AlphaMedixTM, known scientifically as 212Pb-DOTAMTATE, has successfully met all primary efficacy goals in its phase 2 clinical trial, showing promising benefits for patients suffering from gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The investigational treatment demonstrated sustained positive effects in both treatment-naïve and previously treated patients with unresectable or metastatic GEP-NETs, positioning it as a potential new therapy in the realm of Targeted Alpha Therapy.
The promising results emerged from the ALPHAMEDIX-02 phase 2 study (clinical study identifier: NCT05153772), which examined the efficacy and safety of AlphaMedixTM. According to the findings, the treatment not only met all primary efficacy endpoints but also exhibited significant overall response rates (ORR) along with prolonged clinical benefits in the two categories of patients: those new to peptide receptor radionuclide therapy (PRRT-naïve) and those who have previously undergone treatment (PRRT-exposed).
Key secondary outcomes, such as progression-free survival (PFS) and overall survival (OS), also showed positive trends across both groups. Notably, AlphaMedixTM maintained a manageable safety profile consistent among the participants in the study.
Dr. Volker Wagner, Chief Medical Officer at Orano Med, remarked, “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 PRRT-naïve and PRRT-exposed patients.”
Alpha-emitters are currently being evaluated for their effectiveness in targeting tumors while reducing damage to healthy tissue, owing to their limited range. In February 2024, the U.S. Food and Drug Administration (FDA) designated AlphaMedix™ as a Breakthrough Therapy for the treatment of PRRT-naïve patients with progressive, unresectable SSTR-expressing GEP-NETs, underscoring its therapeutic potential.
Dr. Christopher Corsico, Global Head of Development at Sanofi, added, “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.”
As the study continues, the complete findings are set to be unveiled at the 2025 European Society for Medical Oncology (ESMO) Congress. These results will also serve as a foundation for future discussions with health authorities, although it is important to note that AlphaMedix™ has not yet received any regulatory approvals.
Study Details
The ALPHAMEDIX-02 study is a phase 2, open-label, multicenter trial investigating AlphaMedix™ (212Pb-DOTAMTATE) in patients with verified unresectable or metastatic GEP-NETs that express somatostatin receptors and present measurable disease. The trial consisted of two groups: PRRT-naïve patients (35 participants) and PRRT-exposed patients (26 participants). Those who had undergone previous treatment had to have experienced disease progression after receiving up to four doses of 177Lu-DOTATATE, with their most recent dose given at least six months prior to the study’s initiation. In both cohorts, AlphaMedix™ was administered at a dosage of 67.6 μCi/kg every eight weeks for a maximum of four cycles.
Understanding Neuroendocrine Tumors (NETs)
Neuroendocrine tumors (NETs) are a diverse group of malignancies arising from neuroendocrine cells. Although they predominantly occur in the gastrointestinal tract and pancreas, NETs can also develop in various other tissues, including the thymus, lungs, and even rare sites such as the ovaries and heart. While the frequency of NETs is on the rise worldwide, they remain classified as a rare cancer, estimated to affect approximately 35 individuals out of every 100,000 globally. In the United States alone, around 12,000 new cases of neuroendocrine tumors are diagnosed each year, with a five-year survival rate of about 60% in metastatic stages.
About Orano Med
Orano Med, a subsidiary of the Orano Group, is a biotechnology firm focused on advancing targeted therapies for cancer utilizing the specific characteristics of the lead-212 (212Pb) isotope, recognized as a potent treatment option through Targeted Alpha Therapy (TAT). With facilities dedicated to 212Pb manufacturing in both France and the United States, Orano Med is enhancing its capabilities to produce 212Pb radiolabeled pharmaceuticals across North America and Europe.
About Sanofi
Sanofi, an innovative biopharmaceutical company, is committed to improving global health through the development of groundbreaking medicines and vaccines. Listed on EURONEXT: SAN and NASDAQ: SNY, Sanofi employs advanced technology and deep expertise in immunology to address pressing healthcare challenges worldwide.
