AL Amyloidosis - Pipeline Insight, 2024

This “AL Amyloidosis- Pipeline Insight, 2024” 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

In the United States, AL amyloidosis is the most common type, with approximately 4,500 new cases diagnosed every year. It usually affects people from ages 50-80, although there are a few cases of people being diagnosed as early as their late 20s. About two-thirds of the patients are male.

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, or 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.

These misfolded amyloid proteins are deposited in and around tissues, nerves and organs. As the amyloid builds up in an organ, nerve or tissue, it gradually causes damage and affects their function. Each amyloidosis patient has a different pattern of amyloid deposition in their body. It often affects more than one organ. AL amyloidosis does not affect the brain.

Symptoms
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.

Diagnosis
Diagnostic testing for AL amyloidosis involves blood tests, urine tests and biopsies. Blood and/or urine tests can indicate signs of the amyloid protein, but only bone marrow tests or other small biopsy samples of tissue or organs can positively confirm the diagnosis of amyloidosis. Some tests are only done once to establish a diagnosis of AL amyloidosis, while others will be repeated to monitor disease progression and response to therapy.

Treatment
Treatment plans are two-fold for the condition:
Supportive treatment - treating the symptoms and organ damage; and, Source treatment - slowing down, or stopping, the overproduction of amyloid at the source of the disease
Source Treatment
The U.S. Food and Drug Administration (FDA) has approved the use of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide, and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.

Stem Cell Transplant
Stem cell transplant (SCT) is often the preferred therapy, as it can provide long-term control of the underlying disease. However, only a minority of AL patients are eligible for this. For the majority of AL amyloidosis patients, and in many other countries outside the U.S., other chemotherapy-based treatments are considered. These treatment regimens are tailored for each patient and based upon the patients’ organ function, symptoms, and preferences.

Combination Therapy
Patients with AL amyloidosis have benefited from the recent development of new drugs for myeloma, many of which work effectively on the plasma cells that cause AL amyloidosis. Many drug combinations are more effective than single drugs in attacking the abnormal plasma cells and the dosage is tailored to each individual patient, to enable the best course of treatment and possible outcome. Categories of drugs that may be useful include:
Traditional chemotherapy drugs:

• Melphalan, Cyclophosphamide (Cytoxan™), Bendamustine (Treanda™)

Proteasome inhibitors: Bortezomib (Velcade™), MLN9708 (Ixazomib™), Carfilzomib (Kyprolis™)- Immunomodulators: Thalidomide (Thalidomid™), Lenalidomide (Revlimid™), Pomalidomide (Pomalyst™).

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. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival. CAEL-101 has received Orphan Drug Designation from both the U.S. 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).

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.

Phases

This 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

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