Vamorolone Trial for DMD Finishes Pivotal First Part, Results Awaited

Aisha I Abdullah PhD avatar

by Aisha I Abdullah PhD |

Share this article:

Share article via email
gene therapy LION-101

The final patient has finished the first, six-month portion of the pivotal VISION-DMD Phase 2b clinical trial, assessing the anti-inflammatory steroid therapy vamorolone in boys with Duchenne muscular dystrophy (DMD).

Top-line data from this part of the study are expected in the second half of this year, and could support an application to the U.S. Food and Drug Administration requesting vamorolone be approved to treat DMD in early 2022.

The trial is being conducted by ReveraGen Biopharma in collaboration with Santhera Pharmaceuticals, which holds an exclusive worldwide license for vamorolone, a potential alternative to existing corticosteroids.

“The use of glucocorticoids, despite having proven benefits in the treatment of DMD, is severely limited due to side effects and poor tolerability. Our expectation is that vamorolone will have the benefits but avoids many of the tolerability issues that limit the use of this standard of care,” Eric Hoffman, PhD, president and CEO of ReveraGen, said in a press release

The 48-week global clinical trial (NCT03439670) aims to evaluate the efficacy, safety, and tolerability of vamorolone compared with a placebo and the corticosteroid prednisone in 121 Duchenne boys, ages 4 to 6, and able to walk. Its primary outcome is evidence of treatment effectiveness, as seen through changes in motor function and strength using the Time to Stand test at week 24.

Patients in this first trial portion were randomly assigned to oral vamorolone at either 2.0 or 6.0 mg/kg per day, or to prednisone at a daily dose of 0.75 mg/kg, or to a matching placebo for 24 weeks (nearly six months). This part was doubled-blinded, meaning neither the patients nor the investigators know who received what treatment or placebo.

Its second half assigns all to treatment. Boys initially randomized to either prednisone or placebo will switch to vamorolone at 2.0 or 6.0 mg/kg/day for 24 weeks, while those originally given vamorolone will stay with the therapy at their assigned dose.

All will be monitored to assess the treatment’s long-term efficacy and safety, as well as its tolerability.

“Our thanks go out to the study participants, their families and healthcare professionals who, in the midst of the COVID-19 pandemic, are enabling us to advance this pivotal study as intended,” Hoffman said.

ReveraGen received a two-year grant worth $3.3 million from the National Institute of Neurological Disorders and Stroke in November, supporting the development of vamorolone an alternative to corticosteroids, the current standard for DMD. 

The company previously announced that, if the top-line results from the VISION-DMD were positive, it plans to also use the awarded funds to apply for vamorolone’s regulatory approval in the U.S. 

A Phase 2a trial (NCT02760264) and its extension study (NCT02760277) showed improved motor function with vamorolone in boys with DMD. A similar benefit was seen in a two-year extension study (NCT03038399), and without the stunted growth found in boys treated with conventional corticosteroids in the CINRG Duchenne Natural History Study (NCT00468832).

“Based on previously established data, we believe that vamorolone has the potential to become a foundational therapy in DMD for patients irrespective of the underlying gene mutation and a promising alternative to existing corticosteroids,” said Dario Eklund, CEO of Santhera.