Inozyme Targets Underserved Mineralization Disorders Market With First-In-Class Enzyme Therapy
Emerging Company Profile: Fresh from a $73m follow-on offering last year, the US biotech is readying its lead asset for a pivotal trial in ultra-rare but debilitating mineralization disorders that affect infants and children.
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Addressing The Rare Disease Challenge Via Patient Engagement
Following the FDA’s release of draft guidance to enhance patient voice, In Vivo spoke with Ipsen’s rare disease regulation expert on creating a more patient-centric R&D and approval pathway, from modified endpoints to trial decentralization and beyond.
Part One Disappointment For BioXcel’s Igalmi In Pivotal Agitation Trial
The US firm’s drug for schizophrenia- and bipolar-related agitation Igalmi has failed part one of a pivotal trial in at-home patients, but the option for higher dosing in part two combined with placebo-like safety could offer hope.
Mirati’s Pivotal NSCLC Disappointment For Sitravatinib Combo ‘Unsurprising’
The US firm’s kinase inhibitor fell short of overall survival expectations in combination with a PD-1 inhibitor in an advanced lung cancer trial, but upcoming milestones for Krazati may be more of a concern.