Drug Profile

SKY 0515

Alternative Names: SKY-0515

Latest Information Update: 19 Sep 2025

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At a glance

Originator Skyhawk Therapeutics
Class Antiparkinsonians; Small molecules
Mechanism of Action Huntingtin protein inhibitors; PMS1 protein inhibitors; RNA splicing modulators

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Highest Development Phases

Phase II/III Huntington's disease

Most Recent Events

17 Sep 2025 Updated efficacy, pharmacokinetics and adverse events data from a phase I trial in Huntington’s disease released by Skyhawk Therapeutics
18 Jun 2025 Phase-II/III clinical trials in Huntington's disease in New Zealand and Australia (PO) (NCT06873334)
31 Mar 2025 Skyhawk Therapeutics completes enrollment in phase I clinical trials in Huntington's disease (PO)

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