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 | ||
October 30, 2018 |
• | The MEDALIST and BELIEVE Phase 3 trial results in patients with lower-risk MDS and transfusion-dependent beta-thalassemia, respectively, are expected to be presented at the 60th ASH Annual Meeting and Exposition in December 2018. |
• | Acceleron and Celgene plan to submit regulatory applications for both MDS and beta-thalassemia in the United States and Europe in the first half of 2019. |
• | The COMMANDS Phase 3 trial in patients with lower-risk MDS who are treatment naïve has been initiated. |
• | Enrollment is ongoing in the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia as well as the Phase 2 trial in patients with MF. |
• | Final results from Part 1 of each Phase 2 trial with ACE-083 in patients with FSHD and CMT, respectively, were presented at the 2018 World Muscle Society (WMS) Annual Meeting. |
• | Enrollment is ongoing in Part 2 of the Phase 2 FSHD trial, with preliminary 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. |
• | Multiple preclinical abstracts for sotatercept in PAH have been accepted for presentation at the American Heart Association Scientific Sessions on November 10-12, 2018. |
• | On November 16, 2018, the Company will host a PAH Research and Development Deep Dive event in New York City. |
• | Enrollment is ongoing in 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 study, called SPECTRA, in Q1 2019 to provide further understanding of sotatercept’s impact on the disease. |
• | Cash position – Cash, cash equivalents and investments as of September 30, 2018 were $319.8 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 third quarter was $3.3 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 third quarter were $33.4 million. This includes R&D expenses of $24.7 million and G&A expenses of $8.7 million. |
• | Net loss – The Company's net loss for the third quarter ended September 30, 2018 was $29.0 million. |
September 30, 2018 | December 31, 2017 | ||||||
Cash and cash equivalents | $ | 112,427 | $ | 100,150 | |||
Short and long-term investments | 207,380 | 272,800 | |||||
Other assets | 18,310 | 16,227 | |||||
Total assets | $ | 338,117 | $ | 389,177 | |||
Deferred revenue | $ | — | $ | 3,702 | |||
Warrants to purchase common stock | 2,031 | 2,236 | |||||
Other liabilities | 16,974 | 18,021 | |||||
Total liabilities | 19,005 | 23,960 | |||||
Total stockholders’ equity | 319,112 | 365,217 | |||||
Total liabilities and stockholders’ equity | $ | 338,117 | $ | 389,177 |
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2018 | 2017 | 2018 | 2017 | ||||||||||||
Revenue: | |||||||||||||||
Collaboration revenue | $ | 3,258 | $ | 3,014 | $ | 10,175 | $ | 9,776 | |||||||
Costs and expenses: | |||||||||||||||
Research and development | 24,667 | 21,059 | 74,027 | 64,387 | |||||||||||
General and administrative | 8,653 | 7,533 | 23,756 | 26,735 | |||||||||||
Total costs and expenses | 33,320 | 28,592 | 97,783 | 91,122 | |||||||||||
Loss from operations | (30,062 | ) | (25,578 | ) | (87,608 | ) | (81,346 | ) | |||||||
Total other income, net | 1,071 | 86 | 3,481 | 791 | |||||||||||
Loss before income taxes | (28,991 | ) | (25,492 | ) | (84,127 | ) | (80,555 | ) | |||||||
Income tax benefit (provision) | 12 | 41 | (9 | ) | 29 | ||||||||||
Net loss applicable to common stockholders- basic and diluted | $ | (28,979 | ) | $ | (25,451 | ) | $ | (84,136 | ) | $ | (80,526 | ) | |||
Net loss per share applicable to common stockholders- basic and diluted | $ | (0.63 | ) | $ | (0.65 | ) | $ | (1.84 | ) | $ | (2.08 | ) | |||
Weighted-average number of common shares used in computing net loss per share applicable to common stockholders | 46,051 | 39,361 | 45,787 | 38,804 |
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