Voronoi Demonstrates Efficacy of Low-Dose 'VRN11' in EGFR C797S Brain Metastatic Non-Small Cell Lung Cancer Patients

Voronoi announced on April 28 that it has demonstrated the efficacy of its EGFR-targeted therapy for non-small cell lung cancer, VRN11, in patients with the EGFR C797S mutation, based on early clinical data presented at the American Association for Cancer Research (AACR2025).

At AACR2025, Voronoi revealed that during the dose-escalation phase of the phase 1a clinical trial, a low dose of 40mg was administered to a patient with EGFR C797S double mutation (EGFR L858R/C797S) and brain metastasis. The company reported that, without any adverse effects, a partial response of over 50% was observed in the lung lesion, and a complete response was seen in the brain lesion.

Specifically, the tumor in the lung, which measured 22.2mm, was reduced by more than 50% after two months of treatment. The brain lesion, which was 10.6mm, had completely disappeared after four months.

Currently, VRN11 is set to be administered at a dose of 240mg. The clinical trial is designed to escalate the dose up to 320mg, which is eight times higher than the 40mg dose that demonstrated efficacy. It is expected that the antitumor effect will increase as the dose increases.

Among VRN11's targets, the EGFR C797S mutation is an acquired resistance double mutation that occurs in about 10% of patients with primary EGFR mutations (EGFR Del19 or L858R) who have been treated with third-generation non-small cell lung cancer therapies, including Tagrisso and Leclaza. The annual market size for C797S non-small cell lung cancer therapies is estimated to be approximately 1 trillion KRW.

Currently, there are no approved targeted therapies available for patients with the C797S mutation. The existing second-line treatment for non-small cell lung cancer, which combines pemetrexed (Alimta) and platinum-based cytotoxic chemotherapy, has a progression-free survival (PFS) of four months and a rate of grade 3 or higher adverse events of about 50%. This indicates an unmet medical need for second-line therapies for non-small cell lung cancer. Blueprint Medicines, a Nasdaq-listed US company, was a notable competitor, but it failed to demonstrate efficacy in patients with the EGFR C797S mutation.

Voronoi plans to present additional data on EGFR C797S mutant non-small cell lung cancer patients from the ongoing clinical trial at an international conference in July of this year.

A Voronoi representative stated, "The company's Scientific Advisory Board (SAB) and clinical consultants, composed of specialists in lung cancer and solid tumors, have highly praised the efficacy and safety of VRN11," adding, "Based on the data revealed so far, we strongly recommend pursuing aggressive clinical development with the goal of accelerated approval as a treatment for EGFR C797S mutant non-small cell lung cancer, due to its high single-agent antitumor activity and safety margin."

The representative continued, "We plan to conduct a phase 2 clinical trial in 2026 for accelerated approval, aiming to obtain rapid approval in the United States and Europe."