- BLA and MAA filings of luspatercept for
beta-thalassemia- and myelodysplastic syndromes-associated anemia
accepted by U.S. Food and Drug Administration and European
Medicines Agency, respectively -
- Acceleron receives $25 million milestone
payment from Celgene for luspatercept’s BLA acceptance and MAA
validation -
- The ACE-083 Phase 2 trial in
Charcot-Marie-Tooth disease (CMT) has completed enrollment with
topline results anticipated in Q1 2020, and results from the
facioscapulohumeral muscular dystrophy (FSHD) trial are expected in
2H19 -
- PULSAR Phase 2 trial of sotatercept in
pulmonary arterial hypertension (PAH) is fully enrolled, with
topline results expected in Q1 2020 -
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 second
quarter ended June 30, 2019.
“The significant progress across our entire pipeline over the
last few years has us well positioned to execute during one of the
most exciting times in Acceleron’s 16-year history. With the
luspatercept U.S. and European regulatory approval filings under
review, we are now one step closer to the first-ever potential
approval of an Acceleron-discovered medicine,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “Alongside our
global collaboration partner, Celgene, we are focused on preparing
for luspatercept’s potential commercial launch, and we continue to
execute on our ongoing clinical trials in first-line lower-risk
MDS-, non-transfusion-dependent beta-thalassemia- and
myelofibrosis-associated anemia.”
Added Mr. Dable: “In parallel, we have advanced our two
Acceleron-led clinical programs in neuromuscular and pulmonary
disease as we work to establish key proof-of-concept results in
three placebo-controlled Phase 2 trials over the next nine months.
Following robust enrollment in our PULSAR Phase 2 trial in patients
with PAH, we now expect topline results in the first quarter of
2020. For ACE-083, we anticipate topline results in patients with
FSHD and CMT in the second half of this year and early 2020,
respectively.”
Development Program
Highlights
Hematology
Luspatercept: Myelodysplastic Syndromes
(MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is an investigational first-in-class erythroid
maturation agent 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 U.S. Food and Drug Administration (FDA) accepted the
Biologics License Application (BLA) for luspatercept for the
treatment of adult patients with very low- to intermediate-risk
MDS-associated anemia who have ring sideroblasts and require red
blood cell (RBC) transfusions, and for the treatment of adult
patients with beta-thalassemia-associated anemia who require RBC
transfusions.
- The FDA granted priority review for the beta-thalassemia
indication and set a target action date of December 4, 2019, and
set a target action date of April 4, 2020, for the MDS
indication.
- The Marketing Authorization Application (MAA) for luspatercept
in adult patients with MDS- or beta-thalassemia-associated anemia
has been validated by the European Medicines Agency (EMA). The EMA
decision on the MAA is expected in the second half of 2020.
- Results from the Phase 2 trial of luspatercept in patients with
MF are expected later this year.
- Enrollment is ongoing in the COMMANDS Phase 3 trial in patients
with treatment-naïve lower-risk MDS and the BEYOND Phase 2 trial in
patients with non-transfusion-dependent beta-thalassemia. Topline
results from the BEYOND trial are expected by year-end 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.
- Topline results from Part 2 of the Phase 2 trial in patients
with FSHD are expected in the second half of 2019.
- Enrollment was recently completed in Part 2 of the Phase 2
trial in patients with CMT, with topline results expected in the
first quarter of 2020.
- The Phase 2 extension trial is open for patients who
participated in the FSHD and CMT Phase 2 trials of ACE-083.
Pulmonary Disease
Sotatercept: Pulmonary Arterial
Hypertension (PAH) Sotatercept is an investigational
agent designed to be a selective ligand trap for members of the
TGF-beta superfamily to rebalance BMPR2 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.
- The PULSAR Phase 2 trial in patients with PAH has completed
enrollment, with topline results expected in Q1 2020.
- Enrollment is ongoing in the exploratory SPECTRA trial in
patients with PAH, with preliminary results expected in 2020.
Financial Results
- Cash Position – Cash, cash equivalents and investments
as of June 30, 2019 were $500.9 million. This cash balance includes
the receipt of a $25.0 million gross milestone payment for the
acceptance of the luspatercept BLA and MAA filings. As of December
31, 2018, the Company had cash, cash equivalents and investments of
$291.3 million. Based on the Company's current operating plan and
projections, it believes that current cash, cash equivalents and
investments will be sufficient to fund projected operating
requirements until such time as it expects to receive significant
royalty revenue from luspatercept sales.
- Revenue – Second quarter revenue was $27.7 million. The
revenue is all from the Company's collaboration partnership with
Celgene, and is largely related to the milestone payment received,
as well as expenses incurred by the Company in support of
luspatercept.
- Costs and Expenses – Total costs and expenses for the
second quarter were $48.8 million. This includes R&D expenses
of $34.8 million and G&A expenses of $14.0 million.
- Net Loss – The Company's net loss for the second quarter
ended June 30, 2019 was $17.9 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
its second quarter 2019 financial results and provide an update on
recent corporate activities on August 5, 2019, 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 Second Quarter 2019 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 superfamily 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 program with ACE-083,
a locally-acting Myostatin+ agent in Phase 2 development in
facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth
disease and is conducting 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)
June 30, 2019
December 31, 2018
Cash and cash equivalents
$
163,609
$
144,052
Short and long-term investments
337,307
147,260
Operating lease assets
26,549
—
Other assets
23,403
23,509
Total assets
$
550,868
$
314,821
Short-term and long-term operating lease
liabilities
$
29,153
$
—
Warrants to purchase common stock
1,389
1,491
Other liabilities
20,429
21,292
Total liabilities
50,971
22,784
Total stockholders’ equity
499,897
292,037
Total liabilities and stockholders’
equity
$
550,868
$
314,821
ACCELERON PHARMA INC. CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS (Amounts in thousands except per share
data) (unaudited)
Three Months Ended June
30,
Six Months Ended June
30,
2019
2018
2019
2018
Revenue:
Collaboration revenue
$
27,666
$
3,685
$
30,447
$
6,917
Costs and expenses:
Research and development
34,765
25,933
67,536
49,363
General and administrative
14,037
7,658
24,851
15,099
Total costs and expenses
48,802
33,591
92,387
64,462
Loss from operations
(21,136
)
(29,906
)
(61,940
)
(57,545
)
Total other income, net
3,230
979
6,003
2,410
Loss before income taxes
(17,906
)
(28,927
)
(55,937
)
(55,135
)
Income tax benefit (provision)
44
(11
)
24
(21
)
Net loss applicable to common
stockholders- basic and diluted
$
(17,862
)
$
(28,938
)
$
(55,913
)
$
(55,156
)
Net loss per share applicable to common
stockholders- basic and diluted
$
(0.34
)
$
(0.63
)
$
(1.08
)
$
(1.21
)
Weighted-average number of common shares
used in computing net loss per share applicable to common
stockholders
52,689
45,789
51,912
45,654
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190805005585/en/
Acceleron Pharma Inc. Investors: Todd James, IRC, 617-649-9393
Vice President, Investor Relations and Corporate Communications Ed
Joyce, 617-649-9292 Director, Investor Relations Media: Matt
Fearer, 617-301-9557 Director, Corporate Communications
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