Rare Daily Staff

Fulcrum Therapeutics said it entered into a collaboration and license agreement with Acceleron Pharma to use its platform technology to identify small molecules to treat an undisclosed pulmonary disease.

Under the agreement, 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.

Acceleron will use Fulcrum’s proprietary drug discovery platform, which is designed to identify targets for genetically defined rare diseases. They will focus on genes that are known to impact specific pathways associated with the undisclosed pulmonary disease. Fulcrum uses the platform for drug discovery across a wide range of genetic diseases including muscle diseases, central nervous system diseases, and blood disorders.

Fulcrum will receive $10 million in an upfront payment and will be reimbursed 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. It will also receive tiered royalty payments in the mid-single digit to low double-digit range on net sales.

“This collaboration builds on and extends the proven potential of our platform to identify therapies that can address the root cause of diseases,” said Robert Gould, CEO of Fulcrum Therapeutics. “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.”

Photo: Robert Gould, CEO of Fulcrum Therapeutics