-- Part C of MoveDMD® Trial of Edasalonexent in
Duchenne Muscular Dystrophy Ongoing --
-- Continued Advancement of Rare Disease
Pipeline with CAT-5571, a Potential Treatment for Cystic Fibrosis,
and CAT-4001, a Potential Treatment for Neurodegenerative Diseases
--
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced financial results for
the fourth quarter and full year ended December 31, 2016, and
corporate highlights.
“The recently reported safety, tolerability and plasma exposure
data for edasalonexent in patients with Duchenne muscular dystrophy
from Part B of the MoveDMD trial are reassuring, and Part C of the
trial is continuing on track,” said Jill C. Milne, Chief Executive
Officer of Catabasis. “Although we did not achieve the MRI T2
composite endpoint at 12 weeks in Part B, we are encouraged by the
improvements in the function-associated exploratory endpoints,
including the timed function tests, North Star Ambulatory
Assessment, PODCI and muscle strength, and are hopeful that
extended exposure will provide evidence of functional
benefits.”
Dr. Milne continued, “Looking forward in 2017, Part C of the
MoveDMD trial will allow us to collect data for up to 48 weeks of
treatment and provide important information about dose and activity
with extended duration, as well as endpoints for possible future
clinical trials of edasalonexent. Following additional data
analysis from Part C, we will determine the next steps for
edasalonexent in DMD. Catabasis is also moving the development of
our rare disease pipeline forward, with advances in CAT-5571 for
cystic fibrosis and CAT-4001 for neurodegenerative diseases
including Friedreich’s ataxia and ALS.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004) and the MoveDMD Trial
- Announced top-line results from Part B
of the MoveDMD trial of edasalonexent in January. The primary
efficacy endpoint of MRI T2 was not met. The edasalonexent 100
mg/kg/day treatment group consistently showed numerical improvement
versus placebo across multiple timed function tests and the North
Star Ambulatory Assessment, although as expected the changes were
not statistically significant. The 67 mg/kg/day treatment group had
mixed results compared with both the 100 mg/kg/day treatment group
and placebo, which in each case were not statistically
significant.
- Announced two publications about
edasalonexent: Phase 1 clinical data in the Journal of Clinical
Pharmacology and preclinical research in JCI Insight.
- Ongoing open-label extension (Part C)
of the MoveDMD trial in which patients continue on edasalonexent
for 36 weeks following completion of Part B. Catabasis intends to
report the results from Part C in 2017, with an interim update in
Q2.
New Edasalonexent Updates
- Upcoming presentation this month at the
2017 Muscular Dystrophy Association Scientific Conference,
including: Part B data for the pediatric outcomes data collection
instrument (PODCI) and muscle strength function-associated
exploratory endpoints, which showed numerical improvement versus
placebo for both edasalonexent treatment groups at 12 weeks as well
as a review of previously released results from the MoveDMD
trial.
- In the Catabasis and Sarepta joint
research collaboration, established to explore a combination drug
treatment approach for DMD, increased dystrophin protein expression
was seen with an exon-skip modality in combination with
edasalonexent in the designated mouse model of DMD. The companies
believe that these results warrant further research.
Additional Rare Disease Programs
- Announced the publication of research
on CAT-5571, a novel activator of autophagy and potential oral
treatment for cystic fibrosis (CF), in the Journal of Medicinal
Chemistry.
- Presented preclinical data for CAT-5571
at the North American Cystic Fibrosis Conference. CAT-5571, an
activator of autophagy, in combination with lumacaftor/ivacaftor,
enhanced cell-surface trafficking and function of CF transmembrane
conductance regulator (CFTR) in bronchial epithelial cells from CF
patients with the F508del mutation. Catabasis also presented that
CAT-5571 enhanced the clearance of Pseudomonas aeruginosa infection
in preclinical models of CF, irrespective of CFTR mutation
status.
- Ongoing preclinical activities
exploring the potential of CAT-4001 in diseases such as amyotrophic
lateral sclerosis (ALS) and Friedreich’s ataxia.
Corporate
- Catabasis hosted its first Investor Day
in New York on November 17, 2016, focused on its strategy in rare
diseases and its pipeline, including edasalonexent and other
programs.
- Catabasis had three recent executive
team promotions: Ted Hibben to Chief Business Officer, Andrew
Nichols, Ph.D., to Chief Scientific Officer, and Angelika Fretzen,
Ph.D., to Senior Vice President of Product Development.
Fourth Quarter and Full Year 2016 Financial Results
Cash Position: At December 31, 2016, Catabasis had cash,
cash equivalents and marketable securities of $38.5 million,
compared to $47.3 million as of September 30, 2016 and $62.8
million as of December 31, 2015. Catabasis expects that its cash,
cash equivalents and marketable securities at December 31, 2016
will fund operating expenses, debt service and capital expenditure
requirements based on its current operating plan through March 31,
2018, assuming no unscheduled repayment of indebtedness prior to
such date. Net cash used in operating activities for the three
months ended December 31, 2016 was $8.0 million, compared to $8.7
million for the three months ended December 31, 2015. Net cash used
in operating activities for the full year 2016 was $32.9 million,
net of financings, compared to $29.8 million for the full year
2015.
R&D Expenses: Research and development expenses were
$6.3 million for the three months ended December 31, 2016, compared
to $6.7 million for the three months ended December 31, 2015 and
$25.5 million for the full year 2016, compared to $23.0 million for
the full year 2015. The increase in research and development
expenses for the full year 2016 relative to the full year 2015 was
primarily attributable to increased direct program costs related to
the edasalonexent MoveDMD trial. The decrease in research and
development expenses for the fourth quarter of 2016 relative to the
fourth quarter of 2015 was primarily attributable to having one
program in active clinical trials in Q4 2016 compared to two in Q4
2015.
G&A Expenses: General and administrative expenses
were $2.4 million for the three months ended December 31, 2016,
compared to $2.7 million for the three months ended December 31,
2015 and $10.1 million for the full year 2016, compared to $8.6
million for the full year 2015. The increase in general and
administrative expenses for the full year 2016 relative to the
prior year was primarily attributable to increased employee
compensation costs.
Operating Loss: Loss from operations was $8.7 million for
the three months ended December 31, 2016, compared to $9.3 million
for the three months ended December 31, 2015, and $35.6 million for
the full year 2016, compared to $31.7 million for the full year
2015.
Net Loss: Net loss was $8.8 million, or $0.47 per share,
for the three months ended December 31, 2016, compared to a net
loss of $9.6 million for the three months ended December 31, 2015.
Net loss for the full year 2016 was $36.1 million, or $2.22 per
share, compared to $32.6 million for the full year 2015.
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 fourth quarter
and full year 2016 financial results.
Participant Toll-Free Dial-In Number: (877) 388-2733Participant
International Dial-In Number: (541) 797-2984Pass Code: 74976422
Please specify to the operator that you would like to join the
“Catabasis Fourth Quarter and Full Year 2016 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
Duchenne muscular dystrophy (DMD or Duchenne), regardless of their
underlying mutation. Edasalonexent inhibits NF-kB, a protein that
is activated in Duchenne and drives inflammation and fibrosis,
muscle degeneration and suppresses muscle regeneration. In animal
models of DMD, edasalonexent produced beneficial effects in
skeletal, including diaphragm, and cardiac muscle and improved
function. 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. We have previously reported safety,
tolerability and reduction in NF-kB activity in Phase 1 trials in
adults. 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). Part A of the trial evaluated the safety, tolerability
and pharmacokinetics of, and NF-kB target engagement with,
edasalonexent in 17 boys with DMD. Part B of the trial was a
double-blind, placebo-controlled evaluation of the safety and
efficacy of edasalonexent over a 12-week period in 31 boys. The
primary efficacy endpoint for Part B was average change from
baseline to week 12 in MRI T2 measures in boys given edasalonexent
compared to placebo. Additional efficacy endpoints included
age-appropriate timed function tests (10-meter walk/run, 4-stair
climb and time to stand), North Star Ambulatory Assessment (NSAA),
the pediatric outcomes data collection instrument (PODCI) and
muscle strength. Part C is an open-label extension with
edasalonexent for 36 weeks beyond Part B and will evaluate longer
term safety and efficacy with the same clinical end points as Part
B. From the MoveDMD trial, we have reported that edasalonexent was
well tolerated with no safety signals. We reported top-line data
for Part B indicating that the primary efficacy endpoint was not
met. The edasalonexent 100 mg/kg/day treatment group consistently
showed numerical improvement versus placebo across multiple timed
function tests and the North Star Ambulatory Assessment, although
as expected the changes were not statistically significant. The 67
mg/kg/day treatment group had mixed results compared with both the
100 mg/kg/day treatment group and placebo, which in each case were
not statistically significant. Part C of the MoveDMD trial is
ongoing.
About CAT-5571Catabasis 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, irrespective of CFTR
mutation status.
About CAT-4001Catabasis 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 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 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 Annual Report on
Form 10-K for the year ended December 31, 2016, 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 December 31, Year Ended
December 31, 2016
2015 2016 2015
Operating expenses: Research and development $ 6,260 $ 6,670
$ 25,450 $ 23,030 General and administrative 2,413
2,663
10,108
8,629
Total operating expenses
8,673 9,333
35,558
31,659
Loss from operations (8,673 ) (9,333 )
(35,558)
(31,659)
Other (expense) income: Interest expense (175 ) (269 )
(837)
(978)
Interest and investment income 59 -
242
-
Other income, net 11 (4 )
93
7
Total other expense, net (105 ) (273 )
(502)
(971)
Net loss $ (8,778 ) $ (9,606 ) $ (36,060) $ (32,630)
Net loss per share - basic and diluted $ (0.47 ) $ (0.63 ) $
(2.22 ) $ (4.06 )
Weighted-average common sharesoutstanding
used in net loss per share - basicand diluted
18,699,480 15,298,810
16,230,190
8,041,948
Catabasis Pharmaceuticals, Inc.
Condensed Consolidated Balance
Sheets
(In thousands)
(Unaudited)
As of December 31, 2016
2015 Assets Cash and cash equivalents $ 23,596
$ 62,780 Available-for-sale securities 14,931 - Total assets 40,209
64,169
Liabilities and stockholders’ equity Current portion
of notes payable, net of discount 3,243 3,173 Notes payable, net of
current portion and discount 2,479 5,720 Total liabilities 11,123
13,676 Total stockholders’ equity $ 29,086 $ 50,493
Catabasis Pharmaceuticals, Inc.
Condensed Consolidated Statements of
Cash Flows
(In thousands)
(Unaudited)
Year Ended December 31, 2016 2015 Net
cash used in operating activities $ (32,858 ) $ (29,793 ) Net cash
used in investing activities (15,490 ) (421 ) Net cash provided by
financing activities 9,164 78,326 Net
(decrease) increase in cash and cash equivalents $ (39,184 ) $
48,112
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Corporate and Media
ContactCatabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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