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 August 3, 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 | ||
August 3, 2017 |
Exhibit Number | Description of Exhibit | |
99.1 | Press release of Acceleron Pharma Inc. dated August 3, 2017 |
• | Completed patient enrollment in the MEDALIST and BELIEVE Phase 3 clinical trials. The MEDALIST Phase 3 study enrolled 229 patients to evaluate the efficacy and safety of luspatercept in patients with anemia due to lower-risk MDS with ring sideroblasts who require regular RBC transfusions. The BELIEVE Phase 3 study enrolled 336 patients to evaluate the efficacy and safety of luspatercept in patients with anemia due to beta-thalassemia who require regular RBC transfusions. |
• | Results presented at EHA from an ongoing Phase 2 study in lower-risk MDS show increases in hemoglobin, reductions in RBC transfusion burden and RBC transfusion independence. Patients demonstrated a clinically meaningful increase in hemoglobin for up to 26 months in this ongoing clinical trial. |
• | Results presented at EHA from an ongoing Phase 2 study in beta-thalassemia demonstrate increases in hemoglobin and decreases in RBC transfusion burden sustained for up to 24 months in this ongoing clinical trial. |
• | Acceleron and Celgene continue preparations for additional luspatercept clinical trials in new patient populations, including the COMMANDS Phase 3 trial in first-line, lower-risk MDS, the recently initiated Phase 2 trial in myelofibrosis, and the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia. |
• | Enrollment and treatment are ongoing in Part 1 of the Phase 2 trial in FSHD, one of the most prevalent forms of muscular dystrophy in adults. |
• | Achieved pipeline milestone with first patient treated in the ACE-083 Phase 2 study in patients with CMT disease, one of the most common inherited neurological diseases leading to focal muscle weakness. |
• | The Company plans to initiate a Phase 1 healthy volunteer clinical trial this year with ACE-2494. |
• | Report top-line results from MEDALIST and BELIEVE Phase 3 trials in mid-2018 |
• | Initiate the COMMANDS Phase 3 trial in first-line, lower-risk MDS in early 2018 |
• | Enroll the first myelofibrosis patient in Phase 2 by YE 2017 |
• | Initiate the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia by YE 2017 |
• | Present additional Phase 2 MDS and beta-thalassemia study results at medical conferences in 2017 |
• | Report FSHD Phase 2 results for cohort 1 in Part 1 in late 2017 |
• | Report FSHD Phase 2 results for all dose-escalation cohorts in Part 1 in 2018 |
• | Report CMT Phase 2 results from all dose-escalation cohorts in Part 1 by YE 2018 |
• | Initiate Phase 1 healthy volunteer study in 2017 |
• | Acceleron to host R&D Day on September 19, 2017 in New York City |
• | Cash position – Cash, cash equivalents and investments as of June 30, 2017 were $194.0 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 second quarter was $3.1 million. The revenue is all from our Celgene partnership and is primarily due to cost sharing revenue of $2.9 million related to expenses incurred by the Company in support of our partnered programs. |
• | Costs and expenses – Total costs and expenses for the second quarter were $33.0 million. This includes R&D expenses of $21.6 million and G&A expenses of $11.4 million. G&A expense includes a $3.6 million, one-time, non-cash, charge due to modifications of a former executive officer’s equity awards in connection with his change in employment status announced in May 2017. |
• | Net loss – The Company's net loss for the second quarter ended June 30, 2017 was $29.7 million. |
June 30, 2017 | December 31, 2016 | ||||||
Cash and cash equivalents | $ | 30,726 | $ | 20,950 | |||
Short and long-term investments | 163,312 | 213,432 | |||||
Other assets | 14,425 | 13,265 | |||||
Total assets | $ | 208,463 | $ | 247,647 | |||
Deferred revenue | $ | 3,973 | $ | 4,245 | |||
Warrants to purchase common stock | 1,516 | 1,244 | |||||
Other liabilities | 14,442 | 16,561 | |||||
Total liabilities | 19,931 | 22,050 | |||||
Total stockholders’ equity | 188,532 | 225,597 | |||||
Total liabilities and stockholders’ equity | $ | 208,463 | $ | 247,647 |
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
2017 | 2016 | 2017 | 2016 | ||||||||||||
Revenue: | |||||||||||||||
Collaboration revenue | $ | 3,057 | $ | 3,195 | $ | 6,762 | $ | 21,396 | |||||||
Costs and expenses: | |||||||||||||||
Research and development | 21,598 | 16,138 | 43,327 | 32,390 | |||||||||||
General and administrative | 11,370 | 6,712 | 19,203 | 12,618 | |||||||||||
Total costs and expenses | 32,968 | 22,850 | 62,530 | 45,008 | |||||||||||
Loss from operations | (29,911 | ) | (19,655 | ) | (55,768 | ) | (23,612 | ) | |||||||
Total other income (expense) net | 248 | (2,361 | ) | 705 | 6,656 | ||||||||||
Loss before income taxes | (29,663 | ) | (22,016 | ) | (55,063 | ) | (16,956 | ) | |||||||
Income tax provision | (6 | ) | — | (12 | ) | — | |||||||||
Net loss applicable to common stockholders - basic and diluted | $ | (29,669 | ) | $ | (22,016 | ) | $ | (55,075 | ) | $ | (16,956 | ) | |||
Net loss per share applicable to common stockholders - basic and diluted | $ | (0.77 | ) | $ | (0.59 | ) | $ | (1.43 | ) | $ | (0.46 | ) | |||
Weighted-average number of common shares used in computing net (loss) income per share applicable to common stockholders | 38,631 | 37,272 | 38,515 | 37,092 |
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