Tipiracil Hydrochloride/Trifluridine

FDA sets a PDUFA target action date of March 20, 2025Resubmission is supported by the final survival analysis of CARES-310 study, presented at ASCO 2024 FORT LEE, N.J., Oct. 21, 2024 (GLOBE NEWSWIRE) -- Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd., today announced the U.S. Food and Drug Administration (FDA) accepted the resubmission of a new drug application (NDA) for its investigational drug rivoceranib, an oral VEGF-TKI, in combination with camrelizumab, a PD-1 inhibitor, as a first-line systemic treatment for unresectable or metastatic hepatocellular carcinoma (uHCC). The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 20, 2025. “We believe rivoceranib plus camrelizumab has the potential to change the clinical practice in the first-line setting for patients with advanced liver cancer. The results of the pivotal CARES-310 trial demonstrated significant improvements in overall survival, with a very manageable safety profile compared with currently approved uHCC therapies. Elevar is committed to working with the FDA to bring this combination to market for patients and healthcare providers,” commented Chris Galloway, M.D., senior vice president of clinical and medical affairs at Elevar Therapeutics. The resubmission included the final analysis of the Phase 3 CARES-310 study presented during the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting on June 1, which reported median overall survival (mOS) of 23.8 months, the longest mOS for any treatment in a global Phase 3 trial for patients with uHCC, confirming the combination of camrelizumab and rivoceranib continued to show sustained long-term survival as a first-line treatment for patients with uHCC.i With the efficacy results generally consistent across all subgroups, the data suggest the combination has the potential to benefit a global uHCC population. The data also demonstrated consistent efficacy across patients with both viral and non-viral etiologies. About CARES-310 The CARES-310 study, an international, randomized, open-label, Phase 3 trial, with 543 patients with uHCC who had not previously received systemic treatment was the first to demonstrate significant progression-free survival (PFS) and overall survival (OS) benefits with immunotherapy plus an anti-angiogenic tyrosine kinase inhibitor (TKI) over standard TKI as first-line treatment for uHCC. In the primary analysis of PFS (data cut-off [DCO], May 10, 2021) and interim analysis of OS (DCO, Feb. 8, 2022), significant improvements were observed with camrelizumab (C; anti-PD-1 antibody) + rivoceranib (R; VEGFR2-TKI) vs. sorafenib (S). In the final analysis (FA) of the CARES-310 study, after an additional follow-up of ~16 months, median OS was significantly prolonged with C+R vs. S (23.8 mo [95% CI 20.6-27.2] vs. 15.2 mo [95% CI 13.2-18.5]; hazard ratio (HR) 0.64 [95% CI 0.52-0.79]; 1-sided p <0.0001). OS rate with C+R vs. S was 49.0% vs. 32.6% at 24 mo, and 37.7% vs. 24.8% at 36 mo. OS benefits with C+R was generally consistent across subgroups, regardless of geographical region, race, and etiology. Benefits in PFS, objective response rate (ORR) and duration of response (DoR) with C+R vs. S were also sustained after prolonged follow-up. Safety data aligned with the interim OS analysis, with no new signals noted. In the FA, C+R continued to show clinically meaningful survival improvement compared with S, with manageable safety. The extended follow-up further confirmed the favorable benefit-to-risk profile of C+R, supporting it as a new first-line treatment option for uHCC. About Hepatocellular Carcinoma Worldwide each year more than 800,000 people are diagnosed with liver cancerii and the disease is the cause of more than 830,000 deaths.iii Hepatocellular Carcinoma (HCC) is the most common type of liver cancer and most frequently develops in people with chronic underlying liver inflammation which may be from viral and non-viral causes. HCC typically has a poor prognosis with limited treatment options and continues to be a diagnosis with an ongoing urgent medical need. About Rivoceranib Rivoceranib, a small-molecule tyrosine kinase inhibitor (TKI), is a highly selective inhibitor of vascular endothelial growth factor receptors (VEGFRs), a primary pathway for tumor angiogenesis. VEGFR inhibition is a clinically validated target to limit tumor growth and disease progression. Rivoceranib is currently being studied as a monotherapy and in combination with chemotherapy and immunotherapy in various solid tumor indications. Several clinical studies were completed in patients with uHCC (in combination with camrelizumab), gastric cancer (as a monotherapy and in combination with paclitaxel), adenoid cystic carcinoma (as a monotherapy) and colorectal cancer (in combination with Lonsurf®). Rivoceranib, under the name apatinib (Aitan®), was the first TKI approved in gastric cancer in China (October 2014). It was also approved in China in combination with camrelizumab as a first-line treatment for uHCC (January 2023). The drug has been studied in more than 6,000 patients worldwide and was well tolerated in clinical trials with a comparable safety profile to other TKIs and VEGF inhibitors. Orphan drug designations have been granted for gastric cancer (U.S., EU and South Korea), adenoid cystic carcinoma (U.S.) and uHCC (U.S. and EU). Elevar Therapeutics, Inc. holds the global rights (excluding China) to rivoceranib and has partnered for its development and marketing with HLB-LS in South Korea. Jiangsu Hengrui Pharmaceuticals Company Ltd., (Hengrui Pharma), is the Chinese -territory license-holder of rivoceranib. About Camrelizumab Camrelizumab (SHR-1210) is a humanized monoclonal antibody that binds to the programmed death-1 (PD-1) receptor. Blockade of the PD-1/PD-L1 signaling pathway is a therapeutic strategy showing success in a wide variety of solid and hematological cancers. Camrelizumab is developed by Hengrui Pharma and has been studied in more than 5,000 patients. Currently, 50 clinical trials are underway in a broad range of tumors (including liver cancer, lung cancer, gastric cancer, and breast cancer, etc.) and treatment settings. Camrelizumab, under the brand name AiRuiKa®, is currently approved for eight indications in China, including monotherapy for the treatment of HCC (second-line), in combination with rivoceranib as a treatment for uHCC (first-line), relapsed/refractory classic Hodgkin’s lymphoma (third-line), esophageal squamous cell carcinoma (second-line) and nasopharyngeal carcinoma (third-line or further) and in combination with chemotherapy for the treatment of non-small cell lung cancer (non-squamous and squamous), esophageal squamous cell carcinoma and nasopharyngeal carcinoma in the first-line setting. The U.S. Food and Drug Administration granted Orphan Drug Designation to camrelizumab for advanced HCC in April 2021 and by the European Medicines Agency in August 2024. In October 2023, Elevar licensed camrelizumab, an anti-PD-1 antibody, for commercialization from Jiangsu Hengrui Pharmaceuticals Co., Ltd. (Hengrui Pharma) worldwide excluding Greater China and Korea. To learn more, visit ElevarTherapeutics.com. Media Contact Jeanette Bressi Head, Corporate Communications, Elevar Therapeutics jbressi@elevartherapeutics.com609-439-3997 Investor Relations Contact Wade Smith Chief Financial & Business Officer, Elevar Therapeutics wsmith@elevartherapeutics.com i CARES-310 study final analysisii Key Statistics About Liver Cancer | American Cancer Societyiii Key Statistics About Liver Cancer | American Cancer Society