Listed on NCCN within 15 days of FDA approval… Reinforced US prescription base, non-biliary cancer response rate of 37%... Challenge for 'the second Keytruda', undergoing clinical trials for all types of cancer, including pancreatic cancer
HLB (028300)'s new drug for bile duct cancer, 'Lipic2' (ingredient name: lirafugratinib), was listed in the National Comprehensive Cancer Network (NCCN) treatment guidelines following approval by the U.S. Food and Drug Administration (FDA). It was included in the main guidelines that US medical staff refer to when selecting anticancer drugs, strengthening the local prescription base.
Clinical trials are also underway to expand HLB's indications beyond cholangiocarcinoma to other solid cancers. By targeting abnormalities in the FGFR2 gene, we are verifying the effectiveness of treatment in various cancer types, including pancreatic cancer, and are working to develop a treatment for any type of cancer.
#### NCCN listing within 15 days of FDA approval… as a main treatment option
The National Comprehensive Cancer Network (NCCN) included Lipic2 as a follow-up treatment option for bile duct cancer patients with FGFR2 gene fusion or rearrangement in the biliary tract cancer treatment guidelines (Version 2.2026) revised on the 8th. It has been 15 days since it received FDA approval on September 23rd.
The recommendation grade is Category 2A, which is the same as the existing FGFR targeting treatments, pemigatinib and futivatinib. Category 2A means that experts who reviewed the clinical evidence agreed to recommend the treatment.
NCCN stated that Lipictu, pemigatinib, and futivatinib are recommended over erdafitinib, another FGFR-targeted treatment. Although priority was not given among the three treatments, it is noteworthy that Refic2 was included in the list of recommendations alongside existing treatments.
[Source: USA NCCN, lirafugratinib listed as a follow-up treatment option in the biliary tract cancer treatment guidelines.]
Repick2 received FDA approval for patients with previously treated FGFR2 fusion/rearrangement cholangiocarcinoma. In the global phase 1 and 2 clinical trial ReFocus, which served as the basis for approval, the objective response rate (ORR) was approximately 46% and the median response duration was 11.8 months.
This NCCN listing can serve as evidence to support the actual prescription of Refic2 in the United States. NCCN is a widely used practice guideline in cancer treatment settings in the United States, which doctors refer to when selecting an appropriate anticancer drug for a patient.
In the United States, doctors can prescribe anticancer drugs if there is medical evidence, even if the cancer is not approved by the FDA. This is called 'off-label' prescription. For off-label use of anticancer drugs, authorized medical data such as the NCCN drug list are sometimes used as the basis for determining insurance coverage.
In the future, if clinical evidence and related recommendations are secured for other cancer types, the expansion of RepickTwo's prescriptions may also gain momentum. The current NCCN listing is a recommendation for cholangiocarcinoma, and insurance coverage for other cancer types requires separate evidence and review.
#### Bile duct cancer is just the beginning... Lipic2 pursues cancer-agnostic strategy followed by Keytruda
The next step that HLB is pursuing is the development of a treatment for 'any type of cancer.' This is a method that targets patients with specific genetic abnormalities regardless of the organ in which the cancer occurred.
A representative example is MSD's cancer immunotherapy drug Keytruda. Keytruda has expanded its indications to various cancer types, and has also secured indications for any cancer type that can be used regardless of the cancer site based on specific biomarkers.
Refic2 is also expanding its treatment range focusing on FGFR2 gene abnormalities. It is a targeted anti-cancer drug that selectively inhibits FGFR2, blocking abnormal signal transmission caused by fusion and rearrangement of the corresponding gene. Abnormalities in the FGFR2 gene have been found not only in bile duct cancer but also in other solid cancers such as pancreatic cancer and breast cancer, raising the possibility of expanding the indications.
In fact, even in early clinical trials, treatment responses were seen in patients other than cholangiocarcinoma. According to a study published in the international academic journal 'Clinical Cancer Research' in 2026, the objective response rate of Lipic2 in 44 patients with non-biliary tract cancer with FGFR2 rearrangement was reported to be 37%, and the median response duration was 7.3 months.
In particular, responses were confirmed in pancreatic cancer, which has limited treatment options. In the ReFocus clinical trial published at the American Association for Cancer Research (AACR) in 2023, two out of five pancreatic cancer patients with FGFR2 gene abnormalities responded to treatment. Although it is difficult to determine the effectiveness due to the small number of patients, it served as a basis for further verification in other solid cancers, including pancreatic cancer.
Based on these initial results, HLB's U.S. subsidiary Elevar Therapeutics launched the global phase 2 clinical trial 'ReFocus202′ (NCT07359820) last June. We will recruit 30 patients with non-cholangiocarcinoma solid tumors with FGFR2 fusion/rearrangement and evaluate the efficacy and safety of Repick2. The first patient medication was administered at Samsung Seoul Hospital.
[Source: ClinicalTrials.gov, Phase 2 clinical trial registration status for Repick2 regardless of cancer type]
According to Clinical Trials, a U.S. clinical trial registration site, the first evaluation of ReFocus202 is expected to be completed in September 2027. Elevar plans to evaluate five or more patients in at least seven cancer types by combining existing clinical data and new patient data. This is to confirm whether the treatment is effective in patients with FGFR2 gene abnormalities even if the site where the cancer occurs is different.
Repick2 is currently approved by the FDA for bile duct cancer patients with FGFR2 fusion/rearrangement. If the treatment effect is confirmed in other solid cancers, additional clinical evidence will be provided to expand indications in the future.
HLB, which strengthened its prescription base in the U.S. with FDA approval last September and listing in the NCCN treatment guidelines this month, is now focusing on expanding Repick2's treatment area. Based on the results achieved in bile duct cancer, we plan to verify the treatment possibility in other solid cancers, such as pancreatic cancer.
