Acceleron and Fulcrum Strike Deal to Address Pulmonary Diseases

Acceleron Pharma and Fulcrum Therapeutics are entering 2020 as partners to tackle pulmonary diseases. On Monday, the two companies forged a collaboration and license agreement that has the potential to be worth more than $450 million.

The Cambridge, Mass.-based companies inked a deal to identify small molecules designed to modulate specific pathways associated with a targeted indication within the pulmonary disease space. Fulcrum has a product engine and target identification platform that uses computational biology, as well as a proprietary relational database that will help in the identification of small molecules that control the expression of certain genes. Acceleron will use Fulcrum’s platform to focus on genes that are known to impact specific pathways associated with pulmonary disease, the companies said. Fulcrum has also used the platform for drug discovery in a range of genetically defined muscle, central nervous system and blood disorders. Acceleron will be responsible for all development and commercialization activities for any potential therapeutics identified via this platform.

 Habib Dable, chief executive officer of Acceleron, said the collaboration brings in Fulcrum’s skill in identifying drug targets that are based on the modulation of genetic pathways associated with disease. That knowledge, Dable said, will be paired with Acceleron’s “deep expertise in TGF-beta superfamily signaling.” The different skill sets the companies bring together will be aimed at generating what they hope will become disease-modifying therapies.

For Acceleron, the deal comes several months after its mid-stage asset sotatercept received Orphan Drug Designation from the U.S. Food and Drug Administration as a potential treatment in pulmonary arterial hypertension (PAH). Sotatercept is being evaluated in two Phase II trials in patients with PAH, with top-line results from one of those trials expected in the first quarter of 2020. Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH, a rare progressive disorder that is characterized by the constriction of pulmonary arteries and elevated blood pressure in the pulmonary circulation.

“With this agreement, along with the advancement of the Acceleron-discovered assets sotatercept—in Phase II 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,” Dable said in a statement.

Under terms of the agreement, Acceleron will provide Fulcrum with an upfront payment of $10 million and will reimburse the company for relevant R&D costs. Fulcrum will also R&D and commercial milestone payments of up to $295 million for the first product to be approved as a result of this collaboration. Also, Fulcrum will receive up to $143.5 million in additional milestone payments for all subsequent products commercialized, the companies said.

Fulcrum CEO Robert J. Gould said the collaboration with Acceleron builds on the “proven potential” of the company’s platform to identify therapies that can address the root cause of diseases. He noted that the platform led to the discovery of Fulcrum’s mid-stage asset losmapimod, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD) in patients with a genetically confirmed diagnosis of FSHD.

“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,” Gould said in a statement.