- MEDALIST and BELIEVE Phase 3 trial
presentations at the 60th American Society of Hematology (ASH)
Annual Meeting both selected for “Best of ASH” -
- Luspatercept Biologics License Application
(BLA) submission expected in April 2019 -
- Part 2 results from the ACE-083 Phase 2
trials in facioscapulohumeral muscular dystrophy (FSHD) and
Charcot-Marie-Tooth disease (CMT) anticipated during 2019 -
- Pulmonary program on track with two Phase 2
trials of sotatercept in pulmonary arterial hypertension (PAH)
currently active -
Acceleron Pharma Inc. (Nasdaq: XLRN), a leading
biopharmaceutical company in the discovery and development of
TGF-beta superfamily therapeutics to treat serious and rare
diseases, today provided a corporate update and reported financial
results for the fourth quarter and full year ended December 31,
2018.
“A series of significant achievements in 2018 have positioned
Acceleron to execute on a number of key milestones across the
entire pipeline during 2019 and 2020,” said Habib Dable, President
and Chief Executive Officer of Acceleron. “First and foremost, we
and our global collaboration partner, Celgene, plan to submit
marketing applications in the U.S. and E.U. for our lead product
candidate, luspatercept, in lower-risk myelodysplastic syndromes
and transfusion-dependent beta-thalassemia in the first half of the
year. At the same time, we’re continuing to evaluate luspatercept’s
potential to treat a range of anemias, from first-line therapy in
MDS via the ongoing COMMANDS Phase 3 trial, to
non-transfusion-dependent beta-thalassemia, myelofibrosis and
beyond.”
“In addition, our wholly-owned programs in neuromuscular and
pulmonary disease are all advancing. We’re anticipating topline
results from the placebo controlled part of our Phase 2 trials
evaluating our locally-acting muscle agent, ACE-083, in FSHD and
CMT by the end of the year. Lastly, 2020 will bring important Phase
2 trial results in PAH with sotatercept, which we believe has the
potential to alter the treatment landscape for this devastating
disease.”
Development Program
Highlights
Hematology
Luspatercept: Myelodysplastic Syndromes
(MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is an investigational 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, were presented at the 60th ASH
Annual Meeting and Exposition in December 2018.
- The MEDALIST and BELIEVE presentations
were both selected for presentation during the “Best of ASH”
session at the meeting.
- Acceleron and Celgene plan to submit a
Biologics License Application (BLA) to the U.S. Food and Drug
Administration (FDA) for luspatercept in patients with anemia
related lower-risk MDS and beta-thalassemia in April 2019.
- Acceleron and Celgene remain on track
to submit a Marketing Authorization Application (MAA) to the
European Medicines Agency (EMA) for luspatercept in patients with
anemia related to lower-risk MDS and beta-thalassemia in the first
half of 2019.
- The ongoing Phase 2 trial of
luspatercept in patients with MF has completed target enrollment,
with preliminary results expected in the second half of 2019.
- Enrollment is ongoing in the COMMANDS
Phase 3 trial in patients with first-line lower-risk MDS and the
BEYOND Phase 2 trial in patients with non-transfusion-dependent
beta-thalassemia, with preliminary results expected from the BEYOND
trial in 2020.
Neuromuscular Disease
ACE-083: Facioscapulohumeral Muscular
Dystrophy (FSHD) and Charcot-Marie-Tooth Disease
(CMT)
ACE-083 is an investigational 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 superfamily ligands involved in muscle formation.
- Preliminary results from Part 1 of the
Phase 2 trials evaluating ACE-083 in patients with FSHD and CMT,
were presented at the 2018 World Muscle Society (WMS) Annual
Meeting in October 2018.
- Part 2 of the Phase 2 FSHD trial has
completed patient enrollment, with preliminary topline 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: Neuromuscular
Disease
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 superfamily 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 multi-ligand trap for certain members of
the TGF-beta superfamily to rebalance BMPRII signaling, which is a
key molecular driver of PAH. In preclinical studies of PAH,
sotatercept reversed pulmonary vessel muscularization and improved
indicators of right heart failure.
- Preclinical results from multiple
studies of sotatercept in PAH were presented at the American Heart
Association Scientific Sessions in November 2018.
- Enrollment is ongoing in the PULSAR
Phase 2 trial in patients with PAH, with preliminary results
expected in the first half of 2020.
- The exploratory SPECTRA trial in
patients with PAH has been initiated, with preliminary results
expected in 2020.
Corporate Highlights
- The Company recently raised
approximately $264.5 million of gross proceeds in a follow-on
offering of common stock.
Financial Results
- Cash Position – Cash, cash
equivalents and investments as of December 31, 2018 were
$291.3 million. As of December 31, 2017, the Company had cash, cash
equivalents and investments of $372.9 million. Based on the
Company's current operating plan and projections, it believes that
current cash, cash equivalents and investments, together with the
net proceeds of $248.2 million from its recent common stock
offering, will be sufficient to fund projected operating
requirements until such time as it expects to receive significant
royalty revenue from luspatercept sales.
- Revenue – Collaboration revenue
for the year was $14.0 million. The revenue is all from the
Company'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 year were $138.4 million. This includes
R&D expenses of $103.9 million and G&A expenses of $34.5
million.
- Net Loss – The Company’s net
loss for the year ended December 31, 2018 was $118.9
million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
its fourth quarter and full year financial results for 2018 and
provide an update on recent corporate activities on February 27,
2019, at 5:00 p.m. EST.
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 Fourth Quarter and Full Year 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 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)
December 31, 2018
December 31, 2017
Cash and cash equivalents $ 144,052 $ 100,150 Short and long-term
investments 147,260 272,800 Other assets 23,509 16,227 Total
assets $ 314,821 $ 389,177 Deferred revenue $ — $
3,703 Warrants to purchase common stock 1,491 2,236 Other
liabilities 21,293 18,021 Total liabilities 22,784 23,960
Total stockholders’ equity 292,037 365,217 Total liabilities
and stockholders’ equity $ 314,821 $ 389,177
ACCELERON PHARMA INC. CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(Amounts in thousands except per share data)
(unaudited) Three Months Ended December 31,
Year Ended December 31, 2018 2017
2018 2017 Revenue: Collaboration
revenue $ 3,816 $ 3,705 $ 13,991 $ 13,481 Costs and expenses:
Research and development 29,867 25,339 103,902 89,726 General and
administrative 10,755 7,002 34,503 33,738
Total costs and expenses 40,622 32,341 138,405 123,464 Loss
from operations (36,806 ) (28,636 ) (124,414 ) (109,983 ) Total
other income, net 2,036 770 5,516 1,561
Loss before income taxes (34,770 ) (27,866 ) (118,898 ) (108,422 )
Income tax benefit (provision) 36 (60 ) 27 (32 ) Net
loss $ (34,734 ) $ (27,926 ) $ (118,871 ) $ (108,454 ) Net
loss per share - basic and diluted $ (0.75 ) $ (0.62 ) $ (2.59 ) $
(2.68 ) Weighted-average number of common shares used in
computing net loss per share 46,227 45,217 45,898 40,420
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 trials may not be
predictive of the results or success of other 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/20190227005921/en/
Acceleron Pharma Inc.Investors:Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate Communications
Media:Matt Fearer, 617-301-9557Director, Corporate
Communications
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