Catabasis Pharmaceuticals Reports Fourth Quarter and Full Year 2018 Financial Results and Reviews Business Progress

March 14, 2019 Off By BusinessWire

— 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.
  • 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.
  • 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.
Consolidated Statements of
Operations

(In thousands, except share and per share data)
(Audited)

 
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.
Selected Consolidated
Balance Sheets Data

(In thousands)
(Audited)

 
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.
Selected Consolidated
Statements of Cash Flows Data

(In thousands)
(Audited)

 
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
[email protected]