Blastic Plasmacytoid Dendritic cell neoplasm - Pipeline Insight, 2024

This “Blastic Plasmacytoid Dendritic cell neoplasm - Pipeline Insight, 2024” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Blastic Plasmacytoid Dendritic cell neoplasm 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

Blastic Plasmacytoid Dendritic cell neoplasm: Understanding

Blastic Plasmacytoid Dendritic cell neoplasm: Overview

Blastic plasmacytoid dendritic cell neoplasm is a rare entity grouped with the acute myeloid leukemia-related precursor neoplasms in the 2008 World Health Organization classification. It was previously postulated to originate from natural killer cells, T cells, or monocytes but is now believed to arise from the plasmacytoid dendritic cell. The pathogenesis of blastic plasmacytoid dendritic cell neoplasm is not well understood, although the neoplasm demonstrates frequent deletion of tumor suppressor genes, including RB1, CDKN1B, CDKN2A, and TP53. Blastic plasmacytoid dendritic cell neoplasm is a clinically aggressive tumor that often initially presents as cutaneous lesions and subsequently progresses to bone marrow involvement and leukemic dissemination. It is characterized by enhanced expression of CD56, CD4, and CD123, which can be detected by flow cytometry/immunohistochemistry. The differential diagnoses include myeloid sarcoma/acute myeloid leukemia, T-cell lymphoblastic leukemia/lymphoma, NK-cell lymphoma/leukemia, and some mature T-cell lymphomas/leukemias. Patients usually respond to initial chemotherapy but often relapse. Stem cell transplant may improve survival. Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive hematopoietic malignancy derived from the precursors of plasmacytoid dendritic cells. This neoplasm is rare and has only recently been recognized as a distinct neoplastic entity. In the past, it was referred to by several names, including blastic NK-cell lymphoma, agranular CD4+ natural killer cell leukemia, and agranular CD4+CD56+ hematodermic neoplasm, among others. Currently, BPDCN is categorized under “acute myeloid leukemia (AML) and related precursor neoplasms” in the 2008 World Health Organization Classification of Tumours of Haematopoietic and Lymphoid Tissues. Patients typically present with asymptomatic, solitary or multiple skin lesions, such as nodules, plaques, or bruiselike lesions that can range in size from a few millimeters to 10 cm. The skin lesions can be associated with erythema, hyperpigmentation, purpura, or ulceration. Extracutaneous disease is present in most patients at diagnosis, often involving the regional lymph nodes. As the disease continues to progress, the peripheral blood and bone marrow become involved.

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

Blastic Plasmacytoid Dendritic cell neoplasm Emerging Drugs Chapters

This segment of the Blastic Plasmacytoid Dendritic cell neoplasm 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.

Blastic Plasmacytoid Dendritic cell neoplasm Emerging Drugs

Venetoclax : Genentech Venetoclax (RG7601) is a novel small molecule Bcl-2 selective inhibitor designed to restore apoptosis, also known as programmed cell death, by blocking the function of a pro-survival Bcl-2 family protein. The Bcl-2 family proteins, which are expressed at high levels in many tumors, play a central role in regulating apoptosis. This molecule is being developed in collaboration with AbbVie.Currently, the drug is being developed in Phase 1 for the treatment of Blastic Plasmacytoid Dendritic CellNeoplasm.

Pivekimab sunirine : ImmunoGen IMGN632 is a CD123-targeting ADC in clinical development for hematological malignancies, including blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML). IMGN632 uses one of ImmunoGen’s (the “Company”) novel indolinobenzodiazepine (IGN) payloads, which alkylate DNA without crosslinking. IGNs have been designed to have high potency against tumor cells, while demonstrating less toxicity to normal marrow progenitors than other DNA-targeting payloads. The European Medicines Agency (EMA) granted orphan drug designation to IMGN632 for the treatment of BPDCN in June 2020; IMGN632 also holds this designation in the US. In October 2020, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for IMGN632 for the treatment of patients with relapsed or refractory BPDCN. In December 2020, the Company announced that it aligned with FDA on a path to full approval for IMGN632, with an amendment to the ongoing 801 Phase 1/2 study to add a new cohort of up to 20 frontline BPDCN patients, with CR/CRc rate as the primaryendpoint.

MB 102 : Mustang Bio MB102 (CD123) is over expressed in 75-89% of AML patients and over 90% in Blastic plasmacytoid dendritic cell neoplasm (BPDCN) patients. There were an estimated 19,520 new U.S. cases of AML in 2018 with an estimated 25% 5-year survival rate. BPDCN is an ultra-rare disease and aggressive blood cancer with 500-1,000 patients per year in the U.S. and a median overall survival of 9-12 months. MB-102 has shown promising response rates in early small populations of these patients in a physician IND study out of City ofHope.

Blastic Plasmacytoid Dendritic cell neoplasm: Therapeutic Assessment

This segment of the report provides insights about the different Blastic Plasmacytoid Dendritic cell neoplasm drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Blastic Plasmacytoid Dendritic Cell Neoplasm

There are approx. 3+ key companies which are developing the therapies for Blastic Plasmacytoid Dendritic Cell Neoplasm. The companies which have their Blastic Plasmacytoid Dendritic cell neoplasm drug candidates in the most advanced stage, i.e. phase I/ II include, Mustang Bio.

Phases

This report covers around 3+ 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

Blastic Plasmacytoid Dendritic cell neoplasm 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.

Blastic Plasmacytoid Dendritic cell neoplasm: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Blastic Plasmacytoid Dendritic cell neoplasm 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 Blastic Plasmacytoid Dendritic cell neoplasm drugs.

Blastic Plasmacytoid Dendritic cell neoplasm Report Insights

• Blastic Plasmacytoid Dendritic cell neoplasm Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• AbbVie
• ImmunoGen
• Mustang Bio
• Genentech

Key Products

• Venetoclax
• Pivekimab sunirine
• MB 102
• Azacitidine

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