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) |
Exhibit Number | Description of Exhibit | |
99.1 | Press release of Acceleron Pharma Inc. dated May 8, 2017 |
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: May 8, 2017 |
Exhibit Number | Description of Exhibit | |
99.1 | Press release of Acceleron Pharma Inc. dated May 8, 2017 |
• | Enrollment in the MEDALIST Phase 3 clinical trial is expected to complete in the second quarter 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 the 14th International Symposium on MDS from an ongoing Phase 2 study in first-line, lower-risk MDS patients demonstrated positive erythroid response and RBC transfusion independence rates. |
• | Acceleron and Celgene announced plans to initiate a third Phase 3 trial with luspatercept in early 2018. The new Phase 3 trial will evaluate luspatercept treatment versus standard-of-care in the first-line treatment setting for lower-risk MDS patients. |
• | Enrollment in the BELIEVE Phase 3 trial is expected to complete in the second quarter 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. |
• | Enrollment and treatment are ongoing in Part 1 of the ACE-083 Phase 2 study in patients with FSHD, one of the most prevalent forms of muscular dystrophy. |
• | Announced plans to initiate a second Phase 2 clinical trial with ACE-083 in patients with Charcot-Marie-Tooth (CMT) disease, one of the most common inherited neurologic diseases leading to focal muscle weakness, by mid-2017. |
• | The Company plans to initiate a Phase 1 healthy volunteer clinical trial this year with ACE-2494. |
• | Complete enrollment in the MEDALIST Phase 3 trial in Q2 2017 |
• | Complete enrollment in the BELIEVE Phase 3 trial in Q2 2017 |
• | Anticipate top-line data from both Phase 3 trials in mid-2018 |
• | Initiate first-line, lower-risk MDS Phase 3 trial in early 2018 |
• | Initiate Phase 2 trial in myelofibrosis by year-end 2017 |
• | Initiate Phase 2 trial in non-transfusion dependent (NTD) beta-thalassemia by year-end 2017 |
• | Additional Phase 2 results from ongoing trials to be presented at medical conferences in 2017 |
• | Report initial FSHD Phase 2 Part 1 dose-escalation results by late 2017 |
• | Initiate CMT Phase 2 Part 1 dose-escalation trial by mid-2017 |
• | Report top-line results in the DART Phase 2 trial in renal cell carcinoma by Q2 2017 |
• | Initiate Phase 1 healthy volunteer study in 2017 |
• | Host a Research and Development Day to discuss preclinical research and clinical development activities in 2H 2017 |
• | Cash position – Cash, cash equivalents and investments as of March 31, 2017 were $213.2 million. As of December 31, 2016 the Company had cash, cash equivalents and investments of $234.4 million. We believe that existing cash, cash equivalents and investments will be sufficient to fund projected operating requirements into the second half of 2019. |
• | Revenue – Collaboration revenue for the first quarter was $3.7 million. The revenue is all from our Celgene partnership and is primarily due to cost sharing revenue of $3.6 million related to expenses incurred by the Company in support of our partnered programs. |
• | Costs and expenses – Total costs and expenses for the first quarter were $29.6 million. This includes R&D expenses of $21.7 million and G&A expenses of $7.8 million. |
• | Other income, net – Other income, net for the first quarter was $0.5 million primarily due to interest income. |
• | Net loss – The Company's net loss for the first quarter ended March 31, 2017 was $25.4 million. |
March 31, 2017 | December 31, 2016 | ||||||
Cash and cash equivalents | $ | 33,157 | $ | 20,950 | |||
Short and long-term investments | 180,057 | 213,432 | |||||
Other assets | 15,915 | 13,265 | |||||
Total assets | $ | 229,129 | $ | 247,647 | |||
Deferred revenue | $ | 4,108 | $ | 4,245 | |||
Warrants to purchase common stock | 1,289 | 1,244 | |||||
Other liabilities | 15,412 | 16,561 | |||||
Total liabilities | 20,809 | 22,050 | |||||
Total stockholders’ equity | 208,320 | 225,597 | |||||
Total liabilities and stockholders’ equity | $ | 229,129 | $ | 247,647 |
Three Months Ended March 31, | |||||||
2017 | 2016 | ||||||
Revenue: | |||||||
Collaboration revenue | $ | 3,705 | $ | 18,201 | |||
Costs and expenses: | |||||||
Research and development | 21,727 | 16,246 | |||||
General and administrative | 7,836 | 5,911 | |||||
Total costs and expenses | 29,563 | 22,157 | |||||
Loss from operations | (25,858 | ) | (3,956 | ) | |||
Other income, net | 457 | 9,017 | |||||
(Loss) income before income taxes | $ | (25,401 | ) | $ | 5,061 | ||
Income tax provision | (6 | ) | — | ||||
Net (loss) income | $ | (25,407 | ) | $ | 5,061 | ||
Other comprehensive loss: | |||||||
Net unrealized holding gains on short-term and long-term investments during the period | 25 | 245 | |||||
Comprehensive (loss) income | $ | (25,382 | ) | $ | 5,306 | ||
Net (loss) income per share applicable to common stockholders: | |||||||
Basic | $ | (0.66 | ) | $ | 0.14 | ||
Diluted | $ | (0.66 | ) | $ | 0.13 | ||
Weighted-average number of common shares used in computing net (loss) income per share applicable to common stockholders | |||||||
Basic | 38,404 | 36,911 | |||||
Diluted | 38,404 | 38,666 |
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