CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced financial results for the first quarter ended March 31, 2016 and corporate highlights.
“During the first quarter, Catabasis made substantial progress with edasalonexent, formerly known as CAT-1004, and CAT-2054, our two programs in the clinic,” said Jill C. Milne, Chief Executive Officer of Catabasis. “We believe we are approaching drug development for DMD in a very different way with edasalonexent as a potential disease-modifying therapy that may be effective regardless of the underlying mutation. Part B, the Phase 2 portion of the MoveDMD trial of edasalonexent, is underway and is supported by positive results from Part A, which demonstrated safety, tolerability, pharmacokinetics and importantly, proof of concept for inhibition of NF-kB activity with target engagement demonstrated in a dose-dependent manner.”
Dr. Milne continued, “We believe CAT-2054 is well positioned to be effective in both NASH and hypercholesterolemia based on the new data we’ve generated. We completed pre-clinical studies with the CAT-2000 series that provide scientific support for the potential of this Phase 2 asset in NASH. Furthermore, the Phase 2a trial of CAT-2054 in hypercholesterolemia has completed enrollment and is progressing ahead of schedule. Looking forward, we expect to be on target to reach meaningful milestones for Catabasis in the remainder of 2016 as we anticipate reporting top-line results around mid-year from the Phase 2a trial of CAT-2054 and in late 2016 from the MoveDMD trial of edasalonexent.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004): Initiation of Part B of the MoveDMD trial and Positive Safety, Tolerability, Pharmacokinetic and NF-kB Biomarker Results from Part A
- Announced dosing of the first patient in Part B of the MoveDMD trial of edasalonexent and receipt of a grant from the Muscular Dystrophy Association to support travel for trial participants. Part B of MoveDMD is a Phase 2 trial of edasalonexent for boys aged 4-7 with DMD, regardless of the underlying mutation.
- Catabasis also announced positive Part A results for safety, tolerability, pharmacokinetics and NF-kB target engagement via statistically significant reduction in NF-kB controlled gene expression in a dose-dependent manner.
- Top-line results from Part B of the MoveDMD trial are anticipated in late 2016.
CAT-2054: Promising Pre-Clinical Data in NASH; Phase 2a Trial in Hypercholesterolemia Fully Enrolled
- Results from pre-clinical studies of the CAT-2000 series in NASH showed positive effects on liver inflammation, fibrosis and steatosis.
- Catabasis anticipates presenting these pre-clinical results in NASH at an upcoming medical meeting later this quarter.
- The Phase 2a trial of CAT-2054 in hypercholesterolemia has completed enrollment.
- Top-line results from the CAT-2054 Phase 2a trial in hypercholesterolemia are expected around mid-year 2016.
Advancement of the Catabasis Pre-Clinical Pipeline
- In 2016, we plan to continue pre-clinical evaluation of CAT-4001 in animal models of Friedreich’s ataxia and Amyotrophic Lateral Sclerosis (ALS) and to conduct IND-enabling activities. If we are successful in these activities, we intend to advance CAT-4001 into a Phase 1 clinical trial in 2017.
- The Friedreich’s Ataxia Research Alliance awarded us the Kyle Bryant Translational Research Award to evaluate CAT-4001 as a potential treatment for Friedreich’s ataxia.
- We are developing a pipeline of pre-clinical assets using our SMART linker drug discovery platform to potentially treat rare diseases including ALS, Friedreich's ataxia and cystic fibrosis.
Additions to the Catabasis Board of Directors and the Formation of a Science and Technology Committee
- Announced appointment of two highly qualified industry experts, Burt Adelman, M.D. and Michael Kishbauch, to the Catabasis Board of Directors. Michael Kishbauch was appointed as a member of the Audit Committee.
- Formation of a Science and Technology Committee, with Dr. Adelman as the chair and Michael Ross, Ph.D., as a committee member.
First Quarter 2016 Financial Results
Cash Position: At March 31, 2016, Catabasis had cash, cash equivalents and marketable securities of $52.6 million, compared to $62.8 million as of December 31, 2015. We expect that our cash, cash equivalents and marketable securities at March 31, 2016 will enable us to fund our operating expenses and capital expenditure requirements through at least June 30, 2017. Net cash used in operating activities for the three months ended March 31, 2016 was $9.1 million, compared to $7.2 million for the three months ended March 31, 2015.
R&D Expenses: Research and development expenses were $6.4 million for the three months ended March 31, 2016, compared to $4.6 million for the three months ended March 31, 2015. The increase in research and development expenses for the 2016 period relative to the 2015 period was primarily attributable to increased direct program costs related to the edasalonexent MoveDMD trial and the CAT-2054 Phase 2a trial.
G&A Expenses: General and administrative expenses were $2.8 million for the three months ended March 31, 2016, compared to $1.7 million for the three months ended March 31, 2015. The increase in general and administrative expenses for the 2016 period relative to the 2015 period was primarily attributable to increased employee compensation costs and increased consulting and professional expenses to support our more advanced R&D pipeline and overall growth.
Operating Loss: Loss from operations was $9.2 million for the three months ended March 31, 2016, compared to $6.4 million for the three months ended March 31, 2015.
Net Loss: Net loss was $9.4 million, or $0.61 per share, for the three months ended March 31, 2016, compared to a net loss of $6.5 million for the three months ended March 31, 2015.
Conference Call and Webcast
Catabasis will host a conference
call and webcast at 4:30pm ET today to provide an update on corporate
developments and to discuss first quarter 2016 financial results.
Participant Toll-Free Dial-In Number: | (877) 388-2733 | ||
Participant International Dial-In Number: | (541) 797-2984 | ||
Pass Code: | 93873397 | ||
Please specify to the operator that you would like to join the “Catabasis First Quarter 2016 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 oral small molecule that has the potential to be a disease-modifying
therapy for all patients affected by Duchenne muscular dystrophy (DMD or
Duchenne), regardless of the underlying mutation. Edasalonexent inhibits
NF-kB, a protein that is activated in Duchenne and drives inflammation
and fibrosis, muscle degeneration and suppresses muscle regeneration. In
animal models of DMD, edasalonexent inhibited NF-kB, reduced muscle
degeneration and improved muscle regeneration and function, and
beneficial effects were observed in skeletal, diaphragm and cardiac
muscle. 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.
We have previously reported safety, tolerability and reduction in NF-kB
activity in Phase 1 trials in adults. We are currently conducting the
MoveDMDSM trial of edasalonexent in 4-7 year-old boys
affected by Duchenne. From Part A of the MoveDMD trial, we have reported
that edasalonexent was generally well tolerated with no safety signals
observed and successful NF-kB target engagement. Pharmacokinetic results
demonstrated edasalonexent plasma exposure levels consistent with those
previously observed in adults at which inhibition of NF-kB was observed.
About CAT-2054
CAT-2054 is an oral small molecule with a
novel mechanism of action being developed as a potential treatment of
nonalcoholic steatohepatitis (NASH) and hypercholesterolemia. By
inhibiting Sterol Regulatory Element-Binding Protein (SREBP), a master
regulator of lipid metabolism in the body, CAT-2054 has the potential to
significantly reduce LDL-C and liver fat; it may also have beneficial
effects on other metabolic parameters such as triglycerides and glucose.
This profile may differentiate CAT-2054 from currently approved
therapies and others in development. We have shown in pre-clinical
models of NASH that the CAT-2000 series significantly improves liver
inflammation, fibrosis and steatosis. We have previously reported
positive top-line Phase 1 data, including reductions in LDL-C. We are
currently conducting a Phase 2a trial of CAT-2054 in addition to high
intensity statin therapy in patients with hypercholesterolemia, which
may help guide future clinical trials in NASH and hypercholesterolemia.
About MoveDMDSM
MoveDMD is a Phase 1 / 2
clinical trial of edasalonexent (CAT-1004) in boys ages 4-7 affected
with DMD (any confirmed mutation). The MoveDMD trial is a two-part
clinical trial investigating the safety and efficacy of edasalonexent in
DMD. Part A of the MoveDMD trial evaluated the safety, tolerability and
pharmacokinetics of, and NF-kB target engagement with,
edasalonexent and showed positive results. The boys in Part A of the
trial are asked to participate, if eligible, in Part B of the trial.
Part B of the trial is a Phase 2 trial to evaluate the safety and
efficacy of edasalonexent in DMD over a 12-week treatment period and
will enroll approximately 30 boys. The primary end point is changes in
MRI of the leg muscles, and the secondary end points are age-appropriate
timed function tests: 10 meter walk/run, 4-stair climb and time to
stand. Additional assessments include muscle strength, the North Star
Ambulatory Assessment and the pediatric outcomes data collection tool
(PODCI).
About MRI
Magnetic resonance imaging (MRI) is a non-invasive
imaging technique that can visualize muscle structure and composition
and measure disease status in children with DMD. Two MRI measures used
in Duchenne to indicate muscle degeneration are T2 and fat fraction. MRI
is sensitive to changes in muscle structure and composition induced by
disease processes such as the inflammation, edema, muscle damage and fat
infiltration that occur in Duchenne. Changes in T2 may be seen in less
than 12 weeks while changes in fat fraction may take longer. Changes in
these MRI measures have been correlated with longer-term changes in
clinically meaningful measures of functional activity. Changes in MRI
can show the effects of an investigational therapy on disease
progression in Duchenne in an objective and quantifiable manner.
About Catabasis
At Catabasis Pharmaceuticals, our mission is
to bring hope and life-changing therapies to patients and their
families. We have product candidates in both rare diseases and serious
lipid disorders. Our SMART (Safely Metabolized And Rationally Targeted)
linker drug discovery platform enables us to engineer molecules that
simultaneously modulate multiple targets in a disease. We are applying
our SMART linker platform to build an internal pipeline of product
candidates for rare diseases and plan to pursue partnerships to develop
additional product candidates. For more information on the Company's
drug discovery platform and pipeline of drug candidates, 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 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; availability and timing
of results from preclinical studies and clinical trials; 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 period ended March 31,
2016, 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. |
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Three Months Ended March 31, | |||||||||||
2016 | 2015 | ||||||||||
Operating expenses: | |||||||||||
Research and development | $ | 6,436 | $ | 4,616 | |||||||
General and administrative | 2,770 | 1,744 | |||||||||
Total operating expenses | 9,206 | 6,360 | |||||||||
Loss from operations | (9,206 | ) | (6,360 | ) | |||||||
Other (expense) income: | |||||||||||
Interest expense | (243 | ) | (149 | ) | |||||||
Interest and investment income | 53 | - | |||||||||
Other (expense) income, net | (22 | ) | 9 | ||||||||
Total other expense | (212 | ) | (140 | ) | |||||||
Net loss | $ | (9,418 | ) | $ | (6,500 | ) | |||||
Net loss per share - basic and diluted | $ | (0.61 | ) | $ | (13.14 | ) | |||||
Weighted-average common shares outstanding used in net loss per share - basic and diluted | 15,335,516 | 494,590 | |||||||||
Catabasis Pharmaceuticals, Inc. |
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March 31, | December 31, | ||||||||
2016 | 2015 | ||||||||
Assets | |||||||||
Cash and cash equivalents | $ | 23,818 | $ | 62,780 | |||||
Available-for-sale securities | 28,758 | - | |||||||
Total assets | 54,269 | 64,169 | |||||||
Liabilities and stockholders’ equity | |||||||||
Current portion of notes payable, net of discount | 3,190 | 3,173 | |||||||
Notes payable, net of current portion and discount | 4,917 | 5,720 | |||||||
Total liabilities | 12,545 | 13,676 | |||||||
Total stockholders’ equity | $ | 41,724 | $ | 50,493 | |||||
Catabasis Pharmaceuticals, Inc. |
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Three Months Ended March 31, | |||||||||||
2016 | 2015 | ||||||||||
Net cash used in operating activities | $ | (9,147 | ) | $ | (7,228 | ) | |||||
Net cash used in investing activities | (29,069 | ) | (25 | ) | |||||||
Net cash (used in) provided by financing activities | (746 | ) | 16,888 | ||||||||
Net (decrease) increase in cash and cash equivalents | $ | (38,962 | ) | $ | 9,635 | ||||||