Dive Brief:

• Sanofi is adding to its portfolio of rare disease therapies, agreeing on Monday to acquire partial rights to an experimental medicine Fulcrum Therapeutics is developing for a form of muscular dystrophy.
• Per deal terms, Sanofi is paying Fulcrum $80 million for rights outside the U.S. to the medicine, called losmapimod. Fulcrum could get up to $975 million more in downstream payments, as well as royalties on international sales, and will equity split development costs with Sanofi. 
• Fulcrum’s drug is currently in Phase 3 testing for facioscapulohumeral muscular dystrophy, or FSHD, a rare genetic disease that causes muscle weakness. The drug failed a Phase 2 study, but an after-the-fact analysis showed potential effects on a measure of function that’s now the main goal of the ongoing late-stage trial. Results are expected later this year. 

Dive Insight:

Losmapimod was once owned by GSK and thought of as a way to prevent heart attacks in people with cardiovascular disease. 

But Fulcrum, while a privately held biotechnology startup, licensed it in 2019 with a different development plan. It began testing the drug against FSHD, a condition that causes weakness in the muscles of the face, shoulders and arms. Losmapimod blocks an enzyme involved in the production of a protein, DUX4, implicated in disease progression. 

Fulcrum has since gone public and brought losmapimod into Phase 3 tests, but not without some setbacks along the way. Data supporting losmapimod have been mixed and the company has changed leaders multiple times. A second experimental therapy for sickle cell disease has also faced regulatory delays.  

Still, Fulcrum now has Sanofi’s support as it nears the completion of losmapimod’s late-stage trial in FSHD. The company is testing whether the medicine can lead to meaningful improvements in “reachable workspace,” a measure of someone’s range of motion. The Phase 3 trial enrolled 260 people, initially randomized them to receive either losmapimod or a placebo for 48 weeks, and then evaluated their reachable workspace. Afterwards, placebo recipients were given Fulcrum’s drug.

Fulcrum is being chased by Avidity Biosciences, which has an RNA-based medicine for FHSD in early-stage testing. Preliminary study results are expected by the end of June.

In Sanofi, Fulcrum has found a partner with experience marketing neuromuscular disease drugs as well as rare disease medicines. The France-based drugmaker sells two treatments for multiple sclerosis and several therapies for lysosomal storage disorders.