On March 30, 2022 NANOBIOTIX (Euronext: NANO – NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported an update on operational progress and reported financial results1 for the year ended December 31, 2021 (Press release, Nanobiotix, MAR 30, 2022, View Source [SID1234611204]).
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"2021 was a year of acceleration for Nanobiotix as we took important steps towards validating of the broad potential therapeutic benefit of NBTXR3 and reinforced our team." said Laurent Levy, co-founder and chairman of the executive board of Nanobiotix. "Our clinical program evaluating first-in-class radioenhancer, NBTXR3, yielded compelling new data suggesting that our lead therapeutic candidate may improve treatment outcomes for patients both as a single-agent activated by radiotherapy for local diseases and as a combination therapy with anti-PD-1 for systemic disease. We also achieved the launch of our pivotal phase III study, NANORAY-312-a critical milestone as we advance toward global registration in head and neck cancer. 2022 has already seen the enrollment of our first European patients in NANORAY-312, and we expect the first patients in the U.S. and Asia this year. In parallel, we continue to make consistent progress in our Company-led immunotherapy pathway, along with the additional studies evaluating NBTXR3 across tumor indications and therapeutic combinations led by strategic collaborators. We are steadfast in our commitment to bring the potential benefits of NBTXR3 to millions of patients around the world, and in 2022 we will focus on executing our ongoing studies, defining our registration strategy in immunotherapy, and generating more evidence to support the broad applicability of our technology."
1 Financial data taken from the Company’s consolidated financial statements for the fiscal year ending December 31, 2021, which were approved by its executive board and reviewed by its supervisory board on March 30, 2022. The statutory auditors of the Company have completed their audit work on the 2021 financial statements, but have not issued their audit report yet. Such reports should be publicly available in the Company’s 2021 universal registration document and 2021 annual report on Form 20-F.
2021 Operational Highlights, Pipeline Status and Upcoming Milestones
Priority Pathway in Head & Neck Cancer, Local Control as Single Agent Activated by Radiotherapy
Data from Expansion Study 102, a phase I study evaluating NBTXR3 as a single agent activated by radiotherapy (RT) in high-risk, elderly locally advanced head and neck squamous cell carcinoma (LAHNSCC) patients ineligible for cetuximab and intolerant to cisplatin, was presented at the 2021 Annual Meeting of the American Society for Radiation Oncology (ASTRO 21) continued to support NBTXR3 administration as feasible and well-tolerated. At a median follow up of 9.5 months, evaluable patients (n=41) demonstrated a best observed target lesion objective response rate (ORR) of 85.4% and a best observed target lesion complete response rate (CRR) of 63.4%.
A recent review of data from Expansion Study 102 shows, as of February 22, 2022, an on-going median overall survival (mOS) of 17.9 months in the all treated population (n=56) and 23.0 months in evaluable patients (n=44) demonstrating continued improvement relative to the analysis presented at ASTRO 21 and consistent with data reported from the dose escalation phase of Study 102.
Expansion Study 102 is now fully enrolled with 44 evaluable patients. The last treatment visit for the final patient is expected in Q2 2022 and data reflecting one year of follow-up are expected in mid-2023
NANORAY-312, a pivotal phase III global registration study evaluating NBTXR3 as a single-agent activated by RT for elderly patients with LA-HNSCC intolerant to cisplatin initiated in Q1 2022 and actively enrolling patients across multiple European sites with U.S. site activation anticipated in mid-2022.
Priority Pathway in Immunotherapy for Advanced Cancers, Priming Immune Response in Combination with Anti-PD-1 Treatment:
Preclinical data was presented at the 2021 Annual Meeting of the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) showing RT-activated NBTXR3 increased CD8+ T cell infiltration and modulated the T cell receptor repertoire, suggesting stronger immune priming triggered by the therapy compared to radiotherapy alone.
Updated data from Study 1100, a phase I basket study evaluating NBTXR3 activated by RT in combination with nivolumab or pembrolizumab in locoregional recurrent or recurrent metastatic HNSCC (LRR or R/RM HNSCC), lung and/or liver metastasis from any primary tumor presented at ASTRO 21 demonstrated a disease control rate of 81% (n=16) in the evaluable patient population, including 73% in patients with prior primary or secondary resistance to anti-PD-1. In the evaluable population, 3 complete responses and 5 partial responses were reported. Some delayed tumor responses and/or abscopal effects were reported, suggesting NBTXR3 may potentially prime an immune response.
Reporting of recommended Phase II dose for each cohort is expected in 2022 along with the initiation of a new expansion Phase of Study 1100 evaluating NBTXR3 in combination with nivolumab or pembrolizumab in a cohort of patients LRR or R/M HNSCC resistant to prior anti-PD-(L)-1 therapy, a cohort of LRR or R/M HNSCC patients naïve to anti-PD-(L)-1 therapy, and cohort of patients resistant to prior anti-PD-(L)-1 therapy with lung, liver or soft tissue metastasis from select solid tumors.
Expect to provide updated Study 1100 data at a medical conference during the fourth quarter of 2022
Expanding NBTXR3 Opportunity, Collaborating with World-Class Partners to Validate Tumor-Agnostic, Combination-Agnostic Therapeutic Profile:
Formed strategic partnership with LianBio to develop and commercialize NBTXR3 across tumor types and therapeutic combinations in China and other Asian markets. LianBio is expected to begin enrolling patients in NANORAY-312 in China in H2 2022 as the first of up to five registrational studies covered by the collaboration.
Presented preclinical data, developed in collaboration with The University of Texas MD Anderson Cancer Center (MD Anderson), at SITC (Free SITC Whitepaper) 21 suggesting the combination of RT-activated NBTXR3, antiPD-1, anti-LAG3, and anti-TIGIT significantly elevated the activities of anti-tumor immune response in both irradiated and unirradiated tumors, improving local and distant tumor control and increasing survival rate.
Published data from a preclinical study conducted in collaboration between MD Anderson in the International Journal of Radiation Oncology, Biology, Physics (Red Journal) supportive of the hypothesis that RT-activated NBTXR3 in combination with anti-PD-1 could effectively control primary and metastatic tumors, evoke abscopal effect, and reduce the possibility of developing distant lung metastases.
Initiated fifth collaborator-led clinical study at MD Anderson Cancer Center.
Published peer-reviewed clinical case study reported preliminary data on the first-in-human administration of NBTXR3 for the treatment of pancreatic cancer not eligible for surgery, demonstrating feasibility with no treatment-related toxicity.
Determination of recommended phase II dose for NBTXR3 in pancreatic cancer expected in 2022.
Full Year 2021 Financial Results
Cash and Cash Equivalents: As of December 31, 2021, Nanobiotix had €83.9 million in cash and cash equivalents, compared to €119.2 million as of December 31, 2020. This net decrease of €35.3 million primarily reflects €51.8 million of net cash flows used in operating, investing and financing activities of Nanobiotix which was partially offset by the €16.5 million ($20.0 million) upfront payment associated with the LianBio collaboration announced in May 2021. As previously announced, PharmaEngine was eligible for and received a €2.1 million ($2.5 million) payment following the announcement of the LianBio collaboration and has received €3.4 million ($4.0 million) in conjunction with the completion of various administrative steps in connection with the winding-up of the collaboration. PharmaEngine will be eligible and is expected to receive in 2022 an additional $1.0 million in administrative fees and, upon a second regulatory approval of an NBTXR3-containing product, a final payment of $5.0 million.
Based on the current operating plan and financial projections, Nanobiotix anticipates that the cash and cash equivalents of €83.9 million as of December 31, 2021, will fund its operations into the second quarter of 2023.
Revenue: Full year 2021 revenue totaled €9.7k compared to €50.0k for the year ended December 31, 2020. The revenue generated in 2021 was primarily related to the Company’s now concluded collaboration with PharmaEngine, Inc. Other income includes research tax credits which increased from €1.9 million in 2020 to €2.5 million in 2021 due mainly to an increase of research and development expenses.
Research and Development ("R&D") Expenses: R&D expenses consist primarily of preclinical, clinical, and manufacturing expenses related to the development of NBTXR3 and totaled €30.4 million for the twelve-month period ended December 31, 2021, as compared to €24.3 million for the twelve months ended December 31, 2020. The increase in net R&D expenses was primarily due to increases in development costs related to the Company’s priority pathways, including preparation and initiation of its pivotal Phase III registration study, NANORAY-312, continuation of Expansion Study 102 and its ongoing immunotherapy combination Study 1100 as well as increases in personnel related expenses, including share-based compensation.
Selling, General and Administrative ("SG&A") Expenses: SG&A expenses consist primarily of administrative employee-related expenses, legal and other professional fees, patent filing and maintenance fees, and insurance. These expenses were €19.4 million year-ended December 31, 2021, as compared to €14.6 million for twelve-months ended December 31, 2020. The increase in G&A expenses year-over-year was due to increased headcount, consulting fees, legal & compliance expenses resulting from the Nasdaq listing and recruitment expenses.
Net loss: Net loss attributable to shareholders was €47.0 million, or €1.35 per share, for the twelve-month period ended December 31, 2021. This compares to a net loss of €33.6 million, or €1.38 per share for the year ended December 31, 2020.
Conference Call and Webcast
Nanobiotix will host a conference call and live audio webcast on Thursday, March 31, 2022, at 8:00 AM EDT / 2:00 PM CET, prior to the open of the U.S. market. During the call, Laurent Levy, chief executive officer, and Bart Van Rhijn, chief financial officer, will briefly review the Company’s fourth quarter results, year-end results, and an update on business activities before taking questions from analysts and investors. Investors are invited to email their questions in advance to [email protected]
A live webcast of the call may be accessed by visiting the investors section of the company’s website at www.nanobiotix.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the company’s website.
About NBTXR3
NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.
NBTXR3 is being evaluated in locally advanced head and neck squamous cell carcinoma (HNSCC) as the primary development pathway. The company-sponsored phase I dose escalation and dose expansion study has produced favorable safety data and early signs of efficacy; and a phase III global registrational study initiated patient enrollment in 2022. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the planned phase III study.
Nanobiotix has also prioritized an Immuno-Oncology development program—beginning with a Company sponsored phase I dose escalation and dose expansion study evaluating NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors for patients with locoregional recurrent or recurrent/ metastatic HNSCC and lung or liver metastases from any primary cancer eligible for anti-PD-1 therapy.
Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a strategic collaboration strategy with world class partners to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several phase I and phase II studies to evaluate NBTXR3 across tumor types and therapeutic combinations.