This Market Spotlight report covers the Fragile X Syndrome market, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, upcoming events, patent information, probability of success, a discussion on disease epidemiology, and licensing and asset acquisition deals.

Key Takeaways

• Based on cohorts of children with special educational needs, prevalence of fragile X syndrome (FXS) is estimated to be 1 in 4,000 in males, and 1 in 8,000 in females.
• The majority of industry-sponsored drugs in active clinical development for FXS are in Phase II, with only one drug in Phase III. Therapies in active clinical development for FXS focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, GABA-A receptor, insulin-like growth factor-1 receptor, and potassium channels. The majority of these pipeline drugs are administered via the oral route.
• High-impact upcoming events for drugs in the FXS space comprise topline Phase III trial results for Zygel, and topline Phase II trial results for OV101 and BPN14770.
• The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 4%, and the average probability a drug advances from Phase III is 28.6%. Drugs, on average, take 8.4 years from Phase I to approval, compared to 9.8 years in the overall neurology space.
• There have been five licensing and asset acquisition deals involving FXS drugs during 2015–20. The $737.5m exclusive licensing agreement made in 2017 between Boehringer Ingelheim and Autifony Therapeutics for a collaboration on a novel therapeutic approach to a range of CNS disorders was the largest deal.
• All clinical trials for FXS have been in Phase II, with no Phase III trials to date.
• The US leads in terms of the number of FXS clinical trials globally