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.
- 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.
Key Topics Covered:
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
For more information about this report visit https://www.researchandmarkets.com/r/onurxo.