While ESMO 2023 is just around the corner, the leading Pharma players including AstraZeneca, Merck, BMS, Novartis, Dizal Pharma, Amgen, Daiichi Sankyo, etc. are all geared up for this conference to present the data readouts and final analysis. The hype for the session is the unveiling of the first positive Phase III results of Merck’s Belzutifan in advanced renal cell cancer (RCC).

RCC Highlights

• Abstract Number – #LBA88

• Abstract Type - Proffered Paper Session
• Indication - Advanced RCC

Title: Merck’s Belzutifan: First novel mechanism to demonstrate potential in advanced RCC in recent years.

Executive Summary: First positive Phase III results for WELIREG from LITESPARK-005 showed statistically significant improvements in PFS versus everolimus in patients with advanced RCC that has progressed after prior PD-1/L1 and VEGF-targeted therapies.

Main Content - Patients with advanced RCC face low survival rates, and for those whose cancer progresses following PD-1/L1 and VEGF-TKI therapies, there is a need for new treatment options that can reduce their risk of disease progression or death. LITESPARK-005 is the first Phase III trial with positive topline results, evaluating WELIREG, an oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor for the treatment of adult patients with advanced RCC. WELIREG showed a statistically significant and clinically meaningful improvement in PFS compared to everolimus, based on a pre-specified interim analysis. In addition, a statistically significant improvement in the ORR was also demonstrated. However, the OS result did not reach statistical significance. OS will be tested at a subsequent analysis. The safety profile of WELIREG in this trial was consistent. Results will be presented at the upcoming medical meeting and shared with regulatory authorities.

Based on the LITESPARK-005 trial, the FDA has accepted and granted priority review for a supplemental new drug application (sNDA) seeking approval for WELIREG. The FDA has set a Prescription Drug User Fee Act (PDUFA) date of January 17, 2024.