- PULSAR Phase 2 trial results published in New
England Journal of Medicine -
- Updates from the PULSAR and SPECTRA Phase 2
trials of sotatercept in patients with pulmonary arterial
hypertension (PAH) to be presented at the American Thoracic Society
(ATS) 2021 International Conference on May 19, 2021 -
- Acceleron recognized approximately $22.4
million in royalty revenue for Q1 2021 from approximately $112
million in net sales of REBLOZYL® (luspatercept-aamt) -
- REBLOZYL, the first and only erythroid
maturation agent, was approved by Health Canada for the treatment
of transfusion-dependent anemia in adults with myelodysplastic
syndromes (MDS) -
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 first quarter
ended March 31, 2021.
“Acceleron made significant operational progress during the
first quarter,” said Habib Dable, President and Chief Executive
Officer of Acceleron. “With the STELLAR Phase 3 trial already
enrolling patients, we expect to initiate multiple mid- and
late-stage clinical trials across our pipeline in pulmonary disease
throughout 2021. We were thrilled to have data from the PULSAR
Phase 2 trial of sotatercept in PAH recently featured in the New
England Journal of Medicine. These data along with the results from
our ongoing and planned Phase 3 trials will support our vision of
establishing sotatercept as a backbone therapy for patients with
all stages of PAH. We are looking forward to hosting a research and
development day in late June to primarily discuss Acceleron's plans
and vision in rare pulmonary diseases with sotatercept in pulmonary
hypertension and ACE-1334 in systemic sclerosis-associated
interstitial lung disease, or SSc-ILD.”
Added Mr. Dable: “In our hematology program, Acceleron and our
partner, Bristol-Myers Squibb Company (Bristol Myers Squibb), are
leveraging the key learnings from the early success of the
commercial launch of REBLOZYL in the United States. We continue to
transition from the initial bolus patient group earlier in the
launch to underlying new patient demand. Bristol Myers Squibb
expects launches in multiple additional countries across the globe
this year as REBLOZYL receives reimbursement. Most importantly,
assuming success in current and future potential indications
associated with anemia, we continue to estimate that peak annual
sales of REBLOZYL will surpass $4 billion.”
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.
- In April, the New England Journal of Medicine published results
from the PULSAR Phase 2 trial of sotatercept in patients with
PAH.
- The Company plans to present updates from the PULSAR and
SPECTRA Phase 2 trials of sotatercept in patients with PAH at the
American Thoracic Society (ATS) 2021 International Conference, held
May 14-19.
- The Company will also present preclinical research on the
effects of a murine version of sotatercept in animal models of PAH
and pulmonary hypertension (PH Group 2).
- Acceleron plans to host a webcast and conference call for
investors and analysts on May 19, 2021 to discuss highlights from
the PULSAR and SPECTRA trial updates at the congress.
- Enrollment is ongoing in the registrational STELLAR Phase 3
trial in patients with PAH.
- The Company expects to have the HYPERION (newly diagnosed
intermediate and high-risk patients) Phase 3 trial and the ZENITH
(WHO Functional Class IV) Phase 3 trial in expanded PAH populations
initiated by the second 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.
- 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 $22.4 million in royalty
revenue from approximately $112 million in net sales of REBLOZYL in
the first quarter of 2021. This compares with approximately $23.0
million in royalty revenue from approximately $115 million in net
sales of REBLOZYL in the fourth quarter of 2020.
- In February, 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 units over 8 weeks resulting from very low- to
intermediate-risk MDS who have ring sideroblasts and who have
failed or are not suitable for erythropoietin-based therapy.
- Bristol Myers Squibb initiated the INDEPENDENCE Phase 3 trial
in patients with anemia-associated with myelofibrosis.
- The Companies expect to present results from the BEYOND Phase 2
trial in adult patients with non-transfusion-dependent
beta-thalassemia by the end of June 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 on
Tuesday, June 22, 2021 to primarily focus on its rare pulmonary
disease pipeline and long-term vision.
Financial Results
- Cash Position - Cash, cash equivalents and investments
as of March 31, 2021 were $795.4 million, compared with $857.5
million as of December 31, 2020. 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 first quarter of 2021 was
$24.8 million, which includes $2.4 million of cost share revenue
and $22.4 million of royalty revenue from net sales of REBLOZYL.
All revenue was derived from the Company's partnership with Bristol
Myers Squibb.
- R&D Expenses - GAAP R&D expenses were $57.3
million for the first quarter of 2021. Non-GAAP R&D expenses
were $48.2 million for the first quarter of 2021, excluding $8.2
million and $0.9 million in non-cash, stock-based compensation and
depreciation and amortization expense, respectively.
- SG&A Expenses - GAAP SG&A expenses were $31.1
million for the first quarter of 2021. Non-GAAP SG&A expenses
were $23.5 million for the first quarter of 2021, excluding $7.5
million and $0.1 million in non-cash, stock-based compensation and
depreciation and amortization expense, respectively.
- Net Loss - The Company's GAAP net loss for the first
quarter of 2021 was $63.5 million, or $1.05 per share. Non-GAAP
adjusted net loss for the first quarter was $46.8 million, or $0.77
per share, excluding $15.6 million and $1.0 million in non-cash,
stock-based compensation and depreciation and amortization expense,
respectively.
Non-GAAP Financial
Measures
Acceleron supplements its results of operations prepared in
accordance with U.S. generally accepted accounting principles, or
GAAP, with certain non-GAAP financial measures, including non-GAAP
R&D expense, non-GAAP SG&A expense, adjusted net loss and
adjusted net loss per share, that exclude stock-based compensation
expense and depreciation and amortization expense. These results
should not be viewed as a substitute for the Company’s GAAP results
and are provided as a complement to results provided in accordance
with GAAP. Management believes these non-GAAP financial measures
provide investors with additional insight into underlying trends of
the Company's ongoing business, and are important in comparing
current results with prior period results. Investors are cautioned
that there are material limitations associated with the use of
non-GAAP financial measures. In addition, other companies may
report similarly titled non-GAAP measures, but calculate them
differently, which reduces their usefulness as a comparative
measure. In the reconciliation tables below, Acceleron presents
these non-GAAP financial measures reconciled to their comparable
GAAP financial measures.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
its first quarter 2021 financial results on May 6, 2021, 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. To participate in the
conference call, please dial 833-494-1483 (domestic) or
236-714-2620 (international) and reference code #5559908.
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 Phase 1b/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)
March 31, 2021
December 31, 2020
Cash and cash equivalents
$
455,513
$
670,952
Short and long-term investments
339,929
186,536
Operating lease - right of use asset,
net
20,415
21,988
Other assets
59,936
52,861
Total assets
$
875,793
$
932,337
Operating lease liability - right of use,
short-term and long-term
$
22,425
$
24,077
Other liabilities
42,537
53,153
Total liabilities
64,962
77,230
Total stockholders’ equity
810,831
855,107
Total liabilities and stockholders’
equity
$
875,793
$
932,337
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(Amounts in thousands except
per share data)
(unaudited)
Three Months Ended March
31,
2021
2020
Revenue:
Collaboration revenue
$
24,758
$
4,344
Costs and expenses:
Research and development
57,299
37,663
Selling, general and administrative
31,062
18,253
Total costs and expenses
88,361
55,916
Loss from operations
(63,603)
(51,572)
Other, income net
137
648
Loss before income taxes
(63,466)
(50,924)
Income tax provision
(5)
(15)
Net loss
$
(63,471)
$
(50,939)
Net loss per share- basic and diluted
$
(1.05)
$
(0.95)
Weighted-average number of common shares
used in computing net loss per share- basic and diluted
60,579
53,361
ACCELERON PHARMA INC.
RECONCILIATION OF GAAP TO
NON-GAAP COSTS and EXPENSES
(Amounts in thousands)
(unaudited)
Three Months Ended March
31,
2021
2020
GAAP research and development
$
57,299
$
37,663
Less adjustments:
Stock-based compensation
8,158
3,141
Depreciation and amortization
936
932
Non-GAAP research and development
$
48,205
$
33,590
GAAP selling, general and
administrative
$
31,062
$
18,253
Less adjustments:
Stock-based compensation
7,468
3,538
Depreciation and amortization
91
63
Non-GAAP selling, general and
administrative
$
23,503
$
14,652
ACCELERON PHARMA INC.
RECONCILIATION OF GAAP TO
ADJUSTED NON-GAAP NET LOSS AND NET LOSS PER SHARE
(Amounts in thousands except
per share data)
(unaudited)
Three Months Ended March
31,
2021
2020
GAAP net loss
$
(63,471)
$
(50,939)
Adjustments:
Stock-based compensation
15,626
6,679
Depreciation and amortization
1,026
996
Adjusted net loss (non-GAAP)
$
(46,819)
$
(43,264)
GAAP net loss per share- basic and
diluted
$
(1.05)
$
(0.95)
Adjustments:
Stock-based compensation
0.26
0.13
Depreciation and amortization
0.02
0.02
Adjusted net loss per share (non-GAAP)
$
(0.77)
$
(0.80)
Weighted-average number of common shares
used in computing net loss per share
60,579
53,361
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 Company (“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
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/20210506006130/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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