This Market Spotlight report covers the Fragile X Syndromemarket, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, probability of success, a discussion on disease epidemiology, and licensing and asset acquisition deals.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
Published: 11 March 2022
Number of pages:27
This Market Spotlight report covers the Fragile X Syndromemarket, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, key upcoming events, probability of success, a discussion on disease epidemiology, and licensing and asset acquisition deals.
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 largest proportion of industry-sponsored drugs in active clinical development for FXS are in Phase II. Therapies in active clinical development for FXS focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, GABA receptors, insulin-like growth factor-1 receptor, NMDA glutamate receptor, and potassium channels. The majority of these pipeline drugs are administered via the oral route.
The only high-impact upcoming event for drugs in the FXS space is topline Phase III trial results for Zygel. The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 2.6%, and the average probability a drug advances from Phase III is 18.2%. Drugs, on average, take 12.6 years from Phase I to approval, compared to 10.2 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 81% of trials in Phase I–II, and only 19% in Phase III–IV.
The US has a substantial lead in the number of FXS clinical trials globally. France leads the major European markets, while Israel has the top spot in Asia.
Clinical trial activity in the FXS space is dominated by completed trials. Roche has the highest number of completed clinical trials for FXS, with 10 trials.
Novartis and Roche lead industry sponsors with the highest overall number of clinical trials for FXS, followed by Seaside Therapeutics.
6 KEY TAKEAWAYS
7 DISEASE BACKGROUND
10 PIPELINE DRUGS
14 RECENT EVENTS AND ANALYST OPINION
14 BPN14770 for FXS (November 2, 2020)
15 Zygel for FXS (October 15, 2020)
18 KEY UPCOMING EVENTS
19 PROBABILITY OF SUCCESS
20 LICENSING AND ASSET ACQUISITION DEALS
20 Lysogene Gets SATT Conectus’s Fragile X Gene Therapy Candidate
21 CLINICAL TRIAL LANDSCAPE
22 Sponsors by status
23 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
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)
18 Figure 7: Key upcoming events in FXS
19 Figure 8: Probability of success in the neurodevelopmental disorders pipeline
21 Figure 9: Clinical trials in FXS
21 Figure 10: Top 10 drugs for clinical trials in FXS
22 Figure 11: Top 10 companies for clinical trials in FXS
22 Figure 12: Trial locations in FXS
23 Figure 13: FXS trials status
24 Figure 14: 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)
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