This Market Spotlight report covers the Amyotrophic Lateral Sclerosis (ALS) market, comprising key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.

Key Takeaways

• Datamonitor Healthcare estimates that in 2017, there were 47,190 incident cases of amyotrophic lateral sclerosis (ALS) in adults aged 40 years and older worldwide, and forecasts that number to increase to 56,070 incident cases by 2026.
• Oceania and Europe are estimated to have the highest disease incidence (2.33 and 2.16 cases per 100,000 people, respectively), while Asia and Africa have the lowest incidence (0.62 and 0.89 cases per 100,000 people, respectively).
• Italfarmaco’s Tiglutik, Mitsubishi Tanabe’s Radicava, Advanz’s Rilutek, and Corestem’s Neuronata-R are the only marketed drugs available for ALS. These drugs are administered via the intravenous and oral routes.
• The greatest proportion of industry-sponsored drugs in active clinical development for ALS are in Phase II, with only one drug in the NDA/BLA phase.
• Therapies in mid-to-late-stage development for ALS focus on a wide variety of targets. The most popular method of administration is the oral route.
• High-impact upcoming events for drugs in the ALS space comprise topline Phase II and Phase III trial results for a number of drugs, an expected PDUFA date for BHV-0223, and an expected CHMP opinion for Radicava.
• The overall likelihood of approval of a Phase I ALS asset is 5.3%, and the average probability a drug advances from Phase III is 33.3%. Drugs, on average, take 7.9 years from Phase I to approval, compared to 9.6 years in the overall neurology space.
• There have been 24 licensing and asset acquisition deals involving ALS drugs during 2014–19. The $1,225m exclusive collaboration and license agreement signed in October 2018 between Denali and Sanofi to develop and commercialize products containing receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitors for neurological indications such as Alzheimer’s disease, ALS, and multiple sclerosis was the largest deal during the period.
• The distribution of clinical trials across Phase I–IV indicates that the majority of trials for ALS have been in the early and midphases of development, with 81% of trials in Phase I–II, and only 19% in Phase III–IV.
• The US has a substantial lead in the number of ALS clinical trials globally. The UK leads the major EU markets, while Japan has the top spot in Asia.
• Clinical trial activity in the ALS space is dominated by completed trials. Otsuka/Avanir have the highest number of completed clinical trials for ALS, with 11 trials.
• Biogen leads industry sponsors with the highest number of clinical trials for ALS.