Neurofibromatosis Type 1

3
Pipeline Programs
3
Companies
2
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
2
0
1
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
2100%
+ 1 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Healx
HealxUK - Cambridge
1 program
1
HLX-1502Phase 21 trial
Active Trials
NCT06541847RecruitingEst. Jan 2028
M&
Merck & Co.RAHWAY, NJ
1 program
1
SelumetinibPhase 1Small Molecule1 trial
Active Trials
NCT05101148Active Not Recruiting24Est. Apr 2028
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
1 program
1
SelumetinibPhase 1Small Molecule

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
HealxHLX-1502
Merck & Co.Selumetinib

Clinical Trials (2)

Total enrollment: 24 patients across 2 trials

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1

Start: Feb 2025Est. completion: Jan 2028
Phase 2Recruiting

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

Start: Jul 2021Est. completion: Apr 202824 patients
Phase 1Active Not Recruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 actively recruiting trials targeting 24 patients
Small Molecule is the dominant modality (100% of programs)
3 companies competing in this space