On November 4, 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, reported a corporate update and reported financial results for the third quarter ended September 30, 2015 (Press release, Acceleron Pharma, NOV 4, 2015, View Source [SID:1234507937]).
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"We made significant progress across our clinical pipeline in the quarter and are poised, with our partner Celgene, to launch two pivotal Phase 3 programs by year-end," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "We also revealed new clinical data showing unprecedented results with our locally delivered muscle agent, ACE-083. Finally, our discovery team continues to innovate with the introduction of our new IntelliTrap platform, to discover selective and novel compounds targeting the transforming growth factor-beta superfamily of proteins."
Recent Highlights and Current Updates
Development Programs
Hematology
Acceleron and Celgene plan to initiate Phase 3 studies with luspatercept in beta-thalassemia, the BELIEVE trial, and myelodysplastic syndromes (MDS), the MEDALIST trial, by the end of 2015.
Completed enrollment and treatment in the luspatercept Phase 2 dose escalation and expansion studies in MDS and beta-thalassemia.
Treatment continues in Phase 2 long-term extension studies with luspatercept in beta- thalassemia and MDS.
Amended and expanded Phase 2 MDS study. Additional study cohorts have been added to evaluate the effects of luspatercept in lower risk MDS patients that are either erythropoietin-stimulating agent (ESA) treatment naïve or ring sideroblast negative.
Muscle Diseases
Announced initial Phase 1 data on ACE-083, Acceleron’s therapeutic candidate designed to increase muscle mass and strength when delivered locally to targeted muscle groups. Acceleron reported an unprecedented 14.5% mean increase in muscle volume in the injected muscle of the quadriceps. The Company plans to begin a Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD) in mid-2016.
Presented preclinical data on ACE-083 at the 20th Annual International Congress of the World Muscle Society. ACE-083 produced significant increases in muscle mass of approximately 75% in the injected muscle with no observed effect on either the uninjected contralateral muscle or on whole body mass. Increases in muscle mass were associated with a significant increase in muscle force and power of approximately 40%.
Introduced ACE-2494, Acceleron’s first IntelliTrap molecule, designed for systemic administration to increase muscle mass and strength. In mice, ACE-2494 generated dose dependent increases in muscle mass at a magnitude comparable to the powerful effects of soluble ActRIIB fusion proteins. After 4 weeks of treatment, ACE-2494 (10 mg/kg twice weekly) generated increases in muscle mass ranging from 41% to 87% across different muscle groups. The Company expects to begin clinical studies with ACE-2494 by the end of 2016.
Oncology
FDA granted Fast Track Designation for dalantercept in combination with axitinib for the treatment of patients with advanced renal cell carcinoma (RCC), following treatment with an anti-angiogenic agent.
Presented RCC Phase 2 data showing that the combination of dalantercept and axitinib generated encouraging, progression-free survival after one year. The Company reported the 12-month Kaplan Meier estimate for a progression-free survival rate of 39% for the combination of dalantercept plus axitinib in patients with advanced RCC in Part 1 of the Phase 2 DART study. This compares favorably to the 12-month PFS rate of 25% with axitinib alone.
Nephrology
Acceleron and Celgene will refocus the development of sotatercept in the pre-dialysis chronic kidney disease setting. The Companies plan to meet with regulators in the first half of 2016 to discuss study designs.
Research and Development
Acceleron introduced its new IntelliTrap discovery platform. The IntelliTrap platform is designed to generate dozens of highly selective molecules that optimally target the transforming growth factor-beta superfamily to discover innovative new therapies.
Upcoming Milestones and Events
Initiation of the pivotal Phase 3 clinical trials with luspatercept in MDS and beta-thalassemia is expected by year-end.
Additional luspatercept data from ongoing Phase 2 MDS and beta-thalassemia studies will be presented at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), December 5-8, 2015 in Orlando, Florida.
ACE-083 Phase 1 clinical data will be presented at the 8th International Conference on Cachexia, Sarcopenia and Muscle Wasting, December 4-6, 2015, in Paris, France.
Clinical and preclinical data will be presented at the American Society of Nephrology Kidney Week, November 3-8, 2015, in San Diego, CA.
Financial Results
Cash Position – Cash, cash equivalents and investments as of September 30, 2015 were $148.1 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 September 30, 2015 will be sufficient to fund the Company’s operations into the second half of 2017.