SKY-0515 / Skyhawk Therap - LARVOL DELTA

Skyhawk Therapeutics Announces Final Fifteen-Month Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington's Disease Patients (Yahoo Finance) - "At Month 15, the primary timepoint, patients treated with SKY-0515 improved on the cUHDRS by an average of 0.94 points from where they started, while a comparison group of untreated patients from the Enroll-HD natural history database declined by an average of 0.65 points. The gap between the two groups was 1.59 points (95% CI: 1.09, 2.09; p<0.001)...SKY-0515 patients also did better than the comparison group on each of the four measures that make up the cUHDRS: daily function (Total Functional Capacity, +0.98 points), movement (Total Motor Score, −9.38 points, where a lower score is better), processing speed (Symbol Digit Modalities Test, +4.15 points) and reading speed (Stroop Word Reading Test, +4.18 points)...On average, SKY-0515-treated patients' cUHDRS scores held at or above their baseline level at every assessment through Month 15, while the external comparison group's average score declined from Month 6 onward."

P1/2 data • CNS Disorders • Huntington's Disease

The Company's Phase 1/2 program and global Phase 2/3 FALCON-HD 004-ANZ + 004-WW pivotal program for Huntington's disease now have enrolled over 200 patients across twenty trial sites in ten countries. (Yahoo Finance)

Enrollment status • CNS Disorders • Huntington's Disease

Skyhawk Therapeutics...announces it has received additional regulatory approvals to open its Phase 2/3 FALCON-HD (004-ANZ and 004-WW) pivotal trial, with an IND acceptance in the United States and CTA acceptances in each Canada and the United Kingdom. (PRNewswire) - "The FALCON-HD pivotal program (NCT06873334 and NCT07378644) is a randomized, double-blind, placebo-controlled, dose-ranging study evaluating the pharmacodynamics, efficacy and safety of SKY-0515....FALCON-HD 004-WW plans to enroll up to an additional 400 participants with Stage 2 and early Stage 3 HD across more than 40 sites worldwide and is actively treating patients at a number of these sites presently."

IND • Trial status • CNS Disorders • Huntington's Disease

Skyhawk Therapeutics Releases Twelve-Month cUHDRS Subcomponent Results from its Phase 1/2 Clinical Trial of SKY-0515 in Huntington's Disease (PRNewswire) - "The twelve-month data, shared at the European Academy of Neurology, shows favorable and consistent trends for participants on SKY-0515 across all cUHDRS subcomponents, including Total Functional Capacity (TFC), Total Motor Score (TMS), Symbol Digit Modalities Test (SDMT), and Stroop Word Reading Test (SWRT)...At twelve months, participants receiving SKY-0515 demonstrated favorable mean change from baseline TFC score of +0.07, compared with an expected worsening of -0.87 points from propensity score-weighted analyses which use Enroll-HD natural history datasets...At twelve months, participants receiving SKY-0515 demonstrated favorable mean change from baseline TMS score of -2.00, compared with an expected worsening of 2.21 points from propensity score-weighted analyses which use Enroll-HD natural history datasets....The company plans to take additional programs into clinical development by the end of 2027."

P1/2 data • Pipeline update • CNS Disorders • Huntington's Disease

Skyhawk Therapeutics Announces Twelve-Month Interim Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington's Disease (PRNewswire) - "Treatment with SKY-0515 resulted in dose-dependent reductions in mutant huntingtin (mHTT) protein in blood of up to 69% as well as reductions in PMS1 mRNA of up to 26%...At three, six, nine and twelve months, patients receiving SKY-0515 demonstrated positive mean changes from baseline Composite Unified Huntington's Disease Rating Scale (cUHDRS) scores ranging from +0.31 to +0.38. These findings compared favorably with an expected worsening of -0.92 points over twelve months from propensity score-weighted analyses which use Enroll-HD and TRACK-HD natural history datasets."

P1/2 data • CNS Disorders • Huntington's Disease

Investigation of SKY-0515 in Patients with Huntington’s Disease: Interim Results of a Phase 1 Study (EAN 2026) - "These findings support further clinical evaluation of SKY-0515 as a potential disease-modifying therapy for HD. A confirmatory Phase 2/3 study (FALCON-HD) is now enrolling."

Clinical • P1 data • CNS Disorders • Huntington's Disease • Movement Disorders • PMS1

The Company also announces that the Australia and New Zealand (004-ANZ) portion of its Phase 2/3 FALCON-HD pivotal study completed enrollment six months ahead of schedule with 144 patients enrolled, and that the worldwide (004-WW) Phase 2/3 FALCON-HD pivotal study has expanded to eight countries (PRNewswire) - "FALCON-HD 004-ANZ enrolled 144 participants with Stage 2 and early Stage 3 HD across sites in Australia and New Zealand, and enrollment is complete. FALCON-HD 004-WW plans to enroll 400 participants with Stage 2 and early Stage 3 HD across more than 40 sites worldwide and is actively recruiting."

Enrollment closed • Trial status • CNS Disorders • Huntington's Disease

Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease (clinicaltrials.gov) - P2/3 | N=120 | Active, not recruiting | Sponsor: Skyhawk Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Genetic Disorders • Huntington's Disease • Movement Disorders

An Extension of SKY-0515 in Participants With Huntington's Disease (clinicaltrials.gov) - P2/3 | N=500 | Enrolling by invitation | Sponsor: Skyhawk Therapeutics, Inc. | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • CNS Disorders • Huntington's Disease • Movement Disorders

An Extension of SKY-0515 in Participants With Huntington's Disease (clinicaltrials.gov) - P2/3 | N=500 | Not yet recruiting | Sponsor: Skyhawk Therapeutics, Inc.

New P2/3 trial • CNS Disorders • Huntington's Disease • Movement Disorders

Skyhawk Therapeutics Appoints Aaron Deves as Chief Commercial Officer to Lead Commercialization of SKY-0515 for Huntington's Disease (PRNewswire) - "Industry veteran brings more than 30 years of experience launching therapies for neurological diseases, including Huntington's disease, and will prepare Skyhawk for the launch of SKY-0515 as early as 2027....'Skyhawk may receive accelerated approval for SKY-0515 in Australia within the next twelve months, and in other major markets during 2027.'"

Approval • Launch • CNS Disorders • Huntington's Disease

Skyhawk Announces Australia's Therapeutic Goods Administration Has Determined SKY-0515 for Huntington's Disease Meets Eligibility Criteria for Registration via the Provisional Approval Pathway (PRNewswire) - "More than 115 patients are now on trial with SKY-0515...Patients receiving SKY-0515 in the Phase 1C trial of SKY-0515 demonstrate mean Composite Unified Huntington's Disease Rating Scale (cUHDRS) improvement from baseline. At nine months this improvement is +0.64 points in a pooled analysis, compared to expected worsening at nine months of cUHDRS in symptomatic patients of -0.73 points, based on propensity score weighting using Enroll-HD and TRACK-HD."

Non-US regulatory • P1 data • CNS Disorders • Huntington's Disease

Skyhawk also announces today that its SKY-0515 Phase 2/3 FALCON-HD trial, open at twelve sites in Australia and New Zealand, has expanded worldwide. Skyhawk has now dosed more than 90 patients with SKY-0515. (PRNewswire)

Trial status • CNS Disorders • Huntington's Disease

Skyhawk Therapeutics Announces Nine Month Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease (PRNewswire) - "Treatment with SKY-0515 results in dose-dependent reductions of mHTT protein in blood of 62% at the 9mg dose, and dose-dependent PMS1 mRNA reduction of 26%. PMS1 is a key driver of somatic CAG repeat expansion and HD pathology...At three, six and nine months, patients receiving SKY-0515 in the Part C patient cohort of the Phase 1 clinical trial of SKY-0515, demonstrate mean Composite Unified Huntington's Disease Rating Scale (cUHDRS) improvement from baseline....Skyhawk expects to put additional small molecule drugs to treat rare neurological diseases with no approved disease modifying therapies in the clinic by the end of 2027....The first patients were dosed in SKY-0515's Part C in January 2025. Enrollment in Phase 1C of the SKY-0515 trial is now complete."

First-in-human • New trial • P1 data • Trial status • CNS Disorders • Huntington's Disease

FALCON-HD: Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease (clinicaltrials.gov) - P2/3 | N=400 | Recruiting | Sponsor: Skyhawk Therapeutics, Inc.

New P2/3 trial • Genetic Disorders • Huntington's Disease • Movement Disorders

Huntington's disease clinical trials update: October 2025. (PubMed, J Huntingtons Dis) - "In this edition of the Huntington's Disease Clinical Trials Update, we expand on the launch of the phase II/III clinical trial of SKY-0515 from Skyhawk Therapeutics and the phase I/II clinical trial of SPK-10001 from Spark Therapeutics. Further updates include recent developments in Roche's tominersen programme within GENERATION HD2, progress with votoplam (PTC518) in PIVOT-HD by PTC Therapeutics and developments in the collaborative PTC Therapeutics/Novartis programme. We also report positive topline data from uniQure's phase I/II clinical trial of AMT-130 after 36 months of follow-up. We additionally discuss regulatory developments regarding pridopidine following the negative PROOF-HD study. Finally, we provide an updated listing of all registered and ongoing clinical trials in Huntington's disease."

Journal • Huntington's Disease • Movement Disorders

Skyhawk Therapeutics, Inc…announced positive results from the first interim analysis of the Part C patient cohort in its Phase 1 clinical trial of SKY-0515, an investigational treatment for Huntington's disease (HD). (PRNewswire) - "SKY-0515 achieves dose-dependent reductions of mutant huntingtin (mHTT) protein, with 62% lowering at Day 84 on the 9mg daily oral dose; Additional findings include dose-dependent reductions in PMS1 mRNA, excellent brain penetration, and a favorable safety profile....Part C enrollment is complete, and topline data from the blinded active treatment extension are expected in mid-2026."

P1 data • Huntington's Disease

A Phase I Randomised, Double Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effect of Food on the Bioavailability of SKY-0515 in Healthy Volunteers and Patients with Huntington’s Disease (ANZCTR) - P1 | N=50 | Completed | Sponsor: Skyhawk Therapeutics, Inc | Recruiting ➔ Completed

Trial completion • Huntington's Disease • Movement Disorders

Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease (clinicaltrials.gov) - P2/3 | N=120 | Recruiting | Sponsor: Skyhawk Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Huntington's Disease • Movement Disorders

An Open-Label, Crossover, Relative Bioavailability Study Comparing Tablet and Capsule Formulations of SKY-0515 in Healthy Volunteers (ANZCTR) - P1 | N=16 | Completed | Sponsor: Skyhawk Therapeutics, Inc. | Not yet recruiting ➔ Completed

Trial completion • Huntington's Disease • Movement Disorders

Skyhawk Therapeutics Announces First Patient Dosed in Phase 2/3 FALCON-HD Trial of SKY-0515 for Huntington's Disease (PRNewswire) - "Skyhawk Therapeutics, Inc...today announced that the first patient has been dosed in its Phase 2/3 FALCON-HD trial evaluating SKY-0515, an investigational oral RNA splicing modulator for the treatment of Huntington's disease (HD)....The trial includes multiple sites across Australia and New Zealand. The initial dosing took place at Flinders Medical Centre in Adelaide, Australia."

Trial status • Huntington's Disease

Skyhawk Therapeutics Presents at Huntington's Disease Youth Organization HDYO International Congress 2025 (Canada Newswire) - P=NA | N=NA | "Skyhawk Therapeutics...presents on its novel SKY-0515 small molecule RNA splicing modulator targeting Huntington's Disease to members of the Huntington's Disease Youth Organization (HDYO), at the biannual HYDO International Congress in Prague, Czech Republic...'We are excited to present on our Huntingtin-lowering clinical trials at the HDYO International Congress this month'....'with positive results including SKY-0515's average HTT mRNA reduction of 72% at a daily oral dose of 9mg in trials thus far, and the drug's predicted suppression of the PMS1 protein, SKY-0515, if approved, could make a meaningful difference in Huntington's patients' lives.'"

Clinical data • Huntington's Disease

Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants with Huntington's Disease (clinicaltrials.gov) - P2/3 | N=120 | Not yet recruiting | Sponsor: Skyhawk Therapeutics, Inc.

New P2/3 trial • Genetic Disorders • Huntington's Disease • Movement Disorders

An Open-Label, Crossover, Relative Bioavailability Study Comparing Tablet and Capsule Formulations of SKY-0515 in Healthy Volunteers (ANZCTR) - P1 | N=16 | Not yet recruiting | Sponsor: Skyhawk Therapeutics, Inc.

New P1 trial • Huntington's Disease • Movement Disorders

Skyhawk Therapeutics Receives Australian Regulatory Approval to Extend SKY-0515 Treatment Duration in Phase 1 Huntington's Disease Patient Trial (PRNewswire) - P1 | N=50 | ACTRN12624000602527 | "Skyhawk Therapeutics, Inc...today announced that the Australian Human Research Ethics Committees (HREC) have approved SKY-0515 treatment for up to 12 weeks in the Company's ongoing Phase 1 trial in patients with Huntington's Disease (HD). HREC also agreed to the inclusion of Volumetric MRI (vMRI) measurements as an early exploratory endpoint in the trial. vMRI measurement of the brain's regions affected in HD can provide additional data on SKY-0515's effect in HD patients....Topline pharmacodynamic results for Parts A (single-ascending dose) and B (multiple-ascending dose), which evaluated SKY-0515 in healthy volunteers, demonstrated a dose-dependent reduction of HTT mRNA and reached an average reduction of 72% at a daily oral dose of 9mg. SKY-0515 was generally well tolerated at all doses tested."

P1 data • Regulatory • CNS Disorders • Genetic Disorders • Huntington's Disease