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 D. Quisel, J.D., Ph.D. | |
John D. Quisel, J.D., Ph.D. | ||
Senior Vice President and General Counsel | ||
Date: March 1, 2017 |
• | Enrollment is robust in the MEDALIST Phase 3 clinical trial, with completion expected in the second half of this year. The MEDALIST Phase 3 study is enrolling 210 patients to evaluate the efficacy and safety of luspatercept in patients with anemia due to lower-risk MDS with ring sideroblasts who require red blood cell (RBC) transfusions. |
• | Results presented at ASH 2016 from an ongoing Phase 2 extension study in lower-risk MDS patients confirmed previously reported results showing durable safety, RBC transfusion independence and erythroid response per the International Working Group’s (IWG HI-E) criteria. Data were presented showing increases in hemoglobin sustained for as long as 22 months with treatment ongoing. |
• | Initial data reported at ASH in first-line lower-risk MDS patients from an ongoing Phase 2 trial demonstrated encouraging response rates. The Company plans to present additional results at medical conferences in 2017. Acceleron and Celgene continue to evaluate a clinical and regulatory strategy for luspatercept in this MDS patient segment. |
• | Enrollment is robust in the BELIEVE Phase 3 trial, with completion expected in the second half of this year. The BELIEVE Phase 3 study is enrolling 300 patients to evaluate the efficacy and safety of luspatercept in patients with anemia due to beta-thalassemia who require regular RBC transfusions. |
• | The Company presented data at ASH 2016 from its ongoing Phase 2 extension study showing clinically meaningful reductions in RBC transfusion burden compared to baseline in transfusion dependent beta-thalassemia patients. Data presented in non-transfusion dependent (NTD) beta-thalassemia patients showed durable increases in hemoglobin sustained for as long as 18 months with treatment ongoing. |
• | Acceleron and Celgene plan to initiate a new Phase 2 trial in non-transfusion dependent beta-thalassemia patients by the end of the year. |
• | Preliminary data reported at ASH 2016 from an ongoing investigator-initiated Phase 2 study with sotatercept showed that 36% (5/14) of evaluable myelofibrosis patients achieved an anemia response, defined as a composite of RBC transfusion independence and hemoglobin response. |
• | Acceleron and Celgene plan to initiate a Phase 2 trial in myelofibrosis with luspatercept by year-end. |
• | First patient was treated in the ACE-083 Phase 2 study in patients with FSHD, one of the most prevalent forms of muscular dystrophy. |
• | Enrollment continues in randomized Part 2 of the Phase 2 DART study of dalantercept in combination with axitinib. The primary endpoint of this trial, progression-free survival (PFS), is an event-driven assessment with preliminary data expected in the second half of 2017. |
• | The Company plans to initiate a Phase 1 healthy volunteer clinical trial this year with ACE-2494. |
• | IND-enabling development work has begun to advance a new therapeutic candidate to the clinic in 2018. |
• | Habib Dable appointed as President and CEO effective December 1, 2016. Mr. Dable joined Acceleron from Bayer AG, where he most recently served as President of U.S. Pharmaceuticals. |
• | Complete enrollment in the MEDALIST Phase 3 trial in 2H 2017 |
• | Complete enrollment in the BELIEVE Phase 3 trial in 2H 2017 |
• | Develop clinical and regulatory strategy in first-line lower-risk MDS in 2017 |
• | Initiate new Phase 2 trials in myelofibrosis and NTD beta-thalassemia by YE 2017 |
• | Additional Phase 2 extension study data to be presented at medical conferences in 2017 |
• | Initial FSHD Phase 2 Part 1 dose-escalation results by late 2017 |
• | Initiate a Phase 2 clinical trial in a second neuromuscular disease in 2017 |
• | Report topline results in the DART Phase 2 trial in renal cell carcinoma in 2H 2017 |
• | Initiate Phase 1 healthy volunteer study in 2017 |
• | Host an investor and analyst research day to discuss ongoing preclinical research and potential future disease areas in 2017 |
• | Cash Position – Cash, cash equivalents and investments were $234.4 million as of December 31, 2016. We believe that our existing cash, cash equivalents and investments will be sufficient to fund our projected operating requirements into the second half of 2019. |
• | Revenue – Collaboration revenue for the year was $27.8 million. The revenue is all from our Celgene partnership. It includes license and milestone revenue of $15.6 million and cost sharing revenue of $12.2 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 $93.9 million. This includes R&D expenses of $68.6 million and G&A expenses of $25.3 million. |
• | Other Income – Other income, net was $9.1 million and includes a $7.3 million, non-cash, gain on marking the Company's common stock warrant liability to market and $1.8 million in interest income. |
• | Net Loss – The Company’s net loss for the year ended December 31, 2016 was $57.0 million. |
December 31, 2016 | December 31, 2015 | ||||||
Cash and cash equivalents | $ | 20,950 | $ | 27,783 | |||
Short and long-term investments | 213,432 | 108,198 | |||||
Other assets | 13,265 | 10,356 | |||||
Total assets | $ | 247,647 | $ | 146,337 | |||
Deferred revenue | $ | 4,245 | $ | 4,794 | |||
Warrants to purchase common stock | 1,244 | 17,187 | |||||
Other liabilities | 16,561 | 15,093 | |||||
Total liabilities | 22,050 | 37,074 | |||||
Total stockholders’ equity | 225,597 | 109,263 | |||||
Total liabilities and stockholders’ equity | $ | 247,647 | $ | 146,337 |
Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
2016 | 2015 | 2016 | 2015 | ||||||||||||
Revenue: | |||||||||||||||
Collaboration revenue | $ | 3,369 | $ | 3,804 | $ | 27,771 | $ | 18,097 | |||||||
Costs and expenses: | |||||||||||||||
Research and development | 19,088 | 16,146 | 68,580 | 58,404 | |||||||||||
General and administrative | 6,267 | 5,773 | 25,297 | 20,572 | |||||||||||
Total costs and expenses | 25,355 | 21,919 | 93,877 | 78,976 | |||||||||||
Loss from operations | (21,986 | ) | (18,115 | ) | (66,106 | ) | (60,879 | ) | |||||||
Other income (expense), net | 2,742 | (8,967 | ) | 9,116 | (3,015 | ) | |||||||||
Loss before income taxes | (19,244 | ) | (27,082 | ) | (56,990 | ) | (63,894 | ) | |||||||
Income tax provision | (44 | ) | — | (24 | ) | — | |||||||||
Net loss | $ | (19,288 | ) | $ | (27,082 | ) | $ | (57,014 | ) | $ | (63,894 | ) | |||
Other comprehensive loss: | |||||||||||||||
Net unrealized holding losses on short-term and long-term investments during the period | (83 | ) | (199 | ) | (205 | ) | (220 | ) | |||||||
Comprehensive loss | $ | (19,371 | ) | $ | (27,281 | ) | $ | (57,219 | ) | $ | (64,114 | ) | |||
Net loss per share - basic and diluted | $ | (0.51 | ) | $ | (0.81 | ) | $ | (1.52 | ) | $ | (1.92 | ) | |||
Weighted-average number of common shares used in computing net loss per share - basic and diluted | 37,914 | 33,268 | 37,430 | 33,303 |
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