AL Amyloidosis- Pipeline Insight, 2025

DelveInsight's, "Amyloid Light-Chain Amyloidosis - Pipeline Insight, 2025" report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Amyloid Light-Chain 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

Amyloid Light-Chain Amyloidosis: Understanding

Amyloid Light-Chain Amyloidosis: Overview

Amyloid light-chain amyloidosis, (AL) is the most common form of systemic amyloidosis and is associated with an underlying plasma cell dyscrasia. AL amyloidosis is caused by a bone marrow disorder. The bone marrow in the center of bones produces cells in the blood system, including "plasma cells." These plasma cells are the part of the immune system that makes antibodies for fighting infections. The term "immunoglobulin" refers to the class of proteins that function as antibodies. Immunoglobulins are composed of four protein chains: two light chains, either kappa or lambda light chains, and two heavy chains, of which there are several types. These proteins are produced by the plasma cells in the bone marrow. In AL patients, these plasma cells produce an abnormal antibody (immunoglobulin) protein. For AL amyloidosis, it is the "light chains" that become misfolded, and the abnormal, misfolded result is the forming of amyloid. With AL amyloidosis, the "A" is for amyloid and the "L" is for light chain.

The symptoms of AL amyloidosis vary by patient. Initially, the symptoms can be minor or similar to those of many other conditions or systemic diseases. Some symptoms can announce themselves quickly and be very noticeable. For each patient, the symptoms will depend on which organs are affected by the amyloid deposits. It also depends on the degree that the organ function is impaired. Fatigue, weight loss and swelling are the most common symptoms. Impairment of many organs, nerves and soft tissues can cause symptoms, among them the kidneys, heart, the GI tract (the digestive system) and the nervous system.

AL amyloidosis is caused by a clonal expansion of plasma cells that overproduce structurally unstable immunoglobulin light chains, which misfold and aggregate into amyloid fibrils. These misfolded proteins deposit in tissues, disrupting organ function through mechanical infiltration, proteotoxicity, and inflammation. The failure of cellular proteostasis systems to clear the misfolded proteins exacerbates disease progression. Genetic mutations in plasma cells and light chain genes further drive amyloidogenesis and organ damage.

The nonspecific and often vague nature of symptoms that are associated with AL amyloidosis frequently leads to delays in diagnosis such that organ dysfunction is advanced by the time treatment is initiated. The diagnosis of AL amyloidosis should be considered in patients with unexplained proteinuria, cardiomyopathy, neuropathy, or hepatomegaly and in patients with multiple myeloma that has atypical manifestations. The primary treatment goal for systemic amyloidosis, particularly AL (light chain) amyloidosis, is to rapidly reduce the production of amyloidogenic light chains by targeting the underlying plasma cell dyscrasia. This strategy is founded on evidence showing that decreasing the source of the amyloid protein can lead to improved organ function and prolonged survival.

"Amyloid Light-Chain Amyloidosis - Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the Amyloid Light-Chain Amyloidosis pipeline landscape is provided which includes the disease overview and Amyloid Light-Chain Amyloidosis treatment guidelines. The assessment part of the report embraces, in depth Amyloid Light-Chain 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, Amyloid Light-Chain 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 Amyloid Light-Chain Amyloidosis R&D. The therapies under development are focused on novel approaches to treat/improve Amyloid Light-Chain Amyloidosis.

Amyloid Light-Chain Amyloidosis Emerging Drugs Chapters

This segment of the Amyloid Light-Chain Amyloidosis report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, 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.

Amyloid Light-Chain Amyloidosis Emerging Drugs

• Birtamimab: Prothena

Birtamimab is an investigational, humanized monoclonal antibody designed to specifically and selectively target and clear the amyloid that accumulates and causes organ dysfunction and failure in patients with AL amyloidosis. Birtamimab specifically binds to a defined epitope on kappa and lambda AL protein involved in the disease process. Birtamimab is the only investigational drug that has shown a significant survival benefit in patients with Mayo Stage IV AL amyloidosis post-hoc in a placebo-controlled clinical trial. Birtamimab has been granted orphan drug designation for AL amyloidosis by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency and has been granted Fast Track designation by the FDA. A Special Protocol Assessment (SPA) was agreed to between Prothena and the FDA for the AFFIRM-AL clinical trial which represents FDA's agreement that the design and planned analysis for the primary endpoint of time to all-cause mortality adequately address the objectives necessary to support a regulatory submission. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Al Amyloidosis.

• NXC-201: Immix Biopharma

NXC-201 is a next-generation, sterically-optimized B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T (CAR-T) cell therapy designed to treat relapsed or refractory AL amyloidosis. Its mechanism of action involves the genetic modification of a patient's T cells to express a CAR that specifically recognizes and binds to BCMA, a protein highly expressed on plasma cells responsible for producing amyloidogenic light chains in AL amyloidosis. Upon binding to BCMA, NXC-201 CAR-T cells become activated, proliferate, and induce targeted cytotoxicity against the aberrant plasma cells, thereby reducing amyloid production.

• ABBV-383: Abbvie

Etentamig (ABBV-383) is a bispecific antibody that targets both B-cell maturation antigen (BCMA) on malignant plasma cells and CD3 on T cells, facilitating T-cell mediated cytotoxicity in patients with relapsed or refractory multiple myeloma and amyloidosis. Its mechanism of action involves simultaneous binding to BCMA-expressing plasma cells and CD3-positive T cells, bringing them into close proximity to activate and redirect the patient's own T cells to kill the abnormal plasma cells. Etentamig is being evaluated in patients who have received at least three prior lines of therapy, addressing a critical need in heavily pretreated populations.

Amyloid Light-Chain Amyloidosis: Therapeutic Assessment

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

• Major Players in Amyloid Light-Chain Amyloidosis

There are approx. 10+ key companies which are developing the therapies for Amyloid Light-Chain Amyloidosis. The companies which have their Amyloid Light-Chain Amyloidosis drug candidates in the most advanced stage, i.e. Phase III include Prothena.

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

Amyloid Light-Chain 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
• 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.

Amyloid Light-Chain Amyloidosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Amyloid Light-Chain 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 Amyloid Light-Chain Amyloidosis drugs.

Amyloid Light-Chain Amyloidosis Report Insights

• Amyloid Light-Chain Amyloidosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Amyloid Light-Chain 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 Amyloid Light-Chain Amyloidosis drugs?
• How many Amyloid Light-Chain Amyloidosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Amyloid Light-Chain Amyloidosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Amyloid Light-Chain 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 Amyloid Light-Chain Amyloidosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Prothena
• AstraZeneca
• Abbvie
• Immix Biopharma
• Paradox Immunotherapeutics

Key Products

• Birtamimab
• Anselamimab
• ABBV-383
• NXC-201
• LX-96