Thalassemia- Pipeline Insight, 2025

This “Thalassemia- Pipeline Insight, 2025,”report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in 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.

Thalassemia Understanding

Thalassemia: Overview

Thalassemia is a group of inherited blood disorders that affect the body’s ability to produce hemoglobin and red blood cells. Thalassemia is a blood disorder that is inherited. This means it is passed down from one or both parents through their genes. When a person has thalassemia, the body makes less hemoglobin than normal. Hemoglobin is an iron-rich protein in red blood cells. It carries oxygen to all parts of the body. There are 2 main types of thalassemia: alpha and beta. Different genes are affected for each type. Thalassemia can cause mild or severe anemia. Anemia occurs when the body does not have enough red blood cells or hemoglobin. The severity and type of anemia depends on how many genes are affected.

Alpha thalassemia occurs when some or all of the 4 genes that make hemoglobin (the alpha-globin genes) are missing or damaged. There are 4 types of alpha thalassemia: Alpha thalassemia silent carrier: One gene is missing or damaged, and the other 3 are normal. Alpha thalassemia carrier: Two genes are missing. Hemoglobin H disease: Three genes are missing. This leaves just 1 working gene. Alpha thalassemia major. All 4 genes are missing. This causes severe anemia. In most cases, a baby with this condition will die before birth. This is a genetic disease inherited from one or both parents.

The only risk factor is having a family history of the disease. Beta thalassemia is caused by damaged or missing genes. Two specific genes are involved. There are several types of this disorder: Beta thalassemia major (Cooley’s anemia). There are two damaged genes. This is the most severe form of this disorder. Beta thalassemia minor or thalassemia trait. Only one gene is damaged. This causes less severe anemia. People with this type have a 50% chance of passing the gene to their children. If the other parent is not affected, their children will also have this form of the disorder. Many people with this disorder are given iron replacement by mistake. This happens when a lack of iron is believed to cause their anemia. Too much iron can be harmful. So it is important to get the right diagnosis.

Thalassemia Symptoms include: Slow growth in children, wide or brittle bones, enlarged spleen (an organ in your abdomen that filters blood and fights disease), fatigue, weakness, Pale or yellow skin, dark urine and poor appetite. The following tests may help to tell if the person is a carrier, and can pass the disorder on to children: Complete blood count (CBC). This test checks the size, number, and maturity of different blood cells in a set volume of blood, Hemoglobin electrophoresis with A2 and F quantitation. A lab test that tells what type of hemoglobin is present, FEP (free-erythrocyte protoporphyrin) and ferritin. This test is done to rule out iron-deficiency anemia. All of these tests can be done using a single blood sample. In a pregnant woman, the baby is diagnosed using CVS (chorionic villus sampling) or amniocentesis. A DNA test is needed to make a diagnosis of alpha thalassemia. There is no cure. Treatment can reduce symptoms.

'Thalassemia- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Thalassemia pipeline landscape is provided which includes the disease overview and Thalassemia treatment guidelines. The assessment part of the report embraces, in depth 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, 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 Thalassemia R&D. The therapies under development are focused on novel approaches to treat/improve Thalassemia.

Thalassemia Emerging Drugs Chapters

This segment of the Thalassemia 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.

Thalassemia Emerging Drugs

Mitapivat: Agios Pharmaceuticals, Inc

Mitapivat is the potential drug candidate which is a quinolone sulfonamide administered through oral route as a capsule and tablet. It targets pyruvate kinase receptor (PKR). In June 2024, Agios Pharmaceuticals, Inc. presented detailed results from the global Phase III ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia. The ENERGIZE study achieved its primary endpoint, with mitapivat demonstrating a statistically significant increase in hemoglobin response rate compared to placebo. Statistical significance was also achieved for both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration. These improvements were observed across all pre-specified subgroups. Currently it is in Phase III stage of development for the treatment of Thalassemia.

Vamifeport: CSL Vifor

Vamifeport is an oral inhibitor of ferroportin, an enzyme essential for the body’s transport of iron and plays a key role in regulating iron uptake and distribution in the body. Vamifeport binds to ferroportin and blocks it to prevent excessive iron release into the blood. As iron is needed for the formation of hemoglobin, this relative decrease in iron availability is expected to reduce the concentration of abnormal hemoglobin in red blood cells and prevent ensuing unfavorable events. Vamifeport-mediated ferroportin inhibition may lead to less haemolysis, decrease of inflammation and better blood flow with consequent improvement of both the symptoms and clinical outcomes of SCD. Vamifeport is currently also in phase II development for beta-thalassemia, an inherited rare blood disorder that reduces the production of functional hemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body and potentially cause anemia.

BRL-101: BRL MEDICINE

BLR-101 is a type of hematopoietic stem cell therapy based on

HBB gene stimulants (Hemoglobin beta chain gene stimulants) mechanism of action that targets

β-globin (Hemoglobin beta chain) developed for the treatment of patients suffering with thalassemia. On August 16, 2022, the clinical trial application (IND) of BRL Medicine's BRL-101 was officially approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, entering the registration clinical stage. The results of the IIT and IND Phase 1 clinical studies of BRL-101 indicated a significant increase in overall Hb and HbF levels after all patients received gene-edited HSC transplantation. Throughout the treatment process, most adverse events were consistent with known adverse events for hematopoietic stem cell mobilization/apheresis, busulfan myeloablative conditioning, and autologous hematopoietic stem cell transplantation. Currently it is in Phase I/II stage of development for the treatment of Thalassemia.

Thalassemia: Therapeutic Assessment

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

Major Players in Thalassemia

There are approx. 30+ key companies which are developing the therapies for Thalassemia. The companies which have their Thalassemia drug candidates in the most advanced stage, i.e. phase III include, Agios Pharmaceuticals, Inc

Phases

Delve Insight’s report covers around 30+ products under different phases of clinical development like

• Late stage products (Phase II)
• 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

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.

Thalassemia: Pipeline Development Activities

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

Thalassemia Report Insights

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

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

Key Players

• Agios Pharmaceuticals, Inc
• Vifor Pharma Group
• BRL MEDICINE
• Sangamo Therapeutics
• Vifor (International) Inc
• Celgene
• Pharmacosmos A/S
• Bioray Laboratories
• DisperSol Technologies, LLC
• CorrectSequence Therapeutics
• Editas Medicine

Key Products

• Mitapivat
• Vamifeport
• BRL-101
• ST-400
• VIT-2763
• Luspatercept
• SP-420
• BRL-101
• DST-0509
• CS-101
• Renizgamglogene autogedtemcel

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