- Trial Demonstrated Favorable Safety, Tolerability and
Pharmacokinetics in Patients -
- Results Support Initiation of Part B of Trial, Expected in First
Half of 2016 -
Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced positive data from Part A of
the MoveDMD trial of CAT-1004 for Duchenne muscular dystrophy (DMD). The
objectives for Part A were to establish safety, tolerability and
pharmacokinetics in boys with DMD age 4 to 7 to support initiation of
Part B of the MoveDMD trial, a 12-week trial to assess efficacy. All
three doses of CAT-1004 tested were generally well tolerated with no
safety signals observed. The majority of adverse events were mild in
nature, and the adverse events observed were diarrhea (4 events), soft
feces (3 events) and upper abdominal pain (2 events). There were no
serious adverse events and no drug discontinuations. Six of the total of
9 adverse events were observed in the highest dose group. There were no
trends or safety issues in labs, physical exams, EKG or vital signs.
From the pharmacokinetics data, we conclude that doses of 33 mg/kg given
2 or 3 times daily are predicted to achieve systemic exposures at which
NF-kB inhibition was observed in adults. Pharmacokinetics demonstrated
dose-dependent increases in exposure and meal composition had a modest
effect. With single doses of 33 mg/kg, there were minimal differences in
either area under the curve (AUC) or maximum serum concentration (Cmax)
when CAT-1004 was administered either with a high-fat or a low-fat meal.
Based on these results, the daily doses selected for Part B will be 67
or 100 mg/kg administered with food at 33 mg/kg either 2 or 3 times
daily. From these results, Catabasis plans to initiate Part B of the
MoveDMD trial in the first half of 2016, subject to regulatory approval
of the proposed protocol. The Parent Project Muscular Dystrophy and the
Muscular Dystrophy Association are providing funding to support
participant travel for the MoveDMD trial.
“The favorable safety, tolerability and pharmacokinetic results in boys
affected by DMD are encouraging and we look forward to starting Part B
of the MoveDMD trial. CAT-1004 is a potential disease-modifying oral DMD
therapy regardless of dystrophin mutation type,” said Joanne Donovan,
M.D., Ph.D., Chief Medical Officer of Catabasis. “We have received
significant support and enthusiasm for our MoveDMD trial and CAT-1004
from parents, advocacy groups, investigators and study staff, and we
thank them for their participation and support.”
“The unmet medical need in DMD is profound and potential therapies that
could make a meaningful difference are needed” said Richard Finkel,
M.D., Division Chief, Division of Neurology, Department of Pediatrics at
Nemours Children’s Health System. “Showing positive safety, tolerability
and pharmacokinetics results is an important milestone in the
development of CAT-1004. I look forward to the advancement of this novel
potential therapy.”
CAT-1004 is an oral small-molecule that the Company believes has the
potential to be a disease-modifying therapy for the treatment of DMD,
regardless of the underlying dystrophin mutation. CAT-1004 is an
inhibitor of NF-kB, a protein that is chronically activated in DMD as
well as multiple other skeletal muscle disorders and rare diseases. In
animal models of DMD, CAT-1004 inhibited NF-kB, reduced muscle
degeneration and increased muscle regeneration.
The MoveDMD trial is being conducted in two sequential parts, Part A and
Part B. In Part A of the MoveDMD trial, 17 ambulatory boys between ages
4 and 7 with a genetically confirmed diagnosis of DMD across a range of
dystrophin mutations received CAT-1004. The boys were steroid naive or
had not used steroids for at least six months prior to the trial. Part A
of the trial was conducted at three sites in the U.S., and assessed the
safety, tolerability and pharmacokinetics of CAT-1004 in patients at
three dosing levels (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day)
during seven days of dosing. Part B will be a randomized, double-blind,
placebo-controlled trial to evaluate the safety and efficacy of CAT-1004
in DMD over a 12-week period at 5 clinical trial sites in the U.S. at
two dosing levels, 67 mg/kg/day and 100 mg/kg/day, subject to regulatory
approval. The boys that participated in Part A of the MoveDMD trial will
be asked to participate in Part B, and additional participants will also
be enrolled for up to approximately 30 boys. We are currently
identifying additional patients who are interested in participating in
Part B of the trial. Entry criteria are expected to be similar to those
in Part A.
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 CAT-1004
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.
CAT-1004 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, CAT-1004 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 CAT-1004 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 CAT-1004 in 4-7 year-old boys affected
by Duchenne. From Part A of the MoveDMD trial, we have reported that all
three doses of CAT-1004 tested were generally well tolerated with no
safety signals observed. Pharmacokinetic results demonstrated CAT-1004
average plasma exposure levels consistent with those previously observed
in adults at which inhibition of NF-kB was observed.
About MoveDMDSM
MoveDMD is a Phase 1 / 2 clinical trial of 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
CAT-1004 in DMD. Part A of the MoveDMD trial evaluated the safety,
tolerability and pharmacokinetics of CAT-1004 with positive top-line
results. In addition, the Company collected data at baseline on the
muscles of the lower and upper legs using MRI, physical function
(including timed function tests), and muscle strength. The boys in Part
A of the trial will be asked to participate, if eligible, in Part B of
the trial. Part B of the trial will be planned to evaluate the safety
and efficacy of CAT-1004 in DMD over a 12-week treatment period.
Preparations are underway for Part B of the trial.
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. 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 Annual Report
on Form 10-K for the year ended December 31, 2015, and in other filings
that the Company may make with the Securities and Exchange Commission in
the future. The forward-looking statements contained in this
presentation reflect our current views with respect to future events,
and we do not undertake, and specifically disclaim, any obligation to
update any forward-looking statements.
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