Acceleron Pharma Inc., a clinical stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Its therapeutic candidates include luspatercept, which is in Phase III clinical trials for the treatment of myelodysplastic syndromes and beta-thalassemia; Phase II clinical trials for the treatment of patients with transfusion-dependent beta-thalassemia and myelofibrosis. The company is also developing and sotatercept for the treatment of patients with pulmonary arterial hypertension. In addition, the company is developing ACE-083, a neuromuscular candidate that is in Phase II clinical trials for the treatment of patients with facioscapulohumeral dystrophy and Charcot-Marie-Tooth disease; and ACE-2494, which is in Phase I clinical trials for the treatment of systemic muscle disorders. It has a collaboration, license, and option agreement with Celgene Corporation. The company was formerly known as Phoenix Pharma, Inc. Acceleron Pharma Inc. was founded in 2003 and is headquartered in Cambridge, Massachusetts.
(Summary) (Company) (Chart)
(Summary) (Company) (Chart)
| 24 June 2018 Price $37.37 1yr Target $53.92 Analysts 13 Dividend $0.00 Payout Ratio 0.00% 1yr Cap Gain 44.28% Yield 0.00% 1yr Tot Return 44.28% P/E --- PEG --- Beta 1.42 | EPS (ttm) $-2.61 EPS next yr $-1.85 Forward P/E --- EPS next 5yr --- 1yr Price Support --- Market Cap $1.72 Bil Revenues $13.00 Mil Earnings $-109.30 Mil Profit Margin --- Quick Ratio 23.20 Current Ratio 23.20 Debt/Equity 0.00 | 1yr RevGR --- 3yr RevGR --- 5yr RevGR --- 1yr EarnGR --- 3yr EarnGR --- 5yr EarnGR --- 1yr DivGR --- 3yr DivGR --- 5yr DivGR --- ROA -32.40% ROE -34.50% |
Acceleron Pharma is a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases. Their research focuses on key natural regulators of cellular growth and repair, particularly the Transforming Growth Factor-Beta, or TGF-beta, protein superfamily combining their discovery and development expertise, including proprietary knowledge of the TGF-beta superfamily, and their internal protein engineering and manufacturing capabilities, they have generated several innovative therapeutic candidates, all of which encompass novel potential first-in-class mechanisms of action.
Acceleron Pharma has focused and prioritized their research and development activities within three key therapeutic areas: hematologic, neuromuscular and pulmonary. These candidates could have the potential to significantly improve clinical outcomes for patients across these areas of high, unmet need.
Acceleron Pharma has focused and prioritized their research and development activities within three key therapeutic areas: hematologic, neuromuscular and pulmonary. These candidates could have the potential to significantly improve clinical outcomes for patients across these areas of high, unmet need.
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Luspatercept and sotatercept are partnered with Celgene Corporation. Luspatercept is an erythroid maturation agent designed to promote red blood cell production through a novel mechanism, and is being developed to treat chronic anemia and associated complications in myelodysplastic syndromes, or MDS, beta-thalassemia, and myelofibrosis. Celgene is currently conducting two Phase 3 clinical trials with luspatercept; one for the treatment of patients with lower-risk MDS, known as the "MEDALIST" trial, and another for the treatment of patients with beta-thalassemia, also known as the "BELIEVE" trial. Celgene has recently initiated a Phase 2 trial in non-transfusion-dependent beta-thalassemia patients, referred to as the "BEYOND" trial.
The Company further expects Celgene to initiate a Phase 3 clinical trial, the "COMMANDS" trial, in first-line, lower-risk MDS patients in the first half of 2018. Enrollment is also currently ongoing in a Phase 2 clinical trial for the treatment of patients with myelofibrosis, a rare bone marrow disorder. If luspatercept receives regulatory approval for each of these indications in the United States and Europe, the Company believes that there is an aggregate sales opportunity in excess of $2 billion.
For sotatercept, the Company announced in September 2017 that Celgene granted them the rights to fund, develop, and lead the global commercialization of sotatercept in pulmonary hypertension, including pulmonary arterial hypertension, or PAH. PAH is a rare and chronic, rapidly progressing disorder characterized by the constriction of small pulmonary arteries, resulting in abnormally high blood pressure in the pulmonary arteries. If sotatercept is commercialized to treat PAH Celgene will be eligible to receive a royalty in the low 20% range on global net sales. In certain circumstances Celgene may recognize revenue related to the commercialization of sotatercept in PAH, and in this scenario the Company will be eligible to receive a royalty from Celgene such that the economic position of the parties is equivalent to the scenario in which Acceleron Pharma recognizes such revenue. The Company expects to initiate a Phase 2 clinical trial for the treatment of patients with PAH in the first half of 2018.
For luspatercept and, outside of pulmonary hypertension, sotatercept, Celgene is responsible for paying 100% of the development costs for all clinical trials. Acceleron Pharma will receive a maximum of $545.0 million for the potential development, regulatory and commercial milestone payments. If luspatercept and, outside of pulmonary hypertension, sotatercept are commercialized, the Company is eligible for a royalty on net sales in the low-to-mid 20% range and the Company has a co-promotion right in North America in which Celgene will fund all commercialization costs.
Acceleron Pharma is independently developing a wholly-owned neuromuscular candidate, ACE-083. ACE-083 is designed for the treatment of focal muscle disorders, and the Company is currently conducting Phase 2 clinical trials in patients with facioscapulohumeral dystrophy, or FSHD, as well as in patients with Charcot-Marie-Tooth disease, or CMT.
In January 2018 the Company announced preliminary results for the first two cohorts in part 1 of the Phase 2 clinical trial with ACE-083 in patients with FSHD showing marked increases in the mean total muscle volume of the muscles treated with ACE-083 measured using magnetic resonance imaging, or MRI. Acceleron Pharma expects to initiate part 2 of the ACE-083 FSHD Phase 2 trial during the second quarter of this year, and they expect to report preliminary results from all dose-escalation cohorts of part 1 in our FSHD and CMT Phase 2 clinical trials with ACE-083 in the second half of this year.
In addition to the mid- to late-stage clinical programs, the Company initiated a Phase 1 healthy volunteer study in early 2018 with ACE-2494, the Company's wholly-owned systemic muscle agent from their proprietary platform technology, IntelliTrapTM, and they expect to report initial results from this healthy volunteer study in the first half of 2019. They are also conducting research within their three focused disease areas—hematologic, neuromuscular and pulmonary—in order to identify new therapeutic candidates to advance into clinical trials.
As of 31 Dec 2017 operations have been funded by $105.1 million in equity investments from venture investors, $539.7 million from public investors, $123.7 million in equity investments from the Company's collaboration partners and $273.7 million in upfront payments, milestones, and net research and development payments from those collaboration partners.
Acceleron Pharma estimates that they have spent approximately $89.7 million, $68.6 million, and $58.4 million on research and development for the years ended December 31, 2017, 2016, and 2015, respectively.
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