Neurofibromatosis Type 1 Clinical Trials Analysis 2025: Targeted RAS-Pathway and Immuno-Oncology Approaches

The Neurofibromatosis Type 1 (NF1) clinical trial landscape in 2025 focuses on two main goals: controlling plexiform and cutaneous neurofibromas and preventing malignant peripheral nerve sheath tumors (MPNST) while improving quality of life. Current programs include RAS-MAPK pathway inhibitors, next-generation MEK inhibitors, mTOR modulators, immuno-oncology agents for MPNST, and early gene-directed therapies.

Late-stage trials primarily test oral RAS pathway inhibitors for pediatric plexiform neurofibromas, with attention to safety, functional improvement, and pain reduction. MPNST studies explore targeted therapy and immunotherapy combinations to tackle aggressive tumor biology.

Endpoints now incorporate volumetric MRI, patient-reported outcomes, neurocognitive assessments, and biomarkers like NF1 mutation profiling and circulating tumor DNA. Combination strategies and real-world evidence studies are increasingly applied, while upcoming pivotal readouts over the next 12-24 months will guide potential approvals and shifts in standard care.

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Key Takeaways from the Neurofibromatosis Type 1 Pipeline Report
• DelveInsight's neurofibromatosis type 1 pipeline analysis depicts a strong space with 8+ active players working to develop 10+ pipeline drugs for neurofibromatosis type 1 treatment.
• The leading neurofibromatosis type 1 companies include Shanghai Fosun Pharmaceutical, NFlection Therapeutics, Pasithea Therapeutics Corp., Novartis, Pfizer, and others are evaluating their lead assets to improve the neurofibromatosis type 1 treatment landscape.
• Key neurofibromatosis type 1 pipeline therapies in various stages of development include FCN-159, NFX‐179, PAS-004, Trametinib, Binimetinib, and others.
• In September 2025, the FDA approved selumetinib (KOSELUGO, AstraZeneca) granules and capsules for pediatric patients aged 1 and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). Previously, selumetinib capsules were approved for children aged 2 and older with the same condition.
• In February 2025, SpringWorks Therapeutics announced FDA approval of GOMEKLITM (mirdametinib), a MEK inhibitor, for treating symptomatic, inoperable plexiform neurofibromas in NF1 patients aged 2 and older. The approval also earned the company a rare pediatric disease priority review voucher.
• In November 2024, SpringWorks Therapeutics announced that the Phase IIb ReNeu trial results of mirdametinib for NF1-associated plexiform neurofibromas were published in the Journal of Clinical Oncology.

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Neurofibromatosis Type 1 Overview
Neurofibromatosis Type 1 (NF1) is a genetic disorder that affects the growth and development of nerve cells, leading to the formation of benign tumors called neurofibromas along nerves in the skin, brain, and other parts of the body. Mutations in the NF1 gene cause it and is typically inherited in an autosomal dominant pattern, though spontaneous mutations can also occur. Standard features include café-au-lait spots, freckling in the armpits or groin, Lisch nodules (tiny growths on the iris), and learning difficulties. While many cases are mild, some individuals may experience complications such as bone deformities, vision problems, or malignant tumor transformation. There is no cure for NF1, but regular monitoring and supportive treatments can help manage symptoms and improve quality of life.

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Neurofibromatosis Type 1 Treatment Analysis: Drug Profile
FCN-159: Shanghai Fosun Pharmaceutical
FCN-159 is a proprietary, potent MEK inhibitor developed by Shanghai Fosun Pharmaceutical. It targets the RAS/RAF/MEK/ERK signaling pathway, which is essential for cellular processes such as proliferation, survival, and angiogenesis. Dysregulation of this pathway, commonly due to BRAF, KRAS, or NRAS mutations, is linked to various cancers. FCN-159 is currently in Phase III clinical trials for the treatment of Neurofibromatosis Type 1 (NF1).

NFX‐179: NFlection Therapeutics
NFX‐179 is an investigational MEK inhibitor being developed by NFlection Therapeutics. Designed as a "soft drug," NFX‐179 Gel is formulated for topical use, concentrating the active agent at the dermal site while degrading rapidly in systemic circulation to minimize side effects. It is currently in Phase II clinical trials for the treatment of NF1.

PAS-004: Pasithea Therapeutics Corp.
PAS-004 is a small molecule allosteric inhibitor of MEK1/2, key kinases within the MAPK pathway. Since this pathway plays a critical role in abnormal cell growth and tumor formation, PAS-004 is being evaluated for its therapeutic potential in NF1 and other diseases. The drug is currently in Phase I clinical development for Neurofibromatosis Type 1.

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Neurofibromatosis Type 1 Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

By Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Scope of the Neurofibromatosis Type 1 Pipeline Report
• Coverage: Global
• Key Neurofibromatosis Type 1 Companies: Shanghai Fosun Pharmaceutical, NFlection Therapeutics, Pasithea Therapeutics Corp., Novartis, Pfizer, and others.
• Key Neurofibromatosis Type 1 Pipeline Therapies: FCN-159, NFX‐179, PAS-004, Trametinib, Binimetinib, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Neurofibromatosis Type 1 Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Neurofibromatosis Type 1 Pipeline Therapeutics
6. Neurofibromatosis Type 1 Pipeline: Late-Stage Products (Phase III)
7. Neurofibromatosis Type 1 Pipeline: Mid-Stage Products (Phase II)
8. Neurofibromatosis Type 1 Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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This release was published on openPR.