-- Enrollment Complete for MoveDMD® Phase 2
Trial of Edasalonexent, a Potential Disease-Modifying Treatment for
Duchenne Muscular Dystrophy (DMD) --
-- Expanding Rare Disease Pipeline with
CAT-5571, a Potential Treatment for Cystic Fibrosis --
-- Investor Day Scheduled for November 17
--
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced financial results for
the third quarter ended September 30, 2016 and corporate
highlights.
“Catabasis is executing on its mission to bring hope and
life-changing therapies to patients and their families suffering
from rare diseases,” said Jill C. Milne, Chief Executive Officer of
Catabasis. “We completed enrollment in the Phase 2 portion of the
MoveDMD® clinical trial in October and expect to report top-line
safety and efficacy results from the placebo-controlled portion of
the trial in the first half of Q1 2017; we expect to generate a
rich and informative data set in DMD based on the design of the
MoveDMD trial including the 36-week open-label extension. We
believe in the potential of edasalonexent as monotherapy for the
treatment of DMD and also believe that a combination drug approach
may be able to offer additional benefits to patients. In addition
to our work in DMD, we are evaluating other diseases where the
inhibition of NF-kB may be beneficial. There are a number of other
rare diseases where NF-kB plays an important role and edasalonexent
could have the potential to positively impact patients affected by
these diseases. Further, Catabasis recently expanded its rare
disease pipeline by adding CAT-5571, an activator of autophagy, as
a potential treatment of cystic fibrosis.”
Recent and Upcoming Corporate Highlights
Enrollment Complete for MoveDMD Phase 2 Trial of
Edasalonexent (CAT-1004)
- Completed enrollment in Phase 2 (Part
B) of the MoveDMD trial of edasalonexent in October. Top-line
safety and efficacy results from the placebo-controlled portion of
the MoveDMD trial are expected in the first half of Q1 2017.
- Presented positive biomarker gene
expression and proteomic data from Part A of the MoveDMD trial of
edasalonexent at the World Muscle Society Congress.
- Announced the initiation of an
open-label extension for the MoveDMD trial, in which patients will
continue on open-label edasalonexent for 36 weeks following
completion of the 12-week, placebo-controlled portion of the trial.
During the open-label extension, safety will be monitored and
additional assessments including MRI, timed function tests, muscle
strength measures, the North Star Ambulatory Assessment and the
pediatric outcomes data collection instrument (PODCI) will be
performed.
- Announced a joint research
collaboration with Sarepta Therapeutics to explore a combination
drug treatment approach for DMD. The two companies will contribute
their respective expertise to study edasalonexent, an oral NF-kB
inhibitor, developed by Catabasis, together with an exon skipping
treatment developed by Sarepta in a mouse model of DMD.
Additional Rare Disease Programs
- Presented positive preclinical data for
CAT-5571, a potential therapy to treat cystic fibrosis (CF), 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 cystic fibrosis
transmembrane conductance regulator (CFTR) in bronchial epithelial
cells from CF patients with the F508del mutation. Catabasis also
demonstrated that CAT-5571 enhanced the clearance of Pseudomonas
aeruginosa infection in preclinical models of CF, irrespective of
CFTR mutation status.
- Continued CAT-4001 preclinical program
research in rare neurodegenerative diseases. Preclinical activities
exploring the potential of CAT-4001 in diseases such as amyotrophic
lateral sclerosis (ALS) and Friedreich’s ataxia are ongoing.
Corporate
- Catabasis will host its first Investor
Day in New York on November 17, 2016, focused on its strategy in
rare diseases and the pipeline, including edasalonexent and other
programs. The Investor Day is open to members of the professional
investment community. For more information or to register, please
email CATB@macbiocom.com.
- In September 2016, Catabasis sold
2,875,000 shares of common stock in an underwritten registered
direct offering at a price of $4.00 per share and generated net
proceeds of $10.6 million. During third quarter 2016, Catabasis
sold 368,015 shares under an at-the-market offering program for net
proceeds of $1.3 million.
Third Quarter 2016 Financial Results
Cash Position: At September 30, 2016, Catabasis had cash,
cash equivalents and marketable securities of $47.3 million,
compared to $42.8 million as of June 30, 2016. We expect that our
cash, cash equivalents and marketable securities at September 30,
2016 will enable us to fund our operating expenses and capital
expenditure requirements through at least September 30, 2017. Net
cash used in operating activities for the three months ended
September 30, 2016 was $6.9 million, compared to $7.8 million for
the three months ended September 30, 2015. Net cash used in
operating activities for the nine months ended September 30, 2016
was $24.9 million, compared to $21.1 million for the nine months
ended September 30, 2015.
R&D Expenses: Research and development expenses were
$5.9 million for the three months ended September 30, 2016,
compared to $5.8 million for the three months ended September 30,
2015 and $19.2 million for the nine months ended September 30,
2016, compared to $16.4 million for the nine months ended September
30, 2015. The increase in research and development expenses for the
2016 periods relative to the 2015 periods was primarily
attributable to increased direct program costs related to the
edasalonexent MoveDMD trial.
G&A Expenses: General and administrative expenses
were $2.3 million for the three months ended September 30, 2016,
compared to $2.4 million for the three months ended September 30,
2015 and $7.7 million for the nine months ended September 30, 2016,
compared to $6.0 million for the nine months ended September 30,
2015. The increase in general and administrative expenses for the
first three quarters of 2016 relative to the prior year period was
primarily attributable to increased employee compensation costs and
increased consulting and professional expenses to support our
R&D pipeline and overall growth.
Operating Loss: Loss from operations was $8.3 million for
the three months ended September 30, 2016, compared to $8.2 million
for the three months ended September 30, 2015, and $26.9 million
for the nine months ended September 30, 2016, compared to $22.3
million for the nine months ended September 30, 2015.
Net Loss: Net loss was $8.4 million, or $0.54 per share,
for the three months ended September 30, 2016, compared to a net
loss of $8.5 million for the three months ended September 30, 2015.
Net loss for the nine months ended September 30, 2016 was $27.3
million, compared to $23.0 million for the nine months ended
September 30, 2015.
Conference Call and WebcastCatabasis will host a
conference call and webcast at 4:30 pm ET today to
provide an update on corporate developments and to discuss third
quarter 2016 financial results.
Participant Toll-Free Dial-In Number: (877)
388-2733 Participant International Dial-In Number: (541) 797-2984
Pass Code: 99833659
Please specify to the operator that you would like to join the
“Catabasis Third Quarter 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 oral small molecule that has the potential to be a
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, 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 of edasalonexent in 4-7 year-old boys
affected by Duchenne. From Part A of the MoveDMD trial, we have
reported that edasalonexent was generally well tolerated with no
safety signals observed and we observed NF-kB target
engagement. Pharmacokinetic results demonstrated edasalonexent
plasma exposure levels consistent with those previously observed in
adults, at which inhibition of NF-kB was observed.
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 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 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 Quarterly Report on
Form 10-Q for the period ended September 30, 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 September
30, Nine Months Ended September 30, 2016
2015 2016
2015 Operating expenses: Research and
development $ 5,936 $ 5,813 $ 19,190 $ 16,360 General and
administrative 2,347 2,388 7,695
5,966 Total operating expenses 8,283
8,201 26,885 22,326
Loss from operations (8,283 ) (8,201 ) (26,885 ) (22,326 )
Other (expense) income: Interest expense (199 ) (282 ) (662 ) (709
) Interest and investment income 50 - 183 - Other income, net
13 (2 ) 82 11
Total other expense, net (136 ) (284 ) (397 )
(698 ) Net loss $ (8,419 ) $ (8,485 ) $ (27,282 ) $ (23,024
) Net loss per share - basic and diluted $ (0.54 ) $ (0.55 ) $
(1.77 ) $ (4.11 )
Weighted-average common shares outstanding
used innet loss per share - basic and diluted
15,512,608 15,297,794 15,407,747
5,596,412
Catabasis Pharmaceuticals, Inc.
Condensed Consolidated Balance
Sheets
(In thousands)
(Unaudited)
September 30,
December 31, 2016 2015 Assets
Cash and cash equivalents $ 26,464 $ 62,780 Available-for-sale
securities 20,803 - Total assets 48,940 64,169
Liabilities and stockholders’
equity
Current portion of notes payable, net of discount 3,225 3,173 Notes
payable, net of current portion and discount 3,296 5,720 Total
liabilities 12,079 13,676 Total stockholders’ equity $ 36,861 $
50,493
Catabasis Pharmaceuticals, Inc.
Condensed Consolidated Statements of
Cash Flows
(In thousands)
(Unaudited)
Nine Months Ended September 30, 2016
2015 Net cash used
in operating activities $ (24,874 ) $ (21,050 ) Net cash used in
investing activities (21,300 ) (60 ) Net cash provided by financing
activities 9,858 79,151 Net (decrease)
increase in cash and cash equivalents $ (36,316 ) $ 58,041
View source
version on businesswire.com: http://www.businesswire.com/news/home/20161110006123/en/
Catabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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