Acceleron Pharma Inc., a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of therapeutics to treat serious and rare diseases, announced its major corporate research and development goals and priorities for 2017.

“With our Phase 3 luspatercept programs in MDS and beta-thalassemia advancing on plan, 2017 will be a transformational year for Acceleron,” said Habib Dable, president and chief executive officer. “We are looking ahead to the clinical, regulatory and commercial milestones that will help us achieve our vision of becoming a fully integrated biopharmaceutical company.

“Additionally, we continue to advance and expand our wholly-owned portfolio of innovative protein therapeutics for patients with serious diseases. With multiple Phase 3 and Phase 2 trials ongoing and new INDs expected in 2017 and 2018, we believe that our pipeline of therapeutic candidates positions us to create significant value for our shareholders while making a meaningful difference in the lives of patients who have limited treatment options.”

The Company’s major research and development goals and priorities are highlighted below:

Luspatercept in Rare Blood Disorders

Luspatercept is being developed to treat patients who have anemia associated with rare blood disorders, including beta-thalassemia and malignant disorders such as myelodysplastic syndromes (MDS) and myelofibrosis.

Goals for luspatercept in myelodysplastic syndromes (MDS):

• Complete patient enrollment in the MEDALIST Phase 3 clinical trial in the second half of this year
• Release topline results for the MEDALIST Phase 3 trial by the end of next year
• Evaluate and design a clinical and regulatory strategy for luspatercept in first-line lower risk MDS patients

Goals for luspatercept in beta-thalassemia:

• Complete patient enrollment in the BELIEVE Phase 3 clinical trial in the second half of this year
• Release topline results for the BELIEVE Phase 3 trial by the end of next year
• Initiate a Phase 2 trial in patients with non-transfusion dependent beta-thalassemia by the end of this year

Goals for luspatercept in myelofibrosis:

• Initiate a Phase 2 trial in myelofibrosis by the end of this year

ACE-083 in Neuromuscular Disease

ACE-083 is being developed to increase muscle mass and strength in target muscle groups for diseases such as facioscapulohumeral muscular dystrophy (FSHD), where patients experience focal muscle loss. Acceleron plans to:

• Present initial topline results from the open label, dose-escalation stage of the Phase 2 study in FSHD in late 2017
• Initiate the randomized, double-blind, placebo-controlled stage of the Phase 2 study in 2018
• Initiate a Phase 2 clinical trial in a second neuromuscular disease

Pipeline Expansion

Acceleron continues its research on several preclinical protein therapeutics targeting fibrotic disorders, vascular disease, and musculoskeletal disease. Acceleron’s current goals for research and pipeline expansion include:

• Initiate a Phase 1 healthy volunteer study with ACE-2494 this year
• Conduct IND-enabling development work to advance a new protein therapeutic to the clinic in 2018
• Host an investor and analyst research day to discuss ongoing preclinical research and potential future disease areas in the second quarter of this year

Dalantercept in Advanced Renal Cell Carcinoma

Dalantercept is being developed in advanced renal cell carcinoma in combination with axitinib to further inhibit tumor angiogenesis. Acceleron expects to present topline results from the Phase 2 DART study in the second half of 2017. The primary endpoint of this trial, progression-free survival (PFS), is an event-driven assessment.

(Source: Business Wire)