Al Amyloidosis Pipeline Insight

DelveInsight’s, “AL Amyloidosis – Pipeline Insight, 2022,” 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

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, eitherkappa 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. 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 diagnosis of AL amyloidosis requires demonstration of amyloid in tissue and demonstration of a plasma cell dyscrasia. The current therapeutic approach to systemic amyloidosis is based on the observations that organ dysfunction improves and survival increases if the synthesis of the amyloidogenic protein precursor is halted. Therefore, the aim of therapy in AL amyloidosis is to reduce rapidly the supply of amyloidogenic monoclonal light chains by suppressing the underlying plasma cell dyscrasia. Decisions about specific treatment regimens for individual patients must take into consideration the balance between anticipated treatment efficacy and tolerability.

 

"AL Amyloidosis- Pipeline Insight, 2022" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the A L Amyloidosis pipeline landscape is provided which includes the disease overview and A L Amyloidosis treatment guidelines. The assessment part of the report embraces, in depth A L 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, A L 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 A L 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 first-in-class anti-amyloid antibody designed to improve organ function by reducing or eliminating amyloid deposits in patients with amyloid light chain (AL) amyloidosis. Alexion is collaborating with Caelum Biosciences to develop CAEL-101 for light chain (AL) amyloidosis, a rare systemic disorder that causes misfolded immunoglobulin light chain protein to build up in and around tissues, resulting in progressive and widespread organ damage. Alexion and Caelum Biosciences are conducting the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care therapy in AL amyloidosis. 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.

 

Ixazomib: Takeda

Ninlaro (ixazomib) is a proteasome inhibitor. Ixazomib preferentially binds and inhibits the chymotrypsin-like activity of the beta 5 subunit of the 20S proteasome. Ixazomib induced apoptosis of multiple myeloma cell lines in vitro. The drug is being evaluated in Phase III stage of development for the treatment of patients with AL Amyloidosis.

 

Belantamab mafodotin: GlaxoSmithKline

Belantamab mafodotin, or GSK2857916, is an afucosylated monoclonal antibody that targets B cell maturation antigen (BCMA) conjugated to the microtubule distrupter monomethyl auristatin-F (MMAF).1 Afucosylation of the Fc region of monoclonal antibodies enhances binding to the Fc region, which enhances antibody dependant cell mediated cytoxicity. Phase II clinical Study of Belantamab Mafodotin has been initiated to treat Patients with Relapsed or Refractory AL Amyloidosis.

 

STI 6129 : Sorrento Therapeutics

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.

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

AL Amyloidosis: Therapeutic Assessment

This segment of the report provides insights about the different A L 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 A L 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?

Introduction

Executive Summary

AL Amyloidosis: Overview

  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics

  • Comparative Analysis

Therapeutic Assessment

  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

AL Amyloidosis– DelveInsight’s Analytical Perspective

Late Stage Products (Phase III)

  • Comparative Analysis

CAEL-101: Caelum Biosciences

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

Belantamab mafodotin : GlaxoSmithKline

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

STI-6129: Sorrento Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical and Discovery Stage Products

  • Comparative Analysis

Drug name: Company Name

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

AL Amyloidosis Key Companies

AL Amyloidosis Key Products

AL Amyloidosis- Unmet Needs

AL Amyloidosis- Market Drivers and Barriers

AL Amyloidosis- Future Perspectives and Conclusion

AL Amyloidosis Analyst Views

AL Amyloidosis Key Companies

Appendix

List of Table

Table 1: Total Products for AL Amyloidosis

Table 2: Late Stage Products

Table 3: Mid Stage Products

Table 4: Early Stage Products

Table 5: Pre-clinical & Discovery Stage Products

Table 6: Assessment by Product Type

Table 7: Assessment by Stage and Product Type

Table 8: Assessment by Route of Administration

Table 9: Assessment by Stage and Route of Administration

Table 10: Assessment by Molecule Type

Table 11: Assessment by Stage and Molecule Type

Table 12: Inactive Products

List of Figures

Figure 1: Total Products for AL Amyloidosis

Figure 2: Late Stage Products                             

Figure 3: Mid Stage Products

Figure 4: Early Stage Products

Figure 5: Preclinical and Discovery Stage Products

Figure 6: Assessment by Product Type

Figure 7: Assessment by Stage and Product Type

Figure 8: Assessment by Route of Administration

Figure 9: Assessment by Stage and Route of Administration

Figure 10: Assessment by Molecule Type

Figure 11: Assessment by Stage and Molecule Type

Figure 12: Inactive Products   

• Merck & Co
• Janssen Pharmaceutical
• Caelum Biosciences
• Oncopeptides AB
• Bristol-Myers Squibb
• Acrotech biopharma
• Sanofi
• Astellas Pharma GmbH

 

 

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