Innovative DMD therapy reaches the key clinical trial endpoint and is expected to submit a new drug early next year...

Today, Santhera Pharmaceuticals and ReveraGen BioPharma jointly announced that the innovative therapy vamorolone has reached the primary endpoint and multiple key secondary endpoints in a pivotal Phase 2b clinical trial for the treatment of patients with Duchenne muscular dystrophy (DMD).

DMD is one of the most common childhood neuromuscular diseases.

Vamorolone is a potential "first-in-class" steroid drug originally developed by ReveraGen Biopharma.

▲The mechanism of action of Vamorolone (picture source: Santhera's official website)

In this phase 2b clinical trial, a total of 121 DMD patients aged 4-7 years were randomized to receive different doses of vamorolone, placebo or prednisone.

Vamorolone reached the primary endpoint of the trial.

▲Vamorolone significantly improves the speed of patients from supine to standing (picture source: reference [1])

In addition, compared with the placebo group, two different doses of vamorolone also significantly improved the patient's 6-minute walk test (6-MWT) indicators.

▲Vamorolone significantly improved the patient's 6-minute walk test indicators (picture source: reference [1])

In terms of safety, compared with prednisone, vamorolone caused fewer treatment-related adverse events.

▲Compared with prednisone, Vamorolone did not show the effect of delaying the growth of children (picture source: reference [1])

Note: The original text has been deleted

Reference materials:

[1] Presentation on VISION-DMD topline readout with vamorolone.

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