– Top-line results from the MEDALIST and
BELIEVE Phase 3 trials of luspatercept are expected in mid-2018
–
– Plans to advance neuromuscular agent ACE-083
into Part 2 of the ongoing Phase 2 trials in facioscapulohumeral
dystrophy and Charcot-Marie-Tooth disease –
– Expanded pipeline with sotatercept in
pulmonary disease; on track to initiate a Phase 2 trial in
pulmonary arterial hypertension in 1H 2018 –
Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical
company in the discovery and development of TGF-beta 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, 2017.
“2017 was an important year for Acceleron as we laid out our
corporate strategy and vision for future growth. We are committed
to building therapeutic area leadership and advancing our clinical
programs in three areas of high unmet medical need in hematologic,
neuromuscular, and pulmonary disease,” said Habib Dable, Chief
Executive Officer of Acceleron. “With this progress, we are well
positioned for the transformative year ahead. In hematology, we
look forward to announcing top-line results from our MEDALIST and
BELIEVE Phase 3 trials of luspatercept in mid-2018. We and Celgene
continue to drive preparations and key activities for regulatory
and commercial readiness. In neuromuscular, we are advancing
ACE-083, our locally acting ‘Myostatin+’ agent, through Phase 2
trials in two distinct neuromuscular diseases. In pulmonary, we
plan to initiate a Phase 2 study with sotatercept in pulmonary
arterial hypertension. Our entire team remains focused on near-term
execution as we prepare for long-term value creation and aim to
bring new transformative therapies to patients.”
Development Program
Highlights
Hematology
Luspatercept:
Myelodysplastic Syndromes (MDS),
Beta-Thalassemia, and Myelofibrosis (MF)Luspatercept is
designed to treat chronic anemia and reduce red blood cell (RBC)
transfusion burden in adults with rare blood disorders.
Luspatercept is being developed as part of the global collaboration
between Acceleron and Celgene.
- Top-line results from the MEDALIST and
BELIEVE Phase 3 trials in MDS and beta-thalassemia, respectively,
are expected in mid-2018.
- The initiation of the COMMANDS Phase 3
trial in first-line, lower-risk MDS patients is planned for the
first half of 2018.
- The BEYOND Phase 2 trial in
non-transfusion-dependent beta-thalassemia was recently initiated
and enrollment is ongoing in the Phase 2 trial in MF.
- Updated results from the ongoing Phase
2 trial in lower-risk MDS patients were presented at the American
Society of Hematology (ASH) 2017 annual meeting. Multiple patients
are now nearing three years on treatment.
Neuromuscular Disease
ACE-083:
Facioscapulohumeral Muscular Dystrophy
(FSHD) and Charcot-Marie-Tooth (CMT) diseaseACE-083 is a
locally-acting therapeutic, based on the naturally-occurring
protein follistatin, designed to have a concentrated effect on
muscle mass and strength in target muscles for diseases that cause
debilitating focal muscle weakness. ACE-083 utilizes the
"Myostatin+" approach to inhibit multiple TGF-beta ligands.
- Preliminary results from dose cohorts 1
and 2 in Part 1 of the ACE-083 Phase 2 trial in patients with FSHD
demonstrated mean total muscle volume increases of over 12% in the
tibialis anterior and biceps brachii muscle cohorts. In addition,
both of these cohorts demonstrated a reduction in fat fraction.
- Part 1 of the trial is fully enrolled
with treatment ongoing in dose cohort 3; the Company plans to begin
Part 2 of the Phase 2 trial in the second quarter of this
year.
- Enrollment and treatment are ongoing in
Part 1 of the Phase 2 trial in patients with CMT disease. The
Company plans to present preliminary Part 1 results in the second
half of 2018 and to initiate Part 2 of the Phase 2 trial by the end
of 2018.
ACE-2494:ACE-2494 is
designed to have a systemic effect on muscle mass and strength
throughout the body by utilizing the "Myostatin+" approach to
inhibit multiple TGF-beta ligands.
- The Phase 1 clinical trial has been
initiated and preliminary results from this healthy volunteer study
are expected in the first half of 2019.
Pulmonary Disease
Sotatercept:
Pulmonary Arterial Hypertension
(PAH)Sotatercept is an activin receptor type IIA fusion
protein that acts as a ligand trap for members in the TGF-beta
protein superfamily involved in the remodeling of a variety of
different tissues, including the vasculature and fibrotic
tissue.
- Preclinical results were presented at
the American Heart Association 2017 Scientific Sessions
demonstrating that sotatercept decreased vessel muscularization,
improved pulmonary arterial pressures, and decreased indicators of
right heart failure.
- Clinical activities are underway to
initiate the planned Phase 2 trial in PAH in the first half of
2018.
- The Company plans to host an
educational webinar to discuss the Phase 2 trial design in the
first half of 2018.
Financial Results
- Cash Position – Cash, cash
equivalents and investments as of December 31, 2017 were
$372.9 million. As of December 31, 2016 the Company had cash, cash
equivalents and investments of $234.4 million. Cash, cash
equivalents and investments include $215.8 million of net proceeds
from a follow-on public offering of common stock in 2017. We
believe that our existing cash, cash equivalents and investments
will be sufficient to fund projected operating requirements into
2021.
- Revenue – Collaboration revenue
for the year was $13.5 million. The revenue is all from our
Celgene partnership and is primarily due to cost sharing revenue of
$12.9 million related to expenses incurred by the Company in
support of our partnered programs.
- Costs and expenses – Total costs
and expenses for the year were $123.5 million. This includes
R&D expenses of $89.7 million and G&A expenses of $33.7
million.
- Net Loss – The Company’s net
loss for the year ended December 31, 2017 was $108.5
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 2017 and
provide an update on recent corporate activities on February 27,
2018, 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 2017 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 Cambridge-based, 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 pulmonary
program with a planned Phase 2 trial of 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, 2017 December 31, 2016 Cash and
cash equivalents $ 100,150 $ 20,950 Short and long-term investments
272,800 213,432 Other assets 16,227 13,264 Total assets $ 389,177 $
247,647 Deferred revenue $ 3,702 $ 4,245 Warrants to
purchase common stock 2,236 1,244 Other liabilities 18,021 16,562
Total liabilities 23,960 22,050 Total stockholders’ equity 365,217
225,597 Total liabilities and stockholders’ equity $ 389,177 $
247,647
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, 2017 2016 2017
2016 Revenue:
Collaboration revenue $ 3,705 $ 3,369 $ 13,481 $ 27,771 Costs and
expenses: Research and development 25,339 19,088 89,726 68,580
General and administrative 7,002 6,267 33,738
25,297 Total costs and expenses 32,341 25,355 123,464 93,877
Loss from operations (28,636 ) (21,986 ) (109,983 ) (66,106 ) Total
other income, net 770 2,742 1,561 9,116
Loss before income taxes (27,866 ) (19,244 ) (108,422 ) (56,990 )
Income tax provision (60 ) (44 ) (32 ) (24 ) Net loss $ (27,926 ) $
(19,288 ) $ (108,454 ) $ (57,014 ) Net loss per share -
basic and diluted $ (0.62 ) $ (0.51 ) $ (2.68 ) $ (1.52 )
Weighted-average number of common shares used in computing net loss
per share 45,217 37,914 40,420 37,430
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 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 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: http://www.businesswire.com/news/home/20180227006474/en/
For Acceleron Pharma Inc.IRCTodd James, 617-649-9393Vice
President, Investor Relations and Corporate CommunicationsorCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
646-513-3125
Acceleron Pharma (NASDAQ:XLRN)
Gráfica de Acción Histórica
De Jun 2024 a Jul 2024
Acceleron Pharma (NASDAQ:XLRN)
Gráfica de Acción Histórica
De Jul 2023 a Jul 2024