Market Spotlight: Fragile X Syndrome
Market Spotlight: Fragile X Syndrome
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This Market Spotlight report covers the Fragile X Syndrome market, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, upcoming events, probability of success, a discussion on disease epidemiology, and licensing and asset acquisition deals.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- Description
- Contents
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, 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 3%, and the average probability a drug advances from Phase III is 22.2%. Drugs, on average, take 8.2 years from Phase I to approval, compared to 9.8 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.
CONTENTS
5 OVERVIEW
6 KEY TAKEAWAYS
7 DISEASE BACKGROUND
8 TREATMENT
9 EPIDEMIOLOGY
9 Premutation
10 PIPELINE DRUGS
14 RECENT EVENTS AND ANALYST OPINION
14 BPN14770 for FXS (November 2, 2020)
15 Zygel for FXS (October 15, 2020)
17 Zygel for FXS (June 30, 2020)
19 OV101 for FXS (May 7, 2020)
22 PROBABILITY OF SUCCESS
23 LICENSING AND ASSET ACQUISITION DEALS
23 Shionogi To Add CNS Critical Mass With Tetra Buyout
24 CLINICAL TRIAL LANDSCAPE
25 Sponsors by status
26 Sponsors by phase
28 BIBLIOGRAPHY
29 APPENDIX
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
15 Figure 5: BPN14770 for FXS (November 2, 2020): Phase II – Adults
17 Figure 6: Zygel for FXS (October 15, 2020): Phase III – CONNECT-FX (US, AUS, NZ)
19 Figure 7: Zygel for FXS (June 30, 2020): Phase III – CONNECT-FX (US, AUS, NZ)
21 Figure 8: OV101 for FXS (May 7, 2020): Phase II – ROCKET
22 Figure 9: Probability of success in the neurodevelopmental disorders pipeline
24 Figure 10: Clinical trials in FXS
24 Figure 11: Top 10 drugs for clinical trials in FXS
25 Figure 12: Top 10 companies for clinical trials in FXS
25 Figure 13: Trial locations in FXS
26 Figure 14: FXS trials status
27 Figure 15: FXS trials sponsors, by phase
LIST OF TABLES
12 Table 1: Pipeline drugs for FXS in the US
14 Table 2: BPN14770 for FXS (November 2, 2020)
16 Table 3: Zygel for FXS (October 15, 2020)
18 Table 4: Zygel for FXS (June 30, 2020)
20 Table 5: OV101 for FXS (May 7, 2020)
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