A Phase 2, Open-Label, Single-Arm Study of Lirafugratinib in Patients With Previously Treated, Unresectable, Locally Advanced or Metastatic Solid Tumors (Excluding Cholangiocarcinoma) With FGFR2 Fusion or Rearrangement

The goal of this clinical trial is to evaluate if lirafugratinib is efficacious and safe to treat adult patients with previously treated, unresectable, locally advanced or metastatic solid tumors (excluding cholangiocarcinoma) harboring FGFR2 fusion or rearrangement.
Participants will:
* Take lirafugratinib regularly as instructed by their study doctor.
* Visit the clinic as instructed for checkups and tests.
* Keep a diary recording each time a dose of lirafugratinib is taken.

Start Date04 Jun 2026

Sponsor / Collaborator

Elevar Therapeutics, Inc.

ISRCTN27020581

/ CompletedPhase 1

A Phase I, open-label, single-dose study designed to assess the absorption, distribution, metabolism and excretion of [14C]-RLY-4008 in healthy male participants

Start Date12 Jul 2022

Sponsor / Collaborator

Elevar Therapeutics, Inc.

NCT04526106

/ CompletedPhase 1/2

A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients With Intrahepatic Cholangiocarcinoma (ICC) and Other Advanced Solid Tumors

This is a Phase 1/2, open-label, FIH study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDy), and antineoplastic activity of RLY-4008, a potent and highly selective FGFR2 inhibitor, in patients with unresectable or metastatic cholangiocarcinoma (CCA) and other solid tumors. The study consists of 4 parts: a dose escalation (Part 1), a dose expansion (Part 2), and an extension (Part 3) and a rollover (Part 4).

Start Date02 Sep 2020

Sponsor / Collaborator

Elevar Therapeutics, Inc.

100 Clinical Results associated with Lirafugratinib

100 Translational Medicine associated with Lirafugratinib

100 Patents (Medical) associated with Lirafugratinib

Literatures (Medical) associated with Lirafugratinib

01 Jun 2026·ANNALS OF ONCOLOGY

Mechanisms of clinical resistance to selective FGFR2 inhibition by lirafugratinib

Article

Author: Nugent, F W ; Hajian, B ; Silveira, V S ; Wan, J ; Chevalier, N ; Corcoran, R B ; Ellis, H ; Kehlmann, A ; Sellers, W R ; Lau, K ; Balasooriya, E R ; Vijay, V ; Juric, D ; Varkaris, A ; Bardeesy, N ; Gritti, I ; Shekhar, M ; Zhen, Y ; Albertelli, L ; Piot, S

BACKGROUND:

Pan-fibroblast growth factor receptor (FGFR) inhibitors, targeting FGFR1-3 or FGFR1-4, are Food and Drug Administration-approved for FGFR2-driven cholangiocarcinoma. However, acquired resistance and dose-limiting toxicities from systemic FGFR inhibition constrain efficacy. Lirafugratinib (RLY-4008), a first-in-class FGFR2-selective inhibitor with activity against resistance-associated FGFR2 kinase domain mutations, shows promise in patients with FGFR2-altered solid tumors (ReFocus trial, NCT04526106). Defining acquired resistance mechanisms to selective FGFR2 targeting is essential for therapeutic development.

PATIENTS AND METHODS:

Circulating tumor DNA (ctDNA) samples from 28 patients treated with lirafugratinib (16 FGFR inhibitor-naive, 12 FGFR inhibitor-refractory) were analyzed using targeted next-generation sequencing. Genomic alterations observed were compared with those reported in prior studies of pan-FGFR inhibitor resistance and validated in preclinical models.

RESULTS:

Polyclonal FGFR2 kinase domain mutations and receptor tyrosine kinase-mitogen activated protein kinase (RTK-MAPK) bypass alterations emerged as common lirafugratinib resistance mechanisms in the FGFR inhibitor-naive context (8/16 and 9/16 patients, respectively). Resistance profiles differed markedly from pan-FGFR inhibitors, with decreased FGFR2 V565F/L and N550H/K mutations, increased M538I and L618F mutations, and more frequent RTK-MAPK bypass alterations. The variant allele fraction was typically higher for FGFR2 kinase domain mutations, consistent with these alterations serving as primary resistance drivers. Preclinical studies confirmed differential sensitivity of these FGFR2 mutations to lirafugratinib. Importantly, lirafugratinib demonstrated clinical efficacy in the FGFR inhibitor-refractory setting, with ctDNA dynamics showing resolution of multiple FGFR2 mutations and persistence or emergence of others.

CONCLUSIONS:

Lirafugratinib retains activity against multiple mutations that confer clinical resistance to pan-FGFR inhibitors. However, diverse resistance mechanisms, including various kinase domain mutations and RTK-MAPK bypass alterations, remain challenges in the treatment of FGFR2-altered tumors, even with selective FGFR2 kinase inhibition.

15 Apr 2026·CLINICAL CANCER RESEARCH

Recurrent Resistance Mutations to Lirafugratinib Inform Treatment Sequencing in FGFR2 -Driven Tumors

Article

Author: Badoual, Cécile ; Nikolaev, Sergey ; Samaniego, Juliette ; Bourien, Héloise ; Barbé, Rémy ; Nicotra, Claudio ; Alonso-Garcia, Inmaculada ; Boilève, Alice ; Nakazawa, Seshiru ; Jang, Dong Man ; Rodriguez, Julieta ; Cotteret, Sophie ; Bernard, Elsa ; Soares, Miguel ; André, Fabrice ; Italiano, Antoine ; Loriot, Yohann ; Friboulet, Luc ; Ducreux, Michel ; Mosele, Fernanda ; Nobre, Catline ; Beshiri, Kristi ; Da Silva, Alice ; Olaussen, Ken A. ; Facchinetti, Francesco ; Alonso-De-Castro, Beatriz ; Eck, Michael J. ; Brayé, Floriane ; Bigot, Ludovic ; Smolenschi, Cristina ; Delavigne, Mathis ; Ngo-Camus, Maud ; Tselikas, Lambros ; Hollebecque, Antoine ; Méteau, Mélissandre ; Vasseur, Damien

Abstract:

Purpose::

The use of reversible fibroblast growth factor receptor 2 (FGFR) inhibitors leads to the emergence of “undruggable” FGFR2 kinase domain mutations, hampering sequential treatment strategies. Lirafugratinib and futibatinib are irreversible FGFR inhibitors with the most promising clinical activity against FGFR2-driven tumors.

Experimental Design::

We characterized resistance to lirafugratinib with circulating tumor DNA, tissue whole-exome sequencing, and bulk RNA sequencing in 30 patients with FGFR2-driven cancers, treated in the phase I/II ReFocus trial (NCT04526106) and enrolled in the UNLOCK program at Gustave Roussy.

Results::

Among the 30 patients included, 18 (60%) had intrahepatic cholangiocarcinoma and 12 (40%) had other tumor types. Twenty-two patients (73%) were FGFR inhibitor–naïve. Among those experiencing primary resistance to lirafugratinib, we identified potential resistance mechanisms in five of six pretreatment samples. Patients with acquired lirafugratinib resistance manifested an unprecedented emergence of FGFR2 mutations in the M538 and/or L618 residues of the kinase domain, documented in 11 of 16 cases (69%). Compared with futibatinib resistance, FGFR2 molecular brake (N550) and gatekeeper (V565) mutations were rare. Leveraging the spectrum of FGFR2 kinase domain mutations at resistance to lirafugratinib and futibatinib, respectively, we identified the complementarity of the two irreversible inhibitors. On the basis of viability assays in FGFR2::BICC1–dependent Ba/F3 models and in vivo studies on patient-derived xenografts, we propose treatment sequences with the two agents. After lirafugratinib progression, three patients received futibatinib and experienced prolonged disease response.

Conclusions::

The complementary activity of lirafugratinib and futibatinib against FGFR2 kinase domain mutations supports their sequential use when precise resistance mutations are detected in patients.

01 Apr 2026·DRUG METABOLISM AND DISPOSITION

Lirafugratinib attenuates ABCG2-dependent drug efflux and restores chemosensitivity in multidrug-resistant nonsmall cell lung cancer cells

Article

Author: Lin, Bing-Huan ; Wu, Chung-Pu ; Li, Yen-Ching ; Wu, Yu-Shan ; Hung, Tai-Ho ; Ambudkar, Suresh V ; Chang, Yu-Tzu ; Murakami, Megumi

The therapeutic efficacy of many anticancer drugs is frequently compromised by multidrug resistance, a process often driven by elevated activity of ATP-binding cassette (ABC) efflux pumps in tumor cells. These membrane transporters actively expel chemotherapeutic agents in an ATP-dependent fashion, thereby lowering intracellular drug exposure and diminishing treatment responses. The shortage of clinically approved agents capable of overcoming ABC transporter-mediated resistance highlights the urgency of identifying alternative approaches, including the repurposing of small-molecule targeted therapies to inhibit drug efflux. Here, we examine lirafugratinib, an orally available and highly selective fibroblast growth factor receptor 2 inhibitor currently undergoing clinical evaluation for intrahepatic cholangiocarcinoma and other solid tumors, as a potential modulator of ABCG2-mediated drug resistance. Our findings reveal that lirafugratinib, at concentrations that do not impair cell viability, restores sensitivity to ABCG2-substrate chemotherapeutic drugs and enhances apoptosis in ABCG2-overexpressing nonsmall cell lung cancer cells. Mechanistically, lirafugratinib impedes the efflux capability of ABCG2 without altering its protein expression. ATPase experiments and molecular docking analysis further indicate that lirafugratinib engages the drug-binding region of ABCG2 and modulates its ATP hydrolysis cycle. Collectively, these results suggest that lirafugratinib may be utilized as a chemosensitizing agent to counteract multidrug resistance in nonsmall cell lung cancer with high ABCG2 expression, supporting its evaluation in combination therapies. Further in vivo studies and clinical trials are required to substantiate its clinical applicability. SIGNIFICANCE STATEMENT: This work identifies lirafugratinib, a highly selective fibroblast growth factor receptor 2 inhibitor, as a previously unrecognized suppressor of ABCG2-dependent MDR. By limiting efflux-mediated depletion of anticancer drugs while maintaining its own activity, lirafugratinib resensitizes resistant nonsmall cell lung cancer cells to cytotoxic agents, supporting its potential utility in combination regimens for tumors with elevated ABCG2 expression.

News (Medical) associated with Lirafugratinib

23 Jun 2026

·www.biospace.com

Elevar Therapeutics Announces First Patients Dosed in Phase 2 Study of Lirafugratinib Among Non-CCA Solid Tumors With FGFR2 Fusion or Rearrangement

First patient was dosed at Samsung Medical Center in Seoul, South Korea; a second at Moffitt Cancer Center in Tampa, Florida Global multi-site study includes locations in U.S., Korea, the UK, Spain, and France Study aims to confirm lirafugratinib’s potential efficacy among patients with FGFR2 fusion/rearrangement independent of tumor type FORT LEE, N.J., June 22, 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 patients who have limited or inadequate therapeutic options, today announced that the first patients were dosed in its global Phase 2 study of lirafugratinib in non-cholangiocarcinoma (CCA) solid tumors with FGFR2 fusion or rearrangement. “Elevar believes lirafugratinib has much potential as a strong anti-tumor therapy across a wider range of FGFR2 fusion or rearrangement driven tumors. We are committed to do our best to obtain meaningful study results to present to the FDA,” said Dong-Gun Kim, chief executive officer of Elevar. “We are pleased that the first patients have been dosed and we look forward to completing enrollment.” The trial, known as ReFocus202 (Protocol ID ELE-4008-202; NCT07359820 ), is an open-label, single-arm study evaluating the efficacy and safety among a broad scope of tumors containing an FGFR2 fusion or rearrangement. The primary endpoint is objective response rate. The first patient was dosed at Samsung Medical Center in Seoul, South Korea, and a second patient at Moffitt Cancer Center in Tampa, Florida. The multi-site study is set to take place in the U.S., Korea, the UK, Spain, and France. “Patients with advanced solid tumors harboring FGFR2 fusions or rearrangements often have limited treatment options, particularly beyond cholangiocarcinoma,” said Dr. Richard Kim, ReFocus202 principal investigator and service chief of medical gastrointestinal oncology at Moffitt Cancer Center. “This study gives us an important opportunity to better understand whether selective FGFR2 inhibition with lirafugratinib can benefit a broader group of patients whose tumors are driven by FGFR2 alterations. I am pleased that our team was able to enroll the first patient in the U.S. and contribute to this important effort.” Lirafugratinib received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA) for CCA. Priority review of its new drug application for the treatment of patients with CCA with FGFR2 fusion/rearrangement who have received prior therapy is ongoing by the FDA, which set a Prescription Drug User Fee Act date of Sep 27, 2026. ReFocus202 aims to confirm the tumor-agnostic potential of lirafugratinib in patients with FGFR2 fusion/rearrangement. In the Phase 1/2 ReFocus study, data from 42 non-CCA solid tumor patients with FGFR2 fusion/rearrangement (13 tumor types) showed meaningful antitumor activity of lirafugratinib. Elevar plans to leverage that dataset, along with data generated under ReFocus202, to conduct an interim analysis across at least seven tumor types with at least five patients per tumor type. For more information about lirafugratinib, visit ElevarTX.com . About Lirafugratinib Lirafugratinib (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. Preclinically, lirafugratinib demonstrated FGFR2-dependent killing in cancer cell lines and induced regression in in vivo models with minimal inhibition of other targets, including other members of the FGFR family. In addition, lirafugratinib demonstrated strong activity against known clinical on-target resistance mutations in vitro and in vivo preclinical models. Lirafugratinib 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. About Elevar Therapeutics Elevar Therapeutics, Inc. is a fully integrated biopharmaceutical company built on the promise of elevating treatment outcomes for patients who have limited or inadequate therapeutic options. A subsidiary of HLB Group 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. Media Contact: Rosemary Ostmann RoseComm Cell: (201) 615-7751

Orphan DrugFast TrackPhase 2Clinical Result

04 Jun 2026

·www.prnewswire.com

Early Data Show Highly Selective FGFR2 Inhibitor 3HP-2827 Active in Advanced CCA; Chinese Academy of Sciences' Zhou Jian Comments on Potential for Patients With FGFR2 Alterations

CHICAGO, June 2, 2026 /PRNewswire/ -- At the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting, 3H Pharmaceuticals released new clinical data for its wholly owned candidate, 3HP-2827. The ongoing study focuses on patients with advanced cholangiocarcinoma (CCA) and other solid tumors carrying FGFR2 alterations—an area of persistent and significant unmet need. The data showed that 3HP-2827, a highly selective FGFR2 inhibitor, demonstrated encouraging anti-tumor activity in patients with advanced CCA driven by FGFR2 fusions or rearrangements, as well as notable tumor shrinkage in FGFR2-mutated solid tumors. On May 31 in Chicago, on the sidelines of the ASCO meeting, eChinaHealth spoke with Academician Zhou Jian, President of Zhongshan Hospital, Fudan University. He shared his views on the current treatment landscape for FGFR2-altered CCA, the clinical profile and future potential of 3HP-2827, and the prospects for further advances in the field. Continue Reading 3HP-2827 Brings New Hope to Patients with Advanced CCA Harboring FGFR2 Alterations Speed Speed Academician Zhou noted that CCA is a highly aggressive cancer, with incidence rising in both China and worldwide. For patients with advanced disease, the prognosis is extremely poor—even worse than for hepatocellular carcinoma. Currently, the standard first-line treatment for advanced CCA remains chemotherapy plus immunotherapy. In recent years, however, a growing number of new approaches have emerged. Drugs targeting IDH1 and FGFR, for instance, have already received approval. At this year's ASCO Annual Meeting alone, more than 80 abstracts on CCA were presented, with FGFR2-targeted therapy emerging as a particularly active area of research. Earlier studies have validated the efficacy of FGFR2 inhibition in CCA, and as these targeted agents are refined, the efficacy data continue to improve. Looking ahead, FGFR2-directed therapy is expected to play a larger role in the treatment of advanced CCA, offering patients more precise and effective options. 3HP-2827, a novel and highly selective FGFR2 inhibitor, is being evaluated in a multicenter clinical study in China led by Academician Fan Jia and Professor Shen Lin, with RLY-4008 as the active comparator. To date, the efficacy of 3HP-2827 appears comparable to the early-stage clinical results reported for RLY-4008, and both agents have shown superior efficacy compared with pan-FGFR inhibitors. In addition, 3HP-2827 has demonstrated a favorable safety profile. Of note, the patient population in the current study was more heavily pretreated: 80% of patients had previously received an immune checkpoint inhibitor (ICI) in combination with chemotherapy, and 60% had undergone prior anti-angiogenic therapy. Additional clinical data will be important to further evaluate the potential of this approach. Based on available data, both RLY-4008 and Pemigatinib have shown greater efficacy in the first-line treatment of advanced CCA than in the second-line setting. The latest data for single-agent RLY-4008 show an objective response rate (ORR) of 46.5% among 144 second-line patients, while the ORR reached 63.6% in a small cohort of 11 first-line patients—though the limited sample size in the first-line group requires cautious interpretation and further validation. For Pemigatinib, prior phase II and the most recent phase III data report an ORR of 35.5% in 146 second-line patients and 47.0% in 83 first-line patients. To date, 3HP-2827 has generated preliminary efficacy evidence in the second-line setting. Evaluating its single-agent activity in the first-line setting to pursue a first-line indication represents an important next step. Academician Zhou said he expects the future first-line treatment paradigm for CCA to shift toward a multi-drug, multi-modality combination approach integrating chemotherapy, immunotherapy and targeted therapy. Such a strategy, he noted, is scientifically sound and could improve both treatment efficacy and patient outcomes. From a safety perspective, the main adverse reactions linked to 3HP-2827 are FGFR2-related toxicities affecting the skin and mucosa — a profile distinct from that of chemotherapy or immunotherapy. That difference suggests a low risk of overlapping toxicity when used in combination, supporting the potential for 3HP-2827 to be added to existing regimens. In addition, among patients with FGFR2 fusion- or rearrangement-driven CCA treated with 3HP-2827, two achieved complete disappearance of target lesions, and the majority experienced substantial tumor shrinkage. Because most CCA patients are no longer candidates for surgery at initial diagnosis, the pronounced tumor-shrinking effect of 3HP-2827 opens the door to exploring conversion therapy in the future — using preoperative single-agent treatment to downstage tumors and expand the pool of patients eligible for curative resection. Currently, most FGFR2-targeted agents in clinical development are aimed at CCA patients with FGFR2 fusions or rearrangements. In contrast, patients with FGFR2-mutated solid tumors have few options, with no approved therapies to date. FGFR2 mutations occur across a wide range of solid tumors — including biliary tract cancers, gynecologic malignancies, melanoma, colorectal cancer, lung cancer, gastric cancer and urothelial carcinoma — representing a patient population larger than that of FGFR2 fusions or rearrangements. The longstanding lack of a targeted treatment for this group highlights a significant unmet medical need. The activity observed with 3HP-2827 suggests a potential new treatment approach for these patients. The clinical data released by 3H Pharmaceuticals at the meeting showed that among patients with FGFR2-mutated solid tumors, 3HP-2827 achieved a best objective response rate (ORR) of 57.1% and a disease control rate (DCR) of 100%. Although the number of enrolled patients remains small at this stage, the cohort already includes multiple clinically relevant FGFR2 mutation sites, including N549K, Y375C, C382R and F276C. Looking ahead, the clinical development program for 3HP-2827 should continue to gather additional data in FGFR2-mutated solid tumors and initiate a multi-histology basket trial, with the goal of further evaluating its potential in this patient population. In the interview, Academician Zhou Jian of Zhongshan Hospital, Fudan University, discussed the latest clinical data on 3HP-2827, a highly selective FGFR2 inhibitor, in patients with FGFR2-altered advanced CCA and other solid tumors. He noted that while chemotherapy plus immunotherapy remains the current standard first-line treatment for advanced CCA, FGFR2-targeted therapy is emerging as an important precision medicine approach. Looking ahead, Zhou recommended pursuing first-line treatment studies, combination therapy regimens and basket trials targeting FGFR2 mutations — strategies that could further expand the reach of precision oncology. Note: 3HP-2827 is a highly selective FGFR2 inhibitor independently developed by 3H Pharmaceuticals. It works by specifically inhibiting FGFR2 phosphorylation, blocking the FGFR2 signaling pathway and producing anti-tumor activity. The drug is designed to offer a more effective and better-tolerated treatment option for patients with FGFR2 alterations, especially those who have developed resistance to prior therapies. About Academician Zhou Jian Academician Zhou Jian is a member of the Chinese Academy of Sciences and a leading authority in hepatobiliary oncology and liver transplantation. He currently serves as President of Zhongshan Hospital, Fudan University, and Executive Deputy Director of the Liver Cancer Institute at Fudan University. He holds numerous prestigious academic appointments, including Executive Chair of the National Health Commission's Expert Committee for the Guidelines on the Diagnosis and Treatment of Primary Liver Cancer, President of the Chinese Society of Oncology for the Chinese Medical Association, Chairman of the Asia-Pacific Primary Liver Cancer Expert (APPLE) Association, and Chairman of the Chinese Society of Clinical Oncology (CSCO) Expert Committee on Hepatocellular Carcinoma. He also serves on the Executive Council of the Asian Pacific Association for the Study of the Liver (APASL) and is Vice President and Secretary-General of the Chinese College of Surgeons, Chinese Medical Doctor Association. Recognized as one of China's leading physician-scientists, he has been honored with a National Science Fund for Distinguished Young Scholars award, a Chang Jiang Distinguished Professorship from the Ministry of Education, and a National "Ten Thousand Talents Program" designation as a Leading Talent in Scientific and Technological Innovation. He is a five-time recipient of the National Science and Technology Progress Award, having served as the principal investigator for one award and as a co-principal investigator for two others. Academician Zhou has authored or co-authored over 154 peer-reviewed articles as first or corresponding author in journals such as The Lancet, The New England Journal of Medicine, Cell, Journal of Clinical Oncology, The Lancet Gastroenterology & Hepatology, The Lancet Oncology, Signal Transduction and Targeted Therapy, and Cell Research, earning an H-index of 100. Contact: Li Hao [email protected] SOURCE 3H Pharmaceuticals 21% more press release views with Request a Demo

Clinical ResultASCOImmunotherapyPhase 2Phase 3

30 Mar 2026

·www.biospace.com

Elevar Therapeutics Announces FDA Acceptance for Review of New Drug Application for Lirafugratinib as Second-line Cholangiocarcinoma Treatment

With Priority Review designation, FDA set a Sept. 27 PDUFA date for approval decision Lirafugratinib achieved a 46.5% ORR, in CCA patients with FGFR2 fusion and rearrangement Fort Lee, N.J., March 30, 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 patients who have limited or inadequate therapeutic options, today announced that the U.S. Food and Drug Administration (FDA) has completed its filing review of the New Drug Application (NDA) for lirafugratinib, an investigational therapy, for the treatment of patients with cholangiocarcinoma (CCA) with FGFR2 fusions or rearrangements who have received prior therapy. The FDA determined that the NDA is sufficiently complete to permit a substantive review and granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of September 27, 2026. Priority Review designations are given to applications for medicines that, if approved, would lead to “significant improvements in the safety or effectiveness of the treatment” of a serious condition, according to the FDA. The Priority Review designation of the lirafugratinib NDA was supported by positive clinical data from the Phase 1/2 ReFocus trial ( NCT04526106 ), which demonstrated a confirmed objective response rate (ORR) of 46.5% in the patients with the proposed indication. Its safety pro the clinical data has been shown to be predictable and manageable through dose adjustments. “Lirafugratinib has established a compelling clinical pro differentiates it from existing treatment options,” said Dong-Gun Kim, chief executive officer of Elevar. “We are very pleased with the FDA’s priority review designation and focused on advancing the review process efficiently to bring this therapy to patients as quickly as possible.” CCA, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. diagnosed each year, according to the American Cancer Society. Elevar Therapeutics continues to evaluate lirafugratinib in ongoing clinical development programs, including studies in other FGFR2-altered solid tumors. Any future indications will be subject to regulatory review and approval. For more information about lirafugratinib, visit ElevarTX.com . About Lirafugratinib Lirafugratinib (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. Preclinically, lirafugratinib demonstrated FGFR2-dependent killing in cancer cell lines and induced regression in in vivo models with minimal inhibition of other targets, including other members of the FGFR family. In addition, lirafugratinib demonstrated strong activity against known clinical on-target resistance mutations in vitro and in vivo preclinical models. Lirafugratinib 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. About Elevar Therapeutics Elevar Therapeutics, Inc. is a fully integrated biopharmaceutical company built on the promise of elevating treatment outcomes for patients who have limited or inadequate therapeutic options. A subsidiary of HLB Group 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. CONTACT: Media Contact: Rosemary Ostmann RoseComm Cell: (201) 615-7751

Priority ReviewNDA

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Indication	Highest Phase	Country/Location	Organization	Date
FGFR2 fusion or rearranged Cholangiocarcinoma	NDA/BLA	United States	Elevar Therapeutics, Inc.	30 Jan 2026
Advanced Malignant Solid Neoplasm	Phase 2	United States	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	Australia	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	France	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	Germany	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	Hong Kong	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	Italy	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	Netherlands	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	Singapore	Elevar Therapeutics, Inc.	02 Sep 2020
Advanced Malignant Solid Neoplasm	Phase 2	South Korea	Elevar Therapeutics, Inc.	02 Sep 2020

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04526106 (ReFocus, Pubmed \| Ann Oncol)	Phase 1	FGFR2 Mutation Solid Tumors FGFR2 alteration	28	Lirafugratinib (FGFR inhibitor-naive)	ylpzmarrse(ygtxqxfknd) = aicjdqkpfy egawkvssfu (sraaguoblg ) View more	Positive	01 Jun 2026
NCT07359820 (ELE-4008-202, ASCO2026)	Phase 2	FGFR2 fusion or rearranged Cholangiocarcinoma FGFR2 fusion or rearrangement	20	Lirafugratinib 70 mg	xbytfsfroq(jhxrqhbxmt) = mesyegnxcj afhzuylpjk (zpktzqhwow )	Positive	29 May 2026
NCT04526106 (ReFocus, ASCO_GI2026) Manual	Phase 1/2	FGFR2 fusion or rearranged Cholangiocarcinoma FGFR Rearrangement \| FGFR2 Fusion	116	lirafugratinib 70 mg	eajbccyptf(mzilhxqrhc) = dmqyiwwxzu kocfpghjkw (mjdqidpkxk, 37.1 - 56.1) View more	Positive	08 Jan 2026
NCT04526106 (REFOCUS, Corporate Publications) Manual	Phase 1/2	Solid tumor FGFR2 fusions \| FGFR2 amplifications \| FGFR2 mutations	84	Lirafugratinib (patients with FGFR2 fusions)	ytxzkgoqnu(oqbjvogrkv) = zxfimokgoe yibamytgdu (ewxxzyffeq )	Positive	12 Oct 2023
				Lirafugratinib (patients with breast cancer across all FGFR2 alterations, 10 of whom had HR+/HER2- breast cancer)	ytxzkgoqnu(oqbjvogrkv) = xztfhnpyok yibamytgdu (ewxxzyffeq )
NCT04526106 (ReFocus, ASCO 12023 \| ASCO 22023) Manual	Phase 1/2	Solid tumor \| Bile Duct Neoplasms	75	RLY-4008 (FGFRi-naïve CCA f/r)	aceypqtvxn(rbwqszhvri) = 70 mg QD bhngqampto (fepmkrcjqx ) View more	Positive	26 May 2023
				RLY-4008 (Prior-FGFRi CCA f/r)
AACR2023	Phase 1/2	-	-	RLY-4008 (fasted)	jlmvjlgqij(cqhrmyaitc) = ezxueunzhw vgbzohuptj (qxyfkbzknf )	-	04 Apr 2023
				RLY-4008 with food	jlmvjlgqij(cqhrmyaitc) = klhdhkprsv vgbzohuptj (qxyfkbzknf )
NCT04526106 (ReFocus, ESMO_ASIA2022)	Phase 1/2	FGFR2 fusion or rearranged Cholangiocarcinoma	38	RLY-4008	qtivfesyhz(oaakhghtca) = majority of responses ongoing qembynxhsi (qrduejitmf )	-	04 Dec 2022
NCT04526106 (ReFocus, ESMO2022) Manual	Phase 1/2	FGFR2 fusion or rearranged Cholangiocarcinoma First line FGFR2 Rearrangement \| FGFR2 Fusion	195	RLY-4008 20-200 mg QD or BID (RP2D (70 mg QD))	kriihhxnwc(zxmabcxyts) = iiqmquwssz ohceiuiwdp (dvnxkfpauu, 56.6 - 96.2) View more	Positive	11 Sep 2022
				RLY-4008 20-200 mg QD or BID (All dose levels)	kriihhxnwc(zxmabcxyts) = lqjtzjictw ohceiuiwdp (dvnxkfpauu, 40.8 - 73.7) View more
NCT04526106 (ReFocus, AACR2021) Manual	Phase 1/2	Solid tumor \| FGFR2 fusion or rearranged Cholangiocarcinoma FGFR2 Mutation	45	RLY-4008	jkkjutelql(gwdovtvmwr) = AEs occurring in >20% of pts include stomatitis (49%), palmar-plantar erythrodysesthesia (PPE, 38%), dry mouth (29%), and nail toxicities (22%), majority of which were ≤Gr 2. nibwdzxyjh (swolsxrntz ) View more	Positive	01 Dec 2021
				RLY-4008 (FGFRi-naïve, FGFR2-fusion+ CCA)

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Identify the latest clinical trials across global registries.

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Approval

Accelerate your research with the latest regulatory approval information.

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Regulation

Understand key drug designations in just a few clicks with Synapse.

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Synapse data is now integrated into the PatSnap LS Model Context Protocol (MCP) service. Customize your LLM agent now using our MCP server!

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis