Huntington Disease

In honor of Huntington Disease Awareness Month, held annually in May, the director of the Huntington's Disease Center at Northwestern Medicine discussed the design of the INVEST-HD trial.

Interim results from the PIVOT-HD extension study suggested dose-dependent effects of votoplam on measures of disease progression in stage 2 Huntington disease compared with a natural history cohort.

Early-phase 1b trial data showed that ANX005 is generally safe and engages its complement target in Huntington disease.

Neurocrine Biosciences launches a phase 2 trial for NBI-1065890, targeting tardive dyskinesia with promising new treatment options.

Head-to-head PET imaging data indicated that valbenazine achieves significantly higher VMAT2 target occupancy than deutetrabenazine XR at therapeutic doses, with both treatments remaining well tolerated.

uniQure navigates FDA's unexpected shift on AMT-130, aiming for accelerated approval for Huntington's disease despite recent regulatory challenges.

Real-world data from Teva Pharmaceuticals’ IMPACT-TD Registry showed that deutetrabenazine and its extended-release formulation led to significant reductions of involuntary movements and improved quality of life for adults with tardive dyskinesia.

A new study presented at the National Society of Genetic Counselors Annual Conference highlights strong interest among individuals at risk for Huntington disease in undergoing modifier gene testing, underscoring the growing role of genetic insights in personal and clinical decision-making.

UniQure's AMT-130 shows promise in slowing Huntington disease progression, paving the way for a potential breakthrough treatment by 2026.

SKY-0515 shows promising results in reducing mutant huntingtin protein in Huntington disease patients, paving the way for transformative treatment options.

Skyhawk Therapeutics initiates FALCON-HD trial for SKY-0515, a promising treatment for Huntington disease, aiming to modify disease progression.

The phase 2 trial tests Roche’s investigational antisense oligonucleotide therapy tominersen in patients with early stages of Huntington disease, a disease with no FDA-approved therapies available.

Leveraging the Enroll-HD dataset, recent data revealed consistent links between antidopaminergic medication use and worsened cognitive and motor outcomes in Huntington disease.

New 24-month data further showed dose-dependent lowering of neurofilament light and trends on major end points such as cUHDRS, Total Function Capacity, and Symbol Digit Modalities Test.

Roche has updated its phase 2 GENERATION HD2 trial to continue testing only the higher 100 mg dose of tominersen, following interim data favoring its potential clinical benefit in Huntington disease.

A recent study reported that silmitasertib, an investigational drug currently in development for cancer, may help reduce motor deficits and neuropathology in a Huntington disease.

The U.S. HHS announced 15 drugs, including deutetrabenazine, will undergo Medicare Part D price negotiations to lower costs for seniors by 2027.

The FDA has agreed that a scale used in ongoing phase 1/2 clinical trials could potentially serve as an intermediate clinical end point for accelerated approval pathway for uniQure’s investigational gene therapy AMT-130.

The phase 2 DIMENSION study results showed that SAGE-71 was well-tolerated but did not meet its primary end point, leading to Sage Therapeutics’ decision to discontinue its development.

Over a 12-month period, patients showed sustained improvements in disease progression through valbenazine, regardless of concomitant antipsychotic treatment.

VO659 was considered safe and well tolerated, with treated patients showing no sustained increase in neurofilament light protein through day 85 of treatment.

The application is based on results of Prilenia Therapeutics’ phase 3 study that assessed the safety and efficacy of pridopidine in patients with Huntington disease.

Overall, the treatment resulted in dose-dependent trends of improvement on key clinical measures including Total Functional Capacity and Composite Unified Hungtinton's Disease Rating Scale.

AMT-130, uniQure’s investigational gene therapy, is the first therapeutic candidate to receive regenerative medicine advanced therapy designation for Huntington disease.

The original approval for chorea associated with Huntington disease was based on the phase 3 KINECT-HD study, an 128-patient cohort trial in which valbenazine met its primary end point.