- Acceleron gains rights to fund, develop, and lead global commercialization of sotatercept in pulmonary arterial hypertension -
- Acceleron expects to initiate a Phase 2 trial of sotatercept in pulmonary arterial hypertension in 1H 2018 -
- Preclinical results show potential first-in-class disease modifying properties of sotatercept in pulmonary arterial hypertension -
- Sotatercept is a well-defined TGF-beta superfamily ligand trap with a widely established preclinical and clinical profile -
"We are very pleased to announce the execution of this amended agreement
alongside our long-time collaboration partner, Celgene. Sotatercept
shows tremendous potential to be a groundbreaking therapy for pulmonary
arterial hypertension, and we expect sotatercept, as a Phase 2-ready
compound, to be the lead molecule in our new pulmonary disease
"While many therapies are approved for the treatment of pulmonary
arterial hypertension, these therapies all focus on a mechanism of
vasodilation and the prognosis for patients remains poor," said
"This amended agreement unites Acceleron's scientific leadership in the
TGF-beta superfamily with Celgene's commitment to advance novel
molecules, such as sotatercept, for patients in therapeutic areas of
high unmet medical need," said
Under the amended and restated collaboration agreement, Acceleron has the right to fund and conduct all research and development activities for sotatercept in the pulmonary hypertension field. Should sotatercept be approved for an indication in the pulmonary hypertension field, Acceleron will be responsible for global commercialization and Celgene will be eligible to receive royalties on global net sales in that field. The original collaboration deal terms will remain in place with respect to development and commercialization outside of the pulmonary hypertension field.
Acceleron also updated its cash guidance, announcing that its existing cash, cash equivalents and investments will be sufficient to fund projected operating requirements into mid-2019, versus its earlier guidance of the second half of 2019.
Sotatercept is an investigational product that is not approved for use in any country.
Acceleron R&D Day Conference Call and Webcast Information
The live 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 "Acceleron 2017 R&D Day."
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.
Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the transforming growth factor-beta (TGF-β) protein superfamily involved in remodeling and regeneration of a variety of different tissues, including the vasculature and fibrosis.
About Pulmonary Arterial Hypertension
Pulmonary Arterial Hypertension (PAH) is a rare and chronic, rapidly progressing disorder characterized by the constriction of small pulmonary arteries transporting high pressure blood from the heart to the lungs. The high or increased blood pressure causes significant strain on the heart, often leading to limited physical activity, heart failure and reduced life expectancy. The 5-year survival rate for patients with PAH is approximately 57%. There are currently four classes of therapies approved for treatment of PAH: endothelin receptor antagonists (ERAs), phosphodiesterase-5 (PDE-5) inhibitors, prostacyclin analogues and soluble guanylate cyclase (sGC) stimulators. The available therapies all act by promoting the dilation of pulmonary vessels, which have positive effects for patients, but do not impact the underlying cause of the disease. As a result, PAH often progresses rapidly for many patients despite standard of care treatment. A growing body of research has implicated imbalances in BMP and TGF-β signaling as a primary driver of PAH in familial, idiopathic and acquired forms of the disease.
Acceleron is a biopharmaceutical leader in TGF-beta science, focused on the discovery and development of innovative therapeutics to treat patients with serious and rare diseases. Its pioneering research and protein engineering platform engages the target-rich TGF-beta superfamily and its remarkable ability to regulate cellular growth and repair.
Under a global partnership with Celgene, Acceleron is in Phase 3 development with luspatercept, a potential first-in-class chronic anemia therapy for the treatment of rare blood diseases. The Company is also advancing a 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. Acceleron has ongoing preclinical research efforts targeting additional indications in these three disease areas where there is significant unmet medical need.
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," "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
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