Nanobiotix Reports Positive Phase I/II Preliminary Data on Feasibility and Safety of NBTXR3 in Liver Cancers Trial

Good safety and feasibility of the treatment at 10% dose level

Third indication in global development confirming transferability across different cancers

Paris, France and Cambridge, Massachusetts, USA, December 14, 2016 – NANOBIOTIX, a late clinical-stage nanomedicine company pioneering novel approaches for the local treatment of cancer, today announces a positive readout of initial data on the safety and feasibility from the first patients treated in its Phase I/II trial evaluating NBTXR3 in liver cancers, including primary (hepatocellular, HCC) and metastatic tumors.

Patients with either HCC or liver metastases frequently cannot undergo surgery and have very few or no therapeutic options available. Radiation therapy has been shown to improve outcomes of these patients. Clinical trials have shown a direct correlation between higher doses of radiation therapy and increased survival, in both patient populations. The delivery of a high radiation dose is complex and cannot be done in an optimal way in most situations due to toxicity. NBTXR3 aims to amplify the energy dose within the tumor to offer better clinical results and more therapeutic options to improve the poor prognosis of these populations. Nanobiotix’s Phase I/II trial evaluates the safety and preliminary efficacy of NBTXR3 nanoparticles administrated by intra-tumoral (IT) or intra-arterial (IA) injection and activated by high precision radiation therapy, delivered as high dose fractions (stereotactic body radiation therapy (SBRT)) for the treatment of liver cancers.

Elsa Borghi, CMO of Nanobiotix: “At this stage, the safety and feasibility data of NBTXR3 in liver cancers are excellent. Observations are similar to the results of our more advanced trials: Soft Tissue Sarcoma and Head and Neck cancers. This is significant because these trials cover very different patient and disease profiles. Based on the information gathered to date, we anticipate that by the end of this phase, we will have identified the appropriate conditions to use NBTXR3 in these patients populations. Once again, all transferability data show the potential of broad applicability of NBTXR3 for use with radiotherapy in the treatment of solid tumors.”

Preliminary data results:

1. Good safety profile with no serious adverse events recorded. Two sub-groups of patients have been treated at 10% dose of NBTXR3, with intratumoral injections (IT), using either 24 Gy or 45 Gy total radiation dose, based on patients dosimetric constrains. Intra-arterial injection has not been explored so far because the IT injection has been shown to be feasible and successful. Good safety has been demonstrated within these patients as well. To date, all treated patients have completed their radiation therapy course, confirming good local tolerability and no changes in liver hepatic functions (MELD Score evaluation).

2. Treatment feasibility and appropriate distribution demonstrated The data validate the feasibility of the injection with a volume level equivalent to 10% of the baseline tumor volume in both patient populations: primary cancer (HCC) and liver metastasis. The product appears to stay within the tumors with no leakage in the surrounding healthy tissues from the day of injection until end of radiotherapy treatment. It confirms and supports the findings reported from the clinical trials in soft tissue sarcoma or head and neck cancers patients.

3. 10% volume level secured, further levels under evaluation. The 10% dose level was successfully evaluated in HCC and metastatic patients. The 10% dose level is the recommended NBTXR3 volume for the treatment of soft tissue sarcoma (STS) in the act.in.sarc study (www.actinsarc.com), the most advanced indication developed by Nanobiotix (Phase II/III). The trial is now recruiting next dose levels to evaluate safety and feasibility at higher doses along with exploratory efficacy endpoints (complete response rate, progression-free survival, and overall survival).