Report on Patient-Reported Outcomes of INGREZZA® in Huntington’s Disease Chorea Treatment
Introduction
Neurocrine Biosciences, Inc. announced significant findings from patient-reported post-hoc analyses of the Phase 3 KINECT®-HD study. The study demonstrated that once-daily INGREZZA® (valbenazine) capsules significantly reduce both cognitive and motor-related disease burdens in adults with Huntington’s disease (HD) chorea. These results align with Sustainable Development Goal (SDG) 3: Good Health and Well-being, by advancing treatments for neurological disorders and improving patient quality of life.
Key Findings
- INGREZZA® showed greater reduction in cognitive and motor-related disease burden compared to placebo.
- The KINECT®-HD study is the first clinical trial to demonstrate measurable changes in cognitive and motor disease burden using a vesicular monoamine transporter 2 (VMAT2) inhibitor in HD chorea patients.
- Patient-reported outcomes were assessed using the Huntington’s Disease Health Index (HD-HI), a tool developed with patients and caregivers to evaluate disease burden across cognitive, motor, and gastrointestinal domains.
Study Design and Methodology
- Phase 3, randomized, double-blind, placebo-controlled clinical trial involving 128 adults aged 18 to 75 with motor-manifest HD and chorea symptoms.
- Primary efficacy endpoint: Unified Huntington’s Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score.
- Post-hoc analyses evaluated mean changes from baseline to maintenance (average of Week 10 and Week 12) in HD-HI domains:
- Cognition (18 items)
- Mobility/Ambulation (14 items)
- Abnormal Movements (7 items)
- Hand/Arm Function (9 items)
- Gastrointestinal Health (3 items)
- Participants with baseline scores ≥2 on items were considered “affected” and included in analyses.
Results
At maintenance, INGREZZA® demonstrated numerically larger reductions compared to placebo across all 18 cognition-related items and 32 of 33 motor-related items. Statistically significant improvements (p<0.05) were observed in the following key areas:
| HD-HI Item | INGREZZA® Mean Change (n) | Placebo Mean Change (n) |
|---|---|---|
| Cognition-Related | ||
| Memory loss | -0.9 (n=32) | -0.2 (n=30) |
| Reduced decision-making abilities | -1.0 (n=27) | -0.3 (n=26) |
| Trouble finding the right word | -0.9 (n=33) | -0.3 (n=32) |
| Motor-Related | ||
| Limitations with mobility/walking | -0.9 (n=37) | -0.2 (n=27) |
| Abnormal movements | -1.5 (n=33) | -0.8 (n=30) |
| Impaired coordination | -1.0 (n=37) | -0.4 (n=34) |
| Hard time sitting still | -1.6 (n=35) | -0.9 (n=33) |
| Problems knocking things over | -1.0 (n=34) | -0.4 (n=33) |
| Problems swallowing | -1.0 (n=20) | -0.3 (n=22) |
Additional Presentations at the 2025 ATMRD Congress
- Estimation of Minimal Clinically Important Difference (MCID) and longitudinal change in the Tardive Dyskinesia Impact Scale (TDIS), a patient-reported outcome measure.
- Topline results from the Phase 4 KINECT-PRO™ study demonstrating valbenazine’s impact on tardive dyskinesia symptoms.
- Post-hoc analysis of effect sizes of once-daily valbenazine for tardive dyskinesia and HD chorea.
- Sustained chorea improvements with long-term valbenazine treatment in adults with HD.
About Huntington’s Disease and Chorea
Huntington’s disease is a hereditary progressive neurodegenerative disorder causing motor, cognitive, and psychiatric symptoms, typically manifesting between ages 30 and 50. Chorea, characterized by irregular involuntary movements, affects motor coordination, gait, swallowing, and speech. Approximately 41,000 adults in the U.S. are affected, with over 200,000 at risk. Addressing HD chorea supports SDG 3 by improving health outcomes for affected populations.
About INGREZZA® (valbenazine) Capsules
- Selective VMAT2 inhibitor approved by the U.S. FDA for treating tardive dyskinesia and chorea associated with HD.
- Unique therapeutic dose from day one with no titration required.
- Available in capsule and sprinkle formulations to accommodate patients with swallowing difficulties.
- Works by selectively inhibiting dopamine release to reduce uncontrollable movements.
Safety Information
INGREZZA® may cause serious side effects including depression, suicidal thoughts, allergic reactions, somnolence, heart rhythm problems, neuroleptic malignant syndrome, and Parkinson-like symptoms. Patients should inform healthcare providers of all medical conditions and medications. Common side effects include sleepiness, tiredness, rash, and insomnia. Detailed prescribing information and medication guides are available for healthcare providers and patients.
Company Commitment and Alignment with Sustainable Development Goals
Neurocrine Biosciences is dedicated to relieving suffering through innovative neuroscience-focused biopharmaceuticals. Their work addresses SDG 3 by developing treatments for under-addressed neurological disorders, improving patient health and well-being globally. The company’s commitment to brave science and patient-centered research fosters sustainable health advancements.
References and Further Information
- Complete KINECT-HD study results published in The Lancet Neurology.
- Additional clinical trial information available at HuntingtonStudyGroup.org.
- Full prescribing information and medication guides accessible via Neurocrine Biosciences’ official website.
1. Sustainable Development Goals (SDGs) Addressed or Connected
- SDG 3: Good Health and Well-being
- The article focuses on improving health outcomes for patients with Huntington’s disease and tardive dyskinesia through the treatment with INGREZZA (valbenazine).
- It addresses reducing disease burden related to cognitive and motor impairments.
- SDG 9: Industry, Innovation and Infrastructure
- The development and clinical trials of INGREZZA represent innovation in biopharmaceuticals and medical treatments for neurological disorders.
- SDG 10: Reduced Inequalities
- The article mentions treatment options for a rare and under-addressed neurological disorder, contributing to reducing health inequalities.
2. Specific Targets Under the Identified SDGs
- Under SDG 3: Good Health and Well-being
- Target 3.4: By 2030, reduce by one third premature mortality from non-communicable diseases through prevention and treatment and promote mental health and well-being.
- Target 3.8: Achieve universal health coverage, including access to quality essential health-care services and access to safe, effective, quality, and affordable essential medicines and vaccines for all.
- Under SDG 9: Industry, Innovation and Infrastructure
- Target 9.5: Enhance scientific research, upgrade the technological capabilities of industrial sectors, including encouraging innovation and increasing the number of research and development workers.
- Under SDG 10: Reduced Inequalities
- Target 10.2: Empower and promote the social, economic and political inclusion of all, irrespective of age, sex, disability, race, ethnicity, origin, religion or economic or other status.
3. Indicators Mentioned or Implied to Measure Progress
- Unified Huntington’s Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score
- Used as a primary efficacy endpoint to measure severity of chorea symptoms in patients.
- Indicator of motor function improvement.
- Huntington’s Disease Health Index (HD-HI)
- Measures disease burden across cognitive, motor, and gastrointestinal domains.
- Used to assess changes in cognitive and motor-related challenges.
- Clinical Global Impression of Change (CGI-C) and Patient Global Impression of Change (PGI-C)
- Clinician-rated and patient-rated assessments of overall change from baseline.
- Used to evaluate treatment effectiveness.
- Tardive Dyskinesia Impact Scale (TDIS)
- Validated patient-reported outcome measure for tardive dyskinesia impact.
- Used to estimate minimal clinically important difference and longitudinal change.
4. Table of SDGs, Targets, and Indicators
| SDGs | Targets | Indicators |
|---|---|---|
| SDG 3: Good Health and Well-being |
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| SDG 9: Industry, Innovation and Infrastructure |
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| SDG 10: Reduced Inequalities |
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Source: prnewswire.com
