Emerging Therapies

Second-generation antiandrogens have been studied in 6 large, randomized, double-blind, placebo-controlled, phase 3 clinical trials, PROSPER (enzalutamide), SPARTAN (apalutamide), and ARAMIS (darolutamide) in patients with nonmetastatic castration-resistant prostate cancer (nmCRPC) and TITAN (apalutamide), ARCHES (enzalutamide), and ARASENS (darolutamide) in patients with metastatic castration-sensitive prostate cancer (mCSPC) populations. This article will review the primary and secondary end points and adverse events (AEs) in these trials.

Teresa Macarulla, MD, PhD, presented the results of 2 phase 1a/1b dose-escalation/expansion trials, studies that measured the efficacy and safety of brigimadlin in patients with advanced biliary tract cancer.

Zongli Zhang, MD, PhD, presented the preliminary results of an ongoing single-arm, multicenter, open-label, real-world study analyzing the efficacy and safety of surufatinib as a second-line treatment option for patients with biliary tract cancer.

During the 2023 American Association for Cancer Research Annual Meeting, researchers presented results from a phase 2b clinical trial demonstrating significant improvement in recurrence-free survival in patients with high-risk melanoma who were treated with a combination of an investigational personalized mRNA-based vaccine plus the immune checkpoint inhibitor pembrolizumab (Keytruda), compared with pembrolizumab alone.

Results of a phase 1 clinical trial showed that approximately 66% of patients with chronic-phase chronic myeloid leukemia (CML) had a major cytogenetic response (MCyR) to the novel oral BCR-ABL1 tyrosine kinase inhibitor (TKI) vodobatinib, regardless of whether they previously received ponatinib therapy.

Sotorasib, an investigational small-molecule inhibitor of the KRAS p.G12C mutation, demonstrated promising activity and encouraging safety in patients with advanced solid tumors, in particular those with non–small-cell lung cancer (NSCLC), in the preliminary phase 1 CodeBreaK 100 trial.

Xevinapant, an investigational antagonist of IAPs (inhibitor of apoptosis proteins), prolonged overall survival (OS) in patients with locally advanced head and neck squamous-cell carcinoma (HNSCC) when added to chemoradiotherapy, according to an updated analysis of a phase 2 clinical trial that was presented at the 2020 European Society for Medical Oncology congress.

Asingle infusion of the investigational agent lifileucel—an immunotherapy using cryopreserved autologous tumor-infiltrating lymphocytes (TILs)—induced responses in more than 33% of patients with advanced melanoma whose disease progressed with multiple previous therapies, including PD-1 and BRAF/MEK inhibitors.

A novel intratumoral chemotherapy agent delivered by direct injection permits the dispersion of cytotoxic drugs into cancer cells to eradicate tumors.

The small molecule inhibitor of tyrosine kinase WEE1 adavosertib demonstrated promising clinical activity in a single-arm, phase 2 study of patients with unselected uterine serous cancer (USC), with an objective response rate of 29.4%, said Joyce F. Liu, MD, MPH, Director of Clinical Research, Division of Gynecologic Oncology, Dana-Farber Cancer Institute, Boston, MA, at the ASCO 2020 virtual annual meeting.