Catabasis Pharmaceuticals Phase 1 Data on Edasalonexent (CAT-1004), a Potential Disease-Modifying Therapy Being Developed for...
January 19 2017 - 8:00AM
Business Wire
-- Edasalonexent Was Safe, Well Tolerated and
Generated Positive Biomarker Results in Adult Subjects --
-- Top-Line Phase 2 Results in Boys with
Duchenne Muscular Dystrophy On Track: Expected in the First Half of
Q1 2017 --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced the publication of Phase
1 data on edasalonexent in adult subjects. Edasalonexent is a
potential disease-modifying therapy being developed for Duchenne
muscular dystrophy (DMD). The Phase 1 trials demonstrated that
edasalonexent (CAT-1004), an oral inhibitor of NF-kB, was safe,
well tolerated, and inhibited activated NF-kB in adult subjects and
the data are presented in an article titled “A Novel NF-kB
Inhibitor, Edasalonexent (CAT-1004), in Development as a
Disease-Modifying Treatment for Patients with Duchenne Muscular
Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics
in Adult Subjects” in the Journal of Clinical Pharmacology (J Clin
Pharmacol. 2017 Jan 11. doi: 10.1002/jcph.842.)
In Duchenne muscular dystrophy (DMD), NF-kB is activated in
muscle from infancy regardless of the underlying dystrophin
mutation and drives inflammation and muscle degeneration while
inhibiting muscle regeneration. Edasalonexent (CAT-1004) is a
bifunctional orally administered small molecule that covalently
links two compounds known to inhibit NF-kB, salicylic acid and
docosahexaenoic acid (DHA). The three placebo-controlled trials in
adult subjects assessed the safety, pharmacokinetics and
pharmacodynamics of single or multiple edasalonexent doses up to
6000 mg (approximately 100 mg/kg). Seventy-nine adult subjects
received edasalonexent and 25 received placebo. The NF-kB pathway
and proteosome gene expression profiles in peripheral mononuclear
cells were significantly decreased after 2 weeks of edasalonexent
treatment. NF-kB activity was inhibited following a single dose of
edasalonexent but not by equimolar doses of its component
bioactives salicylic acid and DHA dosed in combination.
Edasalonexent was well tolerated, and the most common adverse
events were mild diarrhea and headache.
“These Phase 1 safety, tolerability and positive NF-kB biomarker
results support edasalonexent development in Duchenne muscular
dystrophy and potentially other diseases. The Phase 1 results in
adults informed on the dose and dose schedule for the current
MoveDMD trial in 4-7 year-old boys affected by Duchenne, where
similar Phase 1 results were seen,” said Joanne Donovan, M.D.,
Ph.D., Chief Medical Officer of Catabasis. “We look forward to the
results from the edasalonexent Phase 2 clinical trial in boys
affected by Duchenne, which are expected in the first half of Q1
2017.”
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 CatabasisAt 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 StatementsAny 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 September 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20170119005231/en/
Catabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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