CAMBRIDGE, Mass.--(BUSINESS WIRE)--Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, today provided a corporate update and reported financial results for the first quarter ended March 31, 2017.

“In the first quarter, we made significant progress across our entire pipeline. Our luspatercept program in MDS and beta-thalassemia continues to build momentum, as we look to complete enrollment of the MEDALIST and BELIEVE Phase 3 studies ahead of schedule, and expand into additional patient populations where there are limited treatment options, including first-line, lower-risk MDS and myelofibrosis,” said Habib Dable, President and Chief Executive Officer of Acceleron. "As we continue to explore further expansion opportunities for luspatercept, we are also preparing to expand our wholly-owned pipeline into a second neuromuscular disease, with the expected initiation by mid-year of a Phase 2 study with ACE-083 in patients with Charcot-Marie-Tooth disease.”

DEVELOPMENT PROGRAM HIGHLIGHTS

Hematology

Luspatercept in myelodysplastic syndromes (MDS)

Luspatercept is being developed to treat anemia and reduce or eliminate the need for red blood cell transfusions in patients with MDS, a rare disorder in which bone marrow fails to produce enough healthy blood cells. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

• Enrollment in the MEDALIST Phase 3 clinical trial is expected to complete in the second quarter of this year. The MEDALIST Phase 3 study is enrolling 210 patients to evaluate the efficacy and safety of luspatercept in patients with anemia due to lower-risk MDS with ring sideroblasts who require red blood cell (RBC) transfusions.
• Results presented at the 14th International Symposium on MDS from an ongoing Phase 2 study in first-line, lower-risk MDS patients demonstrated positive erythroid response and RBC transfusion independence rates.
• Acceleron and Celgene announced plans to initiate a third Phase 3 trial with luspatercept in early 2018. The new Phase 3 trial will evaluate luspatercept treatment versus standard-of-care in the first-line treatment setting for lower-risk MDS patients.

Luspatercept in beta-thalassemia

Luspatercept is designed to treat severe, chronic anemia and reduce RBC transfusion-dependence in adults with beta-thalassemia, a rare genetic disorder that reduces the production of hemoglobin. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

• Enrollment in the BELIEVE Phase 3 trial is expected to complete in the second quarter of this year. The BELIEVE Phase 3 study is enrolling 300 patients to evaluate the efficacy and safety of luspatercept in patients with anemia due to beta-thalassemia who require regular RBC transfusions.

Neuromuscular Disease

ACE-083 in facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) disease

ACE-083 is designed to increase muscle mass and strength in target muscles for diseases that cause debilitating focal muscle loss.

• Enrollment and treatment are ongoing in Part 1 of the ACE-083 Phase 2 study in patients with FSHD, one of the most prevalent forms of muscular dystrophy.
• Announced plans to initiate a second Phase 2 clinical trial with ACE-083 in patients with Charcot-Marie-Tooth (CMT) disease, one of the most common inherited neurologic diseases leading to focal muscle weakness, by mid-2017.

Preclinical Research

Acceleron continues its research on several molecules targeting musculoskeletal diseases, fibrotic disorders and other serious diseases.

• The Company plans to initiate a Phase 1 healthy volunteer clinical trial this year with ACE-2494.

NEWSFLOW AND CATALYSTS

Luspatercept

• Complete enrollment in the MEDALIST Phase 3 trial in Q2 2017
• Complete enrollment in the BELIEVE Phase 3 trial in Q2 2017
• Anticipate top-line data from both Phase 3 trials in mid-2018
• Initiate first-line, lower-risk MDS Phase 3 trial in early 2018
• Initiate Phase 2 trial in myelofibrosis by year-end 2017
• Initiate Phase 2 trial in non-transfusion dependent (NTD) beta-thalassemia by year-end 2017
• Additional Phase 2 results from ongoing trials to be presented at medical conferences in 2017

ACE-083

• Report initial FSHD Phase 2 Part 1 dose-escalation results by late 2017
• Initiate CMT Phase 2 Part 1 dose-escalation trial by mid-2017

Dalantercept

• Report top-line results in the DART Phase 2 trial in renal cell carcinoma by Q2 2017

ACE-2494

• Initiate Phase 1 healthy volunteer study in 2017

Corporate

• Host a Research and Development Day to discuss preclinical research and clinical development activities in 2H 2017

Financial Results

• Cash position – Cash, cash equivalents and investments as of March 31, 2017 were $213.2 million. As of December 31, 2016 the Company had cash, cash equivalents and investments of $234.4 million. We believe that existing cash, cash equivalents and investments will be sufficient to fund projected operating requirements into the second half of 2019.
• Revenue – Collaboration revenue for the first quarter was $3.7 million. The revenue is all from our Celgene partnership and is primarily due to cost sharing revenue of $3.6 million related to expenses incurred by the Company in support of our partnered programs.
• Costs and expenses – Total costs and expenses for the first quarter were $29.6 million. This includes R&D expenses of $21.7 million and G&A expenses of $7.8 million.
• Other income, net – Other income, net for the first quarter was $0.5 million primarily due to interest income.
• Net loss – The Company's net loss for the first quarter ended March 31, 2017 was $25.4 million.

About Acceleron

Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases. Its pioneering research platform leverages the powerful biology behind the body’s ability to rebuild and repair its own cells and tissues. This approach to drug discovery has generated four therapeutic candidates that are currently in clinical trials. The Company’s lead therapeutic candidate, luspatercept, is being evaluated in Phase 3 studies for the treatment of the hematologic diseases myelodysplastic syndromes and beta-thalassemia under a global partnership with Celgene Corp. Acceleron is also advancing clinical programs in the fields of oncology and neuromuscular diseases and has a comprehensive preclinical research effort targeting fibrotic and other serious diseases.

For more information, please visit www.acceleronpharma.com.