First European Phase 2/3 trial approval for Nanobiotix in soft tissue sarcoma

Nanobiotix has received approval from the French National Security Agency for Medicines and Health Products (ANSM) to start its Phase 2/3 registration trial of its lead product, NBTXR3, in patients with locally advanced Soft Tissue Sarcoma (STS). NBTXR3 is a selective radioenhancer. The injected nanoparticles penetrate tumor cells and when exposed to radiotherapy make feasible the deposition of a high energy dose within the cancer cell, increasing cell killing and thus improving resectability and disease outcome.

NBTXR3 has the potential to provide a significant clinical benefit by improving radiotherapy efficacy, destroying locally advanced tumors more efficiently and to improve the quality of surgery enabling a more complete removal of the tumor. This international multi-center pivotal study will start in France and then be conducted in around 25-30 sites throughout Europe once further authorizations are received. Patients in the experimental treatment arm (Arm A) will receive an intratumoral injection of NBTXR3 followed by radiotherapy and surgery. Patients in the control arm (Arm B) will be treated with radiotherapy alone followed by surgery only. It is expected to enroll approximately 180 patients to have 78 evaluable patients in each arm. The primary objective of this study is to increase the pathological complete response rate (pCR) of intratumoral injection of NBTXR3 activated by external beam radiation therapy (EBRT), versus EBRT alone in patients with locally advanced STS of the extremity and trunk wall. Secondary objectives are to assess the safety profile of NBTXR3 activated by radiotherapy (incidence of early and late adverse events), to compare the objective response rate (ORR), tumor volume changes after NBTXR3 and carcinological resection rates, and to evaluate limb amputation rates. This pivotal trial is expected to be the final step before registration in Europe (CE mark) and should be –completed towards the end of 2016. In the Asia-Pacific region, as announced recently, PharmaEngine intends to participate in this pivotal study by opening clinical sites in several countries. In parallel, Nanobiotix is establishing industrialization, market access and reimbursement strategies for NBTXR3 to prepare for a potential product launch at the end of 2016.