Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug
development company built on a pathway pharmacology technology platform,
today announced a collaboration to explore the potential utility of
daily physiological activity monitoring in boys affected by Duchenne
muscular dystrophy (DMD) with ActiGraph, a leading provider of objective
physical activity and sleep/wake measurement solutions for the global
scientific community. Catabasis is launching a new observational trial
to measure the daily physical activity of patients affected by Duchenne.
The trial is designed to monitor the activity of young boys who are
affected by Duchenne and collect baseline and longitudinal data using
ActiGraph activity monitoring devices. The ActiGraph monitor uses an
accelerometer to continuously measure movement, and these data can be
analyzed to measure steps and intensity of motion. Catabasis is
currently conducting the MoveDMDSM Phase 1 / 2 clinical trial
of CAT-1004 in 4-7 year-old boys affected by Duchenne. We expect that
the observational trial will enroll patients that have completed Part A
of the MoveDMD trial. Patients enrolled in this observational trial will
receive an ActiGraph activity monitor to be worn for 1 week each month.
Catabasis plans to analyze the data collected and explore the findings
from monitoring the daily physical activity of patients with DMD.
“We at Catabasis are committed not only to developing treatments for
Duchenne; we are also committed to moving the field forward by exploring
the development of non-invasive approaches that measure daily activities
to help assess quality of life for patients with Duchenne,” said Joanne
Donovan, Chief Medical Officer of Catabasis Pharmaceuticals. “Our hope
is that what we learn through the use of the ActiGraph monitors may
eventually have the potential to make the trial participation more
impactful for patients and their families.”
Christine McSherry, Executive Director of the Jett Foundation, an
organization dedicated to increasing worldwide awareness of DMD, added,
“It is extremely exciting to see companies applying innovative
technology to discover new approaches to patient evaluation and activity
assessment in DMD. We applaud both Catabasis and ActiGraph for their
forward thinking and constant consideration of the impact clinical trial
participation has on Duchenne families.”
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. 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 ActiGraph
ActiGraph’s suite of validated hardware and software products are widely
used by prominent scientific, academic, and pharmaceutical organizations
in more than 75 countries in research studies and clinical trials
involving physical activity, energy, expenditure, and sleep/wake
behavior and their relationships to a wide range of health conditions
including obesity, diabetes, cancer, CNS disorders, cardiovascular
disease, and sleep disorders. ActiGraph activity measurement solutions
have delivered objective data to many high profile population studies
including the National Health and Nutrition Examination Survey (NHANES),
the Harvard Women’s Health Study, the German National Cohort, and the
U.K. Millennium Cohort Study. ActiGraph’s activity monitors are
available for prescription use only. ActiGraph’s activity monitors are
small worn activity monitors designed for documenting physical movement
associated with applications in physiological monitoring.
About the Jett Foundation
The Jett Foundation was founded by Christine McSherry soon after her
son, Jett was diagnosed with Duchenne in 2001. McSherry decided that a
short life for Jett and thousands of boys like him was not an option. As
Executive Director of the Jett Foundation, and previously a registered
nurse at Boston hospitals for 12 years before starting the foundation,
her nursing degree and experience with patient advocacy have become
invaluable. Today, the Jett Foundation has grown into a highly effective
organization with a national reputation.
With over 16 years of experience in the fight against Duchenne the Jett
Foundation offers the community valuable experience and insight while
leveraging community resources to find a cure for Duchenne. The Jett
Foundation continues to fill the unmet needs of the Duchenne community
through research collaboration, adaptive services, clinic support,
advocacy and much more.
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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