Date: 2015-06-18
Type of information: Treatment of the first patient
phase: 1-2
Announcement: treatment of the first patient
Company: Catabasis Pharmaceuticals (USA - MA)
Product: CAT -1004 - N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide
Action
mechanism: NF-κB inhibitor. CAT -1004 is a conjugate of salicylate and the omega 3 fatty acid docosahexaenoic acid. CAT -1004 has been designed to inhibit NF-κB, a key mediator of cellular injury. In preclinical studies, it has been shown to reduce inflammation and increase muscle regeneration. In a phase 1 study, CAT -1004 was well-tolerated and demonstrated a significant reduction of activated NF-κB. The FDA has granted CAT-1004 orphan drug designation for the treatment of DMD.
Disease: Duchenne muscular dystrophy (DMD)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country: USA
Trial
details: The purpose of this Phase 1/2 ascending multiple-dose, open-label study is to evaluate the safety and pharmacokinetics (PK) of CAT-1004 in pediatric patients with a confirmed diagnosis of Duchenne muscular dystrophy (DMD). CAT-1004 is an orally administered small molecule targeted to inhibit activated NF-κB, a molecule that is activated from infancy in DMD and which is central to causing muscle damage and preventing muscle regeneration. Data on magnetic resonance imaging of the lower and upper leg muscles, physical function (including timed function tests) and muscle strength will be collected at baseline. Dose escalation between cohorts will only occur after patients in the prior cohort have completed dosing and data have been evaluated by the Sponsor, Investigator and an independent data safety monitoring board (DMSB). The intention is that enrolled patients, if eligible, will return for additional treatment with CAT-1004 in a randomized, placebo-controlled, 12-week extension of the current study, which is anticipated to be submitted as a protocol amendment. (NCT02439216)
Latest
news: * On June 17, 2015, Catabasis Pharmaceuticals, a clinical-stage drug development company built on a pathway pharmacology technology platform, announced that dosing of the first patient has been initiated in 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, by inhibiting activated NF-kB. NF-kB is a protein that plays an important role in regulating muscle health, and chronic activation of NF-kB has been reported in multiple skeletal muscle disorders, such as DMD. Preclinical studies have demonstrated that CAT-1004 can not only reduce muscle inflammation and degeneration, but also promote 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 MoveDMD trial will enroll approximately 18 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD, regardless of the specific dystrophin mutation. The enrolled boys will be steroid naive or have not used steroids for at least six months prior to the trial. The trial will be conducted at three sites in the United States in two sequential parts, Part A and Part B. Part A of the study will assess the safety, tolerability and pharmacokinetics of CAT-1004 in patients at three dosing levels following seven days of dosing. The primary endpoint for Part A is safety. Part B will be a randomized, double-blind, placebo-controlled trial. The primary efficacy endpoint in Part B will be change in muscle inflammation as measured by magnetic resonance imaging (MRI) of leg muscles. Additional measures of physical function and muscle strength will be collected.
