Primary myelofibrosis - Pipeline Insight, 2024

This “Primary myelofibrosis- Pipeline Insight, 2024” report provides comprehensive insights about 55+ companies and 55+ pipeline drugs in Primary myelofibrosis 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

Primary myelofibrosis: Understanding

Primary myelofibrosis: Overview

Primary myelofibrosis (also called chronic idiopathic myelofibrosis, agnogenic myeloid metaplasia) is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialized cells in the bone marrow known as fibroblasts make these fibres. In primary myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result. Primary myelofibrosis is a rare chronic disorder diagnosed in an estimated 1 per 100,000 population. It can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of primary myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).Long-term exposure to high levels of benzene or very high doses of ionizing radiation may increase the risk of primary myelofibrosis in a small number of cases. Around 20 per cent of people have no symptoms of primary myelofibrosis when they are first diagnosed and the disorder is picked up incidentally as a result of a routine blood test. For others, symptoms develop gradually over time. Symptoms of anaemia are common and include unexplained tiredness, weakness, shortness of breath and palpitations. Other nonspecific symptoms include fever, unintended weight loss, pruritus (generalized itching) and excess sweating, especially at night. Virtually all patients with primary myelofibrosis have an enlarged spleen (splenomegaly) when they are first diagnosed. In around a third of cases the spleen is very enlarged. Common symptoms include feelings of discomfort, pain or fullness in the upper left-side of the abdomen. An enlarged spleen may also cause pressure on your stomach causing a feeling of fullness, indigestion and a loss of appetite. Abdominal discomfort can also result from an enlarged liver (hepatomegaly), which occurs in around two-thirds of cases. Other less common symptoms include bone and joint pain, and bleeding problems. Primary myelofibrosis is diagnosed using a combination of a physical examination showing the presence of an enlarged spleen, blood tests and a bone marrow examination. Primary myelofibrosis is generally regarded as an incurable disease but with treatment many people can remain comfortable and symptom-free for some time. The natural course of the disease can vary considerably between individuals. In some people their disease remains stable for long periods and they are free to live a normal life with minimal interruptions from the disease or its treatment. For others, myelofibrosis progresses more quickly and people require treatment to help relieve symptoms of their disease.

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

Primary myelofibrosis Emerging Drugs Chapters

This segment of the Primary myelofibrosis 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.

Primary myelofibrosis Emerging Drugs

NS 018: Nippon Shinyaku Ilginatinib (formerly NS 018), a potent inhibitor of Janus kinase 2 (JAK2) enzyme activity and Src-family kinases is being developed by Nippon Shinyaku.Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of PrimaryMyelofibrosis.

KRT-232: Kartos Therapeutics Navtemadlin (KRT-232), is a novel, potent and selective oral MDM2 inhibitor. The MDM2-p53 interaction represents a compelling therapeutic target with potential to provide a new treatment option for patients with Myelofibrosis (MF), Polycythemia Vera (PV), Acute Myeloid Leukemia (AML), and Merkel Cell Carcinoma (MCC).Currently, the drug is in Phase II/III stage of Clinical trial evaluation for the treatment of Primary Myelofibrosis.

TP 3654: Sumitomo Pharma Oncology TP-3654 is an oral investigational inhibitor of PIM kinases, which has potential antitumor and anti-fibrotic effects through multiple pathways, including induction of apoptosis. Currently, the drug is in Phase I/II stage of Clinical trial evaluation for the treatment of PrimaryMyelofibrosis.

Primary myelofibrosis: Therapeutic Assessment

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

Major Players in Primary myelofibrosis

There are approx. 55+ key companies which are developing the therapies for Primary myelofibrosis. The companies which have their Primary myelofibrosis drug candidates in the most advanced stage, i.e. Phase II/III include, Kartos Therapeutics.

Phases

This report covers around 55+ 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

Primary myelofibrosis 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.

Primary myelofibrosis: Pipeline Development Activities

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

Primary myelofibrosis Report Insights

• Primary myelofibrosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

c Primary myelofibrosis ReportAssessment

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

Key Players

• Nippon Shinyaku
• Kartos Therapeutics
• Sumitomo Pharma Oncology

Key Products

• NS 018
• KRT-232
• TP 3654

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