San­ther­a's Duchenne ef­fort gets a boost on pos­i­tive PhI­Ia for al­ter­na­tive steroid va­morolone — though there's a lot left to prove

Months af­ter San­thera got its hands on a sec­ond Duchenne mus­cu­lar dy­s­tro­phy drug, the Swiss com­pa­ny is get­ting an ear­ly look at how it im­proves mus­cle func­tion. But se­ri­ous ques­tions re­main as it takes on a field stuffed with en­trenched ri­vals and con­tro­ver­sy.

Rever­a­Gen was in charge of the Phase IIa ex­ten­sion, which com­pared dif­fer­ent dos­es of va­morolone with nat­ur­al his­to­ry con­trols. With 12 boys (aged 4 to 7) in each of four treat­ment groups, in­ves­ti­ga­tors test­ed 0.25, 0.75, 2.0 and 6.0 mg/kg/day in the study and pub­lished the re­sults in Neu­rol­o­gy.

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