-- Edasalonexent Phase 3 PolarisDMD Trial in
Duchenne Muscular Dystrophy Underway and Starting Enrollment --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
third quarter ended September 30, 2018, and reviewed recent
business progress.
“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 Jill C. Milne, Ph.D., Chief
Executive Officer of Catabasis. “We presented new data showing
preserved muscle function and stabilized disease progression
following 72 weeks of edasalonexent treatment compared to control.
We also presented data that could potentially contribute to
establishing MRI as an endpoint for clinical outcome in Duchenne.
We are excited to advance our understanding of the potential
cardiac benefits of edasalonexent through a preclinical
collaboration with Dr. Mammen from UT Southwestern who treats
patients with Duchenne and Becker muscular dystrophies. Together,
these advancements support the potential value of edasalonexent and
get us closer to our goal of making edasalonexent available to all
those affected by Duchenne with the hope of enabling patients to
maintain their functional abilities longer.”
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 the World Muscle Society
in October 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.
- 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 the World Muscle Society
in October 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 the Neuromuscular
Cardiomyopathy Clinic at the UT Southwestern Medical Center and
Co-Director of the Nation Institute of Health Sponsored UT
Southwestern Senator Paul D. Wellstone Muscular Dystrophy
Cooperative Research Center.
Third Quarter 2018 Financial Results
Cash Position: As of September 30, 2018, Catabasis had
cash, cash equivalents and short-term investments of $43.2 million,
compared to $49.9 million as of June 30, 2018. Based on the
Company’s current operating plan, Catabasis believes it has
sufficient cash to fund operations into the second quarter of 2020.
Net cash used in operating activities for the three months ended
September 30, 2018 was $5.8 million, compared to $7.4 million for
the three months ended September 30, 2017. Net cash used in
operating activities for the nine months ended September 30, 2018
was $18.2 million, compared to $21.2 million for the nine months
ended September 30, 2017.
Debt: All principal and interest due under the credit
facility Catabasis established in 2014 had been paid as of
September 30, 2018. There are no further payments due under the
credit facility.
R&D Expenses: Research and development expenses were
$3.9 million for the three months ended September 30, 2018,
compared to $4.8 million for the three months ended September 30,
2017 and $13.4 million for the nine months ended September 30,
2018, compared to $14.7 million for the nine months ended September
30, 2017. The decrease in research and development expenses was
primarily attributable to a decrease in post-restructuring employee
compensation and other non-program costs.
G&A Expenses: General and administrative expenses
were $2.1 million for the three months ended September 30, 2018,
compared to $2.4 million for the three months ended September 30,
2017, and $6.9 million for the nine months ended September 30, 2018
compared to $7.2 million for the nine months ended September 30,
2017.
Operating Loss: Loss from operations was $6.0 million for
the three months ended September 30, 2018, compared to $7.0 million
for the three months ended September 30, 2017 and $20.3 million for
the nine months ended September 30, 2018, compared to $21.6 million
for the nine months ended September 30, 2017.
Net Loss: Net loss was $5.7 million, or $0.08 per share,
for the three months ended September 30, 2018, compared to a net
loss of $7.0 million, or $0.31 per share, for the three months
ended September 30, 2017. Net loss for the nine months ended
September 30, 2018 was $19.8 million, compared to $21.9 million for
the nine months ended September 30, 2017.
Conference Call and WebcastCatabasis will host a
conference call and webcast at 8:30am ET today to provide
an update on corporate developments and to discuss third quarter
2018 financial results.
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 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 CatabasisAt 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
development for the treatment of Duchenne muscular dystrophy. Our
global Phase 3 PolarisDMD trial is currently enrolling boys
affected by Duchenne. For more information on edasalonexent and our
Phase 3 PolarisDMD trial, please visit www.catabasis.com or
www.twitter.com/catabasispharma.
Forward Looking StatementsAny 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 September 30, 2018, 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, 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.Condensed Consolidated Balance Sheets(In
thousands)(Unaudited)
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.Condensed Consolidated Statements of Cash Flows(In
thousands)(Unaudited)
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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version on businesswire.com: https://www.businesswire.com/news/home/20181113005340/en/
Investor and Media ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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