On August 6, 2015 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair, provided a corporate update and reported financial results for the second quarter ended June 30, 2015 (Press release, Acceleron Pharma, AUG 6, 2015, View Source [SID:1234507052]).
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"In the second quarter, we presented promising new longer-term treatment data from our luspatercept programs. These results further reinforce our excitement about the potential of luspatercept to help patients with myelodysplastic syndromes and beta-thalassemia. We and our collaboration partner, Celgene, look forward to starting pivotal trials in both of these indications by the end of this year," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "At Acceleron, we remain focused on bringing innovative therapies to patients, and we are continuing to advance and expand our earlier stage pipeline as well. We have scheduled our first Research and Development Day for investors on October 23, 2015. At that meeting, we will review our clinical and preclinical programs, including a first look at top-line data from the phase 1 trial with our candidate muscle agent, ACE-083."
Recent Highlights and Current Updates
Development Programs
Hematology
Completed enrollment and treatment in the luspatercept phase 2 dose escalation study in myelodysplastic syndromes (MDS) – Enrollment and treatment ongoing in the phase 2 dose escalation beta-thalassemia study.
Long-term phase 2 extension studies ongoing with luspatercept in both MDS and beta-thalassemia – Patients who completed their 3-month treatment in the MDS or beta-thalassemia dose escalation studies were eligible to enroll in the 12-month MDS or beta-thalassemia extension study. Enrollment in the MDS extension study is complete and enrollment in the beta-thalassemia extension study is ongoing.
Presented luspatercept phase 2 data supporting the advancement to phase 3 clinical trials at the European Hematology Association (EHA) (Free EHA Whitepaper)’s annual meeting – In lower risk MDS patients, which represent a large majority of patients affected by the disease, longer-term treatment with luspatercept led to sustained increases in hemoglobin levels and transfusion independence. In both transfusion and non-transfusion dependent beta-thalassemia patients, luspatercept generated durable increases in hemoglobin levels, reductions in transfusion burden and improvements in iron overload. Based on these promising data, Acceleron and Celgene are advancing luspatercept to phase 3 clinical trials in MDS and beta-thalassemia.
Luspatercept granted Fast Track designations for beta-thalassemia – The United States Food and Drug Administration (FDA) has granted Fast Track Designations to luspatercept for two separate indications—the use of luspatercept for the treatment of patients with transfusion dependent beta-thalassemia and the use of luspatercept for the treatment of patients with non-transfusion dependent beta-thalassemia.
Oncology
Presented preliminary renal cell carcinoma (RCC) phase 2 data showing that the combination of dalantercept and axitinib generated encouraging, progression-free survival – In 2nd through 4th line RCC patients, the aggregate median progression-free survival (PFS) rate for the combination of dalantercept and axitinib across all three dose levels of dalantercept tested was 8.3 months. The median PFS has not yet been reached for the 0.9 mg/kg dose group, which has been chosen as the dose level for the randomized Part 2 stage of this study.
Muscle Diseases
Completed enrollment and treatment in ACE-083 phase 1 clinical trial – The phase 1 study of ACE-083, a therapeutic candidate designed to selectively increase muscle mass and strength in the muscle into which it is administered, has completed enrollment and treatment in healthy volunteers.
Advanced and expanded pipeline – Acceleron is advancing several promising preclinical programs, particularly those designed to increase muscle mass and strength. Our goal is to advance a fifth, internally discovered therapeutic candidate into clinical trials by the end of 2016.
Upcoming Milestones and Events
Phase 3 clinical trials with luspatercept in MDS and beta-thalassemia expected to start by year-end – Acceleron and Celgene plan to initiate pivotal programs for luspatercept in beta-thalassemia and MDS by year-end.
Acceleron Research and Development (R&D) Day scheduled for Friday October 23, 2015 – Acceleron will hold its first R&D Day on Friday morning, October 23, 2015 in New York City.
ACE-083 phase 1 clinical data – Preliminary, top-line data from the ACE-083 phase 1 clinical trial will be presented at the Acceleron R&D Day on October 23.
Data from a new preclinical muscle program expected to be presented at the World Muscle Society annual meeting – Acceleron plans to present data from a new preclinical program, ACE-2494, at the World Muscle Society’s 20th International Congress in Brighton, UK, which runs from September 30 to October 4, 2015.
Financial Results
Cash Position – Cash, cash equivalents and investments as of June 30, 2015 were $156.6 million. As of December 31, 2014 the company had cash and cash equivalents of $176.5 million. Acceleron expects that its cash, cash equivalents and investments as of June 30, 2015 will be sufficient to fund the Company’s operations into the second half of 2017.