AL Amyloidosis- Pipeline Insight, 2025

DelveInsight’s, “AL Amyloidosis- Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in AL Amyloidosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

AL Amyloidosis: Understanding

AL Amyloidosis: Overview

AL amyloidosis, also known as primary systemic amyloidosis, is a rare yet serious medical condition characterized by the abnormal accumulation of amyloid protein fibrils in tissues and organs throughout the body. This disorder arises from the overproduction of abnormal immunoglobulin light chains, stemming from irregular plasma cells within the bone marrow. These amyloid protein fibrils deposit in vital organs like the heart, kidneys, liver, nerves, and gastrointestinal tract, disrupting their normal function and structure. As a consequence, patients may experience a spectrum of symptoms such as fatigue, shortness of breath, swelling, easy bruising, weight loss, and neurological abnormalities.

Diagnosing AL amyloidosis typically involves a multifaceted approach encompassing clinical evaluation, laboratory tests, imaging studies, and tissue biopsy to confirm amyloid deposits. Timely diagnosis is pivotal for initiating appropriate treatment strategies and averting further organ damage. Treatment modalities for AL amyloidosis primarily focus on reducing the production of abnormal light chains and managing associated symptoms and complications. Therapeutic interventions may encompass chemotherapy, immunomodulatory drugs, stem cell transplantation, and supportive therapies tailored to address specific organ involvement.

Given its chronic nature, AL amyloidosis necessitates long-term management and vigilant monitoring. A collaborative, multidisciplinary approach involving hematologists, nephrologists, cardiologists, neurologists, and other specialists is often imperative to deliver comprehensive care attuned to the individualized needs of patients. Although AL amyloidosis poses significant challenges to quality of life and prognosis, advancements in diagnosis and treatment have led to improved outcomes for many individuals. Ongoing research endeavors aimed at exploring novel therapies and refining management strategies continue to enhance our understanding and management of this intricate disorder.

""AL Amyloidosis- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the AL amyloidosis pipeline landscape is provided which includes the disease overview and AL amyloidosis treatment guidelines. The assessment part of the report embraces, in depth AL amyloidosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, AL amyloidosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence AL amyloidosis R&D. The therapies under development are focused on novel approaches to treat/improve AL amyloidosis.

AL Amyloidosis Emerging Drugs Chapters

This segment of the AL Amyloidosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

AL Amyloidosis Emerging Drugs

• CAEL-101: Caelum Biosciences

CAEL-101 is a fibril-reactive monoclonal antibody (mAb) that is currently in Phase III clinical development for the treatment of patients with amyloid light chain (“AL”) amyloidosis. The Cardiac Amyloid Reaching for Extended Survival (CARES) clinical program includes two parallel Phase III studies – one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease – and will collectively enroll approximately 370 patients globally. The primary objective of the clinical program is to assess overall survival. CAEL-101 has received Orphan Drug Designation from both the US Food and Drug Administration and European Medicine Agency as a therapy for patients with AL amyloidosis.

• NXC-201: Nexcella, Inc.

NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and AL amyloidosis. The design consists of a structurally differentiated CAR-T, with the proprietary BCMA-targeting CAR, which has demonstrated reduced toxicity in NEXICART-1, supporting investigating NXC-201 as an outpatient therapy. It is currently in Phase I NEXICART-1 study of its novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy for the treatment of patients with relapsed or refractory multiple myeloma and light chain amyloidosis (AL).

Further product details are provided in the report……..

AL Amyloidosis: Therapeutic Assessment

This segment of the report provides insights about the different AL Amyloidosis drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in AL Amyloidosis

There are approx. 10+key companies which are developing the therapies for AL Amyloidosis. The companies which have their AL Amyloidosis drug candidates in the most advanced stage, i.e. Phase III include, Caelum Biosciences.

Phases

DelveInsight’s report covers around 10+products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

AL Amyloidosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal
• Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

AL Amyloidosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses AL Amyloidosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging AL Amyloidosis drugs.

AL Amyloidosis Report Insights

• AL Amyloidosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

AL Amyloidosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing AL Amyloidosis drugs?
• How many AL Amyloidosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of AL Amyloidosis?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the AL Amyloidosis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for AL Amyloidosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Caelum Biosciences
• Nexcella, Inc.
• HaemaLogiX Ltd.
• Prothena
• ZENTALIS PHARMACEUTICALS
• Sorrento Therapeutics, Inc.

Key Products

• CAEL-101
• NXC-201
• LambdaMab 10B3 & 7F11
• Birtamimab
• ZN-d5
• STI-6129