According to Spherix Global Insights’ first market landscape study on Huntington’s disease, neurologists are staking their hopes on gene therapy.
EXTON, Pa., January 3, 2024 – Huntington’s disease (HD) could be called the perfect storm of neurodegenerative diseases. It causes a loss of critical striatal neurons which affect not only motor function – causing symptoms like the hallmark chorea (rapid, jerky involuntary body movements) – but also cognitive and emotional function. Thus, often without full awareness, patients lose motor control, experience psychiatric symptoms, and cognitive decline. A neurologist’s perspective from a recent research study suggests:
“A Huntington’s patient is almost a combination of Alzheimer’s, tardive dyskinesia, Parkinson’s – all in one. Basically, every symptom has an unmet need, save for perhaps- CHOREA.” (General Neurologist)
The only treatments for HD are so-called “symptomatics” – they address abnormal movements and some psychiatric symptoms, but do not alter the worsening of the disease or the myriad other symptoms. Generic tetrabenazine has been used for years to help with abnormal movements and fairly recently, Teva’s Austedo (deutetrabenazine) was approved as another movement disorder alternative. Beyond that, the only other pharmaceutical interventions used for HD are antipsychotic and antidepressant therapies for emotional and behavioral symptoms.
Recognizing the significant treatment unmet needs, Spherix Global Insights conducted a market landscape study, seeking insights from neurologists managing HD. The study revealed that over 80% of neurologists agreed that a top unmet need in HD is to stop neurodegeneration prior to the onset of symptoms. Furthermore, an equal number of neurologists also expressed frustration with the absence of meaningful treatments to slow cognitive decline, a critical concern given patients’ delayed seeking of treatment due to a lack of insight into their symptoms.
“Usually, chorea in Huntington’s, isn’t something that patients always notice and certainly as a result isn’t always something that bothers them. Its family who says you’re moving all the time and it’s a problem and the patient isn’t actually disabled by it.” (Movement Disorder’s Specialist)
Study results show that practicing neurologists have little to no knowledge of the assets in clinical development – less than one in twenty neurologists can name a specific HD therapy in development. However, one-fifth are aware that gene therapy, conceptually, is one of the potential solutions being studied for HD and two-thirds are very interested in bringing a gene therapy to market.
“Looking at the pathology, we know there’s a genetic mutation. The repeats, we know the accumulation, the huntingtin protein. If there’s a way that pathology in the brain could be halted or stopped or altered genetically where you would have lesser repeats, you wouldn’t have the atrophy we talked about and the progression in the caudate nucleus in the basal ganglia.” (Movement Disorder Specialist)
Neurologists answered questions about their familiarity, interest, and degree of advance over current treatments for five pipeline agents: Hoffman-La Roche/Genentech’s tominersen, PTC Therapeutics PTC518, Sage Therapeutics SAGE-718, UniQure Biopharma’s AMT-130, and Wave Life Sciences’ WVE-003. Additionally and unaided, neurologists also mentioned Annexon Biosciences’ ANX005, Azevan Pharmaceuticals’ SRX246, SOM Biotech’s SOM3355, and Vaccinex, Inc.’s pepinemab.
Research from Market Dynamix™: Huntington’s Disease (US) affirms neurologists’ optimism (perhaps buoyed by the dearth of therapies today) about effective disease-modifying treatments coming from biopharmaceutical pipelines within the next five years.
About Market Dynamix™
Market Dynamix™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years, highlighting market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents.
About Spherix Global Insights
Spherix Global Insights is a leading provider of market research, business intelligence and advisory services to the global life sciences industry. The company’s unique service offerings are powered by deep therapeutic knowledge, the Spherix Network specialty physician panel, and commercially relevant analyses to enable strategic decision-making by our valued customers.
A trusted advisor and industry thought leader, Spherix Global Insights provides specialized market expertise in six (6) focused therapeutic areas including: dermatology, gastroenterology, nephrology, neurology, rheumatology, and ophthalmology.
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Spherix Global Insights Contact
Blaine Cloud, Ph.D., Neurology Franchise Head,
blaine.cloud@spherixglobalinsights.com
NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement by any company, brand, or product aforementioned.
