Investigational New Drug (IND) application submitted to U.S. Food and Drug Administration (FDA) for ELX-02 for the treatment of Alport syndrome with nonsense mutations
Rebound in average UPCR 3-months post treatment provides further evidence that proteinuria remission in one out of three patients was drug related
All 3 Alport patients (100% response rate) treated with ELX-02 had biopsy confirmed disease regression, suggesting clinical benefit likely with longer treatment duration
Significant strategic interest in ZKN-013 following FDA approval to start Phase 1 single ascending dose (SAD) trial
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