罕見神經肌肉疾病的新治療市場：管道分析

本報告探討了罕見神經肌肉疾病的全球新興治療市場，並提供了市場概覽、戰略要務和增長機會。

內容

戰略要務

• 為什麼增長越來越難？
• 戰略要務
• 罕見神經肌肉疾病的三大戰略要務的影響
• 增長機會加速增長管道引擎

增長機會分析

• 分析範圍
• 細分
• NMD 中主要的新興生物學類別
• 增長動力
• 抑制增長的因素

成長環境

• 投資者對罕見神經肌肉疾病治療的興趣激增
• 風險投資支持的公司推動生物製品進步的概況
• 神經肌肉疾病的生物專利
• 專利狀態 - 神經肌肉疾病的生物製劑
• 主要市場進入者

肌萎縮側索硬化 (ASL)：生物製品管道分析

• 肌萎縮側索硬化症的生物療法
• 肌萎縮側索硬化細胞療法，2022 年
• 肌萎縮側索硬化症寡核甘酸療法，2022 年
• 肌萎縮側索硬化蛋白療法，2022 年
• 近期治療肌萎縮側索硬化症的生物學研究要點
• 探索治療肌萎縮側索硬化症的新治療範式

重症肌無力 (MG)：生物製劑管道分析

• 用於治療重症肌無力的未來生物製劑
• 重症肌無力的蛋白質療法
• 重症肌無力生物製劑臨床試驗進展
• 領先的重症肌無力生物製劑

杜氏肌營養不良症 (DMD)：生物製劑管道分析

• Duchenne 肌營養不良症新興生物學景觀中的基因治療
• 杜氏肌營養不良症的外顯子跳躍療法
• 杜氏肌營養不良症的微肌營養不良蛋白基因療法
• 引領杜氏肌營養不良症治療的寡核甘酸療法

脊髓性肌萎縮症 (SMA)：生物製劑管道分析

• 已獲批准和新興的脊髓性肌萎縮症生物學類別
• 改變脊髓性肌萎縮症臨床狀況的生物製劑
• 利用突破性療法治療脊髓性肌萎縮症
• Novartis Zolgensma：脊髓性肌萎縮症的臨床突破

糖尿病腎病 (DN) 和慢性炎症性脫髓鞘性多發性神經病 (CIDP)：生物製劑產品線分析

• 用於治療糖尿病腎病的生物製劑
• 用於糖尿病腎病個性化治療的生物製劑
• 一種用於治療慢性炎症性脫髓鞘性多發性神經病的新型生物製劑
• 用於治療慢性炎症性脫髓鞘性多發性神經病的免疫球蛋白

增長機會領域

• 增長機會 1：RNA 療法
• 成長機會 2：細胞和基因療法
• 增長機會 3：蛋白質療法

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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