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Report Overview

CNS Lymphoma is a rare, aggressive non‑Hodgkin lymphoma confined to the brain, spinal cord, cerebrospinal fluid or eyes, representing about 3-4% of primary CNS tumors with an annual incidence of ~0.4-0.5 per 100,000 people globally. Prevalence increases with age and in immunocompromised patients, particularly those with HIV/AIDS. CNS Lymphoma pipeline analysis by Expert Market Research highlights ongoing epidemiology trends and unmet needs, with incidence projected to rise in aging populations and a growing pipeline of targeted therapies and clinical studies.

  • Major companies involved in the CNS Lymphoma pipeline analysis include Nurix Therapeutics, Inc., AVM Biotechnology Inc, and others.

  • Leading drugs currently in the pipeline include NX-2127, 18F-AV-1451, and others.

  • A unique market driver for the pipeline is the identification of molecular subtypes and actionable biomarkers (e.g., MYD88, CD79B mutations), enabling precision-targeted therapies. Advances in drug delivery across the blood–brain barrier and increasing use of novel immunotherapies such as CAR-T and bispecific antibodies tailored to CNS penetration are accelerating clinical development and investment interest.

Report Coverage

The CNS Lymphoma Pipeline Analysis Report by Expert Market Research gives comprehensive insights into CNS Lymphoma therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for CNS Lymphoma. The CNS Lymphoma report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The CNS Lymphoma pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with CNS Lymphoma treatment guidelines to ensure optimal care practices.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to CNS Lymphoma.

CNS Lymphoma Pipeline Analysis by Drug Class

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CNS Lymphoma Pipeline Outlook

The CNS lymphoma pipeline outlook reflects growing innovation in a disease traditionally managed with high-dose methotrexate-based chemotherapy, whole-brain radiotherapy, and consolidation with autologous stem cell transplantation. Limitations related to neurotoxicity and relapse have accelerated the development of targeted therapies. Notably, Bruton’s tyrosine kinase inhibitors are reshaping treatment paradigms. In this context, tirabrutinib has emerged as a key pipeline asset. In May 2025, Ono Pharmaceutical reported positive Phase II PROSPECT study results in relapsed or refractory primary CNS lymphoma, demonstrating meaningful response rates. Earlier approvals and launches in Japan further strengthen its clinical and commercial potential.

CNS Lymphoma Epidemiology

Central nervous system lymphoma (PCNSL) is a rare but aggressive subtype of non‑Hodgkin lymphoma. It has an estimated annual incidence of approximately 0.5 per 100,000 persons in the United States, equating to about 1,700 new cases per year. PCNSL accounts for roughly 3% of all primary brain tumors and 2-3% of all non‑Hodgkin lymphoma cases. Immunocompetent patients are typically diagnosed between the ages of 50 and 70, while immunocompromised individuals present earlier. Men are affected more often than women.

CNS Lymphoma – Pipeline Therapeutic Assessment

This section of the report covers the analysis of CNS Lymphoma drug candidates based on several segmentations, including:

By Phase

The pipeline assessment report covers 50+ drug analyses based on phase:

  • Late-Stage Products (Phase 3 and Phase 4)
  • Mid-Stage Products (Phase 2)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

By Drug Class

The CNS Lymphoma pipeline analysis report covers 50+ drug analyses based on drug classes:

  • Small Molecules
  • Monoclonal Antibodies
  • Antibody-Drug Conjugates
  • Bispecific Antibodies

By Route of Administration

The pipeline assessment report covers 50+ drug analyses based on the route of administration:

  • Oral
  • Parenteral
  • Others

CNS Lymphoma Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II covers a major share of the total CNS Lymphoma clinical trials. Phase I accounts for 35% of the pipeline, while Phase III represents 6%, indicating strong clinical activities. This phase distribution highlights a robust early- and mid-stage development landscape, underscoring sustained research momentum and long-term growth potential in the CNS lymphoma therapeutic pipeline.

CNS Lymphoma Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the CNS Lymphoma pipeline analysis include small molecules, monoclonal antibodies, antibody-drug conjugates, and bispecific antibodies. The CNS Lymphoma report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for CNS Lymphoma. Following regulatory approval, tirabrutinib was commercially launched in Japan in May 2020 under the brand name Velexbru®. The launch strengthened the CNS lymphoma pipeline by introducing an oral, brain-penetrant BTK inhibitor, supporting targeted disease management and reinforcing Japan’s leadership in advancing PCNSL-specific therapeutic innovations.

CNS Lymphoma Clinical Trials – Key Players

The EMR report for the CNS Lymphoma pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed CNS Lymphoma therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in CNS Lymphoma clinical trials:

  • InnoCare Pharma
  • Sichuan Baili Pharmaceutical Co., Ltd.
  • Ono Pharmaceutical Co. Ltd
  • Estrella Biopharma, Inc.
  • Nurix Therapeutics, Inc.
  • AVM Biotechnology Inc
  • Cellectar Biosciences, Inc.
  • Mayo Clinic

CNS Lymphoma – Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for CNS Lymphoma. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of CNS Lymphoma drug candidates.

Drug: AVM0703

AVM0703 is a suprapharmacologic, high‑dose formulation of dexamethasone designed as an immunomodulatory small‑molecule therapeutic with antineoplastic and lymphoablative activity. It works by binding glucocorticoid receptors and triggering mobilization of endogenous bispecific γδ TCR+ invariant TCR+ Natural Killer T‑like cells that can recognize and destroy tumor cells and potentially autoreactive immune cells. AVM0703 is being advanced by clinical‑stage biotech AVM Biotechnology Inc., which is conducting Phase 2 trials in relapsed/refractory Non‑Hodgkin’s lymphoma and exploring broader oncology and autoimmune indications.

Drug: CUDC-907

CUDC-907, also known as fimepinostat, is an orally available small‑molecule dual inhibitor of phosphatidylinositol 3‑kinase (PI3K) and histone deacetylases (HDACs), classifying it as an antineoplastic epigenetic regulator. It works by simultaneously inhibiting PI3K signaling and HDAC‑mediated chromatin remodeling to induce cancer cell apoptosis and impede survival pathways. Initially developed by Curis, Inc. for hematologic malignancies including relapsed/refractory diffuse large B‑cell lymphoma, it has received regulatory designations and undergone early clinical studies, though development activity has varied over time.

*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*

Key Questions Answered in the CNS Lymphoma Pipeline Insight Report

  • Which companies/institutions are leading the CNS Lymphoma drug development?
  • Which company is leading the CNS Lymphoma pipeline development activities?
  • What is the current CNS Lymphoma commercial assessment?
  • What are the opportunities and challenges present in the CNS Lymphoma pipeline landscape?
  • What is the efficacy and safety profile of CNS Lymphoma pipeline drugs?
  • Which company is conducting major trials for CNS Lymphoma drugs?
  • Which companies/institutions are involved in CNS Lymphoma collaborations aimed at providing enhanced therapeutic alternatives for patients?
  • What are the geographies covered for clinical trials in CNS Lymphoma?

Reasons To Buy This Report

The CNS Lymphoma Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for CNS Lymphoma. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into CNS Lymphoma collaborations, regulatory environments, and potential growth opportunities.

Related Reports

Global Clinical Trials Market

CNS Lymphoma Epidemiology Forecast

Central Nervous System (CNS) Stimulant Drugs Market

*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*

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Report Summary

Explore our key highlights of the report and gain a concise overview of key findings, trends, and actionable insights that will empower your strategic decisions.

Key Highlights of the Report

Please note that the figures mentioned in the description serve as estimates and may vary from the actual figures presented in the final report.

Scope of the Report

Details

Drug Pipeline by Clinical Trial Phase

  • Late-Stage Products (Phase III and Phase IV)
  • Mid-Stage Products (Phase II)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

Route of Administration

  • Oral
  • Parenteral
  • Others

Drug Classes

  • Small Molecules
  • Monoclonal Antibodies
  • Antibody-Drug Conjugates
  • Bispecific Antibodies

Leading Sponsors Covered

  • InnoCare Pharma
  • Sichuan Baili Pharmaceutical Co., Ltd.
  • Ono Pharmaceutical Co. Ltd
  • Estrella Biopharma, Inc.
  • Nurix Therapeutics, Inc.
  • AVM Biotechnology Inc
  • Cellectar Biosciences, Inc.
  • Mayo Clinic

Geographies Covered

  • North America
  • Europe
  • Asia Pacific
  • Others

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