Once-daily topical treatment of target cutaneous neurofibromas (cNFs) with NFX‑179 Gel 1.5% achieved statistically significant reduction in size

用NFX-179凝胶1.5%每日一次局部治疗靶皮肤神经纤维瘤（CNF）达到统计学显着的尺寸减小

First randomized, double-blind, vehicle-controlled Phase 2b clinical trial to report positive results in the treatment of cutaneous neurofibromas

首次随机，双盲，车辆控制的2b期临床试验报告皮肤神经纤维瘤治疗阳性结果

Multiple secondary endpoints met; significant reductions in the volume and height of cNFs correlates with patient-reported meaningful improvement in their cNFs

满足多个次要终点；Cnf体积和高度的显著减少与患者报告的cNFs有意义的改善相关

Efficacy and tolerability results support advancing NFX‑179 Gel program to Phase 3 development in 2024

功效和耐受性结果支持将NFX-179凝胶计划推进到2024年的3期开发

BOSTON, Nov. 13, 2023 /PRNewswire/ -- NFlection Therapeutics Inc., a company developing topical MEK inhibitors for RAS-mediated skin conditions, today announced positive topline results from a randomized, double-blind, vehicle-controlled Phase 2b clinical trial (NCT05005845) evaluating NFX‑179 Gel as a treatment for cutaneous neurofibromas (cNFs) in people with neurofibromatosis type 1 (NF1), a rare genetic condition.

波士顿，2023年11月13日/PRNewswire/-NFlection Therapeutics Inc.，一家开发用于RAS介导的皮肤病的局部MEK抑制剂的公司，今天宣布了一项随机，双盲，媒介物对照的2b期临床试验的阳性结果。（NCT05005845）评估NFX-179凝胶治疗1型神经纤维瘤病（NF1）患者的皮肤神经纤维瘤（cNFs），罕见的遗传病。

Cutaneous neurofibromas appear in over 95% of people with NF1 and are physically disfiguring, painful, cause extensive psychological harm, and negatively impact quality of life. There are currently no FDA-approved therapeutics for the treatment of cNFs..

皮肤神经纤维瘤出现在95%以上的NF1患者中，身体毁容，疼痛，造成广泛的心理伤害，并对生活质量产生负面影响。目前还没有FDA批准的治疗cNFs的疗法。。

The Phase 2b study was a 6‑month double-blind, randomized, vehicle-controlled study of 199 subjects to determine the safety and effectiveness of NFX‑179 Gel 0.5% and 1.5% compared with vehicle in patients with cNFs. The study was conducted at 24 investigational centers in the U.S.

2b期研究是对199名受试者进行的为期6个月的双盲，随机，载体对照研究，以确定NFX-179凝胶与CNF患者载体相比0.5%和1.5%的安全性和有效性。该研究在美国的24个研究中心进行。

NFX‑179 Gel was applied topically to target cNF tumors for six months, enabling delivery of high concentrations of NFX‑179 to the tumors. The highest concentration of NFX‑179 tested, 1.5%, achieved the study's primary efficacy endpoint, with a statistically significant improvement over vehicle in the shrinkage of cNFs in people with NF1 that was highly correlated with patient-reported meaningful improvements in their cNFs.

将NFX-179凝胶局部施用于靶向cNF肿瘤6个月，使得能够将高浓度的NFX-179递送至肿瘤。测试的NFX-179浓度最高，为1.5%，达到了研究的主要疗效终点，与NF1患者CNF收缩率高度相关的统计学显着改善与患者报告的CNF有显着改善相关。

Consistent with its design as a soft, or metabolically labile, MEK inhibitor, NFX‑179 Gel was generally well tolerated, and plasma drug concentrations were orders of magnitude lower than those typically observed for oral MEK inhibitors..

与其作为软的或代谢不稳定的MEK抑制剂的设计一致，NFX-179凝胶通常具有良好的耐受性，血浆药物浓度比口服MEK抑制剂通常观察到的低几个数量级。。

'Cutaneous neurofibromas, which present in over 95 percent of people with NF1, are associated with significant psychosocial impact and are often the most burdensome symptom of the condition,' said study investigator Miriam Bornhorst, MD, Assistant Professor of Pediatrics, and the Clinical Director of the Gilbert Family Neurofibromatosis Institute at Children's National Hospital.

研究调查人员Miriam Bornhorst博士，儿科助理教授和临床研究员说：“皮肤神经纤维瘤存在于95%以上的NF1患者中，与显着的社会心理影响有关，并且通常是该病最繁重的症状。”儿童国立医院吉尔伯特家族神经纤维瘤病研究所主任。

'The data shows that NFX‑179 Gel significantly reduced the size of cNFs across multiple efficacy endpoints and was generally well tolerated. We are excited to see the potential for NFX‑179 Gel as the first topical therapy for the treatment of cNFs in people with NF1.'.

'数据显示NFX-179凝胶显着降低了多个功效终点的CNF大小，并且通常具有良好的耐受性。我们很高兴看到NFX-179凝胶作为治疗NF1患者CNF的第一种局部疗法的潜力。

The primary efficacy endpoint of the study was a subject-level responder analysis, with a response defined as at least 50% reduction in cNF volume above the surrounding non-tumor skin for five or more of the ten treated tumors, after 6 months of once-daily application of NFX‑179 Gel.

该研究的主要疗效终点是受试者水平响应者分析，其响应定义为在一次治疗6个月后，对于10个治疗的肿瘤中的5个或更多个，在周围非肿瘤皮肤上方的cNF体积减少至少50%每日应用NFX-179凝胶。

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).

该研究达到了主要疗效终点，NFX-179凝胶1.5%的剂量依赖性应答率为44.2%，NFX-179凝胶0.5%的剂量依赖性应答率为34.8%，载体为24.1%。高剂量具有统计学显着高于载体的应答率（p=0.03）。

'We are pleased to report success from our Phase 2b NF1 patient trial that supports the first-in-class potential of NFX‑179,' said William Hodder, Chief Executive Officer of NFlection. 'Based on the favorable efficacy and tolerability profile NFX‑179 Gel demonstrated in this study, we plan to meet with U.S.

“我们很高兴报告支持NFX-179一流潜力的2b期NF1患者试验的成功，”NFlection首席执行官William Hodder说基于本研究中证明的有利功效和耐受性概况NFX-179凝胶，我们计划与美国会面。

and European regulators and move the program to Phase 3 development in 2024. We would like to thank the patients, our investigators and collaborators, including The Children's Tumor Foundation, who made this study possible. We look forward to continuing the development of this important therapy for the NF1 community.'.

和欧洲监管机构，并于2024年将该计划移至第3阶段开发。我们要感谢使这项研究成为可能的患者，我们的研究人员和合作者，包括儿童肿瘤基金会。我们期待继续为NF1社区开发这种重要疗法。”。

'In addition to developing efficient preclinical and clinical neurofibromatosis (NF) test platforms, CTF is dedicated to investing in new medicines for people with NF. Our support for NFlection demonstrates that commitment,' said Annette Bakker, PhD, President of The Children's Tumor Foundation. 'People with NF1 with cutaneous neurofibromas have been overlooked for a long time and are desperate for safe and easy-to-apply treatment options.'.

“除了开发有效的临床前和临床神经纤维瘤病（NF）测试平台外，CTF还致力于为NF患者投资新药。我们对NF选择的支持证明了这一承诺，”儿童肿瘤总裁Annette Bakker博士说。基金会'患有皮肤神经纤维瘤的NF1患者长期以来一直被忽视，迫切需要安全且易于应用的治疗方案。

About NFX‑179 Topical GelNFX‑179 is an investigational mitogen-activated protein kinase kinase (MEK) inhibitor. NFX‑179 is a 'soft' (metabolically labile) drug, which, when formulated as NFX‑179 Gel for topical application, is designed to concentrate at the dermal site of action but degrade in systemic circulation, thereby significantly reducing side effects routinely seen with systemically available MEK inhibitors.

关于NFX-179外用GelNFX-179是一种研究性丝裂原活化蛋白激酶激酶（MEK）抑制剂。NFX-179是一种“软”（代谢不稳定）药物，当配制成局部应用的NFX-179凝胶时，其设计用于集中在皮肤作用部位但在体循环中降解，从而显着减少常规副作用用全身可用的MEK抑制剂观察。

NFlection is developing NFX‑179 Gel for the treatment of cutaneous neurofibromas in people with neurofibromatosis type 1 and has received Orphan Designation in the United States and European Union for this indication..

NFlection正在开发NFX-179凝胶，用于治疗1型神经纤维瘤病患者的皮肤神经纤维瘤，并已在美国和欧盟获得孤儿称号。。

About NFlection Therapeutics Inc.NFlection is a clinical-stage biopharmaceutical company focused on the development of novel therapies to address the needs of patients with neurofibromatosis type 1, immunosuppressant-mediated squamous cell carcinoma, and congenital birthmarks such as keratinocytic epidermal nevi and nevi sebacei.

关于NFlection Therapeutics Inc.NFlection是一家临床阶段的生物制药公司，专注于开发新疗法，以满足1型神经纤维瘤病，免疫抑制剂介导的鳞状细胞癌和先天性胎记如角质形成细胞表皮痣和痣sebacei患者的需求。

To address these RAS-mediated disorders driven by the aberrant activation of the Ras/Raf/MEK/ERK pathway, we are developing first-in-class MEK (mitogen-activated protein kinase kinase) inhibitors for topical treatment of these conditions. To learn more about the company, please visit www.nflection.com..

为了解决由RAS/Raf/MEK/ERK途径异常激活驱动的这些RAS介导的疾病，我们正在开发用于局部治疗这些疾病的一流MEK（促分裂原活化蛋白激酶激酶）抑制剂。要了解有关公司的更多信息，请访问www.nflection.com。。

SOURCE NFlection Therapeutics

来源选择疗法