Lead Product(s): Inzomelid
Therapeutic Area: Genetic Disease
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 26, 2020
The Phase I study demonstrated Inzomelid has an excellent safety, tolerability and pharmacokinetic profile in healthy subjects, with dose-dependent target engagement.