The Targeted Pulse: FDA Updates and Conference Highlights

This week in oncology, the FDA expanded the treatment landscape for platinum-resistant ovarian cancer with a new approval, and significant clinical data emerged regarding novel inhibitors and antibody-drug conjugates (ADCs) across lung and gastrointestinal cancers. Additionally, a new regulatory clearance aims to streamline the administration of gene therapy in the community setting.

FDA Approves Relacorilant for Platinum-Resistant Ovarian Cancer

The FDA has approved relacorilant (Lifyorli), a first-in-class selective glucocorticoid receptor (GR) antagonist, for the treatment of adult patients with platinum-resistant ovarian cancer. The approval is based on data demonstrating that the addition of relacorilant to nab-paclitaxel significantly improves progression-free survival compared with chemotherapy alone. By modulating the GR, relacorilant is designed to reverse the anti-apoptotic effects of cortisol, which often contributes to chemotherapy resistance in solid tumors.

Setidegrasib Shows Early Efficacy in KRAS G12D-Mutant NSCLC

From this year’s European Lung Cancer Congress (ELCC), early results from a phase 1/2 study of setidegrasib indicate promising activity and a manageable safety profile in patients with non–small cell lung cancer (NSCLC) harboring the KRAS G12D mutation. As KRAS G12D remains a high-priority target with limited approved options compared to G12C, the observed objective response rates (ORR) offer a potential new pathway for patients who have progressed on standard-of-care immunotherapy and platinum-based regimens.

Trastuzumab Rezetecan Shows Promise in HER2-Expressing GI Cancers

The ADC trastuzumab rezetecan (SHR-A1811) has demonstrated encouraging antitumor activity in patients with HER2-expressing gastrointestinal (GI) cancers, including gastric and colorectal malignancies. Data presented this week highlighted a robust safety profile and significant tumor shrinkage in heavily pretreated populations. The results further validate the role of HER2-directed ADCs as a versatile backbone for treating solid tumors beyond breast and lung cancers.

Iza-bren Plus Serplulimab Hits High Marks in First-Line ES-SCLC

In the realm of small cell lung cancer (SCLC), the combination of iza-bren and serplulimab has shown high efficacy in the first-line treatment of extensive-stage disease, according to data also presented at ELCC. The combination therapy achieved a notable ORR and durable responses in early trial cohorts. This dual-immunotherapy approach aims to build upon the current standard of care by enhancing the immune system's ability to target the aggressive neuroendocrine features of SCLC.

FDA Clears Faster Thawing Method for Nadofaragene in NMIBC

The FDA has cleared a new, faster thawing method for nadofaragene firadenovec-vncw (Adstiladrin), a gene therapy used to treat high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non–muscle invasive bladder cancer (NMIBC). The update reduces the preparation time required in the clinic, addressing a common logistical hurdle in the administration of viral vector-based therapies. This clearance is expected to improve clinic throughput and patient access to this intravesical treatment.