Rare Disease Report

DMD Clinical Trial Update at PPMD Patient Connect Meeting Part 2

JUNE 30, 2017
James Radke
The Friday afternoon at Parent Project Muscular Dystrophy (PPMD) Patient Connect Meeting in Chicago, Illinois saw more input from a variety of companies showing off their latest clinical data. Below is a summary of those presentations. To view our summary of the clinical data presented Friday morning, click here.
Joanne Donovan, MD, PhD, chief medical officer at Catabasis discussed the latest clinical data for their compound, edasalonexent, a NF-κB inhibitor, is currently being studied in a Phase 2 study involving DMD patients.
NF-κB is activated by mechanical stress and triggers a series of inflammatory processes as well as suppress muscle regeneration while driving muscle degeneration.
Regarding the Phase 2 MoveDMD study, Dr. Donovan said that in the placebo-controlled 12-week study, the drug was found to be superior to placebo in a variety of functional tests.
Dr. Donavan noted that while their Phase 2 study is continuing, they are also collaborating with Sarepta to see if exon skipping therapy plus edasalonexent is beneficial.
Moving forward, the company is currently designing its Phase 3 study that they hope to start in 2018.
Elias Kouchakji, MD, vice president, clinical development and drug safety at Fibrogen presented data for their drug pamrevlumab, a monoclonal antibody against Connective Tissue Growth Factor (CTGF).
The drug is being studied in a variety of patient populations, including pancreatic cancer, idiopathic pulmonary fibrosis, as well as DMD.
The rationale for testing pamrevlumab in DMD is that the drug can block CTGF, which is high in higher than normal in DMD boys.
A Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) is currently underway to test the safety and efficacy of the drug in non-ambulatory patients with DMD. Currently, 12 boys are enrolled in the study. They hope to enroll 22 boys with DMD with the primary endpoint being change in pulmonary function, as well muscle function tests and change in fibrosis.
Dr. Kouchakji noted that the company has struggled to get boys enrolled in the study. To learn more about the trial, click here.
George Mulligan, PhD, vice president, translational medicine at Mitobridge talked about their compound MA-0211which is in preclinical development.
Mitobridge is a relatively new company, started in 2013, that is focused on mitochondrial diseases. With regard to DMD, Dr. Mulligan stated that mitochondrial dysfunction has been reported in DMD patients.
Mitobridge focus was to manipulate PPARd, which regulates mitochondrial function in skeletal muscle.  Early studies by Mitobridge showed that their compound, MA-0211, which binds to PPARd, reduced muscle damage and cell death in a mouse model for DMD. Mouse studies also showed MA-0211 improved cardiac function and the animal’s endurance.
Moving forward, Mitobrige is partnering with Astellas to start clinical studies in humans, beginning with a phase 1 study in the summer of 2017 in healthy volunteers with a clinical study starting in 2018 that involves ambulatory DMD patients.

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