- Updates from ongoing PULSAR and SPECTRA Phase
2 clinical trials of sotatercept in patients with pulmonary
arterial hypertension (PAH) presented at the American Heart
Association (AHA) 2020 Scientific Sessions -
- STELLAR Phase 3 trial initiated with
sotatercept in PAH -
- Acceleron recognized approximately $23.0
million in royalty revenue from approximately $115 million in net
sales of REBLOZYL® (luspatercept-aamt) for Q4 2020 and
approximately $54.8 million in royalty revenue from approximately
$274 million in net sales of REBLOZYL for full year 2020 -
- Multiple clinical presentations on REBLOZYL
reviewed at the 62nd American Society of Hematology (ASH) Annual
Meeting & Exposition -
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 reported financial results for the
fourth quarter and full year ended December 31, 2020.
“2020 was a very productive year for Acceleron as we generated
important results in our lead pulmonary program to advance
sotatercept into a registrational Phase 3 trial and executed on a
successful first full year of the commercial launch of REBLOZYL in
the United States,” said Habib Dable, President and Chief Executive
Officer of Acceleron. “I would like to acknowledge our team's
strong commitment to our company and mission as these important
milestones along with many others were achieved despite the many
challenges resulting from the ongoing global pandemic. Throughout
the year, we presented updates from our two Phase 2 trials with our
lead pulmonary program, supporting our long-term vision of
establishing sotatercept as a backbone therapy for patients with
pulmonary arterial hypertension across all stages of the
disease."
Added Mr. Dable: "As we continue to grow our capabilities in
rare pulmonary disease, we are also advancing ACE-1334 into a
Phase1b/Phase 2 trial in patients with systemic
sclerosis-associated interstitial lung disease later this year. We
look forward to hosting a research and development day to provide
additional details on our long-term vision and programs in rare
pulmonary disease in the middle of 2021.”
Program Highlights
Pulmonary
Sotatercept: Pulmonary Arterial
Hypertension (PAH)
Sotatercept is an investigational reverse-remodeling 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. The PULSAR Phase 2 trial evaluating sotatercept in
combination with approved PAH-specific medicines in patients with
PAH achieved its primary endpoint of improvement in pulmonary
vascular resistance and its key secondary endpoint of improvement
in 6-minute walk distance.
- In November, the Company presented the 24-week echocardiography
results from the PULSAR Phase 2 trial at the virtual 2020 American
Heart Association (AHA) Scientific Sessions, which earned the AHA's
"Cardiopulmonary Best Abstract" award. In addition, preliminary
interim results from the SPECTRA Phase 2 trial were presented at
the congress.
- In December, the European Commission (EC) granted Orphan Drug
designation to sotatercept for the treatment of patients with
PAH.
- In December, Acceleron initiated its registrational STELLAR
Phase 3 trial in patients with PAH.
- The Company expects to initiate the HYPERION (early
intervention) Phase 3 trial and the ZENITH (WHO Functional Class
IV) Phase 3 trial in expanded PAH populations in the middle of
2021.
- Results from the open-label extension period of the PULSAR
Phase 2 trial and additional results from the SPECTRA Phase 2 trial
are expected in the first half of 2021.
ACE-1334: Systemic Sclerosis-associated
Interstitial Lung Disease (SSc-ILD)
ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based
ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands
but not TGF-beta 2. ACE-1334 has shown robust anti-fibrotic
activity in multiple preclinical models of fibrosis. ACE-1334
recently completed an ascending-dose Phase 1 clinical trial in
healthy volunteers.
- In December, the United States Food and Drug Administration
(FDA) granted Orphan Drug designation to ACE-1334 for the treatment
of patients with systemic sclerosis.
- Acceleron expects to start a Phase 1b/Phase 2 study to evaluate
the activity of ACE-1334 in patients with SSc-ILD in 2021.
Hematology
REBLOZYL
(luspatercept-aamt):
REBLOZYL is the first and only approved erythroid maturation
agent designed to promote late-stage red blood cell (RBC)
production. REBLOZYL is part of the global collaboration between
Acceleron and Bristol Myers Squibb.
- The Company recognized approximately $23.0 million in royalty
revenue from approximately $115 million in net sales of REBLOZYL in
the fourth quarter of 2020. This compares with approximately $19.3
million in royalty revenue from approximately $96 million in net
sales of REBLOZYL in the third quarter of 2020.
- For the full year 2020, Acceleron recognized approximately
$54.8 million in royalty revenue from approximately $274 million in
net sales of REBLOZYL.
- In February 2021, Acceleron and partner Bristol Myers Squibb
announced that Health Canada approved REBLOZYL for the treatment of
adult patients with transfusion-dependent anemia requiring at least
two RBC red blood cell units over 8 weeks resulting from very low-
to intermediate-risk myelodysplastic syndromes (MDS) who have ring
sideroblasts and who have failed or are not suitable for
erythropoietin-based therapy.
- Six clinical abstracts on REBLOZYL were presented at the 62nd
American Society of Hematology (ASH) Annual Meeting &
Exposition held virtually in December 2020.
- The INDEPENDENCE Phase 3 trial is initiating in patients with
myelofibrosis.
- The Companies expect to present results from the BEYOND Phase 2
trial in adult patients with non-transfusion-dependent
beta-thalassemia in the first half of 2021.
- Enrollment is ongoing in the COMMANDS Phase 3 trial in patients
with first-line lower-risk MDS, with topline results expected in
2022+.
Corporate Highlights
- Acceleron is planning to host a research and development day in
the middle of 2021 to provide a deep dive into all of the Company's
programs.
Financial Results
- Cash Position – Cash, cash equivalents and investments
as of December 31, 2020 were $857.5 million. Based on Acceleron's
current operating plan and projections, the Company believes that
its current cash, cash equivalents and investments, along with the
expected royalty revenue from REBLOZYL sales, will be sufficient to
fund the Company’s projected operating requirements for the
foreseeable future.
- Revenue – Revenue for the fourth quarter of 2020 was
$25.9 million, which includes $2.9 million of cost share revenue
and $23.0 million of royalty revenue from net sales of REBLOZYL.
Revenue for the full year was $92.5 million, which includes $12.7
million of cost share revenue, $54.8 million of royalty revenue
from net sales of REBLOZYL, and the recognition of a $25.0 million
regulatory-based milestone for the approval of REBLOZYL in Europe.
All revenue was derived from the Company's partnership with Bristol
Myers Squibb.
- Costs and Expenses – Total costs and expenses for the
fourth quarter of 2020 were $83.5 million. This includes R&D
expenses of $57.3 million and SG&A expenses of $26.2 million.
Total costs and expenses for the full year were $259.8 million.
This includes R&D expenses of $173.9 million and SG&A
expenses of $85.9 million.
- Net Loss – The Company's net loss for the fourth quarter
of 2020 was $57.4 million. The Company’s net loss for the year
ended December 31, 2020 was $166.0 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
its fourth quarter and full year 2020 financial results on February
25, 2021, 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. To
participate in the conference call, please dial 833-494-1483
(domestic) or 236-714-2620 (international) and reference code
#4282162.
An archived version of the webcast will be available for replay
on the Company's website for approximately one year.
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 research, development, and
commercialization efforts in pulmonary and hematologic diseases. In
pulmonary, Acceleron is developing sotatercept for the treatment of
pulmonary arterial hypertension (PAH), having reported positive
topline results of the PULSAR Phase 2 trial. The Company is
currently planning multiple Phase 3 trials with the potential to
support its long-term vision of establishing sotatercept as a
backbone therapy for patients with PAH at all stages of the
disease. Acceleron is also investigating the potential of its
early-stage pulmonary candidate, ACE-1334, which it plans to
advance into a Phase1b/Phase 2 trial in systemic
sclerosis-associated interstitial lung disease (SSc-ILD) this
year.
In hematology, REBLOZYL® (luspatercept-aamt) is the first and
only erythroid maturation agent approved in the United States,
Europe, and Canada for the treatment of anemia in certain blood
disorders. REBLOZYL is part of a global collaboration partnership
with Bristol Myers Squibb. The Companies co-promote REBLOZYL in the
United States and are also developing luspatercept for the
treatment of anemia in patient populations of myelodysplastic
syndromes, beta-thalassemia, and myelofibrosis.
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,
2020
2019
Cash and cash equivalents
$
670,952
$
237,677
Short and long-term investments
186,536
216,169
Operating lease assets
21,988
23,908
Other assets
52,861
27,152
Total assets
$
932,337
$
504,906
Short-term and long-term operating lease
liabilities
$
24,077
$
26,384
Warrants to purchase common stock
—
1,856
Other liabilities
53,153
27,190
Total liabilities
77,230
55,430
Total stockholders’ equity
855,107
449,476
Total liabilities and stockholders’
equity
$
932,337
$
504,906
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(Amounts in thousands except
per share data)
(unaudited)
Three Months Ended December
31,
Year Ended December
31,
2020
2019
2020
2019
Revenue:
Collaboration revenue
$
25,866
$
39,338
$
92,523
$
73,993
Costs and expenses:
Research and development
57,254
48,852
173,917
153,953
Selling, general and administrative
26,207
16,067
85,912
56,485
Total costs and expenses
83,461
64,919
259,829
210,438
Loss from operations
(57,595)
(25,581)
(167,306)
(136,445)
Total other income, net
190
2,002
1,297
11,525
Loss before income taxes
(57,405)
(23,579)
(166,009)
(124,920)
Income tax (provision) benefit
9
4
(21)
62
Net loss
$
(57,396)
$
(23,575)
$
(166,030)
$
(124,858)
Net loss per share - basic and diluted
$
(0.95)
$
(0.44)
$
(2.92)
$
(2.38)
Weighted-average number of common shares
used in computing net loss per share
60,269
46,227
56,800
52,453
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 may take longer and/or cost more than planned or
accelerate faster than currently expected, that the Company or its
collaboration partner, Bristol Myers Squibb (“BMS”), may 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 may 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/20210225006022/en/
Acceleron Pharma Inc. Investors: Jamie Bernard, IRC, (617)
301-9650 Associate Director, Investor Relations
Media: Matt Fearer, (617) 301-9557 Senior Director, Corporate
Communications
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