-- Improvements Seen Across Functional
Assessments in Boys Treated with Edasalonexent for 12 Weeks in Part
B of the MoveDMD® Trial in Duchenne Muscular Dystrophy --
-- MoveDMD Trial Progressing, Part C Interim
Results Expected in Q3 2017 --
-- Rare Disease Pipeline Programs Advancing in
Cystic Fibrosis and Neurodegenerative Diseases --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
first quarter ended March 31, 2017, and reviewed recent business
progress.
“We made important progress across our portfolio in the first
quarter, including the presentation of encouraging results from our
edasalonexent MoveDMD trial and the advancement of our rare disease
pipeline,” said Jill C. Milne, Ph.D., Chief Executive Officer of
Catabasis. “Our recently completed prespecified analysis of
functional assessments in Part B of the MoveDMD trial strengthens
our confidence in the potential of edasalonexent as a novel
treatment for boys with Duchenne muscular dystrophy.”
Dr. Milne continued, “We see consistent numerical improvements
across prespecified functional analyses of boys treated with
edasalonexent. We expect to report interim results from Part C of
the MoveDMD trial in the third quarter, and we look forward to
gaining important information on the persistence of treatment
signals with edasalonexent as well as determining functional
endpoints for possible future clinical trials. The MoveDMD trial is
progressing as planned, and we are focused on the next steps for
the development of edasalonexent while continuing to advance our
pipeline programs in cystic fibrosis and neurodegenerative
diseases.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004) and the MoveDMD Trial
- Continued execution of open-label
extension (Part C) of the MoveDMD trial in which patients are
treated with edasalonexent following completion of Part B. The
institutional review boards have approved moving all boys in Part C
to the higher 100 mg/kg/day dose group of edasalonexent and
extending Part C to 60 weeks. Catabasis intends to report results
from Part C in 2017, with an interim update in Q3 after all boys
participating have completed 24 weeks of dosing with
edasalonexent.
- In April, presented data from the
MoveDMD trial of edasalonexent at the American Academy of Neurology
69th Annual Meeting. The prespecified analysis of Part B data
showed numerical improvement in rates of change across five
functional assessments for patients on 12 weeks of edasalonexent
compared to off-treatment prior to Part B dosing. These results are
in addition to and consistent with numerical improvements in the
same functional assessments with edasalonexent compared to placebo
as reported in the top-line results from Part B in January. These
functional assessments are meaningful to boys affected by Duchenne
and are known to correlate with loss of milestones and disease
progression. The MoveDMD trial was not powered for functional
assessments and these analyses were generally not statistically
significant.
- In March, reported results from the
Catabasis and Sarepta joint research collaboration showing
increased dystrophin expression in the mdx mouse with edasalonexent
in combination with an exon-skip modality. The companies believe
that these results warrant further research.
- In January, the primary endpoint for
Part B of MRI T2, an exploratory early biomarker, was not met.
However, we continue to see numerical improvements across the
functional assessments, as described above, which have precedence
as endpoints in pivotal trials in DMD.
Additional Rare Disease Programs
- In January, published research on
CAT-5571, a novel activator of autophagy and potential oral
treatment for cystic fibrosis (CF), in the Journal of Medicinal
Chemistry; upcoming presentation on CAT-5571 planned at the
European Cystic Fibrosis Conference in June.
- Continued ongoing preclinical
activities exploring the potential of CAT-4001 in diseases such as
amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.
First Quarter 2017 Financial Results
Cash Position: As of March 31, 2017, Catabasis had cash
and cash equivalents of $31.8 million, compared to $38.5 million in
cash, cash equivalents and available-for-sale securities as of
December 31, 2016. Catabasis expects that its current cash and cash
equivalents as of April 30, 2017, will fund operating expenses,
debt service and capital expenditure requirements based on its
current operating plan for at least 12 months from today. Net cash
used in operating activities for the three months ended March 31,
2017 was $8.1 million, compared to $9.1 million for the three
months ended March 31, 2016.
R&D Expenses: Research and development expenses were
$5.4 million for the three months ended March 31, 2017, compared to
$6.4 million for the three months ended March 31, 2016. The
decrease in research and development expenses was primarily
attributable to the completion of certain clinical activities.
G&A Expenses: General and administrative expenses
were $2.4 million for the three months ended March 31, 2017,
compared to $2.8 million for the three months ended March 31, 2016.
The decrease in general and administrative expenses was primarily
attributable to decreased G&A headcount.
Operating Loss: Loss from operations was $7.8 million for
the three months ended March 31, 2017, compared to $9.2 million for
the three months ended March 31, 2016.
Net Loss: Net loss was $7.9 million, or $0.41 per share,
for the three months ended March 31, 2017, compared to a net loss
of $9.4 million for the three months ended March 31, 2016.
Conference Call and Webcast
Catabasis will host a conference call and webcast at 4:30pm
ET today to provide an update on corporate developments and to
discuss first quarter 2017 financial results.
Participant Toll-Free Dial-In Number: (877) 388-2733Participant
International Dial-In Number: (541) 797-2984Pass Code: 99023963
Please specify to the operator that you would like to join the
“Catabasis First Quarter 2017 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. We are currently
conducting the MoveDMD trial, a three-part clinical trial
investigating the safety and efficacy of edasalonexent in boys ages
4 – 7 affected with DMD (any confirmed mutation). The third part of
the trial, an open-label extension with edasalonexent, is ongoing.
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 CAT-5571
Catabasis is developing CAT-5571 as a potential oral treatment
for cystic fibrosis (CF) with potential effects on both the cystic
fibrosis transmembrane conductance regulator (CFTR) and on the
clearance of Pseudomonas aeruginosa. CAT-5571 is a small molecule
that activates autophagy, a process that maintains cellular
homeostasis and host defense mechanisms, and is known to be
impaired in CF. Catabasis has shown in preclinical studies that
CAT-5571, in combination with lumacaftor/ivacaftor, enhances
cell-surface trafficking and function of CFTR with the F508del
mutation. Catabasis has also shown that CAT-5571 enhances the
clearance of P. aeruginosa infection in preclinical models of
CF.
About CAT-4001
Catabasis is developing CAT-4001 as a potential treatment for
neurodegenerative diseases such as Friedreich’s ataxia (FA) and
amyotrophic lateral sclerosis (ALS). CAT-4001 is a small molecule
that activates Nrf2 and inhibits NF-kB, two pathways that have been
implicated in FA and ALS. Catabasis has shown that CAT-4001
modulates the Nrf2 and NF-kB pathways in both cellular assays and
animal models.
About Catabasis
At 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. We are applying our SMART
linker platform to build an internal pipeline of product candidates
for rare diseases and plan to pursue partnerships to develop
additional product candidates. For more information on the
Company's drug discovery platform and pipeline of drug candidates,
please visit www.catabasis.com.
Forward Looking Statements
Any statements in this press release about future expectations,
plans and prospects for the Company, including statements about
future clinical trial plans 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; availability and
timing of results from preclinical studies and clinical trials;
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 period ended March 31, 2017,
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,
2017 2016
Operating expenses: Research and development $ 5,398 $ 6,436
General and administrative 2,363 2,770
Total operating expenses 7,761 9,206
Loss from operations (7,761 ) (9,206 ) Other (expense) income:
Interest expense (149 ) (243 ) Interest and investment income 39 53
Other expense, net (5 ) (22 ) Total other expense,
net (115 ) (212 ) Net loss $ (7,876 ) $ (9,418 ) Net
loss per share - basic and diluted $ (0.41 ) $ (0.61 )
Weighted-average common shares outstanding used in net loss per
share - basic and diluted 19,093,273
15,335,516
Catabasis Pharmaceuticals, Inc.
Condensed Consolidated Balance
Sheets
(In thousands)
(Unaudited)
March 31, December 31,
2017 2016 Assets Cash and
cash equivalents $ 31,795 $ 23,596 Available-for-sale securities -
14,931 Total assets 33,422 40,209
Liabilities and stockholders’
equity Current portion of notes payable, net of discount 3,260
3,243 Notes payable, net of current portion and discount 1,657
2,479 Total liabilities 9,467 11,123 Total stockholders’ equity $
23,955 $ 29,086
Catabasis Pharmaceuticals, Inc.
Condensed Consolidated Statements of
Cash Flows
(In thousands)
(Unaudited)
Three Months Ended March 31,
2017 2016 Net cash used
in operating activities $ (8,105 ) $ (9,147 ) Net cash provided by
(used in) investing activities 14,901 (29,069 ) Net cash provided
by (used in) financing activities 1,403 (746 )
Net increase (decrease) in cash and cash equivalents $ 8,199
$ (38,962 )
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version on businesswire.com: http://www.businesswire.com/news/home/20170511005997/en/
Investor and Media ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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