Ipsen receives positive CHMP opinion for Cabometyx ® in previously treated advanced neuroendocrine tumors

Ipsen announced that the EMA's CHMP issued a positive opinion for Cabometyx (cabozantinib) for advanced neuroendocrine tumors (NETs) after prior systemic therapy, based on the CABINET Phase III trial. The trial demonstrated statistically significant reductions in disease progression or death compared to placebo, with median progression-free survival of 13.8 months versus 4.4 months in pancreatic NETs and 8.4 months versus 3.9 months in extra-pancreatic NETs; if approved by the European Commission, Cabometyx would be the first systemic therapy in the EU for previously treated NETs regardless of tumor site or grade.

Analysis

Ipsen (IPSEY) has achieved a significant regulatory milestone with the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issuing a positive opinion for Cabometyx® (cabozantinib) for treating adult patients with advanced, well-differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumors who have progressed following prior systemic therapy. This recommendation, critically underpinned by the CABINET Phase III trial results, positions Cabometyx to potentially become the first and only systemic therapy approved in the European Union for this broad NET patient population, regardless of tumor site, grade, or specific previous non-somatostatin analogue-based systemic therapy. The CABINET trial demonstrated compelling efficacy, with Cabometyx significantly reducing the risk of disease progression or death compared to placebo: median progression-free survival (PFS) reached 13.8 months for Cabometyx versus 4.4 months for placebo in the pNET cohort (hazard ratio [HR] 0.23; p<0.001), and 8.4 months versus 3.9 months in the epNET cohort (HR 0.38; p<0.001). This development is particularly salient given the increasing diagnosed prevalence of NETs and the often-limited treatment options available upon disease progression, especially for challenging subgroups such as the 27% of patients with lung NETs who currently lack approved options after non-somatostatin analogue-based systemic therapy. While overall survival data were not mature at the time of analysis and were potentially confounded by the trial's crossover design, the safety profile of Cabometyx was consistent with its known characteristics, with no new safety signals identified, and health-related quality of life was reported as maintained or improved. A final decision on approval from the European Commission is anticipated in the third quarter of 2025, which, if favorable, would notably expand Cabometyx's market reach for Ipsen, the licensee for commercialization and further clinical development outside the U.S. and Japan from originator Exelixis (EXEL).

Key Decisions for Investors

• Investors should interpret the CHMP positive opinion as a substantial de-risking event for Ipsen (IPSEY), significantly bolstering the probability of European Commission approval for Cabometyx in this expanded NET indication by Q3 2025 and facilitating access to a market with considerable unmet medical need.
• It is advisable to monitor Ipsen's execution towards final EC approval and its subsequent commercialization strategy, as the robust progression-free survival data from the CABINET trial suggest a strong clinical value proposition, while acknowledging the current immaturity of overall survival data as a factor for ongoing observation.
• Consider the potential for incremental revenue contributions to Ipsen from Cabometyx post-EU approval, and peripherally for Exelixis (EXEL) through royalties or milestone payments, given Cabometyx's positioning as a potential first-in-class systemic therapy for this broad NET patient group in Europe.