On November 3, 2016 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, reported a corporate update and reported financial results for the third quarter ended September 30, 2016 (Press release, Acceleron Pharma, NOV 3, 2016, View Source [SID1234516218]).
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"In the third quarter, we continued to make strong progress across our pipeline of clinical and preclinical programs," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "Enrollment continues to go well in both of the ongoing luspatercept Phase 3 studies in patients with myelodysplastic syndromes and beta-thalassemia. We look forward to providing new and updated preliminary results from several Phase 2 studies at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December. Within our muscle program, we’re on track to initiate the ACE-083 Phase 2 study in patients with facioscapulohumeral muscular dystrophy before year-end."
Continued Dr. Knopf, "I would also like to extend a warm welcome to the Company’s newly appointed Chief Executive Officer, Habib Dable, who will assume this role on December 1st. Habib’s global pharmaceutical operational and commercial experience will serve Acceleron well as we advance toward becoming a commercial-stage company."
THIRD QUARTER 2016 HIGHLIGHTS
Development Programs
Hematology
Enrollment is ongoing in the pivotal luspatercept Phase 3 clinical trials for the treatment of myelodysplastic syndromes (MDS) patients (the MEDALIST study) and beta-thalassemia patients (the BELIEVE study).
Enrollment is ongoing in Phase 2 trial cohorts evaluating luspatercept in a first-line treatment setting in low- or intermediate-risk MDS patients.
Data from five clinical abstracts on luspatercept and sotatercept will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held in San Diego, California on December 3-6, 2016.
Neuromuscular Disease
Initiation of the ACE-083 Phase 2 study in facioscapulohumeral muscular dystrophy (FSHD) patients expected to start by the end of the year.
Oncology
Enrollment continues in randomized part 2 of the Phase 2 DART study of dalantercept in combination with axitinib in patients with advanced renal cell carcinoma (RCC). The primary endpoint of this trial, progression-free survival (PFS), is an event-driven assessment with preliminary data expected in the first half of 2017.
Preclinical Research and Development
Acceleron continues its research on several molecules targeting musculoskeletal diseases, fibrotic disorders and other serious diseases. The most advanced molecule from the IntelliTrap platform, ACE-2494, continues to advance through IND-enabling activities. The Company plans to initiate a Phase 1 clinical trial with ACE-2494 in mid-2017.
Other Corporate Updates
Habib Dable appointed as President and CEO effective December 1, 2016. Mr. Dable joins Acceleron from Bayer AG, where he most recently served as President of U.S. Pharmaceuticals.
John Knopf retiring as President and CEO. Dr. Knopf is expected to apply his scientific expertise as an advisor to the Company.
Upcoming Events in Q4 2016
Present data at the ASH (Free ASH Whitepaper) annual meeting from ongoing luspatercept Phase 2 studies and an ongoing sotatercept Phase 2 investigator initiated trial in patients with myelofibrosis.
Initiate ACE-083 Phase 2 study in patients with FSHD, one of the most prevalent forms of muscular dystrophy.
Provide sotatercept development plan update before year-end.
Present at the 25th Annual Credit Suisse Healthcare Conference on Monday, November 7, 2016 in Scottsdale, Arizona.
Participate at the Citi Global Healthcare Conference to be held on December 7-8, 2016 in New York.
Financial Results
Cash position – Cash, cash equivalents and investments as of September 30, 2016 were $251.0 million. As of December 31, 2015 the Company had cash, cash equivalents and investments of $136.0 million. The Company believes that existing cash, cash equivalents and investments will be sufficient to fund its projected operating requirements into the second half of 2019.
Revenue – Collaboration revenue for the third quarter was $3.0 million. Cost sharing reimbursement revenue from the Company’s Celgene partnership was $2.9 million and is related to expenses incurred by the Company in support of its partnered programs.
Costs and expenses – Total costs and expenses for the third quarter were $23.5 million. This includes R&D expenses of $17.1 million and G&A expenses of $6.4 million.
Other expense, net – Other expense for the third quarter was $0.3 million and includes a $0.8 million, non-cash, loss on marking the Company’s common stock warrant liability to market.
Net loss – The Company posted a net loss for the third quarter ended September 30, 2016 of $20.8 million.