Retinoblastoma

42

Pipeline Programs↗

13

Active Trials↗

17

Companies Involved↗

Retinoblastoma is a moderately active landscape with 42 programs across 17 companies. The most active players are Eli Lilly, Chengdu Kanghong Biotech Co., Ltd., Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA). 2 therapies are approved with 12 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

58/100

Pipeline Velocity

0/100

Mech. Concentration

15/100

Completion Rate

62/100

Attrition Rate

21/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved

Phase 3

Phase 2

Phase 1

Preclinical

Key Players

Top Companies

Eli Lilly

2 programs

Chengdu Kanghong Biotech Co., Ltd.

1 program

Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

1 program

Pfizer

1 program

Novartis

1 program

Roche

1 program

Pipeline

Programs by Phase

Preclinical9

Cytarabine

Teva

Dexmedetomidine

McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Topotecan Hydrochloride

Gynecologic Oncology Group

Photodynamic Therapy

Roswell Park Cancer Institute

Nitroglycerin

Viatris Specialty LLC

Phase 15

Topotecan and Pazopanib

The Hospital for Sick Children

Episcleral Celecoxib

Targeted Therapy Technologies, LLC

Maxitrol

St. Jude Children's Research Hospital

GPC2 CAR T Cells

Stanford University

Targeting NUDT21 Sirna Drugs

Eye & ENT Hospital of Fudan University

Phase 214

Abemaciclib

Eli Lilly

Temozolomide

Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Conbercept

Chengdu Kanghong Biotech Co., Ltd.

Doxorubicin

Pfizer

Vincristine

Eli Lilly

Phase 312

Carboplatin

Avyxa

Cisplatin

McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Etoposide

Avyxa

Vincristine Sulfate

Eastern Cooperative Oncology Group

Melphalan

Apotex Inc.

Approved2

Carboplatin AUC=5

Sun Yat-sen University

CEV Chemotherapy

Sun Yat-sen University

Analysis

Modality Breakdown

92%

Small molecule

47 of 51 classified programs — the dominant modality here.

Small molecule leads; the ring shows all 51 classified programs, each share of the total. Modality classified for 100% of 51 pipeline programs.

Analysis

Trial Status

48

clinical trials

Completed16 · 33%

Recruiting9 · 19%

Unknown status9 · 19%

Terminated8 · 17%

Active, not recruiting4 · 8%

Withdrawn2 · 4%

Clinical Trials

Trial Activity

48 trials

High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed

Recruiting·EEye & ENT Hospital of Fudan University·Phase 1·NCT06972602·10 enrolled

Adjuvant Chemotherapy for High-risk Postenucleation Retinoblastoma

Recruiting·SSun Yat-sen University·Phase 3·NCT05080010·500 enrolled

A Study to Give Treatment Inside the Eye to Treat Retinoblastoma

Recruiting·Children's Oncology Group·Phase 2·NCT05504291·26 enrolled

Retinoblastoma Phase II Expanded Access Clinical Trial

Recruiting·TTargeted Therapy Technologies, LLC·Phase 2·NCT06679634·30 enrolled

GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma

Recruiting·SStephan Grupp MD PhD·Phase 1·NCT05650749·45 enrolled

Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma

Recruiting·EEye & ENT Hospital of Fudan University·N/A·NCT04903678·18 enrolled

Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma

Recruiting·EEye & ENT Hospital of Fudan University·Phase 1·NCT06424301·2 enrolled

Topotecan and Melphalan for Retinoblastoma

Recruiting·SSun Yat-sen University·Phase 3·NCT04799002·50 enrolled

Ocular Conservative Treatment for Retinoblastoma : Efficacy of the New Management Strategies and Visual Outcome

Recruiting·IInstitut Curie·Phase 2/3·NCT04681417·225 enrolled

Conservative Treatments of Retinoblastoma

Active, not recruiting·IInstitut Curie·Phase 2·NCT02866136·133 enrolled

Combination Chemotherapy and Cyclosporine Followed by Focal Therapy for Bilateral Retinoblastoma

Active, not recruiting·TThe Hospital for Sick Children·Phase 2·NCT00110110·71 enrolled

Adjuvant Chemotherapy for High-risk Retinoblastoma After Enucleation

Active, not recruiting·SSun Yat-sen University·Phase 3·NCT01906814·179 enrolled

Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma

Active, not recruiting·SSt. Jude Children's Research Hospital·Phase 2·NCT01783535·174 enrolled

A Study of the Effectiveness of a Local Injection of Chemotherapy for Retinoblastoma

Completed·—·N/A·NCT00857519·10 enrolled

Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma

Completed·SSt. Jude Children's Research Hospital·Phase 3·NCT00186888·107 enrolled

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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)