– Top-line results from the MEDALIST and
BELIEVE Phase 3 trials of luspatercept expected in mid-2018 –
– ACE-083 advances into Part 2 of the ongoing
Phase 2 trial in facioscapulohumeral muscular dystrophy –
– PULSAR Phase 2 trial with sotatercept in
pulmonary arterial hypertension on track to initiate 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 provided a corporate
update and reported financial results for the first quarter ended
March 31, 2018.
“2018 is an important year for our Company, as we prepare for
the upcoming top-line results from our MEDALIST and BELIEVE Phase 3
trials with luspatercept expected mid-year. We and Celgene continue
to invest heavily in the overall luspatercept opportunity by
targeting myelodysplastic syndromes, beta-thalassemia, and
myelofibrosis,” said Habib Dable, Chief Executive Officer of
Acceleron. “The joint collaboration teams recently kicked off
strategic preparations for the anticipated regulatory and
commercial activities planned to follow the luspatercept Phase 3
results.”
Added Mr. Dable: “Our team continues to make meaningful progress
in the advancement of our wholly-owned clinical programs. In
neuromuscular disease, we presented preliminary results from our
Phase 2 trial of ACE-083 in FSHD for the first time and recently
initiated Part 2 of the trial. In pulmonary, we expect to initiate
our first Phase 2 trial with sotatercept in pulmonary arterial
hypertension in the second quarter.”
Development Program
Highlights
Hematology
Luspatercept:
Myelodysplastic Syndromes (MDS),
Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is a first-in-class erythroid maturation agent
(EMA) designed to treat the late-stage red blood cell (RBC)
maturation defect that results in chronic anemia and the need for
regular RBC transfusions in adults with rare blood disorders.
Luspatercept is being developed as part of the global collaboration
between Acceleron and Celgene.
- Top-line results from the MEDALIST and
BELIEVE Phase 3 trials in MDS and beta-thalassemia, respectively,
are expected in mid-2018.
- The initiation of the COMMANDS Phase 3
trial in first-line, lower-risk MDS patients is planned for the
first half of 2018.
- Enrollment and treatment are ongoing in
the BEYOND Phase 2 trial in non-transfusion-dependent
beta-thalassemia and the Phase 2 trial in MF.
- The Company expects to provide updates
from the ongoing long-term Phase 2 extension trials in MDS and
beta-thalassemia at the 2018 American Society of Clinical Oncology
(ASCO) Annual Meeting and 23rd Congress of the European Hematology
Association (EHA) in June.
Neuromuscular Disease
ACE-083:
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 focal muscle weakness. ACE-083 utilizes the
"Myostatin+" approach to inhibit multiple TGF-beta ligands.
- Preliminary results from dose cohorts 1
and 2 in Part 1 of the ACE-083 Phase 2 trial in patients with FSHD
were highlighted in a “Best of Neuromuscular Disease” clinical
session at the American Academy of Neurology (AAN) 70th Annual
Meeting on April 26, 2018.
- The Company plans to present additional
preliminary results from all Part 1 dose cohorts during the second
half of 2018.
- Part 2 of the Phase 2 trial was
recently initiated and preliminary results are expected in the
second half of 2019.
- ACE-083 received FDA Fast Track
designation in FSHD.
- Enrollment and treatment are ongoing in
Part 1 of the Phase 2 trial in patients with CMT disease.
- The Company plans to present
preliminary Part 1 results in the second half of 2018 and to
initiate Part 2 of the Phase 2 trial by the end of 2018.
ACE-2494:
ACE-2494 is designed to have a systemic effect on muscle mass
and strength for diseases that cause muscle weakness throughout the
body. ACE-2494 utilizes the "Myostatin+" approach to inhibit
multiple TGF-beta ligands.
- Enrollment and treatment are ongoing in
the Phase 1 healthy volunteer trial.
- Preliminary results from the Phase 1
trial are expected in the first half of 2019.
Pulmonary Disease
Sotatercept:
Pulmonary Arterial Hypertension
(PAH)
Sotatercept is a ligand trap for members in the TGF-beta
superfamily involved in remodeling a variety of different tissues,
including the vasculature and fibrosis. In multiple preclinical
studies in PAH, sotatercept decreased vessel muscularization,
improved pulmonary arterial pressures, and decreased indicators of
right heart failure.
- The Company expects to initiate the
PULSAR Phase 2 trial during the second quarter of 2018.
- Preliminary results from the PULSAR
Phase 2 trial are expected in the first half of 2020.
Financial Results
- Cash position – Cash, cash
equivalents and investments as of March 31, 2018 were $353.3
million. As of December 31, 2017, the Company had cash, cash
equivalents and investments of $372.9 million. The Company believes
that existing cash, cash equivalents and investments will be
sufficient to fund projected operating requirements into 2021.
- Revenue – Collaboration
revenue for the first quarter was $3.2 million. The revenue is
all from Acceleron’s partnership with Celgene and is primarily
related to expenses incurred by the Company in support of
luspatercept.
- Costs and expenses – Total
costs and expenses for the first quarter were $30.8 million. This
includes R&D expenses of $23.4 million and G&A
expenses of $7.4 million.
- Net loss – The Company's
net loss for the first quarter ended March 31,
2018 was $26.2 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
its first quarter financial results for 2018 and provide an update
on recent corporate activities on May 8, 2018, at 5:00 p.m.
EDT.
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
referring to the “Acceleron First Quarter 2018 Earnings Call.”
The archived webcast will be available for replay on the
Acceleron website approximately two hours after the event.
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 an ongoing Phase 2 trial of sotatercept in pulmonary
arterial hypertension.
For more information, please visit www.acceleronpharma.com. Follow Acceleron on
Social Media: @AcceleronPharma and LinkedIn.
ACCELERON PHARMA INC. CONDENSED CONSOLIDATED BALANCE
SHEET (Amounts in thousands) (unaudited)
March 31, 2018 December 31, 2017 Cash and cash
equivalents $ 69,736 $ 100,150 Short and long-term investments
283,607 272,800 Other assets 16,691 16,227 Total assets $
370,034 $ 389,177 Deferred revenue $ — $ 3,702
Warrants to purchase common stock 1,320 2,236 Other liabilities
14,934 18,021 Total liabilities 16,254 23,960 Total
stockholders’ equity 353,780 365,217 Total liabilities and
stockholders’ equity $ 370,034 $ 389,177
ACCELERON PHARMA INC. CONDENSED CONSOLIDATED STATEMENTS
OF OPERATIONS (Amounts in thousands except per share
data) (unaudited) Three Months Ended March
31, 2018 2017 Revenue:
Collaboration revenue $ 3,232 $ 3,705 Costs and expenses: Research
and development 23,431 21,727 General and administrative 7,441
7,836 Total costs and expenses 30,872
29,563 Loss from operations (27,640 ) (25,858 ) Total other
income, net 1,431 457 Loss before income taxes
(26,209 ) (25,401 ) Income tax provision (10 ) (6 ) Net loss
applicable to common stockholders- basic and diluted $ (26,219 )
$ (25,407 ) Net loss per share applicable to common
stockholders- basic and diluted $ (0.58 ) $ (0.66 )
Weighted-average number of common shares used in computing net loss
per share applicable to common stockholders 45,516 38,404
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 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: https://www.businesswire.com/news/home/20180508006320/en/
Acceleron Pharma Inc.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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