DelveInsight’s, “Essential Thrombocythemia (ET) – Pipeline Insight, 2020,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Essential Thrombocythemia 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
Essential Thrombocythemia Understanding
Essential Thrombocythemia (ET): Overview
Essential thrombocythemia, also known as ET, is a rare disease. The most important first fact about Essential thrombocythemia: on average, people with Essential thrombocythemia have a normal life expectancy. Patients with Essential thrombocythemia have increased numbers of platelets. Platelets are the smallest of the three types of blood cells and are needed for successful blood clotting after an injury. The two other types of blood cells are red blood cells, which carry oxygen to all tissues in the body, and white blood cells, which help to fight infections. Red blood cell numbers (often measured as a percentage of whole blood, called a hematocrit) are generally normal in Essential thrombocythemia, while white blood cell numbers are normal or slightly elevated in Essential thrombocythemia. Importantly, most people with an elevated platelet count do not have Essential thrombocythemia. Common alternative causes of an elevated platelet count are iron deficiency, infection or generalized inflammation; less common causes are blood disorders such as Essential thrombocythemia or other related blood diseases.
Symptoms
Abnormal blood clotting (thrombosis) is common in people with essential thrombocythemia and causes many signs and symptoms of this condition. Clots that block blood flow to the brain can cause strokes or temporary stroke-like episodes known as transient ischemic attacks. Thrombosis in the legs can cause leg pain, swelling, or both. In addition, clots can travel to the lungs (pulmonary embolism), blocking blood flow in the lungs and causing chest pain and difficulty breathing (dyspnea). Another problem in essential thrombocythemia is abnormal bleeding, which occurs more often in people with a very high number of platelets. Affected people may have nosebleeds, bleeding gums, or bleeding in the gastrointestinal tract.
Diagnosis
• CBC and peripheral blood smear
• Exclusion of causes of secondary thrombocytosis
• Cytogenetic studies
• JAK2 mutation by polymerase chain reaction, and, if negative, CALR or MPL mutation analysis
• Possibly a bone marrow examination
Treatment
• Aspirin
• Platelet-lowering drugs (eg, hydroxyurea, anagrelide)
• Rarely plateletpheresis
• Rarely cytotoxic agents
• Rarely interferon
• Rarely stem cell transplantation
For mild vasomotor symptoms (eg, headache, mild digital ischemia, erythromelalgia) and to decrease the risk of thrombosis in low-risk patients, aspirin 81 mg po once/day is usually sufficient, but a higher dose may be used if necessary.
Essential Thrombocythemia Emerging Drugs Chapters
This segment of the Essential Thrombocythemia 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.
Essential Thrombocythemia Emerging Drugs
• Ropeginterferon alfa-2b: PharmaEssentia
Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). Its pharmacokinetic properties offer a new level of tolerability. Ropeginterferon alfa-2b is designed to be self-administered subcutaneously with a pen once every two weeks, or monthly during long-term maintenance. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons. PharmaEssentia Corporation is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of ET.
Available clinical data and experience with P1101 in PV shows that the compound, with proper dose modifications, is effective in controlling disease in a significant proportion of subjects with ET. Further, its increased serum half-life presents distinct advantages for ET treatment over that of standard IFN-α and other available PEG IFN-α therapy. This pivotal Phase 3 study will establish the efficacy and safety of P1101 in ET subjects.
• IMG-7289: Imago BioSciences, Inc.
Bomedemstat is a small molecule developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A). Bomedemstat (IMG-7289) is an investigational agent currently being evaluated in ongoing clinical trials. Bomedemstat has FDA Orphan Drug and Fast Track Designation for the treatment of essential thrombocythemia.
Further product details are provided in the report……..
Essential Thrombocythemia: Therapeutic Assessment
This segment of the report provides insights about the different Essential Thrombocythemia drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Essential Thrombocythemia
There are approx. 10+ key companies which are developing the therapies for Essential Thrombocythemia. The companies which have their Essential Thrombocythemia drug candidates in the mid to advanced stage, i.e. phase III and Phase II include, PharmaEssentia, Imago BioSciences, NS Pharma, Novartis, CTI BioPharma, Celgene, Samus Therapeutics etc.
Phases
DelveInsight’s report covers around 10+ products under different phases of clinical development like
• Mid-stage products (Phase II and Phase I/II)
• 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
Essential Thrombocythemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
• Subcutaneous
• Intravenous
• Oral
• Intramuscular
• Molecule Type
Products have been categorized under various Molecule types such as
• Small molecules
• Pyridines
• Pyrazoles
• Polyethylene glycols
• Interferons

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

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

Report Highlights
• The companies and academics are working to assess challenges and seek opportunities that could influence Essential Thrombocythemia R&D. The therapies under development are focused on novel approaches to treat/improve Essential Thrombocythemia.
Essential Thrombocythemia Report Insights
• Essential Thrombocythemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs
Essential Thrombocythemia 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 Essential Thrombocythemia drugs?
• How many Essential Thrombocythemia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Essential Thrombocythemia?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Essential Thrombocythemia 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 Essential Thrombocythemia and their status?
• What are the key designations that have been granted to the emerging drugs?
Key Players
• PharmaEssentia
• Imago BioSciences,Inc.
• NS Pharma, Inc.
• Novartis
• Celgene
Key Products
• Ropeginterferon alfa-2b
• Bomedemstat
• Ruxolitinib