Clinical Trials Highlight Advances in Neuroendocrine Tumor Research
Research into neuroendocrine tumors (NETs) continues to progress, with clinical trials exploring both new treatment approaches and improvements to existing therapies. Recent updates highlight progress in targeted radiopharmaceutical therapies, regulatory developments that could expand treatment access, and early-stage studies investigating innovative immune-based treatments. In this roundup, we highlight several studies and policy milestones that reflect the expanding landscape of neuroendocrine cancer research and represent important steps toward improving care for those living with the disease.
Phase 3 COMPETE trial analysis shows promising results for ITM-11 in pancreatic NETs (Pan-NETs)
Updated findings from the Phase 3 COMPETE trial suggest that the investigational radiopharmaceutical therapy 177Lu-edotreotide (ITM-11), developed by ITM Isotope Technologies Munich, may improve outcomes for some people living with pancreatic neuroendocrine tumors (Pan-NETs).
Researchers presented updated findings at the 2026 Annual Meeting of the European Neuroendocrine Tumor Society (ENETS). In a subgroup analysis of patients with Pan-NETs, ITM-11 demonstrated longer progression-free survival and higher objective response rates compared with the targeted therapy everolimus.
The Phase 3 COMPETE trial enrolled 309 participants with inoperable or progressive grade 1 or grade 2 gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Participants were randomly assigned to receive either ITM-11 or everolimus.
Among the participants, 178 had Pan-NETs. In this group, participants treated with ITM-11 experienced a median progression-free survival of 24.5 months, compared with 14.7 months for those receiving everolimus. Objective response rates were also higher: 33.3% of participants receiving ITM-11 had a measurable response, compared with 3.6% of those treated with everolimus.
Key takeaways:
- A subgroup analysis from the Phase 3 COMPETE trial showed longer progression-free survival with ITM-11 compared with everolimus in Pan-NETs.
- Participants receiving ITM-11 also experienced higher objective response rates and longer overall survival.
- Ongoing follow-up will help determine long-term outcomes.
FDA grants tentative approval for potential generic PRRT therapy
A recent regulatory decision could eventually expand access to peptide receptor radionuclide therapy (PRRT), a targeted radiopharmaceutical treatment used for some neuroendocrine tumors.
The U.S. Food and Drug Administration (FDA) has granted tentative approval for lutetium Lu 177 dotatate (PNT2003), developed by Lantheus, a therapy designed to be radioequivalent to LUTATHERA®, an FDA-approved PRRT used to treat certain NETs.
Tentative approval means the FDA has completed its review and found that PNT2003 meets the requirements for approval. However, it cannot yet be marketed in the United States because final approval is delayed by patent-related litigation protections now in place through June 2026. If final approval is granted, PNT2003 could become available as an additional PRRT option for eligible patients in the United States.
Key takeaways:
- The FDA has granted tentative approval for PNT2003, a therapy designed to be radioequivalent to the PRRT drug LUTATHERA®.
- The therapy cannot yet be marketed because of patent protections tied to the reference product.
- If approved for marketing in the future, PNT2003 could become an additional PRRT option for patients with NETs.
Early clinical trial advances CDH17 CAR T-cell therapy for gastrointestinal cancers and NETs
Researchers are also exploring new cell-based therapies that could one day be used to treat neuroendocrine tumors and other gastrointestinal cancers.
A Phase 1/2 clinical trial investigating CHM CDH17, a CAR T-cell therapy developed by Chimeric Therapeutics, has advanced to a higher dose level after early testing showed encouraging safety findings. The therapy targets the CDH17 cell surface protein and is being studied in participants with advanced colorectal cancer, gastric cancer, and intestinal neuroendocrine tumors.
Early results from the first two dose levels showed no safety concerns or off-target effects. Some participants experienced disease control and evidenceof anti-tumor activity. Researchers have now advanced the study to a third dose level, in which participants receive a higher dose of CAR T-cells.
The Phase 1 portion of the study is designed to determine a recommended Phase 2 dose and evaluate the therapy’s safety and effectiveness.
Key takeaways:
- A Phase 1/2 clinical trial of CHM CDH17, a CDH17-targeting CAR T-cell therapy, has advanced to a higher dose level.
- Early testing showed encouraging safety findings and preliminary evidence of anti-tumor activity.
The study includes participants with gastrointestinal cancers, including intestinal neuroendocrine tumors.
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Interested in clinical trials? To explore studies that are currently enrolling patients with neuroendocrine cancer, visit NETRF’s Clinical Trial Finder.