Nanobiotix (NANO) Study Update summary

Study background and product overview

• Phase 1 Study 1100 evaluated NBTXR3, a first-in-class radioenhancer activated by radiation and designed for intratumoral injection, in recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC), including both anti-PD-1 naïve and resistant patients.

• The product aims to improve local tumor control and prime a systemic immune response, with potential for tumor-agnostic and combination-agnostic use.

• NBTXR3 has demonstrated proof of concept in humans and is being developed in partnership with Johnson & Johnson and MD Anderson Cancer Center.

• The study included heavily pretreated patients, with 33 anti-PD-1 naïve and 35 anti-PD-1 resistant participants.

• Data cutoff for this update was April 17, 2024.

Patient population and trial design

• The trial enrolled 68 patients with R/M HNSCC, both anti-PD-1 naïve and resistant, with significant prior treatment histories and high tumor burden.

• Most patients had a low CPS score (<20), indicating poor prognosis, and a significant proportion were HPV-positive.

• The expansion phase focused on two cohorts: anti-PD-1 naïve and anti-PD-1 resistant, both receiving NBTXR3, radiation (SBRT), and anti-PD-1 therapy (nivolumab or pembrolizumab).

• Over 80% of anti-PD-1 resistant patients had progressive disease at study entry.

• Primary endpoints were safety and recommended phase 2 dose; secondary endpoints included ORR, safety, feasibility, and pharmacokinetics.

Safety findings

• NBTXR3 with radiation and anti-PD-1 showed a favorable safety profile, with less than 10% grade 3 or higher adverse events related to the regimen.

• No unexpected or new side effects emerged compared to previous studies or literature.

• A single intra-tumoral injection of NBTXR3 followed by SBRT activation was well tolerated.

• Serious grade 3+ adverse events related to the regimen occurred in 8.8% (6/68) of patients.