Amended Collaboration Deal Gives Acceleron the Global Rights to Potential PAH Therapy Sotatercept

Celgene has given Acceleron Pharma the global rights to develop and commercialize sotatercept as a  treatment for pulmonary arterial hypertension (PAH) and other lung diseases.

Acceleron obtained the rights under a revision of the collaboration agreement that the companies signed in February 2008.

The terms of the original agreement will apply to sotatercept’s development and commercialization as a treatment for conditions other than lung diseases.

Sotatercept, also known as ACE-011, is an engineered protein that binds to members of the TGF-β protein superfamily, inhibiting their activity. Preclinical-trial studies and early trials suggest that it could be an effective PAH therapy.

TGF-β is involved in tissue remodeling and regeneration processes, including those affecting vessels and fibrosis. Mutations of the gene that encodes the protein can influence the age at which PAH strikes, and whether it will run in families. These findings further support the notion that TGF-β can play a critical role in the development of PAH, highlighting its potential as therapeutic target.

“While many therapies are approved for the treatment of pulmonary arterial hypertension, these therapies all focus on a mechanism of vasodilation [widening of blood vessels], and the prognosis for patients remains poor,” Dr. Eric Austin, director of the Vanderbilt Pediatric Pulmonary Hypertension Program, said in a press release.

“Sotatercept’s mechanism is intended to rectify the deficits in molecular signaling that underlie both the familial [inherited] and idiopathic [unknown cause] forms of this disease,” he said. “The preclinical data with sotatercept is very encouraging, and I look forward to seeing data from clinical trials.”

Under the amended collaboration agreement, Celgene will receive royalties on global sales of sotatercept. Regulators must approve the therapy before the sales can occur, of course.

“This amended agreement unites Acceleron’s scientific leadership in the TGF-beta superfamily with Celgene’s commitment to advance novel molecules, such as sotatercept, for patients in therapeutic areas of high unmet medical need,” said Scott Smith, Celegene’s chief operating officer.

Acceleron will start Phase 2 trials in the first half of 2018 on sotatercept’s safety and ability to treat PAH.

“We are very pleased to announce the execution of this amended agreement alongside our long-time collaboration partner, Celgene,” said Habib Dable, Acceleron’s chief executive officer. “Sotatercept shows tremendous potential to be a groundbreaking therapy for pulmonary arterial hypertension, and we expect sotatercept, as a Phase 2-ready compound, to be the lead molecule in our new pulmonary disease franchise.”