Rare Disease Report

DMD Clinical Trial Update at PPMD Patient Connect Meeting Part 1

JUNE 30, 2017
James Radke
The first of 4 clinical trial update sessions was held this morning at Parent Project Muscular Dystrophy (PPMD) Patient Connect Meeting in Chicago, Illinois. Below is a summary of those presentations.
Paolo Bettica, MD, PhD, vice president research and development at Italfarmaco talked about  the company’s givinostat for children with DMD.
Givinostat is a histone deacetylase inhibitor that has anti-inflammatory activities. In DMD it is believed that givinostat improved muscle integrity.
The have an ongoing Phase 2 study and have just started a Phase 3 study.
Providing an update on their Phase 2 study (Study 43), 20 boys with DMD were enrolled.
Muscle biopsies showed after 1 year of givinostat treatment, boys had increased muscle fiber area fraction and decreased necrosis, fibrosis, and fatty replacement.

And of the 20 boys, only 2 boys have to date lost ambulation.
Thomas Meier, PhD, Chief Executive Officer of Santhera provided an update on clinical trial with Raxone (idebenone) and its ability to improve lung function in DMD patients.
As background, from the ages of 10 to 20, boys with DMD dramatically lose their pulmonary functions that creates a plethora of other problems. Idelbenone was tested in a Phase 3 DELOS trial a few years ago involving patients not taking glucocorticoids and while the results were not significant, Dr. Meier noted that post-hoc analysis in that study showed patients who more severe symptoms appeared to benefit from idebenone treatment.

The new phase 3 study is currently underway and only includes patients also taking glucocorticoids.
For more information about the phase 3 study, visit SIDEROSDMD
Eric Hoffman, PhD, president and chief executive officer of ReveraGen summarizes the lastest data with vamorolone, a first in class steroid that they hope will be as effective as glucocorticoids in DMD but without the side effects normally seen with the latter treatment.
Based on their phase 1 study, the drug appears to have much fewer side effects (stunted growth, delayed puberty, adrenal suppression, metabolic disturbance, mineral corticoid receptor activation, immune suppression).
Dr Hoffman provided an update on their phase 2 studies. The phase 2a study is fully enrolled and a phase 2b study will be a 6-month pivotal study that will begin in September 2017. They plan to enroll 100 DMD boys aged 4 to 7 years of age and are steroid naïve. For more information, click here.


Linda Marban, PhD, president and chief executive officer of Capricor Therapeutics talked about CAP-1002, a cardiac cell therapy.
In the HOPE-Duchenne trial, 13 boys were given CAP-1002 and 12 boys given placebo to determine if treatment would improve cardiac function in DMD boys. Interim results reported in April showed that CAP-1002 was safe and well tolerated. Further, the study showed the drug not only helped cardiac muscle health (reduced scar tissue, increased wall thickening).

Finally, Dr. Marban noted that animal studies indicate that CAP-1002 may not only improve the health of cardiac tissue but may also improve skeletal muscle function. Further, measuring skeletal performance in the boys in the HOPE-Duchenne trial did suggest that the drug may improve skeletal muscles as well as cardiac muscles.
In the next study, the HOPE-2 study should begin soon.
Michael Binks, MD Vice President for Rare Disease Clinical Research at Pfizer provided an update on domagrozumab, a monoclonal anti-myostatin antibody. Myostatin restrains muscle growth, meaning a therapy that inhibits could benefit DMD patients.
At the morning session, Binks provided an update on the company’s clinical trial, most notably mentioning that the study is ongoing and, at present, no data is available yet. The study is, however, fully enrolled.

BMS-986089 / RG6206
Cliff Bechtold, MS of Bristol-Myers Squibb (BMS) spoke about the company’s anti-myostatin adnectin program. Bechtold noted that BMS is partnering with Roche on this program (hence the 2 working names for the drug).
In a phase 1 study, the drug was well-tolerated and also observed increased thigh muscle volume and total lean body mass.
In 2015, BMS started an ongoing study in DMD boys and, at present, no results are available. A Phase 2/3 a 48-week, placebo-controlled study involving boys with DMD has started enrolling patients, though, and it will include patients aged 6 to 11 years of age.

For more information, visit BMStrialDMD.com
Diana Escolar, MD, chief medical officer at Akashi Therapeutics summarized the latest data with HT-100, a drug in development to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD boys.
During her presentation, Escolar noted that HT-100 is an oral drug that has been tested in 3 clinical trials, a 7-day dosing study, a 12-month safety study, and an open label study. She presented data that showed boys taking HT-100 had increased muscle strength, including patients who were non-ambulatory.

Further, exploratory analysis using data from the HT-100 studies and comparing it to an external control group showed that HT-100 may also improve muscle function.
Dr. Escolar noted that in the study, 1 patient in the higher dose cohort died, and after an extensive analysis, it was discovered that the patient had extremely high levels of HT-100 (halofuginone) in his or her blood. It is unclear why that was the case, however.

In January 2016, the FDA asked the company to suspend the trial, but after a yearlong investigation, the FDA lifted that clinical hold with the caveat that the company do a clinical pharmacokinetic study at a dose of 150 µg HT-100 (which the company is doing), and that all studies be conducted at that lower dose.

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