This Market Spotlight report covers the Fragile X Syndrome market, comprising key pipeline drugs, clinical trials, upcoming events, patent information, recent events and analyst opinion, a discussion on disease epidemiology, and licensing and asset acquisition deals.
- 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 the industry-sponsored drugs in active clinical development for FXS are in Phase II, with only one drug in Phase III.
- Therapies in mid- and late-stage development focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, insulin-like growth factor-1 receptor, and GABA-A receptor. The majority of the pipeline drugs are administered via the oral and intravenous routes, while one drug is available in a topical formulation.
- High-impact upcoming events in the FXS space comprise topline Phase III trial results for ZYN-002, and topline Phase II trial results for OV101.
- The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 4.8%, and the average probability a drug advances from Phase III is 28.6%. Drugs, on average, take 8.3 years from Phase I to approval, compared to 9.6 years in the overall neurology space.
- There have been five licensing and asset acquisition deals involving FXS drugs during 2014-19. The exclusive licensing agreement in 2017 between Boehringer Ingelheim and Autifony Therapeutics, worth $737.5m, for a collaboration on a novel therapeutic approach to a range of CNS disorders, was the largest deal during the period.
- 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.
Key Topics Covered
RECENT EVENTS AND ANALYST OPINION
- ZYN-002 for FXS (September 28, 2017)
KEY UPCOMING EVENTS
PROBABILITY OF SUCCESS
LICENSING AND ASSET ACQUISITION DEALS
- Shionogi Picks Up Asia Rights To Tetra’s Cognitive Candidate
CLINICAL TRIAL LANDSCAPE
- Sponsors by status
- Sponsors by phase
LIST OF FIGURES
Figure 1: Overview of pipeline drugs for FXS in the US
Figure 2: Pipeline drugs for FXS, by company
Figure 3: Pipeline drugs for FXS, by drug type
Figure 4: Pipeline drugs for FXS, by classifications
Figure 5: ZYN-002 for FXS (September 28, 2017): Phase II – FAB-C
Figure 6: Key upcoming events in FXS
Figure 7: Probability of success in the FXS pipeline
Figure 8: Licensing and asset acquisition deals in FXS, 2014-19
Figure 9: Parent patents in FXS
Figure 10: Clinical trials in FXS
Figure 11: Top 10 drugs for clinical trials in FXS
Figure 12: Top 10 companies for clinical trials in FXS
Figure 13: Trial locations in FXS
Figure 14: FXS trials status
Figure 15: FXS trials sponsors, by phase
LIST OF TABLES
Table 1: Pipeline drugs for FXS in the US
Table 2: ZYN-002 for FXS (September 28, 2017)
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