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) |
Title of each class | Ticker Symbol(s) | Name of each exchange on which registered |
Common Stock, $0.001 per share | XLRN | The Nasdaq Global Market |
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 | ||
Date: May 9, 2019 |
• | Celgene recently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for luspatercept in patients with MDS- and beta-thalassemia-associated anemia based on the safety and efficacy results of the pivotal Phase 3 studies MEDALIST and BELIEVE. |
• | The Companies expect to announce preliminary topline results from the Phase 2 trial of luspatercept in patients with MF in the second half of 2019. |
• | Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS and the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia, with preliminary results expected from the BEYOND trial in 2020. |
• | Previously presented results from Part 1 of the Phase 2 trial evaluating ACE-083 in patients with FSHD were highlighted in an encore presentation at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in April 2019. |
• | Preliminary results from Part 2 of the Phase 2 trial in patients with FSHD are expected in the second half of 2019. |
• | Previously presented results from Part 1 of the Phase 2 trial evaluating ACE-083 in patients with CMT will be highlighted in a platform presentation at the American Academy of Neurology (AAN) 71st Annual Meeting on May 10, 2019. |
• | Enrollment is ongoing in Part 2 of the Phase 2 trial in patients with CMT, with preliminary results expected in the first quarter of 2020. |
• | Enrollment is ongoing in the PULSAR Phase 2 trial in patients with PAH, with topline results expected in the first half of 2020. |
• | Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020. |
• | A preclinical abstract of sotatercept in PAH has been accepted for presentation at the American Thoracic Society (ATS) 2019 International Conference on May 21, 2019. |
• | Cash Position – Cash, cash equivalents and investments as of March 31, 2019 were $513.1 million. As of December 31, 2018, the Company had cash, cash equivalents and investments of $291.3 million. Based on the Company's current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from luspatercept sales. |
• | Revenue – Collaboration revenue for the first quarter was $2.8 million. The revenue is all from the Company'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 first quarter were $43.6 million. This includes R&D expenses of $32.8 million and G&A expenses of $10.8 million. |
• | Net loss – The Company's net loss for the first quarter ended March 31, 2019 was $38.1 million. |
March 31, 2019 | December 31, 2018 | ||||||
Cash and cash equivalents | $ | 214,490 | $ | 144,052 | |||
Short and long-term investments | 298,615 | 147,260 | |||||
Operating lease assets | 27,817 | — | |||||
Other assets | 19,408 | 23,509 | |||||
Total assets | $ | 560,330 | $ | 314,821 | |||
Short-term and long-term operating lease liabilities | $ | 30,455 | $ | — | |||
Warrants to purchase common stock | 1,607 | 1,491 | |||||
Other liabilities | 17,733 | 21,292 | |||||
Total liabilities | 49,795 | 22,784 | |||||
Total stockholders’ equity | 510,535 | 292,037 | |||||
Total liabilities and stockholders’ equity | $ | 560,330 | $ | 314,821 |
Three Months Ended March 31, | |||||||
2019 | 2018 | ||||||
Revenue: | |||||||
Collaboration revenue | $ | 2,780 | $ | 3,232 | |||
Costs and expenses: | |||||||
Research and development | 32,771 | 23,431 | |||||
General and administrative | 10,814 | 7,441 | |||||
Total costs and expenses | 43,585 | 30,872 | |||||
Loss from operations | (40,805 | ) | (27,640 | ) | |||
Total other income, net | 2,772 | 1,431 | |||||
Loss before income taxes | (38,033 | ) | (26,209 | ) | |||
Income tax provision | (20 | ) | (10 | ) | |||
Net loss applicable to common stockholders- basic and diluted | $ | (38,053 | ) | $ | (26,219 | ) | |
Net loss per share applicable to common stockholders- basic and diluted | $ | (0.74 | ) | $ | (0.58 | ) | |
Weighted-average number of common shares used in computing net loss per share applicable to common stockholders | 51,126 | 45,516 |
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