Sanofi and Fulcrum Therapeutics have entered into a collaboration and licence agreement for the development and commercialisation of an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
Losmapimod, a selective p38α/β mitogen activated protein kinase inhibitor that was recently granted Fast Track Designation by the US Food and Drug Administration, is currently being investigated as a potential transformative therapy for FSHD.
Currently one of the most common forms of muscular dystrophy, estimated to affect around 30,000 people in the US, FSHD is a rare, progressive and debilitating disease characterised by fat infiltration of skeletal muscle, which can lead to muscular atrophy.
Results from the phase 2 ReDUX4 trial showed that losmapimod demonstrated a slowing of disease progression and improved function in FSHD and is currently being evaluated in a phase 3 study, REACH, in people living with the condition.
Under the terms of the agreement, Sanofi will obtain exclusive rights to commercialise losmapimod outside of the US, while Fulcrum retains full US commercialisation rights.
In exchange, Fulcrum will receive an upfront payment of $80m and will be eligible to receive up to an additional $975m in specified regulatory and sales-based milestones, plus tiered escalating royalties on annual net sales of losmapimod outside of the US.
In addition, both companies will equally share future global development costs.
Burcu Eryilmaz, global head, rare diseases, Sanofi, commented: “This partnership provides an exciting opportunity to… deliver the first approved FSHD treatment to patients.”
She continued: “Losmapimod has shown meaningful clinical benefits that underscore the disease-modifying potential and opportunity to address the high unmet need for a safe and effective drug that slows disease progression.”
Alex Sapir, president and chief executive officer, Fulcrum, said: “We are excited about the potential to provide the first approved treatment for FSHD patients and we look forward to working with Sanofi to bring losmapimod to patients globally.”
The biopharmaceutical company expects to report topline data from the global REACH clinical trial in late 2024.
Following positive data, both companies plan to submit marketing applications in the US, Europe and Japan, as well as other markets.