- Luspatercept Phase 3 trials to complete enrollment in both the MDS ("MEDALIST") and beta-thalassemia ("BELIEVE") trials in 2H 2017 -
- Presented data from five abstracts on luspatercept and sotatercept
- Treated first patient in ACE-083 Phase 2 study for facioscapulohumeral muscular dystrophy -
- Outlined plans to initiate several new clinical trials in 2017 -
- Maintained strong balance sheet with
"Acceleron made tremendous progress in 2016 across our entire portfolio
of potentially first- and best-in-class therapies for patients with
serious hematologic, neuromuscular and fibrotic diseases without
effective treatments," said
DEVELOPMENT PROGRAM HIGHLIGHTS
Luspatercept in myelodysplastic syndromes (MDS)
Luspatercept is being developed to treat anemia and reduce or eliminate the need for red blood cell transfusions in patients with MDS, a rare disorder in which bone marrow fails to produce enough healthy blood cells. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.
Luspatercept in beta-thalassemia
Luspatercept is designed to treat severe, chronic anemia and reduce RBC transfusion-dependence in adults with beta-thalassemia, a rare genetic disorder that reduces the production of hemoglobin. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.
Luspatercept and sotatercept in myelofibrosis
Myelofibrosis is a malignant disease which results in fibrotic bone marrow, multiple cytopenias and severe anemia often requiring RBC transfusions. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.
ACE-083 in facioscapulohumeral muscular dystrophy (FSHD)
ACE-083 is designed to increase muscle mass and strength in target muscles for diseases that cause debilitating focal muscle loss.
Dalantercept in Advanced Renal Cell Carcinoma
Dalantercept is being developed in combination with axitinib to further inhibit tumor angiogenesis.
Acceleron continues its research on several molecules targeting musculoskeletal diseases, fibrotic disorders and other serious diseases.
OTHER CORPORATE UPDATES
NEWSFLOW AND CATALYSTS
Conference Call and Webcast Information
The Company will host a live conference call and webcast to discuss its
fourth quarter and full year financial results for 2016 and provide a
corporate update on
The live webcast can be accessed on the Investors page of the Company's website at www.acceleronpharma.com.
A replay of the webcast will be archived on the Company's website and accessible approximately two hours after the event.
Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases. Its pioneering research platform leverages the powerful biology behind the body's ability to rebuild and repair its own cells and tissues. This approach to drug discovery has generated four therapeutic candidates that are currently in clinical trials. The Company's lead therapeutic candidate, luspatercept, is being evaluated in Phase 3 studies for the treatment of the hematologic diseases, myelodysplastic syndromes (MDS) and beta-thalassemia under a global partnership with Celgene Corp. Acceleron is also advancing clinical programs in the fields of oncology and neuromuscular diseases and has a comprehensive preclinical research effort targeting fibrotic and other serious diseases.
For more information, please visit www.acceleronpharma.com.
|Cash and cash equivalents||$||20,950||$||27,783|
|Short and long-term investments||213,432||108,198|
|Warrants to purchase common stock||1,244||17,187|
|Total stockholders' equity||225,597||109,263|
|Total liabilities and stockholders' equity||$||247,647||$||146,337|
Three Months Ended
|Costs and expenses:|
|Research and development||19,088||16,146||68,580||58,404|
|General and administrative||6,267||5,773||25,297||20,572|
|Total costs and expenses||25,355||21,919||93,877||78,976|
|Loss from operations||(21,986||)||(18,115||)||(66,106||)||(60,879||)|
|Other income (expense), net||2,742||(8,967||)||9,116||(3,015||)|
|Loss before income taxes||(19,244||)||(27,082||)||(56,990||)||(63,894||)|
|Income tax provision||(44||)||—||(24||)||—|
|Other comprehensive loss:|
|Net unrealized holding losses on short-term and long-term investments during the period||(83||)||(199||)||(205||)||(220||)|
|Net loss per share - basic and diluted||$||(0.51||)||$||(0.81||)||$||(1.52||)||$||(1.92||)|
|Weighted-average number of common shares used in computing net loss per share - basic and diluted||37,914||33,268||37,430||33,303|
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, including sotatercept, luspatercept, dalantercept, ACE-083, ACE-2494, the Company's IntelliTrap™ drug discovery platform, and the Company's TGF-beta superfamily program generally, the timeline for clinical development and regulatory approval of the Company's compounds, the expected timing for the reporting of data from ongoing trials, and the structure of the Company's planned or pending clinical trials. The words "anticipate," "appear," "believe," "continue," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "potential," "predict," "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.
Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks that the Company's cash, cash equivalents and investments will be insufficient to fund operations into the second half of 2019, 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 data may not be available when the Company expects it to be, that the Company or its collaboration partner, Celgene, will be unable to successfully complete the clinical development of the Company's compounds, that the development of the Company's compounds will take longer or cost more than planned, that the Company or Celgene may be delayed in initiating, enrolling or completing any clinical trials, that the Company's drug discovery activities may not yield drug candidates for which the Company can commence clinical trials at the rate at which the Company currently anticipates or at all, and that the Company's compounds will not receive regulatory approval or become commercially successful products.
Other risks and uncertainties include those identified under the heading
"Risk Factors" included in the Company's Annual Report on Form 10-K
which was filed with the
Senior Director, Investor Relations and Corporate Communications
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