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 2016, there were 46,080 incident cases of amyotrophic lateral sclerosis (ALS) in adults aged 40 years and older worldwide, and forecasts that number to increase to 54,750 incident cases by 2025.
• 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).
• Mitsubishi Tanabe’s Radicava, Concordia’s Rilutek, and Corestem’s Neuronata-R are the only marketed drugs available for ALS.
• Half of all industry-sponsored drugs in active clinical development for ALS are in Phase II. Therapies in mid-to-late-stage development for ALS focus on a wide variety of targets. The most popular method of administration for pipeline drugs in mid-to-late-stage development is the oral route.
• High impact upcoming events for drugs in the ALS space comprise topline Phase I/II, Phase II, and Phase III trial results, an expected CHMP opinion, and a trial announcement.
• The overall likelihood of approval of a Phase I ALS asset is 2.9%, and the average probability of success in Phase III is 20%. Drugs, on average, take 7.7 years from Phase I to approval, compared to 9.6 years in the overall neurology space.
• There have been 21 licensing and asset acquisition deals involving ALS drugs during 2013–18. The $1,050m exclusive strategic collaboration and option agreement signed in 2018 between AbbVie and Voyager to develop and commercialize vectorized antibodies directed against tau for the treatment of Alzheimer’s disease and other neurodegenerative diseases was the largest deal.
• 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 has 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, followed by Otsuka.