Acceleron Receives FDA Orphan Drug Designation for ACE-1334 for the Treatment of Systemic Sclerosis

Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation to ACE-1334 for the treatment of patients with systemic sclerosis. The Company intends to initiate a Phase 1b/Phase 2 trial in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in 2021.

“We’re excited that ACE-1334 has received Orphan Drug designation, as it aligns with our vision to develop novel therapies for pulmonary diseases of high unmet medical need,” said Habib Dable, President and Chief Executive Officer of Acceleron. “It’s clear that 2021 will be an important year in this regard, as we seek to expand our portfolio in this important therapeutic area. In addition to advancing our Phase 3 clinical program to investigate sotatercept’s potential to reverse vascular remodeling associated with pulmonary arterial hypertension, we’re pleased to be planning for a Phase 1b/Phase 2 study to determine whether the anti-fibrotic activity ACE-1334 has demonstrated in preclinical models of fibrosis can be replicated in patients with SSc-ILD.”

Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website.

ACE-1334 is an investigational therapy that is not approved for any use in any country.

About ACE-1334

ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. TGF-beta 1 and 3 are believed to be key signaling factors in the pathogenesis of fibrotic disease. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis. ACE-1334 recently completed an ascending-dose Phase 1 clinical trial in healthy volunteers. The FDA has granted Fast Track designation to ACE-1334 in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) as well as Orphan Drug designation for the treatment of systemic sclerosis.

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