AlphaMedix™ (212Pb-DOTAMTATE) has successfully met all primary efficacy endpoints in a pivotal phase 2 clinical trial aimed at treating patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The positive outcomes from the ALPHAMEDIX-02 study highlight the significant potential of this innovative treatment for both naïve and previously treated patients with unresectable or metastatic tumors. These findings suggest that Targeted Alpha Therapy utilizing lead-212 could offer a meaningful new treatment option for individuals battling this challenging form of cancer.
The Paris-based biotechnology firm Orano Med reported on October 8, 2025, that AlphaMedix™ not only met all primary efficacy goals but also demonstrated impressive overall response rates (ORR) and sustained clinical benefits. These advantages were observed in both cohorts of patients participating in the trial: those who had never received peptide receptor radionuclide therapy (PRRT)-naïve patients, and those who had previously undergone PRRT (PRRT-exposed). Significant improvements were also noted in secondary endpoints such as progression-free survival (PFS) and overall survival (OS).
Dr. Volker Wagner, Chief Medical Officer at Orano Med, expressed enthusiasm about the results: “The positive results from the ALPHAMEDIX-02 study represent a pivotal movement for the Orano Med 212Pb-based platform and underscore the profound potential of lead-212-based radiopharmaceuticals in addressing critical unmet needs for patients with GEP-NETs. We are very encouraged by AlphaMedix™’s consistent and clinically meaningful activity across both peptide receptor radionuclide therapy (PRRT)-naïve and PRRT-exposed patients.”
One of the critical advantages of alpha-emitters, like lead-212, is their ability to deliver potent therapeutic effects directly to cancer cells while minimizing damage to surrounding healthy tissue. This characteristic is linked to the short range of alpha particles, which enhances the treatment’s specificity. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to AlphaMedix™ in February 2024 for its potential to treat PRRT-naïve patients with unresectable or metastatic, progressive SSTR-expressing GEP-NETs.
Dr. Christopher Corsico, Global Head of Development at Sanofi, who is collaborating with Orano Med on this project, stated, “The promising ALPHAMEDIX-02 results represent a significant step forward, reinforcing the potential of targeted alpha therapy to deliver precise treatment for GEP-NETs. These data, demonstrating clinically meaningful activity and a manageable safety profile, underscore our unrelenting commitment to developing innovative therapies for patients with difficult-to-treat cancers. We look forward to advancing AlphaMedix™ and working with Orano Med and regulators to bring this important treatment to the GEP-NET community as soon as possible.”
The ALPHAMEDIX-02 study is currently ongoing, and a comprehensive presentation of its full results is scheduled for the 2025 European Society for Medical Oncology (ESMO) Congress. The outcomes of this study will also play a crucial role in forthcoming discussions with healthcare authorities about the potential approval of AlphaMedix™.
Study Details
The ALPHAMEDIX-02 trial is an open-label, multicenter phase 2 study designed to evaluate the efficacy and safety of AlphaMedix™ in patients whose tumors are histologically confirmed to be unresectable or metastatic GEP-NETs. The study enrolled two distinct cohorts: 35 PRRT-naïve patients and 26 who had previously received PRRT but exhibited progressive disease after up to four doses of 177Lu-DOTATATE, with their last treatment occurring at least six months prior to Day 1 of the study. In both groups, AlphaMedix™ was administered at a dosage of 67.6 μCi/kg every eight weeks for up to four cycles, with a maximum allowable dosage of 6 mCi per cycle. The primary endpoints included overall response rate as determined by RECIST1.1 criteria and safety measures, while secondary endpoints focused on progression-free and overall survival rates.
Understanding Neuroendocrine Tumors (NETs)
Neuroendocrine tumors (NETs) are a diverse group of cancers derived from neuroendocrine cells, often arising in the gastrointestinal tract and pancreas, but they can also be seen in other organs, including the thymus and lungs. Although NETs are categorized as a rare form of cancer, with an estimated incidence of about 35 cases per 100,000 individuals globally, the prevalence is on the rise. In the U.S., approximately 12,000 new cases are diagnosed each year, with the average five-year survival rate for metastatic patients at 60%.
About Orano Med
Orano Med, a subsidiary of the Orano Group, is a clinical-stage biotechnology company that focuses on developing targeted cancer therapies utilizing lead-212, a potent alpha-emitting radioisotope. The company is actively expanding its capabilities for manufacturing 212Pb-based therapies in both North America and Europe.
About Sanofi
Sanofi is a research-driven biopharmaceutical company dedicated to improving lives through innovative medicines. With a commitment to addressing critical healthcare challenges, Sanofi continues to pursue advancements in drug development to benefit countless patients around the world. Their ongoing partnership with Orano Med is aimed at enhancing treatment options for patients facing challenging cancers.
