Acceleron Pharma Reports Third Quarter 2017 Operational and Financial Results
-Presented vision and strategic focus in hematological, neuromuscular, and pulmonary diseases at September R&D Day-
-Obtained the rights to fund, develop, and lead global commercialization of sotatercept in pulmonary arterial hypertension; robust preclinical results show potential for first-in-class disease modifying approach-
"As we think about and plan to execute on our long-term vision and
strategy, there were several significant corporate events in the third
quarter. At our September R&D day, we outlined our core research and
development focus in three disease areas of high unmet medical need:
hematology, neuromuscular, and pulmonary disease. We announced that we
gained rights to sotatercept, an internally discovered Phase 2 asset,
for the development in pulmonary arterial hypertension. We also
completed a successful equity offering that will provide sufficient
funding through key inflection points in each of our clinical programs,"
Development Program Highlights
Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis
Luspatercept is designed to treat chronic anemia and reduce red blood cell (RBC) transfusion burden in adults with rare blood disorders. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.
- In addition to the ongoing MEDALIST and BELIEVE Phase 3 trials, Acceleron and Celgene continue to prepare for clinical trial expansion in new patient populations, including the COMMANDS Phase 3 trial in first-line, lower-risk MDS, regardless of ring sideroblast status, the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia, and the Phase 2 trial in myelofibrosis.
- Results from the Phase 2 trial of luspatercept for the treatment of anemia in patients with lower-risk MDS were recently published in The Lancet Oncology.
Data from two clinical abstracts on luspatercept and sotatercept will
be presented at the 59th
American Society of Hematology(ASH) Annual Meeting and Exposition in Atlanta, GAon December 9-12, 2017.
Facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) disease
ACE-083 is a locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause debilitating focal muscle loss by utilizing the "Myostatin+" approach to inhibit multiple TGF-beta ligands.
- Enrollment and treatment are ongoing in Part 1 of the Phase 2 trial in patients with FSHD, one of the most prevalent forms of muscular dystrophy in adults.
- Enrollment and treatment are ongoing in Part 1 of the Phase 2 trial in patients with CMT disease, one of the most common inherited neurological diseases leading to focal muscle weakness.
ACE-2494 is a protein therapeutic designed to have a systemic effect on muscle mass and strength throughout the body by utilizing the "Myostatin+" approach to inhibit multiple TGF-beta ligands.
- The Company plans to initiate a Phase 1 healthy volunteer clinical trial this year and is actively evaluating a potential first indication.
Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the TGF-beta protein superfamily involved in remodeling and regeneration of a variety of different tissues, including the vasculature and fibrotic tissue.
- Acceleron gained development and commercialization rights for pulmonary arterial hypertension (PAH).
- Preclinical results presented at R&D day show potential for sotatercept to be a first-in-class disease-modifying therapy that addresses fundamental molecular causes of disease in PAH.
Preclinical results of sotatercept in PAH will be highlighted in an
oral presentation at the American Heart Association Scientific
Sessions 2017 in
Anaheim, CAon November 14, 2017.
Key Corporate Priorities
- Report top-line results from MEDALIST and BELIEVE Phase 3 trials in mid-2018
- Initiate the COMMANDS Phase 3 trial in first-line, lower-risk MDS in 1H 2018
- Enroll the first myelofibrosis patient in Phase 2 by YE 2017
- Initiate the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia by YE 2017
Report FSHD Phase 2 results for cohort 1 in Part 1 in
- Report FSHD Phase 2 results for all dose-escalation cohorts in Part 1 in 2018
- Report CMT Phase 2 results from all dose-escalation cohorts in Part 1 by YE 2018
- Initiate Phase 1 healthy volunteer trial in 2017
- Initiate Phase 2 trial in PAH first half of 2018
Cash position - Cash, cash equivalents and investments as of
September 30, 2017were $366.6 million. As of December 31, 2016the Company had cash, cash equivalents and investments of $234.4 million. Cash, cash equivalents and investments include $187.6 millionof net proceeds from a follow-on public offering of common stock in September 2017. In October 2017, the underwriters exercised the over-allotment option in the offering which resulted in additional net proceeds of $28.2 million. We believe that existing cash, cash equivalents and investments, including the net proceeds from the offering and the exercise of the underwriters' over-allotment option, will be sufficient to fund projected operating requirements into 2021.
Revenue - Collaboration revenue for the third quarter was $3.0
million. The revenue is all from our Celgene partnership and is
primarily due to cost sharing revenue of
$2.9million related to expenses incurred by the Company in support of our partnered programs.
Costs and expenses - Total costs and expenses for the
third quarter were
$28.6 million. This includes R&D expenses of $21.1 million and G&A expenses of $7.5 million.
- Net loss - The Company's net loss for the third quarter ended September 30, 2017 was $25.5 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its third
quarter 2017 financial results and provide an update on recent clinical
development and corporate activities on
The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company's website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the "Acceleron Third Quarter Earnings Call".
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.
Acceleron is a
Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its neuromuscular franchise with two distinct Myostatin+ agents, ACE-083 and ACE-2494, and a pulmonary program with a Phase 2 trial of sotatercept planned in pulmonary arterial hypertension.
|CONDENSED CONSOLIDATED BALANCE SHEET|
|(Amounts in thousands)|
|Cash and cash equivalents||$||238,959||$||20,950|
|Short and long-term investments||127,638||213,432|
|Warrants to purchase common stock||1,927||1,244|
|Total stockholders' equity||358,541||225,597|
|Total liabilities and stockholders' equity||$||381,277||$||247,647|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(Amounts in thousands except per share data)|
Three Months Ended
Nine Months Ended
|Costs and expenses:|
|Research and development||21,059||17,102||64,387||49,492|
|General and administrative||7,533||6,411||26,735||19,029|
|Total costs and expenses||28,592||23,513||91,122||68,521|
|Loss from operations||(25,578||)||(20,508||)||(81,346||)||(44,119||)|
|Total other income (expense) net||86||(282||)||791||6,374|
|Loss before income taxes||(25,492||)||(20,790||)||(80,555||)||(37,745||)|
|Income tax benefit||41||20||29||20|
|Net loss applicable to common stockholders - basic and diluted||$||(25,451||)||$||(20,770||)||$||(80,526||)||$||(37,725||)|
Net loss per share applicable to common stockholders - basic and
Weighted-average number of common shares used in computing net
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements about the Company's strategy, future plans and prospects, including statements regarding the development of the Company's compounds, the timeline for clinical development and regulatory approval of the Company's compounds and the expected timing for reporting of data from ongoing clinical trials. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Actual results could differ materially from those included in the
forward-looking statements due to various risks and uncertainties,
including, but not limited to, that preclinical testing of the Company's
compounds and data from clinical trials may not be predictive of the
results or success of ongoing or later clinical trials, that the
development of the Company's compounds will take longer and/or cost more
than planned, that the Company or its collaboration partner, Celgene,
will be unable to successfully complete the clinical development of the
Company's compounds, that the Company or Celgene may be delayed in
initiating, enrolling or completing any clinical trials, and that the
Company's compounds will not receive regulatory approval or become
commercially successful products. These and other risks and
uncertainties are identified under the heading "Risk Factors" included
in the Company's most recent Annual Report on Form 10-K, and other
filings that the Company has made and may make with the
The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions and projections with respect to future events, and the Company does not undertake and specifically disclaims any obligation to update any forward-looking statements.
Vice President, Investor Relations and Corporate Communications
Manager, Investor Relations and Corporate Communications
News Provided by Acquire Media