LG Chem wins FDA approval for clinical trial of drug targeting untreated cancer mutation

LG Chem has entered one of oncology's most underserved areas — a cancer mutation for which no approved treatment currently exists, leaving patients with severely limited options for extending survival.

The company said Tuesday it had received final approval from the US Food and Drug Administration for an investigational new drug application to begin a Phase 1/2 clinical trial of its anticancer drug candidate LG00313112.

LG Chem secured global exclusive development and commercialization rights to LG00313112 — excluding Greater China (mainland China, Hong Kong, Macao and Taiwan) — through a licensing agreement with US-based Frontier Medicines in April. The compound is the basis of the newly approved trial.

LG00313112 targets the TP53 Y220C mutation, found in 1 to 3 percent of all cancer patients. The drug works by stabilizing the p53 protein, which becomes structurally unstable due to the mutation, and restoring its tumor-suppressing function to normal levels. LG Chem said the compound is the first in its class to use a covalent-binding design, which the company expects will deliver stable target engagement and sustained therapeutic effect.

Preclinical studies showed superior antitumor efficacy and durable drug response even at low doses. Strong anticancer activity was also confirmed in tumor models carrying concurrent KRAS mutations, which frequently co-occur across multiple cancer types.

According to cancer genomic data from The Cancer Genome Atlas, compiled by the US National Cancer Institute, patients with TP53 gene mutations have a median post-treatment survival of just 29 months — less than half the 63-month median seen in patients without the mutation. Despite this, no targeted therapy for the mutation has been approved anywhere in the world.

To shorten the global development timeline, LG Chem designed a combined Phase 1/2 protocol integrating both stages under a single study plan, allowing early confirmation of dosing and efficacy. The Phase 1 portion will enroll patients with advanced solid tumors — including ovarian, lung and breast cancers — carrying the TP53 Y220C mutation, and will evaluate safety, tolerability, the recommended Phase 2 dose and preliminary efficacy. A full efficacy assessment will follow in Phase 2.

"We will improve the probability of success in drug development by efficiently identifying patients most likely to respond through a biomarker-driven precision medicine approach," said Kim Hye-jin, head of LG Chem's clinical development group. "We will accelerate development of LG00313112 so that cancer patients with limited treatment options can live longer, healthier lives."