AL Amyloidosis – Pipeline Insight, 2020

DelveInsight’s, “AL Amyloidosis – Pipeline Insight, 2020,” report provides comprehensive insights about 15+ companies and 15+ 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
Amyloidosis is a rare and serious protein deposition disease. It is caused by an abnormal protein called amyloid that builds up in tissues or organs. As the amount of amyloid protein deposits increase in a tissue or organ, they interfere with the tissue or organ’s healthy function. Eventually, the amyloid protein deposits cause symptoms and organ failure. Amyloidosis is sometimes fatal. Light chain (AL) amyloidosis. This is the most common type of amyloidosis in the United States. The amyloid proteins that build up in the tissues in this condition are known as light chains. They can either be kappa or lambda light chains. AL amyloidosis is a disorder of the plasma cells. Plasma cells are a type of white blood cell responsible for the production of immunoglobulins or antibodies, which are a type of protein that fights infection. In AL amyloidosis, the light chain proteins are misshapen and produced in excess. They deposit in tissues and can damage 1 or more organs. The heart, kidneys, nerves, and gastrointestinal system are the most common organs affected.
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. Chronic kidney disease is common in patients with AL amyloidosis. Amyloid deposits in the heart can cause it to become unusually thickened and stiff, making it unable to function efficiently. This results in shortness of breath, which may occur with only minor activity. The digestive system is also called the gastrointestinal tract (or GI tract). Amyloid deposits in the digestive system can cause nausea, diarrhea or constipation, weight loss, loss of appetite, or a feeling of fullness in the stomach after eating small amounts. Amyloid deposits can affect the nerves of the hands, feet and lower legs and may cause pain, numbness and tingling.
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 for AL amyloidosis is tailored to the patient with their individual health in mind. The type of treatment is based upon disease progression and seriousness of the patient’s organ, tissue and nerve involvement.
Treatment plans are two-fold:
• Supportive treatment – treating your symptoms and organ damage; and,
• Source treatment – slowing down, or stopping, the overproduction of amyloid at the source of the disease.
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 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.
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.
• Bendamustine - Astellas Pharma GmbH
Bendamustine is being developed by Astellas Pharma GmbH for the treatment of AL amyloidosis. It is currently in phase II stage of development.
• STI-6129: Sorrento Therapeutics
STI-6129, a CD38-targeting antibody drug conjugate (ADC) that is being developed by Sorrento Therapeutics for the treatment of AL amyloidosis. It is currently in phase I stage of development.
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. 15+ 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 and others
• Phases
DelveInsight’s report covers around 15+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage products (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
• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical.
• Molecule Type
Products have been categorized under various Molecule types such as
• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies

• 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 III, 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.

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.
• 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.
• In January 2019 Alexion Pharmaceuticals and Caelum Biosciences announced a collaboration to develop CAEL-101 for light chain (AL) amyloidosis.
• In May 2020, Sorrento Therapeutics announced it has received clearance from the U.S. Food and Drug Administration (FDA) for its investigational new drug (IND) application for STI-6129.
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
• Sorrento Therapeutics
• Prothena Therapeutics
• Spectrum Pharmaceuticals
• Takeda
• Millennium Pharmaceuticals
• Caelum Biosciences
• Astellas Pharma GmbH

Key Products
• STI-6129
• NEOD001
• Propylene Glycol-Free Melphalan Hydrochloride
• Ixazomib
• CAEL-101
• Bendamustine