-- Favorable Results Seen in 12-week
Edasalonexent Phase 2 MoveDMD® Trial in Duchenne Muscular
Dystrophy; Phase 3 Clinical Trial Plan Expected Second Half of 2017
--
-- Preclinical Data Support CAT-5571 as a
Potential Treatment to Enhance Host Defenses by Restoring Autophagy
in Cystic Fibrosis --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
second quarter ended June 30, 2017, and reviewed recent business
progress.
“In the second quarter, we presented an important prespecified
crossover analysis of data from boys with Duchenne in our Phase 2
edasalonexent trial. We are very excited to see improvements in the
rate of decline of muscle function across multiple assessments in
boys treated with edasalonexent for 12 weeks. The results are very
consistent with and support our earlier analysis of functional
assessments in boys treated with edasalonexent for 12 weeks as
compared to placebo and further strengthen our confidence in the
potential of edasalonexent as a novel treatment for DMD. We
continue to advance the open-label extension and expect to share
24-week edasalonexent results in the third quarter as well as
announce our Phase 3 clinical trial plan for edasalonexent in the
second half of 2017,” said Jill C. Milne, Ph.D., Chief Executive
Officer of Catabasis.
Dr. Milne continued, “We are also encouraged by the results of
our preclinical studies of CAT-5571, supporting its potential as an
oral treatment for cystic fibrosis, and with our progress across
our research programs driven by our SMART Linker℠ drug discovery
technology.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004) for the Treatment of Duchenne
Muscular Dystrophy (DMD)
- In the MoveDMD Phase 2 edasalonexent
trial, a crossover analysis of the rates of change across five
assessments of muscle function in patients after 12 weeks of
treatment compared to off-treatment prior to Phase 2 dosing showed
clinically meaningful numerical improvements in rates of decline.
The analysis was presented at the American Academy of Neurology
69th Annual Meeting in April and was the second of two prespecified
analyses of the 12-week data. The first analysis was presented in
March and showed meaningful improvements in assessments of muscle
function in boys treated with edasalonexent compared to placebo.
Functional assessments have precedence as endpoints in pivotal
trials in DMD. The MoveDMD 12-week Phase 2 results are consistent
with the therapeutic goal of treatment, to delay the predictable,
sequential loss of function in DMD. There were no safety signals
and edasalonexent was well tolerated in this study.
- The open-label extension of the MoveDMD
trial is progressing as planned and results from 24 weeks of
edasalonexent treatment are expected to be announced in the third
quarter of 2017. All boys participating in the open-label extension
have now moved to the higher 100 mg/kg/day edasalonexent treatment
group. Pending IRB approval, the open-label extension will be
extended for an additional 52 weeks so that participating boys can
continue to receive edasalonexent.
- Catabasis expects to announce the Phase
3 clinical trial plan for edasalonexent in DMD in the second half
of 2017.
- The first boy in the MoveDMD trial who
is amenable to exon 51 skipping treatment has started EXONDYS 51™
treatment along with edasalonexent in the open-label extension. The
Catabasis and Sarepta joint research collaboration previously
showed increased dystrophin expression in the mdx mouse with
edasalonexent in combination with an exon-skip modality.
Edasalonexent may have the potential to increase dystrophin levels
in combination with dystrophin-targeted therapies.
CAT-5571 for the Treatment of Cystic Fibrosis (CF)
- CAT-5571 demonstrated in preclinical
studies improved intracellular clearance of bacterial pathogens
that are clinically important in CF, as reported at the European
Cystic Fibrosis Society Conference in June. This activity has the
potential to improve lung function by reducing the intracellular
load of multiple types of bacteria, including the
pathogens, Pseudomonas aeruginosa and Burkholderia
cenocepacia, which are the leading cause of morbidity and mortality
for patients with CF. CAT-5571 restores autophagy, a host defense
mechanism, which is known to be impaired in CF. Catabasis expects
to initiate a Phase 1 trial for CAT-5571 in 2018.
Second Quarter 2017 Financial Results
Cash Position: As of June 30, 2017, Catabasis had cash
and cash equivalents of $29.4 million, compared to $31.8 million in
cash, cash equivalents and available-for-sale securities as of
March 31, 2017. Catabasis expects that its current operating plan
provides for cash to fund operations through August, 2018. Net cash
used in operating activities for the three months ended June 30,
2017 was $5.7 million, compared to $8.8 million for the three
months ended June 30, 2016. Net cash used in operating activities
for the six months ended June 30, 2017 was $13.8 million, compared
to $18.0 million for the six months ended June 30, 2016.
R&D Expenses: Research and development expenses were
$4.5 million for the three months ended June 30, 2017, compared to
$6.8 million for the three months ended June 30, 2016 and $9.9
million for the six months ended June 30, 2017, compared to $13.3
million for the six months ended June 30, 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 June 30, 2017,
compared to $2.6 million for the three months ended June 30, 2016
and $4.8 million for the six months ended June 30, 2017, compared
to $5.3 million for the six months ended June 30, 2016. The
decrease in general and administrative expenses was primarily
attributable to headcount reductions.
Operating Loss: Loss from operations was $6.9 million for
the three months ended June 30, 2017, compared to $9.4 million for
the three months ended June 30, 2016, and $14.7 million for the six
months ended June 30, 2017, compared to $18.6 million for the six
months ended June 30, 2016.
Net Loss: Net loss was $7.0 million, or $0.32 per share,
for the three months ended June 30, 2017, compared to a net loss of
$9.4 million, or $0.61 per share, for the three months ended June
30, 2016. Net loss for the six months ended June 30, 2017 was $14.9
million, compared to $18.9 million for the six months ended June
30, 2016.
Conference Call and WebcastCatabasis will host a
conference call and webcast at 4:30pm ET today to provide
an update on corporate developments and to discuss second quarter
2017 financial results.
Participant Toll-Free Dial-In Number: (877) 388-2733
Participant International Dial-In Number: (541) 797-2984 Pass Code:
47693255
Please specify to the operator that you would like to join the
“Catabasis Second 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 enrolled at 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-5571Catabasis is developing CAT-5571 as a
potential oral treatment for CF with potential effects on both the
cystic fibrosis transmembrane conductance regulator (CFTR) and on
the clearance of multiple types of bacteria including 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
observed 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
observed that CAT-5571 enhances the clearance of P. aeruginosa
infection in preclinical models of CF.
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. 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 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 our plans to
identify, develop and commercialize novel therapeutics based on our
SMART Linker drug discovery platform, our plans to continue to
evaluate data from the open-label extension of our MoveDMD®
clinical trial of edasalonexent for the treatment of DMD, our plans
for ongoing and planned clinical trials for edasalonexent and other
product candidates, whether conducted by us or by any future
collaborators, including the timing of initiation of these trials
and of the anticipated results, 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 June 30, 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 June 30, Six
Months Ended June 30, 2017 2016
2017 2016 Operating expenses: Research
and development $ 4,519 $ 6,818 $ 9,917 $ 13,254 General and
administrative 2,400 2,578 4,763
5,348 Total operating expenses 6,919
9,396 14,680 18,602
Loss from operations (6,919 ) (9,396 ) (14,680 ) (18,602 )
Other (expense) income: Interest expense (127 ) (220 ) (276 ) (463
) Interest and investment income 44 80 83 133 Other income, net
28 91 23 69
Total other expense, net (55 ) (49 ) (170 )
(261 ) Net loss $ (6,974 ) $ (9,445 ) $ (14,850 ) $ (18,863
) Net loss per share - basic and diluted $ (0.32 ) $ (0.61 ) $
(0.73 ) $ (1.23 )
Weighted-average common shares
outstandingused in net loss per share - basic and diluted
21,796,194 15,373,964 20,452,200
15,354,740
Catabasis Pharmaceuticals,
Inc.Condensed Consolidated Balance Sheets(In
thousands)(Unaudited)
June 30,
December 31, 2017 2016 Assets
Cash and cash equivalents $ 29,369 $ 23,596 Available-for-sale
securities - 14,931 Total assets 30,816 40,209
Liabilities and
stockholders’ equity Current portion of notes payable, net of
discount 3,278 3,243 Notes payable, net of current portion and
discount 831 2,479 Total liabilities 9,266 11,123 Total
stockholders’ equity $ 21,550 $ 29,086
Catabasis Pharmaceuticals,
Inc.Condensed Consolidated Statements of Cash Flows(In
thousands)(Unaudited)
Six Months Ended June 30, 2017
2016 Net cash used in operating activities $ (13,785 ) $
(17,976 ) Net cash provided by (used in) investing activities
14,901 (18,971 ) Net cash provided by (used in) financing
activities 4,657 (1,555 ) Net increase
(decrease) in cash and cash equivalents $ 5,773 $ (38,502 )
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170810006001/en/
Investor and Media ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
Catabasis Pharmaceuticals (NASDAQ:CATB)
Historical Stock Chart
From Mar 2024 to Apr 2024
Catabasis Pharmaceuticals (NASDAQ:CATB)
Historical Stock Chart
From Apr 2023 to Apr 2024