CAMBRIDGE, Mass.--(BUSINESS WIRE)--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.”
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.
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.
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.
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.