Santhera scores in Duchenne

Santhera has long claimed that, in vamorolone, it has a safer alternative to steroids for the treatment of Duchenne muscular dystrophy. This was borne out today by topline data from the pivotal Vision-DMD study, which compared vamorolone versus placebo and the steroid prednisone. The trial met its primary endpoint, time to stand, with vamorolone 6mg statistically superior to placebo at 24 weeks; a 2mg dose also hit on this measure. Also importantly, vamorolone was linked with fewer adverse events than prednisone, while the 6mg dose was similar on efficacy, Santhera said. Steroids are a mainstay of DMD therapy but can cause side effects including stunted growth and weight gain. Santhera plans to file vamorolone in the US in the first quarter of 2022. The Swiss group reckons vamorolone could bring in over $500m at peak in DMD, putting it among the top-selling therapies for the disorder, according to Evaluate Pharma. The company intends to market the drug alone in the US and EU; this could prove trickier than gaining approval, considering Santhera only has SFr26.4m ($29.4m) of available cash, including up to SFr18m that it can draw down from a Highbridge loan. The group’s stock shot up 50% this morning – still, it is only worth SFr46m.

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