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▎ WuXi AppTec content team editor On May 26, 2021, NFlection Therapeutics announced that its topical NFX-179 gel has achieved positive results in a phase 2a clinical trial for the treatment of neurofibromatosis type 1 (NF1) patients.
The test results showed that NFX-179 gel at a dose of 0.
5% had the best inhibitory effect on p-ERK, a biomarker that drives the growth of subcutaneous neurofibromas, reducing the level of p-ERK by 47%.
In addition, after 28 days of treatment, the experimental group observed a reduction in the tumor volume of patients, and 22% of the patients in the 0.
5% NFX-179 group reduced their tumor volume by an average of at least 50%.
NFlection plans to launch a Phase 2b clinical trial in the second half of this year to study the impact of longer treatment time on tumor shrinkage.
This study provides hope for NF1 patients who suffer from pain or disfigurement of the mass.
NFX-179 gel is designed to deliver a MEK inhibitor that is easily metabolically degraded to tumors by topical application on the skin surface to inhibit the excessive activation of the Ras/Raf/MEK/ERK pathway in these tumors.
NF1 is caused by mutations in the NF1 gene that synthesizes neurofibromin (neurofibromin), which can disrupt the RAS-RAF-MEK-ERK signal and lead to tumor growth.
NFX-179 can be degraded naturally in the human circulation, so it can locally inhibit the RAS-RAF-MEK-ERK signaling pathway while reducing the systemic side effects of MEK inhibitors.
In this multicenter, randomized double-blind, parallel grouping, placebo-controlled Phase 2a clinical trial, 48 patients were randomized to receive 0.
05%, 0.
15%, or 0.
5% NFX-179 gel or placebo once a day 28 days.The primary endpoints are safety, tolerability, and inhibition of p-ERK levels in tumors.
p-ERK is a key biomarker known to promote the growth of subcutaneous neurofibromas.
The test results show that, compared with the control group, 0.
5% and 0.
15% NFX-179 have statistically significant inhibitory effects on p-ERK.
0.
5% and 0.
15% of NFX-179 reduced p-ERK by 47% (p=0.
0001) and 26% (p=0.
04), respectively.
The 0.
05% NFX-179 in the lowest dose group reduced p-ERK by 10%, which was not statistically different from the control group (p=0.
4).
In addition, the secondary endpoint of the study showed that despite only 28 days of treatment, the patient's tumor volume had decreased.
The tumor volume in the 0.
5% NFX-179 group was reduced by an average of 17% compared with baseline (8% in the control group, p=0.
073).
In the 0.
5% NFX-179 gel group, 22% of patients had an average reduction in tumor volume of at least 50% (6% in the control group, p=0.
051).
Image source: In terms of safety and tolerance of 123RF, NFX-179 gel is well tolerated.
No serious adverse events were reported, and no adverse events were classified as related to NFX-179.
All adverse events were mild to moderate, with similar frequency in the experimental group and the control group.
Unlike systemic MEK inhibitors that can cause severe acne-like rashes, no acne-like rashes were observed during NFX-179 treatment.
Cutaneous neurofibromas are tumors that grow from small nerves in or under the skin.
Large or small masses usually appear around puberty.
Patients with NF1 usually develop more cutaneous neurofibromas as they age.
They do not become malignant tumors, but they may cause disfigurement, itching or pain.
They may also cause serious problems such as depression and isolation, and may require surgical removal. ▲ NFlection’s Chief Medical Officer, Dr.
Guy Webster (Image source: NFlection’s official website) NFlection’s Chief Medical Officer, Dr.
Guy Webster, said, “We are very satisfied with these data.
They prove that NFX-179 gel has good tolerance and can Induce cutaneous neurofibromas to produce clinically significant p-ERK inhibition.
Whether it is the powerful p-ERK biomarker data or the early trend of tumor volume reduction after only 28 days of treatment, all support our hypothesis that NFX- 179 gel is an important new therapy for patients with NF1.
We look forward to advancing the clinical research of NFX-179 gel to Phase 2b to determine the effect of longer treatment time on the regression of cutaneous neurofibromas.
"For more rare disease related knowledge and research progress, please click to visit our mini program reference materials: [1] 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.
Retrieved May 26, 2021, from -reduction-in-p-erk-a-key-biomarker-driving-neurofibroma-tumor-growth-in-positive-data-from-a-28-day-phase-2a-clinical-trial-301299521.
htmlNote: This article aims to introduce the progress of medical and health research, not to recommend treatment plans.
If you need guidance on treatment plans, please go to a regular hospital for treatment.
The test results showed that NFX-179 gel at a dose of 0.
5% had the best inhibitory effect on p-ERK, a biomarker that drives the growth of subcutaneous neurofibromas, reducing the level of p-ERK by 47%.
In addition, after 28 days of treatment, the experimental group observed a reduction in the tumor volume of patients, and 22% of the patients in the 0.
5% NFX-179 group reduced their tumor volume by an average of at least 50%.
NFlection plans to launch a Phase 2b clinical trial in the second half of this year to study the impact of longer treatment time on tumor shrinkage.
This study provides hope for NF1 patients who suffer from pain or disfigurement of the mass.
NFX-179 gel is designed to deliver a MEK inhibitor that is easily metabolically degraded to tumors by topical application on the skin surface to inhibit the excessive activation of the Ras/Raf/MEK/ERK pathway in these tumors.
NF1 is caused by mutations in the NF1 gene that synthesizes neurofibromin (neurofibromin), which can disrupt the RAS-RAF-MEK-ERK signal and lead to tumor growth.
NFX-179 can be degraded naturally in the human circulation, so it can locally inhibit the RAS-RAF-MEK-ERK signaling pathway while reducing the systemic side effects of MEK inhibitors.
In this multicenter, randomized double-blind, parallel grouping, placebo-controlled Phase 2a clinical trial, 48 patients were randomized to receive 0.
05%, 0.
15%, or 0.
5% NFX-179 gel or placebo once a day 28 days.The primary endpoints are safety, tolerability, and inhibition of p-ERK levels in tumors.
p-ERK is a key biomarker known to promote the growth of subcutaneous neurofibromas.
The test results show that, compared with the control group, 0.
5% and 0.
15% NFX-179 have statistically significant inhibitory effects on p-ERK.
0.
5% and 0.
15% of NFX-179 reduced p-ERK by 47% (p=0.
0001) and 26% (p=0.
04), respectively.
The 0.
05% NFX-179 in the lowest dose group reduced p-ERK by 10%, which was not statistically different from the control group (p=0.
4).
In addition, the secondary endpoint of the study showed that despite only 28 days of treatment, the patient's tumor volume had decreased.
The tumor volume in the 0.
5% NFX-179 group was reduced by an average of 17% compared with baseline (8% in the control group, p=0.
073).
In the 0.
5% NFX-179 gel group, 22% of patients had an average reduction in tumor volume of at least 50% (6% in the control group, p=0.
051).
Image source: In terms of safety and tolerance of 123RF, NFX-179 gel is well tolerated.
No serious adverse events were reported, and no adverse events were classified as related to NFX-179.
All adverse events were mild to moderate, with similar frequency in the experimental group and the control group.
Unlike systemic MEK inhibitors that can cause severe acne-like rashes, no acne-like rashes were observed during NFX-179 treatment.
Cutaneous neurofibromas are tumors that grow from small nerves in or under the skin.
Large or small masses usually appear around puberty.
Patients with NF1 usually develop more cutaneous neurofibromas as they age.
They do not become malignant tumors, but they may cause disfigurement, itching or pain.
They may also cause serious problems such as depression and isolation, and may require surgical removal. ▲ NFlection’s Chief Medical Officer, Dr.
Guy Webster (Image source: NFlection’s official website) NFlection’s Chief Medical Officer, Dr.
Guy Webster, said, “We are very satisfied with these data.
They prove that NFX-179 gel has good tolerance and can Induce cutaneous neurofibromas to produce clinically significant p-ERK inhibition.
Whether it is the powerful p-ERK biomarker data or the early trend of tumor volume reduction after only 28 days of treatment, all support our hypothesis that NFX- 179 gel is an important new therapy for patients with NF1.
We look forward to advancing the clinical research of NFX-179 gel to Phase 2b to determine the effect of longer treatment time on the regression of cutaneous neurofibromas.
"For more rare disease related knowledge and research progress, please click to visit our mini program reference materials: [1] 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.
Retrieved May 26, 2021, from -reduction-in-p-erk-a-key-biomarker-driving-neurofibroma-tumor-growth-in-positive-data-from-a-28-day-phase-2a-clinical-trial-301299521.
htmlNote: This article aims to introduce the progress of medical and health research, not to recommend treatment plans.
If you need guidance on treatment plans, please go to a regular hospital for treatment.
