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Market Spotlight: Fragile X Syndrome

  • ID: 4775087
  • Report
  • June 2021
  • Region: Global
  • 30 Pages
  • Pharma Intelligence
Fragile X syndrome (FXS) is a genetic disorder leading to various developmental problems ranging from learning disabilities to cognitive impairment. FXS is caused by mutation of the fragile X mental retardation 1 (FMR1) gene, which usually makes a protein known as fragile X mental retardation protein (FMRP). FMRP is required for normal brain development, and individuals with FXS do not make this protein. Individuals who have other fragile X-associated disorders (FXTAS) have mutations in their FMR1 gene, but they generally make some FMRP.

Key Takeaways

  • Based on cohorts of children with special educational needs, the 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 two drugs 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, GABA-B receptor, 11-beta-hydroxylase, 11 betahydroxysteroid dehydrogenase type 1, and potassium channels. The majority of these pipeline drugs are administered via the oral route.
  • The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 2.7%, and the average probability a drug advances from Phase III is 20%. Drugs, on average, take 12.4 years from Phase I to approval, compared to 10.0 years in the overall neurology space.
  • The distribution of clinical trials across Phase I-IV indicates that the majority of trials for FXS have been in the early and midphases of development, with 77% of trials in Phase I-II, and only 23% in Phase III-IV.
  • The US has a substantial lead in the number of FXS clinical trials globally. France and Spain lead the major European markets, while Israel has the top spot in Asia.
  • Clinical trial activity in the FXS space is dominated by completed trials. Seaside Therapeutics and Novartis have the highest number of completed clinical trials for FXS, with four trials each.
  • Seaside Therapeutics leads industry sponsors with the highest overall number of clinical trials for FXS, followed by Novartis.

Table of Contents

OVERVIEWKEY TAKEAWAYSDISEASE BACKGROUNDTREATMENT
EPIDEMIOLOGY
  • Premutation

PIPELINE DRUGS
RECENT EVENTS AND ANALYST OPINION
  • BPN14770 for FXS (November 2, 2020)
  • Zygel for FXS (October 15, 2020)
  • Zygel for FXS (June 30, 2020)
  • OV101 for FXS (May 7, 2020)

PROBABILITY OF SUCCESS
LICENSING AND ASSET ACQUISITION DEALS
  • Shionogi To Add CNS Critical Mass With Tetra Buyout

CLINICAL TRIAL LANDSCAPE
  • Sponsors by status
  • Sponsors by phase

BIBLIOGRAPHYAPPENDIX
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 classification
Figure 5: BPN14770 for FXS (November 2, 2020): Phase II - Adults
Figure 6: Zygel for FXS (October 15, 2020): Phase III - CONNECT-FX (US, AUS, NZ)
Figure 7: Zygel for FXS (June 30, 2020): Phase III - CONNECT-FX (US, AUS, NZ)
Figure 8: OV101 for FXS (May 7, 2020): Phase II - ROCKET
Figure 9: Probability of success in the neurodevelopmental disorders pipeline
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: BPN14770 for FXS (November 2, 2020)
Table 3: Zygel for FXS (October 15, 2020)
Table 4: Zygel for FXS (June 30, 2020)
Table 5: OV101 for FXS (May 7, 2020)