澱粉樣變性的治療進展

本報告提供澱粉樣變性研究分析，深入了解新療法、趨勢、挑戰、臨床管道以及增長機會分析。

內容

戰略要務

• 為什麼成長越來越難？
• 戰略要務 8 (TM)
• 三大戰略要務對開發澱粉樣變性新療法的影響
• 增長機會推動增長管道引擎 (TM)
• 調查方法

增長機會分析

• 分析範圍
• 細分
• 增長驅動力
• 抑制增長的因素

成長環境分析

• 澱粉樣變性簡介
• 現有的澱粉樣變性治療方法
• 澱粉樣變性的現有挑戰

澱粉樣變性的新療法 - 技術概覽

• 應對挑戰的管道療法
• 重新利用藥物來應對澱粉樣變性治療的挑戰
• 採用 AL 澱粉樣變性藥物類別
• 採用 ATTR 澱粉樣變性藥物類別
• 單克隆抗體 (mAb) 開發概覽
• 單克隆抗體的發展
• 基因治療發展概況
• RNAi 藥物開發概覽
• 開發 RNAi/siRNA 療法
• 開發 RNAi 和 ASO 療法
• 小分子開發快照
• 小分子的開發
• CAR-T 細胞療法開發概覽
• CAR-T 細胞療法的發展
• 正在開發的其□□他藥物
• 定向投放系統的新方法

管道/臨床試驗分析

• 獲批用於治療澱粉樣變性的藥物
• 正在臨床試驗中研究的藥物
• AL/ATTR 澱粉樣變性臨床試驗藥物分佈
• 臨床試驗中 AL 澱粉樣變性的主要治療方法
• 臨床試驗中 ATTR 澱粉樣變性的主要治療方式
• 臨床試驗中 ATTR 澱粉樣變性中心肌病和神經病的初步治療

利益相關者生態系統

• 戰略聯盟加強產品開發和商業化
• 衍生公司從重要的研發中成長
• 製造商與各利益相關方簽訂合同以促進市場進入
• 聯盟和公私合作夥伴關係提高了對罕見病的認識並傳授知識

資金情況

• 資助和研究基金的獲得者和組織
• 大學和生物製藥/製藥公司對澱粉樣變性的研究資助
• 來自製藥/生物製藥公司的貸款
• 患者支持計劃
• 我們可以從利益相關者環境的發展和趨勢中學到什麼
• 我們可以從資金狀況中學到什麼

增長機會

• 增長機會 1：與利益相關方簽署更多戰略協議以改善市場准入
• 增長機會 2：加速先進療法的研究
• 增長機會 3：不斷發現新的治療方式

下一步

Research Efforts and Technology Developments through Partnerships and Collaborations will Push New Therapeutic Modalities into Treatment Regimens.

Amyloidosis is a rare disease caused by the accumulation and the deposition of proteins such as immunoglobulin light-chain protein in AL amyloidosis and misfolded transthyretin (TTR) protein in ATTR amyloidosis. There are a few other types of amyloidosis as well, which are either a result of a preexisting condition or are not highly prevalent. Various challenges exist in terms of finding a cure for this debilitating disease, such as multiple mutations in the TTR gene, resulting in diverse etiologies across different geographies. Research efforts have decreased disease progression significantly, and many researchers are focusing on treatment options to deplete amyloid deposition on organs that cause their dysfunction.

Developments in amyloidosis treatment have resulted in many pharmaceutical and biotechnological companies trying different drug classes targeting various stages of pathways that lead to misfolded protein, including stabilizers, inhibitors, and silencers. Recently, gene editing drugs, such as RNA interference drugs, have been receiving approval, indicating the success rate of targeted therapies and creating a manufacturer monopoly for the development of a certain class of drugs for treatment.

Therapeutic options being considered are a mix of drug combinations, drugs used in multiple myeloma, other repurposed drugs, novel drug molecules, advanced therapeutics such as gene editing, CAR T-cells, and transplants. As healthcare advances toward modular methods, several upcoming companies are being spun off of universities that have developed technology platforms to target amyloid formation and deposition.

This Frost & Sullivan research service provides an overview of therapeutic advancements enabling amyloidosis research.

For new targets to be explored, research efforts have to run in parallel to the growing number of treatment approvals. Several funding organizations exist in parts of the United States and the United Kingdom, and grants are being offered by leading pharmaceutical companies for this effort. Drug development is also translating into collaborations and acquisitions between companies.

One of the many challenges associated with patients is the high cost of these therapies, which necessitates financial assistance programs by manufacturers. In addition, the ongoing COVID-19 pandemic is having a major impact, and the fear of contracting infection due to hospital exposure is making people averse to hospitalization; this is a major hindrance as many drugs are delivered as infusions.

Development of new target drugs and increased understanding of the disease have to be coupled with evidence-based treatment pathways so that there is uniformity in treatment and ease in measuring outcomes to facilitate streamlined treatment over the next 5-10 years. Furthermore, a treatment that is suitable for patients at all stages of the disease should be developed.

Growth opportunities exist in terms of having clinical trials and disease registries in geographies where the disease is prevalent as this will boost the market access of certain drugs based on the manifestation that the drug can treat. Screening of members at high risk and asymptomatic transthyretin gene mutations can curb the disease at its onset.

This study discusses various new and emerging therapeutics for amyloidosis, trends, challenges, and clinical pipelines; it also makes recommendations to pharmaceutical/biotechnological and research organizations to leverage growth opportunities.

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 the Development of New Therapies for Amyloidosis
• Growth Opportunities Fuel the Growth Pipeline Engine™
• Research Methodology

Growth Opportunity Analysis

• Scope of Analysis
• Segmentation
• Growth Drivers
• Growth Restraints

Growth Environment Analysis

• Introduction to Amyloidosis
• Existing Therapies in Amyloidosis
• Existing Challenges in Amyloidosis

Emerging Therapeutic Modalities in Amyloidosis-Technology Snapshot

• Therapies in the Pipeline to Address Challenges
• Therapies in the Pipeline to Address Challenges (continued)
• Repurposed Drugs to Address Amyloidosis' Treatment Challenges
• Drug Class Adoption for AL Amyloidosis
• Drug Class Adoption for ATTR Amyloidosis
• Snapshot of Monoclonal Antibody (mAb) Development
• Developments in mAb
• Snapshot of Developing Gene Therapies
• Snapshot of RNAi Drug Development
• Developments in RNAi and siRNA Therapy
• Developments in RNAi and ASO Therapy
• Snapshot of Small Molecule Development
• Developments in Small Molecules
• Snapshot of CAR T-Cell Therapy Development
• Developments in CAR T-Cell Therapy
• Other Drugs under Development
• New Approaches in Targeted Delivery Systems

Pipeline/Clinical Trial Analysis

• Drugs Approved for Amyloidosis Treatment
• Drugs under Investigation in Clinical Trials
• Drug Distribution in Clinical Trials for AL and ATTR Amyloidosis
• Top Therapeutic Modalities for AL Amyloidosis in Clinical Trials
• Top Therapeutic Modalities for ATTR Amyloidosis in Clinical Trials
• Top Therapeutic Modalities for Cardiomyopathy and Neuropathy in ATTR Amyloidosis in Clinical Trials

Stakeholder Ecosystem

• Strategic Collaborations Bolster Product Developments and Commercialization
• Spin-off Companies Grow from Significant Research Developments
• Manufacturers Enter into Contracts with Various Stakeholders to Ease Market Access
• Consortia and Public/Private Partnerships to Expand Awareness and Impart Knowledge of Rare Diseases

Funding Landscape

• Grants and Research Funding Recipients and Organizations
• Grants and Research Funding Recipients and Organizations (continued)
• Research Grants by Universities and Biopharma/Pharma Companies for Amyloidosis
• Research Grants by Universities and Biopharma/Pharma Companies for Amyloidosis (continued)
• Financing from Pharmaceutical/Biopharmaceutical Companies
• Patient Assistance Programs
• Takeaways from Developments and Trends in the Stakeholder Environment
• Takeaways from the Funding Landscape

Growth Opportunity Universe

• Growth Opportunity 1: Creating More Strategic Contracts with Stakeholders to Improve Market Access
• Growth Opportunity 1: Creating More Strategic Contracts with Stakeholders to Improve Market Access (continued)
• Growth Opportunity 2: Acceleration of Advanced Therapeutics Research
• Growth Opportunity 2: Acceleration of Advanced Therapeutics Research (continued)
• Growth Opportunity 3: Continued Discovery of New Modalities for Treatment
• Growth Opportunity 3: Continued Discovery of New Modalities for Treatment (continued)

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