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) |
ACCELERON PHARMA INC. | ||
By: | /s/ John Quisel | |
John Quisel | ||
Senior Vice President and General Counsel | ||
Date: August 12, 2014 |
• | Positive interim data from phase 2 study of dalantercept in combination with axitinib in renal cell carcinoma (RCC) presented at ASCO – Acceleron presented encouraging safety and efficacy data from the dose escalation stage of the ongoing phase 2 clinical trial at the annual meeting of the American Society of Clinical Oncology (ASCO) in June. |
• | Initiation of the randomized, placebo-controlled stage of the ongoing Phase 2 study of dalantercept in combination with axitinib in RCC patients – The Company initiated the randomized, placebo-controlled study (part 2) of dalantercept in combination with axitinib versus placebo plus axitinib. The primary endpoint of part 2 of the trial will be progression-free survival. |
• | Initiation of the Phase 1b study of dalantercept in hepatocellular carcinoma (HCC) – Acceleron initiated a clinical trial of dalantercept in combination with sorafenib, a VEGF antagonist approved for treatment of advanced HCC, in first-line patients. The endpoint for the study is safety, tolerability and a recommended dose for a phase 2b study. Similar to the therapeutic strategy being used in the renal cell carcinoma study, we believe the combination of a VEGFR TKI plus dalantercept will more fully inhibit angiogenesis than just a VEGF antagonist and thereby may provide a better clinical outcome for patients. |
• | Positive interim data showing increases in hemoglobin or reductions in transfusion burden from the sotatercept and ACE-536 clinical trials in beta-thalassemia and myelodysplastic syndromes (MDS) presented at EHA – Acceleron and Celgene investigators gave three oral presentations highlighting encouraging safety and efficacy data from the sotatercept and ACE-536 clinical trials in beta thalassemia patients and the ACE-536 clinical trial in MDS patients at the annual meeting of the European Hematology Association (EHA) in June. |
• | Early signs of activity from phase 2a study of sotatercept in end-stage renal disease patients presented at two clinical meetings – An interim analysis from an ongoing Phase 2a clinical trial of sotatercept in end-stage renal disease patients on hemodialysis indicated that sotatercept produced dose dependent increases in hemoglobin. These data were presented at the National Kidney Foundation Spring Clinical Meeting and at the European Renal Association – European Dialysis Transplantation Association annual meeting. |
• | ACE-536 granted orphan designation by EMA – The European Medicines Association (EMA) granted orphan designation to ACE-536 for the treatment of beta-thalassemia patients and myelodysplastic syndromes patients. Collectively, FDA orphan designation has been granted for both sotatercept and |
• | ACE-536 nonproprietary name is luspatercept – The United States Adopted Names (USAN) Council has approved the name “luspatercept” for ACE-536 and we expect the World Health Organization’s International Nonproprietary Name (INN) group to also approve the name in the near future. |
• | Positive preclinical data with ACE-536 in a murine model of sickle cell disease presented at EHA – Acceleron scientists presented data in a mouse model of sickle cell disease. Following one month of treatment, RAP-536 (murine version of ACE-536) significantly increased red blood cell (RBC) number and hemoglobin compared to placebo treatment in sickle cell disease (SCD) mice. Other clinically important parameters improved, such as a decrease in number of reticulocytes and irreversibly sickled cells. Sickle cell disease, like beta-thalassemia, is a hemoglobinopathy, a category of red blood cell diseases, which is among the most common inherited diseases. |
• | Antibody discovery collaboration with Adimab – Acceleron continues to leverage its unique understanding of the importance and therapeutic potential of the TGF-β superfamily to create new product candidates. To this end, Acceleron and Adimab have signed a multi-target antibody discovery collaboration against targets selected by Acceleron. Acceleron has the worldwide rights to develop and commercialize antibodies resulting from the collaboration. |
• | Interim data from sotatercept phase 2a clinical trial in end-stage renal disease patients at ASN – Acceleron’s collaboration partner Celgene intends to present additional interim data at the annual meeting of the American Society of Nephrology (ASN) in November. |
• | Interim data from sotatercept and ACE-536 phase 2 clinical trials in beta-thalassemia and MDS patients at ASH – Acceleron and Celgene intend to present additional interim data at the annual meeting of the American Society of Hematology (ASH) in December for these programs in patients with beta-thalassemia and MDS. |
• | Initiation of a phase 1 clinical trial with ACE-083 – Acceleron is conducting development activities in preparation for the submission of an Investigational New Drug (IND) application for its clinical candidate ACE-083, a locally acting protein therapeutic designed to increase muscle mass and strength. Acceleron plans to initiate a phase 1 clinical trial in the second half of 2014. |
• | Cash Position – Cash and cash equivalents as of June 30, 2014 were $204.3 million. Acceleron expects that its cash and cash equivalents balance as of June 30, 2014, will be sufficient to fund the Company’s operations into the second half of 2017. |
• | Revenue - Collaboration revenue was $4.1 million for the second quarter of 2014 compared to $26.4 million for the comparable period in 2013. The decrease for the second quarter of 2014 compared to the comparable period in 2013 was driven by recognition of $22.8 million of deferred revenue resulting from termination of the ACE-031 collaboration agreement in June 2013. |
• | R&D Expenses – Research and development expenses were $12.7 million in the second quarter of 2014, compared to $8.9 million in the same period in 2013. This $3.8 million increase was primarily due to increases in direct program expenses. |
• | Litigation settlement – In the three months ended June 30, 2014 we recorded $5.0 million as a condition of a settlement with the Salk Institute for Biological Studies compared to zero in the same period in 2013. |
• | G&A Expenses – General and administrative expenses were $3.7 million in the second quarter of 2014, compared to $3.4 million for the same period in 2013. |
• | Net Loss – Our net loss was $16.6 million for the second quarter of 2014, compared to a net gain of $13.1 million for the comparable periods in 2013. The second quarter of 2014 results were affected by a |
June 30, 2014 | December 31, 2013 | |||||||
Cash and cash equivalents | $ | 204,250 | $ | 113,163 | ||||
Other assets | 10,394 | 10,569 | ||||||
Total assets | $ | 214,644 | $ | 123,732 | ||||
Accrued expenses | 11,475 | 6,927 | ||||||
Notes payable, net of discount | — | 16,868 | ||||||
Deferred revenue | 6,629 | 7,651 | ||||||
Warrants to purchase common stock | 21,512 | 30,753 | ||||||
Other liabilities | 5,201 | 3,721 | ||||||
Total liabilities | 44,817 | 65,920 | ||||||
Total stockholders’ equity | 169,827 | 57,812 | ||||||
Total liabilities and stockholders’ equity | $ | 214,644 | $ | 123,732 |
Three Months Ended June 30, | |||||||||
2014 | 2013 | ||||||||
Collaboration revenue | 4,078 | 26,427 | |||||||
Costs and expenses: | |||||||||
Research and development | 12,677 | 8,911 | |||||||
Litigation settlement | 5,000 | — | |||||||
General and administrative | 3,712 | 3,365 | |||||||
Total costs and expenses | 21,389 | 12,276 | |||||||
(Loss) income from operations | (17,311 | ) | 14,151 | ||||||
Other income (expense), net | 761 | (1,073 | ) | ||||||
Net (loss) income | $ | (16,550 | ) | $ | 13,078 | ||||
Accretion of dividends, interest, redemption value and issuance costs on redeemable convertible preferred stock | — | (6,843 | ) | ||||||
Net (loss) income applicable to participating securities | — | (5,492 | ) | ||||||
Net (loss) income applicable to common stockholders—basic | $ | (16,550 | ) | $ | 743 | ||||
Net (loss) income per share applicable to common stockholders: | |||||||||
Net (loss) income | $ | (16,550 | ) | $ | 13,078 | ||||
Accretion of dividends, interest, redemption value and issuance costs on redeemable convertible preferred stock | — | (6,843 | ) | ||||||
Net (loss) income applicable to participating securities | — | (5,000 | ) | ||||||
Net (loss) income applicable to common stockholders—diluted | $ | (16,550 | ) | $ | 1,235 | ||||
Net (loss) income per share applicable to common stockholders— basic and diluted: | |||||||||
Basic | $ | (0.52 | ) | $ | 0.30 | ||||
Diluted | $ | (0.52 | ) | $ | 0.28 | ||||
Weighted-average number of common shares used in computing net (loss) income per share applicable to common stockholders: | |||||||||
Basic | 31,552 | 2,438 | |||||||
Diluted | 31,552 | 4,457 |
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