Cold Agglutinin Disease - Pipeline Insight, 2024

This “Cold Agglutinin Disease- Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Cold Agglutinin Disease 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

Cold Agglutinin Disease Understanding

Cold Agglutinin Disease: Overview

Cold agglutinin disease (CAD) is a rare autoimmune disorder characterized by the premature destruction of red blood cells (hemolysis). Autoimmune diseases occur when one’s own immune system attacks healthy tissue. More specifically, CAD is a subtype of autoimmune hemolytic anemia. In this type of disorder, red blood cells are “tagged” by antibodies and are then destroyed by other types of immune cells. The disease is termed “cold” because the antibodies are active and cause hemolysis at cold temperatures, usually 3 to 4oC (37 to 39oF), which is not necessarily the case in other types of autoimmune hemolytic anemia. CAD affects about one person per million every year, and mostly develops between the ages of 40 and 80 years. Normally, the red blood cells have a life span of approximately 120 days before they are destroyed by the spleen. In individuals with CAD, the red blood cells are destroyed prematurely and the rate of production of new cells in the bone marrow can no longer compensate for their loss. A decreased number of red blood cells (anemia) may cause fatigue, weakness, a pale skin color (pallor), dizziness, palpitations, and shortness of breath (dyspnea). Hemolysis leads to an increased release from red blood cells of hemoglobin, a protein responsible for carrying oxygen in the blood. Degradation of hemoglobin into bilirubin can result in yellowing of the skin and whites of the eyes (jaundice). Hemoglobin can also pass in the urine and give it a dark brown color. Other symptoms that can be triggered by exposure to cold include sweating and coldness of the fingers and/or toes (digits) and painful bluish or reddish discoloration of the skin of the digits, ankles, and wrists (acrocyanosis or Raynaud sign). Treatment of CAD includes avoidance of cold temperatures, treating anemia and hemolysis (if needed) and medications that modulate the immune system to decrease the production of antibodies against red blood cells. CAD typically develops in individuals between the age of 40 and 80, and is more common in elderly individuals. The symptoms associated with the disease are mostly the result of either hemolysis or circulatory symptoms, both of which are triggered by exposure to cold temperatures. Some individuals, especially those with mild hemolysis and a gradual onset of anemia, may not have any obvious symptoms (asymptomatic). Symptoms of anemia include paleness of the skin (pallor), fatigue, shortness of breath (dyspnea), dizziness and palpitations. In cases of brisk and severe hemolysis, chest pain, decreased alertness (lethargy), confusion, transient loss of consciousness (syncope), and deregulation of heart rate and blood pressure (hemodynamic instability) might occur. CAD most commonly affects people between the ages of 40 and 80. The median age at symptom onset is around 65 years, meaning that half of affected individuals develop symptoms before this age, and the other half after this age. The disease is present in about 16 people per million (prevalence), and develops in one person per million every year (incidence). The disease is almost twice as common in women compared to men.

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

Cold Agglutinin Disease Emerging Drugs Chapters

This segment of the Cold Agglutinin Disease 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.

Cold Agglutinin Disease Emerging Drugs

BIVV020: Bioverativ BIVV020 is a humanized monoclonal antibody that targets complement C1s. A phase 1b clinical trial that was testing the safety and tolerability of the experimental drug BIVV020 in adult patients with cold agglutinin disease (CAD) has been completed. The primary objective of the trial was to assess the safety and tolerability of the treatment as measured by the number of participants with adverse events. Secondary objectives were the effect of the treatment on complement-mediated hemolysis, as well as its pharmacodynamics, pharmacokinetics, and immunogenicity. The drug is being developed by Bioverativ, a Sanoficompany.

Iptacopan: Novartis Pharmaceuticals Iptacopan is an oral investigational therapy by Novartis. It is a first-in-class, potent, and highly selective factor B inhibitor of the alternative complement pathway. It binds to factor B and suppresses the activity of C3 convertase. This way, signaling from the alternative complement pathway and the activation of the amplification loop are also suppressed. This inhibits the generation of the downstream C5 convertase complex, opsonization, and the formation of C3a and C5a anaphylatoxins and membrane attack complex. Because the direct classical and lectin pathway signaling remains intact, the risk of meningococcal infection is lower with Iptacopan compared to terminal complement pathway inhibitors. Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of Cold Agglutinin Disease.

Cold Agglutinin Disease: Therapeutic Assessment

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

Major Players in Cold Agglutinin Disease

There are approx. 5+ key companies which are developing the therapies for Cold Agglutinin Disease. The companies which have their Cold Agglutinin Disease drug candidates in the most advanced stage, i.e. phase II include, Novartis Pharmaceuticals.

Phases

This report covers around 5+ 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

Cold Agglutinin Disease 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.

Cold Agglutinin Disease: Pipeline Development Activities

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

Cold Agglutinin Disease Report Insights

• Cold Agglutinin Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Bioverativ
• Novartis Pharmaceuticals

Key Products

• BIVV020
• Iptacopan

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