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 |
ACCELERON PHARMA INC. | ||
By: | /s/ John D. Quisel, J.D., Ph.D. | |
John D. Quisel, J.D., Ph.D. | ||
Executive Vice President and Chief Business Officer | ||
August 2, 2018 |
• | The MEDALIST and BELIEVE Phase 3 trials in patients with lower-risk MDS and transfusion-dependent beta-thalassemia, respectively, met all primary and key secondary endpoints. |
• | Data will be submitted to a future medical meeting for presentation in late 2018. |
• | Acceleron and Celgene plan to submit regulatory filings in the United States and Europe in the first half of 2019. |
• | Updated results from the ongoing Phase 2 trials in MDS and beta-thalassemia were presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting and the 23rd Congress of the European Hematology Association (EHA) in June 2018. |
• | The initiation of the COMMANDS Phase 3 trial in patients with lower-risk MDS who are treatment naïve is planned for the third quarter of 2018. |
• | Enrollment and treatment are ongoing in the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia and the Phase 2 trial in MF. |
• | Preliminary results from Part 1 of the ACE-083 Phase 2 trial in patients with CMT disease were highlighted in oral and poster presentations at the 2018 Peripheral Nerve Society (PNS) Annual Meeting in July. |
• | Part 2 of the Phase 2 trial in patients with CMT was recently initiated with preliminary results expected by the end of 2019. |
• | The Company plans to present results from all Part 1 dose cohorts in the FSHD Phase 2 trial in October 2018. |
• | Enrollment and treatment are ongoing in Part 2 of the Phase 2 trial in patients with FSHD and preliminary results are expected in the second half of 2019. |
• | ACE-083 received FDA Fast Track status and Orphan Drug designation in FSHD. |
• | Enrollment and treatment are ongoing in the Phase 1 healthy volunteer trial with preliminary results expected in the first half of 2019. |
• | The Company initiated the PULSAR Phase 2 trial in patients with PAH with preliminary results expected in the first half of 2020. |
• | The Company plans to initiate an exploratory imaging study in Q1 2019 to provide additional understanding of endpoints in anticipation of a potential pivotal trial in the future. |
• | In November 2018, the Company will host a PAH Research and Development Deep Dive event in New York City. |
• | The event will include internal and external expert presentations to discuss disease background, current treatment landscape, key disease pathways including BMP signaling, Acceleron’s clinical development activities, and the latest sotatercept preclinical results. |
• | Robert K. Zeldin, M.D., was appointed Chief Medical Officer (CMO). He brings 20 years of industry experience and joined from Ablynx, where he served as Chief Medical Officer. Prior to Ablynx, Dr. Zeldin served in senior roles at Novartis, Merck, and the U.S. Food & Drug Administration’s Center for Biologics Evaluation and Research. |
• | Janethe Pena, M.D., Ph.D., recently joined us as Vice President of Pulmonary to lead the company’s clinical development efforts in this area. Dr. Pena most recently served as Vice President and Group Head of Pulmonology Clinical Development at Bayer Pharmaceuticals. At Bayer, she was responsible for the pulmonary portfolio, including leading clinical trials with riociguat (Adempas®) in different pulmonary hypertension indications and the overall program life cycle management. |
• | Cash position – Cash, cash equivalents and investments as of June 30, 2018 were $332.3 million. As of December 31, 2017, the Company had cash, cash equivalents and investments of $372.9 million. The Company believes that existing cash, cash equivalents and investments will be sufficient to fund projected operating requirements into 2021. |
• | Revenue – Collaboration revenue for the second quarter was $3.7 million. The revenue is all from Acceleron's partnership with Celgene and is primarily related to expenses incurred by the Company in support of luspatercept. |
• | Costs and expenses – Total costs and expenses for the second quarter were $33.6 million. This includes R&D expenses of $25.9 million and G&A expenses of $7.7 million. |
• | Net loss – The Company's net loss for the second quarter ended June 30, 2018 was $28.9 million. |
June 30, 2018 | December 31, 2017 | ||||||
Cash and cash equivalents | $ | 79,592 | $ | 100,150 | |||
Short and long-term investments | 252,666 | 272,800 | |||||
Other assets | 19,283 | 16,227 | |||||
Total assets | $ | 351,541 | $ | 389,177 | |||
Deferred revenue | $ | — | $ | 3,702 | |||
Warrants to purchase common stock | 1,689 | 2,236 | |||||
Other liabilities | 16,743 | 18,021 | |||||
Total liabilities | 18,432 | 23,960 | |||||
Total stockholders’ equity | 333,109 | 365,217 | |||||
Total liabilities and stockholders’ equity | $ | 351,541 | $ | 389,177 |
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
2018 | 2017 | 2018 | 2017 | ||||||||||||
Revenue: | |||||||||||||||
Collaboration revenue | $ | 3,685 | $ | 3,057 | $ | 6,917 | $ | 6,762 | |||||||
Costs and expenses: | |||||||||||||||
Research and development | 25,933 | 21,598 | 49,363 | 43,327 | |||||||||||
General and administrative | 7,658 | 11,370 | 15,099 | 19,203 | |||||||||||
Total costs and expenses | 33,591 | 32,968 | 64,462 | 62,530 | |||||||||||
Loss from operations | (29,906 | ) | (29,911 | ) | (57,545 | ) | (55,768 | ) | |||||||
Total other income, net | 979 | 248 | 2,410 | 705 | |||||||||||
Loss before income taxes | (28,927 | ) | (29,663 | ) | (55,135 | ) | (55,063 | ) | |||||||
Income tax provision | (11 | ) | (6 | ) | (21 | ) | (12 | ) | |||||||
Net loss applicable to common stockholders- basic and diluted | $ | (28,938 | ) | $ | (29,669 | ) | $ | (55,156 | ) | $ | (55,075 | ) | |||
Net loss per share applicable to common stockholders- basic and diluted | $ | (0.63 | ) | $ | (0.77 | ) | $ | (1.21 | ) | $ | (1.43 | ) | |||
Weighted-average number of common shares used in computing net loss per share applicable to common stockholders | 45,789 | 38,631 | 45,654 | 38,515 |
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