Alpha Thalassemia - Pipeline Insight, 2024

This “Alpha Thalassemia - Pipeline Insight, 2024,” report provides comprehensive insights about 4+ companies and 5+ pipeline drugs in Alpha Thalassemia 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

Alpha Thalassemia Understanding

Alpha Thalassemia: Overview

Thalassemias are a group of disorders caused by abnormal production of globin chains. The production can be diminished or can be absent for one or more of the globin chains. This imbalance of globin chain production impairs the production of normal hemoglobin. This impairment causes ineffective erythropoiesis with intramedullary hemolysis. Alpha thalassemia also known as Hemoglobin H Disease (HbH) refers specifically to the abnormal or absent manufacturing of alpha-globin chains. These are associated with more than 15 different genetic mutations. The severity of the clinical condition is based on the mutation type. The severity of mutation is based on which of the two alpha-globin loci is affected. Mutations can also be deletion or non-deletion. In deletion mutation, there is an inheritance of a single alpha-globin gene. With the non-deletion type, a patient has inherited two alpha-globin genes, but one gene carries a non-deletion abnormality, for example, point mutation. In non-deletion, the severity of clinical expression is also affected depending on whether the mutation blocks the production of the remaining normal alpha chains partially or fully. Hemoglobin H disease occurs when only one normal alpha gene has been inherited. One of these most common non-deletion subtypes of Hemoglobin H is called Hemoglobin Constant Spring. HbH disease tends to be more severe in patients with the non-deletion-type likely due to interference with the transcription of the normal alpha chain gene by the abnormal one.

Hemoglobin H can cause chronic hypochromic microcytic anemia and hemolytic anemia, which can worsen in periods of oxidant stress. This can be effectively broken down as ineffective erythropoiesis and increased hemolysis. The microcytic hypochromic anemia is due to impaired hemoglobin production due to decreased alpha chain synthesis and hyperhydration of the cell. The cause of the hyperhydration in alpha thalassemia is not clear. One theory argues that the K-Cl cotransporter stops early, thereby preventing the usual loss of K-Cl and water that is part of the red blood cell remodeling process.

The management of alpha thalassemia is ideally done by an interprofessional team that consists of primary care providers, hematologists, geneticists, nurses, pharmacists, and dietitians. This hemoglobinopathy can affect multiple organs, and close monitoring is required. Supplementation with folic acid should be given because there is hemolytic anemia. Patients with HbH are at risk of clinical manifestations with oxidative damage. Blood counts should be monitored, and transfusional intervention may be required during periods of oxidant stress, such as infection or the use of oxidant drugs.

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

Alpha Thalassemia Emerging Drugs Chapters

This segment of the Alpha Thalassemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.

Alpha Thalassemia Emerging Drugs

Mitapivat: Agios Pharmaceuticals Mitapivat is a pyruvate kinase activator. It works to increase the activity of erythrocyte pyruvate kinase, a key enzyme involved in the survival of red blood cells. Defects in the pyruvate kinase enzyme in various red blood cells disorders lead to the lack of energy production for red blood cells, leading to lifelong premature destruction of red blood cells or chronic hemolytic anemia.

The pyruvate kinase enzyme is an ATP-generating enzyme involved in the Embden-Meyerhof glycolytic pathway: it catalyzes the conversion of phosphoenolpyruvate to pyruvate in the final step of glycolysis, generating adenosine triphosphate (ATP), which is critical for cellular maintenance and survival. One of the four isoforms of pyruvate kinase - erythrocyte pyruvate kinase or PKR - is dedicated to red blood cells (RBCs). Compared to most human cells, RBCs lack the metabolic machinery required for aerobic metabolism of glucose and generation of ATP; thus, they rely on anaerobic glycolysis for ATP production. The deficiency of ATP due to glycolytic enzyme defects leads to shortened lifespan and premature destruction of RBCs in the form of chronic hemolytic anemia and ineffective erythropoiesis. Upon binding to pyruvate kinase, Mitapivat stabilizes the active tetrameric form of the enzyme and enhances its affinity for its substrate, phosphoenolpyruvate. Mitapivat upregulates erythrocyte pyruvate kinase activity, increases ATP production, and reduces levels of 2,3-DPG. The Drug is currently in Phase III stage of its clinical development for the treatment of Alpha Thalassemia.

Alpha Thalassemia: Therapeutic Assessment

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

Major Players in Alpha Thalassemia

There are approx. 4+ key companies which are developing the therapies for Alpha Thalassemia. The companies which have their Alpha Thalassemia drug candidates in the most advanced stage, i.e. phase III include, Agios 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

Alpha Thalassemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• intravenous
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Alpha Thalassemia: Pipeline Development Activities

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

Alpha Thalassemia Report Insights

• Alpha Thalassemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Agios Pharmaceuticals
• Silence Therapeutics plc
• Bristol-Myers Squibb
• Novo Nordisk

Key Products

• Mitapivat
• Etavopivat
• Luspatercept
• SLN124

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