Clinical trial data is extremely helpful in understanding the safety, efficacy, and tolerability of various potential treatments. According to Muscular Dystrophy News, top-line data from the Phase 2b VISION-DMD clinical trial highlights how vamorolone was beneficial in pediatric patients with Duchenne muscular dystrophy (DMD). Altogether, the investigational treatment showed efficacy in both its primary and secondary endpoints.

Vamorolone

Currently, vamorolone is being developed in conjunction between Santhera Pharmaceuticals (“Santhera”) and ReveraGen BioPharma (“ReveraGen”). According to Santhera, vamorolone is:

a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore could emerge as a valuable alternative to corticosteroids, the current standard of care in children and adolescent patients with DMD.

Thus far, vamorolone has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations within the United States; and Promising Innovative Medicine (PIM) and Orphan Drug status within Europe. Moving forward, the partnership also looks forward to requesting Priority Review in the United States in 2022.

Duchenne Muscular Dystrophy (DMD)

Altogether, vamorolone is being evaluated as a potential therapeutic option for patients with Duchenne muscular dystrophy (DMD). DMD is one of nine forms of muscular dystrophy and is characterized by progressive muscle weakness caused by dystrophin deficiency. Patients with DMD have DMD gene mutations preventing them from creating dystrophin, which is needed for muscle strength, protection, and function. In most cases, DMD symptoms appear before a child is 6 years old. By teenage years, many patients with DMD must utilize mobility assistance. Beyond muscle weakness, DMD symptoms can include intellectual and cognitive delays, enlarged calf muscles, delayed motor development, and cardiomyopathy. Because of the way DMD is inherited, it mostly affects males. Females with DMD are rare.

Evaluating Vamorolone in VISION-DMD

During the 48-week VISION-DMD clinical trial, researchers evaluated the safety, efficacy, and tolerability of 2mg/kg and 6mg/kg vamorolone. The orally administered liquid therapy was compared to prednisone and a placebo. Altogether, 121 patients enrolled. Patients in the VISION-DMD trial were able to walk and were ages 4-6.

Top-line data from the VISION-DMD Phase 2b clinical trial evaluating vamorolone in boys with Duchenne muscular dystrophy (DMD), showed efficacy across primary and secondary study goals. The investigational therapy was also well-tolerated with a favorable safety profile. Overall, top-line data from the first 24 weeks (6 months) showed that:

• In terms of efficacy, 6mg/kg vamorolone showed similar efficacy to prednisone.
• Patients receiving 6mg/kg daily showed an improvement in Time-to-Stand (TTSTAND) velocity, from 6.0 seconds at baseline to 4.6 seconds with treatment. In patients who received 2mg/kg vamorolone, researchers also saw a significant improvement in TTSTAND velocity.
  • Ultimately, this highlights an improvement in muscle function allowing patients to stand faster.
  • Alternately, patients receiving a placebo saw a worse TTSTAND score.
• Both vamorolone doses improved walking distance, while 6mg/kg improved the time it took to walk or run 10 meters.
• Altogether, vamorolone was relatively safe and well-tolerated. No severe adverse reactions occurred during the trial. While some milder adverse reactions did occur, there were nearly 30~ more adverse reactions reported in patients treated with prednisone.
• Unlike the current standard-of-care (corticosteroids), vamorolone did not stunt patient growth.
• Researchers believe vamorolone treatment could inhibit DMD progression by approximately 2 years.

This top-line data is promising. Moving forward, researchers will continue the VISION-DMD clinical trial for the remaining 24 weeks. Additional data should be available before the end of 2021.