Lirafugratinib

Lyrfigtu demonstrated a confirmed ORR of 46% and mDOR of 11.8 months, with a predictable and manageable safety profile CCA, also known as bile duct cancer, is diagnosed in about 8,000 people in the U.S. each year FORT LEE, N.J., Sept. 23, 2026 (GLOBE NEWSWIRE) -- Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for cancer patients, today announced the U.S. Food and Drug Administration (FDA) granted approval for LYRFIGTU™ (lirafugratinib) as a treatment option for patients with cholangiocarcinoma (CCA) with FGFR2 fusion or other rearrangement. CCA, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. newly diagnosed each year, according to the American Cancer Society. “The FDA approval of Lyrfigtu introduces a vital second-line treatment option for patients living with CCA and their families,” said Dong-Gun Kim, chief executive officer of Elevar. “Our team is thrilled to bring this therapy to market and focused on getting Lyrfigtu to doctors and patients as quickly as possible, while continuing to deliver on our mission to improve treatment for patients whose therapeutic options were previously limited.” Lyrfigtu is expected to be available to patients in the U.S. by Q4 2026. In the Phase 1/2 ReFocus trial ( NCT04526106 ), Lyrfigtu demonstrated a confirmed objective response rate (ORR) of 46% and a median duration of response of 11.8 months in patients with the proposed indication. Median progression-free survival was 11.3 months (95% CI, 9.2, 14.8), with a 12-month rate of 49.2%. Its safety pro the clinical data was shown to be predictable and manageable through dose adjustments. Lyrfigtu in March 2026 was given a priority review designation by the FDA, which is reserved for drugs that if approved would lead to “significant improvements in the safety or effectiveness of the treatment” of a serious condition. Expert Perspectives on Lyrfigtu Approval Lipika Goyal, M.D., lead author on the ReFocus study and director of gastrointestinal oncology at the Stanford Cancer Center: “ Lyrfigtu’s unique, irreversible, covalent-binding mechanism enables potent and sustained inhibition of FGFR2, including many resistance mutations that can emerge with earlier FGFR inhibitors. And unlike earlier pan-FGFR inhibitors, it selectively targets FGFR2 while minimizing off-isoform toxicities. This FDA approval brings an important next-generation precision medicine option directly to patients with advanced bile duct cancer.” Robin Kate Kelley, M.D., professor of medicine in the division of Hematology & Oncology, Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco: “ Lyrfigtu achieved remarkably deep and durable treatment responses in patients on the ReFocus trial. Beyond the confirmed ORR of 45.7% which speaks for itself, I’ve witnessed, first-hand, many of my own patients who were able to regain meaningful quality of life and precious time with their loved ones, owing to the robust tumor shrinkage they achieved on this treatment.” Alison Schram, M.D., gynecologic medical oncologist at Memorial Sloan Kettering Cancer Center: "These results represent a meaningful step forward for patients with FGFR2-fusion-positive CCA, a disease where treatment options have historically been limited and outcomes poor. Lyrfigtu demonstrated durable responses and a manageable safety profile, providing a much-needed treatment option for patients. The results also reinforce the importance of molecular testing at diagnosis so that patients can be matched to therapies most likely to benefit them." Elevar continues to evaluate Lyrfigtu for other indications in ongoing clinical development programs, including studies in other FGFR2-altered solid tumors. Any future indications will be subject to regulatory review and approval. Important Safety Information for LYRFIGTU™ (lirafugratinib) Warnings and Precautions: Ocular Toxicity LYRFIGTU can cause retinal pigment epithelial detachment (RPED), which may cause symptoms such as blurred vision. Among 385 patients who received LYRFIGTU, RPED occurred in 31% of patients, including Grade 3 events in 1.8%. The median time to first onset was 57 days. RPED led to dose interruption in 15% of patients and dose reduction in 10%. Perform a comprehensive ophthalmological examination, including OCT of the macula, prior to initiation of therapy, every 2 months for the first 13 months, and every 4 months thereafter. For onset of visual symptoms, obtain ophthalmologic evaluation urgently, with follow-up every 3 weeks until resolution or discontinuation of LYRFIGTU. Withhold, reduce the dose, or discontinue LYRFIGTU based on severity. Among 385 patients who received LYRFIGTU, blurred vision occurred in 18% of patients with Grade 3 events in 1.3% of patients. Dry eye occurred in 38% of patients. Treat patients with ocular demulcents as needed. Among 385 patients who received LYRFIGTU, corneal toxicity/keratitis occurred in 11% of patients. Treat patients with ocular demulcents as needed. Hyperphosphatemia and Soft Tissue Mineralization LYRFIGTU can cause hyperphosphatemia leading to soft tissue mineralization, calcinosis, nonuremic calciphylaxis, and vascular calcification. Hyperphosphatemia occurred in 21% of patients. The median time to onset was 15 days. Hyperphosphatemia led to dose interruption in one (0.3%) patient and no permanent discontinuation. Monitor serum phosphate throughout treatment and manage as clinically appropriate. Embryo-Fetal Toxicity Based on its mechanism of action and findings from animal studies, LYRFIGTU can cause fetal harm or loss of pregnancy when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment and for 3 months after the last dose. Contraindications: None. Adverse reactions: Serious adverse reactions occurred in 32% of patients. Serious adverse reactions reported in ≥2% of patients were infection (6%), pneumonia (3.4%), fatigue (2.6%), and hemorrhage (2.6%). A fatal adverse reaction of hemorrhage occurred in one patient. The most common adverse reactions (≥20%) were nail toxicity, palmar-plantar erythrodysesthesia syndrome, stomatitis, alopecia, dry eye, dry mouth, fatigue, dysgeusia, retinal pigment epithelial detachment, constipation, dry skin, infection, rash, abdominal pain, hemorrhage, blurred vision, diarrhea, musculoskeletal pain, nausea, and decreased appetite. The most common laboratory abnormalities (≥20%) were increased phosphate, increased alanine aminotransferase, increased creatinine, decreased hemoglobin, decreased sodium, decreased lymphocytes, increased blood bilirubin, increased aspartate aminotransferase, decreased platelets, increased glucose, decreased leukocytes, increased alkaline phosphatase, decreased albumin, decreased phosphate, decreased neutrophils, decreased bicarbonate. Reporting Suspected Adverse Events: To report SUSPECTED ADVERSE REACTIONS, contact Elevar Therapeutics at 1-866-4ELEVAR or contact the FDA at 1-800-FDA-1088 , or visit . Please see full Prescribing Information , including Patient Information, for Lyrfigtu . For more information about Elevar, visit ElevarTX.com . About LYRFIGTU™ (Lirafugratinib) Lyrfigtu (lirafugratinib, aka RLY-4008) is a potent, selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Lyrfigtu is currently being evaluated in a clinical trial to enroll additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 fusion or rearrangement, who have not been treated with prior FGFR inhibitors. Elevar has an exclusive license to lirafugratinib from Relay Therapeutics, Inc. for commercialization worldwide. About Elevar Therapeutics Elevar Therapeutics, Inc. is a fully integrated biopharmaceutical company built on the promise of elevating treatment outcomes for cancer patients. A subsidiary of HLB Co., Ltd. based in Fort Lee, New Jersey, Elevar’s expertise is rooted in oncology and grounded in compassion. Our team of experienced scientists and industry leaders is highly focused on identifying and developing medicines for complex yet under-treated health conditions, with a shared goal of making a meaningful difference in the lives of patients. For more information, follow us on LinkedIn and Twitter or visit ElevarTX.com. Forward-Looking Statements This press release contains forward-looking statements including, without limitation, statements regarding the anticipated commercial launch, market acceptance, patient access, future development activities, regulatory obligations, commercial opportunities, and the expected benefits and potential of Lyrfigtu. These forward-looking statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to materially differ from those expressed or implied by such statements. Such risks and uncertainties include, among others, the timing and success of commercialization activities; manufacturing capacity and product supply; market acceptance; the availability and adequacy of reimbursement coverage; competition from existing or future therapies; safety findings; post-marketing regulatory requirements and commitments; and other risks and uncertainties affecting Elevar's business and operations. FDA approval does not guarantee commercial success or any particular level of market adoption, reimbursement, revenue generation, or clinical outcomes in broader patient populations. Except as required by applicable law, Elevar undertakes no obligation to update or revise any forward-looking statements to reflect events or circumstances after the date of this press release. Additional information regarding these and other risks and uncertainties can be found in HLB Co., Ltd.'s periodic reports, material event disclosures, and other public filings made with the Korea Exchange ("KRX") and the Financial Supervisory Service's Data Analysis, Retrieval and Transfer System ("DART"). Readers should review those disclosures caref ully, including risk factors relating to product commercialization, regulatory compliance, reimbursement, manufacturing, competition, and financial performance. All forward-looking statements contained in this press release are expressly qualified by these cautionary statements. Media Contact: Rosemary Ostmann RoseComm Cell: (201) 615-7751 US-LIRA-26-0022 09/26

On September 23, 2026, the Food and Drug Administration approved lirafugratinib (Lyrfigtu, Elevar Therapeutics, Inc.), a kinase inhibitor, for the treatment of adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement.Full prescribing information for Lyrfigtu will be posted on Drugs@FDA.Efficacy and SafetyEfficacy was evaluated in REFOCUS (NCT04526106), a multicenter, open-label, single-arm trial in 116 patients with unresectable or metastatic cholangiocarcinoma who were naïve to FGFR inhibitor treatment and had received prior chemotherapy or chemoimmunotherapy. Patients received lirafugratinib 70 mg once daily until disease progression or unacceptable toxicity.The major efficacy outcome measures were objective response rate (ORR) and duration of response (DOR) as determined by an independent review committee according to RECIST v1.1. ORR was 46% (95% CI: 36, 55) and median DOR was 11.8 months (95% CI: 7.5, 13.0).The prescribing information includes warnings and precautions for ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity.Recommended DosageThe recommended lirafugratinib dose is 70 mg orally once daily until disease progression or unacceptable toxicity.This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.This application was granted priority review. Lirafugratinib received breakthrough therapy and orphan drug designations. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email  OncProjectFacilitate@fda.hhs.gov.