罕见神经肌肉疾病的新治疗市场：管道分析

本报告探讨了罕见神经肌肉疾病的全球新兴治疗市场，并提供了市场概览、战略要务和增长机会。

内容

战略要务

• 为什么增长越来越难？
• 战略要务
• 罕见神经肌肉疾病的三大战略要务的影响
• 增长机会加速增长管道引擎

增长机会分析

• 分析范围
• 细分
• NMD 中主要的新兴生物学类别
• 增长动力
• 抑制增长的因素

成长环境

• 投资者对罕见神经肌肉疾病治疗的兴趣激增
• 风险投资支持的公司推动生物製品进步的概况
• 神经肌肉疾病的生物专利
• 专利状态 - 神经肌肉疾病的生物製剂
• 主要市场进入者

肌萎缩侧索硬化 (ASL)：生物製品管道分析

• 肌萎缩侧索硬化症的生物疗法
• 肌萎缩侧索硬化细胞疗法，2022 年
• 肌萎缩侧索硬化症寡核甘酸疗法，2022 年
• 肌萎缩侧索硬化蛋白疗法，2022 年
• 近期治疗肌萎缩侧索硬化症的生物学研究要点
• 探索治疗肌萎缩侧索硬化症的新治疗范式

重症肌无力 (MG)：生物製剂管道分析

• 用于治疗重症肌无力的未来生物製剂
• 重症肌无力的蛋白质疗法
• 重症肌无力生物製剂临床试验进展
• 领先的重症肌无力生物製剂

杜氏肌营养不良症 (DMD)：生物製剂管道分析

• Duchenne 肌营养不良症新兴生物学景观中的基因治疗
• 杜氏肌营养不良症的外显子跳跃疗法
• 杜氏肌营养不良症的微肌营养不良蛋白基因疗法
• 引领杜氏肌营养不良症治疗的寡核甘酸疗法

脊髓性肌萎缩症 (SMA)：生物製剂管道分析

• 已获批准和新兴的脊髓性肌萎缩症生物学类别
• 改变脊髓性肌萎缩症临床状况的生物製剂
• 利用突破性疗法治疗脊髓性肌萎缩症
• Novartis Zolgensma：脊髓性肌萎缩症的临床突破

糖尿病肾病 (DN) 和慢性炎症性脱髓鞘性多发性神经病 (CIDP)：生物製剂产品线分析

• 用于治疗糖尿病肾病的生物製剂
• 用于糖尿病肾病个性化治疗的生物製剂
• 一种用于治疗慢性炎症性脱髓鞘性多发性神经病的新型生物製剂
• 用于治疗慢性炎症性脱髓鞘性多发性神经病的免疫球蛋白

增长机会领域

• 增长机会 1：RNA 疗法
• 成长机会 2：细胞和基因疗法
• 增长机会 3：蛋白质疗法

下一步

Strategic partnering and new product development will enable better biologics for personalized and targeted treatment

Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs). The disorders are largely classified as motor neuron diseases, hereditary ataxias, peripheral nerve disorders, neuromuscular junction transmission disorders, and myopathies. NMDs present significant clinical challenges because of disease heterogeneity and rarity of occurrence. The lack of measurable early disease markers and clinical outcomes further complicates the management of such disorders.

Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class. An overview of the global clinical trial landscape for emerging Phase 1, 2, and 3 treatments revealed that Amyotrophic Lateral Sclerosis (ALS), Myasthenia Gravis (MG), Duchenne Muscular Dystrophy (DMD), Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), Diabetic Neuropathy (DN), and Spinal Muscular Atrophy (SMA) are the key hotspots for clinical studies. These indications are likely to witness new drug developments. Frost & Sullivan's research, "Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis" explores emerging biologics across these six NMDs.

Key biologics emerging for these rare NMDs include: stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments. There were 3 recent US FDA approvals for NMD RNA therapies: Nusinersen for SMA and Eteplirsen for DMD in 2016, and Golodirsen for DMD in 2019. Furthermore, Sarepta's Casimersen for DMD and argenx's efgartigimod for MG were approved as recently as 2021. Biologics have opened doors for ground-breaking disease-modifying treatments and will transform the quality of life for patients across the world.

While biologics may appear to cost more than small molecule treatments, they provide a personalized and, in some cases, the only treatment option. They can also be less expensive than lifetime treatment costs for certain rare NMDs. For example, Zolgensma's $2.1 million price tag (approximate) is still lower than the standard medical costs incurred for SMA patients in a lifetime. Furthermore, the evolving payer landscape is likely to get more standardized in the coming years to offer greater access to biologicals. Therefore, biologics are suitably poised to transform the rare NMD landscape with personalized and targeted treatment strategies and will provide life-saving treatment options for fatal neuromuscular conditions.

Key Points Discussed:

• What are the key emerging biologics for NMDs with high clinical activity?

• What are the key drivers or challenges for biologics development across NMDs?

• How do venture funding and patent landscapes look for NMD biologics?

• Who are the key industry participants developing biologics for NMDs?

• What are the clinical trends emerging across each biologic category for NMDs?

• Which biologic categories provide promising growth opportunities for NMD management?

Table of Contents

Strategic Imperatives

• Why Is It Increasingly Difficult to Grow?The Strategic Imperative 8™: Factors Creating Pressure on Growth
• The Strategic Imperative 8™
• The Impact of the Top 3 Strategic Imperatives on Rare Neuromuscular Disease Management
• Growth Opportunities Fuel the Growth Pipeline Engine™
• Research Methodology

Growth Opportunity Analysis

• Scope of Analysis
• Segmentation
• Predominant Emerging Biologic Classes Across NMDs
• Growth Drivers
• Growth Restraints

Growth Environment

• Sharp Growth in Investor Interest for Rare NMD Treatments
• Snapshot of Venture-backed Companies that Enable Biologics Progress
• Biologic Patents for NMDs
• Patent Landscape-Biologics for NMDs
• Top Participants

ALS: Biologics Pipeline Analysis

• Biological Therapies for ALS
• ALS Cell Therapies, 2022
• ALS Oligonucleotide Therapies, 2022
• ALS Protein Therapies, 2022
• Highlights of Recent Biologics Studies for ALS Treatment
• Exploring New Therapy Paradigms for ALS Treatment

MG: Biologics Pipeline Analysis

• Upcoming Biologics for MG Treatment
• Protein Therapies for MG
• Clinical Trial Progress in Biologics* for MG
• Dominant MG Biologics

DMD: Biologics Pipeline Analysis

• Gene Therapies in the Emerging DMD Biologics Landscape
• Exon-Skipping Therapies for DMD
• Microdystrophin Gene Therapies for DMD
• Oligonucleotide Therapies to Lead DMD Management

SMA: Biologics Pipeline Analysis

• Approved and Emerging Biologics* Classes for SMA
• Biologics Transforming the Clinical Landscape for SMA
• SMA Management Through Groundbreaking Therapies
• Novartis' Zolgensma: A Clinical Breakthrough for SMA
• Novartis' Zolgensma: A Clinical Breakthrough for SMA (continued)
• Novartis' Zolgensma: A Clinical Breakthrough for SMA (continued)

DN & CIDP: Biologics Pipeline Analysis

• Biologics for DN Treatment
• Biologics for Treatment Personalization in DN
• Emerging Biologics for CIDP Treatment
• Immunoglobulins for CIDP Treatments

Growth Opportunity Universe

• Growth Opportunity 1: RNA Therapeutics
• Growth Opportunity 1: RNA Therapeutics (continued)
• Growth Opportunity 2: Cell and Gene Therapeutics
• Growth Opportunity 2: Cell and Gene Therapeutics (continued)
• Growth Opportunity 3: Protein Therapeutics
• Growth Opportunity 3: Protein Therapeutics (continued)

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