Acceleron's long-running collaboration with Celgene on sotatercept has taken another turn, with an amendment of their agreement to give Acceleron the right to develop the drug for pulmonary diseases.

Acceleron plans to start a phase 2 trial of sotatercept (ACE-011) in pulmonary arterial hypertension (PAH) in the first half of 2018.

The biopharma's CEO Habib Dable told analysts that PAH has a high unmet need, with around 30,000 patients in the U.S. alone and no disease-modifying treatment approved, with most therapies focused on dilating the blood vessels to alleviate symptoms. Sotatercept could become the first disease-modifying therapy for PAH, which typically strikes in middle age and has an average survival of just five to seven years.

"Sotatercept will be the lead family in our new pulmonary disease franchise," he said, adding that this will sit alongside its established hematology and neuromuscular programs. The company plans to have three programs in phase 3 development in the next three years—in other words, by the end of 2020.

With sotatercept, Acceleron gets a phase 2-ready project with clinical experience in 300 patients to date, said a spokesman for the company. Celgene has retained rights in non-pulmonary diseases and will get royalties on sales in pulmonary indications in the low 20s if sotatercept reaches the market.

"While many therapies are approved for the treatment of pulmonary arterial hypertension, these therapies all focus on a mechanism of vasodilation and the prognosis for patients remains poor," said Eric Austin, M.D., director of the Vanderbilt Pediatric Pulmonary Hypertension Program.

"Sotatercept's mechanism is intended to rectify the deficits in molecular signaling that underlie both the familial and idiopathic forms of this disease."

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