This Market Spotlight report covers the Fragile X Syndromemarket, comprising key pipeline drugs, clinical trials, upcoming and regulatory events, patent information, a discussion on disease epidemiology, and licensing and acquisition deals.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
Published: 27 May 2020
Number of pages:26
This Market Spotlight report covers the Fragile X Syndromemarket, 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.
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
6 KEY TAKEAWAYS
7 DISEASE BACKGROUND
10 PIPELINE DRUGS
14 RECENT EVENTS AND ANALYST OPINION
14 Campral for FXS (June 25, 2019)
16 KEY UPCOMING EVENTS
17 PROBABILITY OF SUCCESS
18 LICENSING AND ASSET ACQUISITION DEALS
19 PARENT PATENTS
20 CLINICAL TRIAL LANDSCAPE
21 Sponsors by status
22 Sponsors by phase
LIST OF FIGURES
10 Figure 1: Overview of pipeline drugs for FXS in the US
10 Figure 2: Pipeline drugs for FXS, by company
11 Figure 3: Pipeline drugs for FXS, by drug type
11 Figure 4: Pipeline drugs for FXS, by classification
16 Figure 5: Key upcoming events in FXS
17 Figure 6: Probability of success in the FXS pipeline
18 Figure 7: Licensing and asset acquisition deals in FXS, 2015–20
19 Figure 8: Parent patents in FXS
20 Figure 9: Clinical trials in FXS
20 Figure 10: Top 10 drugs for clinical trials in FXS
21 Figure 11: Top 10 companies for clinical trials in FXS
21 Figure 12: Trial locations in FXS
22 Figure 13: FXS trials status
23 Figure 14: FXS trials sponsors, by phase
LIST OF TABLES
12 Table 1: Pipeline drugs for FXS in the US
14 Table 2: Campral for FXS (June 25, 2019)
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