This Market Spotlight report covers the Huntington’s Disease market, comprising key marketed and pipeline drugs, clinical trials, upcoming events, recent events and analyst opinion, probability of success, licensing and asset acquisition deals, and a 10-year disease prevalence forecast, as well as presenting drug-specific revenue forecasts.

Key Takeaways

• Datamonitor Healthcare estimates that in 2021, there were 79,600 prevalent cases of Huntington’s disease (HD) in adults aged 30 years and older worldwide, and forecasts that number to increase to 87,700 prevalent cases by 2028.
• Northern America is estimated to have the highest disease prevalence (0.0073%), while Asia has the lowest prevalence (0.0004%).
• Teva’s Austedo and Bausch Health’s Xenazine, which target vesicular monamine transporters, are the only marketed drugs for HD. These drugs are administered via the oral route.
• The largest proportion of industry-sponsored drugs in active clinical development for HD are in Phase II. Therapies in active clinical development for HD focus on targets such as huntingtin, vesicular monamine transporters, p38 MAP kinase, SIRT1, vasopressin receptors, dopamine 2 receptor, sigma-1 receptor, immune system, semaphorin 4D/CD100, NMDA glutamate receptor, PPAR delta, PPAR gamma, complement component 1q, complement pathway, and survival of motor neuron protein. The majority of the pipeline drugs are administered via the oral route, with the remainder being intrathecal, intravenous, and intracerebral/cerebroventricular formulations.
• High-impact upcoming events for drugs in the HD space comprise topline Phase I/II trial results for AMT-130 and topline Phase III trial results for TV-7820.
• The overall likelihood of approval of a Phase I neurodegenerative asset is 7.6%, and the average probability a drug advances from Phase III is 49.5%. Drugs, on average, take 11.9 years from Phase I to approval, compared to 10.2 years in the overall neurology space.
• The distribution of clinical trials across Phase I–IV indicates that the majority of trials for HD have been in the early and midphases of development, with 76% of trials in Phase I–II, and only 24% in Phase III–IV.
• The US has a substantial lead in the number of HD clinical trials globally, while the UK leads the major European markets. Clinical trial activity in the HD space is dominated by completed trials. Pfizer has the highest number of completed clinical trials for HD, with 14 trials.
• Pfizer leads industry sponsors with the highest overall number of clinical trials for HD.