- Clinical development and research efforts focused on hematological,
neuromuscular and pulmonary diseases –
CAMBRIDGE, Mass. -- (Business Wire)
Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company
in the discovery and development of TGF-beta therapeutics to treat
serious and rare diseases, hosted its Research and Development (R&D) Day
in New York City today.The Company discussed clinical
development plans to build therapeutic area leadership, and its
long-term strategy to advance key programs in hematologic,
neuromuscular, and pulmonary diseases.
“We have several, near-term milestones in our mid- to late-stage
pipeline that could transform our business from a development-stage
organization to a fully-integrated, commercial biopharmaceutical
company,” said Habib Dable, Chief Executive Officer of Acceleron. “With
the opportunity to launch and co-promote luspatercept in North America,
and the continued advancement of our neuromuscular candidate as well as
our recent entry into pulmonary disease, we are positioned to bring
potentially life-changing new treatments to patients and drive value for
our shareholders. We expect to report top-line Phase 3 results of
luspatercept in myelodysplastic syndromes and beta-thalassemia alongside
our collaboration partner Celgene, in mid-2018. ACE-083 continues to
advance through multiple Phase 2 clinical trials, and ACE-2494, the
second asset in our neuromuscular franchise, is expected to initiate a
clinical trial later this year. The recent announcement of our plan to
develop sotatercept as a Phase 2 therapeutic candidate for the potential
treatment of pulmonary arterial hypertension is a tremendous opportunity
for us, given that it launches our therapeutic focus in pulmonary
disease and enhances our commitment to develop new therapies that could
transform patients’ lives.”
Key R&D Day Highlights:
Hematology
-
Luspatercept
-
Significant market expansion efforts across multiple new
indications are being pursued by Acceleron and Celgene in addition
to the potential blockbuster market opportunity in initial
MEDALIST and BELIEVE Phase 3 indications
-
A third Phase 3 trial, also known as the COMMANDS trial, is
expected to begin in early 2018
-
The COMMANDS trial will evaluate luspatercept versus
erythropoiesis-stimulating agent (ESA) treatment in
first-line, lower-risk MDS, and include patients regardless of
ring sideroblast (RS) status
-
A Phase 2 trial in non-transfusion-dependent (NTD)
beta-thalassemia patients, also known as the BEYOND trial, is
expected to begin later this year, and the Phase 2 trial in
myelofibrosis is underway
Neuromuscular
-
ACE-083 and ACE-2494
-
ACE-083 and ACE-2494 utilize the Company’s “Myostatin+” approach
to increase muscle strength and function by inhibiting multiple
ligands of the TGF-beta superfamily
-
ACE-083, a targeted muscle agent being developed for the treatment
of facioscapulohumeral dystrophy (FSHD) and Charcot-Marie-Tooth
(CMT) disease, has shown increases in muscle mass of 9% to 15% in
healthy volunteers
-
Company expects Phase 2 FSHD part 1 preliminary results from
cohort 1 to be available in late 2017
-
ACE-2494, a systemic muscle agent, has demonstrated preclinical
activity across multiple neuromuscular diseases
Pulmonary
-
Sotatercept
-
Acceleron gains rights for pulmonary hypertension indications from
Celgene
-
Preclinical results show potential for sotatercept to be a
first-in-class disease-modifying therapy that addresses
fundamental molecular causes of disease in pulmonary arterial
hypertension (PAH)
-
Company expects to initiate a Phase 2 trial in PAH during the
first half of 2018
-
ACE-1334
-
Multiple, ongoing preclinical studies in pulmonary disease
About Acceleron
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 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.
For more information, please visit www.acceleronpharma.com/.
Follow Acceleron on Social Media: @AcceleronPharma and
LinkedIn.
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," "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 SEC in the
future.
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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170919006177/en/
Contacts:
Acceleron Pharma Inc.
Todd James, IRC, 617-649-9393
Vice
President, Investor Relations and Corporate Communications
or
Candice
Ellis, 617-649-9226
Manager, Investor Relations and Corporate
Communications
or
Media:
BMC Communications
Brad
Miles, 646-513-3125
Source: Acceleron Pharma
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