Catabasis
Pharmaceuticals, Inc. (NASDAQ: CATB), a clinical-stage drug
development company built on a pathway pharmacology technology platform,
today announced that enrollment is complete in Part A of the MoveDMD
trial, a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne
muscular dystrophy (DMD). CAT-1004 is an oral small-molecule that the
Company believes has the potential to be a disease-modifying therapy for
DMD patients, regardless of the underlying dystrophin mutation. CAT-1004
is an inhibitor of NF-kB, a protein that is activated in DMD as well as
multiple other skeletal muscle disorders. In animal models of DMD,
CAT-1004 inhibited activated NF-kB, reduced muscle inflammation and
degeneration and increased muscle regeneration. DMD is a rare disease
that involves progressive muscle degeneration that eventually leads to
death and for which there are no approved therapies in the United States.
“The completion of enrollment in Part A of our MoveDMD trial is an
important milestone in our development program for CAT-1004 and we look
forward to reporting the safety, tolerability and pharmacokinetics
results from this study in early Q1 2016,” said Joanne Donovan, M.D.,
Ph.D., chief medical officer at Catabasis. “We are grateful to the
participants and their families as well as the clinical trial site staff
who have made this possible and appreciate the enthusiasm and support
that we have received from the DMD community.”
The MoveDMD trial enrolled ambulatory boys between ages 4 and 7 with a
genetically confirmed diagnosis of DMD across a range of dystrophin
mutations. The enrolled boys are steroid naive or have not used steroids
for at least six months prior to the trial. The trial is being conducted
at three sites in the United States in two sequential parts, Part A and
Part B. Part A of the study assessed the safety, tolerability and
pharmacokinetics of CAT-1004 in patients at three dosing levels
following 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. The boys in the first
part of the trial will be asked to participate, if eligible, in the
second part of the trial, and additional participants will also be
enrolled. 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
inhibits activated NF-kB, a protein that coordinates cellular response
to muscular damage, stress and inflammation and plays an important role
in muscle health. In skeletal muscle, activated NF-kB drives muscle
degeneration and suppresses muscle regeneration. In animal models of
DMD, CAT-1004 inhibited activated NF-kB, reduced muscle inflammation and
degeneration and increased muscle regeneration. In Phase 1 clinical
trials, CAT-1004 inhibited activated NF-kB and was well tolerated with
no observed safety concerns. The FDA has granted CAT-1004 orphan drug,
fast track and rare pediatric disease designations for the treatment of
DMD. The European Commission has granted CAT-1004 orphan medicinal
product designation for DMD. Catabasis is currently conducting the
MoveDMD Phase 1 / 2 trial of CAT-1004 in 4-7 year-old boys with DMD.
About MoveDMD
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 will be a two-part clinical trial investigating the
safety and efficacy of CAT-1004 in DMD. The first part of the MoveDMD
trial will include 7 days of treatment with CAT-1004 with the goal of
evaluating the safety, tolerability and pharmacokinetics of CAT-1004. In
addition, the Company will collect 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 the first part of the
trial will be asked to participate, if eligible, in the second part of
the trial. The second part of the trial will be planned to evaluate the
safety and efficacy of CAT-1004 in DMD over a 12-week period. Additional
details of the second part of the trial will be available once the first
part is complete and the protocol is finalized.
About Catabasis
Catabasis Pharmaceuticals is a
clinical-stage biopharmaceutical company focused on the discovery,
development and commercialization of novel therapeutics using its
proprietary Safely Metabolized And Rationally Targeted, or SMART, linker
technology platform. The Company's SMART linker technology platform is
based on the concept of treating diseases by simultaneously modulating
multiple targets in one or more related disease pathways. The Company
engineers bi-functional product candidates that are conjugates of two
molecules, or bioactives, each with known pharmacological activity,
joined by one of its proprietary SMART linkers. The SMART linker
conjugates are designed for enhanced efficacy and improved safety and
tolerability. The Company’s focus is on treatments for rare diseases.
The Company is also developing other product candidates for the
treatment of serious lipid disorders. For more information on the
Company's technology 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,” 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 three months ended
September 30, 2015, 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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