Catabasis Pharmaceuticals Reports First Quarter 2020 Financial Results and Reviews Business Progress
Top-Line Results Expected in Q4 2020 from Fully Enrolled Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy
“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
Recent and Upcoming Corporate Highlights
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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.
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Catabasis closed a
$26.5 million underwritten public offering inFebruary 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.
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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 atOregon 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. DuchenneUK 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
R&D Expenses: Research and development expenses were
G&A Expenses: General and administrative expenses were
Operating Loss: Loss from operations was
Net Loss: Net loss was
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
About Catabasis
At
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
Consolidated Statements of Operations (In thousands, except share and per share data) (Unaudited) |
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Three Months Ended |
||||||
|
|
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 |
|
Selected Consolidated Balance Sheets Data (In thousands) (Unaudited) |
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|
|
|
|
|
||
|
|
|
|
|
||
|
|
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 |
Selected Consolidated Statements of Cash Flows Data (In thousands) (Unaudited) |
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|
|
|
||||||
|
|
Three Months Ended |
||||||
|
|
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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Investors and Media
T: (617) 349-1971
amatthews@catabasis.com
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