VivoSeq
Neurology

Neurofibromatosis

22

Pipeline Programs↗

6

Active Trials↗

17

Companies Involved↗

Neurofibromatosis is an emerging landscape with 22 programs across 17 companies. The most active players are Roche, Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA), Graceway Pharmaceuticals, LLC. 1 therapy is approved with 4 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

49/100

Pipeline Velocity

6/100

Mech. Concentration

20/100

Completion Rate

67/100

Attrition Rate

24/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Roche logo

Roche

1 program

Phase 1C

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

1 program

Phase 1G

Graceway Pharmaceuticals, LLC

1 program

Phase 1Novartis logo

Novartis

2 programs

Phase 2N

NFlection Therapeutics, Inc.

1 program

Phase 2P

PTC Therapeutics

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

88%

Small molecule

22 of 25 classified programs — the dominant modality here.

12%ALL OTHERSMonoclonal antibody14%Protein / enzyme14%Cell therapy14%

Small molecule leads; the ring shows all 25 classified programs, each share of the total. Modality classified for 96% of 26 pipeline programs.

Analysis

Trial Status

25

clinical trials

Completed12 · 48%
Terminated5 · 20%
Not yet recruiting3 · 12%
Recruiting3 · 12%
Withdrawn1 · 4%
Unknown status1 · 4%

Clinical Trials

Trial Activity

25 trials

Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
Recruiting·JJohns Hopkins University·Phase 1/2·NCT06159166·24 enrolled
A Prospective Natural History Study of Patients With Neurofibromatosis Type 2 (NF2).
Recruiting·National Institute of Neurological Disorders and Stroke (NINDS)·N/A·NCT00598351·269 enrolled
Clinical and Demographic Characteristics of Adult Patients With NEurofibromatosis in RUSsia
Recruiting·AstraZeneca logoAstraZeneca·N/A·NCT07088991·200 enrolled
Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule
Not yet recruiting·SSun Yat-Sen Memorial Hospital of Sun Yat-Sen University·Phase 2·NCT06620354·33 enrolled
The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsule
Not yet recruiting·SSun Yat-Sen Memorial Hospital of Sun Yat-Sen University·Phase 2·NCT06621082·19 enrolled
Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II
Not yet recruiting·PPRG Science & Technology Co., Ltd.·Phase 1/2·NCT07131722·25 enrolled
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
Completed·NNFlection Therapeutics, Inc.·Phase 2·NCT05005845·199 enrolled
Icotinib Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
Completed·—·Phase 2·NCT02934256·10 enrolled
Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
Completed·NNYU Langone Health·Phase 2·NCT00973739·21 enrolled
Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
Completed·MMayo Clinic·Phase 2·NCT00754780·24 enrolled
Endostatin Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
Completed·—·Phase 2·NCT02104323·20 enrolled
NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
Completed·NNFlection Therapeutics, Inc.·Phase 2·NCT04435665·48 enrolled
Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)
Completed·UUniversity of California, Los Angeles·Phase 1·NCT00352599·44 enrolled
Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2
Completed·NNYU Langone Health·Phase 2·NCT01419639·10 enrolled
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T
Completed·TThe University of Texas Health Science Center, Houston·Phase 2·NCT02332902·24 enrolled
Page 1 of 2

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