– MEDALIST and BELIEVE Phase 3 trial results in
myelodysplastic syndromes (MDS) and beta-thalassemia, respectively,
are expected to be presented at the 60th American Society of
Hematology (ASH) Annual Meeting –
– COMMANDS Phase 3 trial in patients with
lower-risk MDS who are treatment naïve has been initiated –
– Part 1 results from the ACE-083 Phase 2 trial
in facioscapulohumeral muscular dystrophy (FSHD) and
Charcot-Marie-Tooth disease (CMT) were presented at the World
Muscle Society Annual Meeting –
– Pulmonary Arterial Hypertension (PAH) R&D
Deep Dive Event scheduled for Friday, November 16th in New York
City –
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 third quarter ended
September 30, 2018.
“2018 is proving to be a pivotal year for Acceleron, and with
several key milestones approaching, we are well-positioned for
future growth. Along with our global collaboration partner,
Celgene, we expect to share results from the MEDALIST and BELIEVE
Phase 3 trials of luspatercept with the global hematology community
at the upcoming ASH meeting in December. With the recent initiation
of the COMMANDS Phase 3 trial, we now have ongoing clinical trials
with luspatercept in three additional patient populations. We
remain committed to further exploring luspatercept’s potential to
be a platform treatment for a range of anemias,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “Our
neuromuscular and pulmonary teams have done a tremendous job in
getting multiple Phase 2 trials underway for ACE-083 in patients
with FSHD and CMT, and sotatercept in patients with PAH. Both
programs will have multiple important inflection points over the
next two years.”
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 address a late-stage erythroid maturation defect
that results in chronic anemia and the need for regular red blood
cell transfusions in adults with serious hematologic diseases.
Luspatercept is part of the global collaboration between Acceleron
and Celgene.
- The MEDALIST and BELIEVE Phase 3 trial
results in patients with lower-risk MDS and transfusion-dependent
beta-thalassemia, respectively, are expected to be presented at the
60th ASH Annual Meeting and Exposition in December 2018.
- Acceleron and Celgene plan to submit
regulatory applications for both MDS and beta-thalassemia in the
United States and Europe in the first half of 2019.
- The COMMANDS Phase 3 trial in patients
with lower-risk MDS who are treatment naïve has been
initiated.
- Enrollment is ongoing in the BEYOND
Phase 2 trial in patients with non-transfusion-dependent
beta-thalassemia as well as the Phase 2 trial in patients with
MF.
Neuromuscular Disease
ACE-083:
Facioscapulohumeral Muscular Dystrophy
(FSHD) and Charcot-Marie-Tooth Disease (CMT)
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 involved
in muscle formation.
- Final results from Part 1 of each Phase
2 trial with ACE-083 in patients with FSHD and CMT, respectively,
were presented at the 2018 World Muscle Society (WMS) Annual
Meeting.
- Enrollment is ongoing in Part 2 of the
Phase 2 FSHD trial, with preliminary results expected in the second
half of 2019.
- Enrollment is ongoing in Part 2 of the
Phase 2 CMT trial, with preliminary results expected by the end of
2019.
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 involved in muscle formation.
- Enrollment is ongoing in the Phase 1
healthy volunteer trial, with preliminary results expected in the
first half of 2019.
Pulmonary Disease
Sotatercept:
Pulmonary Arterial Hypertension
(PAH)
Sotatercept acts as a ligand trap for members of the TGF-beta
superfamily that control the BMPRII signaling pathway, which is
critical for maintaining healthy pulmonary vasculature. In multiple
preclinical studies in PAH, sotatercept significantly decreased
pulmonary vessel muscularization, improved pulmonary arterial
pressures, and decreased indicators of right heart failure.
- Multiple preclinical abstracts for
sotatercept in PAH have been accepted for presentation at the
American Heart Association Scientific Sessions on November 10-12,
2018.
- On November 16, 2018, the Company will
host a PAH Research and Development Deep Dive event in New York
City.
- Enrollment is ongoing in the PULSAR
Phase 2 trial in patients with PAH, with preliminary results
expected in the first half of 2020.
- The Company plans to initiate an
exploratory study, called SPECTRA, in Q1 2019 to provide further
understanding of sotatercept’s impact on the disease.
Financial Results
- Cash position – Cash, cash
equivalents and investments as of September 30, 2018 were
$319.8 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 third quarter was $3.3 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 third quarter were $33.4 million. This
includes R&D expenses of $24.7 million and G&A
expenses of $8.7 million.
- Net loss – The Company's
net loss for the third quarter ended September 30, 2018
was $29.0 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
its third quarter financial results for 2018 and provide an update
on recent corporate activities on October 30, 2018, at 4:30 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 Third 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 Phase 2
pulmonary program with 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) September 30, 2018
December 31, 2017 Cash and cash equivalents $ 112,427
$ 100,150 Short and long-term investments 207,380 272,800 Other
assets 18,310 16,227 Total assets $ 338,117 $ 389,177
Deferred revenue $ — $ 3,702 Warrants to purchase common
stock 2,031 2,236 Other liabilities 16,974 18,021 Total
liabilities 19,005 23,960 Total stockholders’ equity 319,112
365,217 Total liabilities and stockholders’ equity $ 338,117
$ 389,177
ACCELERON PHARMA INC. CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS (Amounts in thousands
except per share data) (unaudited)
Three Months Ended September
30,
Nine Months Ended September
30,
2018 2017 2018 2017
Revenue: Collaboration revenue $ 3,258 $ 3,014 $ 10,175 $
9,776 Costs and expenses: Research and development 24,667 21,059
74,027 64,387 General and administrative 8,653 7,533
23,756 26,735 Total costs and expenses 33,320
28,592 97,783 91,122 Loss from operations
(30,062 ) (25,578 ) (87,608 ) (81,346 ) Total other income, net
1,071 86 3,481 791 Loss before income
taxes (28,991 ) (25,492 ) (84,127 ) (80,555 ) Income tax benefit
(provision) 12 41 (9 ) 29 Net loss applicable
to common stockholders- basic and diluted $ (28,979 ) $ (25,451 ) $
(84,136 ) $ (80,526 ) Net loss per share applicable to
common stockholders- basic and diluted $ (0.63 ) $ (0.65 ) $ (1.84
) $ (2.08 )
Weighted-average number of common shares
used in computingnet loss per share applicable to common
stockholders
46,051 39,361 45,787 38,804
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 factors, 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 results of any clinical trial may not be
predictive of the results or success of other clinical trials of
the same product candidate, 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: https://www.businesswire.com/news/home/20181030006026/en/
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate CommunicationsOrCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
Communications
Media:Matt Fearer, 617-301-9557Director, Corporate
Communications
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