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Acceleron's (XLRN) PAH Candidate Gets Orphan Designation in EU

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Zacks Equity Research
·3 min read
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Acceleron Pharma Inc. XLRN announced that the European Commission has granted Orphan Designation to its investigational pipeline candidate, sotatercept for the treatment of patients with pulmonary arterial hypertension (“PAH”), a rare disease of high unmet medical need.

In the EU, an Orphan status is given to products targeting a life-threatening or chronically debilitating rare disease that affects fewer than 5 in 10,000 individuals for which no suitable treatment is available.

Shares of Acceleron were up 4.6% following this news on Monday. In fact, so far this year, the stock has skyrocketed 145.2% compared with the industry’s increase of 7.1%.

price chart for XLRN
price chart for XLRN


We remind investors that in September 2019, sotatercept received an Orphan Drug tag in the United States for the treatment of PAH. The candidate also received a Breakthrough Therapy designation from the FDA for the same indication in April 2020.

Meanwhile, the European Medicines Agency bestowed a Priority Medicines (PRIME) designation to sotatercept for PAH in May 2020.

Please note that sotatercept is being developed as part of Acceleron’s licensing agreement with Bristol Myers Squibb BMY.

Sotatercept is currently being evaluated in several mid-to-late-stage studies for treating PAH. The phase II PULSAR study, which evaluated sotatercept in combination with approved PAH-specific medicines, met its primary endpoint in patients with PAH as well key secondary endpoints. Meanwhile, enrollment is also ongoing in an exploratory phase II study SPECTRA to understand sotatercept's potential impact on PAH with additional results expected in the first half of 2021.

Acceleron is currently planning to begin 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 plans to initiate a phase III registrational STELLAR study on sotatercept in patients with PAH by 2020-end. The company also expects to begin the HYPERION (early intervention) phase III study and the ZENITH (later intervention) phase III study in PAH patients in the middle of 2021.

Zacks Rank & Stocks to Consider

Acceleron currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector include Repligen Corporation RGEN and Aptose Biosciences, Inc. APTO, both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Repligen’s earnings estimates have moved 12.5% and 16.9% north for 2020 and 2021, respectively, over the past 60 days. The stock has rallied 99% year to date.

Aptose Biosciences’ loss per share estimate has been narrowed 10.3% for 2020 and 4.7% for 2021 over the past 60 days.

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Bristol Myers Squibb Company (BMY) : Free Stock Analysis Report
 
Repligen Corporation (RGEN) : Free Stock Analysis Report
 
Acceleron Pharma Inc. (XLRN) : Free Stock Analysis Report
 
Aptose Biosciences, Inc. (APTO) : Free Stock Analysis Report
 
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