Sanofi and Fulcrum Collaborate to Develop and Commercialize FSHD Therapeutic

Rare Daily Staff

Fulcrum Therapeutics entered into a collaboration and license agreement with Sanofi for the development and commercialization of losmapimod, an investigational oral small molecule in late-stage development for the treatment of facioscapulohumeral muscular dystrophy.

The deal combines Fulcrum’s expertise in FSHD with Sanofi’s global reach and commitment to treating patients with rare diseases.

As part of the agreement, Sanofi obtains global commercialization rights outside the United States. Sanofi will pay Fulcrum $80 million upfront in cash. Fulcrum is also eligible to receive up to an additional $975 million in specified regulatory and sales-based milestones, along with tiered escalating royalties starting in the low-teens on annual net sales of losmapimod outside the United States. In addition, Fulcrum and Sanofi will equally share future global development costs.

Facioscapulohumeral muscular dystrophy (FSHD) is a serious, rare, progressive and debilitating disease for which there are no approved treatments. It is characterized by fat infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, scapula and shoulders, upper arms, and abdomen. Impact on patients includes profound decreases in the ability to perform activities of daily living, loss of upper limb function, loss of mobility and independence and chronic pain.

Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor currently being evaluated in a global phase 3 clinical trial for the treatment of FSHD. Results from ReDUX4, the phase 2 clinical trial evaluating losmapimod for the treatment of FSHD, demonstrated a slowing of disease progression and improved muscle health. Fulcrum expects to report topline data from REACH, the global phase 3 clinical trial, in the fourth quarter of 2024. Following positive data from the phase 3 trial, Fulcrum and Sanofi plan to submit marketing applications in the United States, Europe, Japan, and other geographies.

Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Losmapimod has been granted U.S. Food and Drug Administration Fast Track and Orphan Drug designations for the treatment of FSHD.

“This deal aligns with our core strategy, allowing Fulcrum to remain focused on preparations for commercialization of losmapimod in the U.S., while leveraging Sanofi’s global commercial capabilities and established infrastructure in key markets around the world,” said Alex Sapir, president and CEO of Fulcrum Therapeutics.

Photo: Alex Sapir, president and CEO of Fulcrum Therapeutics