- In April 2025, A new Phase 1 clinical trial has opened for enrollment at Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, for patients with high-grade neuroendocrine tumors. This trial will test a novel combination of immunotherapy drugs (nivolumab and ipilimumab) paired with an oncolytic (tumor-busting) virus, SVV-001 (Seneca Valley Virus-001), injected directly into the tumors
- In February 2025, The U.S. Food and Drug Administration (FDA) approved mirdametinib (Gomekli) for the treatment of neurofibromatosis type 1 (NF1) in patients who have symptomatic plexiform neurofibromas not amenable to complete surgical resection. While not directly a neuroendocrine tumor, NF1 is a genetic disorder that can cause various tumors, including some with neuroendocrine features. This approval provides a significant non-surgical option for a challenging tumor type within a related patient population
- In April 2024, Novartis received U.S. Food and Drug Administration (FDA) approval for Lutathera for the treatment of gastroenteropancreatic neuroendocrine tumors in pediatric patients. This approval marks a significant advancement, as Lutathera becomes the first radioligand therapy specifically approved for children with GEP-NETs, offering new hope for young patients and expanding the scope of precision medicine in pediatric oncology
- In March 2024, Crinetics Pharmaceuticals reported positive topline results from its Phase 2 open-label study of paltusotine, an investigational oral somatostatin receptor type 2 (SST2) nonpeptide agonist, in carcinoid syndrome. Paltusotine was shown to result in rapid and sustained reductions in the frequency and severity of flushing episodes and bowel movements associated with neuroendocrine tumors
- In December 2023, Bristol Myers Squibb (BMS) announced the acquisition of RayzeBio for approximately USD 4.1 billion. RayzeBio is a clinical-stage radiopharmaceutical company with a lead asset, RYZ101, targeting somatostatin receptor 2 (SSTR2), which is over-expressed in GEP-NETs and extensive stage small cell lung cancer (ES-SCLC). This acquisition significantly expands BMS's presence in the radiopharmaceutical therapeutics (RPTs) space and aims to strengthen its oncology pipeline
