Systemic Amyloidosis - Pipeline Insight, 2024

This “Systemic Amyloidosis- Pipeline Insight, 2024” report provides comprehensive insights about 7+ companies and 7+ pipeline drugs in Systemic 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

Systemic Amyloidosis: Understanding

Systemic Amyloidosis: Overview

Amyloidosis constitutes a large spectrum of diseases characterized by an extracellular deposition of a fibrillar aggregate, generating insoluble and toxic amasses that may be deposited in tissues in bundles with an abnormal cross-ß-sheet conformation, known as amyloid. Amyloid may lead to a cell damage and an impairment of organ function. Several different proteins are recognized as able to produce amyloid fibrils with a different tissue tropism related to the molecular structure. The deposition of amyloid may occur as a consequence of the presence of an abnormal protein, caused by high plasma levels of a normal protein, or as a result of the aging process along with some environmental factors. Tissue deposition of protein fibrils causes a group of rare diseases called systemic amyloidosis. Systemic light chain (AL) amyloidosis is the most common of these conditions, but wild-type transthyretin cardiac amyloidosis (ATTRwt) is increasingly being diagnosed. Typing of amyloid fibrils, a critical determinant of therapy, has improved with the wide availability of laser capture and mass spectrometry from fixed histological tissue sections. Specific and accurate evaluation of cardiac amyloidosis is now possible using cardiac magnetic resonance imaging and cardiac repurposing of bone scintigraphy tracers. Survival in AL amyloidosis has improved markedly as novel chemotherapy agents have become available, but challenges remain in advanced disease.

Systemic Amyloidosis- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Systemic Amyloidosis pipeline landscape is provided which includes the disease overview and Systemic Amyloidosis treatment guidelines. The assessment part of the report embraces, in depth Systemic 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, Systemic 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 Systemic Amyloidosis R&D. The therapies under development are focused on novel approaches to treat/improve
Systemic Amyloidosis.

Systemic Amyloidosis Emerging Drugs Chapters

This segment of the Systemic 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.

Systemic Amyloidosis Emerging Drugs

STI-6129: Sorrento Therapeutics, Inc.STI-6129 is an ADC with covalently bound duostatin tubulin inhibitors (Duostatin 5) using a proprietary site-specific C-LOCK™ chemical linker designed to reduce the premature systemic release of duostatin and avoid the potential for toxicity, particular ocular toxicity, seen with other ADCs, especially first-generation products. STI-6129 has demonstrated an improved therapeutic index in animal models, as compared to traditional non-selective conjugates. STI-6129 has the potential for being a first-line treatment for amyloidosis as well as second line in those patients who have developed daratumumab resistance, an anti-CD38 mAb alone. STI-6129 binds to different epitopes than daratumumab and the addition of the targeted delivery of the duostatin can potentially manage those patients who have become refractory to such treatments. Currently, the drug is in Phase I stage of Clinical trial evaluation for the treatment of Systemic AL Amyloidosis.

AT-03: Attralus AT-03 is a fusion of the company’s PAR-SAP (Serum Amyloid Protein) technology with a single-chain Fc. The PAR-SAP component mediates binding to all types of amyloid deposits, and the single-chain Fc stimulates the immune system to remove amyloid deposits that are bound by AT-03. Attralus obtained exclusive rights to develop, manufacture and commercialize AT-03 under license agreements with University of Limoges. Currently, the drug is in Phase I stage of Clinical trial evaluation for the treatment of SystemicAmyloidosis.

Systemic Amyloidosis: Therapeutic Assessment

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

Major Players in Systemic Amyloidosis

There are approx. 7+ key companies which are developing the therapies for Systemic Amyloidosis. The companies which have their Systemic Amyloidosis drug candidates in the most advanced stage, i.e. Phase I, Attralus.

Phases

This report covers around 7+ 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

Systemic 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.

Systemic Amyloidosis: Pipeline Development Activities

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

Systemic Amyloidosis Report Insights

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

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

Key Players

• Attralus
• Sorrento Therapeutics, Inc.
• Proclara Bioscience
• Takeda

Key Products

• STI-6129
• AT-03
• AT- 01
• NPT189
• IXAZOMIB
• Bendamustine Hydrochloride