 230 adults with FSHD. Patients will be randomized 1:1 to receive either losmapimod, administered orally as a 15 mg tablet twice a day, or placebo, and evaluated over a 48-week treatment period. RWS is the primary endpoint of the trial. Secondary endpoints include muscle fat infiltration (MFI) measured by MRI, Patient Global Impression of Change (PGIC), and Quality of Life in Neurological Disorders of the upper extremity (Neuro QOL-UE). REACH will also include patient-centered assessments of healthcare utilization.

In addition to the presentation on RWS, Fulcrum has multiple presentations at the MDA conference demonstrating its scientific and clinical leadership in FSHD drug discovery and development.

[Source(s): Fulcrum Therapeutics Inc, GlobeNewswire]