Global Market Spotlight, 2019: Fragile X Syndrome - ResearchAndMarkets.com

DUBLIN--()--The "Market Spotlight: Fragile X Syndrome" report has been added to ResearchAndMarkets.com's offering.

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.

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 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

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

EPIDEMIOLOGY

PIPELINE DRUGS

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

PARENT PATENTS

CLINICAL TRIAL LANDSCAPE

  • Sponsors by status
  • Sponsors by phase

BIBLIOGRAPHY

APPENDIX

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)

For more information about this report visit https://www.researchandmarkets.com/r/wlezq0

Contacts

ResearchAndMarkets.com
Laura Wood, Senior Press Manager
press@researchandmarkets.com
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
Related Topics: Central Nervous System Drugs , Neurology

Contacts

ResearchAndMarkets.com
Laura Wood, Senior Press Manager
press@researchandmarkets.com
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
Related Topics: Central Nervous System Drugs , Neurology