- REBLOZYL® (luspatercept-aamt), a
first-in-class erythroid maturation agent, was approved by the U.S.
FDA to treat anemia in adults with lower-risk myelodysplastic
syndromes (MDS) -
- REBLOZYL received positive CHMP opinion for
the treatment of adults with transfusion-dependent anemia in
lower-risk MDS and beta-thalassemia -
- Pivotal Phase 3 MEDALIST and BELIEVE trial
results published in New England Journal of Medicine -
- Sotatercept granted Breakthrough Therapy
designation by FDA and Priority Medicines (PRIME) designation by
EMA for the treatment of patients with pulmonary arterial
hypertension (PAH) -
- PULSAR Phase 2 topline results are expected
to be presented in an American Thoracic Society (ATS) 2020
web-based conference session by the end of June 2020 -
Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company
dedicated to the discovery, development, and commercialization of
TGF-beta superfamily therapeutics to treat serious and rare
diseases, today provided a corporate update and reported financial
results for the first quarter ended March 31, 2020.
“Acceleron is off to a strong start in 2020, propelled by one of
the most eventful time periods in the Company’s history,” said
Habib Dable, President and Chief Executive Officer of Acceleron.
“The breadth and depth of our hematology program has been on full
display, owing to the publication of the MEDALIST and BELIEVE Phase
3 luspatercept trial results in the prestigious New England Journal
of Medicine and the recent FDA approval of REBLOZYL for the
treatment of anemia in a population of patients with lower-risk
MDS. We’re thrilled that this first-in-class erythroid maturation
agent, having received approvals in two distinct indications over a
span of just five months, is now available in the U.S. to patients
who have long needed a new option for treating their chronic
anemias.”
Added Mr. Dable: “Our scientific expertise in leveraging the
therapeutic potential of the TGF-beta superfamily of proteins is
delivering equally exciting results in pulmonary disease. With
great enthusiasm, we announced that the PULSAR Phase 2 trial of
sotatercept in patients with PAH met its primary and key secondary
endpoints - underscoring our belief that sotatercept could
eventually alter the current treatment paradigm. Following the
results, sotatercept was granted Breakthrough Therapy designation
by the FDA and PRIME designation by the EMA. We look forward to
presenting topline results from PULSAR in a virtual meeting of the
ATS as well as interactions with health authorities as we prepare
for future Phase 3 development of sotatercept on our path to
potential global registration.”
Program Highlights
Hematology
REBLOZYL (luspatercept-aamt):
Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and
Myelofibrosis (MF) REBLOZYL is the first and only U.S.
FDA approved erythroid maturation agent designed to promote red
blood cell production through a novel mechanism. Luspatercept-aamt
is being developed to treat anemia in patients with
beta-thalassemia, MDS, and MF. REBLOZYL is part of the global
collaboration between Acceleron and Bristol Myers Squibb.
- In April, Acceleron and partner Bristol Myers Squibb announced
the FDA approved REBLOZYL for the treatment of anemia failing an
erythropoiesis stimulating agent and requiring 2 or more red blood
cell (RBC) units over 8 weeks in adult patients with very low- to
intermediate-risk MDS with ring sideroblasts (MDS-RS) or with
myelodysplastic/myeloproliferative neoplasm with ring sideroblasts
and thrombocytosis (MDS/MPN-RS-T).
- The New England Journal of Medicine published results from the
pivotal Phase 3 MEDALIST and BELIEVE trials.
- In April, REBLOZYL received a positive opinion from the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) for the treatment of adult patients
with:
- transfusion-dependent anemia due to very low-, low- and
intermediate-risk MDS with ring sideroblasts, who had an
unsatisfactory response or are ineligible for erythropoietin-based
therapy; and
- transfusion-dependent anemia associated with
beta-thalassemia
- Bristol Myers Squibb expects to initiate the Phase 3
INDEPENDENCE trial in patients with MF on concomitant JAK 2
inhibitor therapy who require RBC transfusions by year-end
2020.
- The BEYOND Phase 2 trial in adult patients with
non-transfusion-dependent beta-thalassemia is ongoing, with topline
results expected by year-end 2020 or early 2021.
- Enrollment is ongoing in the COMMANDS Phase 3 trial in patients
with treatment-naïve, lower-risk MDS, with topline results expected
in 2022.
Pulmonary
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
(RAP-011) reversed pulmonary vessel muscularization and improved
indicators of right heart failure.
- In January, Acceleron reported that the PULSAR Phase 2 trial in
patients with PAH met its primary and key secondary endpoints.
Sotatercept was generally well tolerated in the trial and adverse
events observed were generally consistent with previously published
data on sotatercept in other diseases.
- PULSAR Phase 2 topline results are expected to be presented in
an American Thoracic Society (ATS) 2020 web-based session by the
end of June 2020.
- Acceleron plans to host a webcast and conference call for
investors and analysts following the ATS 2020 virtual presentation
to discuss highlights from the PULSAR trial results.
- In April, sotatercept was granted Breakthrough Therapy
designation by the FDA for the treatment of patients with PAH
(World Health Organization Group 1).
- The EMA recently granted Priority Medicines (PRIME) designation
to sotatercept for the treatment of patients with PAH.
- 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 March 31, 2020 were $415.6 million, as compared to $453.8
million as of December 31, 2019. 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 REBLOZYL sales.
- Revenue - Revenue for the first quarter of 2020 was $4.3
million, which includes $2.8 million of cost share revenue, and
$1.5 million of royalty revenue from net U.S. sales of REBLOZYL.
All revenue was derived from the Company's partnership with Bristol
Myers Squibb.
- Costs and Expenses - Total costs and expenses for the
first quarter of 2020 were $55.9 million. This includes R&D
expenses of $37.7 million and SG&A expenses of $18.3
million.
- Net Loss - The Company's net loss for the first quarter
of 2020 was $50.9 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
its first quarter 2020 financial results and provide an update on
recent corporate activities on May 11, 2020, 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 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 2020
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 biopharmaceutical company dedicated to the
discovery, development, and commercialization of therapeutics to
treat serious and rare diseases. Acceleron’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 commercialization, research, and
development efforts in hematologic and pulmonary diseases. In
hematology, Acceleron and its global collaboration partner, Bristol
Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the
first and only approved erythroid maturation agent, in the United
States for the treatment of anemia in certain blood disorders. The
Companies are also developing luspatercept for the treatment of
anemia in patient populations of MDS, beta-thalassemia, and
myelofibrosis. In pulmonary, Acceleron is developing sotatercept
for the treatment of pulmonary arterial hypertension, having
recently reported positive topline results of the Phase 2 PULSAR
trial.
For more information, please visit acceleronpharma.com. Follow
Acceleron on social media: @AcceleronPharma and LinkedIn.
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED BALANCE
SHEET
(Amounts in thousands)
(unaudited)
March 31, 2020
December 31, 2019
Cash and cash equivalents
$
277,486
$
237,677
Short and long-term investments
138,077
216,169
Operating lease - right of use asset,
net
22,531
23,908
Other assets
26,068
27,152
Total assets
$
464,162
$
504,906
Operating lease liability - right of use,
short-term and long-term
$
24,918
$
26,384
Warrants to purchase common stock
3,304
1,856
Other liabilities
22,118
27,190
Total liabilities
50,340
55,430
Total stockholders’ equity
413,822
449,476
Total liabilities and stockholders’
equity
$
464,162
$
504,906
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(Amounts in thousands except
per share data)
(unaudited)
Three Months Ended March 31,
2020
2020
2019
Revenue:
Collaboration revenue:
$
4,344
$
2,780
Costs and expenses:
Research and development
37,663
32,771
Selling, general and administrative
18,253
10,814
Total costs and expenses
55,916
43,585
Loss from operations
(51,572
)
(40,805
)
Other income, net
648
2,772
Loss before income taxes
(50,924
)
(38,033
)
Income tax provision
(15
)
(20
)
Net loss
$
(50,939
)
$
(38,053
)
Net loss per share- basic and diluted
$
(0.95
)
$
(0.74
)
Weighted-average number of common shares
used in computing net loss per share- basic and diluted
53,361
51,126
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 and commercialization of the
Company's compounds, the timeline for clinical development and
regulatory approval of the Company's compounds, the expected timing
for reporting of data from ongoing clinical trials, the Company's
future cash position and the potential of REBLOZYL®
(luspatercept-aamt) as a therapeutic drug. 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 other clinical
trials, that regulatory approval of the Company’s compounds in one
indication or country may not be predictive of approval in another
indication or country, that the development of the Company’s
compounds will take longer and/or cost more than planned or
accelerate faster than currently expected, that the Company or its
collaboration partner, Bristol-Myers Squibb Corporation (“BMS”),
will be unable to successfully complete the clinical development of
the Company’s compounds, that the Company or BMS 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,
Quarterly Report on Form 10-Q, 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/20200511005852/en/
Acceleron Pharma Inc. Investors: Todd James, 617-649-9393 Senior
Vice President, Corporate Affairs and Investor Relations
Media: Matt Fearer, 617-301-9557 Director, Corporate
Communications
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