Waldenstrom's Macroglobulinemia - Pipeline Insight, 2024

This “Waldenstrom’s Macroglobulinemia - Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 22+ pipeline drugs in Waldenstrom’s Macroglobulinemia 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

Waldenstrom’s Macroglobulinemia: Understanding

Waldenstrom’s Macroglobulinemia: Overview

Waldenstrom Macroglobulinemia (WM) is a lymphoproliferative disorder characterized by the proliferation of lymphoplasmacytic elements in the bone marrow and the presence of monoclonal immunoglobulin M (IgM) gammopathy. The World Health Organization (WHO) classification defined WM as lymphoplasmacytic lymphoma (LPL) screening IgM proteins, belonging to the category of Non-Hodgkin B Lymphomas (NHL) with indolent course. The disease is rare, representing approximately 2% of all cases of non-Hodgkin Lymphoma, and presents distinctive clinical and laboratory features related to the presence of the monoclonal IgM.

The malignant cells in lymphoplasmacytic lymphoma are believed to originate from cells at a late stage of B-cell differentiation. These cells derive from a B-cell arrest after somatic hypermutation in the germinal center and before terminal differentiation to a plasma cell. The pathological findings in lymphoplasmacytic lymphoma are due to infiltration of the bone marrow with small lymphocytes and IgM monoclonal gammopathy. Visual and neurological symptoms are related to hyperviscosity and sluggishness of blood flow. Bleeding encountered in Waldenstrom macroglobulinemia is due to IgM binding to the clotting factors. Cryoglobulinemia in Waldenstrom macroglobulinemia patients leads to Raynaud phenomena and cold urticaria.

The diagnosis of lymphoplasmacytic lymphoma/Waldenstrom macroglobulinemia is usually challenging due to the lack of specific morphologic, immunophenotypic, or chromosomal changes. This lack makes the differentiation of this disease entity from other small B cell lymphomas based on exclusion. As a rule, the diagnosis of lymphoplasmacytic lymphoma should be considered in elderly individuals with unexplained weakness, bleeding, neurological deficits, neuropathies, and visual difficulties.

Treatment of Waldenstrom macroglobulinemia depends on the extent of end-organ involvement and symptoms. Asymptomatic patients are managed by close follow-up every 1 to 2 months including serial IgM measurements and assessment of serum viscosity. Symptomatic patients are managed by chemotherapy, immunotherapy, and/or an autologous bone marrow stem cell transplant. Chemotherapeutic agents that are effective in managing Waldenstrom macroglobulinemia include dexamethasone, fludarabine, bortezomib, and cyclophosphamide. A bone marrow transplant is usually reserved for younger patients with more extensive bone marrow involvement. Treatment algorithms for Waldenstrom macroglobulinemia recommend rituximab and bendamustine as a first-line treatment. Several therapeutic novelties have radically changed WM scenario during the last years. Furthermore, the discoveries of two mutations, myeloid differentiation primary response 88 (MYD88) and C-X-C chemokine receptor type 4 (CXCR4) in WM patients has improved disease characterisation helping to deeper understand the biology of the disease.

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

Waldenstrom’s Macroglobulinemia Emerging Drugs Chapters

This segment of the Waldenstrom’s Macroglobulinemia 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.

Waldenstrom’s Macroglobulinemia Emerging Drugs

Lisaftoclax: Ascentage Pharma Lisaftoclax is a highly selective Bcl-2 inhibitor that is capable of inducing apoptosis and suppressing cell growth. In the global Phase Ib/II study, lisaftoclax monotherapy showed encouraging antitumor activity and satisfactory safety profile in patients with relapsed/refractory (R/R) WM who had received prior treatment with BTKis. In addition, a response rate of 100% was achieved when in combination with BTKi for patients with treatment-naïve WM. Currently, the drug is in Phase II stage of development to treat Waldenstrom’sMacroglobulinemia.

ABT199: Abbvie ABT-199 (venetoclax), which is a Bcl-2 inhibitor. Bcl-2 is an apoptosis regulator expressed in high levels in leukemia cells, and it is responsible for the prolonged survival of these cells. Venetoclax will block and modulate the activity of Bcl-2. The clinical trials for ABT199 are being evaluated in phase II stage of development for the treatment of Waldenstrom’s Macroglobulinaemia in collaboration with Dana-Farber CancerInstitute.

TT-01488: Trans Thera Biosciences TT-01488 is a potential best-in-class, internally developed, novel non-covalent, reversible BTK inhibitor to overcome acquired resistance developed from marketed covalent BTKinhibitors in various types of relapsed or refractory hematological malignancies. In a head-to-head kinase panel screening, in addition to its higher potency, TT-01488 demonstrated better kinase selectivity than ARQ-531 (the frontrunner non-covalent reversible BTK inhibitor currently in Phase II trial) on EGFR and TEC, indicating its fewer off-target side effects and a better safety profile. In the DLBCL CDX model, TT-01488 showed a superior antitumor effect compared to ARQ-531. Currently, the drug is in the Phase I stage of its development for the treatment ofWaldenstrom’sMacroglobulinaemia.

Waldenstrom’s Macroglobulinemia: Therapeutic Assessment

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

Major Players in Waldenstrom’s Macroglobulinemia

• There are approx. 20+ key companies which are developing the therapies for Waldenstrom’s Macroglobulinemia. The companies which have their Waldenstrom’s Macroglobulinemia drug candidates in the most advanced stage, i.e. phase II include, Ascentage Pharma.

Phases

This report covers around 22+ 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

Waldenstrom’s Macroglobulinemia 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

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

Waldenstrom’s Macroglobulinemia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Waldenstrom’s Macroglobulinemia 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 Waldenstrom’s Macroglobulinemia drugs.

Waldenstrom’s Macroglobulinemia Report Insights

• Waldenstrom’s Macroglobulinemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Amgen
• Abbvie
• Roche
• Bristol-Myers Squibb
• X4 Pharmaceuticals
• Trans TheraSciences
• BeiGene
• Janssen
• Ascentage Pharma
• Gilead Sciences
• ADC Therapeutics
• Inno CarePharma
• Mustang Bio

Key Products

• Carfilzomib
• ABT199
• Obinutuzumab
• Dasatinib
• Mavorixafor
• TT-01488
• BGB 16673
• Zydelig
• Daratumumab
• APG-2575
• loncastuximab tesirine
• Orelabrutinib
• MB-106

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