Catabasis Pharmaceuticals Reports Third Quarter 2018 Financial Results and Reviews Business Progress
-- Edasalonexent Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy Underway and Starting Enrollment --
“We have made great advancements across our edasalonexent program over
the last quarter. Clinical trial sites for our Phase 3 PolarisDMD trial
are now open for enrollment and we are receiving tremendous interest and
positive feedback from families and physicians, reflecting the high
level of unmet need in Duchenne and enthusiasm for edasalonexent,” said
Recent and Upcoming Corporate Highlights
-
Multiple sites open for enrollment for the Phase 3 PolarisDMD clinical
trial evaluating edasalonexent in boys affected by Duchenne muscular
dystrophy (DMD)
- The Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial. Catabasis plans to enroll approximately 125 patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who have not been on steroids for at least 6 months.
- Top-line results from the Phase 3 PolarisDMD trial are expected in the second quarter of 2020, and the trial is intended to support an application for commercial registration of edasalonexent.
-
New MoveDMD Phase 2 trial and open-label extension data through 72
weeks of edasalonexent treatment
-
Preserved muscle function and sustained disease-modifying effects
in all assessments of physical function in boys with DMD was seen
through 72 weeks of edasalonexent treatment compared to the
off-treatment control period. Data were presented at the
International Congress of theWorld Muscle Society inOctober 2018 . - Significant decreases in muscle enzymes through 72 weeks were also seen in boys treated with edasalonexent, supporting the durability of edasalonexent treatment effects and positive effects on muscle integrity.
- Significantly decreased heart rate towards age-normative values was observed and supports the potential beneficial cardiac effects of edasalonexent. Boys with DMD in this age range typically have elevated heart rates, which is the first cardiac manifestation in boys with DMD. Cardiomyopathy is the leading cause of mortality in DMD.
- Edasalonexent continued to be well tolerated with no safety signals observed in the trial. Boys treated with edasalonexent continued to follow age-appropriate growth curves with age-appropriate increases in weight and height.
- Catabasis expects to present additional clinical data from the MoveDMD trial with edasalonexent in 2019.
-
Preserved muscle function and sustained disease-modifying effects
in all assessments of physical function in boys with DMD was seen
through 72 weeks of edasalonexent treatment compared to the
off-treatment control period. Data were presented at the
-
New magnetic resonance imaging (MRI) data support MRI T2 as a
potential marker of clinical outcome in DMD
- Data from ImagingDMD, the largest natural history database of MRI measurements in more than 150 boys with DMD, show a strong correlation of lower leg composite MRI T2 with clinically relevant functional abilities and DMD disease progression.
- These results highlight the clinical importance of the significant improvement observed in the Catabasis Phase 2 MoveDMD trial and open-label extension following 12, 24, 36 and 48 weeks of edasalonexent treatment for lower leg MRI T2 compared to the off-treatment control period.
-
ImagingDMD and MoveDMD data were presented at the
International Congress of theWorld Muscle Society inOctober 2018 .
-
Preclinical collaboration with
University of Texas Southwestern (UT Southwestern) to explore the potential benefits of edasalonexent on cardiac function in Duchenne and Becker muscular dystrophies-
The collaboration is with
Pradeep Mammen , M.D., founder and Medical Director of theNeuromuscular Cardiomyopathy Clinic at theUT Southwestern Medical Center and Co-Director of the Nation Institute of Health Sponsored UT Southwestern Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center.
-
The collaboration is with
Third Quarter 2018 Financial Results
Cash Position: As of
Debt: All principal and interest due under the credit facility
Catabasis established in 2014 had been paid 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
Conference Call and Webcast
Catabasis will host a conference
call and webcast at
Participant Toll-Free Dial-In Number: | (877) 388-2733 | |||
Participant International Dial-In Number: | (541) 797-2984 | |||
Pass Code: | 5269109 | |||
Please specify to the operator that you would like to join the “Catabasis Third Quarter 2018 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 new standard of care for all patients affected by DMD,
regardless of their underlying mutation. Edasalonexent inhibits NF-kB,
which is a key link between loss of dystrophin and disease progression
in DMD. NF-kB has a fundamental role in skeletal and cardiac muscle
disease in DMD. We are currently enrolling our global Phase 3 PolarisDMD
trial to evaluate the efficacy and safety of edasalonexent for
registration purposes. 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. Edasalonexent continues to be dosed in the open-label
extension of the MoveDMD trial. 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 future clinical trial plans including, among
other things, statements about the Company’s global Phase 3 PolarisDMD
trial in DMD to evaluate the efficacy and safety of edasalonexent for
registration purposes, the Company’s plans to report top-line results
from this trial in the second quarter of 2020, and the Company’s
expectation that its current operating plan provides for cash to fund
operations into the second quarter of 2020, 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; 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 quarter ended
Catabasis Pharmaceuticals, Inc. |
||||||||||||
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||
2018 | 2017 | 2018 | 2017 | |||||||||
Revenue | $ | - | $ | 250 | $ | - | $ | 250 | ||||
Operating expenses: | ||||||||||||
Research and development | 3,897 | 4,776 | 13,383 | 14,693 | ||||||||
General and administrative | 2,111 | 2,426 | 6,900 | 7,189 | ||||||||
Total operating expenses | 6,008 | 7,202 | 20,283 | 21,882 | ||||||||
Loss from operations | (6,008) | (6,952) | (20,283) | (21,632) | ||||||||
Other income (expense): | ||||||||||||
Interest expense | (10) | (105) | (100) | (381) | ||||||||
Interest and investment income | 177 | 45 | 252 | 128 | ||||||||
Other income (loss), net | 162 | (5) | 321 | 18 | ||||||||
Total other income (expense), net | 329 | (65) | 473 | (235) | ||||||||
Net loss | $ | (5,679) | $ | (7,017) | $ | (19,810) | $ | (21,867) | ||||
Net loss per share - basic and diluted | $ | (0.08) | $ | (0.31) | $ | (0.45) | $ | (1.03) | ||||
Weighted-average common shares outstanding used in net loss per share - basic and diluted | 71,038,419 | 22,563,174 | 43,603,950 | 21,163,591 | ||||||||
Catabasis Pharmaceuticals, Inc. |
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September 30, | December 31, | |||||
2018 | 2017 | |||||
Assets | ||||||
Cash and cash equivalents | $ | 19,876 | $ | 16,369 | ||
Short-term investments | 23,363 | - | ||||
Total assets | 44,699 | 17,897 | ||||
Liabilities and stockholders’ equity | ||||||
Current portion of notes payable, net of discount | - | 2,479 | ||||
Total liabilities | 4,180 | 6,105 | ||||
Total stockholders’ equity | $ | 40,519 | $ | 11,792 | ||
Catabasis Pharmaceuticals, Inc. |
||||||
Nine Months Ended September 30, | ||||||
2018 | 2017 | |||||
Net cash used in operating activities | $ | (18,197) | $ | (21,199) | ||
Net cash (used in) provided by investing activities | (22,999) | 14,883 | ||||
Net cash provided by financing activities | 44,703 | 4,433 | ||||
Net increase in cash and cash equivalents | $ | 3,507 | $ | (1,883) |
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Source:
Investor and Media Contact
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com