Thrombotic Microangiopathy - Pipeline Insight, 2024

This “Thrombotic Microangiopathy- Pipeline Insight, 2024” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Thrombotic Microangiopathy 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

Thrombotic Microangiopathy: Understanding

Thrombotic Microangiopathy: Overview

Thrombotic microangiopathies (TMA) are clinical syndromes defined by the presence of hemolytic anemia (destruction of red blood cells), low platelets, and organ damage due to the formation of microscopic blood clots in capillaries and small arteries. The kidneys are commonly affected, although virtually any organ may be involved. Smoldering TMA will sometimes result in kidney damage without significant anemia or low platelets. Under the microscope, the blood demonstrates injured red blood cells known as schistocytes or fragments. Kidney disease can be severe, with over 50% of individuals requiring dialysis with a cause of TMA known as atypical hemolytic uremic syndrome (a HUS). Historically, TMA were often referred to as TTP/HUS, or thrombotic thrombocytopenic purpura/hemolytic uremic syndrome. It is now recognized that a large number of different diseases can result in TMA. TMA are associated with a large number of diseases. A common cause is thrombotic thrombocytopenic purpura (TTP) which is due to low activity of a protein called ADAMTS13. Some individuals are born with a mutation in the gene for ADAMTS13, although most affected patients have an acquired auto-antibody (a form of autoimmune disease) that blocks the activity of ADAMTS13. Another major cause for TMA is atypical hemolytic uremic syndrome (a HUS), a disorder caused by dysregulation of a part of the immune system known as complement. Approximately 50% of aHUS patients are found to have either a genetic mutation in the complement system or an auto-antibody that interferes with the regulation of complement. Other notable causes for TMA include infection (e.g., bloody diarrhea associated with E. coli infection), medications (e.g., quinine, bevacizumab), connective tissue diseases (e.g., systemic lupus erythematosus, antiphospholipid antibody syndrome, scleroderma), cancer, vasculitis, pregnancy, malignant hypertension, organ transplant, and metabolic disorders. TMA often present very suddenly and result in severe illness in many patients. Patients are often hospitalized at the time of diagnosis. Diagnosis requires blood tests to confirm red blood cell destruction, the presence of schistocytes on blood smears, and organ damage that can be attributed to the TMA. Typical organ damage includes very high blood pressure (malignant hypertension), kidney injury, abdominal pain, diarrhea, stroke, confusion, heart injury, and eye damage. Identifying the specific cause for TMA requires specialized blood and genetic testing to evaluate for the different causative diseases. Thrombotic Microangiopathy (often known simply as TMA) is a rare but seriousmedicaldisease.

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

Thrombotic Microangiopathy Emerging Drugs Chapters

This segment of the Thrombotic Microangiopathy 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.

Thrombotic Microangiopathy Emerging Drugs

Pegcetacoplan: Swedish Orphan Biovitrum Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across haematology, ophthalmology, nephrology, and neurology. Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of Transplant-associated thromboticmicroangiopathy.

Nomacopan: Akari Therapeutics Nomacopan is a recombinant small protein (16,740 Da) that acts on complement component-C5, preventing release of C5a and formation of C5b-9 (also known as the membrane attack complex, or MAC), and independently also inhibits leukotriene B4, or LTB4, activity, both elements that are co-located as part of the immune/inflammatory response.

Narsoplimab: Omeros Corporation Narsoplimab, also known as OMS721, is a fully human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system. The lectin pathway is activated primarily by tissue damage or microbial infection. Importantly, unlike other complement-targeting drugs on the market or in development, inhibition of MASP-2 by narsoplimab does not interfere with the classical complement pathway, a critical component of the acquired immune response to infection. Narsoplimab is designed to prevent complement-mediated inflammation and endothelial damage without affecting other pathways of innate immunity. In the US, the FDA has granted narsoplimab the following designations: (1) Breakthrough Therapy designation in patients who have persistent TMA despite modification of immunosuppressive therapy, (2) Orphan Drug designation for the prevention (inhibition) of complement-mediated TMAs and (3) Orphan Drug designation for the treatment ofHSCT-TMA.

Thrombotic Microangiopathy: Therapeutic Assessment

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

Major Players in Thrombotic Microangiopathy

There are approx. 15+ key companies which are developing the therapies for Thrombotic Microangiopathy. The companies which have their Thrombotic Microangiopathy drug candidates in the most advanced stage, i.e. Pre-Registration include, Omeros Corporation.

Phases

This report covers around 15+ 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

Thrombotic Microangiopathy 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.

Thrombotic Microangiopathy: Pipeline Development Activities

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

Thrombotic Microangiopathy Report Insights

• Thrombotic Microangiopathy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Swedish Orphan Biovitrum
• Akari Therapeutics
• Omeros Corporation
• Novartis Pharmaceuticals
• Takeda
• UCB
• Harbour BioMed

Key Products

• Pegcetacoplan
• Nomacopan
• Narsoplimab
• Iptacopan
• BAX930
• UCB7665
• ADAMTS13

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