On May 19, 2021 Guardant Health, Inc. (Nasdaq: GH), reported to present data demonstrating the use of the company’s proprietary blood tests to advance precision oncology, including cancer screening, detecting residual or recurrent disease in early-stage cancer, and treatment selection and treatment response monitoring in advanced cancer (Press release, Guardant Health, MAY 19, 2021, View Source;and-Late-Stage-Cancers [SID1234580319]). Data, from 20 abstracts, will be presented along with other leading medical institutions and pharmaceutical companies, during the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held from June 4-8, 2021.

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"The data at ASCO (Free ASCO Whitepaper) not only demonstrate the advantages of blood-based comprehensive genomic profiling in advanced cancer, but also the growing body of evidence showing the power of our blood tests across the continuum of care. We are pleased to share data that not only informs treatment decisions, but that also shows value in assessing treatment response, screening for early-stage colorectal cancer in asymptomatic patients, and detecting residual or recurrent disease in early-stage cancers, including colorectal, non-small cell lung, and bladder," said Helmy Eltoukhy, Guardant Health CEO. "The data also illustrates the value of our liquid biopsy tests and our real-world clinical-genomic platform to gain new insights which help accelerate research and development of the next generation of cancer therapeutics."

LUNAR-2 Blood Test Detects Early-Stage Colorectal Cancer (CRC) with High Accuracy in Largest Cohort to Date

The data (N=705) show that the LUNAR-2 assay achieved overall sensitivity of 91% in early-stage CRC (stage I, II, and III), and specificity of 94%. The performance in this new large cohort of CRC cases, and cancer-free controls, is consistent with previously reported data.1-2 Notably no differences in sensitivity for CRC detection were observed in patients presenting with asymptomatic disease, compared to those patients who were symptomatic, despite the lower cell-free DNA (cfDNA) tumor fractions observed in asymptomatic patients, suggesting the test will have clinically meaningful performance in an average-risk screening population (Abstract 3536).

Guardant Reveal Blood Test Detects ctDNA in Early-Stage Non-Small Cell Lung and Bladder Cancers, and Predicts Recurrence in Oligometastatic CRC in Post-Surgical Setting

The data show that Guardant Reveal, a first of its kind, blood-only minimal residual disease (MRD) test for solid tumors, detects circulating tumor DNA (ctDNA) in pre-treatment, early-stage non-small cell lung cancer and bladder cancer, without the need for tumor tissue, and with comparable sensitivity to tissue-dependent approaches (Abstract 3045).

Additionally, in a study (Abstract 3565) with the University of California San Francisco (UCSF) and Massachusetts General Hospital, the Guardant Reveal test detected MRD in patients with oligometastatic colorectal cancer undergoing curative intent surgery or radiotherapy. Detection of ctDNA post-procedure had a high positive predictive value (PPV) for cancer recurrence, with a median lead time of six months compared to surveillance imaging.

Guardant Health will also provide an update on the COBRA interventional trial (NRG-G1005: Circulating Tumor DNA as a Predictive Biomarker in Adjuvant Chemotherapy in Stage II Colon Cancer) (Abstracts TPS3622, TPS148).

The Guardant Reveal test is commercially available for the detection of residual and recurrent disease in early-stage colorectal cancer. The test identifies patients with residual disease who may benefit most from adjuvant therapy, and has been shown to detect recurrence months earlier than current standard-of-care methods like carcinoembryonic antigen (CEA) tests or imaging.3-8 The Guardant Reveal test achieves industry-leading sensitivity (91%)9 for detecting ctDNA by simultaneously interrogating both genomic alterations and methylation. Test results are obtained from a simple blood draw and returned in as little as seven days without the need for a tissue biopsy.

Guardant360 Blood Tests Demonstrate Clinical Utility in the Treatment and Management of Locally Advanced and Metastatic Cancers

Fifteen abstracts, now online, highlight the use of the Guardant360 test to detect clinically actionable mutations and to inform treatment options for patients. Key findings include data showing the ability of Guardant360 liquid biopsy to robustly detect tumor mutational burden across a wide range of solid tumors (Abstract 3040) and to predict clinical outcomes in a prospectively collected cohort of non-small cell lung cancer patients treated with targeted therapy (Abstract 9027). In addition, several abstracts highlight the ability of the Guardant360 test to predict molecular response in patients across a variety of cancer types and therapy classes (Abstract 4130, Abstract 9011).

GuardantINFORM Real-World Evidence Platform Used to Identify Unmet Need for Patients with Lung Adenocarcinomas Harboring STK11 and KRAS G12C Mutations Treated with Checkpoint Inhibitors

A study in collaboration with faculty from Massachusetts General Hospital and Mirati Therapeutics, Inc. (NASDAQ: MRTX) using clinical-genomic information from the GuardantINFORM platform demonstrated that patients with metastatic lung adenocarcinoma and co-occurring STK11 and KRAS G12C mutations had significantly worse outcomes to treatment with first-line checkpoint inhibitor-based treatment regimens (including combination with platinum-based chemotherapy), highlighting the need for effective targeted and/or combination therapies to address this patient population (Abstract 9106).

The GuardantINFORM clinical-genomic platform is intended to help accelerate research and development of the next generation of cancer therapeutics by offering biopharma partners an in-silico platform that combines de-identified longitudinal clinical information and genomic data collected from the Guardant360 liquid biopsy test. With data from more than 160,000 patients diagnosed with locally advanced and metastatic cancers, this robust dataset offers real-world insights into anti-cancer therapy use in the clinic, tumor evolution, and treatment resistance throughout each patient’s treatment journey for many advanced solid tumor cancers, including non-small cell lung, breast, colon, and prostate.

Data at ASCO (Free ASCO Whitepaper)

Abstract#

Title

LUNAR-2

Abstract No. 3536

Multimodal circulating tumor DNA (ctDNA) colorectal neoplasia detection assay for asymptomatic and early-stage colorectal cancer (CRC)

Guardant Reveal

Abstract No. 3045

Multiomic, plasma-only ctDNA NGS assay for minimal residual disease (MRD) detection in solid tumors.

Abstract No. 3565

Circulating tumor derived cell-free DNA (ctDNA) to predict recurrence of metastatic colorectal cancer (mCRC) following curative intent surgery or radiation.

Abstract No. TPS3622, TPS148

Phase II/III study of circulating tumor DNA as a predictive biomarker in adjuvant chemotherapy in patients with stage II colon cancer: NRG-G1005 (COBRA).

Guardant 360

Abstract No. 3040

Blood-based tumor mutational burden from circulating tumor DNA (ctDNA) across advanced solid malignancies using a commercially available liquid biopsy assay.

Abstract No. 9027

Clinical outcomes for plasma-based comprehensive genomic profiling versus tissue testing in advanced lung adenocarcinoma.

Abstract No. 4130

Circulating cell free tumor DNA detection as a prognostic tool in advanced pancreatic cancer.

Abstract No. 9011

Early circulating tumor DNA dynamics and efficacy of lorlatinib: Analysis from the CROWN study.

Abstract No. 1028

Identification of pathogenic CDK12 alterations in cell-free DNA (cfDNA) from patients with breast cancer.

Abstract No. 8577

Cell-free circulating tumor DNA (cfDNA) analysis of advanced thymic epithelial tumors (TETs).

Abstract No. 4585

Circulating tumor DNA (ctDNA) in patients with advanced adrenocortical carcinoma.

Abstract No. 9108

Genomic landscape differences in patients with advanced non-small cell lung cancer by sex and age.

Abstract No. 3523

Circulating tumor DNA-based genomic profiling of small bowel

adenocarcinoma.

Abstract No. e17018

Serial ctDNA profiling in patients with metastatic prostate cancer undergoing treatment with Radium-223.

Abstract No.

3572

Serial circulating tumor DNA (ctDNA) monitoring in metastatic colorectal cancer (mCRC) reveals dynamic profile of actionable alterations.

Abstract No. 5038

Complementary detection of genomic alterations in metastatic castration‑resistant prostate cancer (mCRPC) from CheckMate 9KD through analyses of tumor tissue and plasma DNA.

Abstract No. 3589

Assessment of HER2 (ERBB2) amplification (HER2amp) using blood-based circulating tumor DNA (ctDNA) next generation sequencing (NGS) and correlation with tissue-based testing in metastatic colorectal cancer (mCRC)

Abstract No. 4058

Early predictors of benefit to dual anti-PD1/HER2 inhibition: Biomarker analysis from phase 2 trial of pembrolizumab/trastuzumab in HER2-positive metastatic esophagogastric (mEG) cancer.

Abstract No. 3555

Pertuzumab plus trastuzumab and real-world standard of care (SOC) for patients (pts) with treatment refractory metastatic colorectal cancer (mCRC) with HER2 (ERBB2) amplification (amp) confirmed by tumor tissue or ctDNA analysis.

GuardantINFORM

Abstract No. 9106

Impact of STK11 mutation on first-line immune checkpoint inhibitor outcomes in a real-world KRAS G12C mutant lung adenocarcinoma cohort.

On May 19, 2021 Bicara Therapeutics, a clinical-stage biotechnology company developing dual-action biologics designed to spur a potent and durable immune response in the tumor microenvironment, reported that a digital presentation with clinical data from its ongoing Phase 1/2 study of BCA101, a bifunctional antibody designed to target the TGFβ trap to EGFR+ tumors, will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting, to be held virtually from June 4-8, 2021 (Press release, Bicara Therapeutics, MAY 19, 2021, View Source [SID1234580318]).

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"We’re excited to share clinical data on our lead therapeutic candidate BCA101, a first-in-class antibody with strong potential to achieve superior anti-tumor efficacy by promoting immune response directly at the tumor site," said Liviu Niculescu, M.D., Chief Medical Officer of Bicara Therapeutics. "Data from the ongoing Phase 1/2 clinical trial of BCA101 support our biologic rationale and point to the potential to provide treatment options for patients who have not benefitted from other approaches within the standard of care."

Details of the digital presentation are as follows:

Abstract: 3074
Title: First-in-human phase I study of the bifunctional EGFR/TGFβ fusion protein BCA101 in patients with EGFR-driven advanced solid cancers
Session: Developmental Therapeutics – Molecularly Targeted Agents and Tumor Biology
Lead Authors: Filip Janku, M.D. and Philippe Bedard, M.D.

Key findings:

BCA101 demonstrates target engagement at the tumor site and decreases SMAD2 phosphorylation as a proximal PD endpoint.
A partial response has been observed in combination with pembrolizumab in a PD-(L)1 naïve subject.
BCA101 achieves dose-proportional PK and exhibits encouraging biomarkers in both plasma and tissue.
Dose escalation currently continues at the 1250mg dose level in single agent and 750mg dose level in combination with pembrolizumab.
The ASCO (Free ASCO Whitepaper) e-poster website will be launched on Friday, June 4, 2021, and will remain available for viewing through Tuesday, July 6, 2021. Bicara’s presentation will also be available on the Bicara website.

About BCA101

BCA101 is a first-in-class EGFR / TGFβ-trap bifunctional antibody designed to enhance both innate and adaptive immune responses directly at the site of the tumor by binding to the well-validated EGFR antigen and disabling TGFβ, a signaling molecule that plays a key role in suppressing the immune response in the tumor microenvironment. Promising preclinical data suggest that BCA101 is superior to the anti-EGFR antibody cetuximab in preventing tumor recurrence, as well as in restoring immune activation. An ongoing Phase 1/2 clinical trial of BCA101, initiated in July 2020, has dosed the first four cohorts of patients in a dose-escalation study with BCA101 as a single agent. A second arm of the study began enrolling patients for combination treatment with BCA101 and pembrolizumab, a PD-1 inhibitor, in January 2021. For more information, please visit www.clinicaltrials.gov.

On May 19, 2021 Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, reported that it will present the latest research from its early stage clinical development programs in two presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place virtually from June 4-8, 2021 (Press release, Seres Therapeutics, MAY 19, 2021, View Source [SID1234580317]). In an oral presentation, Seres will share data from its collaboration with the University of Cologne (Köln, Germany) assessing the association of the microbiome on clinical outcomes in allogeneic hematopoietic stem cell transplantation (HSCT) recipients. A separate poster presentation will include data from its collaboration with Memorial Sloan Kettering Cancer Center (New York, NY) evaluating the correlation of microbiome composition with response to immune checkpoint inhibitor treatment in patients who have metastatic lung, urothelial or renal cancer, or metastatic melanoma.

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"Research has shown that disruption in the gut microbiome can affect clinical outcomes in patients treated with certain cancer treatments, including allogeneic hematopoietic stem cell transplantation and cancer immunotherapy," said Matthew Henn, Ph.D., Chief Scientific Officer at Seres. "We are looking forward to presenting data that deepens this understanding and brings further attention to the opportunity of microbiome therapeutics to modulate patient immune responses to improve outcomes as well as to reduce subsequent antibiotic resistant bacterial infections that can lead to blood stream infections during the course of various cancer treatments."

Presentation details are as follows:

Oral Abstract Presentation: Prospective longitudinal evaluation of microbiome diversity in patients with hematological malignancy undergoing allogeneic hematopoietic stem cell transplantation (HSCT), June 4, 2021, 2:30 – 5:30 pm ET, lead co-authors: Emily Walsh, Anastasia Tsakmaklis (Abstract: 7005)
Poster Presentation: Assessment of cancer-specific microbiome signature of response to immune checkpoint inhibitors, June 4, 2021, 9:00 am ET, lead co-authors: Michal Sarfaty, Christopher A. Desjardins (Abstract: 2574)

On May 19, 2021 The Janssen Pharmaceutical Companies of Johnson & Johnson reported new data from the fixed-duration cohort of the investigational Phase 2 CAPTIVATE study (PCYC-1142), showing 95 percent of patients treated with fixed duration combined IMBRUVICA (ibrutinib) plus venetoclax were alive and progression-free at two years (Press release, Johnson & Johnson, MAY 19, 2021, View Source;Based-Combination-Regimen-as-a-Fixed-Duration-First-Line-Treatment-for-Chronic-Lymphocytic-Leukaemia-Demonstrates-High-Rates-of-Disease-Control [SID1234580316]).1 Deep remissions were seen across all subgroups, including patients with high risk chronic lymphocytic leukaemia (CLL).1 In addition, long-term data from the RESONATE-2 (PCYC-1115/1116) study will be presented, providing the longest follow-up Phase 3 data for any Bruton tyrosine kinase (BTK) inhibitor to date.2 These data reinforce the long-term survival benefits and well-established safety profile of single-agent ibrutinib for patients with CLL, a type of non-Hodgkin lymphoma and the most common form of leukaemia in adults.1,2

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Both studies will be presented during the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, June 4-8, and following at the 26th European Hematology Association (EHA) (Free EHA Whitepaper) Congress, June 9-17.

"Ibrutinib was the first BTK inhibitor approved in Europe and has been used to treat more than 230,000 patients worldwide. It is now also the first BTK inhibitor to be studied as a fixed-duration combination treatment option," said Edmond Chan, EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Ltd. "The latest data to be presented at ASCO (Free ASCO Whitepaper) reinforce the potential of ibrutinib as a foundational treatment option across the CLL landscape and add to the breadth of evidence regarding its efficacy and safety profile."

First Data from the Fixed-Duration Cohort of the Phase 2 CAPTIVATE (PCYC-1142) Study of Ibrutinib-Based Combination Regimen in Previously Untreated CLL Patients (Abstract #7501)1

The CAPTIVATE study evaluated previously untreated patients with CLL who were 70 years or younger, including patients with high-risk disease.1 In the fixed-duration cohort (N=159; median age 60 years), patients received three cycles of ibrutinib lead-in therapy, followed by 12 cycles of combination ibrutinib plus venetoclax therapy, and then stopped therapy regardless of minimal residual disease (MRD) status.1 More than 90 percent of patients completed planned therapy of ibrutinib plus venetoclax treatment.

At a median follow-up of 27.9 months, the complete response (CR) rate in the overall population was 56 percent (n=88; 95 percent Confidence Interval [CI]: 48–64) and was consistent across high-risk subgroups.1 Of the patients who achieved a CR, 89 percent had a durable CR of at least one year.1 Among the remaining 11 percent, one patient had progressive disease; the remaining patients with response follow-up of less than one year were not evaluable.1 The overall response rate (ORR) was 96 percent.1 Estimated 24-month progression-free survival (PFS) with ibrutinib plus venetoclax was 93 percent for patients with unmutated IGHV and 97 percent for patients with mutated IGHV (unmutated IGHV 95 percent CI: 85‒97; mutated IGHV 95 percent CI: 88‒99) and overall survival (OS) was 98 percent (95 percent CI: 94-99) for all treated patients.1 Seventy-seven and 60 percent of patients achieved undetectable minimal residual disease (uMRD) at any time in the peripheral blood and bone marrow, respectively.1

"The use of continuous treatment with ibrutinib in CLL has well been established as the standard of care for patients, including those with high-risk disease," said Paolo Ghia*, M.D., Ph.D., Professor of Medical Oncology, Università Vita-Salute San Raffaele, Italy and principal study investigator. "The latest data from the CAPTIVATE study underscore that ibrutinib in an all-oral fixed duration combination with venetoclax also delivers a high rate of progression-free survival at two years while enabling treatment-free remission for patients."

Of note, 94 percent of patients with high baseline tumour lysis syndrome (TLS) risk based on tumour burden shifted to medium- or low-risk after ibrutinib lead-in therapy, and no TLS events occurred.1 Adverse events (AEs) were primarily Grade 1/2.1 The most common Grade 3/4 AEs were neutropaenia (33 percent), infections (eight percent), hypertension (six percent), and neutrophil count decrease (five percent).1 Discontinuations due to AEs were infrequent (three percent for ibrutinib).1

Findings from the uMRD-guided cohort of the CAPTIVATE study were presented at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The phase 3 GLOW study (NCT03462719) is also evaluating fixed-duration ibrutinib plus venetoclax, with a comparison to chlorambucil plus obinutuzumab, for first-line treatment of younger unfit patients or elderly patients with CLL, regardless of fitness criteria. These studies are part of a comprehensive development programme exploring the potential of ibrutinib-based fixed-duration therapy.

Long-Term Data from the Phase 3 RESONATE-2 Study Demonstrate Efficacy and Safety of Single-Agent Ibrutinib in Previously Untreated CLL Patients (Abstract #7523)2

The RESONATE-2 study evaluated 269 previously untreated patients with CLL aged 65 years or older, without del(17p), who were randomly assigned to receive continuous ibrutinib or chlorambucil up to 12 cycles.2 With up to seven years of follow-up, PFS benefit with single-agent ibrutinib was sustained (PFS Hazard Ratio [HR] 0.160 [95 percent CI: 0.111–0.230]).2 At 6.5 years, the median PFS with ibrutinib has not been reached; 61 percent of patients treated with single-agent ibrutinib were alive and progression-free compared with nine percent of patients treated with chlorambucil.2 The PFS benefit for patients treated with ibrutinib was seen in all subgroups, including those with high-risk genomic features of TP53 mutation, unmutated IGHV or 11q deletion (HR 0.091 [95 percent CI: 0.054–0.152]).2 Additionally, 78 percent of patients in the ibrutinib treatment-arm were alive at 6.5 years. The CR rate with ibrutinib treatment has increased over time to 34 percent.2 Nearly half of patients continue to receive ibrutinib treatment with up to seven years of follow-up.2

Single-agent ibrutinib was well tolerated as a long-term treatment with no new safety signals.2 Ongoing rates of Grade 3 or higher AEs of interest remained low for hypertension (five-to-six-year interval: n=20; six-to-seven-year interval: n=15) and atrial fibrillation (five-to-six-year interval: n=7; six-to-seven-year interval: n=5).2 Additionally, no Grade 3 or higher major haemorrhage occurred in the five-to-seven-year interval.2 Any-grade AEs leading to discontinuations were seen in three percent (n=2) of patients from year five to year six and no patients discontinued treatment in the ibrutinib arm due to AEs from year six to year seven.2

"The positive results from the CAPTIVATE study demonstrate the potential of ibrutinib and venetoclax, with complementary mechanisms of action, to provide deep responses with a once-daily, fixed-duration combination that can be administered in the outpatient setting for younger, fit patients," said Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology, Janssen Research & Development, LLC. "The results from RESONATE-2 further support the long-term benefit of ibrutinib monotherapy in front line CLL for which the breadth and maturity of data continue to grow in support of this standard-of-care treatment and its impact on progression free and overall survival."

#ENDS#

About Ibrutinib

Ibrutinib is a once-daily, first-in-class Bruton’s tyrosine kinase (BTK) inhibitor that is administered orally, and is jointly developed and commercialised by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.3 Ibrutinib blocks the BTK protein; the BTK protein sends important signals that tell B cells to mature and produce antibodies. BTK signalling is needed by specific cancer cells to multiply and spread.4 By blocking BTK, ibrutinib may help move abnormal B cells out of their nourishing environments in the lymph nodes, bone marrow, and other organs.5

Ibrutinib was first approved by the European Commission (EC) in 2014, and approved indications to date include:3

Chronic lymphocytic leukaemia (CLL): As a single agent or in combination with rituximab or obinutuzumab for the treatment of adult patients with previously untreated CLL, and as a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy.
Mantle cell lymphoma (MCL): As a single agent for the treatment of adult patients with relapsed or refractory MCL.
Waldenström’s macroglobulinemia (WM): As a single agent for the treatment of adult patients who have received at least one prior therapy or in first-line treatment for patients unsuitable for chemo-immunotherapy, and in combination with rituximab for the treatment of adult patients.
Ibrutinib is approved in more than 100 countries, and, to date, has been used to treat more than 230,000 patients worldwide.6 Ibrutinib is the only BTKi that has demonstrated overall survival benefits in three CLL clinical trials, with response durability persisting up to 8 years,7,8,9 and more than seven out of ten patients alive and without disease progression after six and a half years.2 Ibrutinib is the only BTKi that has been shown to mediate short- and long-term immune restoration.10

Ibrutinib is the most comprehensively studied BTKi, with more than 150 active clinical trials in several blood cancers and other serious diseases. For a full list of side effects and information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics for further information.

About Chronic Lymphocytic Leukaemia

Chronic lymphocytic leukaemia (CLL) is typically a slow-growing blood cancer of the white blood cells.11 The overall incidence of CLL in Europe is approximately 4.92 cases per 100,000 persons per year and is about 1.5 times more common in men than in women.12 CLL is predominantly a disease of the elderly, with a median age of 72 years at diagnosis.13

The disease eventually progresses in the majority of patients, and they are faced with fewer treatment options with each relapse. Patients are often prescribed multiple lines of therapy as they relapse or become resistant to treatments.

On May 19, 2021 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 the upcoming presentation of updated results from the Company’s priority development pathways in head and neck cancer (head and neck squamous cell carcinoma; HNSCC) and in immunotherapy for advanced cancers at the 2021 Annual Meeting of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) (Press release, Nanobiotix, MAY 19, 2021, View Source [SID1234580315]). The Company will also present a poster with long-term safety analysis from its pivotal phase II/III study in soft tissue sarcoma.

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"The data we will present at ASCO (Free ASCO Whitepaper) provide further support for the paradigm-shifting potential of NBTXR3 as a foundational solid tumor-agnostic and combination-agnostic cancer therapeutic," said Laurent Levy, co-founder and chief executive officer of Nanobiotix. "We are excited to present both long-term safety data from our phase III soft tissue sarcoma study, along with updated safety and efficacy data from our second single-agent registration pathway in head and neck cancer. Moreover, we are particularly eager to present a growing body of data suggesting that after activation by radiotherapy, NBTXR3 may prime an immune response that could enhance the efficacy of immune checkpoint inhibitors as a first-line therapy, overcome resistance for refractory patients, and meaningfully expand the tumor types that respond to the class by transforming cold tumors into hot tumors."

Local Control as a Single-Agent for Patients with Head and Neck Cancer

Abstract #6051: Phase I Dose Expansion Study of Functionalized Hafnium Oxide Nanoparticles (NBTXR3) in Cisplatin-Ineligible Locally Advanced HNSCC Patients

The number of elderly patients diagnosed with locally advanced HNSCC (LA-HNSCC) is increasing. While concurrent chemoradiation is the non-surgical standard of care, not all patients can tolerate platinum-based chemotherapy (e.g., cisplatin). The Nanobiotix phase I dose expansion study in patients with LA-HNSCC (Study 102) is evaluating a single dose of NBTXR3 at 22% of baseline tumor volume (the recommended phase II dose; RP2D). Primary endpoints of the study are objective response rate (ORR) and complete response rate (CRR) of the primary tumor. Study 102 is expected to recruit a total of 44 evaluable patients. To date, 52 total patients have been injected with NBTXR3 in the study overall, of which 40 have been evaluable.

Updated data presented at ASCO (Free ASCO Whitepaper) further support NBTXR3 administration, followed by activation with radiotherapy, as feasible and well-tolerated. Six (6) serious adverse events (SAEs) related to NBTXR3 were observed across five (5) patients. A total of ten (10) deaths related to adverse events were reported. Four (4) deaths related to radiotherapy were observed, along with one (1) death from sepsis that was investigator-assessed as possibly related to NBTXR3, radiotherapy, and cancer.

At a median follow up of 8.1 months, evaluable patients demonstrated a high primary tumor ORR of 82.5% and a 62.5% CRR. These results are consistent with those observed in the dose escalation part of the study and suggest durability of efficacy.

Nanobiotix plans to launch a pivotal phase III global registration trial evaluating NBTXR3 as a single-agent activated by radiotherapy for patients with LA-HNSCC in 2021.

Priming Immune Response and Immunotherapy Combination Across Oncology

Abstract #2590: A Phase I Study of NBTXR3 Activated by Radiotherapy for Patients with Advanced Cancers Treated with an Anti-PD-1 Therapy
Abstract #2591: Overcoming Resistance to Anti-PD-1 with Tumor-Agnostic NBTXR3: From Bench to Bedside

Cancer immunotherapies such as anti-PD-1 have shown promising clinical outcomes over the past two decades and are often used to treat advanced cancers once other therapies have reached the end of their effectiveness. However, across tumor indications, the significant majority of patients (80-85% according to published data) receive only a temporary benefit from anti-PD-1—or no benefit at all—as they either develop resistance to the therapy over time or are non-responsive to treatment altogether.

Previously reported data from the Company’s phase I immunotherapy study in advanced cancers (Study 1100) and its preclinical collaboration with The University of Texas MD Anderson Cancer Center support NBTXR3 activated by radiotherapy as a potential primer of immune response. These data suggest that when combined with anti-PD-1, NBTXR3 could contribute to tumor regression in patients with advanced and metastatic tumors regardless of the patient’s prior exposure to anti-PD-1.

Nanobiotix will provide an update on Study 1100 with additional patients and further follow up prior to the conference (abstract #2590). The Company will also present a compilation of preclinical and clinical data supporting NBTXR3 as a potentially tumor-agnostic, therapeutic combination-agnostic agent that could overcome resistance to immune checkpoint inhibitors and increase response rates across tumor indications (abstract #2591).

Local Control as a Single-Agent for Patients with Soft Tissue Sarcoma

Abstract #11544: Long-Term Evaluation of the Novel Radioenhancer NBTXR3 plus Radiotherapy in Patients with Locally Advanced Soft Tissue Sarcoma Treated in the Phase III Act.in.Sarc Trial

A long-term safety analysis following the Nanobiotix phase II/III pivotal study evaluating NBTXR3 as a single-agent activated by radiotherapy in patients with locally advanced soft tissue sarcoma (STS) did not observe a negative impact on patient quality of life and long-term morbidity. The long-term safety profile of NBTXR3, together with its efficacy data, further supported a favorable benefit-risk ratio for patients with STS. The analysis highlighted potential for future indications, including non-resectable sarcoma, pediatric tumors, and re-irradiation.

Nanobiotix Investor Event

Nanobiotix will host a virtual investor event featuring several key opinion leaders, including study investigators, after the ASCO (Free ASCO Whitepaper) Annual Meeting on Friday, June 11, 2021 at 8 am ET. The discussion will expand on the new immunotherapy results from Study 1100 that will be reported prior to ASCO (Free ASCO Whitepaper), providing additional detail and clinical perspective, following the ASCO (Free ASCO Whitepaper) presentation. Register here.

***

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 is planned to launch in 2021. 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 clinical 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.