Delaware (State or other jurisdiction of incorporation) | 001-36065 (Commission File Number) | 27-0072226 (I.R.S. Employer Identification Number) | ||
128 Sidney Street Cambridge, MA (Address of principal executive offices) | 02139 (Zip Code) |
ACCELERON PHARMA INC. | ||
By: | /s/ John Quisel | |
John Quisel | ||
Senior Vice President and General Counsel | ||
Date: February 25, 2016 |
• | 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 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. |
• | 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 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. |
• | 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. |
• | 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. |
• | 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) Annual Meeting and ASCO 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. |
• | 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. |
• | 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. |
• | 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. |
• | 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). |
• | Update long-term treatment results at major medical conferences. |
• | Present Phase 1 data from two new cohorts (Mid 2016). |
• | Initiate Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD) patients (H2 2016). |
• | Submit IND for first-in-human study for ACE-2494 (YE 2016). |
• | Present preliminary Part 2 DART study results (dalantercept in combination with axitinib) (YE 2016). |
• | Provide an update on development strategy (H2 2016). |
• | 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. |
December 31, 2015 | December 31, 2014 | ||||||
Cash and cash equivalents | $ | 27,783 | $ | 176,460 | |||
Short and long-term investments | 108,198 | — | |||||
Other assets | 10,356 | 9,836 | |||||
Total assets | $ | 146,337 | $ | 186,296 | |||
Deferred revenue | $ | 4,794 | $ | 5,978 | |||
Warrants to purchase common stock | 17,187 | 14,124 | |||||
Other liabilities | 15,093 | 9,909 | |||||
Total liabilities | 37,074 | 30,011 | |||||
Total stockholders’ equity | 109,263 | 156,285 | |||||
Total liabilities and stockholders’ equity | $ | 146,337 | $ | 186,296 |
Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
2015 | 2014 | 2015 | 2014 | ||||||||||||
Revenue: | |||||||||||||||
Collaboration revenue | $ | 3,804 | $ | 3,739 | $ | 18,097 | $ | 14,632 | |||||||
Costs and expenses: | |||||||||||||||
Research and development | 16,146 | 14,579 | 58,404 | 50,897 | |||||||||||
Litigation settlement | — | — | — | 5,000 | |||||||||||
General and administrative | 5,773 | 3,714 | 20,572 | 14,199 | |||||||||||
Total costs and expenses | 21,919 | 18,293 | 78,976 | 70,096 | |||||||||||
Loss from operations | (18,115 | ) | (14,554 | ) | (60,879 | ) | (55,464 | ) | |||||||
Other (expense) income, net | (8,967 | ) | (3,063 | ) | (3,015 | ) | 4,205 | ||||||||
Net loss | $ | (27,082 | ) | $ | (17,617 | ) | $ | (63,894 | ) | $ | (51,259 | ) | |||
Net unrealized holding (losses) on short-term and long-term investments during the period | (199 | ) | — | (220 | ) | — | |||||||||
Comprehensive loss | $ | (27,281 | ) | $ | (17,617 | ) | $ | (64,114 | ) | $ | (51,259 | ) | |||
Net loss per share applicable to common stockholders-basic and diluted | $ | (0.81 | ) | $ | (0.55 | ) | $ | (1.92 | ) | $ | (1.63 | ) | |||
Weighted-average number of common shares used in computing net loss per share applicable to common stockholders-basic and diluted | 33,268 | 32,293 | 33,303 | 31,515 |
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