- Mean total muscle volume increases of over
12% in the tibialis anterior and biceps brachii muscle cohorts
-
- Company plans to initiate Part 2 of the FSHD
Phase 2 trial in Q2 2018 -
Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical
company in the discovery and development of TGF-beta therapeutics
to treat serious and rare diseases, today announced positive
preliminary results for the first two cohorts in Part 1 of the
Phase 2 clinical trial with ACE-083 in patients with
facioscapulohumeral dystrophy (FSHD), a rare genetic muscle
disorder that results in progressive focal muscle loss and
weakness. The Company plans to initiate Part 2 of the ACE-083 FSHD
Phase 2 trial during the second quarter of 2018.
“Preliminary results of ACE-083 in FSHD patients demonstrated
positive safety and tolerability along with unprecedented mean
increases in total muscle volume of over 12% in the two distinct
muscles evaluated,” said Matthew Sherman, M.D., Chief Medical
Officer of Acceleron. “These data support our decision to advance
to Part 2 of the Phase 2 trial, which we expect to initiate in the
second quarter of this year. We look forward to fully exploring
functional outcomes in the larger, placebo-controlled Part 2 of the
trial.”
Part 1 of the Phase 2 trial is an open-label, dose-escalation
study of ACE-083 designed to evaluate safety as well as changes in
total muscle volume in up to 36 patients with FSHD. Preliminary
results include data from 23 patients evaluable for magnetic
resonance imaging (MRI) among two different cohorts (11 patients
with tibialis anterior weakness and 12 patients with biceps brachii
weakness). Each patient received ACE-083 (150 mg or 200 mg) as a
unilateral intramuscular injection once every three weeks for 12
weeks. Total muscle volume changes were measured by MRI relative to
baseline at 3 weeks after the last injection of ACE-083. Based on
overlap in dosing on a milligram per gram muscle analysis, dose
cohorts were pooled for the analyses of each muscle.
Tibialis Anterior Part 1 Cohorts (150 mg and 200 mg pooled)
Preliminary Results (n=11):
The tibialis anterior (TA), which is located in the lower leg,
is the main muscle responsible for ankle dorsiflexion, or the
ability to lift the front of the foot when taking a step. Over 70%
of FSHD patients experience tibialis anterior weakness over the
course of their disease, which can lead to general decreased
mobility and an increased frequency of falling.
- The TA cohorts generated a mean total
muscle volume increase of 12.6%.
- The TA cohorts produced a mean decrease
or improvement in muscle fat fraction of 5.3%
Biceps Brachii Part 1 Cohorts (150 mg and 200 mg pooled)
Preliminary Results (n=12):
The biceps brachii (BB), which is located in the upper arm, is a
major muscle responsible for elbow flexion, or the ability to lift
the lower arm. A majority of FSHD patients experience biceps
brachii weakness early in their disease, which leads to the
inability to lift objects or perform other important daily
activities without assistance.
- The BB cohorts generated a mean total
muscle volume increase of 13.2%.
- The BB cohorts produced a mean decrease
or improvement in muscle fat fraction of 0.6%.
In the BB cohorts, the majority of patients had less
intramuscular fat at baseline relative to the patients in the TA
cohorts. Patients with higher fat fraction in the BB cohorts at
baseline demonstrated larger decreases in fat fraction with
treatment.
“I am encouraged by the preliminary safety and tolerability
results of ACE-083 in Part 1 of the trial. There are currently no
FDA approved therapies for FSHD, therefore limiting our treatment
of patients to supportive options such as physical therapy and
bracing," said Dr. Jeffrey Statland, M.D., Associate Professor of
Neurology at the University of Kansas Medical Center and the
ACE-083 FSHD Phase 2 trial principal investigator. “ACE-083 is
demonstrating encouraging activity in its ability to increase
muscle volume, and I look forward to its advancement in Part 2 of
the trial.”
Strength and Function Tests:
Strength and function tests are being explored in Part 1 to
assist with the design of the randomized, double-blind,
placebo-controlled Part 2 of the study. Given the lack of placebo
control and small sample size of patients in Part 1, no conclusions
can be made on the strength and function assessments at this time.
Effects of ACE-083 on strength and function versus a
placebo-control will be evaluated in Part 2 of the study.
Part 1 Preliminary Safety Results (n=25):
There were no serious adverse events reported. The most common
adverse events were injection site related and grades 1-2. One
patient experienced a related grade 3 non-serious adverse event of
lower leg intramuscular swelling. This patient fully recovered and
was discontinued from the study.
Acceleron plans to present an in-depth review of Part 1 data at
a medical conference in 2018.
For additional information on this clinical trial, please visit
clinicaltrials.gov, identifier NCT02927080.
About ACE-083
ACE-083 is a therapeutic candidate, based on the
naturally-occurring protein follistatin, which utilizes the
“Myostatin+” approach to inhibit multiple TGF-beta ligands. It is
designed to have a concentrated effect along targeted muscles to
maximize growth and strength selectively in the muscles into which
the drug is administered. Acceleron is developing ACE-083 for
diseases such as facioscapulohumeral dystrophy (FSHD) and
Charcot-Marie-Tooth (CMT) disease, in which improved muscle
strength in target muscles may provide a clinical benefit and
enhance quality of life.
About Facioscapulohumeral Dystrophy (FSHD)
FSHD is a rare genetic muscle disorder affecting approximately
20,000 people in the United States for which there are currently no
approved treatments. The primary clinical presentation of FSHD is
debilitating skeletal muscle weakness and loss. The symptoms of
FSHD develop in a descending pattern, beginning with the face and
upper body and progressing to the lower body in a "muscle by
muscle" fashion. The disease is typically diagnosed by a
characteristic pattern of muscle weakness and other clinical
symptoms, as well as through genetic testing.
About Acceleron
Acceleron is a Cambridge-based, clinical-stage biopharmaceutical
company dedicated to the discovery, development, and
commercialization of therapeutics to treat serious and rare
diseases. The Company’s leadership in the understanding of TGF-beta
biology and protein engineering generates innovative compounds that
engage the body’s ability to regulate cellular growth and
repair.
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.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social Media: @AcceleronPharma and
LinkedIn.
Forward-Looking Statements
Cautionary Note Regarding 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 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20180108005723/en/
For Acceleron:Investors:Todd James,
IRC, (617) 649-9393Vice President, Investor Relations and Corporate
CommunicationsorCandice Ellis, 617-649-9226Manager, Investor
Relations and Corporate CommunicationsorMedia:BMC
CommunicationsBrad Miles, (646) 513-3125
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