On July 9, 2019 NANOBIOTIX (Euronext: NANO – ISIN: FR0011341205), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported that the previously reported results from the Phase II/III Act.In.Sarc trial evaluating NBTXR3 in patients with advanced STS have been published online in The Lancet Oncology (Press release, Nanobiotix, JUL 9, 2019, View Source [SID1234537453]).

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The registration study met its primary (pCR) and secondary (R0 rate) endpoints. The increased proportion of patients with pCR among those given NBTXR3 as a single injection prior to standard of care RT when compared to RT alone (approximately twice as many), provides robust justification for the efficacy of nanoparticle-enhanced tumor cell death. The overall safety profile of NBTXR3 activated by RT was similar to RT alone, with manageable and reversible transient immune reactions observed in those treated with NBTXR3 and RT. More detail on the results can be found here.

The data from the Act.In Sarc trial were the basis for first European market approval (CE marking) of NBTXR3 in advanced STS of the extremity and chest wall, under the brand name Hensify. With STS results validating the efficacy and safety profile of the product, the company will continue its development strategy to evaluate NBTXR3 in multiple global (US, EU and APAC) clinical trials with a focus including but not limited to: head and neck, liver, lung and prostate cancer, as well as tumors that may benefit from NBTXR3 in combination with immune-oncology agents.

About Phase II/III Act.In.Sarc Study

Nanobiotix and its partner, PharmaEngine treated 179 patients in 32 sites across 11 countries in Europe and Asia. The Global Principal Investigator is Pr. Sylvie Bonvalot, MD, PhD (Institut Curie, Paris, France).

Primary Endpoint
Pathological Complete Response Rate (pCR): A pathological complete response is defined as the presence of less than 5% of residual malignant viable cells in the surgically removed tissue. The primary endpoint compared the proportion of patients with pCR between the two randomized arms. This was determined by an independent pathological central review according to Wardelmann et al., 2016.

Key Secondary Endpoint
Resection Margin Status: The resection margin status is evaluating the quality of surgery. Surgery remains the mainstay of care for advanced soft tissue sarcoma. The primary surgical objective is the complete removal of the tumor with negative resection margins (R0). Several retrospective studies suggest that surgical margin status predicts the risk of local and distant recurrence. In particular, negative surgical margins are significantly correlated to increased patients’ survival.

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 standards of radiation care. The physical mode of action of NBTXR3
makes it applicable across solid tumors such as lung, prostate, liver, glioblastoma, and breast cancers.

NBTXR3 is actively being evaluated in head and neck cancer with locally advanced squamous cell carcinoma of the oral cavity or
oropharynx in elderly and frail patients unable to receive chemotherapy or cetuximab with very limited therapeutic options.
Promising results have been observed in the phase I/II trial regarding the local control of the tumors. In the United States,
based on the discussions with the Food and Drug Administration that occurred in the first half of 2019, the Company plans to
begin the clinical trial authorization process in the second half of 2019 and commence a phase II/III clinical trial in locally
advanced head and neck cancers.

Nanobiotix is also running an Immuno-Oncology development program. In the United States, the Company received approval
from the Food and Drug Administration to launch a clinical trial of NBTXR3 activated by radiotherapy in combination with antiPD-1 antibodies in lung, and head and neck cancer patients (head and neck squamous cell carcinoma and non-small cell lung
cancer).

The other ongoing NBTXR3 trials are treating patients with liver cancers (hepatocellular carcinoma and liver metastasis), locally
advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with
concurrent chemotherapy, and prostate adenocarcinoma.
Further, the company has a large-scale, comprehensive clinical research collaboration with The University of Texas MD
Anderson Cancer Center (9 new phase I/II clinical trials in the United States) to evaluate NBTXR3 across head and neck,
pancreatic, thoracic, lung, gastrointestinal and genitourinary cancers.