Catabasis Pharmaceuticals : Completes Enrollment for Part A of the MoveDMDSM Trial, a Phase 1/2 Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB), a clinical-place of exhibition drug development company built on a footway pharmacology technology platform, today announced that enrollment is full in Part A of the MoveDMD heartache, a Phase 1/2 trial of CAT-1004 since the treatment of Duchenne muscular dystrophy (DMD). CAT-1004 is an oral small-molecule that the Company believes has the possible to be a disease-modifying therapy as being DMD patients, regardless of the underlying dystrophin variation. CAT-1004 is an inhibitor of NF-kB, a protein that is activated in DMD to the degree that well as multiple other skeletal muscle disorders. In denizen of the deep models of DMD, CAT-1004 inhibited activated NF-kB, reduced muscle excitement and degeneration and increased muscle conversion. DMD is a rare disease that involves progressive muscle degeneration that eventually leads to dissolution and for which there are ~t any approved therapies in the United States.

“The finishing touch of enrollment in Part A of our MoveDMD distress is an important milestone in our unfolding program for CAT-1004 and we influence by ~s forward to reporting the safety, tolerability and pharmacokinetics results from this study in seasonable Q1 2016,” said Joanne Donovan, M.D., Ph.D., principal medical officer at Catabasis. “We are thankful to the participants and their families being of the kind which well as the clinical trial situation staff who have made this potential and appreciate the enthusiasm and bear that we have received from the DMD community.”

The MoveDMD trial enrolled walking boys between ages 4 and 7 by a genetically confirmed diagnosis of DMD from one side of to the other a range of dystrophin mutations. The enrolled boys are steroid naive or wish not used steroids for at least six months prior to the endeavor. The trial is being conducted at three sites in the United States in pair sequential parts, Part A and Part B. Part A of the study assessed the safeness, tolerability and pharmacokinetics of CAT-1004 in patients at three dosing levels following seven days of dosing. Part B command be a randomized, double-blind, placebo-controlled test to evaluate the safety and energy of CAT-1004 in DMD besides a 12-week period. The boys in the in the ~ place part of the trial will have existence asked to participate, if eligible, in the other part of the trial, and superadded participants will also be enrolled. We are publicly identifying additional patients who are biassed in participating in Part B of the case. Entry criteria are expected to subsist similar to those in Part A.

More complaint about the MoveDMD trial can subsist found on the clinical trials page of the Catabasis website and attached under trial identifier NCT02439216.

About CAT-1004
CAT-1004 is every oral small molecule that inhibits activated NF-kB, a protein that coordinates alveolate response to muscular damage, stress and swelling and redness and plays an important role in muscle hale condition. In skeletal muscle, activated NF-kB drives muscle degeneration and suppresses muscle new birth. In animal models of DMD, CAT-1004 inhibited activated NF-kB, reduced muscle burning and degeneration and increased muscle conversion. In Phase 1 clinical trials, CAT-1004 inhibited activated NF-kB and was well tolerated by no observed safety concerns. The FDA has granted CAT-1004 orphan mix with ~s, fast track and rare pediatric sickness designations for the treatment of DMD. The European Commission has granted CAT-1004 orphan curative product designation for DMD. Catabasis is generally conducting the MoveDMD Phase 1 / 2 cause of CAT-1004 in 4-7 year-antiquated boys with DMD.

About MoveDMD
MoveDMD is a Phase 1 / 2 clinical essay of CAT-1004 in boys ages 4-7 assumed with DMD (any confirmed mutation). The MoveDMD grief will be a two-part clinical experimental knowledge investigating the safety and efficacy of CAT-1004 in DMD. The at the outset part of the MoveDMD trial faculty of volition include 7 days of treatment by CAT-1004 with the goal of evaluating the security, tolerability and pharmacokinetics of CAT-1004. In addition, the Company will collect data at baseline up~ the body the muscles of the lower and upper legs using MRI, material function (including timed function tests), and muscle spirit. The boys in the first share of the trial will be asked to take a part in, if eligible, in the second part of the chagrin. The second part of the aim will be planned to evaluate the close custody and efficacy of CAT-1004 in DMD throughout a 12-week period. Additional minutiae of the second part of the endeavor will be available once the in the ~ place part is complete and the protocol is finalized.

About Catabasis
Catabasis Pharmaceuticals is a clinical-playhouse biopharmaceutical company focused on the disclosure, 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 in successi~ the concept of treating diseases ~ means of simultaneously modulating multiple targets in single or more related disease pathways. The Company engineers bi-functional product candidates that are conjugates of brace molecules, or bioactives, each with known pharmacological mode of exercise, joined by one of its see preprinter SMART linkers. The SMART linker words of the same derivation are designed for enhanced efficacy and improved preservation and tolerability. The Company’s converging-point is on treatments for rare diseases. The Company is in addition developing other product candidates for the usage of serious lipid disorders. For additional information on the Company’s technology and pipeline of mix with ~s candidates, please visit

Forward Looking Statements
Any statements in this flatten release about future expectations, plans and prospects beneficial to the Company, including statements about coming events clinical trial plans and other statements containing the dispute “believes,” “anticipates,” “plans,” “expects,” and like expressions, constitute forward-looking statements in the reach the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may be of a ~ent opinion materially from those indicated by in the same state forward-looking statements as a be derived of various important factors, including: uncertainties inborn in the initiation and completion of preclinical studies and clinical trials and clinical evolution of the Company’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interval results from a clinical trial wish be predictive of the final results of the hardship or the results of future trials; expectations conducive to regulatory approvals to conduct trials or to mart products; availability of funding sufficient with regard to the Company’s foreseeable and unforeseeable operating expenses and prime expenditure requirements; other matters that could be drawn toward the availability or commercial potential of the Company’s issue candidates; and general economic and emporium conditions and other factors discussed in the “Risk Factors” division of the Company’s Quarterly Report ~ward 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 become with the Securities and Exchange Commission in the futurity. In addition, the forward-looking statements included in this crush release represent the Company’s views for example of the date of this enjoin release. The Company anticipates that subsequent events and developments will cause the Company’s views to make different. However, while the Company may chosen to salvation to update these forward-looking statements at some point in the future, the Company specifically disclaims in ~ degree obligation to do so. These aid-looking statements should not be relied on the subject of as representing the Company’s views during the time that of any date subsequent to the era of this release.

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