On August 4, 2016 Acceleron Pharma Inc. (NASDAQ: XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of highly innovative therapeutics to treat serious and rare diseases, reported a corporate update and reported financial results for the second quarter ended June 30, 2016 (Press release, Acceleron Pharma, AUG 4, 2016, View Source [SID:1234514228]).

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"In the second quarter, we presented updated luspatercept data that builds upon the encouraging Phase 2 results and we have also reinforced the initial results with ACE-083 by showing impressive increases in muscle volume in the second muscle studied in the Phase 1 clinical trial," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "With luspatercept now in two Phase 3 clinical trials, ACE-083 heading toward its first Phase 2 clinical trial in the second half of 2016, a rapidly expanding and advancing pipeline of preclinical programs and capital to finance the Company into the second half of 2019, Acceleron is well-positioned for both near- and long-term growth."

SECOND QUARTER 2016 HIGHLIGHTS

Development Programs

Hematology

• Updated results from ongoing luspatercept Phase 2 clinical trial in myelodysplastic syndromes (MDS) presented at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) confirm activity and durability of response. Results highlighted in an oral presentation showed that 51% (25/49) of patients with lower-risk MDS treated with luspatercept achieved increased hemoglobin levels, and 35% (14/40) of patients achieved transfusion independence in the 3-month base study. In the ongoing extension study, 81% (26/32) of patients had increased hemoglobin levels and 50% (11/22) achieved transfusion independence with luspatercept treatment. Data was presented showing hemoglobin increases being sustained for 15 months.

• Updated results from ongoing luspatercept Phase 2 clinical trial in beta-thalassemia also presented at EHA (Free EHA Whitepaper). Results presented showed that 67% (20/30) of patients achieved at least a 33% reduction in transfusion burden, and 36% (8/22) of patients achieved a hemoglobin increase of at least 1.5 g/dL in the luspatercept 3-month base study. In the ongoing long-term extension study, 83% (20/24) of patients achieved at least a 33% reduction in transfusion burden and 56% (15/27) of patients achieved a hemoglobin increase of at least 1.5 g/dL. Data was presented showing hemoglobin increases being sustained for 12 months.

• Enrollment is ongoing to explore luspatercept activity in low- or intermediate-risk MDS patients who are erythropoiesis-stimulating agent (ESA) naïve or ring sideroblast negative (RS-). Patients who are eligible to be treated with an ESA but have not yet received such treatment or who are RS-, are being enrolled in a Phase 2 clinical trial. Acceleron plans to present the initial data from these patients by the end of the year.

Musculoskeletal Diseases

• Results from Phase 1 study of ACE-083 in healthy volunteers were presented at the International Congress on Neuromuscular Diseases. Results demonstrated that ACE-083 produced statistically significant, dose-dependent increases in muscle volume, assessed by magnetic resonance imaging (MRI), of the tibialis anterior muscle. At the highest dose level, ACE-083 generated a mean increase in muscle volume of 8.9% (p<0.001 vs placebo).

• Progress continues toward the initiation of a Phase 2 trial with ACE-083 in facioscapulohumeral muscular dystrophy (FSHD), a neuromuscular disorder, in the second half of 2016.

Nephrology

• Acceleron and Celgene expect to provide a development plan update for sotatercept in the second half of 2016. The companies met with health authorities in the first half of the year as part of assessing the potential future investigation of sotatercept in patients with pre-dialysis chronic kidney disease (CKD) due to diabetic nephropathy.

Oncology

• Enrollment continues in Part 2 of the Phase 2 DART study, a randomized, double-blind study of dalantercept plus axitinib, compared to placebo plus 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 data potentially available by the end of the year.

Preclinical Research

• 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 the first half of 2017.

Other Corporate Updates

• Thomas McCourt appointed to Acceleron’s board of directors. Mr. McCourt brings 30 years of experience building commercial strategies and capabilities across multiple companies for new therapies. He serves as chief commercial officer and senior vice president of marketing and sales of Ironwood Pharmaceuticals. Prior to joining Ironwood in 2009, Mr. McCourt held commercial roles at Amgen, Novartis AG and Astra Merck Inc.
Financial Results

• Cash position – Cash, cash equivalents and investments as of June 30, 2016 were $262.7 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 second quarter was $3.2 million. License and milestone revenue was $0.1 million. Cost sharing reimbursement revenue from the Company’s Celgene partnership was $3.1 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 second quarter were $22.8 million. This includes R&D expenses of $16.1 million and G&A expenses of $6.7 million.

• Other expense, net – Other expense for the second quarter was $2.4 million and includes a $2.9 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 second quarter ended June 30, 2016 of $22.0 million.