– Completed luspatercept myelodysplastic
syndromes phase 2 clinical trial –
– FDA Fast Track Designations granted to
luspatercept for the treatment of patients with beta-thalassemia
–
– Phase 3 clinical trials with luspatercept in
lower risk myelodysplastic syndromes and beta-thalassemia expected
to begin by year-end 2015 –
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 second quarter ended
June 30, 2015.
“In the second quarter, we presented promising new longer-term
treatment data from our luspatercept programs. These results
further reinforce our excitement about the potential of
luspatercept to help patients with myelodysplastic syndromes and
beta-thalassemia. We and our collaboration partner, Celgene, look
forward to starting pivotal trials in both of these indications by
the end of this year,” said John Knopf, Ph.D., Chief Executive
Officer of Acceleron. “At Acceleron, we remain focused on bringing
innovative therapies to patients, and we are continuing to advance
and expand our earlier stage pipeline as well. We have scheduled
our first Research and Development Day for investors on October 23,
2015. At that meeting, we will review our clinical and preclinical
programs, including a first look at top-line data from the phase 1
trial with our candidate muscle agent, ACE-083.”
Recent Highlights and Current Updates
Development Programs
Hematology
- Completed enrollment and treatment
in the luspatercept phase 2 dose escalation study in
myelodysplastic syndromes (MDS) – Enrollment and treatment
ongoing in the phase 2 dose escalation beta-thalassemia study.
- Long-term phase 2 extension studies
ongoing with luspatercept in both MDS and beta-thalassemia –
Patients who completed their 3-month treatment in the MDS or
beta-thalassemia dose escalation studies were eligible to enroll in
the 12-month MDS or beta-thalassemia extension study. Enrollment in
the MDS extension study is complete and enrollment in the
beta-thalassemia extension study is ongoing.
- Presented luspatercept phase 2 data
supporting the advancement to phase 3 clinical trials at the
European Hematology Association's annual meeting – In lower
risk MDS patients, which represent a large majority of patients
affected by the disease, longer-term treatment with luspatercept
led to sustained increases in hemoglobin levels and transfusion
independence. In both transfusion and non-transfusion dependent
beta-thalassemia patients, luspatercept generated durable increases
in hemoglobin levels, reductions in transfusion burden and
improvements in iron overload. Based on these promising data,
Acceleron and Celgene are advancing luspatercept to phase 3
clinical trials in MDS and beta-thalassemia.
- Luspatercept granted Fast Track
designations for beta-thalassemia – The United States Food and
Drug Administration (FDA) has granted Fast Track Designations to
luspatercept for two separate indications—the use of luspatercept
for the treatment of patients with transfusion dependent
beta-thalassemia and the use of luspatercept for the treatment of
patients with non-transfusion dependent beta-thalassemia.
Oncology
- Presented preliminary renal cell
carcinoma (RCC) phase 2 data showing that the combination of
dalantercept and axitinib generated encouraging, progression-free
survival – In 2nd through 4th line RCC patients, the aggregate
median progression-free survival (PFS) rate for the combination of
dalantercept and axitinib across all three dose levels of
dalantercept tested was 8.3 months. The median PFS has not yet been
reached for the 0.9 mg/kg dose group, which has been chosen as the
dose level for the randomized Part 2 stage of this study.
Muscle Diseases
- Completed enrollment and treatment
in ACE-083 phase 1 clinical trial – The phase 1 study of
ACE-083, a therapeutic candidate designed to selectively increase
muscle mass and strength in the muscle into which it is
administered, has completed enrollment and treatment in healthy
volunteers.
- Advanced and expanded pipeline –
Acceleron is advancing several promising preclinical programs,
particularly those designed to increase muscle mass and strength.
Our goal is to advance a fifth, internally discovered therapeutic
candidate into clinical trials by the end of 2016.
Upcoming Milestones and Events
- Phase 3 clinical trials with
luspatercept in MDS and beta-thalassemia expected to start by
year-end – Acceleron and Celgene plan to initiate pivotal
programs for luspatercept in beta-thalassemia and MDS by
year-end.
- Acceleron Research and Development
(R&D) Day scheduled for Friday October 23, 2015 – Acceleron
will hold its first R&D Day on Friday morning, October 23, 2015
in New York City.
- ACE-083 phase 1 clinical data –
Preliminary, top-line data from the ACE-083 phase 1 clinical trial
will be presented at the Acceleron R&D Day on October 23.
- Data from a new preclinical muscle
program expected to be presented at the World Muscle Society annual
meeting – Acceleron plans to present data from a new
preclinical program, ACE-2494, at the World Muscle Society's 20th
International Congress in Brighton, UK, which runs from September
30 to October 4, 2015.
Financial Results
- Cash Position – Cash, cash
equivalents and investments as of June 30, 2015 were $156.6
million. As of December 31, 2014 the company had cash and cash
equivalents of $176.5 million. Acceleron expects that its
cash, cash equivalents and investments as of June 30, 2015
will be sufficient to fund the Company’s operations into the second
half of 2017.
Conference Call and Webcast
The company will host a conference call and live audio webcast
to report its second quarter financial results for 2015 and provide
a corporate update on August 6, 2015, at 8:00 AM EDT. To
participate by teleconference, please dial 877-312-5848 (domestic)
or 253-237-1155 (international) and refer to the “Acceleron Q2
Earnings Call.” To access the live webcast, please select “Events
& Presentations” in the Investors & Media section on the
Company’s website (www.acceleronpharma.com) at least 10 minutes
beforehand to ensure time for any downloads that may be
required.
An archived webcast recording will be available on the Acceleron
website beginning 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-beta) 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)
June 30, 2015 December 31, 2014
Cash, cash equivalents and investments $ 156,595 $ 176,460 Other
assets 10,923 9,836 Total assets $ 167,518 $ 186,296
Accrued expenses 10,167 7,572 Deferred revenue 5,175 5,978
Warrants to purchase common stock 10,680 14,124 Other liabilities
2,093 2,337 Total liabilities 28,115 30,011 Total
stockholders’ equity 139,403 156,285 Total liabilities and
stockholders’ equity $ 167,518 $ 186,296
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS
(Amounts in thousands except per share
data)
(unaudited)
Three Months Ended June 30, Six Months
Ended June 30, 2015 2014 2015
2014 Revenue: Collaboration revenue $ 5,717 $ 4,078 $
10,139 $ 7,385 Costs and expenses: Research and development 14,150
12,677 28,930 24,442 Litigation settlement — 5,000 — 5,000 General
and administrative 4,661 3,712 9,360 7,462
Total costs and expenses 18,811 21,389 38,290 36,904 Loss
from operations (13,094 ) (17,311 ) (28,151 ) (29,519 ) Total other
income, net 2,711 761 3,196 3,849 Net
loss applicable to common stockholders $ (10,383 ) $ (16,550 ) $
(24,955 ) $ (25,670 ) Net loss per share applicable to
common stockholders-basic and diluted $ (0.32 ) $ (0.52 ) $ (0.76 )
$ (0.83 ) Weighted-average number of common shares used in
computing net loss per share applicable to common
stockholders-basic and diluted 32,870 31,552 32,754
30,939
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, ACE-083, or
ACE-2494 and the Company’s TGF-beta 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 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 the Company expects 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 or Celgene 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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20150806005199/en/
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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