VivoSeq
Neurology

Neurofibromatosis Type 1

38

Pipeline Programs↗

12

Active Trials↗

18

Companies Involved↗

Neurofibromatosis Type 1 is a moderately active landscape with 38 programs across 18 companies. The most active players are AbbVie, Pasithea Therapeutics Corp., Bayer. 8 therapies are approved.

Intelligence

Pipeline Metrics

Competitive Density

57/100

Pipeline Velocity

0/100

Mech. Concentration

17/100

Completion Rate

82/100

Attrition Rate

10/100

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.

AbbVie logo

AbbVie

2 programs

Phase 1P

Pasithea Therapeutics Corp.

1 program

Phase 1Bayer logo

Bayer

1 program

Phase 1Daiichi Sankyo logo

Daiichi Sankyo

1 program

Phase 1AstraZeneca logo

AstraZeneca

1 program

Phase 2S

Shanghai Kechow Pharma, Inc.

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Trial Status

30

clinical trials

Completed14 · 47%
Active, not recruiting8 · 27%
Recruiting3 · 10%
Terminated3 · 10%
Approved for marketing1 · 3%
Not yet recruiting1 · 3%

Clinical Trials

Trial Activity

30 trials

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
Recruiting·HHealx Limited·Phase 2·NCT06541847·25 enrolled
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
Recruiting·PPasithea Therapeutics Corp.·Phase 1·NCT06961565·56 enrolled
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Recruiting·SShanghai Kechow Pharma, Inc.·Phase 2·NCT05331105·70 enrolled
Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
Active, not recruiting·AstraZeneca logoAstraZeneca·Phase 1·NCT05101148·24 enrolled
MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Active, not recruiting·National Cancer Institute (NCI)·Phase 2·NCT02407405·36 enrolled
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
Active, not recruiting·SShanghai Fosun Pharmaceutical Industrial Development Co. Ltd.·Phase 3·NCT05913037·167 enrolled
Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1
Active, not recruiting·—·Phase 2·NCT04481048·58 enrolled
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
Active, not recruiting·AstraZeneca logoAstraZeneca·Phase 1·NCT04590235·32 enrolled
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
Active, not recruiting·SShanghai Fosun Pharmaceutical Industrial Development Co. Ltd.·Phase 1/2·NCT04954001·160 enrolled
Natural History Study and Longitudinal Assessment of Children, Adolescents, and Adults With Neurofibromatosis Type 1
Active, not recruiting·National Cancer Institute (NCI)·N/A·NCT00924196·259 enrolled
Neurobiology and Treatment of Reading Disability in NF1
Active, not recruiting·VVanderbilt University·Phase 2·NCT02964884·120 enrolled
Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1
Not yet recruiting·SShanghai Fosun Pharmaceutical Industrial Development Co. Ltd.·Phase 1/2·NCT07024394·65 enrolled
A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
Completed·UUniversity of Alabama at Birmingham·Phase 2·NCT00634270·58 enrolled
Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
Completed·MMassachusetts General Hospital·Phase 1·NCT04730583·34 enrolled
Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas
Completed·MMassachusetts General Hospital·Phase 1·NCT06132165·13 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)