• First results from the expansion part of the Company’s phase I trial, evaluating the potential of first-in-class NBTXR3 to improve treatment outcomes for elderly patients with locally advanced head and neck cancer ineligible for chemotherapy or intolerant to cetuximab, were presented today at ASCO 2020
• NBTXR3 has been administered to 40 patients in the trial and was well tolerated, maintaining the safety profile observed in the dose escalation part of the phase I study
• Analysis of 30 evaluable patients for efficacy showed a primary tumor objective response rate of 83%, including a complete response rate of 60% in the target lesion, which are the co-primary endpoints of the study
• The high rate of ORR observed in the escalation phase (69%) has been further improved in the expansion phase part, to reach 83%
• Preliminary safety and efficacy data further reinforce NBTXR3 as a potential new option for head and neck cancer patients
“Elderly head and neck cancer patients are currently underserved by the standard of care. Those ineligible to receive chemotherapy or intolerant to cetuximab are treated with radiation therapy alone, which is limited in its ability to offer satisfactory treatment outcomes. I am encouraged that the data from the expansion part of our phase I study shows that NBTXR3 activated by radiation therapy could be a new option to improve therapeutic outcomes in this area. Continuing the development pathway into phase III will be an exciting step, and I am optimistic that what we have seen will continue.” – Professor Christophe Le Tourneau, Curie Institute and Principal Investigator for the Study
Paris, France ; Cambridge, Massachusetts (USA) ; May 29, 2020 – NANOBIOTIX (Euronext: NANO – ISIN: FR0011341205 – the “Company”), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, today announced new data from the expansion part of its phase I trial, evaluating the potential of first-in-class NBTXR3 activated by radiation therapy to improve treatment outcomes for elderly patients with locally advanced head and neck cancer ineligible for chemotherapy or intolerant to cetuximab. The data were published as part of the virtual scientific program at the 2020 annual meeting of the American Society for Clinical Oncology (ASCO).
Study 102: A phase I trial of hafnium oxide nanoparticles activated by radiotherapy in cisplatin-ineligible locally advanced HNSCC patients
Authors: Christophe Le Tourneau, Valentin Calugaru, Edith Borcoman, Victor Moreno, Emiliano Calvo, Xavier Liem, Sebastien Salas, Bernard Doger, Olivier Choussy, Maria Lesnik, Xavier Mirabel, Nathalie Badois, Samar Krhili, Josefin Blomkvist, Nicolas Fakhry, Stéphanie Wong-Hee-Kam, Caroline Hoffmann.
J Clin Oncol 38: 2020 (suppl; abstr 6573). DOI: 10.1200/JCO.2020.38.15_suppl.6573.
Abstract Number: 6573
Study Design
After reporting promising early signs of activity from the dose escalation part of its phase I trial evaluating the safety and feasibility of NBTXR3 activated by radiation therapy in elderly patients with locally advanced head and neck squamous cell carcinoma (HNSCC), the Company launched an expansion cohort to confirm results and observed trends with a larger population treated at the recommended dose. This part of the study is expected to recruit a total of 44 evaluable patients. To date, 40 patients have been recruited, 30 of whom are evaluable for efficacy and are included in the data presented at ASCO 2020.
Topline Results
As previously announced, in the dose escalation part of the study NBTXR3 activated by radiation therapy was safe and well tolerated. The recommended phase 2 dose (RP2D) was determined to be 22% of baseline tumor
volume. Among 16 evaluable patients, injected lesion complete response rate was 56% and overall objective
response rate was 69%.
Regarding the new, expansion part data, analysis of 40 patients dosed showed that NBTXR3 activated by
radiation therapy remains safe and well tolerated. In terms of efficacy, for the 30 evaluable patients, investigators
observed, at a median time of 5 months after NBTXR3 injection, an overall objective response rate of 83% and
an overall complete response rate of 43% and objective response rate of the primary tumor (target lesion) of
83% with a complete response rate of the primary tumor of 60%. The safety profile was consistent with the dose
escalation part and the efficacy data improved (i.e. an increase in overall objective response rate from 69% in
the dose escalation part to 83% in the dose expansion part).
In the safety population (all treated patients, N=40), three serious adverse events (SAEs) related to NBTXR3
were observed (0.7% of all AEs), comprising one case each of: Grade 4 tumor hemorrhage also related to
radiotherapy, Grade 3 mucosal inflammation and Grade 2 swollen tongue also related to the injection procedure.
Two SAEs related to the injection procedure were reported (0.5% of all AEs), comprising: two cases of swollen
tongue, of which one was Grade 2 and also related to NBTXR3, and one was Grade 4. The radiotherapyassociated
safety profile was as expected with the most frequently occurring AEs being stomatitis and skin
injury. Three deaths due to AEs related to radiotherapy and other causes were observed. Four other patients
died of non-oncologic or non-toxicity-related reasons.
Next Steps in Head and Neck Cancer
The expansion part of the phase I trial will continue to recruit until reaching 44 evaluable patients. In parallel,
subject to the FDA’s pending review, the Company intends to globally launch a pivotal phase III trial. In the
planned phase III trial, a futility analysis is expected 18 months after the first patient is randomized, and an
interim analysis of progression-free survival (PFS) is expected at 24-30 months.
NBTXR3 for the treatment of locally advanced HNSCC patients who are not eligible for platinum-based
chemotherapy received Fast Track designation from the FDA in February 2020. Fast Track designation is a
process designed to facilitate the development and accelerate the review of drugs for serious conditions that
have the potential to address unmet medical needs. The purpose is to expedite the availability of new treatment
options for patients.
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About NBTXR3
NBTXR3 is a first-in-class product designed to destroy tumors through physical cell death when activated by radiotherapy.
NBTXR3 has a high degree of biocompatibility, requires one single administration before the first radiotherapy treatment
session, and has the ability to fit into current worldwide radiation therapy standards of care. The physical mode of action of
NBTXR3 makes it applicable across solid tumors.
NBTXR3 is being evaluated in locally advanced head and neck squamous cell carcinoma (HNSCC) of the oral cavity or
oropharynx in elderly patients unable to receive chemotherapy or cetuximab with limited therapeutic options. Promising
results have been observed in the phase I trial regarding local control. In the United States, the Company has started the
regulatory process to commence a phase III clinical trial in locally advanced head and neck cancers.
Nanobiotix is also running an Immuno-Oncology development program. The Company has launched a Phase I clinical trial
of NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors in locoregional recurrent (LRR) or
recurrent and metastatic (R/M) HNSCC amenable to re-irradiation of the HN and lung or liver metastases (mets) from any
primary cancer eligible for anti-PD-1 therapy.
Other ongoing NBTXR3 trials are treating patients with hepatocellular carcinoma (HCC) or liver metastases, locally
advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with
concurrent chemotherapy, and pancreatic cancer. The Company has a broad, comprehensive clinical research collaboration
with The University of Texas MD Anderson Cancer Center (initially expected to support 9 new clinical trials in the United
States) to evaluate NBTXR3 across several cancer types.
About NANOBIOTIX: www.nanobiotix.com
Incorporated in 2003, Nanobiotix is a leading, clinical-stage nanomedicine company pioneering new approaches to
significantly change patient outcomes by bringing nanophysics to the heart of the cell.
The Nanobiotix philosophy is rooted in designing pioneering, physical-based approaches to bring highly effective and
generalized solutions to address unmet medical needs and challenges.
Nanobiotix’s first-in-class, proprietary lead technology, NBTXR3, aims to expand radiotherapy benefits for millions of cancer
patients. Nanobiotix’s Immuno-Oncology program has the potential to bring a new dimension to cancer immunotherapies.
Nanobiotix is listed on the regulated market of Euronext in Paris (Euronext: NANO / ISIN: FR0011341205; Bloomberg:
NANO: FP). The Company’s headquarters are in Paris, France, with a US affiliate in Cambridge, MA, and European affiliates
in France, Spain and Germany.