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IO Biotech melanoma trial signals strong PFS but misses primary significance
Phase 3 results show a 19.4 month median PFS with Cylembio plus pembrolizumab in first-line advanced melanoma, but the primary endpoint narrowly missed significance; an FDA meeting is planned this fall.
Phase 3 results show a strong progression free survival signal with Cylembio plus pembrolizumab, yet the primary endpoint missed statistical significance.
IO Biotech Melanoma Trial Shows 19.4 Months PFS but Misses Primary Significance
IO Biotech reported topline results from the pivotal IO B-013/KN-D18 trial, which compared Cylembio in combination with pembrolizumab against pembrolizumab alone in 407 patients with newly diagnosed unresectable or metastatic melanoma. The primary endpoint was progression free survival (PFS) by RECIST v1.1, with a hazard ratio of 0.77 and a p-value of 0.056. Median PFS was 19.4 months for the combination versus 11.0 months for the control.
In a post hoc analysis of patients without prior anti-PD-1 therapy (n=371), the PFS improvement remained, with a median of 24.8 months versus 11.0 months and a nominal p-value of 0.037. A striking effect was seen in PD-L1 negative tumors (n=67 vs 63) with a hazard ratio of 0.54 and a median PFS of 16.6 months compared with 3.0 months on control. The combination was reported to be well tolerated, with no new safety signals. IO Biotech plans discussions with the FDA this fall to determine next steps for a potential BLA submission and will present more detailed results at future meetings.
Key Takeaways
"The magnitude and durability of clinical effect observed consistently across subgroups supports our confidence in Cylembio and its potential as a treatment for advanced melanoma patients."
statement from the IO Biotech CEO after topline results
"The significant benefit seen across patients with poor prognostic factors, including PD-L1 negative patients, cannot be overlooked."
comment from Omid Hamid on key subgroup results
"We were thrilled to play such an important part in this study and to have had the ability to offer our patients an investigational therapy that potentially offers improvements in PFS while not adding significant systemic toxicity."
quote from Jessica Hassel, lead enrolling investigator
"Delaying progression and improving survival is the ultimate treatment goal for patients and although overall survival is not yet mature, the trend we are seeing in OS with separation of the curves is encouraging."
remark from Qasim Ahmad, chief medical officer
The data show a meaningful improvement in progression free survival across most subgroups, including those with adverse prognostic factors. Yet the primary endpoint narrowly missed the predefined significance threshold, which injects caution about declaring a formal benefit. The strong signals in PD-L1 negative patients and in PD-1 treatment naive populations are encouraging, but regulators will demand robust, mature survival data and a clear safety profile before any approval. The company’s framing emphasizes potential impact on standard of care, while investors will be watching how these results translate into a regulatory path and ultimately patient access. If OS continues to favor the combination as data mature, the results could shift how melanoma is treated in the first line. For now, the results invite careful optimism rather than a verdict of success.
Highlights
- A durable PFS signal changes the way we view combination vaccines
- PD-L1 negative patients see a real difference in progression
- This result invites cautious optimism and a careful regulatory review
- The data push the field toward a new standard in first line therapy
Regulatory and investor risk after borderline results
The primary endpoint narrowly missed the statistical threshold, though subgroup signals are strong. Regulators will require robust, mature outcomes and full safety data before approving a vaccine approach in melanoma. Investors may reassess risk until a clear regulatory path is defined and OS data mature.
Regulators will weigh the full data package before any approval decision, while investors monitor the regulatory timeline.
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