-- Edasalonexent Phase 3 PolarisDMD Trial in
Duchenne Muscular Dystrophy Expected to Initiate in the Second Half
of 2018 --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
second quarter ended June 30, 2018, and reviewed recent business
progress.
“We are thrilled to have achieved important milestones that
enable the Phase 3 PolarisDMD trial of edasalonexent for
registration purposes. We are now completing final preparations and
expect to initiate the Phase 3 trial in the coming months,” said
Jill C. Milne, Ph.D., Chief Executive Officer of
Catabasis. “Based on muscle effects and stabilization of
function seen with edasalonexent treatment in Phase 2, we believe
edasalonexent could fundamentally change the course of Duchenne
disease progression and the treatment paradigm. We look forward to
advancing edasalonexent into the PolarisDMD trial as we progress
towards our goal of making edasalonexent available to all those
affected by Duchenne with the hope of enabling patients to maintain
their functional abilities longer.”
Recent and Upcoming Corporate Highlights
- Announced plans for edasalonexent Phase
3 PolarisDMD trial in Duchenne muscular dystrophy (DMD), expected
to initiate in the second half of 2018 with top-line results
expected in the second quarter of 2020. The trial design was
informed by discussions with the U.S. Food and Drug
Administration (FDA) as well as input from treating physicians
and families of boys affected by Duchenne.
- The PolarisDMD trial will evaluate the
efficacy and safety of edasalonexent in patients with DMD and is
intended to support an application for commercial registration of
edasalonexent.
- The randomized, double-blind,
placebo-controlled PolarisDMD trial is expected to enroll
approximately 125 patients ages 4 to 7 (up to 8th birthday)
regardless of mutation type who have not been on steroids for at
least 6 months. Boys on a stable dose of eteplirsen may be eligible
to enroll.
- The primary efficacy endpoint will be
change in the North Star Ambulatory Assessment score
after 12 months of treatment with edasalonexent compared to
placebo. Key secondary endpoints are planned to include the
age-appropriate timed function tests time to stand, 4-stair climb
and 10-meter walk/run. Assessments of growth, cardiac and bone
health are also planned.
- Presented new edasalonexent clinical
biomarker data demonstrating NF-kB target engagement in the MoveDMD
trial in boys affected by DMD at the New Directions in Biology and
Disease of Skeletal Muscle Conference in June 2018. NF-kB is a
fundamental driver of disease progression in DMD.
- Presented new magnetic resonance
imaging (MRI) data through one year of edasalonexent treatment at
the American Academy of Neurology (AAN) 70th Annual Meeting in
April 2018. Edasalonexent significantly slowed DMD disease
progression as measured by MRI compared to the off-treatment
control period.
- Catabasis closed a $42 million
underwritten public offering in June 2018. The proceeds will be
used for the Phase 3 PolarisDMD clinical trial of edasalonexent for
the treatment of DMD, as well as for working capital and general
corporate purposes.
Second Quarter 2018 Financial Results
Cash Position: As of June 30, 2018, Catabasis had cash
and cash equivalents of $49.9 million, compared to $17.0 million as
of March 31, 2018. Based on the Company’s current operating plan,
Catabasis believes it has sufficient cash to fund operations into
Q2 2020. Net cash used in operating activities for the three months
ended June 30, 2018 was $5.6 million, compared to $5.7 million for
the three months ended June 30, 2017. Net cash used in operating
activities for the six months ended June 30, 2018 was $12.4
million, compared to $13.8 million for the six months ended June
30, 2017.
R&D Expenses: Research and development expenses were
$4.2 million for the three months ended June 30, 2018, compared to
$4.5 million for the three months ended June 30, 2017 and $9.5
million for the six months ended June 30, 2018, compared to $9.9
million for the six months ended June 30, 2017. The decrease in
research and development expenses was primarily attributable to a
decrease in direct program costs.
G&A Expenses: General and administrative expenses
were $2.4 million for the three months ended June 30, 2018 and
2017, respectively, and $4.8 million for the six months ended June
30, 2018 and 2017, respectively.
Operating Loss: Loss from operations was $6.6 million for
the three months ended June 30, 2018, compared to $6.9 million for
the three months ended June 30, 2017 and $14.3 million for the six
months ended June 30, 2018, compared to $14.7 million for the six
months ended June 30, 2017.
Net Loss: Net loss was $6.5 million, or $0.20 per share,
for the three months ended June 30, 2018, compared to a net loss of
$7.0 million, or $0.32 per share, for the three months ended June
30, 2017. Net loss for the six months ended June 30, 2018 was $14.1
million, compared to $14.9 million for the six months ended June
30, 2017.
Conference Call and WebcastCatabasis will host a
conference call and webcast at 8:30am ET today to provide
an update on corporate developments and to discuss second quarter
2018 financial results.
Participant Toll-Free Dial-In Number: (877) 388-2733
Participant International Dial-In Number: (541) 797-2984 Pass Code:
5876459
Please specify to the operator that you would like to join the
“Catabasis Second Quarter 2018 Results Call.”
Interested parties may access a live audio webcast of the
conference call via the investor section of the Catabasis
website, www.catabasis.com. Please connect to the Catabasis
website several minutes prior to the start of the broadcast to
ensure adequate time for any software download that may be
necessary. The webcast will be archived for 90 days.
About Edasalonexent (CAT-1004)Edasalonexent (CAT-1004) is
an investigational oral small molecule that is being developed as a
potential foundational therapy for all patients affected by DMD,
regardless of their underlying mutation. Edasalonexent inhibits
NF-kB, a protein that is activated in DMD and drives inflammation,
fibrosis and muscle degeneration and suppresses muscle
regeneration. Edasalonexent continues to be dosed in an open-label
extension of the MoveDMD Phase 2 clinical trial, and Catabasis is
preparing to initiate a single global Phase 3 trial in the second
half of 2018 to evaluate the efficacy and safety of edasalonexent
for registration purposes. The FDA has granted orphan drug, fast
track and rare pediatric disease designations and the European
Commission has granted orphan medicinal product designation to
edasalonexent for the treatment of DMD. For a summary of clinical
results reported to-date, please visit www.catabasis.com.
About CatabasisAt Catabasis Pharmaceuticals, our mission
is to bring hope and life-changing therapies to patients and their
families. Our lead program is edasalonexent, an NF-kB inhibitor in
development for the treatment of Duchenne muscular dystrophy. The
global Phase 3 PolarisDMD trial is expected to initiate in the
second half of 2018. For more information on edasalonexent and our
Phase 3 PolarisDMD trial, please visit www.catabasis.com.
Forward Looking StatementsAny statements in this press
release about future expectations, plans and prospects for the
Company, including statements about future clinical trial plans
including, among other things, statements about the Company’s plans
to commence the global Phase 3 PolarisDMD trial in DMD to evaluate
the efficacy and safety of edasalonexent for registration purposes,
and the Company’s expectation that based on its current operating
plan it has sufficient cash to fund operations into the second
quarter of 2020 and other statements containing the words
“believes,” “anticipates,” “plans,” “expects,” “may” and similar
expressions, constitute forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including: uncertainties inherent in the initiation and
completion of preclinical studies and clinical trials and clinical
development of the Company’s product candidates; whether interim
results from a clinical trial will be predictive of the final
results of the trial or the results of future trials; expectations
for regulatory approvals to conduct trials or to market products;
availability of funding sufficient for the Company’s foreseeable
and unforeseeable operating expenses and capital expenditure
requirements; other matters that could affect the availability or
commercial potential of the Company’s product candidates; and
general economic and market conditions and other factors discussed
in the “Risk Factors” section of the Company’s Quarterly Report on
Form 10-Q for the quarter ended June 30, 2018, which is on file
with the Securities and Exchange Commission, and in other filings
that the Company may make with the Securities and Exchange
Commission in the future. In addition, the forward-looking
statements included in this press release represent the Company’s
views as of the date of this press release. The Company anticipates
that subsequent events and developments will cause the Company’s
views to change. However, while the Company may elect to update
these forward-looking statements at some point in the future, the
Company specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing the Company’s views as of any date subsequent to the
date of this release.
Catabasis Pharmaceuticals,
Inc.Condensed Consolidated Statements of Operations(In
thousands, except share and per share data)
(Unaudited)
Three Months EndedJune
30,
Six Months EndedJune 30,
2018 2017 2018
2017 Operating expenses: Research and development
4,239 4,519 9,486 9,917 General and administrative 2,397
2,400 4,789 4,763
Total operating expenses 6,636 6,919
14,275 14,680 Loss from operations
(6,636 ) (6,919 ) (14,275 ) (14,680 ) Other income (expense):
Interest expense (33 ) (127 ) (90 ) (276 ) Interest and investment
income 43 44 75 83 Other income, net 147 28
159 23 Total other income
(expense), net 157 (55 ) 144
(170 ) Net loss $ (6,479 ) $ (6,974 ) $ (14,131 ) $ (14,850
) Net loss per share - basic and diluted $ (0.20 ) $ (0.32 ) $
(0.48 ) $ (0.73 ) Weighted-average common shares outstanding used
in net loss per share - basic and diluted 32,728,771
21,796,194 29,659,358 20,452,200
Catabasis Pharmaceuticals,
Inc.Condensed Consolidated Balance Sheets(In
thousands)(Unaudited)
June 30, December 31, 2018 2017
Assets Cash and cash equivalents $ 49,931 $ 16,369 Total
assets 50,847 17,897
Liabilities and stockholders’ equity
Current portion of notes payable, net of discount 831 2,479 Total
liabilities 5,035 6,105 Total stockholders’ equity $ 45,812 $
11,792
Catabasis Pharmaceuticals,
Inc.Condensed Consolidated Statements of Cash Flows(In
thousands)(Unaudited)
Six Months Ended June 30,
2018 2017 Net cash used in operating activities $
(12,421 ) $ (13,785 ) Net cash provided by investing activities 8
14,901 Net cash provided by financing activities 45,975
4,657 Net increase in cash and cash
equivalents $ 33,562 $ 5,773
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version on businesswire.com: https://www.businesswire.com/news/home/20180809005095/en/
Investor and Media ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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