On February 25, 2016 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 fourth quarter and year ended December 31, 2015 (Press release, Acceleron Pharma, FEB 25, 2016, View Source [SID:1234509189]).
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"We made significant progress over the past year in advancing our pipeline, as marked by successful clinical results across our programs and the recent launch of Phase 3 programs in MDS and beta-thalassemia with our partner Celgene," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "At the start of 2016, we raised $150 million in gross proceeds from an equity financing which will be used to fund our wholly owned programs, including our compounds for muscle diseases. Our locally acting muscle agent, ACE-083, showed unprecedented results in increasing muscle volume, and we plan to present new Phase 1 data later this year and initiate a Phase 2 trial in FSHD, a form of muscular dystrophy."
Added Dr. Knopf, "In late 2015, we introduced ACE-2494, a systemic muscle therapeutic created from our new IntelliTrap discovery platform. IntelliTrap is a powerful discovery engine, and we expect it to enable us to introduce a novel therapeutic into the clinic every 12 to 18 months."
2015 HIGHLIGHTS AND CURRENT UPDATES
DEVELOPMENT PROGRAMS
Hematology
Luspatercept in myelodysplastic syndromes (MDS)
Luspatercept is a protein therapeutic that increases hemoglobin levels and is being developed to help patients reduce or eliminate their need for red blood cell (RBC) transfusions
Initiated Phase 3 MEDALIST study in MDS with partner Celgene. MEDALIST is a 210-patient, global, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept versus placebo in subjects with anemia due to very low, low, or intermediate-risk MDS with ring sideroblasts (≥ 15%) who require red blood cell transfusions.
Presented data at ASH (Free ASH Whitepaper) 2015 annual meeting from ongoing Phase 2 extension study showing that 50% of MDS patients achieved transfusion independence and 69% of patients achieved sustained increases in hemoglobin levels.
Expanded the Phase 2 MDS study with additional cohorts in lower risk MDS patients that are either erythropoietin-stimulating agent (ESA) treatment naïve or ring sideroblast negative.
Received FDA Fast Track Designation.
Luspatercept in beta-thalassemia
Initiated Phase 3 BELIEVE study in beta-thalassemia with partner Celgene. BELIEVE is a 300-patient, global, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept versus placebo in adults who require regular red blood cell transfusions due to beta-thalassemia.
Presented data at ASH (Free ASH Whitepaper) 2015 annual meeting from ongoing Phase 2 studies showing luspatercept reduced transfusion burden, improved health-related quality of life measures, had beneficial effects on liver iron concentration and demonstrated a favorable safety profile.
Received FDA Fast Track Designation.
Muscle Diseases
ACE-083
Protein therapeutic designed to increase muscle mass and strength in the muscles in which it is administered
First-in-human Phase 1 study results showed an unprecedented 14.5% mean increase in muscle volume in the injected rectus femoris muscle of the quadriceps. The data were presented in the Late Breaking Clinical Trials Session of the 8th International Conference on Cachexia, Sarcopenia, and Muscle Wasting in December 2015.
Preclinical data in mice showed that ACE-083 produced significant increases in muscle mass 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. The data were presented at the 20th International Annual Congress of the World Muscle Society in October 2015.
ACE-2494
Systemic muscle therapeutic designed to increase muscle mass and strength in a range of muscle diseases
Introduced ACE-2494, Acceleron’s first IntelliTrap molecule. Preclinical data in mice presented at the 2015 World Muscle Society Congress showed that after 4 weeks of treatment, ACE-2494 generated substantial dose-dependent mean increases in muscle mass: 41% in rectus femoris, 53% in gastrocnemius, and 87% in pectoralis.
Oncology
Dalantercept in renal cell carcinoma (RCC)
Protein therapeutic that inhibits angiogenesis and is being developed in combination with approved VEGF-based anti-angiogenesis compounds to improve patient outcomes.
Enrollment is ongoing 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.
Results from Part 1 of the DART study with dalantercept plus axitinib demonstrated a median progression free survival of 8.3 months across all dose levels tested versus the historic control of 4.8 months for axitinib alone. The data were presented at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and ASCO (Free ASCO Whitepaper) 2015 Genitourinary Cancers Symposium.
Received FDA Fast Track Designation for dalantercept in combination with axitinib for the treatment of patients with advanced RCC following treatment with one anti-angiogenic agent.
Nephrology
Sotatercept in chronic kidney disease
Protein therapeutic that has effects on fibrosis, vascular calcification, bone mineral density and red blood cell (RBC) levels
Presented preliminary data from ongoing Phase 2a clinical studies of sotatercept in End-Stage Kidney Disease patients, including effects on hemoglobin, vascular calcification, bone mineral density and safety and tolerability at the American Society of Nephrology Kidney Week in October 2015.
Acceleron and Celgene assessing the opportunity for the development of sotatercept in the pre-dialysis chronic kidney disease (CKD) setting.
RESEARCH AND DEVELOPMENT
Introduced the Company’s IntelliTrap platform for discovery of selective and novel compounds targeting the transforming growth factor-beta superfamily of proteins.
Identified ACE-2494 as the first compound to be developed from the platform and is expected to be Acceleron’s fifth internally discovered therapeutic to enter the clinic.
Acceleron plans to have a new internally discovered compound enter the clinic every 12 to 18 months.
CORPORATE UPDATES
Raised $150 million in gross proceeds in an underwritten public offering of common stock in January 2016.
Hosted first Research & Development Day in October 2015. Company executives and a panel of outside experts briefed the investment community on Acceleron’s clinical programs in MDS, beta-thalassemia, cancer and muscular dystrophies.
UPCOMING 2016 PROGRAM MILESTONES AND EVENTS
We anticipate the following milestones and events in 2016:
Hematology
Luspatercept in MDS
Will update long-term treatment results at major medical conferences.
Initial data on ring sideroblast negative and ESA treatment naïve (front-line) patients (YE 2016).
Luspatercept in beta-thalassemia
Update long-term treatment results at major medical conferences.
Muscle Diseases
ACE-083
Present Phase 1 data from two new cohorts (Mid 2016).
Initiate Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD) patients (H2 2016).
ACE-2494
Submit IND for first-in-human study for ACE-2494 (YE 2016).
Oncology
Dalantercept in RCC
Present preliminary Part 2 DART study results (dalantercept in combination with axitinib) (YE 2016).
Nephrology
Sotatercept in CKD
Provide an update on development strategy (H2 2016).
Financial Results
Cash Position – Cash, cash equivalents and investments were $136.0 million as of December 31, 2015. Net cash used in operating activities in 2015 was $44.2 million. We believe that our existing cash, cash equivalents and investments, including the net proceeds of $140.4 million from our January 2016 offering, will be sufficient to fund our projected operating requirements into the second half of 2019.
Revenue – Collaboration revenue for the year was $18.1 million. This includes license and milestone amortization of $1.2 million and cost sharing reimbursement revenue from our Celgene partnership of $16.9 million related to expenses incurred by the Company in support of our partnered programs.
Costs and expenses – Total costs and expenses for the year were $79.0 million. This includes R&D expenses of $58.4 million and G&A expenses of $20.6 million.
Net Loss – The Company’s net loss for the year ended December 31, 2015 was $63.9 million.