アミロイドーシスの治療の進歩

当レポートでは、アミロイドーシスについて調査分析し、新しい治療法、動向、課題、臨床パイプラインについての考察や、成長機会の分析などを提供しています。

目次

戦略的必須要件

• 成長がますます困難になっているのはなぜか
• The Strategic Imperative 8 (TM)
• アミロイドーシスの新しい治療法の開発に対する上位3つの戦略的必須要件の影響
• 成長機会がGrowth Pipeline Engine (TM) を促進する
• 調査手法

成長機会の分析

• 分析の範囲
• セグメンテーション
• 成長促進要因
• 成長抑制要因

成長環境分析

• アミロイドーシスのイントロダクション
• アミロイドーシスの既存の治療法
• アミロイドーシスの既存の課題

アミロイドーシスの新たな治療法 - 技術スナップショット

• 課題を対処するためのパイプラインの治療法
• アミロイドーシス治療の課題に対処するために転用された医薬品
• ALアミロイドーシスに対する医薬品クラスの採用
• ATTRアミロイドーシスに対する医薬品クラスの採用
• モノクローナル抗体（mAb）開発のスナップショット
• mAbにおける発展
• 遺伝子治療開発のスナップショット
• RNAi医薬品開発のスナップショット
• RNAi・siRNA治療の発展
• RNAiとASO治療の発展
• 低分子開発のスナップショット
• 低分子における発展
• CAR-T細胞治療開発のスナップショット
• CAR-T細胞治療における発展
• その他の開発中の医薬品
• 標的型デリバリーシステムの新しいアプローチ

パイプライン/臨床試験分析

• アミロイドーシスの治療に向けて承認されている医薬品
• 臨床試験で調査中の医薬品
• AL・ATTRアミロイドーシスの臨床試験における医薬品の流通
• 臨床試験におけるALアミロイドーシスの主な治療法
• 臨床試験におけるATTRアミロイドーシスの主な治療法
• 臨床試験におけるATTRアミロイドーシスの心筋症と神経障害の主な治療法

ステークホルダーのエコシステム

• 戦略的提携が製品開発と商品化を強化する
• スピンオフ企業は重要な研究開発から成長する
• メーカーは市場参入を容易にするためにさまざまなステークホルダーと契約を結ぶ
• コンソーシアムと官民パートナーシップは希少疾患の認識を広げ、知識を与える

資金調達情勢

• 助成金と研究費の受取人と組織
• アミロイドーシスに向けた大学とバイオ医薬品/製薬企業による研究費
• 医薬品/バイオ医薬品企業からの融資
• 患者支援プログラム
• ステークホルダーの環境における発展と動向から学べること
• 資金調達情勢から学べること

成長機会

• 成長機会1：市場参入の改善に向けた、ステークホルダーとのより戦略的な契約の締結
• 成長機会2：先進治療法の研究の加速
• 成長機会3：治療のための新しいモダリティの継続的な発見

次のステップ

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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