焦点市场分析:X染色体脆折症候群

根据需要特殊教育的儿童相关世代调查的结果，男儿4000人，女孩儿8000人有一人，X染色体脆折症候群 (FXS) 发病的可能性。复数的未来潜力治疗药物正在进行临床实验中，某些产品已进入II期。还有2015年至2020年之间已达成五项许可协议和资产收购交易，提高了对FXS治疗剂开发的期望。就各国来看，美国在药物开发方面处于领先地位。

本报告提供全球X染色体脆折症候群 (FXS) 治疗药的市场相关分析，汇整疾病概要和治疗方法，目前发病情形，主要的已上市/开发平台医药品的概要，近来临床实验趋势与今后的预测，近几年的授权合约·资本交易的动向，专利趋势，市场规模趋势预测 (今后5年份)等资讯，为您概述为以下内容。

概要

主要的要点

疾病的背景

治疗

流行病学

• 前突然变异

开发平台医药品

近来的主要市场活动和分析师的见解

今后主要的活动

成功的可能性

授权合约·资产收购交易

母专利

临床实验环境

• 临床试验赞助者一览:各状况
• 临床试验赞助者一览:各期

参考文献

附录

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

CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

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

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 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)