Their lead drug failed both main goals in key study, but Orphazyme plans to pitch subgroup analyses to regulators
Orphazyme attempted on Wednesday to enthuse investors about the results of a Phase II/III study testing their lead drug, arimoclomol, in patients with a rare genetic disorder, underscoring some positive subgroup analyses — even though the experimental treatment missed its primary endpoints. The company is now preparing marketing filings and planning to engage with regulators to get arimoclomol on the market at the earliest.
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