Elevar Therapeutics Inc., a majority-owned subsidiary of HLB Co., Ltd. (028300.KQ), announced that it has submitted a New Drug Application to the U.S. Food and Drug Administration for its investigational drug Lirafugratinib as a second-line treatment option for cholangiocarcinoma patients with Fibroblast Growth Factor Receptor 2 fusion or FGFR2 rearrangement.
The New Drug Application (NDA) submission was supported by positive clinical data from the phase 1/2 ReFocus trial in which lirafugratinib demonstrated clinically meaningful results.
Cholangiocarcinoma is a rare type of bile duct cancer, with about 8,000 people in the U.S. diagnosed each year, according to the American Cancer Society.
Lirafugratinib (RLY-4008) is a selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers.
Phase 1/2 ReFocus Trial
ReFocus is an open-label, Phase 1/2 trial designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and antineoplastic activity of Lirafugratinib in patients with unresectable or metastatic cholangiocarcinoma and other solid tumours.
The study consists of 4 parts: a dose escalation (Part 1), a dose expansion (Part 2), an extension (Part 3) and a rollover (Part 4). (Source-clinicaltrials.gov)
Trial Findings
An independent review committee-assessed objective response rate was 46.5%, and the median duration of response was 11.8 months, where 76.2% of responses lasted more than 6 months.
Median progression-free survival was 11.3 months with a 12-month rate of 49.2%.
Median overall survival was 22.8 months, with a 12-month rate of 74.6%. The disease control rate was 96.5%.
The most common severe treatment-related adverse events were hand-foot skin reactions (32.8%) and mouth sores (12.1%). These adverse events led to dose reductions in 75.9% of patients, dose interruptions in 82.8%, and treatment discontinuation in 4.3%.
Lirafugratinib's Journey So Far
The abstract "Efficacy and safety of lirafugratinib in FGFRi-naïve cholangiocarcinoma (CCA) patients harbouring FGFR2 fusions/rearrangements (FGFR2 f/r)" was presented earlier this month at the American Society of Clinical Oncology's Gastrointestinal Cancers Symposium. In December 2024, Elevar acquired worldwide rights to develop and commercialise lirafugratinib from Relay Therapeutics.
Earlier that year, Relay Therapeutics met with the FDA to discuss data from the ReFocus trial and potential regulatory pathways, and the FDA recommended the company first file an NDA for FGFR2-driven CCA, followed by a supplemental NDA for FGFR2-altered other solid tumours.
Elevar is also developing lirafugratinib for patients with other FGFR2-altered solid tumours.
"We are excited to work with the FDA as it reviews the submission while simultaneously preparing for a potential commercial launch," said Dong-Gun Kim, the company's chief executive officer.
On Thursday, 028300.KQ traded in the day's range of 63,200 Korean Won and 68,200 Korean Won on KOSDAQ and closed trading at 65,300 Korean Won, down 2.83%.
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Business News
June 19, 2026 16:46 ET Major central banks continued to dominate the economic news flow this week too, led by the Federal Reserve, as they announced their latest policy decisions. The Federal Reserve policy session was in focus as it was the first to be led by the new chief Kevin Warsh. In Europe, central banks of the U.K. and Switzerland announced their rate decisions. In Asia, the Bank of Japan drew attention for its policy moves, while data out of China threw some light on the state of the economy.