-- MoveDMD® Trial Data Through One Year of
Treatment Reinforce Edasalonexent Potential as Disease-Modifying
Therapy for Duchenne Muscular Dystrophy --
-- 2018 Priorities Focused on Advancing
Edasalonexent and Improving the Lives of Boys Affected by Duchenne
Muscular Dystrophy --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
first quarter ended March 31, 2018, and reviewed recent business
progress.
“We are pleased to see additional positive clinical data from
our MoveDMD trial early in 2018 that continue to demonstrate the
potential of edasalonexent as a disease-modifying therapy for all
patients affected by Duchenne, regardless of mutation. As evidenced
by sustained improvements in all assessments of physical function
and in biomarkers of muscle health and inflammation, edasalonexent
has slowed the progression of Duchenne in the MoveDMD trial,” said
Jill C. Milne, Ph.D., Chief Executive Officer of
Catabasis. “We believe that these effects ultimately will
translate to boys with Duchenne maintaining functional abilities
longer.”
Recent and Upcoming Corporate Highlights
- Edasalonexent significantly slowed
Duchenne muscular dystrophy (DMD) disease progression as measured
by MRI through one year of treatment, as reported at the American
Academy of Neurology 70th Annual Meeting in April 2018.
Statistically significant improvements in MRI T2 rate of change
through 48 weeks of oral 100 mg/kg of edasalonexent treatment
compared to control were observed in the MoveDMD trial. The rate of
fat fraction accumulation slowed in both the soleus and vastus
lateralis through 48 weeks of edasalonexent treatment compared to
the off-treatment control period. There was a greater reduction in
the rate of fat fraction accumulation in boys on edasalonexent
treatment than in a population of boys in a separate ImagingDMD
natural history study who were largely on steroids. Edasalonexent
continued to be well tolerated with no safety signals observed
throughout the trial.
- Height and weight through 60 weeks of
edasalonexent treatment was on track with standard growth curves
for unaffected boys, as presented at the 2018 Muscular Dystrophy
Association (MDA) Clinical Conference in March 2018. This profile
is favorably differentiated from the typical profile associated
with the corticosteroid standard of care in DMD, which includes
weight gain and curtailed growth.
- Heart rate data from boys treated with
edasalonexent decreased toward age-normative values through 48
weeks of treatment, as reported at the MDA Clinical Conference in
March 2018. In the 4-7 year old age range, boys typically have
resting tachycardia, a heart rate that exceeds the normal resting
rate, which is the first cardiac manifestation in DMD. Cardiac
failure is a leading cause of mortality in DMD.
- Observations from an ImagingDMD natural
history study, as presented at the MDA Clinical Conference in March
2018, were generally consistent with the absolute functional
abilities as well as declines in abilities experienced by boys in
the off-treatment control period of the Catabasis MoveDMD trial. We
believe that these data provide important corroboration that the
MoveDMD off-treatment control period observations are
characteristic of the expected natural history and provide added
confidence in the slowing of disease progression treatment effects
observed with edasalonexent.
- Preservation of muscle function and
substantially slowed DMD disease progression through more than a
year of edasalonexent treatment, as reported at the XVI
International Conference on Duchenne and Becker Muscular Dystrophy
in February 2018. Consistent improvements in all assessments of
muscle function (North Star Ambulatory Assessment, time to stand,
4-stair climb and 10-meter walk/run) were observed following 48 and
60 weeks of edasalonexent treatment compared to the rates of change
in the off-treatment control period. Statistically significant
improvements in multiple non-effort based biomarkers of muscle
health and inflammation (muscle enzymes and C-reactive protein)
were observed compared to baseline.
- Company resources have been aligned to
focus on its lead program edasalonexent for the treatment of DMD.
Catabasis is preparing for a global Phase 3 trial to evaluate the
safety and efficacy of edasalonexent for registration
purposes.
First Quarter 2018 Financial Results
Cash Position: As of March 31, 2018, Catabasis had cash
and cash equivalents of $17.0 million, compared to $16.4 million as
of December 31, 2017. Based on the Company’s current operating
plan, Catabasis believes that it has sufficient cash to fund
operations through December 2018. To advance edasalonexent in the
Phase 3 trial, Catabasis expects to seek additional funds through
equity or debt financings and/or through partnering or licensing
transactions. Net cash used in operating activities for the three
months ended March 31, 2018 was $6.8 million, compared to $8.1
million for the three months ended March 31, 2017.
R&D Expenses: Research and development expenses were
$5.2 million for the three months ended March 31, 2018, compared to
$5.4 million for the three months ended March 31, 2017.
G&A Expenses: General and administrative expenses
remained consistent at $2.4 million for the three months ended
March 31, 2018 and for the three months ended March 31, 2017.
Operating Loss: Loss from operations was $7.6 million for
the three months ended March 31, 2018, compared to $7.8 million for
the three months ended March 31, 2017.
Net Loss: Net loss was $7.7 million, or $0.29 per share,
for the three months ended March 31, 2018, compared to a net loss
of $7.9 million, or $0.41 per share, for the three months ended
March 31, 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 first quarter
2018 financial results.
Participant Toll-Free Dial-In Number: (877) 388-2733
Participant International Dial-In Number: (541) 797-2984 Pass Code:
2192025
Please specify to the operator that you would like to join the
“Catabasis First 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 disease-modifying 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 and fibrosis, muscle degeneration and suppresses
muscle regeneration. Edasalonexent continues to be dosed in the
open-label extension of the MoveDMD Phase 2 clinical trial and
Catabasis is preparing for a single global Phase 3 trial to
evaluate the efficacy and safety of edasalonexent for registration
purposes, dependent on raising capital. 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 SMART (Safely Metabolized And Rationally Targeted)
Linker drug discovery platform enables us to engineer molecules
that simultaneously modulate multiple targets in a disease. Our
lead program in development is edasalonexent for the treatment of
Duchenne muscular dystrophy. For more information on edasalonexent
and our pipeline of drug candidates, 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 a single global Phase 3 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 through December 2018,
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; the Company’s
ability to obtain financing on acceptable terms and in a timely
manner to fund the Company’s planned Phase 3 trial of edasalonexent
in DMD for registration purposes; 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 March 31, 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 Ended March 31, 2018
2017 Operating expenses: Research and development
5,247 5,398 General and administrative 2,392 2,363
Total operating expenses 7,639 7,761 Loss from
operations (7,639) (7,761) Other (expense) income: Interest expense
(57) (149) Interest and investment income 32 39 Other income
(expense), net 12 (5) Total other expense, net
(13) (115) Net loss $ (7,652) $ (7,876) Net loss per share -
basic and diluted $ (0.29) $ (0.41) Weighted-average common shares
outstanding used in net loss per share - basic and diluted
26,555,840 19,093,273
Catabasis Pharmaceuticals,
Inc.Condensed Consolidated Balance Sheets(In
thousands)(Unaudited)
March 31, December 31,
2018 2017 Assets Cash and cash equivalents $
17,030 $ 16,369 Total assets 18,290 17,897
Liabilities and
stockholders’ equity Current portion of notes payable, net of
discount 1,657 2,479 Total liabilities 5,359 6,105 Total
stockholders’ equity $ 12,931 $ 11,792
Catabasis Pharmaceuticals,
Inc.Condensed Consolidated Statements of Cash Flows(In
thousands)(Unaudited)
Three Months Ended March 31, 2018
2017 Net cash used in operating activities $ (6,819) $
(8,105) Net cash provided by investing activities - 14,901 Net cash
provided by financing activities 7,480 1,403 Net
increase in cash and cash equivalents $ 661 $ 8,199
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version on businesswire.com: https://www.businesswire.com/news/home/20180510005248/en/
Investor and Media ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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