-- Top-Line Results Expected in Q4 2020 from
Fully Enrolled Edasalonexent Global Phase 3 PolarisDMD Trial in
Duchenne Muscular Dystrophy --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
second quarter ended June 30, 2020 and reviewed recent business
progress.
“We remain on track for top-line results from our Phase 3
PolarisDMD trial for edasalonexent in Duchenne and we are looking
forward to reporting the results later this year,” said Jill C.
Milne, Ph.D., Chief Executive Officer of Catabasis. “In
anticipation of data, we are continuing to advance the program and
are working on preparations for a New Drug Application as well as
our strategy for commercialization and establishment of our supply
chain. We see edasalonexent as a potential foundational therapy for
all patients with DMD, regardless of underlying mutation.”
Phase 3 PolarisDMD Trial and GalaxyDMD Open-Label
Extension
- Top-line results from the fully enrolled Phase 3 PolarisDMD
trial of edasalonexent in Duchenne muscular dystrophy (DMD) are
expected in Q4 2020.
- The Phase 3 trial enrolled 131 boys enrolled across 8 countries
and is intended to support a new drug application (NDA) in 2021 for
commercial registration of edasalonexent.
- Due to the COVID-19 pandemic, Catabasis has implemented steps
designed to enable continued safe conduct for patients and maintain
study integrity, including the option to deliver study drug to
patients’ homes, increased flexibility in the timing of patient
visits, and utilization of telehealth.
- The open-label extension GalaxyDMD trial is enrolling boys who
have completed the Phase 3 PolarisDMD trial as well as their
interested eligible siblings. The trial is progressing as planned,
with visits every six months.
Edasalonexent Highlights
- Catabasis presented information on the edasalonexent program at
the 2020 Muscular Dystrophy Association Virtual Poster Session and
the Parent Project Muscular Dystrophy Virtual Annual Conference.
- North Star Ambulatory Assessment (NSAA), the primary endpoint
in the Phase 3 PolarisDMD trial, was shown to be a consistent and
reproducible measure of function in young boys with DMD in an
analysis of the screening and baseline values from the Phase 3
PolarisDMD trial before treatment with edasalonexent.
- The Phase 3 PolarisDMD trial was found to have enrolled a
similar patient population as the Phase 2 MoveDMD trial. An
analysis found overall similar baseline characteristics in the
patient populations in the two trials. There were no significant
differences between the two trials in baseline age, North Star
Ambulatory Assessment (NSAA) score and all three timed function
test values. These findings are believed to support the assumptions
on which the Phase 3 trial was powered.
- In the Phase 2 MoveDMD trial and open-label extension, clinical
findings support edasalonexent being associated with age-normative
growth without negative impact on bone health with no evidence of
adrenal insufficiency or clinically significant changes in adrenal
function.
- Plans are progressing for the edasalonexent non-ambulatory
trial in partnership with Duchenne UK and the trial is expected to
initiate in 2021.
Corporate Highlights
- Catabasis entered into an agreement with the Bill & Melinda
Gates Medical Research Institute to study CAT-5571 in
drug-sensitive and drug-resistant tuberculosis.
- Catabasis named Ben Harshbarger as Senior Vice President,
adding extensive senior legal and compliance experience to the
executive team.
- The Company plans to host a virtual investor and analyst event
with a leading key opinion leader on Friday, September 11, 2020, to
discuss edasalonexent and its potential in patients with DMD.
Second Quarter 2020 Financial Results
Cash Position: As of June 30, 2020, Catabasis had cash,
cash equivalents and short-term investments of $53.9 million,
compared to $55.1 million as of March 31, 2020. Based on the
Company’s current operating plan, Catabasis expects that it has
sufficient cash to fund operations through a potential NDA filing
and through the third quarter of 2021. Net cash used in operating
activities for the three months ended June 30, 2020 was $7.5
million, compared to $5.7 million for the three months ended June
30, 2019.
R&D Expenses: Research and development expenses were
$6.8 million for the three months ended June 30, 2020, compared to
$5.2 million for the three months ended June 30, 2019.
G&A Expenses: General and administrative expenses
were $2.8 million for the three months ended June 30, 2020,
compared to $2.2 million for the three months ended June 30,
2019.
Operating Loss: Loss from operations was $9.6 million for
the three months ended June 30, 2020, compared to $7.3 million for
the three months ended June 30, 2019.
Net Loss: Net loss was $9.5 million, or $0.53 per share,
for the three months ended June 30, 2020, compared to a net loss of
$7.1 million, or $0.62 per share, for the three months ended June
30, 2019.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small
molecule designed to inhibit NF-kB that is being developed as a
potential foundational therapy for all patients affected by DMD,
regardless of their underlying mutation. In DMD the loss of
dystrophin leads to chronic activation of NF-kB, which is a key
driver of skeletal and cardiac muscle disease progression. The
ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy
and safety of edasalonexent for registration purposes.
Edasalonexent is also being evaluated in the GalaxyDMD open-label
extension trial. In the MoveDMD Phase 2 trial and open-label
extension, the Company observed that edasalonexent preserved muscle
function and substantially slowed disease progression compared to
rates of change in a control period, and significantly improved
biomarkers of muscle health and inflammation. 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, please visit www.catabasis.com.
About Catabasis
At Catabasis Pharmaceuticals, our mission is to bring hope and
life-changing therapies to patients and their families. Our lead
program is edasalonexent, an NF-kB inhibitor in Phase 3 development
for the treatment of Duchenne muscular dystrophy. For more
information on edasalonexent and our Phase 3 PolarisDMD trial,
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:
the potential impact of the COVID-19 pandemic on the Company’s
business and operations; future clinical trial plans and potential
regulatory activities including, among other things, statements
about the potential commencement of the Company’s planned trial in
non-ambulatory patients, the Company’s global Phase 3 PolarisDMD
trial in DMD to evaluate the efficacy and safety of edasalonexent
for registration purposes and related statements, including the
anticipated timing for top-line results, steps that have been
implemented to address the Covid-19 pandemic and the potential
timing for the filing of an NDA; commercialization and supply chain
preparations for edasalonexent; edasalonexent potentially being a
foundational therapy for DMD patients; plans for an analyst and
investor event; and having sufficient cash to fund operations
through a potential NDA filing and through the third quarter of
2021, along with other statements containing the words “believes,”
“anticipates,” “plans,” “expects,” “may” and similar expressions,
constitute forward-looking statements within the meaning of
applicable securities regulations and laws. Actual results may
differ materially from those indicated by such forward-looking
statements as a result of various important factors, including
risks and uncertainties: related to the impact of the COVID-19
pandemic and the effectiveness of the steps we have implemented to
address the pandemic, including the use of telehealth visits;
inherent in the initiation and completion of clinical trials and
clinical development; related to whether the results of earlier
stage clinical trials will be predictive of the results of later
stage trials; related to the regulatory review and approval
process; inherent in the commercialization of marketed products;
related to successfully managing the Company’s potential
transformation into a fully integrated company; related to
competitive products, including those already approved and those in
development; inherent in transitioning from a clinical to
commercial supply chain, including the ability to enter into
long-term agreements with key contract manufacturers, overseeing
such manufacturers, and managing inventory, particularly where the
Company expects to use sole source manufacturers for the
foreseeable future; related to the availability of funding
sufficient for the Company’s foreseeable and unforeseeable
operating expenses and capital expenditure requirements; related to
other matters that could affect the clinical development,
regulatory status, availability or commercial potential of the
Company’s product candidates; and related to general market and
economic conditions, as well as the risks and uncertainties
discussed in the “Risk Factors” section of the Company’s Quarterly
Report on Form 10-Q for the period ended June 30, 2020, 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.
Consolidated Statements of
Operations
(In thousands, except share and
per share data)
(Unaudited)
Three Months Ended June 30, Six Months Ended June
30,
2020
2019
2020
2019
Operating expenses: Research and development
$
6,750
$
5,160
$
12,039
$
9,357
General and administrative
2,803
2,165
5,555
4,302
Total operating expenses
9,553
7,325
17,594
13,659
Loss from operations
(9,553
)
(7,325
)
(17,594
)
(13,659
)
Other income (expense): Interest and investment income
60
257
227
483
Other (expense) income, net
(15
)
(63
)
(93
)
7
Total other income, net
45
194
134
490
Net loss
$
(9,508
)
$
(7,131
)
$
(17,460
)
$
(13,169
)
Net loss per share - basic and diluted
$
(0.53
)
$
(0.62
)
$
(1.03
)
$
(1.24
)
Weighted-average common shares outstanding used in net loss per
share - basic and diluted
17,967,495
11,505,542
16,933,079
10,600,909
Catabasis Pharmaceuticals,
Inc.
Selected Consolidated Balance
Sheets Data
(In thousands)
(Unaudited)
June 30,
December 31,
2020
2019
Assets Cash and cash equivalents
$
51,885
$
9,899
Short-term investments
2,001
26,345
Right-of-use asset
1,326
2,349
Other current and long-term assets
1,875
3,187
Total assets
57,087
41,780
Liabilities and stockholders’ equity
Current portion of operating lease liabilities
645
1,225
Long-term portion of operating lease liabilities
718
1,028
Other current and long-term liabilities
4,864
3,807
Total liabilities
6,227
6,060
Total stockholders’ equity
$
50,860
$
35,720
Catabasis Pharmaceuticals,
Inc.
Selected Consolidated
Statements of Cash Flows Data
(In thousands)
(Unaudited)
Six Months Ended June 30,
2020
2019
Net cash used in operating activities
$
(14,455
)
$
(12,325
)
Net cash provided by (used) in investing activities
24,310
(14,229
)
Net cash provided by financing activities
31,889
20,875
Net increase (decrease) in cash, cash equivalents and restricted
cash
$
41,744
$
(5,679
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200810005500/en/
Investor and Media Contact Andrea
Matthews Catabasis Pharmaceuticals, Inc. T: (617) 349-1971
amatthews@catabasis.com
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