Sanofi's AlphaMedix Meets Primary Endpoints in Phase 2 Study for Neuroendocrine Tumors

Sanofi has reported positive results from the ALPHAMEDIX-02 Phase 2 study, where its investigational drug AlphaMedix (212Pb-DOTAMTATE) met all primary efficacy endpoints. The study focused on patients with unresectable or metastatic gastroenteropancreatic neuroendocrine tumors (GEP-NETs). AlphaMedix, a somatostatin receptor (SSTR)-targeted alpha therapy using the lead-212 (212Pb) isotope, showed clinically meaningful objective response rates (ORR) and prolonged clinical benefits in both peptide receptor radionuclide therapy (PRRT)-naïve and PRRT-exposed patient groups.

The study (clinical study identifier: NCT05153772) also observed benefits in key secondary endpoints, including progression-free survival (PFS) and overall survival (OS), across both cohorts. The safety profile of AlphaMedix was manageable and similar between the groups. Volker Wagner, MD, PhD, Chief Medical Officer at Orano Med, which developed the 212Pb-based platform, stated that the results underscore the profound potential of lead-212-based radiopharmaceuticals for addressing critical unmet needs in GEP-NET patients.

Alpha-emitters are being studied for their potential to deliver highly potent and targeted tumor activity while potentially reducing exposure to surrounding healthy tissue due to the short range of alpha particles. In February 2024, AlphaMedix received a Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its potential to treat PRRT-naïve patients with unresectable or metastatic, progressive SSTR-expressing GEP-NETs.

Christopher Corsico, MD, Global Head of Development at Sanofi, described the results as a significant step forward in advancing targeted alpha therapy for GEP-NETs. Sanofi plans to advance AlphaMedix and engage in discussions with health authorities. Full study results are scheduled for presentation at the 2025 European Society for Medical Oncology (ESMO) Congress.