Acceleron & Fulcrum Therapeutics Announce Pulmonary Research & Discovery Collaboration Agreement

Acceleron Pharma Inc. and Fulcrum Therapeutics, Inc. recently announced they have entered into a collaboration and license agreement to identify small molecules designed to modulate specific pathways associated with a targeted indication within the pulmonary disease space.

“This collaboration brings together Fulcrum’s skill in identifying drug targets based on modulation of genetic pathways associated with disease and Acceleron’s deep expertise in TGF-beta superfamily signaling in an effort to generate potentially disease-modifying therapeutics,” said Habib Dable, Chief Executive Officer of Acceleron Pharma. “With this agreement, along with the advancement of the Acceleron-discovered assets sotatercept—in Phase 2 trials in pulmonary arterial hypertension—and ACE-1334, we underscore our growing commitment to the development of novel therapies for patients with pulmonary diseases of high unmet medical need.”

Under the agreement, Acceleron will have access to Fulcrum’s unique, proprietary product engine and target identification platform with the potential to identify small molecules that control the expression of genes known to impact specific pathways associated with a pulmonary disease of interest. Acceleron and Fulcrum will collaborate on the identification of therapeutic targets and small molecule drug candidates for those targets. Acceleron will be responsible for all development and commercialization activities for any potential therapeutics identified via this platform. Fulcrum will receive a one-time, upfront payment of $10 million as well as reimbursement for relevant R&D costs. Fulcrum will also be eligible to receive research, development and commercial milestone payments of up to $295 million for a first product commercialized and up to a maximum of $143.5 million in additional milestone payments for all subsequent products commercialized. Fulcrum will additionally receive tiered royalty payments in the mid-single-digit to low double-digit range on net sales.

“We are very pleased to partner with Acceleron on this important research initiative,” said Robert J. Gould, PhD, Chief Executive Officer of Fulcrum Therapeutics. “This collaboration builds on and extends the proven potential of our platform to identify therapies that can address the root cause of diseases, including our progress with losmapimod, currently in a Phase 2 clinical trial for FSHD and extensive preclinical and early stage research targeting other genetically defined diseases. This new opportunity to screen and identify pulmonary disease-specific therapies is another reflection of the broad potential applications of the Fulcrum platform in gene modulation.”

Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron’s leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body’s ability to regulate cellular growth and repair.

Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol-Myers Squibb, are co-promoting newly approved REBLOZYL (luspatercept-aamt), the first and only approved erythroid maturation agent, in the US and are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes and myelofibrosis. Acceleron is also advancing its neuromuscular program with ACE-083, a locally-acting Myostatin+ agent in Phase 2 development in Charcot-Marie-Tooth disease and is conducting a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension. For more information, visit www.acceleronpharma.com.

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. Fulcrum has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia. For more information, visit www.fulcrumtx.com.