According to results of a recent survey, more than 80% of U.S.-based neurologists agreed that a major unmet need in confronting Huntington’s disease is halting neurodegeneration prior to the onset of symptoms.
Furthermore, an equal number of professionals in the specialty expressed frustration with the absence of meaningful treatments to slow cognitive decline.
Spherix Global Insights conducted a market landscape study to gather insights from neurologists responsible for treatment of the condition. Due to a broad range of symptoms affecting motor function, cognitive abilities and emotional regulation, HD could be called the “perfect storm” of neurodegenerative diseases, Spherix said in a release.
Without full awareness of the severity of their disease progression and with little insight into the nature of their symptoms, patients often lose motor control and experience psychiatric symptoms and cognitive decline.
Neurologists answered questions regarding 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. Without further prompting, neurologists who responded to the poll also mentioned Annexon Biosciences’ ANX005, Azevan Pharmaceuticals’ SRX246, SOM Biotech’s SOM3355 and Vaccinex Inc.’s pepinemab as potential treatments.
Currently, according to the Spherix release, the only treatments for HD are “symptomatics,” which solely address abnormal movements and a limited number of psychiatric symptoms, but do not slow disease progression. To date, generic tetrabenazine has been the go-to in addressing abnormal movements and, fairly recently, Teva’s Austedo (deutetrabenazine) was approved as a treatment for movement disorders. Pharmaceutical options specifically to target HD symptoms have thus far been limited to antidepressants and antipsychotics.
Further results from the survey revealed that respondents possess little working knowledge of current assets in the pipeline, with fewer than 5% of those polled able to name a specific HD therapy currently in development. Conversely, 20% of neurologists included in the study were aware that gene therapy is being studied for HD and 66% expressed particular interest in bringing a gene therapy to market.
Source: Healio: https://www.healio.com/news/neurology/20240105/survey-halting-neurodegeneration-chief-concern-in-huntingtons-disease
