IO Biotech (IOBT) Piper Sandler 37th Annual Healthcare Conference summary

Key clinical developments

• Phase III trial of IO102-IO103 with pembrolizumab in metastatic melanoma showed clinically meaningful PFS improvement (19.4 vs. 11 months), though statistical significance was narrowly missed.

• Subgroup analyses revealed consistent drug effect across all stratification markers, with a favorable safety profile and no added toxicity.

• OS data is trending positively (hazard ratio 0.79), with more mature results expected next year.

• First therapeutic cancer vaccine to show clear benefit in metastatic disease setting in a randomized Phase III trial.

• Feedback from physicians and KOLs has been highly positive despite the primary endpoint miss.

Regulatory and trial strategy updates

• FDA recommended against BLA submission due to lack of statistical significance, despite clear clinical benefit and safety.

• New global adaptive Phase II/III trial planned, combining IO102-IO103 with Opdualag, reflecting updated standard of care and FDA input.

• EU regulatory engagement ongoing, with potential for MAA submission in 2026, leveraging different guidelines and possible subgroup filings.

• Phase II basket studies in neoadjuvant/adjuvant melanoma and SCCHN are underway, with initial data expected by year-end and presentations in 2026.

Pipeline and platform insights

• IO102-IO103 demonstrates pan-tumor activity, with positive data in head and neck and encouraging results in lung cancer.

• Early immune modulatory signatures may help identify responsive patient populations.

• Pipeline assets IO112 (Arginase 1) and IO170 (TGF-beta) have generated strong interest, with IO112 nearing IND readiness.

• Focus remains on melanoma, but broader indications and partnerships are being considered for the future.