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News on Research


Catabasis Pharmaceuticals Announces Positive Top-Line Results from Part A of the MoveDMDSM Trial, a Phase 1 / 2 Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy 

- Trial Demonstrated Favorable Safety, Tolerability and Pharmacokinetics in Patients

- Results Support Initiation of Part B of Trial, Expected in First Half of 2016


CAMBRIDGE, MA, January 25, 2016 – Catabasis Pharmaceuticals, Inc. [...] today announced positive top-line results from Part A of the MoveDMD trial, a Phase 1 / 2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD or Duchenne). All three doses of CAT-1004 tested were generally well tolerated with no safety signals observed. [...]. Based on these results, Catabasis plans to initiate Part B of the MoveDMD trial in the first half of 2016. [...]


CAT-1004 is an oral small-molecule that the Company 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.

Ezutromid - SMT C1100


Summit Therapeutics Receives Regulatory Approval to Initiate PhaseOut DMD, a Phase 2 Clinical Trial of SMT C1100 in Patients With DMD 

January 21, 2016 

OXFORD, UK - Summit Therapeutics plc (NM) [...] announces that it has received approval from the UK Medicines and Healthcare products Regulatory Agency and the Research Ethics Committee to initiate PhaseOut DMD, a Phase 2 proof of concept clinical trial of SMT C1100 in patients with DMD.


SMT C1100 is an orally administered, small molecule utrophin modulator that the Company believes has the potential to treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation. Utrophin is functionally and structurally similar to dystrophin, a protein which is essential for the healthy function of muscles.

First patient enrolled in Summit's PhaseOut DMD, a phase 2 clinical trial of Ezutromid in boys with DMD

Oxford, UK, 17 June 2016

Summit Therapeutics plc [...] today announces that it has enrolled the first patient in PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid (formerly SMT C1100) in patients with DMD. Ezutromid dosing is expected to follow a screening period of up to 28 days.

Ezutromid is an orally administered small molecule that is designed to modulate utrophin, a protein that is structurally and functionally similar to the dystrophin protein. Dystrophin is essential for the healthy function of all muscles but is missing in patients with DMD. Utrophin modulation is a potential disease-modifying approach that could treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation.

"Ezutromid has garnered considerable interest from the patient, family and healthcare communities, which we believe is due to its potential to slow or stop progression of DMD regardless of the underlying dystrophin gene mutation," said Ralf Rosskamp, MD, Chief Medical Officer of Summit. "The enrolment of the first patient in PhaseOut DMD is a significant milestone in the clinical development of ezutromid, with the aim of studying long-term dosing of ezutromid in boys with DMD. We look forward to the possibility of demonstrating ezutromid's effect on utrophin with the initial set of 24-week biopsy data." 

The Company anticipates reporting data periodically during this trial with 24-week muscle biopsy data from the first group of patients enrolled expected to be reported in January 2017.


Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find

DEC. 31, 2015

After decades of disappointingly slow progress, researchers have taken a substantial step toward a possible treatment for Duchenne muscular dystrophy with the help of a powerful new gene-editing technique. [...]


Because the disease is devastating and incurable, and common for a hereditary illness, it has long been a target for gene therapy, though without success. An alternative treatment, drugs based on chemicals known as antisense oligonucleotides, is in clinical trials.


But gene therapy — the idea of curing a genetic disease by inserting the correct gene into damaged cells — is making a comeback. A new technique, known as Crispr-Cas9, lets researchers cut the DNA of chromosomes at selected sites to remove or insert segments.

Comment bidouiller un gène pour soigner la myopathie


Il y a trois semaines, en faisant le bilan de 2015, la revue Science décernait son titre de percée scientifique de l'année à la technologie d'édition des gènes nommée CRISPR/Cas9. Derrière ce sigle quasi imprononçable se cache une astucieuse et complexe boîte à outils permettant d'aller faire du couper-coller dans l'ADN. [...]


[...] une rafale de trois études réalisées par trois équipes américaines différentes ont toutes exploité ce traitement de texte génétique afin de soigner, avec un certain succès, des souris génétiquement modifiées pour développer la plus connue et aussi la plus grave de toutes les myopathies, la myopathie de Duchenne, aussi appelée DMD pour dystrophie musculaire de Duchenne

Le Soir, 14/01/2016

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