Novartis (NVS) said Thursday a phase I/II study of its experimental therapy, del-brax, met both primary and key secondary endpoints in patients with facioscapulohumeral muscular dystrophy.
The drugmaker said the drug reduced levels of KHDC1L, a biomarker linked to the disease's underlying cause, and lowered creatine kinase levels, a marker of muscle damage, compared with placebo.
Del-brax, designed to target DUX4, a protein that drives the condition was acquired through Novartis' purchase of Avidity Biosciences earlier this year, the company said.
Based on study, Novartis said it is enrolling about 200 patients in the phase 3 study to evaluate the drug's safety and effectiveness.
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