irpagratinib (ABSK011) / Abbisko - LARVOL DELTA

Irpagratinib (ABSK-011) plus atezolizumab in first-line (1L) and immune checkpoint inhibitors (ICIs) treated advanced hepatocellular carcinoma (HCC) with FGF19 overexpression (+): Updated results of the phase II ABSK-011-201 study (ESMO-GI 2025) - P2 | "Irpagratinib combined with Atezolizumab was safe and demonstrated promising anti-tumor activity in both 1L and ICI-treated FGF19+ HCC. The remarkable response rate along with an evident PFS benefit underscores the potential of FGFR4i-based combinations for further development in both ICI-treated and treatment-naïve settings."

Checkpoint inhibition • Clinical • IO biomarker • Metastases • P2 data • Hepatocellular Cancer • Oncology • Solid Tumor • FGF19 • FGFR4

IAPETUS: A Prospective Study to Evaluate the Safety and Efficacy of the Combination Therapy of Irpagratinib, Atezolizumab, and Bevacizumab in Patients With Hepatocellular Carcinoma (clinicaltrials.gov) - P2 | N=33 | Recruiting | Sponsor: Asan Medical Center | Not yet recruiting ➔ Recruiting | Initiation date: Nov 2025 ➔ Mar 2026

Enrollment open • Trial initiation date • Hepatocellular Cancer • Oncology • Solid Tumor • FGF19

Abbisko Therapeutics…announced that the first patient has been dosed in a Phase II study evaluating…irpagratinib (ABSK-011), in combination with standard of care (toripalimab plus bevacizumab biosimilar) as first-line treatment for patients with advanced or unresectable hepatocellular carcinoma ('HCC') (HKEXnews)

Trial status • Hepatocellular Cancer

ABSK-011-201: A Phase 2, Open-Label Study of ABSK-011 Combined Atezolizumab or SOC in HCC Patients (clinicaltrials.gov) - P2 | N=118 | Enrolling by invitation | Sponsor: Abbisko Therapeutics Co, Ltd | Recruiting ➔ Enrolling by invitation | N=62 ➔ 118 | Trial completion date: Oct 2024 ➔ Dec 2029 | Trial primary completion date: Jul 2024 ➔ Dec 2028

Enrollment change • Enrollment status • Trial completion date • Trial primary completion date • Hepatocellular Cancer • Oncology • Solid Tumor • FGF19

Abbisko Therapeutics…announced that the first patient in the United States has been successfully dosed in the expansion part of the global, multicenter Phase I clinical study (ABSK-011-101) of its independently developed, highly selective small-molecule FGFR4 inhibitor irpagratinib (ABSK-011) for patients with FGF19 overexpression advanced hepatocellular carcinoma (HCC) (Abbisko Therap Press Release) - "ABSK-011-101 is an open-label, global, multicenter Phase I study (NCT04906434) designed to assess the safety, tolerability, and pharmacokinetics of irpagratinib in patients with advanced solid tumors. The study consists of dose-escalation and dose-expansion parts. In the U.S., patients will be treated at the recommended dose determined from the completed dose-escalation part to further assess safety, tolerability, and preliminary efficacy in FGF19 overexpression advanced HCC."

Trial status • Hepatocellular Cancer

Abbisko Therapeutics' FGFR4 Inhibitor Irpagratinib Granted FDA Fast Track Designation for Advanced HCC Patients with FGF19 Overexpression Previously Treated with ICIs and mTKIs Therapies (Abbisko Therap Press Release) - "The FDA's Fast Track Designation was primarily based on positive results from a Phase I clinical study presented at the 2024 European Society for Medical Oncology (ESMO) Annual Congress."

Fast track • Hepatocellular Cancer

Updated safety and efficacy of ABSK-011 in advanced hepatocellular carcinoma (aHCC) with FGF19 overexpression from a phase I study (ESMO 2024) - P1 | "ABSK-011 demonstrated a manageable safety profile and promising anti-tumor activity as a single agent. These findings support further development of ABSK-011 at 220 mg BID in FGF19+ aHCC with prior ICI treatments."

Clinical • Metastases • P1 data • Gastrointestinal Cancer • Hepatocellular Cancer • Oncology • Solid Tumor • FGF19 • FGFR4

ABSK-011+BSC vs. Placebo+BSC in Previously Treated Advanced HCC With FGF19 Overexpression (clinicaltrials.gov) - P2 | N=141 | Recruiting | Sponsor: Abbisko Therapeutics Co, Ltd

New P2 trial • Hepatocellular Cancer • Oncology • Solid Tumor • FGF19

A Clinical Trial to Evaluate the Relative Bioavailability of New and Old ABSK-011 Capsules (clinicaltrials.gov) - P1 | N=40 | Completed | Sponsor: Abbisko Therapeutics Co, Ltd | Recruiting ➔ Completed | N=73 ➔ 40 | Trial completion date: Dec 2024 ➔ Sep 2025 | Trial primary completion date: Dec 2024 ➔ Sep 2025

Enrollment change • Trial completion • Trial completion date • Trial primary completion date

Fibroblast growth factor 19-fibroblast growth factor receptor 4 axis: From oncogenesis to targeted-immunotherapy in advanced hepatocellular carcinoma. (PubMed, World J Gastrointest Oncol) - "Phase I clinical trials of Irpagratinib (ABSK-011) demonstrated an objective response rate of 43.5%, which increased to 55.6% combined with atezolizumab. It also firstly synthesizes combination strategies and underlying mechanisms between FGFR4 inhibitors and targeted-immunotherapy, addressing critical gaps in existing reviews. Additionally, we discuss the potential of FGF19 as a predictive biomarker, integrating mechanistic and clinical evidence to advance precision HCC therapeutics."

IO biomarker • Journal • Review • Hepatocellular Cancer • Oncology • Solid Tumor • FGF19 • FGFR4 • PD-L1

ABSK-011-101: A Study to Assess the Safety, Tolerability, and Pharmacokinetics of ABSK-011 in Patients With Advanced Solid Tumor (clinicaltrials.gov) - P1 | N=200 | Recruiting | Sponsor: Abbisko Therapeutics Co, Ltd | N=50 ➔ 200 | Trial completion date: Aug 2025 ➔ Dec 2028 | Trial primary completion date: Dec 2023 ➔ Dec 2027

Enrollment change • Trial completion date • Trial primary completion date • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor • FGF19

Irpagratinib/Atezolizumab Active in FGF19+ Advanced HCC (Oncology Nursing News) - P2 | N=62 | NCT05441475 | Sponsor: Abbisko Therapeutics Co, Ltd | "The combination of irpagratinib (ABSK011) and atezolizumab (Tecentriq) led to deep, durable responses in patients with advanced hepatocellular carcinoma (HCC) and FGF19 overexpression...according to phase 2 data presented at the 2025 ESMO GI Congress. At a median follow-up of 7.1 months, among response-evaluable patients in the treatment-naive population (n = 12/15), the overall response rate (ORR) was 50.0%; best responses included partial response (PR; n = 6), stable disease (SD; n = 5), and progressive disease (PD; n = 1). The confirmed ORR was 33.3%. The median duration of response (DOR) was not reached (NR), the disease control rate (DCR) was 91.7%, and the median progression-free survival (PFS) was 7.0 months (95% CI, 2.8-not evaluable [NE]). Among response-evaluable patients in the pretreated population (n = 17/18), the ORR was 52.9%; best responses included PR (n = 9), SD (n = 2), and PD (n = 5)."

P2 data • Hepatocellular Cancer

ABBISKO THERAPEUTICS TO PRESENT UPDATED RESULTS FROM THE PHASE 2 STUDY OF IRPAGRATINIB IN COMBINATION WITH ATEZOLIZUMAB FOR THE TREATMENT OF ADVANCED HCC AT THE 2025 ESMO GI CONGRESS (HKEXnews) - P2 | N=62 | NCT05441475 | Sponsor: Abbisko Therapeutics Co, Ltd | "...Abbisko Therapeutics...announced that updated results from the phase 2 study of irpagratinib (ABSK-011) in combination with atezolizumab for the treatment of patients with advanced hepatocellular carcinoma ('HCC') will be presented at the 2025 European Society for Medical Oncology Gastrointestinal Cancers Congress ('2025 ESMO GI Congress'....The oral presentation will highlight the safety and efficacy of irpagratinib in combination with atezolizumab in both treatment-naive and previously treated FGF19+ HCC patients. The treatment combination with irpagratinib showcases an objective response rate of ≥50% and a median progression-free survival of ≥7 months. These results further demonstrate the clinical benefit of irpagratinib in both previously treated and treatment-naive settings, offering renewed hope for patients with advanced HCC."

P2 data • Hepatocellular Cancer

Abbisko Therapeutics Completes First Patient Dosing in Registrational Study of Irpagratinib for HCC (PRNewswire) - "Abbisko Therapeutics...announced that it has completed first patient dosing in a registrational study of irpagratinib...for the treatment of Hepatocellular Carcinoma (HCC)....The registrational study of irpagratinib (ABSK-011-205) is a multi-center, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the efficacy and safety of irpagratinib in combination with Best Supportive Care (BSC) versus placebo in combination with BSC, in patients with advanced or unresectable HCC who exhibit FGF19 overexpression and have previously been treated with ICIs and mTKIs. Eligible patients will be randomized in a 2:1 ratio to receive irpagratinib or placebo."

Trial status • Hepatocellular Cancer

IAPETUS: A Prospective Study to Evaluate the Safety and Efficacy of the Combination Therapy of Irpagratinib, Atezolizumab, and Bevacizumab in Patients With Hepatocellular Carcinoma (clinicaltrials.gov) - P2 | N=33 | Not yet recruiting | Sponsor: Asan Medical Center

New P2 trial • Hepatocellular Cancer • Oncology • Solid Tumor

Abbisko Therapeutics Receives CDE Approval of Breakthrough Therapy Designation for Irpagratinib (ABSK011) in the Treatment of HCC (PRNewswire) - "Abbisko Therapeutics...announced that its self-developed, highly selective small molecule FGFR4 inhibitor, irpagratinib (ABSK011), has received approval of Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of Hepatocellular Carcinoma (HCC). Irpagratinib is the first therapeutic agent to leverage molecularly defined biomarkers for precision-targeted treatment in patients with HCC....The approval of irpagratinib for Breakthrough Therapy Designation is based on its promising Phase I clinical trial data."

Breakthrough therapy • Hepatocellular Cancer

FOCUS-19: FGF19 Overexpression Combination Unified Study in HCC-19 (clinicaltrials.gov) - P2 | N=42 | Recruiting | Sponsor: RenJi Hospital

IO biomarker • New P2 trial • Hepatocellular Cancer • Oncology • Solid Tumor

Abbisko Therapeutics to Present Updated Results from the Phase 2 Study of Irpagratinib in Combination with Atezolizumab for the Treatment of Advanced Hepatocellular Carcinoma at the 2025 ESMO GI Congress (PRNewswire) - "Abbisko Therapeutics Co., Ltd...today announced that updated results from the phase 2 study of irpagratinib (ABSK011) in combination with atezolizumab for the treatment of advanced hepatocellular carcinoma (HCC) will be presented at the 2025 ESMO GI Congress, taking place in Barcelona, Spain from July 2 to July 5. The oral presentation will highlight the regimen's favorable safety and promising anti-tumor activity in both treatment-naive and previously treated FGF19+ HCC patients. The evident objective response rate (ORR) and progression free survival (PFS) benefit underscores the potential of irpagratinib-based combinations in the treatment of HCC."

P2 data • Hepatocellular Cancer

A Mass Balance Study of [14C]ABSK011 (clinicaltrials.gov) - P1 | N=6 | Not yet recruiting | Sponsor: Abbisko Therapeutics Co, Ltd

New P1 trial

Hong Kong Stocks Move | Heyu-B (02256) rose nearly 4% and was approved by the CDE to conduct a registration clinical study of irpagratinib in the treatment of hepatocellular carcinoma [Google translation] (Sina Corp) - "...Heyu Pharmaceuticals recently announced that its independently developed highly selective small molecule FGFR4 inhibitor irpagratinib (ABSK011) has been approved by the Center for Drug Evaluation (CDE) of the China National Drug Administration to conduct a registration clinical study for the treatment of hepatocellular carcinoma (HCC). This study will serve as a key basis to support the launch of irpagratinib, and also marks an important step for irpagratinib in the treatment of advanced or unresectable hepatocellular carcinoma (HCC)."

New trial • Hepatocellular Cancer

Heyu-B (02256.HK) subsidiary released the latest clinical progress of irpagratinib and pimicotinib at the ESMO Annual Meeting [Google translation] (Sina Corp) - P1 | N=50 | NCT04906434 | Sponsor: Abbisko Therapeutics Co, Ltd | "Heyu-B...announced that it will announce the latest clinical data on the safety and efficacy of its independently developed small molecule FGFR4 inhibitor irpagratinib (ABSK011) in a Phase I clinical trial for advanced hepatocellular carcinoma...at the 2024 European Society of Oncology Annual Meeting...The data released this time showed that the 220mg irpagratinib twice daily ('BID') group showed excellent efficacy in FGF19 overexpressing aHCC patients treated with immune checkpoint inhibitors ('ICIs') and multi-target small molecule tyrosine kinase inhibitors ('mTKIs'), with an overall response rate ('ORR') of 44.8%....In addition, the design of a Phase II clinical study of the small molecule CSF-1R inhibitor pimicotinib (ABSK021) combined with chemotherapy with or without toripalimab as a first-line treatment for advanced pancreatic ductal adenocarcinoma (PDAC) will also be made public at this conference."

Clinical protocol • P1 data • Gastrointestinal Cancer • Hepatocellular Cancer • Liver Cancer • Oncology • Pancreatic Cancer • Pancreatic Ductal Adenocarcinoma • Solid Tumor

Hong Kong Stock Market Movement | Heyu-B (02256) rose more than 5% and is expected to make more than 200 million yuan in profit in the first half of the year, turning losses into profits year-on-year [Google translation] (Sina Corp) - "On the news front, Heyu will release its 2024 interim results on August 12. The company previously issued a profit warning, expecting total revenue in the first half of the year to be approximately RMB 497 million, a significant year-on-year increase; profit of RMB 200 million to RMB 220 million, a year-on-year turnaround. In addition to improving cost control by improving business operating efficiency, it also benefited from the out-licensing agreement for pimicotinib (ABSK021) and the first payment of US$70 million (RMB 497 million) with Merck....In addition, the company will announce the latest clinical data on the safety and efficacy of its independently developed highly selective small molecule FGFR4 inhibitor Irpagratinib in a Phase I clinical trial for advanced hepatocellular carcinoma with overexpression of FGF19 at the European Society of Oncology (ESMO) Annual Meeting in 2024."

Commercial • P1 data • Gastrointestinal Cancer • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor

50% ORR: Impressive Clinical Trial Data for Irpagratinib Combined with Atezolizumab in Advanced Hepatocellular Carcinoma Stuns ESMO-GI Congress (PRNewswire) - P2 | N=62 | NCT05441475 | Sponsor: Abbisko Therapeutics Co, Ltd | "Abbisko Therapeutics Co., Ltd...announced that it has presented new phase II clinical data of its highly selective FGFR4 inhibitor irpagratinib (ABSK011) in combination with atezolizumab for the treatment of advanced hepatocellular carcinoma (HCC) at the 2024 ESMO-GI Congress. The presentation highlights that 220mg BID of irpagratinib in combination with atezolizumab demonstrated promising antitumor activity with an objective response rate (ORR) of 50% in FGF19+ HCC patients....Given the encouraging preliminary results from this study, Abbisko plans to explore dual/triple combinations with irpagratinib in earlier lines of therapy for HCC."

P2 data • Trial status • Gastrointestinal Cancer • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor

A phase II study of ABSK-011 plus atezolizumab in patients with advanced hepatocellular carcinoma (HCC) (ESMO-GI 2024) - P2 | "ABSK-011 in combination with atezolizumab showed encouraging anti-tumor activity and manageable safety profile which supports further development of the regimen in FGF19+ HCC and exploration of ABSK-011 based double/triple combinations in earlier lines."

Clinical • IO biomarker • Metastases • P2 data • Gastrointestinal Cancer • Hepatocellular Cancer • Oncology • Solid Tumor • FGF19

Improvements in advanced hepatocellular carcinoma to repeat implementation of primary protocol after cancer progression occurs following sequential systemic therapy and a clinical trial: A case report. (PubMed, Medicine (Baltimore)) - "The combined treatment of TACE plus systemic therapy might be an appropriate subsequent treatment."

Journal • Metastases • Fatigue • Gastrointestinal Cancer • Hepatitis B • Hepatocellular Cancer • Hepatology • Infectious Disease • Oncology • Solid Tumor • AFP • FGFR4