HLB (028300) has expanded the potential reach of its liver cancer treatment, Riboceranib, to include rare cancers. The long-term follow-up results of the phase 2 clinical trial for metastatic thymic epithelial tumors, presented at the European Society for Medical Oncology (ESMO 2026), revealed a median progression-free survival (PFS) of 11.8 months. This metric was not observable last year due to shorter observation periods.
The company's FDA-approved biliary tract cancer drug, Rifect, has also entered the commercialization phase. HLB is preparing for direct sales in the United States while simultaneously pursuing European approval and expanding the treatment scope to other cancer types.
#### Beyond Liver Cancer: Riboceranib Shows Promising Long-Term Results for Rare Cancers
The standout study at this year's ESMO is the phase 2 clinical trial 'THRIVE' for Riboceranib's treatment of metastatic thymic epithelial tumors. Professor Park, Se-Hoon of Samsung Medical Center presented the additional follow-up results on the 26th. This study, involving 11 domestic institutions, targeted 40 patients who required further treatment after receiving immunochemotherapy. Notably, 40% of these patients had received systemic treatment three or more times.
HLB's Ribociclib Shows Promise in Treating Rare Cancers
Ribociclib, a VEGFR-2 targeted anti-cancer drug, inhibits the formation of blood vessels that supply nutrients to cancer cells. HLB has been developing this drug in combination with the immunotherapy camrelizumab as a first-line treatment for liver cancer. The THRIVE study aimed to evaluate the potential of ribociclib monotherapy in treating rare cancers.
Thymic epithelial tumors are a rare form of cancer that originates in the thymus, located in the chest's central region. These tumors are classified as either thymic cancer or thymoma, and treatment options are limited when the cancer progresses or becomes resistant to standard therapies.
When initially presented at ESMO 2025 last year, the median follow-up period was 3.7 months. The median progression-free survival (PFS), which indicates the duration of time post-treatment where the cancer does not worsen, was not yet determined.
This year, with an extended follow-up period of 13.6 months, the median PFS was calculated at 11.8 months. This means that, on average, patients experienced a disease progression or death after 11.8 months.
Financial Insights: Ribociclib's Performance in ESMO 2026
The objective response rate (ORR), which measures the percentage of patients with a tumor reduction above a certain threshold, stood at 35%, while the disease control rate (DCR), including patients with tumor reduction or stabilization, reached 85%. The median duration of response (DOR) for patients exhibiting tumor reduction was 11.2 months.
While these results represent a slight decrease from the previously announced figures of 38.9% ORR and 94.4% DCR, the actual number of responding patients remained consistent at 14, and the number of disease-controlled patients was 34. This discrepancy can be attributed to the expanded analysis pool, which increased from 36 to 40 patients.
[Source: ESMO 2026 Presentation Materials, Ribociclib THRIVE Phase 2 Clinical Trial Tumor Response Results]
For patients with thymic cancer, the median progression-free survival (PFS) was 10 months. The thymoma patient group had not yet reached the median, and their disease control rate stood at 93.8%. The median overall survival (OS) has yet to be determined.
In terms of safety evaluation, treatment-related adverse events of grade 3 or higher occurred in 55% of patients. The primary severe adverse events were hypertension (22.5%) and proteinuria (17.5%). A significant proportion of patients, 67.5%, required dose reductions due to adverse events, and 7.5% permanently discontinued treatment. Notably, there were no treatment-related deaths reported.
Late-Stage Clinical Trial Results for Ribociclib Show Promise
This study, a single-arm Phase 2 trial with 40 participants, lacks a comparison treatment group, making it challenging to determine superiority over existing therapies. However, the long-term follow-up provides additional evidence to evaluate ribociclib's anti-tumor activity and potential for disease control.
Rifiktu Prepares for Direct Sales in the US, Following NCCN Listing
Rifiktu (generic name: lirafugratinib) received FDA approval on September 23rd. The approval is for adult patients with advanced biliary tract cancer who have FGFR2 gene abnormalities and have received prior treatment. Specifically, this includes patients with FGFR2 fusion or other rearrangements and unresectable locally advanced or metastatic biliary tract cancer.
Rifiktu is a selective inhibitor of the FGFR2 protein, which plays a role in the growth of cancer cells. In the ReFocus clinical trial, which led to its approval, it demonstrated an objective response rate of 46% and a median response duration of 11.8 months.
Following FDA approval, Rifiktu was listed as a subsequent treatment option in the NCCN's guidelines for biliary tract cancer. As the NCCN guidelines are widely used by healthcare professionals in the US to guide their chemotherapy choices and treatment directions, this listing provides a significant foundation for Rifiktu's entry into the prescription market.
Elleva Therapeutics Makes Waves at ESMO, Unveiling New Insights and Strategies
Elleva Therapeutics took center stage at the recent European Society for Medical Oncology (ESMO) conference, operating an exclusive booth to share valuable clinical data with medical professionals. The company's ambitious plan includes direct sales of its flagship product, Refictu, in the United States, marking a significant step towards commercialization.
The conference also revealed additional safety analysis from the ongoing ReFocus clinical trial. Notably, the incidence of dermatitis was higher (48%) in patients who had previously received immune checkpoint inhibitor therapy compared to those with no prior treatment experience (14%). However, the overall rate of grade 3 or higher skin and mucosal adverse reactions was statistically similar, at 33% and 37%, respectively.
This research underscores the potential correlation between patients' previous treatment histories and certain adverse reaction patterns. It provides crucial insights for developing tailored safety management strategies during treatment.
Furthermore, Elleva is actively pursuing an expansion of its treatment scope. The company is currently conducting a global phase 2 clinical trial, ReFocus202, targeting other solid tumors with FGFR2 fusion or rearrangement. This innovative 'cancer-agnostic' approach aims to treat tumors regardless of their origin, focusing on common genetic abnormalities.
Late-Stage Cancer Treatment: A Promising Pipeline
As the treatment response and durability are confirmed across various cancer types, further clinical evidence is expected to be accumulated for potential expansion of indications. In Europe, the review process for the marketing authorization application submitted to the European Medicines Agency (EMA) is currently underway.
Dong-Gun Kim, CEO of Eleva Therapeutics, stated, "Following the FDA approval of Rifiktu, we are preparing for its launch in the U.S. market. We have engaged in scientific exchanges with medical professionals and industry experts at ESMO, and we plan to continue our research and regulatory activities in regions beyond the U.S."
Hepatocellular Carcinoma Combination Therapy: FDA Re-Challenge with Phase 3 Results
HLB is actively developing a first-line treatment for hepatocellular carcinoma by combining ribociclib and the immunotherapeutic camrelizumab.
In the global Phase 3 trial, 'CARES-310', the median overall survival of the combination therapy group was 23.8 months, which was 8.6 months longer than the 15.2 months of the sorafenib control group. The risk of death was also 36% lower (HR 0.64) compared to the control group.
ESMO Sub-Analysis: Examining Treatment Efficacy and Antibiotic Usage
A sub-analysis presented at ESMO explored the correlation between early antibiotic use and the therapeutic effectiveness of combination therapies. It's important to note that the aforementioned survival data was derived from the overall patient population in the previous Phase 3 trial.
Eleva Pharmaceuticals resubmitted their New Drug Application to the FDA for their primary liver cancer treatment on September 9th. The new review deadline for this resubmission has not yet been disclosed.
HLB presented at ESMO, providing additional clinical evidence for their rare cancer treatment, Ribociclib, which is being developed as a liver cancer therapy. They also disclosed the preparation status for the U.S. launch of their FDA-approved drug, Rifect, which is now entering the commercialization phase, requiring actual prescriptions and revenue generation to demonstrate its market viability. Additionally, Ribociclib is expanding its development scope beyond liver cancer to other cancer types, aiming for a broader approval.
