SKY-0515

SKY-0515 is investigational and has no established place in therapy. Its studied use is as a once-daily oral therapy intended to slow disease progression, added to symptomatic care, in adults with symptomatic early disease: the phase 1/2 trial enrolled HD-ISS stage 1, stage 2, or mild stage 3 participants, and the FALCON-HD phase 2/3 trials enroll participants with stage 2 and early stage 3 disease, with registry eligibility thresholds of TFC 10 or higher and TMS 6 or higher. FALCON-HD 004-ANZ enrolled 144 participants in Australia and New Zealand; planned enrollment in FALCON-HD 004-WW was stated as up to an additional 400 participants in a July 2026 announcement and as approximately 600 participants in the September 2026 fifteen-month announcement. In HD-ISS terms, stage 2 denotes a measurable clinical phenotype and stage 3 denotes functional decline; clinical motor diagnosis corresponds to the latter part of stage 2. If approved, SKY-0515 would be used alongside, not in place of, symptomatic VMAT2 inhibitors, antipsychotics, antidepressants, and rehabilitation. Because administration is oral, SKY-0515 would not require the intrathecal injections used for tominersen or the MRI-guided neurosurgical procedure used for AMT-130. Specialist HD care in the United States is concentrated in the HDSA Centers of Excellence network (60 centers and 9 partner sites covering 37 states plus the District of Columbia), and the multidisciplinary care described in guidelines would continue regardless of disease-modifying treatment.