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Acceleron net loss for the first quartere 2019 $38.1M, R&D expenses $32.8

Luspatercept Biologics License Application (BLA) and Marketing
Authorization Application (MAA) submitted in April 2019

Part 2 results from the ACE-083 Phase 2 trials in
facioscapulohumeral muscular dystrophy (FSHD) expected in the second
half of 2019 and Charcot-Marie-Tooth disease (CMT) anticipated in Q1
2020

PULSAR and SPECTRA Phase 2 trials of sotatercept in pulmonary
arterial hypertension (PAH) on track with topline results expected from
PULSAR in 1H 2020

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (Nasdaq:XLRN), a leading biopharmaceutical company
in the discovery and development of TGF-beta superfamily therapeutics to
treat serious and rare diseases, today provided a corporate update and
reported financial results for the first quarter ended March 31, 2019.

“With the submission of marketing applications for luspatercept in the
U.S. and E.U. last month, we and our global collaboration partner,
Celgene, are excited about the potential to bring a new therapy to
patients with myelodysplastic syndromes and beta-thalassemia within the
next year,” said Habib Dable, President and Chief Executive Officer of
Acceleron. “At the same time, our pulmonary program remains on track,
with enrollment ongoing in two Phase 2 trials of sotatercept in PAH, and
we are anticipating topline results from the placebo-controlled part of
the Phase 2 trial of our locally-acting muscle agent, ACE-083, in FSHD
during the second half of this year.”

Development Program Highlights

Hematology

Luspatercept: Myelodysplastic Syndromes (MDS),
Beta-Thalassemia, and Myelofibrosis (MF)

Luspatercept is an investigational first-in-class erythroid
maturation agent (EMA) designed to address a late-stage erythroid
maturation defect that results in chronic anemia and the need for
regular red blood cell transfusions in adults with serious hematologic
diseases. Luspatercept is part of the global collaboration between
Acceleron and Celgene.

Neuromuscular Disease

ACE-083: Facioscapulohumeral Muscular Dystrophy
(FSHD) and Charcot-Marie-Tooth Disease (CMT)

ACE-083 is an investigational locally-acting therapeutic designed to
have a concentrated effect on muscle mass and strength in target muscles
for diseases that cause focal muscle weakness. ACE-083 utilizes the
“Myostatin+” approach to inhibit multiple TGF-beta superfamily ligands
involved in muscle formation.

Pulmonary Disease

Sotatercept: Pulmonary Arterial Hypertension
(PAH)

Sotatercept is an investigational agent designed to be a selective
ligand trap for members of the TGF-beta superfamily to rebalance BMPR2
signaling, which is a key molecular driver of PAH. In preclinical
studies of PAH, sotatercept reversed pulmonary vessel muscularization
and improved indicators of right heart failure.

Financial Results

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its first
quarter 2019 financial results and provide an update on recent corporate
activities on May 9, 2019, at 5:00 p.m. EDT.

The webcast will be accessible under “Events & Presentations” in the
Investors/Media page of the Company’s website at www.acceleronpharma.com.
Individuals can participate in the conference call by dialing
877-312-5848 (domestic) or 253-237-1155 (international) and referring to
the “Acceleron First Quarter 2019 Earnings Call.”

The archived webcast will be available for replay on the Acceleron
website approximately two hours after the event.

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