- Luspatercept MEDALIST and BELIEVE Phase 3
trials expected to complete enrollment in Q2 2017 -
- Presented results from ongoing Phase 2 trial
with luspatercept at the 14th International Symposium on MDS -
- Announced plans to initiate luspatercept
Phase 3 trial in first-line, lower-risk MDS in early 2018 -
- Announced plans to initiate ACE-083 Phase 2
study in Charcot-Marie-Tooth disease -
- Maintained strong balance sheet with $213.2
million in cash, cash equivalents and investments to fund projected
operating requirements into the second half of 2019 -
Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage
biopharmaceutical company focused on the discovery, development and
commercialization of innovative therapeutics to treat serious and
rare diseases, today provided a corporate update and reported
financial results for the first quarter ended March 31,
2017.
“In the first quarter, we made significant progress across our
entire pipeline. Our luspatercept program in MDS and
beta-thalassemia continues to build momentum, as we look to
complete enrollment of the MEDALIST and BELIEVE Phase 3 studies
ahead of schedule, and expand into additional patient populations
where there are limited treatment options, including first-line,
lower-risk MDS and myelofibrosis,” said Habib Dable, President and
Chief Executive Officer of Acceleron. "As we continue to explore
further expansion opportunities for luspatercept, we are also
preparing to expand our wholly-owned pipeline into a second
neuromuscular disease, with the expected initiation by mid-year of
a Phase 2 study with ACE-083 in patients with Charcot-Marie-Tooth
disease.”
DEVELOPMENT PROGRAM
HIGHLIGHTS
Hematology
Luspatercept in myelodysplastic
syndromes (MDS)
Luspatercept is being developed to treat anemia and reduce or
eliminate the need for red blood cell transfusions in patients with
MDS, a rare disorder in which bone marrow fails to produce enough
healthy blood cells. Luspatercept is being developed as part of the
global collaboration between Acceleron and Celgene.
- Enrollment in the MEDALIST Phase 3
clinical trial is expected to complete in the second quarter of
this year. The MEDALIST Phase 3 study is enrolling 210 patients
to evaluate the efficacy and safety of luspatercept in patients
with anemia due to lower-risk MDS with ring sideroblasts who
require red blood cell (RBC) transfusions.
- Results presented at the
14th International Symposium on MDS from an ongoing
Phase 2 study in first-line, lower-risk MDS patients demonstrated
positive erythroid response and RBC transfusion independence
rates.
- Acceleron and Celgene announced
plans to initiate a third Phase 3 trial with luspatercept in early
2018. The new Phase 3 trial will evaluate luspatercept
treatment versus standard-of-care in the first-line treatment
setting for lower-risk MDS patients.
Luspatercept in
beta-thalassemia
Luspatercept is designed to treat severe, chronic anemia and
reduce RBC transfusion-dependence in adults with beta-thalassemia,
a rare genetic disorder that reduces the production of hemoglobin.
Luspatercept is being developed as part of the global collaboration
between Acceleron and Celgene.
- Enrollment in the BELIEVE Phase 3
trial is expected to complete in the second quarter of this
year. The BELIEVE Phase 3 study is enrolling 300 patients to
evaluate the efficacy and safety of luspatercept in patients with
anemia due to beta-thalassemia who require regular RBC
transfusions.
Neuromuscular Disease
ACE-083 in facioscapulohumeral muscular
dystrophy (FSHD) and Charcot-Marie-Tooth (CMT)
disease
ACE-083 is designed to increase muscle mass and strength in
target muscles for diseases that cause debilitating focal muscle
loss.
- Enrollment and treatment are ongoing
in Part 1 of the ACE-083 Phase 2 study in patients with FSHD,
one of the most prevalent forms of muscular dystrophy.
- Announced plans to initiate a second
Phase 2 clinical trial with ACE-083 in patients with
Charcot-Marie-Tooth (CMT) disease, one of the most common
inherited neurologic diseases leading to focal muscle weakness, by
mid-2017.
Preclinical Research
Acceleron continues its research on several molecules targeting
musculoskeletal diseases, fibrotic disorders and other serious
diseases.
- The Company plans to initiate a Phase 1
healthy volunteer clinical trial this year with ACE-2494.
NEWSFLOW AND CATALYSTS
Luspatercept
- Complete enrollment in the MEDALIST
Phase 3 trial in Q2 2017
- Complete enrollment in the BELIEVE
Phase 3 trial in Q2 2017
- Anticipate top-line data from both
Phase 3 trials in mid-2018
- Initiate first-line, lower-risk MDS
Phase 3 trial in early 2018
- Initiate Phase 2 trial in myelofibrosis
by year-end 2017
- Initiate Phase 2 trial in
non-transfusion dependent (NTD) beta-thalassemia by year-end
2017
- Additional Phase 2 results from ongoing
trials to be presented at medical conferences in 2017
ACE-083
- Report initial FSHD Phase 2 Part 1
dose-escalation results by late 2017
- Initiate CMT Phase 2 Part 1
dose-escalation trial by mid-2017
Dalantercept
- Report top-line results in the DART
Phase 2 trial in renal cell carcinoma by Q2 2017
ACE-2494
- Initiate Phase 1 healthy volunteer
study in 2017
Corporate
- Host a Research and Development Day to
discuss preclinical research and clinical development activities in
2H 2017
Financial Results
- Cash position – Cash, cash
equivalents and investments as of March 31, 2017 were $213.2
million. As of December 31, 2016 the Company had cash, cash
equivalents and investments of $234.4 million. We believe that
existing cash, cash equivalents and investments will be sufficient
to fund projected operating requirements into the second half of
2019.
- Revenue – Collaboration
revenue for the first quarter was $3.7 million. The revenue is
all from our Celgene partnership and is primarily due to cost
sharing revenue of $3.6 million related to expenses incurred
by the Company in support of our partnered programs.
- Costs and expenses – Total
costs and expenses for the first quarter were $29.6 million. This
includes R&D expenses of $21.7 million and G&A
expenses of $7.8 million.
- Other income, net – Other
income, net for the first quarter was $0.5 million primarily due to
interest income.
- Net loss – The Company's
net loss for the first quarter ended March 31,
2017 was $25.4 million.
Conference Call and Webcast
The Company will host a live conference call and webcast to
discuss its first quarter 2017 financial results and provide a
corporate update on May 8, 2017, at 8:00 a.m. EDT.
Participants can access the live conference call by dialing
877-312-5848 (domestic) or 253-237-1155 (international) and refer
to the “Acceleron Earnings Call.”
The live webcast can be accessed on the Investors page of the
Company’s website at www.acceleronpharma.com.
A replay of the webcast will be available approximately two
hours after the event on the Company's website.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused
on the discovery, development and commercialization of innovative
therapeutics to treat serious and rare diseases. Its pioneering
research platform leverages the powerful biology behind the body’s
ability to rebuild and repair its own cells and tissues. This
approach to drug discovery has generated four therapeutic
candidates that are currently in clinical trials. The Company’s
lead therapeutic candidate, luspatercept, is being evaluated in
Phase 3 studies for the treatment of the hematologic diseases
myelodysplastic syndromes and beta-thalassemia under a global
partnership with Celgene Corp. Acceleron is also advancing clinical
programs in the fields of oncology and neuromuscular diseases and
has a comprehensive preclinical research effort targeting fibrotic
and other serious diseases.
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, 2017
December 31, 2016 Cash and cash equivalents $ 33,157 $
20,950 Short and long-term investments 180,057 213,432 Other assets
15,915 13,265 Total assets $ 229,129 $ 247,647
Deferred revenue $ 4,108 $ 4,245 Warrants to purchase common stock
1,289 1,244 Other liabilities 15,412 16,561 Total
liabilities 20,809 22,050 Total stockholders’ equity 208,320
225,597 Total liabilities and stockholders’ equity $ 229,129
$ 247,647
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS
(Amounts in thousands except per share
data)
(unaudited)
Three Months Ended March 31, 2017
2016 Revenue: Collaboration revenue $ 3,705 $ 18,201
Costs and expenses: Research and development 21,727 16,246 General
and administrative 7,836 5,911 Total costs and
expenses 29,563 22,157 Loss from operations (25,858 ) (3,956 )
Other income, net 457 9,017 (Loss) income
before income taxes $ (25,401 ) $ 5,061 Income tax provision (6 ) —
Net (loss) income $ (25,407 ) $ 5,061 Other
comprehensive loss: Net unrealized holding gains on short-term and
long-term investments during the period 25 245
Comprehensive (loss) income $ (25,382 ) $ 5,306 Net
(loss) income per share applicable to common stockholders: Basic $
(0.66 ) $ 0.14 Diluted $ (0.66 ) $ 0.13 Weighted-average
number of common shares used in computing net (loss) income per
share applicable to common stockholders Basic 38,404 36,911 Diluted
38,404 38,666
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,
including sotatercept, luspatercept, dalantercept, ACE-083,
ACE-2494, the Company's IntelliTrap™ drug discovery platform, 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," "believe,"
"continue," "could," "estimate," "expect," "forecast," "goal",
"intend," "may," "plan," "potential," "project," "should,"
"strategy," "target," "will," "would," 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, cash
equivalents and investments will be insufficient to fund operations
into the second half of 2019, 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 the
Company's 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, that the Company's drug discovery activities may
not yield drug candidates for which the Company can commence
clinical trials at the rate at which the Company currently
anticipates or at all, 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 1, 2017, 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/20170508005243/en/
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Senior
Director, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
646-513-3125
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