- Positive NF-kB Biomarker Data from Part A of the MoveDMD Trial in
Patients -
“Catabasis announced that dosing of the first patient has been initiated
in Part B of the MoveDMD trial. They also announced positive biomarker
results from Part A of the trial.”
Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced that dosing of the first
patient has been initiated in Part B of the MoveDMD trial, a 12-week
trial to assess the efficacy of CAT-1004 in Duchenne muscular dystrophy
(DMD). Catabasis also announced positive biomarker results from Part A
of the MoveDMD trial, demonstrating successful NF-kB target engagement.
The biomarker assay piloted in boys affected by DMD showed a
statistically significant decrease in NF-kB gene expression markers
compared to baseline at the 67 mg/kg and 100 mg/kg per day doses as well
as a statistically significant dose response in the gene expression data
measured from whole blood samples. Catabasis has previously reported
positive safety, tolerability and pharmacokinetics results from Part A
of the trial.
“We are very glad to advance CAT-1004 into Phase 2 with the initiation
of Part B of the MoveDMD trial in boys affected by DMD,” said Jill C.
Milne, Ph.D., Chief Executive Officer of Catabasis. “We are pleased with
the positive NF-kB biomarker results in Part A of the trial
demonstrating target engagement as well as the 5 - 10 fold higher
CAT-1004 concentration that we have seen in muscle compared to plasma in
pre-clinical models.”
“I am glad to see the advancement of this novel potential therapy in
boys affected by Duchenne,” said H. Lee Sweeney, Ph.D., Professor,
Director, Myology Institute at the University of Florida. “Therapies
that have the potential to make a meaningful difference are needed to
address the profound unmet medical need in DMD.”
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 is 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. The boys that
participated in Part A of the MoveDMD trial are asked to participate in
Part B and additional participants are expected to be enrolled for a
total of approximately 30 boys. We are currently identifying additional
patients who are interested in participating in Part B of the trial.
Entry criteria are similar to those in Part A. The Parent Project
Muscular Dystrophy 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 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
CAT-1004 was generally well tolerated with no safety signals observed
and successful NF-kB target engagement. 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 results. 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 and will enroll approximately 30 boys. The primary end point is
changes in MRI of the leg muscles, and the secondary end point is
age-appropriate timed function tests. 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. 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20160412005768/en/
Copyright Business Wire 2016