Phase I Study Results Show First-in-class NBTXR3 Could Present as a Valuable Option for Patients With Hepatocellular Carcinoma or Liver Metastasis

On September 18, 2019 NANOBIOTIX (Euronext : NANO – ISIN : FR0011341205 – the ‘‘Company’’), reported encouraging findings from the Company’s phase I study evaluating first-in-class radioenhancer NBTXR3 activated by stereotactic body radiation (SBRT) (Press release, Nanobiotix, SEP 18, 2019, View Source [SID1234539628]). These data were presented at the ASTRO 2019 Annual Meeting by investigator Dr. Enrique Chajon.

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Hafnium oxide nanoparticles activated by SBRT for the treatment of hepatocellular carcinoma and liver metastasis: a phase I/II trial

Enrique Chajon1, Marc Pracht1, Yann Rolland1, Thierry de Baere2, France Nguyen2, Jérôme Durand-Labrunie2, Jean-Pierre Bronowicki3, Véronique Vendrely4, Antonio Sa Cunha5, Anne-Sophie Baumann6, Valérie Croisé-Laurent3, Emanuel Rio7, Samuel Le Sourd1, Pierre Gustin2, Patricia Said8, Christophe Perret7, Didier Peiffert5, Eric Deutsch2

1Radiation Oncology, Centre Eugene – Marquis, Rennes, FR, 2Radiation oncology, Institut Gustave Roussy, Villejuif, FR, 3Hepatology and Gastroenterology, Hôpital de Brabois, Vandoeuvre Les Nancy, FR, 4Radiotherapy, Groupe Hospitalier Sud – Hôpital Haut-Lévêque, Pessac, FR, 5Centre Hépato-Biliaire Paul Brousse, Villejuif, FR, 6Radiotherapy, Institut de Cancérologie de Lorraine, Nancy, FR, 7Radiotherapy, Institut de cancérologie de l’Ouest, Nantes, FR, 8Nanobiotix, SA, Paris, Fr

The population for this study included (i) hepatocellular carcinoma (HCC) patients with a minimum life expectancy of three (3) months that were unsuitable for local surgery or local treatment and (ii) liver metastasis (mets) patients with a minimum life expectancy of six (6) months whose tumors are unresectable. The total number of HCC patients was 11, the total numbers of mets patients was 6.

The HCC patient population has high unmet medical needs as they have underlying liver dysfunction and concomitant malignancies that limit treatment options. Stereotactic body radiation therapy (SBRT) is a well-tolerated alternative therapy, however the dose is limited by hepatic function which is widely impaired in this group. Therefore, these patients need a way to increase the dose delivered to the tumor while sparing surrounding healthy tissues without additional strain on the liver. Moreover, given the short life expectancy of these patients, the need is particularly urgent.

Given that NBTXR3 is inert unless exposed to radiotherapy, is not metabolized by the liver, and can be administered regardless of liver and renal function, the first-in-class treatment offers an opportunity to increase

the efficacy of SBRT while maintaining tolerable dose levels for the patient. In this study, the safety profile of NBTXR3 activated by SBRT was similar to SBRT alone with no dose-limiting toxicity (DLT). Additionally, NBTXR3 also proved to remain within the injected tumor with no leakage to surrounding tissue.

To date, 62.5% of evaluable HCC patients have shown a complete response while the rest have shown partial response. These results show that NBTXR3 activated by SBRT may present as a valuable option in patients with HCC not amenable to curative local treatment or with unresectable liver metastases.

In liver mets, 50% of patients have shown partial response and 50% show stable disease.

Given that the safety profile was very good and there were no DLTs, the protocol was amended to include a fifth dose escalation level of 42%. Three (3) patients have already been recruited and recruitment is expected to be complete in Q4. Results for all patients are expected in Q1 2020.

These results are significant for the Company as they increase the scope of patients NBTXR3 may treat beyond Soft Tissue Sarcoma and Head and Neck cancer. The Company remains focused on its head and neck plan but will now accelerate integration of HCC into its go-to-market strategy.

***

About NBTXR3

NBTXR3 is a first-in-class product designed to destroy tumors through physical cell death when activated by radiotherapy. NBTXR3 has a high degree of biocompatibility, requires one single administration before the first radiotherapy treatment session, and has the ability to fit into current worldwide standards of radiation care. The physical mode of action of NBTXR3 makes it applicable across solid tumors such as lung, prostate, liver, glioblastoma, and breast cancers.

NBTXR3 is actively being evaluated in head and neck cancer with locally advanced squamous cell carcinoma of the oral cavity oropharynx in elderly and frail patients unable to receive chemotherapy or cetuximab with very limited therapeutic options. Promising results have been observed in the phase I/II trial regarding the local control of the tumors. In the United States, based on the discussions with the Food and Drug Administration that occurred in the first half of 2019, the Company plans to begin the clinical trial authorization process in the second half of 2019 and commence a phase II/III clinical trial in locally advanced head and neck cancers.

Nanobiotix is also running an Immuno-Oncology development program. In the United States, the Company received approval from the Food and Drug Administration to launch a clinical trial of NBTXR3 activated by radiotherapy in combination with antiPD-1 antibodies in lung, and head and neck cancer patients (head and neck squamous cell carcinoma and non-small cell lung cancer).

The other ongoing NBTXR3 trials are treating patients with liver cancers (hepatocellular carcinoma and liver metastasis), locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and prostate adenocarcinoma. Further, the company has a large-scale, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center (9 new phase I/II clinical trials in the United States) to evaluate NBTXR3 across head and neck, pancreatic, thoracic, lung, gastrointestinal and genitourinary cancers.

Dr. Robert Petit Follows His Passion to OS Therapies as Chief Medical & Scientific Advisor

On September 18, 2019 OS Therapies, a clinical-stage biopharmaceutical company focused on discovering and developing innovative therapies to treat and cure Osteosarcoma (OS), reported the appointment of Robert Petit, Ph.D., as its Chief Medical and Scientific Advisor. He is continuing to support the technology that he helped to develop at Advaxis (Press release, OS Therapies, SEP 18, 2019, View Source [SID1234539627]).

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Dr. Petit is a highly successful C-level executive and biopharma leader with a diverse background in research and development with over 25 years of experience in clinical development, and pre-clinical and translational research. In addition, he has helped to build, grow and secure investments for respected academic organizations, as well as private and publicly traded biopharma companies. An accomplished immuno-oncology scientist, innovator and drug developer, he has been integral to 7 NDA/BLA/EMA filings in the immuno-oncology space and was part of team at BMS who established the first checkpoint inhibitor therapy. Most recently, Dr. Petit served as Chief Scientific Officer of Advaxis, Inc., contributing to their successful NASDAQ IPO, raising significant capital, and evolving their platform technology to leverage both personal and shared neoantigens.

"I am excited to join the OS Therapies team and contribute toward realizing the potential of the OS Therapies lead program in the treatment of osteosarcoma," said Dr. Petit. "I also look forward to furthering the mission of OS Therapies by assisting in building a pipeline of assets that will improve treatment options and outcomes for patients with osteosarcoma."

"From our first meeting with Advaxis to discuss out-licensing for the Osteosarcoma indication, we knew Robert was the driving force behind the development of the technology," said Paul Romness, Chief Executive Officer of OS Therapies. "Already, he has been a crucial contributor to our team in bringing knowledge and passion for the technology. As our Chief Medical and Scientific Advisor, he is set to bring even more confidence and institutional knowledge to our investors and clinical researchers alike."

Biodesix Announces New Clinical Data Demonstrating VeriStrat Testing Predicts Survival in Advanced NSCLC and Immunotherapy

On September 18, 2019 Biodesix, Inc. reported findings from multiple studies demonstrating the significance and clinical utility of its VeriStrat blood-based proteomic test as a predictive biomarker for patient survival outcomes and as a diagnostic tool to improve the clinical management of patients with non-small cell lung cancer (NSCLC) (Press release, Biodesix, SEP 18, 2019, View Source [SID1234539624]). This information is vital to implementing optimal treatment strategies for patients and supports disease state monitoring. To date, VeriStrat testing has helped more than 50,000 patients in determining the best treatment for their lung cancer.

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Biomarker Identification for Immunotherapy

Data from a clinical study that examined the impact of VeriStrat testing on patients receiving immune checkpoint inhibitors (ICIs) as part of their NSCLC treatment strategy, showed that the VeriStrat MS-based proteomic signature has potential as a biomarker for survival outcomes in NSCLC patients receiving immunotherapy. Among NSCLC patients treated with ICIs, VeriStrat-Good classification was associated with significantly increased PFS in comparison to VeriStrat-Poor classification (median PFS of 6.2 vs. 3.0 months, p=0.012), while the differences in OS trended towards significance (median OS, not reached vs. 16.5 months p=0.076). These results were presented by Northwestern University Feinberg School of Medicine Professor Young Kwang Chae, MD, MPH, MBA at the International Association for the Study of Lung Cancer (IASLC) 2019 World Conference on Lung Cancer.

"As an oncologist, my goal is to identify the treatment course that will give each of my patients the best possible outcome," Dr. Chae stated. "By using VeriStrat, I am able to get a more complete clinical picture of my patient’s state of health, which allows me to work with that individual to determine the path forward that is best for them."

Improved Clinical Management of Advanced NSCLC Patients Unsuited for Chemotherapy

A recent University College London (UCL) study published in the European Journal of Cancer found that the VeriStrat test improved prognosis accuracy for patients with advanced NSCLC who were considered to be unsuitable candidates for chemotherapy. NSCLC accounts for 80-85 percent of all lung cancers and many patients are not diagnosed until their cancer has advanced to a late stage. Siow Ming Lee, PhD, UCL Professor of Medical Oncology and Chief Investigator of the TOPICAL trial, reported a strong correlation between the results of the VeriStrat test and patient outcomes. This VeriStrat correlation held even when the patients were split into the good and poor ECOG performance status subgroups; VeriStrat Good versus VeriStrat Poor in ECOG 0-1; HR 0.25, p=0.006 and ECOG 2-3; HR 0.60, p=0.002.

"The relationship between the VeriStrat test and survival is a major finding within this patient population, who have generally poor health and are difficult to treat," Dr. Lee commented. "The VeriStrat test seems to distinguish patients who have a shorter or longer survival. This has never been reported before in this particular patient group."

The VeriStrat test is a multivariate, mass-spectrometry based test that measures circulating proteins in the blood serum or plasma of patients with NSCLC. Test results assign a good (VS Good) or poor (VS Poor) classification to patient samples. Multiple studies support that patients with a VS Good result have a better prognosis than patients with a VS Poor result, independent of current clinical prognostic indicators and treatment choice. VeriStrat testing has been validated in more than 85 studies with more than 6,600 patients and is covered by Medicare and many private health insurers.

"VeriStrat blood testing continues to help guide treatment decisions as evidenced through these studies and many others," said Scott Hutton, Biodesix COO. "The potential for VeriStrat continues to expand as studies demonstrate relevancy in lung cancer with the latest therapies in development."

Veristrat is part of the Biodesix Lung Reflex Strategy (BLR), which integrates the GeneStrat genomic test. The BLR strategy has been widely adopted across oncology clinics due primarily to its ability to provide a more comprehensive understanding of patients’ health. Through a series of case studies, physicians detail the value offered through GeneStrat and VeriStrat testing, highlighting their ability to optimize treatment strategies, and to inform conversations on prognoses and treatment options with patients.

Genprex to Present Via Webcast at the Singular Research Midwestern Values Conference on September 19, 2019

On September 18, 2019 Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company, reported that it will present at the Singular Research Midwestern Values Conference on September 19, 2019 via webcast (Press release, Genprex, SEP 18, 2019, View Source [SID1234539623]).

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Genprex’s Chairman and Chief Executive Officer, Rodney Varner, will lead the company’s webcast presentation.

Genprex will present at the Singular Research Midwestern Values Conference as follows:

Date: Thursday, September 19, 2019

Time: 4:00-4:30 p.m. CDT

Webcast Link: http://bit.ly/2kpgxlu

Registration for the webcast can be completed on the Singular Research website at www.SingularResearch.com.

Janssen to Highlight Depth of Solid Tumour Portfolio with Multiple Data Presentations at ESMO 2019

On September 18, 2019 The Janssen Pharmaceutical Companies of Johnson & Johnson reported multiple data presentations from its solid tumour portfolio, including key prostate cancer data, will be featured at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Annual Congress, taking place 27th September to 1st October in Barcelona, Spain (Press release, Janssen Pharmaceuticals, SEP 18, 2019, View Source [SID1234539622]). Among Janssen’s 12 accepted abstracts is an oral presentation reporting overall survival from the Phase 3 SPARTAN study investigating the use of apalutamide in patients with high-risk non-metastatic castration-resistant prostate cancer (nmCRPC); patient-reported outcomes from the Phase 3 TITAN study in patients with metastatic hormone-sensitive prostate cancer (mHSPC) demonstrating maintenance of overall health-related quality of life with apalutamide; and a late-breaking interim analysis from the Phase 2 GALAHAD study evaluating niraparib in the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC) and biallelic DNA-repair gene defects (DRD).

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"We are delighted to share key data from our solid tumour portfolio at this year’s ESMO (Free ESMO Whitepaper) Congress, including new findings for apalutamide and niraparib in the treatment of prostate cancer," said Dr Joaquín Casariego, Janssen Therapeutic Area Lead Oncology for Europe, Middle East & Africa, Janssen-Cilag S.A.. "We continue to pursue areas of oncology where there is the greatest unmet need, and the data being presented in Barcelona reflects our ongoing passion and commitment to improving patient outcomes."

Company-sponsored abstracts to be presented at the meeting include:

Abstract No.

Title

Date/Time

Apalutamide

Oral Presentation

Abstract #843O

Apalutamide and Overall Survival in Patients with Nonmetastatic Castration-Resistant Prostate Cancer (nmCRPC): Updated Results from the Phase 3 SPARTAN Study

Friday 27th September

14:00 – 14:15 CET

Poster Presentations

Abstract #851PD

Patient-Reported Outcomes (PROs) From TITAN: A Phase 3, Randomized, Double-Blind Study of Apalutamide Versus Placebo

Added to Androgen Deprivation Therapy in Patients with Metastatic Castration-Sensitive Prostate Cancer (mCSPC)

Sunday 29th September

09:20 CET

Abstract #883P

Androgen Receptor Aberrations in Patients with Metastatic Castration-Sensitive Prostate Cancer (mCSPC) Treated with Apalutamide Plus Androgen Deprivation Therapy in TITAN

Monday 30th September

12:00 – 13:00 CET

Abstract #900TiP

A Phase 2 randomized, open-label study comparing salvage radiotherapy in combination with 6 months of androgen-deprivation therapy with LHRH agonist or antagonist versus anti-androgen therapy with apalutamide in patients with biochemical progression after radical prostatectomy

Monday 30th September

12:00 – 13:00CET

Abiraterone acetate

Poster Presentation

Abstract #95P

Evaluation of markers associated with efficacy of abiraterone acetate plus prednisone in patients with castration-sensitive prostate cancer (mCSPC) from the LATITUDE study

Monday 30th September

12:00 – 13:00 CET

Niraparib

Poster Presentations

Abstract #LBA50

Pre-specified interim analysis of GALAHAD: A Phase 2 study of niraparib in patients with metastatic castration-resistant prostate cancer (mCRPC) and biallelic DNA-repair gene defects

Sunday, 29th September

08:30 CET

Abstract #897TiP

A Phase 3 randomized, placebo-controlled, double-blind study of niraparib plus abiraterone acetate and prednisone versus abiraterone acetate

and prednisone in patients with metastatic prostate cancer (NCT03748641)

Monday 30th September

12:00 – 13:00 CET

Abstract #1412P

Analytical performance of the Resolution-HRD plasma assay used to identify mCRPC patients with biallelic disruption of DNA repair genes for

treatment with niraparib

Monday 30th September

12:00 – 13:00 CET

Erdafitinib

Poster Presentations

Abstract #925P

Analysis of response to prior therapies and therapies after treatment with erdafitinib in fibroblast growth factor receptor (FGFR)-positive patients with metastatic urothelial carcinoma

Monday 30th September

12:00 CET

Abstract #926P

Erdafitinib versus available therapies in advanced urothelial cancer: A matching adjusted indirect comparison

Monday 30th September 30

12:00 CET

Abstract #932P

Hyperphosphatemia due to Erdafitinib (a Pan-FGFR Inhibitor) and Antitumor Activity Among Patients with Advanced Urothelial Carcinoma

Monday 30th September

12:00 CET

Solid Tumor Portfolio

Poster Presentation

Abstract #488P

Correlation of Progression Free Survival-2 and Overall Survival in Solid Tumors

Saturday 28th September

12:00 CET

About ERLEADA (apalutamide)

ERLEADA (apalutamide) is an androgen receptor (AR) inhibitor indicated for use in Europe for the treatment of patients with high-risk non-metastatic castration-resistant prostate cancer (nmCRPC).1 In the U.S. apalutamide is indicated for the treatment of nmCRPC.2

About ZYTIGA (abiraterone acetate)

ZYTIGA (abiraterone acetate) in combination with prednisone is indicated in Europe and the U.S. for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC) and newly-diagnosed high-risk metastatic hormone-sensitive prostate cancer (mHSPC).3,4

About BALVERSATM (erdafitinib)

BALVERSA (erdafitinib) is a once-daily, oral fibroblast growth factor receptor (FGFR) kinase inhibitor that is currently being studied for the treatment of patients with advanced or metastatic urothelial cancer.5 In the U.S. it is indicated for the treatment of adults with locally advanced or metastatic urothelial carcinoma (mUC) that has susceptible FGFR3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.6

About niraparib

Niraparib is an orally-administered selective poly ADP ribose polymerase (PARP) inhibitor that is currently being studied by Janssen for the treatment of patients with prostate cancer.7 In April 2016, Janssen entered a worldwide (except Japan) collaboration and license agreement with TESARO, Inc., for exclusive rights to niraparib in prostate cancer.8 In the U.S., niraparib is indicated for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.9 Niraparib is currently marketed by TESARO, an oncology-focused business within GSK, devoted to providing transformative therapies to people facing cancer.10