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
first quarter ended March 31, 2020 and reviewed recent business
progress.
“Our fully enrolled Phase 3 PolarisDMD trial for edasalonexent
in Duchenne is progressing well and we are continuing to prepare
for top-line Phase 3 results in the fourth quarter of this year and
a subsequent NDA filing in 2021. We have initial commercialization
and supply chain preparations underway and recently strengthened
our financial position,” said Jill C. Milne, Ph.D., Chief Executive
Officer of Catabasis. “In the current environment, our priorities
are focused on the safety of patients as well as maintaining study
integrity. Advantages of our Phase 3 trial include that enrollment
was completed last year, clinical trial site visits are only every
three months, and that patients take the oral study drug at home.
Together with our clinical trial sites and consistent with recent
regulatory guidance, we have developed contingency plans that we
are implementing as needed to enable the continued conduct of the
Phase 3 trial as well as the open-label extension GalaxyDMD
trial.”
Recent and Upcoming Corporate Highlights
- The fully enrolled Phase 3 PolarisDMD trial of edasalonexent in
Duchenne muscular dystrophy (DMD) is progressing with top-line
results expected in Q4 2020.
- Patient enrollment was completed in 2019 with 131 boys enrolled
across 8 countries.
- The Phase 3 PolarisDMD trial is intended to support a new drug
application (NDA) for commercial registration of edasalonexent in
2021.
- Contingency plans are in place to enable the continued conduct
of our Phase 3 PolarisDMD and GalaxyDMD clinical trials. These
plans include the delivery of study drug to patients’ homes,
increased flexibility in the timing of patient visits, and use of
telehealth for remote visits to monitor safety and assess patients
where in-person visits are not available.
- The open-label extension GalaxyDMD trial continues to enroll
boys who have completed treatment in the Phase 3 PolarisDMD trial.
Their eligible siblings up to age 12 have the option to enroll as
well.
- Catabasis closed a $26.5 million underwritten public offering
in February 2020. The proceeds will be used for clinical trial and
other research and development activities; initial
commercialization preparations; and for working capital and other
general corporate purposes. Based on the Company’s current
operating plan, Catabasis expects that it has sufficient cash to
fund operations through a potential NDA filing and into Q3
2021.
- Catabasis plans to commercialize edasalonexent in the US and we
are evaluating our commercialization strategy outside of the US. We
are encouraged by the favorable feedback we have received from
healthcare providers and payors on the potential profile for
edasalonexent and the meaningful role it could play as a
foundational therapy for all patients with DMD, regardless of
mutation.
- Clinical findings supporting edasalonexent treatment in young
boys being associated with age-normative growth and normal adrenal
function from the Phase 2 MoveDMD trial and open-label extension
were presented at the Muscular Dystrophy Association Virtual
Clinical Trials Session by Dr. Erika Finanger, M.D., Division of
Neurology at Oregon Health and Science University and a Principal
Investigator for the Phase 2 MoveDMD, Phase 3 PolarisDMD, and
open-label extension GalaxyDMD trials of edasalonexent in DMD.
- Catabasis and Duchenne UK entered into a partnership to
evaluate edasalonexent in a Phase 2 trial in non-ambulatory DMD
patients. Duchenne UK granted Catabasis over $600,000 in funding to
support patient and clinical trial site costs. This planned Phase 2
trial is designed to assess safety and pharmacokinetics of
edasalonexent and exploratory measures of function including
cardiac, skeletal muscle and pulmonary function in non-ambulatory
DMD patients.
First Quarter 2020 Financial Results
Cash Position: As of March 31, 2020, Catabasis had cash,
cash equivalents and short-term investments of $55.1 million,
compared to $36.2 million as of December 31, 2019. Based on the
Company’s current operating plan, Catabasis expects that it has
sufficient cash to fund operations through a potential NDA filing
and into Q3 2021. Net cash used in operating activities for the
three months ended March 31, 2020 was $7.0 million, compared to
$6.6 million for the three months ended March 31, 2019.
R&D Expenses: Research and development expenses were
$5.3 million for the three months ended March 31, 2020, compared to
$4.2 million for the three months ended March 31, 2019.
G&A Expenses: General and administrative expenses
were $2.8 million for the three months ended March 31, 2020,
compared to $2.1 million for the three months ended March 31,
2019.
Operating Loss: Loss from operations was $8.0 million for
the three months ended March 31, 2020, compared to $6.3 million for
the three months ended March 31, 2019.
Net Loss: Net loss was $8.0 million, or $0.50 per share,
for the three months ended March 31, 2020, compared to a net loss
of $6.0 million, or $0.62 per share, for the three months ended
March 31, 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. Our
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 our MoveDMD Phase 2 trial and open-label
extension, we 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, statements about future clinical trial plans
including, among other things, statements about the potential
commencement of the Company’s planned Phase 2 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, including the anticipated timing for
top-line results, the potential timing for the filing of an NDA,
the Company's cash expectations, the Company’s planned transition
to a commercial-stage organization 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: risks and uncertainties related to the impact
of the COVID-19 pandemic, uncertainties inherent in the initiation
and completion of preclinical studies and clinical trials and
clinical development of the Company’s product candidates; 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, 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 March
31,
2020
2019
Operating expenses: Research and development
5,289
4,197
General and administrative
2,753
2,137
Total operating expenses
8,042
6,334
Loss from operations
(8,042
)
(6,334
)
Other income (expense): Interest and investment income
167
226
Other (expense) income, net
(77
)
70
Total other income, net
90
296
Net loss
$
(7,952
)
$
(6,038
)
Net loss per share - basic and diluted
$
(0.50
)
$
(0.62
)
Weighted-average common shares outstanding used in net loss per
share - basic and diluted
15,898,664
9,686,224
Catabasis Pharmaceuticals,
Inc.
Selected Consolidated Balance
Sheets Data
(In thousands)
(Unaudited)
March 31,
December 31,
2020
2019
Assets Cash and cash equivalents
$
13,344
$
9,899
Short-term investments
41,759
26,345
Right-of-use asset
1,799
2,349
Other current and long-term assets
2,274
3,187
Total assets
59,176
41,780
Liabilities and stockholders’ equity Current portion of
operating lease liabilities
879
1,225
Long-term portion of operating lease liabilities
875
1,028
Other current and long-term liabilities
3,711
3,807
Total liabilities
5,465
6,060
Total stockholders’ equity
$
59,176
$
41,780
Catabasis Pharmaceuticals,
Inc.
Selected Consolidated
Statements of Cash Flows Data
(In thousands)
(Unaudited)
Three Months Ended March
31,
2020
2019
Net cash used in operating activities
$
(6,989
)
$
(6,587
)
Net cash used in investing activities
(15,432
)
(17,738
)
Net cash provided by financing activities
25,624
20,683
Net increase (decrease) in cash, cash equivalents and restricted
cash
$
3,203
$
(3,642
)
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version on businesswire.com: https://www.businesswire.com/news/home/20200512005585/en/
Investors and Media Andrea Matthews
Catabasis Pharmaceuticals, Inc. T: (617) 349-1971
amatthews@catabasis.com
Catabasis Pharmaceuticals (NASDAQ:CATB)
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