Nanobiotix(US:NBTX)2023-04-24 21:15Financials and Expenditures - Capital expenditures for 2020, 2021, and 2022 were €0.08 million, €1.6 million, and €0.5 million respectively, with expectations for increased expenditures in 2023[311]. - The company plans to expand its manufacturing capabilities in 2024 to support clinical trials and initial commercialization of NBTXR3[488]. - The company has not yet established commercial infrastructure in the US or EU and is defining its commercialization strategy[489]. Clinical Trials and Studies - NBTXR3 demonstrated a statistically significant improvement in pathological complete response, with approximately twice as many patients achieving this outcome compared to those receiving radiotherapy alone[321]. - The median overall survival for patients in the Phase 1 trial of NBTXR3 was reported at 23 months, showing continued improvement in outcomes[322]. - The NANORAY-312 Phase 3 trial aims to enroll approximately 500 patients, focusing on elderly patients with locally advanced head and neck squamous cell carcinoma[323]. - NBTXR3 has been administered to over 300 patients as of December 31, 2022, indicating significant clinical engagement[328]. - Nanobiotix is conducting Study 1100, a Phase 1 multi-cohort trial of RT-activated NBTXR3 followed by anti-PD-1 checkpoint inhibitors in patients with recurrent or metastatic head and neck squamous cell carcinoma[334]. - The company is conducting a Phase 2/3 trial (Act.In.Sarc) to measure the anti-tumor activity of NBTXR3 activated by radiotherapy in patients with locally advanced STS[368]. - The NANORAY-312 trial includes stratification factors such as investigator's choice of cetuximab addition, HPV status, and age-adjusted Charlson Comorbidity Index[389]. - The study aims to evaluate safety and tolerability of NBTXR3, with secondary endpoints including overall response rate, local progression, and quality of life[394][387]. - The ongoing Phase 1 trial (Study 1100) aims to evaluate the safety and efficacy of RT-activated NBTXR3 followed by anti-PD-1 checkpoint inhibitors in approximately 141 evaluable patients[434]. - The ongoing dose expansion part of the study is evaluating RT-activated NBTXR3 plus anti-PD-1 in patients with LRR or R/M HNSCC[446]. Mechanism of Action and Technology - NBTXR3 is designed to enhance the efficacy of radiotherapy by increasing localized tumor cell death without harming surrounding healthy tissues[314]. - NBTXR3 nanoparticles are designed to enhance the efficacy of radiotherapy by increasing the absorption of radiation within malignant tumors without causing additional damage to surrounding healthy tissues[344]. - The technology utilizes nanoparticles with a high electron density core of crystallized hafnium oxide, which allows for greater energy absorption during radiotherapy[345]. - The company believes that NBTXR3 could improve systemic treatment outcomes by enhancing the effectiveness of immune checkpoint inhibitors in patients[332]. - Preclinical data indicated that RT-activated NBTXR3 may enhance the immune response, potentially converting "cold" tumors into "hot" tumors[423]. Market Potential and Collaborations - Approximately 60% of cancer patients in developed countries receive radiotherapy, highlighting a substantial market opportunity for NBTXR3[315]. - The company aims to build a treatment franchise for head and neck cancer, where 74% of patients are expected to receive radiotherapy, representing a significant market potential[319]. - The company has established a collaboration with LianBio to develop and commercialize NBTXR3 in key Asian markets, including Mainland China, Taiwan, and South Korea[328]. - The company has entered into a collaboration with MD Anderson to support multiple clinical trials involving approximately 312 patients[361]. - The company has terminated its collaboration with PharmaEngine, regaining all rights to NBTXR3 in the Asia-Pacific region[472]. Regulatory Designations and Approvals - NBTXR3 received Fast Track designation from the FDA in February 2020 for the treatment of locally advanced head and neck cancers[333]. - The company received Fast Track designation from the FDA for NBTXR3 for treating locally advanced head and neck cancers not eligible for platinum-based chemotherapy[391]. - NBTXR3 has received CE mark approval for commercialization in the EU for the treatment of locally advanced STS of the extremities and trunk wall under the brand name Hensify[376]. Patient Outcomes and Efficacy - NBTXR3 nanoparticles demonstrated a pathological complete response rate of 16.1% in the treatment arm compared to 7.9% in the control arm, achieving statistical significance with a p-value of 0.0448[370]. - In patients with higher histology grades, the pathological complete response was achieved in 17.1% of the NBTXR3 arm versus 3.9% in the control arm[371]. - The rate of tumor surgery with R0 margins was 77% in the NBTXR3 arm compared to 64% in the control arm, with a p-value of 0.0424 indicating statistical significance[373]. - The five-year survival rate for patients with locally advanced head and neck cancers is estimated at 68%[378]. - Approximately 54,000 patients were diagnosed with oral or oropharyngeal cancer in the United States in 2022, with an estimated 11,230 deaths from the disease[378]. - Among evaluable patients with oropharyngeal head and neck cancer, the objective response rate was 100%, with 66.7% achieving complete response and 33.3% partial response[405]. - The median overall survival (mOS) for evaluable patients was 18.1 months, while the median progression-free survival (mPFS) was 10.6 months[406]. - In the "all patients treated" population, the mOS was 14.1 months, with 63% of patients having a modified Charlson Comorbidity Index (mCCI) of four or more, indicating a higher risk of early death[408]. - As of February 22, 2022, the mOS improved to 17.9 months in the all treated population and 23.0 months in evaluable patients[411]. Intellectual Property and Patents - The company holds over 450 issued or pending patents across 23 patent families related to NBTXR3 technology and nanomedicine applications[498]. - The company holds 73 patent families related to its technologies, with expiration dates ranging from 2025 to 2041[499]. - The company relies on trade secrets to protect proprietary technologies and maintains confidentiality agreements with employees and consultants[507]. - The patent family for NBTXR3 includes applications for its use in immuno-oncology and in combination with other checkpoint inhibitors[505]. - The company has patent applications pending in multiple countries, which may affect the expiration dates of certain patents[502]. - The company has a diverse geographical patent coverage, including countries such as the United States, Canada, Australia, and various European nations[499][500].