— Edasalonexent Phase 3 PolarisDMD Trial in Duchenne Muscular
Dystrophy Enrolling Boys Globally —
— Sustained Slowing of DMD Disease Progression with Edasalonexent
Reinforced by Long Term MoveDMD MRI Data —
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported financial results for the
first quarter ended March 31, 2019, and reviewed recent business
progress.
“We are making strong progress with enrollment in our Phase 3 PolarisDMD
trial for edasalonexent, and the trial is now enrolling patients in
seven countries. There has been significant interest from families and
enthusiasm from investigators globally, and we believe that
approximately 90% of the patients have been identified. We anticipate
all patients will be identified in the coming weeks and expect all
participating sites to be open for enrollment within a month, which will
further support scheduling the remaining families. Based on our current
projections, we expect to have top-line results from the study in the
second half of 2020, which are anticipated to support an NDA filing in
early 2021,” said Jill C. Milne, Ph.D., Chief Executive Officer of
Catabasis. “In addition, we continue to be encouraged by the long-term
MoveDMD muscle function, biomarker and growth data that reinforce
edasalonexent’s potential as a therapy for all affected by Duchenne,
regardless of mutation type.”
Recent and Upcoming Corporate Highlights
-
The Phase 3 PolarisDMD clinical trial evaluating edasalonexent in boys
affected by Duchenne muscular dystrophy (DMD) is enrolling patients at
clinical trial sites in seven countries.-
All patients are expected to be identified in the coming weeks and
all participating clinical trial sites are expected to be open for
enrollment within a month. -
Top-line results from the Phase 3 PolarisDMD trial are expected in
the second half of 2020, and the trial is intended to support an
application for commercial registration of edasalonexent in early
2021.
-
All patients are expected to be identified in the coming weeks and
-
The GalaxyDMD trial has been initiated and is enrolling boys from the
MoveDMD open-label extension and their eligible siblings. Boys who
complete the PolarisDMD trial as well as their eligible siblings will
also have the opportunity to participate in the GalaxyDMD open-label
extension trial. -
Sustained slowing of DMD disease progression with edasalonexent
treatment was reinforced by recent MoveDMD trial open-label extension
data.-
Improvements in lower leg composite MRI T2 were observed through
72 weeks of edasalonexent treatment compared to the off-treatment
control period in the Phase 2 MoveDMD trial and open-label
extension. These data were presented last week at the 2019
American Academy of Neurology Annual Meeting in Philadelphia, PA.
The lower leg composite MRI T2 is highly correlated with
functional abilities in boys with DMD in the ImagingDMD natural
history database. -
The improvements in MRI T2 are consistent with the slowing of
disease progression and preserved muscle function observed in all
assessments of muscle function (the North Star Ambulatory
Assessment, 4-stair climb, time to stand and 10-meter walk/run)
through 72 weeks of edasalonexent treatment compared to the
off-treatment control period. -
Encouraging growth data from the MoveDMD trial also showed that
boys treated with edasalonexent grew age-appropriately in both
height and weight, resulting in decreased BMI that approached the
average BMI for boys not affected by DMD.
-
Improvements in lower leg composite MRI T2 were observed through
First Quarter 2019 Financial Results
Cash Position:
As of March 31, 2019, Catabasis had cash, cash equivalents and
short-term investments of $51.7 million, compared to $37.6 million as of
December 31, 2018. Based on the Company’s current operating plan,
Catabasis expects that it has sufficient cash to fund operations through
top-line Phase 3 results and into the fourth quarter of 2020. Net cash
used in operating activities for the three months ended March 31, 2019
was $6.6 million, compared to $6.8 million for the three months ended
March 31, 2018.
R&D Expenses: Research and development expenses were $4.2
million for the three months ended March 31, 2019, compared to $5.2
million for the three months ended March 31, 2018.
G&A Expenses: General and administrative expenses were $2.1
million for the three months ended March 31, 2019, compared to $2.4
million for the three months ended March 31, 2018.
Operating Loss: Loss from operations was $6.3 million for the
three months ended March 31, 2019, compared to $7.6 million for the
three months ended March 31, 2018.
Net Loss: Net loss was $6.0 million, or $0.62 per share, for the
three months ended March 31, 2019, compared to a net loss of $7.7
million, or $2.88 per share, for the three months ended March 31, 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 first quarter 2019 financial results.
| Participant Toll-Free Dial-In Number: | (877) 388-2733 | |||
| Participant International Dial-In Number: | (541) 797-2984 | |||
| Pass Code: | 3195905 | |||
Please specify to the operator that you would like to join the
“Catabasis First Quarter 2019 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 therapy 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.
Edasalonexent is also being dosed in the open-label extension trial
GalaxyDMD. 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 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, including the anticipated timing for completion
of enrollment and 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: 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-Q for the year ended March 31, 2019, 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. |
||||||||
| Three Months Ended March 31, | ||||||||
| 2019 | 2018 | |||||||
| Operating expenses: | ||||||||
| Research and development | 4,197 | 5,247 | ||||||
| General and administrative | 2,137 | 2,392 | ||||||
| Total operating expenses | 6,334 | 7,639 | ||||||
| Loss from operations | (6,334) | (7,639) | ||||||
| Other income (expense): | ||||||||
| Interest expense | – | (57) | ||||||
| Interest and investment income | 226 | 32 | ||||||
| Other income, net | 70 | 12 | ||||||
| Total other income (expense), net | 296 | (13) | ||||||
| Net loss | $ | (6,038) | $ | (7,652) | ||||
| Net loss per share – basic and diluted | $ | (0.62) | $ | (2.88) | ||||
|
Weighted-average common shares outstanding used in net loss per share – basic and diluted |
9,686,224 | 2,655,584 | ||||||
|
Catabasis Pharmaceuticals, Inc. |
||||||||
| March 31, | December 31, | |||||||
| 2019 | 2018 | |||||||
| Assets | ||||||||
| Cash and cash equivalents | $ | 11,652 | $ | 15,294 | ||||
| Short-term investments | 40,012 | 22,276 | ||||||
| Right-of-use asset | 1,577 | – | ||||||
| Other current and long-term assets | 1,256 | 1,599 | ||||||
| Total assets | 54,497 | 39,169 | ||||||
| Liabilities and stockholders’ equity | ||||||||
| Current portion of operating lease liabilities | 1,259 | – | ||||||
| Long-term portion of operating lease liabilities | 330 | – | ||||||
| Other current and long-term liabilities | 3,039 | 4,227 | ||||||
| Total liabilities | 4,628 | 4,227 | ||||||
| Total stockholders’ equity | $ | 49,869 | $ | 34,942 | ||||
|
Catabasis Pharmaceuticals, Inc. |
||||||||||
| Three Months Ended March 31, | ||||||||||
| 2019 | 2018 | |||||||||
| Net cash used in operating activities | $ | (6,587 | ) | $ | (6,819 | ) | ||||
| Net cash used in investing activities | (17,738 | ) | – | |||||||
| Net cash provided by financing activities | 20,683 | 7,480 | ||||||||
| Net (decrease) increase in cash, cash equivalents and restricted cash | $ | (3,642 | ) | $ | 661 | |||||
Contacts
Andrea Matthews
Catabasis Pharmaceuticals, Inc.
T: (617)
349-1971
amatthews@catabasis.com