CAMBRIDGE, Mass. & CHICAGO--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug development company built on a pathway pharmacology technology platform, and the Muscular Dystrophy Association (MDA), today announced a collaboration to support the Part B portion of the MoveDMD clinical trial of CAT-1004, a novel product candidate for the treatment of patients with Duchenne muscular dystrophy (DMD). Under the collaboration, MDA will provide funding for transportation to participants who need to travel to take part in the study. Part A of the MoveDMD trial has completed and Catabasis recently reported positive top-line results for safety, tolerability and pharmacokinetics. Part B of the trial is expected to initiate in the first half of 2016. The boys in Part A of the trial will be asked to participate in Part B and additional participants will also be enrolled. Catabasis is currently identifying additional patients who are interested in participating in Part B of the trial. Enrollment criteria for Part B are expected to be similar to those for Part A.
The incidence of DMD in the U.S. population is estimated to be between one in every 3,500 to one in 6,000 male births. The loss of a key muscle protein called dystrophin causes muscle wasting and weakness, eventually leading to the loss of ambulation, difficulty breathing, and heart failure. Life expectancy is currently the mid- to late 20s.
CAT-1004 is an oral small-molecule that Catabasis believes has the potential to be a disease-modifying therapy for the treatment of Duchenne, 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. In animal models of DMD, CAT-1004 inhibited NF-kB, reduced muscle degeneration and increased muscle regeneration.
“Catabasis and the Muscular Dystrophy Association share the mission to provide all boys suffering from DMD with the hope of a new and effective treatment,” said Joanne Donovan, M.D., Ph.D., Catabasis Chief Medical Officer. “We are grateful for the support from MDA for this study and look forward to success in this collaboration for our patients, their families and all of those who are impacted by this disease.”
“The DMD community desperately needs and deserves treatment options that will help families by allowing for everyday freedoms that many take for granted such as fastening a button without assistance, moving into a more comfortable position independently, or just being able to hug the people you love,” said MDA Executive Vice President and Chief Medical and Scientific Officer Valerie Cwik, M.D. “We are proud to partner with Catabasis and look forward to what is sure to be an exciting partnership that will work toward bringing urgently needed treatments to our families.”
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 in adults. 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 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. 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 period. Additional details of the second part of the
trial will be available once the protocol is finalized.
About the Muscular Dystrophy Association
MDA is leading the
fight to free individuals — and the families who love them — from the
harm of muscular dystrophy, ALS and related muscle-debilitating diseases
that take away physical strength, independence and life. We use our
collective strength to help kids and adults live longer and grow
stronger by finding research breakthroughs across diseases; caring for
individuals from day one; and empowering families with services and
support in hometowns across America. Learn how you can fund cures, find
care and champion the cause at mda.org.
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.
