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 | ||
Date: February 27, 2019 |
• | The MEDALIST and BELIEVE Phase 3 trial results in patients with lower-risk MDS and transfusion-dependent beta-thalassemia, respectively, were presented at the 60th ASH Annual Meeting and Exposition in December 2018. |
• | The MEDALIST and BELIEVE presentations were both selected for presentation during the “Best of ASH” session at the meeting. |
• | Acceleron and Celgene plan to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for luspatercept in patients with anemia related lower-risk MDS and beta-thalassemia in April 2019. |
• | Acceleron and Celgene remain on track to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for luspatercept in patients with anemia related to lower-risk MDS and beta-thalassemia in the first half of 2019. |
• | The ongoing Phase 2 trial of luspatercept in patients with MF has completed target enrollment, with preliminary results expected 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. |
• | Preliminary results from Part 1 of the Phase 2 trials evaluating ACE-083 in patients with FSHD and CMT, were presented at the 2018 World Muscle Society (WMS) Annual Meeting in October 2018. |
• | Part 2 of the Phase 2 FSHD trial has completed patient enrollment, with preliminary topline results expected in the second half of 2019. |
• | Enrollment is ongoing in Part 2 of the Phase 2 CMT trial, with preliminary results expected by the end of 2019. |
• | Enrollment is ongoing in the Phase 1 healthy volunteer trial, with preliminary results expected in the first half of 2019. |
• | Preclinical results from multiple studies of sotatercept in PAH were presented at the American Heart Association Scientific Sessions in November 2018. |
• | Enrollment is ongoing in the PULSAR Phase 2 trial in patients with PAH, with preliminary results expected in the first half of 2020. |
• | The exploratory SPECTRA trial in patients with PAH has been initiated, with preliminary results expected in 2020. |
• | The Company recently raised approximately $264.5 million of gross proceeds in a follow-on offering of common stock. |
• | Cash Position – Cash, cash equivalents and investments as of December 31, 2018 were $291.3 million. As of December 31, 2017, the Company had cash, cash equivalents and investments of $372.9 million. Based on the Company's current operating plan and projections, it believes that current cash, cash equivalents and investments, together with the net proceeds of $248.2 million from its recent |
• | Revenue – Collaboration revenue for the year was $14.0 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 year were $138.4 million. This includes R&D expenses of $103.9 million and G&A expenses of $34.5 million. |
• | Net Loss – The Company’s net loss for the year ended December 31, 2018 was $118.9 million. |
December 31, 2018 | December 31, 2017 | ||||||
Cash and cash equivalents | $ | 144,052 | $ | 100,150 | |||
Short and long-term investments | 147,260 | 272,800 | |||||
Other assets | 23,509 | 16,227 | |||||
Total assets | $ | 314,821 | $ | 389,177 | |||
Deferred revenue | $ | — | $ | 3,703 | |||
Warrants to purchase common stock | 1,491 | 2,236 | |||||
Other liabilities | 21,293 | 18,021 | |||||
Total liabilities | 22,784 | 23,960 | |||||
Total stockholders’ equity | 292,037 | 365,217 | |||||
Total liabilities and stockholders’ equity | $ | 314,821 | $ | 389,177 |
Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
2018 | 2017 | 2018 | 2017 | ||||||||||||
Revenue: | |||||||||||||||
Collaboration revenue | $ | 3,816 | $ | 3,705 | $ | 13,991 | $ | 13,481 | |||||||
Costs and expenses: | |||||||||||||||
Research and development | 29,867 | 25,339 | 103,902 | 89,726 | |||||||||||
General and administrative | 10,755 | 7,002 | 34,503 | 33,738 | |||||||||||
Total costs and expenses | 40,622 | 32,341 | 138,405 | 123,464 | |||||||||||
Loss from operations | (36,806 | ) | (28,636 | ) | (124,414 | ) | (109,983 | ) | |||||||
Total other income, net | 2,036 | 770 | 5,516 | 1,561 | |||||||||||
Loss before income taxes | (34,770 | ) | (27,866 | ) | (118,898 | ) | (108,422 | ) | |||||||
Income tax benefit (provision) | 36 | (60 | ) | 27 | (32 | ) | |||||||||
Net loss | $ | (34,734 | ) | $ | (27,926 | ) | $ | (118,871 | ) | $ | (108,454 | ) | |||
Net loss per share - basic and diluted | $ | (0.75 | ) | $ | (0.62 | ) | $ | (2.59 | ) | $ | (2.68 | ) | |||
Weighted-average number of common shares used in computing net loss per share | 46,227 | 45,217 | 45,898 | 40,420 |
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