ReveraGen’s vamorolone improves muscle function in DMD trial

7th October 2019 (Last Updated December 23rd, 2019 12:07)

ReveraGen BioPharma has reported positive results from the ongoing VBP15-LTE clinical trial where vamorolone improved muscle function after 18 months of therapy in Duchenne muscular dystrophy (DMD) patients.

ReveraGen’s vamorolone improves muscle function in DMD trial
Histopathology of gastrocnemius muscle from a patient with pseudohypertrophic muscular dystrophy, Duchenne type. Credit: Dr. Edwin P. Ewing, Jr.

ReveraGen BioPharma has reported positive results from the ongoing VBP15-LTE clinical trial where vamorolone improved muscle function after 18 months of therapy in Duchenne muscular dystrophy (DMD) patients.

The results were drawn from 23 patients who received a 2mg/kg or 6mg/kg a day dose of the drug for a minimum of 18 months. The drug was compared to corticosteroids during the study.

In addition to motor function, the results showed improved tolerability with fewer corticosteroid-specific side effects.

Vamorolone is an investigational, steroidal anti-inflammatory drug being developed to treat DMD.

VBP15-LTE is an extension study of the VBP15-003 trial, which involved 48 DMD patients treated for six months with 0.25mg/kg to 6mg/kg a day dose and showed dose-related improvements in various gross motor outcomes.

In the extension study, the drug led to consistent and significant improvements in standardised motor function outcomes from baseline to month 18.

It was observed that the outcomes for patients treated with ReveraGen’s drug were consistently better compared with age-matched, steroid naïve participants of an external natural history study.

Motor function outcomes of vamorolone-treated DMD patients showed similar improvements when compared with age-matched patients treated with prednisone from an external control group.

Vamorolone also showed normal growth rates and lesser reports of weight gain and Cushingoid features than published prednisone and deflazacort studies.

ReveraGen CEO Eric Hoffman said: “These data demonstrate that vamorolone treatment results in persistently improved motor function in DMD patients, similar to that of corticosteroids.

“Importantly, however, vamorolone treatment over a period of 18 months showed better tolerability with less corticosteroid-specific side effects, including no stunting of the growth of DMD children.”

The drug is also being studied in the ongoing 48-week Phase IIb VISION-DMD trial in 120 boys who did not receive previous treatment with corticosteroids, compared with prednisone and placebo.