Voronoi Unveils Clinical Results of EGFR-Mutant Lung Cancer Drug VRN11 at ESMO
2026.10.27
■ Complete remission of brain lesions in 20% of patients with brain metastases; intracranial PFS of 9.1 months
■ Objective response in 7 of 8 patients (87.5%) with the C797S mutation; PFS of 11 months
■ First-line treatment development under way based on brain penetration and clinical results
Voronoi (310210) announced on the 27th that it presented Phase 1 clinical results of VRN11, its treatment for EGFR-mutant non-small cell lung cancer, as a poster at the European Society for Medical Oncology (ESMO 2026) held in Madrid, Spain.
In this presentation, VRN11 produced multiple cases in which brain lesions disappeared completely in patients with brain metastases, and it also showed antitumor effects in patients who had developed resistance to existing EGFR-targeted therapies. Based on these clinical results, the company is running first-line clinical trials in multiple countries, targeting patients with no prior treatment in addition to patients with prior treatment experience.
● Brain tumors disappeared completely in 4 of 20 patients with brain metastases
Among 20 C797S-negative patients with brain metastases who received 160 mg or more, the intracranial disease control rate was 95% (19/20). Of these, 4 patients (20%) showed intracranial complete response (intracranial CR), in which the brain lesions disappeared completely.
The median intracranial progression-free survival (iPFS), which indicates the duration of dosing without brain lesion worsening, was 9.1 months. The 6-month and 12-month intracranial progression-free survival rates were 79.8% and 49.9%, respectively. Notably, 85% of this group were patients enrolled after treatment with a third-generation EGFR-targeted therapy.
A 71-year-old woman, presented as a representative case, had received three prior treatments including Tagrisso and Lazcluze. Her brain lesion disappeared completely at week 4 of VRN11 treatment, and systemic partial response was confirmed at week 8.
[Image caption] Case of an EGFR-mutant non-small cell lung cancer patient with brain metastases (71-year-old woman)
By contrast, Tagrisso, a third-generation targeted therapy, was reported in a trial of patients who developed brain metastases after first- or second-generation targeted therapy to have an iPFS of 3.5 months, a 6-month intracranial progression-free survival rate of 31%, and a complete response rate of 0%. The treatment effect of VRN11 in patients with brain metastases was therefore even more pronounced.
● Antitumor effect confirmed even in patient groups with limited treatment options
VRN11 also showed antitumor effects in patients with limited further treatment options after existing EGFR-targeted therapies, including Tagrisso.
Among 8 patients who received 160 mg or more and had the C797S resistance mutation confirmed after prior treatment, 7 showed tumor reduction. The median progression-free survival (mPFS) was 11.0 months, and the 6-month progression-free survival rate was 66.7%.
In one disclosed case, a 63-year-old man had received six prior treatments, and tumor reduction was confirmed in all 4 lesions in the lungs and lymph nodes at week 4 of VRN11 treatment.
[Image caption] Case of an EGFR C797S-mutant non-small cell lung cancer patient (63-year-old man)
Patients without a confirmed C797S mutation also recorded an mPFS of 7.3 months. Among 17 patients who received 320 mg or more, the 6-month progression-free survival rate was 52.5%. Of these, 82.4% were patients enrolled after treatment with a third-generation EGFR-targeted therapy.
● Grade 3 or higher treatment-related adverse events at 2%; high safety with zero treatment discontinuations
Among all 82 patients, grade 3 or higher treatment-related adverse events were only 2%. No dose-limiting toxicity occurred during dose escalation up to 560 mg, and there were no treatment discontinuations due to treatment-related adverse events.
In particular, in the group that received 160 mg or more, 90% of all dosing days involved adverse events of grade 1 or lower, and 45% of them involved no treatment-related adverse events. No pattern of accumulating grade 3 or higher adverse events was observed even with longer treatment duration.
The adverse events mainly reported were mild skin and gastrointestinal symptoms known from EGFR-targeted therapies, such as diarrhea, itching, and rash, and no lung-related adverse events of special interest, such as interstitial lung disease, were reported.
● Expanding first-line treatment development based on excellent brain penetration
VRN11 is designed so that the drug can reach the brain effectively. Measured in patients, Kp,uu,CSF, an indicator of how well the drug is delivered into the cerebrospinal fluid, was 2.0, about 10 times higher than that of Tagrisso (0.22). Based on this, the company calculated that VRN11 at 320 mg achieves target engagement about 4 times higher than Tagrisso systemically and about 40 times higher in the central nervous system.
The company is expanding development into the first-line treatment setting based on the efficacy, target engagement, and safety data confirmed so far. For the Phase 1b dose optimization evaluation, it selected 320 mg as the low dose, and the high dose for comparison will be decided later. In Phase 1b, the company also plans to run a first-line cohort, a C797X cohort for patients receiving second-line or later treatment, and a central nervous system metastasis cohort, among others. The first-line cohort is currently enrolling patients.
A Voronoi representative said, "VRN11 has confirmed antitumor effects not only in patients who developed resistance after existing targeted therapies but also in patients with brain metastases. Based on these results, we plan to expand development into the first-line treatment setting and continue to confirm its potential as a new therapy that can overcome the limitations of existing treatments."
■ Objective response in 7 of 8 patients (87.5%) with the C797S mutation; PFS of 11 months
■ First-line treatment development under way based on brain penetration and clinical results
Voronoi (310210) announced on the 27th that it presented Phase 1 clinical results of VRN11, its treatment for EGFR-mutant non-small cell lung cancer, as a poster at the European Society for Medical Oncology (ESMO 2026) held in Madrid, Spain.
In this presentation, VRN11 produced multiple cases in which brain lesions disappeared completely in patients with brain metastases, and it also showed antitumor effects in patients who had developed resistance to existing EGFR-targeted therapies. Based on these clinical results, the company is running first-line clinical trials in multiple countries, targeting patients with no prior treatment in addition to patients with prior treatment experience.
● Brain tumors disappeared completely in 4 of 20 patients with brain metastases
Among 20 C797S-negative patients with brain metastases who received 160 mg or more, the intracranial disease control rate was 95% (19/20). Of these, 4 patients (20%) showed intracranial complete response (intracranial CR), in which the brain lesions disappeared completely.
The median intracranial progression-free survival (iPFS), which indicates the duration of dosing without brain lesion worsening, was 9.1 months. The 6-month and 12-month intracranial progression-free survival rates were 79.8% and 49.9%, respectively. Notably, 85% of this group were patients enrolled after treatment with a third-generation EGFR-targeted therapy.
A 71-year-old woman, presented as a representative case, had received three prior treatments including Tagrisso and Lazcluze. Her brain lesion disappeared completely at week 4 of VRN11 treatment, and systemic partial response was confirmed at week 8.
[Image caption] Case of an EGFR-mutant non-small cell lung cancer patient with brain metastases (71-year-old woman)
By contrast, Tagrisso, a third-generation targeted therapy, was reported in a trial of patients who developed brain metastases after first- or second-generation targeted therapy to have an iPFS of 3.5 months, a 6-month intracranial progression-free survival rate of 31%, and a complete response rate of 0%. The treatment effect of VRN11 in patients with brain metastases was therefore even more pronounced.
● Antitumor effect confirmed even in patient groups with limited treatment options
VRN11 also showed antitumor effects in patients with limited further treatment options after existing EGFR-targeted therapies, including Tagrisso.
Among 8 patients who received 160 mg or more and had the C797S resistance mutation confirmed after prior treatment, 7 showed tumor reduction. The median progression-free survival (mPFS) was 11.0 months, and the 6-month progression-free survival rate was 66.7%.
In one disclosed case, a 63-year-old man had received six prior treatments, and tumor reduction was confirmed in all 4 lesions in the lungs and lymph nodes at week 4 of VRN11 treatment.
[Image caption] Case of an EGFR C797S-mutant non-small cell lung cancer patient (63-year-old man)
Patients without a confirmed C797S mutation also recorded an mPFS of 7.3 months. Among 17 patients who received 320 mg or more, the 6-month progression-free survival rate was 52.5%. Of these, 82.4% were patients enrolled after treatment with a third-generation EGFR-targeted therapy.
● Grade 3 or higher treatment-related adverse events at 2%; high safety with zero treatment discontinuations
Among all 82 patients, grade 3 or higher treatment-related adverse events were only 2%. No dose-limiting toxicity occurred during dose escalation up to 560 mg, and there were no treatment discontinuations due to treatment-related adverse events.
In particular, in the group that received 160 mg or more, 90% of all dosing days involved adverse events of grade 1 or lower, and 45% of them involved no treatment-related adverse events. No pattern of accumulating grade 3 or higher adverse events was observed even with longer treatment duration.
The adverse events mainly reported were mild skin and gastrointestinal symptoms known from EGFR-targeted therapies, such as diarrhea, itching, and rash, and no lung-related adverse events of special interest, such as interstitial lung disease, were reported.
● Expanding first-line treatment development based on excellent brain penetration
VRN11 is designed so that the drug can reach the brain effectively. Measured in patients, Kp,uu,CSF, an indicator of how well the drug is delivered into the cerebrospinal fluid, was 2.0, about 10 times higher than that of Tagrisso (0.22). Based on this, the company calculated that VRN11 at 320 mg achieves target engagement about 4 times higher than Tagrisso systemically and about 40 times higher in the central nervous system.
The company is expanding development into the first-line treatment setting based on the efficacy, target engagement, and safety data confirmed so far. For the Phase 1b dose optimization evaluation, it selected 320 mg as the low dose, and the high dose for comparison will be decided later. In Phase 1b, the company also plans to run a first-line cohort, a C797X cohort for patients receiving second-line or later treatment, and a central nervous system metastasis cohort, among others. The first-line cohort is currently enrolling patients.
A Voronoi representative said, "VRN11 has confirmed antitumor effects not only in patients who developed resistance after existing targeted therapies but also in patients with brain metastases. Based on these results, we plan to expand development into the first-line treatment setting and continue to confirm its potential as a new therapy that can overcome the limitations of existing treatments."