RARE Daily

Fulcrum Enters Exclusive Global License Agreement in Rare Hematologic Anemia

July 10, 2023

Rare Daily Staff

Fulcrum Therapeutics has entered into a worldwide, exclusive license agreement with Camp4 Therapeutics to advance the discovery, development, and commercialization of novel therapeutic agents against an undisclosed target for the potential treatment of Diamond-Blackfan anemia.

Diamond-Blackfan anemia (DBA) is a rare, congenital, blood disorder that affects an estimated 5,000 individuals worldwide. DBA is caused by genetic mutations in ribosomal subunits that halt red blood cell maturation and lead to anemia. Patients are usually diagnosed in infancy with the presentation of severe anemia and potential developmental abnormalities. Patients with DBA require lifelong management with corticosteroids and blood transfusions that are known for their toxicities and long-term complications.

“This agreement further strengthens our discovery pipeline and reinforces our strategy of addressing rare genetic conditions through small molecules,” said Jeff Jacobs, chief scientific officer, Fulcrum Therapeutics.

Under the terms of the agreement, Fulcrum has been granted an exclusive, worldwide license to intellectual property arising from CAMP4’s preclinical DBA program, including the right to research, develop, manufacture, and commercialize investigational compounds against an undisclosed target. In exchange, Camp4 Therapeutics will receive an undisclosed upfront payment and up to $70 million in additional payments, upon the achievement of certain development, regulatory, and commercial milestones. Fulcrum will assume sole responsibility for research, development, manufacturing, and commercialization costs and activities, and will pay tiered royalties on future commercial sales.

“Our mission is to harness the power of the cell’s transcriptional regulators to unlock new options for people living with genetic diseases like DBA,” said Josh Mandel-Brehm, CEO of Camp4 Therapeutics. “By uniting our scientific insights with Fulcrum’s deep expertise in hematology and small molecule development, this agreement will help accelerate the discovery and delivery of ground-breaking therapies for this underserved community.”

Photo: Josh Mandel-Brehm, CEO of Camp4 Therapeutics

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