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Acceleron (XLRN) Gets Orphan Drug Tag for ACE-1334 by FDA

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Acceleron Pharma Inc. (XLRN - Free Report) announced that the FDA granted orphan drug designation to ACE-1334 for the treatment of patients with systemic sclerosis.

Shares of the company have surged 126.3% compared with the industry’s growth of 4.3%.


Notably, an orphan drug designation is usually granted to drugs that are capable of treating rare diseases that affect less than 200,000 people in the United States. This tag also makes the company entitled to certain other benefits, including tax credits related to clinical trial expenses and an exemption from the FDA user fee.

ACE-1334 is a transforming growth factor (TGF)-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands, which are believed to be key signaling factors in the pathogenesis of the fibrotic disease.

In preclinical studies, ACE-1334 has been observed to have antifibrotic activity in multiple models of fibrosis. The investigational drug was recently evaluated in an ascending-dose phase I study in healthy volunteers. The company intends to initiate a phase Ib/phase II study in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in 2021. The company intends to initiate the 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.

Apart from ACE-1334, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension (PAH). The company reported positive top-line results from the PULSAR phase II study. It is currently planning multiple phase III studies to support its long-term vision of establishing sotatercept as a backbone therapy for patients with PAH at all stages of the disease.

The company’s drug Reblozyl is a novel erythroid maturation agent (EMA) approved to treat anaemia associated with beta-thalassemia in adult patients who require regular red blood cell (RBC) transfusions.

Zacks Rank & Stocks to Consider

Acceleron currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the healthcare sector include ASLAN Pharmaceuticals Ltd. (ASLN - Free Report) , Aptose Biosciences Inc. (APTO - Free Report) and Alimera Sciences Inc. (ALIM - Free Report) . All of them carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

ASLAN’s loss per share estimates have narrowed from 42 cents to 40 cents for 2020 and from 60 cents to 57 cents for 2021 over the past 60 days.

Aptose’s loss per share estimates have narrowed from 77 cents to 69 cents for 2020 and from 85 cents to 81 cents for 2021 over the past 60 days.

Alimera’s loss per share estimates have narrowed from $1.31 to 96 cents for 2020 over the past 60 days.


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