Fulcrum Collabs With Sanofi to Develop and Commercialize of losmapimod in FSHD

Fulcrum Collabs With Sanofi to Develop and Commercialize of losmapimod in FSHD

By, Admin 15 May 2024

Fulcrum Therapeutics inks collaboration and license agreement with Sanofi to develop and commercialize of losmapimod in FSHD

Overview

Fulcrum Therapeutics, Inc. (Fulcrum), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, announced that it has entered into a collaboration and license agreement with Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Under the collaboration and license agreement, Sanofi obtains exclusive commercialization rights for losmapimod outside of the US.

Losmapimod: Phase 2 results

  • The collaboration and license agreement combines Fulcrum’s expertise in FSHD with Sanofi’s global reach and unparalleled commitment to treating patients with rare diseases. 
  • Losmapimod is currently being evaluated in a global phase 3 clinical trial for the treatment of FSHD, a chronic and progressive genetic muscular disorder that is characterized by significant muscle cell death and fat infiltration into muscle tissue. 
  • 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 US, Europe, Japan, and other geographies.

Words from CEO: Fulcrum Therapeutics

  • “Sanofi is a proven leader in developing therapeutics for rare neuromuscular diseases and is the ideal partner to maximize the opportunity and reach of losmapimod outside the US,” said Alex C. Sapir, Fulcrum’s president and chief executive officer. 
  • “This deal aligns with our core strategy, allowing Fulcrum to remain focused on preparations for commercialization of losmapimod in the US, while leveraging Sanofi’s exceptional global commercial capabilities and established infrastructure in key markets around the world. 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.”

Words from Sanofi

  • “This partnership provides an exciting opportunity to expand Sanofi’s rare disease franchise and deliver the first approved FSHD treatment to patients with the strength and reach of our commercial organization,” said Burcu Eryilmaz, Sanofi’s global head of rare diseases. 
  • “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. With a deep commitment to bringing hope and new treatment options to patients, we look forward to working closely with Fulcrum as losmapimod advances through late-stage development.”

Terms of the Agreement

Per the terms of the agreement, Fulcrum will receive an upfront payment of $80.0 million and is eligible to receive up to an additional $975.0 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 US. In addition, Fulcrum and Sanofi will equally share future global development costs.

Losmapimod

  • Losmapimod is a selective p38a/ß mitogen activated protein kinase (MAPK) inhibitor. 
  • Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38a/ß inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. 
  • Results reported from the phase 2 ReDUX4 trial demonstrated a slowing of disease progression and improved function, including positive impacts on upper extremity strength and functional measures supporting losmapimod’s potential to be a transformative therapy for the treatment of FSHD. 
  • Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, with no safety signals attributed to losmapimod. 
  • Losmapimod has been granted US Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD. 
  • Losmapimod is currently being evaluated in a Phase 3 multi-center randomized, double-blind, placebo-controlled, 48-week parallel-group study in people with FSHD (NCT05397470).

FSHD

  • 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. 
  • FSHD is one of the most common forms of muscular dystrophy and has an estimated patient population of 30,000 in the United States alone.

Fulcrum Therapeutics

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need.

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