DelveInsight’s, “Immune Thrombocytopenia – Pipeline Insight, 2020,” report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Immune Thrombocytopenia 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
Immune Thrombocytopenia Understanding
Immune Thrombocytopenia: Overview
Immune thrombocytopenia or ITP is a bleeding disorder. In ITP, the blood doesn't clot as it should. This is due to a low number of blood cell fragments called platelets or thrombocytes. People who have ITP often have purple bruises called purpura. These bruises appear on the skin or mucous membranes. Bleeding from small blood vessels under the skin causes purpura. People who have ITP also may have bleeding that causes tiny red or purple dots on the skin. These pinpoint-sized dots are called petechiae. Petechiae may look like a rash. The two types of ITP are acute (temporary or short-term) and chronic (long-lasting).
• Acute ITP generally lasts less than 6 months. It mainly occurs in children—both boys and girls—and is the most common type of ITP. Acute ITP often occurs after a viral infection.
• Chronic ITP lasts 6 months or longer and mostly affects adults. However, some teenagers and children do get this type of ITP. Chronic ITP affects women two to three times more often than men.
Symptoms
Immune thrombocytopenia (ITP) may not cause any signs or symptoms. However, ITP can cause bleeding inside the body (internal bleeding) or underneath or from the skin (external bleeding). Signs of bleeding may include:
• Bruising or purplish areas on the skin or mucous membranes (such as in the mouth). These bruises are called purpura. They're caused by bleeding under the skin, and they may occur for no known reason.
• Pinpoint red spots on the skin called petechiae. These spots often are found in groups and may look like a rash. Bleeding under the skin causes petechiae.
• A collection of clotted or partially clotted blood under the skin that looks or feels like a lump. This is called a hematoma.
• Nosebleeds or bleeding from the gums (for example, during dental work).
• Blood in the urine or stool (bowel movement).
Diagnosis
The doctor will diagnose immune thrombocytopenia (ITP) based on medical history, a physical exam, and test results. The doctor will want to make sure that your low platelet count isn't due to another condition (such as an infection) or medicines you're taking (such as chemotherapy medicines or aspirin).
• A complete blood count: This test checks the number of red blood cells, white blood cells, and platelets in your blood. In ITP, the red and white blood cell counts are normal, but the platelet count is low.
• A blood smear: For this test, some of your blood is put on a slide. A microscope is used to look at your platelets and other blood cells.
Treatment
Treatment for immune thrombocytopenia (ITP) is based on how much and how often the patient is bleeding and platelet count. Adults who have mild ITP may not need any treatment, other than watching their symptoms and platelet counts. Adults who have ITP with very low platelet counts or bleeding problems often are treated. The acute (short-term) type of ITP that occurs in children often goes away within a few weeks or months. Children who have bleeding symptoms, other than merely bruising (purpura), usually are treated. Corticosteroids, such as prednisone, are commonly used to treat ITP. These medicines, called steroids for short, help increase platelet count. However, steroids have many side effects. Some people relapse (get worse) when treatment ends. Other medicines also are used to raise the platelet count. Some are given through a needle inserted into a vein. These medicines include rituximab, immune globulin, and anti-Rh (D) immunoglobulin. If needed, doctors can surgically remove the spleen. If ITP hasn't responded to medicines, removing the spleen will reduce the destruction of platelets. However, it also may raise your risk for infections.
Immune Thrombocytopenia Emerging Drugs Chapters
This segment of the Immune Thrombocytopenia 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.
Immune Thrombocytopenia Emerging Drugs
• Efgartigimod: Argenx
Efgartigimod is designed as a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Efgartigimod is being evaluated for the treatment of patients with severe autoimmune diseases with confirmed presence of pathogenic immunoglobulin G, IgG autoantibodies, where a severe unmet medical need exists. It is currently in phase III stage of development.
• Rozanolixizumab: UCB pharma
Rozanolixizumab is a subcutaneously administered, humanized monoclonal antibody that specifically binds, with high affinity, to human FcRn. It has been designed to block the interaction of FcRn and IgG, inhibiting IgG recycling and inducing the removal of pathogenic IgG autoantibodies. It is currently in phase III stage of development.
• TAK-079: Takeda
TAK-079 is being tested to treat people who have primary immune thrombocytopenia (ITP). This study will evaluate the safety and biologic activity of TAK-079 or matching placebo in combination with stable ITP background therapy. It is currently in phase II stage of development.
• HL161: HanAll Biopharma
HL161 is a fully human monoclonal antibody targeting neonatal Fc receptor (FcRn). The FcRn rescues IgGs from intracellular degradation and maintains the high serum levels, even pathogenic auto-IgGs. Blocking the FcRn-IgG interaction accelerates the degradation of autoantibodies and alleviates the flare-up in wide array of pathogenic IgG-mediated autoimmune diseases such as myasthenia gravis, pemphigus, immune thrombocytopenia purpura, neuromyelitis optica, and etc. HanAll discovered HL161 by using transgenic animal producing fully human monoclonal antibodies. The company is currently conducting phase I trial.
Further product details are provided in the report……..
Immune Thrombocytopenia: Therapeutic Assessment
This segment of the report provides insights about the different Immune Thrombocytopenia drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Immune Thrombocytopenia
There are approx. 50+ key companies which are developing the therapies for Immune Thrombocytopenia. The companies which have their Immune Thrombocytopenia drug candidates in the most advanced stage, i.e. phase III include Argenx, UCB Pharma and others
• Phases
DelveInsight’s report covers around 50+ products under different phases of clinical development like
• Late-stage products (Phase II and Phase II/III)
• Mid-stage products (Phase II and Phase II/III)
• Early-stage products (Phase I/II and Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

• Route of Administration
Immune Thrombocytopenia 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
• 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.

Immune Thrombocytopenia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Immune Thrombocytopenia 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 Immune Thrombocytopenia drugs.

Report Highlights
• The companies and academics are working to assess challenges and seek opportunities that could influence Immune Thrombocytopenia R&D. The therapies under development are focused on novel approaches to treat/improve Immune Thrombocytopenia.
• Rozanolixizumab, an investigational monoclonal antibody, was granted orphan drug designation for the treatment of ITP by the US Food and Drug Administration on 30 April 2018 and by the European Commission on 11 January 2019.
• In September 2017, HanAll Biopharma and Harbour BioMed announced that they have entered into a strategic collaboration and license agreement to develop, manufacture and commercialize in Greater China (including Hong Kong, Macau and Taiwan) HanAll's two novel biologics, the anti-FcRn monoclonal antibody, HL161, for the treatment of pathogenic IgG-mediated autoimmune diseases’
Immune Thrombocytopenia Report Insights
• Immune Thrombocytopenia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs
Immune Thrombocytopenia 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 Immune Thrombocytopenia drugs?
• How many Immune Thrombocytopenia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Immune Thrombocytopenia?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Immune Thrombocytopenia 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 Immune Thrombocytopenia and their status?
• What are the key designations that have been granted to the emerging drugs?
Key Players
• Argenx
• HanAll Biopharma
• Eris Lifesciences
• Momenta Pharmaceuticals
• Taiho Pharmaceutical
• Takeda
• UCB Pharma
• Immunomedics
• Oscotech
• Bioverativ, a Sanofi company
• Novartis
• Pfizer
• Kissei Pharmaceutical Co., Ltd.

Key Products
• Efgartigimod
• Rozanolixizumab
• TAK-079
• HL161
• Romiplostim biosimilar
• R788
• M254
• Eltrombopag
• PRN1008
• HMPL-523
• SKI-O-703
• HBM9161
• BIVV009