Dive Brief:
- Nanobiotix shares shot up over 50% on the Paris stock exchange Friday after its experimental drug NBTXR3 outperformed standard-of-care (radiation therapy alone) in soft tissue sarcoma, meeting both its primary and secondary endpoint. NBTXR3 is activated at the tumor site using radiotherapy.
- In the Phase 2/3 act.in.sarc trial, twice as many patients met the primary endpoint (pathological complete response) compared with radiotherapy alone.
- The secondary endpoint was cancer cell-free surgical margins, and significantly more NBTXR3-treated patients showed these compared with radiotherapy alone. Cancer-free margins are a surrogate endpoint for long-term and systemic benefits for patients. The results will be presented at upcoming conferences.
Dive Insight:
Nanibiotix's NBTXR3 has a dual mode of attack, with both physical destruction of tumor cells and immune system activation for local and systemic control. The market applauded the results Friday, although shares were down by more than 5% Monday.
"Data … show without any doubt an improvement of radiation therapy impact with a significant number of complete response. NBTXR3 can bring real benefit to patients and it can change the standard of care. This innovation will play a role in many other indications and particularly where radiotherapy is used alone," said Sylvie Bonvalot, head of Sarcoma and Complex Tumor Surgery Unit at Institut Curie, Paris, France and global principal investigator of the Phase 2/3 study, in a statement.
Nanobiotix has Food and Drug Administration approval to run a clinical trial of NBTXR3 in combination with anti-PD1 antibodies in non-small cell lung cancer and in head and neck cancer. Other ongoing studies include liver cancers, prostate cancer, and rectal cancer and head and neck cancer in combination with chemotherapy. NBTXR3 is in seven clinical trials in total and is pending marketing approval in Europe for soft tissue sarcoma.
In 2018 roughly 13,000 new soft tissue sarcomas are expected to be diagnosed in the U.S. and about 5,000 people will die of this form of cancer. There are more than 50 subtypes of an already rare cancer and limited treatment options, making it a challenging field with significant unmet need.
Eli Lilly’s Lartruvo (olaratumab), given the thumbs up by the FDA in October 2016, was the first new front-line drug for soft tissue sarcoma in 40 years.
Immunotherapeutic approaches are being studied in this area, with Memorial Sloan Kettering Cancer Center looking into the checkpoint inhibitors Yervoy (ipilimumab), Opdivo (nivolumab), and Keytruda (pembrolizumab). Other approaches use traditional chemotherapy agents but delivered differently. The Liposome Company is developing TLC178, a NanoX formulation of vinorelbine, for the treatment of rhabdomyosarcoma, a rare form of soft tissue sarcoma most commonly seen in children. This is in Phase 1/2