— Edasalonexent Phase 3 PolarisDMD Trial Globally Enrolling Boys
with Duchenne Muscular Dystrophy —
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
fourth quarter and full year ended December 31, 2018, and reviewed
recent business progress.
“We are off to a strong start in 2019, executing on our Phase 3 trial
and further strengthening our financial position and Board of Directors
to support our plans for transitioning to a commercial-stage
organization. We continue to generate valuable data on edasalonexent
that further reinforce its potential as a foundational therapy for
Duchenne,” said Jill C. Milne, Ph.D., Chief Executive Officer of
Catabasis. “We look forward to building on this momentum as we work
toward our goal of bringing hope and life-changing therapies to patients
and their families.”
Recent and Upcoming Corporate Highlights
-
The Phase 3 PolarisDMD clinical trial evaluating edasalonexent in boys
affected by Duchenne muscular dystrophy (DMD) is progressing well with
patients enrolling at clinical trial sites globally.-
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.
-
The Phase 3 PolarisDMD trial is a one-year, randomized,
-
Recent data from the MoveDMD trial continue to support edasalonexent
as a potential foundational treatment for DMD.-
Boys treated with corticosteroids, the current standard of care in
DMD, typically experience excess weight gain, curtailed growth and
substantially increased body mass index (BMI). In contrast,
positive growth data from the MoveDMD trial showed boys treated
with edasalonexent grew age appropriately in both height and
weight, resulting in decreased BMI that approached the average BMI
for unaffected boys. -
In addition to positive efficacy and safety results with
edasalonexent as a monotherapy, MoveDMD also provided the
foundation for combination therapy for the treatment of DMD. The
combination of edasalonexent and the approved therapy EXONDYS 51®,
developed by Sarepta Therapeutics, was well tolerated with no
safety signals. Two boys received this combination for an average
of 1 year. These clinical safety data combined with preclinical
data showing edasalonexent increased dystrophin expression in
combination with exon-skipping therapy support the potential of
edasalonexent to enhance dystrophin-targeted therapies such as
EXONDYS 51 and other therapies in development.
-
Boys treated with corticosteroids, the current standard of care in
-
Catabasis’ Board of Directors was strengthened with new appointments
of Gregg Lapointe and Joanne T. Beck, Ph.D. -
Catabasis closed a $20 million underwritten public offering in
February 2019. The proceeds will be used for clinical trial and
certain NDA-enabling activities; initial investments in commercial and
medical affairs infrastructure to support our planned transition to a
commercial-stage company; and for working capital and other general
corporate purposes.
Fourth Quarter and Full Year 2018 Financial Results
Cash
Position: As of December 31, 2018, Catabasis had cash, cash
equivalents and short-term investments of $37.6 million, compared to
$43.2 million as of September 30, 2018 and $16.4 million as of December
31, 2017. Following December 31, 2018, Catabasis raised an additional
$20.5 million in net proceeds from equity financings. Based on the
Company’s current operating plan, Catabasis believes it has sufficient
cash to fund operations into the fourth quarter of 2020. Net cash used
in operating activities for the three months ended December 31, 2018 was
$5.3 million, compared to $5.6 million for the three months ended
December 31, 2017. Net cash used in operating activities for the full
year 2018 was $23.5 million, compared to $26.8 million for the full year
2017.
R&D Expenses: Research and development expenses were $3.7
million for the three months ended December 31, 2018, compared to $4.0
million for the three months ended December 31, 2017 and $17.0 million
for the full year 2018, compared to $18.7 million for the full year 2017.
G&A Expenses: General and administrative expenses were $2.4
million for the three months ended December 31, 2018, compared to $1.7
million for the three months ended December 31, 2017 and $9.3 million
for the full year 2018, compared to $8.9 million for the full year 2017.
Operating Loss: Loss from operations was $6.1 million for the
three months ended December 31, 2018, compared to $5.5 million for the
three months ended December 31, 2017, and $26.4 million for the full
year 2018, compared to $27.1 million for the full year 2017.
Net Loss: Net loss was $6.1 million, or $0.85 per share, for the
three months ended December 31, 2018, compared to a net loss of $5.5
million, or $2.37 per share, for the three months ended December 31,
2017. Net loss for the full year 2018 was $25.9 million, or $5.12 per
share, compared to $27.4 million for the full year 2017. All per share
figures give effect to the one-for-ten reverse stock split of Catabasis
common stock that was effective on December 28, 2018.
Conference Call and Webcast
Catabasis will host a conference
call and webcast at 8:30am ET today to provide an update on corporate
developments and to discuss fourth quarter and full year 2018 financial
results.
| Participant Toll-Free Dial-In Number: | (877) 388-2733 | ||||
| Participant International Dial-In Number: | (541) 797-2984 | ||||
| Pass Code: | 3873249 | ||||
Please specify to the operator that you would like to join the
“Catabasis Fourth Quarter and Full Year 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 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
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.
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 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:
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 Annual Report on Form 10-K for the
year ended December 31, 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. |
||||||||
| Year Ended December 31, | ||||||||
| 2018 | 2017 | |||||||
| Revenue | $ | – | $ | 500 | ||||
| Operating expenses: | ||||||||
| Research and development | 17,042 | 18,682 | ||||||
| General and administrative | 9,329 | 8,912 | ||||||
| Total operating expenses | 26,371 | 27,594 | ||||||
| Loss from operations | (26,371) | (27,094) | ||||||
| Other income (expense): | ||||||||
| Interest expense | (100) | (462) | ||||||
| Interest and investment income | 425 | 160 | ||||||
| Other income, net | 176 | 32 | ||||||
| Total other income (expense), net | 501 | (270) | ||||||
| Net loss | $ | (25,870) | $ | (27,364) | ||||
| Net loss per share – basic and diluted | $ | (5.12) | $ | (12.62) | ||||
|
Weighted-average common shares outstanding used in net loss per share – basic and diluted |
5,054,823 | 2,168,153 | ||||||
|
Catabasis Pharmaceuticals, Inc. |
||||||||
| December 31, | December 31, | |||||||
| 2018 | 2017 | |||||||
| Assets | ||||||||
| Cash and cash equivalents | $ | 15,294 | $ | 16,369 | ||||
| Short-term investments | 22,276 | – | ||||||
| Total assets | 39,169 | 17,897 | ||||||
| Liabilities and stockholders’ equity | ||||||||
| Current portion of notes payable, net of discount | – | 2,479 | ||||||
| Total liabilities | 4,227 | 6,105 | ||||||
| Total stockholders’ equity | $ | 34,942 | $ | 11,792 | ||||
|
Catabasis Pharmaceuticals, Inc. |
||||||||||
| Year Ended December 31, | ||||||||||
| 2018 | 2017 | |||||||||
| Net cash used in operating activities | $ | (23,465 | ) | $ | (26,836 | ) | ||||
| Net cash (used in) provided by investing activities | (21,905 | ) | 14,883 | |||||||
| Net cash provided by financing activities | 44,295 | 4,726 | ||||||||
| Net decrease in cash and cash equivalents | $ | (1,075 | ) | $ | (7,227 | ) | ||||
Contacts
Andrea Matthews
Catabasis Pharmaceuticals, Inc.
T: (617)
349-1971
amatthews@catabasis.com