Denali Therapeutics recently reported in The New England Journal of Medicine that its investigational Hunter syndrome therapy, tividenofusp alfa, showed strong reductions in key disease biomarkers and encouraging neurological and physical outcomes in a Phase 1/2 trial, underpinning an ongoing FDA Priority Review with an accelerated approval decision expected by April 5, 2026.
The therapy’s ability to cross the blood–brain barrier using Denali’s TransportVehicle platform and address neurological manifestations of MPS II sets it apart from conventional enzyme replacement approaches being compared in the ongoing COMPASS Phase 2/3 study.
We will now examine how tividenofusp alfa’s blood–brain barrier-crossing design and Priority…
