– Phase 2 data from luspatercept trials in
β-thalassemia and MDS patients given as oral presentations at ASH
and MDS abstract included in “Best of ASH” session –
– Plans with Celgene to initiate phase 3
clinical trials in both β-thalassemia and MDS in 2015 –
– Sotatercept slowed the progression of
vascular calcification in patients with end stage kidney disease
–
– Preliminary data from dalantercept phase 2
trial in renal cell carcinoma patients demonstrated encouraging
progression-free survival rate –
– Initiated phase 1 clinical trial with
innovative muscle drug ACE-083 –
Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage
biopharmaceutical company focused on the discovery, development and
commercialization of novel therapeutic candidates that regulate
cellular growth and repair, today provided a corporate update and
reported financial results for the fourth quarter and year ended
December 31, 2014.
“Acceleron made enormous progress in 2014 by demonstrating
encouraging proof-of-concept data in both MDS and β-thalassemia,
providing the basis to advance into phase 3 clinical trials in each
disease in 2015. This will be transformative for Acceleron and the
patients we aim to serve with our innovative therapeutic
candidates,” said John Knopf, Ph.D., Chief Executive Officer of
Acceleron. “Additionally, we generated promising data in several
other ongoing phase 2 clinical trials while expanding our robust
pipeline by bringing a fourth internally discovered therapeutic
candidate into phase 1 clinical development. We look forward to
building on the positive momentum generated in 2014 for our entire
pipeline in 2015.”
Recent Highlights and Current Updates
Clinical Development Programs
- Completed enrollment in the
luspatercept myelodysplastic syndromes (MDS) phase 2 clinical
trial – The ongoing phase 2 clinical trial of luspatercept in
MDS patients is now fully enrolled. This clinical trial was
designed as a two-stage study. The first stage was a dose
escalation design and the second stage, the dose expansion stage,
allows each patient’s dose of luspatercept to be titrated to
maximize the potential clinical benefit for that patient. Data from
the dose expansion cohort of patients will inform the design of the
phase 3 trial in MDS patients that is expected to start by the end
of this year.
- Luspatercept and sotatercept
increased hemoglobin levels and established transfusion
independence in patients with lower risk MDS – Preliminary data
from the ongoing phase 2 clinical trials in patients with lower
risk MDS presented at the 56th American Society of Hematology (ASH)
Annual Meeting and Exposition in San Francisco, CA demonstrated
increases in hemoglobin levels, transfusion independence, and the
identification of potential biomarkers that may be used to select
patients most likely to respond to treatment.
- Luspatercept phase 2 clinical data
in patients with lower risk MDS was selected for “Best of ASH” at
the 56th ASH Annual Meeting and Exposition – The
“Best of ASH” session, led by the 2014 Annual Meeting Scientific
Program Co-Chairs, highlighted twenty-four of the top hematology
breakthroughs selected from the meeting’s more than 4,000 abstract
presentations.
- Luspatercept increased hemoglobin
levels, reduced transfusion burden and improved measures of iron
overload in β-thalassemia patients – Preliminary data from the
ongoing phase 2 clinical trial in patients with β-thalassemia
presented at the 56th ASH Annual Meeting and Exposition
demonstrated increases in hemoglobin levels, reductions in
transfusion burden, reductions in markers of iron overload and
substantial improvement in clinical complications such as leg
ulcers.
- Phase 3 studies planned for 2015
– In December, Celgene, Acceleron’s collaboration partner for the
sotatercept and luspatercept programs, announced its intention to
initiate phase 3 clinical trials in β-thalassemia patients and MDS
patients in 2015, based on the encouraging data from the ongoing
phase 2 clinical trials.
- Initiated long-term extension
studies of luspatercept in β-thalassemia patients and MDS
patients – Acceleron initiated two extension studies to
evaluate the long-term safety and tolerability of luspatercept in
patients with β-thalassemia and MDS.
- Sotatercept slowed the progression
of vascular calcification and improved abnormal changes in bone
mineral density – Preliminary clinical data demonstrating
encouraging effects of sotatercept on vascular calcification, bone
mineral density, and hemoglobin levels in patients with end-stage
renal disease on hemodialysis were presented at the American
Society of Nephrology (ASN) Kidney Week 2014 meeting in
Philadelphia, PA. Acceleron’s collaboration partner, Celgene, is
conducting the phase 2 clinical trial of sotatercept in patients
with end-stage renal disease on hemodialysis.
- Preliminary data from dalantercept
phase 2 clinical trial in renal cell carcinoma patients
demonstrated encouraging progression-free survival rates –
Preliminary data from Part 1 of the DART study were presented in an
oral session at the American Society of Clinical Oncology (ASCO)
2015 Genitourinary Cancers Symposium in Orlando, FL. The
preliminary median overall progression-free survival (PFS) of
dalantercept plus axitinib was 8.3 months across all dose levels
tested in Part 1. The median PFS of the 0.9 mg/kg cohort has not
yet been reached.
- Initiated a phase 1 clinical trial
with innovative muscle drug, ACE-083 – ACE-083 is designed to
increase muscle mass and strength selectively in muscles in which
the drug is directly administered by blocking proteins in the
Transforming Growth Factor-β superfamily that inhibit muscle
growth.
Upcoming Clinical Data Presentations in the 2nd
Quarter of 2015
- International Symposium on MDS –
Presentation of new clinical data from the luspatercept and
sotatercept phase 2 clinical trials in patients with lower risk MDS
at the 13th International Symposium on Myelodysplastic Syndromes in
Washington, D.C. in April 2015.
- ERA-EDTA Congress – Presentation
of new clinical data on changes in vascular calcification and bone
mineral density from the ongoing phase 2a study of sotatercept in
end-stage renal disease patients on hemodialysis will be given at
the 52nd European Renal Association – European Dialysis and
Transplant Association (ERA-EDTA) Congress in London, UK in May
2015.
- EHA 20th Annual
Congress – Presentation of new clinical data from the ongoing
phase 2 clinical trials in patients with β-thalassemia and MDS will
be presented at the European Hematology Association 20th Annual
Congress in Vienna, Austria in June 2015.
- ASCO Annual Meeting – From the
dalantercept phase 2 clinical trial in patients with advanced renal
cell carcinoma, we expect to provide a presentation at the American
Society of Clinical Oncology (ASCO) 51st Annual Meeting in Chicago,
IL in June 2015.
Other Business Highlights
- Francois Nader, M.D. appointed to
the Acceleron Board of Directors – Dr. Francois Nader was
appointed to the Board in December 2014. Dr. Nader served as the
President and Chief Executive Officer of NPS Pharmaceuticals, Inc.,
or NPS, from 2008 through February 2015 when NPS was acquired by
Shire plc. Dr. Nader joined NPS in 2006 and served as Executive
Vice President and Chief Operating Officer until 2008. He has
served as a Director and Chair of the Compensation Committee of
Trevena, Inc. since January 2014, and as a Director of Clementia
Pharmaceuticals, Inc. since March 2014. Before joining NPS, Dr.
Nader was a venture partner at Care Capital, LLC, where he served
as Chief Medical Officer of its Clinical Development Capital unit
from 2005 to 2006. From 2000 to 2004, he served as Senior Vice
President, Integrated Healthcare Markets and Senior Vice President,
North America Medical and Regulatory Affairs with Aventis
Pharmaceuticals. He also held similar positions at Hoechst Marion
Roussel and served as Head of Global Commercial Operations at the
Pasteur Vaccines division of Rhone-Poulenc. Dr. Nader is the
immediate past Chair of the Board of BioNJ, a trade association
representing the biotechnology industry in New Jersey, and a Board
member of the New Jersey Chamber of Commerce. He received a French
State Doctorate in Medicine from St. Joseph University (Lebanon)
and a Physician Executive M.B.A. from the University of Tennessee.
We believe Dr. Nader's extensive experience in the life sciences
industry and his background in research and development qualify him
to serve as a member of our Board of Directors.
Financial Results
- Cash Position – Cash and cash
equivalents as of December 31, 2014 were $176.5 million. Acceleron
expects that its cash and cash equivalents will be sufficient to
fund the Company’s operations into the second half of 2017.
- Revenue – Collaboration revenue
for the year was $14.6 million. This includes license and milestone
amortization of $1.7 million and cost sharing reimbursement revenue
from our Celgene partnership of $13.0 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 $70.1 million. This includes R&D
expenses of $50.9 million, G&A expenses of $14.2 million and a
one-time litigation settlement expense of $5.0 million.
- Net Loss – The Company’s net
loss for the year ended December 31, 2014 was $51.3 million.
Conference Call, Webcast and Prepared Statement
Information
The company will host a conference call and live audio webcast
to report its fourth quarter and full year financial results for
2014 and provide a corporate update on March 2, 2015, at 8:00 AM
EST. To participate by teleconference, please dial 877-312-5848
(domestic) or 253-237-1155 (international) and refer to the
“Acceleron Q4 Earnings Call.” To access the live webcast and read
the Company’s prepared statement for this earnings call, please
select “Events & Presentations” in the “Investors & Media”
section on the Company’s website (www.acceleronpharma.com). To ensure a timely
connection, it is recommended that users register at least 15
minutes prior to the scheduled webcast.
A replay of the webcast will be archived on the Company’s
website and accessible approximately two hours after the event.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused
on the discovery, development and commercialization of novel
therapeutic candidates that regulate cellular growth and repair.
The company is a leader in understanding the biology of the
Transforming Growth Factor-Beta (TGF-β) protein superfamily, a
large and diverse group of molecules that are key regulators in the
growth and repair of tissues throughout the human body, and in
targeting these pathways to develop important new medicines.
Acceleron has built a highly productive R&D platform that has
generated innovative clinical and preclinical therapeutic
candidates with novel mechanisms of action. These therapeutic
candidates have the potential to significantly improve clinical
outcomes for patients with cancer and rare diseases.
We routinely post information that may be important to investors
in the “Investors and Media” section of our website at www.acceleronpharma.com. We encourage investors
and potential investors to regularly consult our website for
important information about us.
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED BALANCE
SHEET
(Amounts in thousands)
(unaudited)
December 31,2014
December 31,2013
Cash and cash equivalents $ 176,460 $ 113,163 Other assets 9,836
10,569 Total assets $ 186,296 $ 123,732
Deferred revenue $ 5,978 $ 7,651 Notes payable, net of discount —
16,868 Warrants to purchase common stock 14,124 30,753 Other
liabilities 9,909 10,648 Total liabilities 30,011 65,920
Total stockholders’ equity 156,285 57,812 Total liabilities
and stockholders’ equity $ 186,296 $ 123,732
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS
(Amounts in thousands except per share
data)
(unaudited)
Three Months EndedDecember
31,
Year Ended December 31, 2014
2013 2014 2013
Revenue: Collaboration revenue $ 3,739 $ 11,521 $ 14,632 $ 57,230
Costs and expenses: Research and development 14,579 10,216 50,897
36,051 Litigation settlement — — 5,000 — General and administrative
3,714 4,756 14,199 14,227 Total costs
and expenses 18,293 14,972 70,096 50,278 Loss from operations
(14,554 ) (3,451 ) (55,464 ) 6,952 Other (expense) income, net
(3,063 ) (14,659 ) 4,205 (28,850 ) Net loss $ (17,617 ) $
(18,110 ) $ (51,259 ) $ (21,898 )
Accretion of dividends, interest,
redemption value and issuancecosts on redeemable convertible
preferred stock
— — — (19,870 ) Gain on extinguishment of redeemable convertible
preferred stock — — — 2,765 Net loss
applicable to common stockholders—basic and diluted $ (17,617 ) $
(18,110 ) $ (51,259 ) $ (39,003 )
Net loss per share applicable to common
stockholders-basic anddiluted
$ (0.55 ) $ (0.64 ) $ (1.63 ) $ (4.15 )
Weighted-average number of common shares
used in computingnet loss per share applicable to common
stockholders-basic anddiluted
32,293 28,123 31,515 9,407
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements about the
Company’s strategy, future plans and prospects, including
statements regarding the development of the Company’s compounds,
including sotatercept, luspatercept, dalantercept, or ACE-083 and
the Company’s TGF-β superfamily program generally, the timeline for
clinical development and regulatory approval of the Company’s
compounds, the expected timing for the reporting of data from
ongoing trials, and the structure of the Company’s planned or
pending clinical trials. The words “anticipate,” “appear,”
“believe,” “estimate,” “expect,” “intend,” “may,” “plan,”
“predict,” “project,” “target,” “potential,” “will,” “would,”
“could,” “should,” “continue,” and similar expressions are intended
to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Each
forward-looking statement is subject to risks and uncertainties
that could cause actual results to differ materially from those
expressed or implied in such statement. Applicable risks and
uncertainties include the risks that the Company’s cash position
will be insufficient to fund operations into the second half of
2017, that preclinical testing of the Company’s compounds and
preliminary data from clinical trials may not be predictive of the
results or success of ongoing or later clinical trials, that data
may not be available when we expect it to be, that the Company or
its collaboration partner, Celgene, will be unable to successfully
complete the clinical development of its compounds, that the
development of the Company’s compounds will take longer or cost
more than planned, that the Company may be delayed in initiating or
completing any clinical trials, and that the Company’s compounds
will not receive regulatory approval or become commercially
successful products. Other risks and uncertainties include those
identified under the heading “Risk Factors” included in the
Company’s Annual Report on Form 10-K which was filed with the
Securities and Exchange Commission (SEC) on March 2, 2015, 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 reflect the Company’s current views with respect to
future events, and the Company does not undertake and specifically
disclaims any obligation to update any forward-looking
statements.
Acceleron Pharma Inc.Kevin F. McLaughlin, 617-649-9204Senior
Vice President and Chief Financial OfficerorMedia:Suda
Communications LLCMaureen L. Suda, 585-387-9248
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