CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB), a clinical-stage pharmaceutical company, today announced that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy of edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD). Edasalonexent is an oral small molecule that the Company believes has the potential to be a disease-modifying therapy for DMD patients, regardless of their underlying dystrophin mutation. Catabasis has previously reported positive safety, tolerability, pharmacokinetics and biomarker results from Part A of the trial. Based on scheduling of the enrolled patients as of September 30, 2016, the Company expects to report top-line safety and efficacy results from this Phase 2 trial in the first half of Q1 2017.
“Completing target enrollment in Part B of our MoveDMD trial is another important milestone in the development of edasalonexent. We appreciate the exceptional support that we have received from the DMD community that has resulted in enrolling Part B of the trial,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer and Senior Vice President, Clinical Development at Catabasis. “We are grateful to the participants and their families as well as the clinical trial site staff involved.”
“It is encouraging to see the progress of this potential therapy given the profound unmet medical need in Duchenne and our desire to have therapies with the potential to benefit all of those affected regardless of the underlying mutation type,” said Pat Furlong, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD).
Edasalonexent is an inhibitor of NF-kB, a protein that is activated in DMD as well as multiple other diseases. Inhibition of NF-kB has the potential to slow muscle degeneration and enhance muscle regeneration.
In the first portion of the MoveDMD trial (Part A), 17 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received edasalonexent. The boys were steroid naive or had not used steroids for at least six months prior to the trial. This portion of the trial was conducted at three sites in the U.S., and assessed the safety, tolerability and pharmacokinetics of edasalonexent in patients at three dosing levels (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day) during seven days of dosing. Sixteen of the boys who participated in the first part of the MoveDMD trial (Part A) are participating in Part B. Phase 2 of the MoveDMD trial (Part B) is a randomized, double-blind, placebo-controlled trial of approximately 30 boys to evaluate the safety and efficacy of edasalonexent in DMD over a 12-week period at five clinical trial sites in the U.S. at two dosing levels, 67 mg/kg/day and 100 mg/kg/day.
The open-label extension (Part C) was initiated in July and includes dosing with edasalonexent for 36 weeks beyond the 12-week placebo-controlled portion of the trial and will evaluate longer term safety and efficacy with the same clinical end points. PPMD and the Muscular Dystrophy Association are providing funding to support participant travel for the MoveDMD trial.
More information about the MoveDMD trial can be found on the clinical trials page of the Catabasis website and on ClinicalTrials.gov under trial identifier NCT02439216.
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 their 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 produced beneficial
effects in skeletal, diaphragm and cardiac muscle and improved function.
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 MoveDMD®
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 we observed
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 MoveDMD®
The MoveDMD trial is a
three-part clinical trial investigating the safety and efficacy of
edasalonexent in boys ages 4 – 7 affected with DMD (any confirmed
mutation). Part A of the MoveDMD trial evaluated the safety,
tolerability and pharmacokinetics of, and NF-kB target engagement
with, edasalonexent and showed positive results. Part B of the trial is
a Phase 2 trial to evaluate the safety and efficacy of edasalonexent in
DMD over a 12-week period in approximately 30 boys. The primary end
point is change in MRI of the lower 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 instrument (PODCI). Part C is an open-label
extension that includes dosing with edasalonexent for 36 weeks beyond
the 12-week placebo-controlled portion of the trial (Part B) and will
evaluate longer term safety and efficacy with the same clinical end
points as Part B.
About MRI
Magnetic resonance imaging (MRI) is a non-invasive
imaging technique that can assess 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. 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, including any
impact of scheduling and potential rescheduling of patient assessments
on the timing of the release of data from 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 June
30, 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.
