NFX-179 / NFlection Therap - LARVOL DELTA

12-week Study of NFX-179 Gel in Subjects With Epidermal Nevi (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Albert Chiou | N=12 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Rare Diseases

Effect of NFX-179 MEK inhibitor on cutaneous neurofibromas in persons with neurofibromatosis type 1. (PubMed, Sci Adv) - "In addition, 20% of cNFs treated with 0.5% NFX-179 Topical Gel showed a ≥50% reduction in volume compared to 6% in the vehicle group by ruler measurement with calculated volume (P = 0.021). Thus, NFX-179 Topical Gel demonstrated significant inhibition of MEK in cNF with excellent safety and potential therapeutic benefit."

Clinical • Journal • P2 data • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor

NFX-179 topical gel for the treatment of cutaneous neurofibromas in neurofibromatosis type 1: a multicenter phase 2b study (SID 2024) - No abstract available

Clinical • Late-breaking abstract • P2b data • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor

Qualitative Insights Into Disease Burden and Meaningful Change Associated With Cutaneous Neurofibromas (CNFs) in Adults With Neurofibromatosis Type 1 (ISPOR 2024) - "To support development of a novel treatment of cNF, NFX-179 Topical Gel, NFlection wanted to understand what is most important to individuals in respect to the impact/bother associated with cNFs and explore the participant experience of meaningful change in cNFs within a Phase 2b trial... Size was shown to be the key aspect of cNFs, and changes in size, in particular height, are important to people living with NF1. The PGI-C scales captured this change, and a 1-category improvement was reported to reflect meaningful change on these scales."

Clinical • CNS Disorders • Depression • Fibrosis • Genetic Disorders • Mood Disorders • Neurofibromatosis • Pain • Psychiatry • Solid Tumor • NF1

NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) (clinicaltrials.gov) - P2 | N=199 | Completed | Sponsor: NFlection Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor • NF1

NFlection Therapeutics Announces Positive Results from Phase 2b Study of NFX‑179 Topical Gel in the Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 (PRNewswire) - P2 | N=199 | NCT05005845 | Sponsor: NFlection Therapeutics, Inc. | "'The data shows that NFX‑179 Gel significantly reduced the size of cNFs across multiple efficacy endpoints and was generally well tolerated....The study met the primary efficacy endpoint with dose-dependent responder rates of 44.2% with NFX‑179 Gel 1.5%, 34.8% with NFX‑179 Gel 0.5%, and 24.1% with vehicle. The high dose had a statistically significant higher responder rate than vehicle (p = 0.03)....'Based on the favorable efficacy and tolerability profile NFX‑179 Gel demonstrated in this study, we plan to meet with U.S. and European regulators and move the program to Phase 3 development in 2024."

European regulatory • FDA event • P2b data • Neurofibromatosis • Oncology • Solid Tumor

Development of a MEK inhibitor, NFX-179, as a chemoprevention agent for squamous cell carcinoma. (PubMed, Sci Transl Med) - "Our systematic computational drug repurposing screen predicted that selumetinib, a MAPK (mitogen-activated protein kinase) kinase inhibitor (MEKi), would reverse transcriptional signatures associated with cSCC development, consistent with our genomic analysis implicating MEK as a chemoprevention target. NFX-179 inhibits the growth of human SCC cell lines in a dose-dependent manner, and topical NFX-179 application penetrates human skin and inhibits MAPK signaling in human cSCC explants. Together, our data provide a compelling rationale for using topical MEK inhibition through the application of NFX-179 gel as an effective strategy for cSCC chemoprevention."

Journal • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer • Squamous Cell Carcinoma • Squamous Cell Skin Cancer

NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) (clinicaltrials.gov) - P2 | N=199 | Active, not recruiting | Sponsor: NFlection Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Feb 2023 ➔ Sep 2023 | Trial primary completion date: Jan 2023 ➔ Sep 2023

Enrollment closed • Trial completion date • Trial primary completion date • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor • NF1

12-week Study of NFX-179 Gel in Subjects With Epidermal Nevi (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: Albert Chiou | Trial completion date: Dec 2023 ➔ Dec 2024 | Initiation date: Jun 2022 ➔ Jun 2023 | Trial primary completion date: Jun 2023 ➔ Jun 2024

Trial completion date • Trial initiation date • Trial primary completion date • Rare Diseases

12-week Study of NFX-179 Gel in Subjects With Epidermal Nevi (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: Albert Chiou | Initiation date: Feb 2022 ➔ Jun 2022 | Trial primary completion date: Feb 2023 ➔ Jun 2023

Trial initiation date • Trial primary completion date • Rare Diseases

12-week Study of NFX-179 Gel in Subjects With Epidermal Nevi (clinicaltrials.gov) - P2; N=12; Not yet recruiting; Sponsor: Albert Chiou

Clinical • New P2 trial • Rare Diseases

NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) (clinicaltrials.gov) - P2; N=168; Recruiting; Sponsor: NFlection Therapeutics, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor • NF1

NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) (clinicaltrials.gov) - P2; N=168; Not yet recruiting; Sponsor: NFlection Therapeutics, Inc.

Clinical • New P2 trial • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor • NF1

NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) (clinicaltrials.gov) - P2a; N=48; Completed; Sponsor: NFlection Therapeutics, Inc.; Recruiting ➔ Completed

Trial completion • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor • NF1

NFlection Therapeutics’ NFX-179 Gel Demonstrates Safety and Significant Reduction in p-ERK, a Key Biomarker Driving Neurofibroma Tumor Growth, in Positive Data From a 28-Day Phase 2a Clinical Trial (PRNewswire) - P2, N=44; NCT04435665; Sponsor: NFlection Therapeutics; "Data supports progression to Phase 2b, set to commence in the second half of 2021…NFX-179 Gel showed a statistically significant suppression in p-ERK. A 47% reduction (p = 0.0001) in p-ERK was observed in tumors treated with 0.5% NFX-179 Gel, and a 26% reduction (p = 0.04) in p-ERK was observed in tumors treated with 0.15% NFX-179 Gel. The lowest dose group, 0.05% NFX-179 Gel, gave a 10% reduction of p-ERK that was not statistically different from vehicle (p = 0.4).”

New P2b trial • P2a data • Dermatology • Genetic Disorders • Neurofibromatosis • Oncology • Solid Tumor • Squamous Cell Carcinoma

"Topical #MEKInhibitor, #NFX179, Prevents #CutaneousSquamousCellCarcinoma in Pre-Clinical Model https://t.co/GtD8yiZei7" (@1stOncology)

Non-melanoma Skin Cancer • Oncology • Squamous Cell Skin Cancer

Topical MEK Inhibitor, NFX-179, Prevents Cutaneous Squamous Cell Carcinoma in Pre-Clinical Model (PRNewswire) - "NFX-179 gel reduced the formation of new cutaneous squamous cell carcinomas by an average of 92% at doses of 0.5% and greater at 28 days. Near-complete suppression of cutaneous squamous cell carcinoma was observed from treatment of NFX-179 0.5% gel in a split-back randomized controlled study in mice. NFX-179 is a topical, soft compound that is precision-targeted and has the potential to be well-tolerated, especially in immunosuppressed patients, because it is rapidly degraded when it reaches the blood."

Preclinical • Oncology • Squamous Cell Carcinoma

[VIRTUAL] Topical MEK inhibition as precision targeted chemoprevention (SID 2021) - "Consistent with this, our computational drug repositioning screen predicted the FDA-approved MEK1, 2 inhibitor, selumetinib, as a therapeutic for cuSCC based on its ability to reverse transcriptional signatures associated with cuSCC development. NFX-179 inhibits the growth of human cSCC cell lines in a dose-dependent manner and topical NFX-179 application penetrates human skin and inhibits ERK signaling in human cuSCC explants. Our data provide compelling evidence that topical application of NFX-179 is a safe, effective strategy for SCC chemoprevention in high risk individuals."

Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Skin Cancer

NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) (clinicaltrials.gov) - P2a; N=48; Recruiting; Sponsor: NFlection Therapeutics, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Genetic Disorders • Neurofibromatosis • Solid Tumor • Uterine Leiomyoma

[VIRTUAL] Development of topical MEK inhibitor, NFX-179, as a chemopreventive agent for squamous cell carcinoma (SID 2020) - "These results support the development of topical NFX-179 for as a chemopreventative agent for SCC. Plans are underway to conduct human clinical trials in 2021."

Late-breaking abstract • Genetic Disorders • Oncology • Squamous Cell Carcinoma

NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) (clinicaltrials.gov) - P2a; N=48; Not yet recruiting; Sponsor: NFlection Therapeutics, Inc.

Clinical • New P2a trial • Genetic Disorders • Neurofibromatosis • Solid Tumor • Uterine Leiomyoma