On May 29, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX), reported the final results from the GENUINE Phase 3 study evaluating the combination of ublituximab, the Company’s novel glycoengineered anti-CD20 monoclonal antibody, plus ibrutinib compared to ibrutinib alone in patients with previously treated high-risk chronic lymphocytic leukemia (CLL) at the 56thAmerican Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting (Press release, TG Therapeutics, MAY 29, 2020, View Source [SID1234558738]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "We are extremely pleased with the final results of the GENUINE Phase 3 trial presented today. Not only is this the first randomized trial to show a PFS benefit with the addition of an anti-CD20 antibody to ibrutinib, compared to ibrutinib monotherapy, it was also highly encouraging to see the combination significantly improved overall response rate, complete response rate, and importantly increased the rates of undetectable minimal residual disease." Mr. Weiss continued, "We believe these data support the potential of combination therapies to improve outcomes for patients with high risk CLL, especially those with 17p deletion/TP53 mutation, who continue to progress more rapidly than those without high risk cytogenetics."

Details of the data presentation are included below.

Presentation Title: Effect of Adding Ublituximab to Ibrutinib on PFS, ORR, and MRD Negativity in Previously Treated High-Risk Chronic Lymphocytic Leukemia: Final Results of the GENUINE Phase III Study

The GENUINE trial is an open-label, multicenter, randomized, Phase III study in relapsed or refractory high-risk CLL (del17p, del11q, or TP53mutation). This presentation includes data from 117 patients treated with either ublituximab plus ibrutinib (n=59) or ibrutinib alone (n=58). As of the cut-off date of September 1, 2019, patients had a median follow-up time of 41.9 months. The primary endpoint for this trial was overall response rate (ORR) as determined by an independent review committee (IRC). The secondary endpoints included progression free survival (PFS) and complete response (CR) rate as determined by an IRC, and undetectable minimal residual disease (uMRD) assessed by central lab.

Efficacy and safety highlights include:

The addition of ublituximab to ibrutinib compared to ibrutinib monotherapy significantly improved ORR (93% compared to 78%; p=0.019), complete response/complete response with incomplete blood count recovery (CR/CRi) rate (20% vs. 5%; p=0.024), and increased rates of uMRD (46% vs. 7%; p<0.001) in patients with relapsed/refractory CLL with high-risk cytogenetics
At a median follow-up of 41.9 months, median PFS was not reached in the ublituximab plus ibrutinib arm and was 35.9 months in the ibrutinib monotherapy arm (hazard ratio 0.46), with del17p/TP53mut patients seeing the greatest difference in PFS
The addition of ublituximab to ibrutinib did not significantly alter the known safety profile of ibrutinib however the combination resulted in slightly higher rates of neutropenia (36% compared to 21%) and atrial fibrillation (14% compared to 7%)
The data presented is available on the Publications page, located within the Pipeline section, of the Company’s website at www.tgtherapeutics.com/publications.cfm.

ABOUT THE GENUINE PHASE 3 STUDY
The GENUINE Phase 3 study is a randomized, open label, multicenter clinical trial to evaluate the safety and efficacy of ublituximab plus ibrutinib compared to ibrutinib alone in adult patients with high-risk chronic lymphocytic leukemia (CLL) who received at least one prior therapy for their disease.

Patients received ibrutinib orally at 420 mg once daily in both arms and in the combination arm those patients also received intravenous infusions of ublituximab at 900 mg dosed on days 1, 8 and 15 of cycle 1 and day 1 of cycles 2-6. Patients in the combination arm who had not progressed received quarterly infusions of ublituximab maintenance at 900 mg.

On May 29, 2020 Rakuten Medical, Inc. (Rakuten Medical), a global biotechnology company developing precision, cell-targeting investigational therapies on its Illuminox technology platform, reported that established a strategic cancer research alliance network, the Illuminox Alliance Institutes (IAI), in order to advance the development of new cancer therapies (Press release, Rakuten Medical, MAY 29, 2020, View Source [SID1234558737]). The National Cancer Center Japan (NCC) signed a memorandum of understanding (MOU) to become the first institution to join the IAI network. Rakuten Medical will expand the IAI network to include premier cancer centers from around the world.

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NCC is currently conducting clinical trials with ASP-1929, Rakuten Medical’s first investigational drug developed on the Illuminox platform. Under the signed MOU, NCC and Rakuten Medical will pursue multiple avenues to advance Illuminox-based cancer therapies. NCC will designate a lead principal who will act as an Illuminox Advisor. The Illuminox Advisor will, based on their expertise, actively participate in various research plans and Advisory board meetings, support identification of additional investigators from the IAI and assist with education and training related to the use of Illuminox platform therapies.

Hiroshi Mikitani, Chairman and CEO of Rakuten Medical, remarked, "There are no international borders in the mission to conquer cancer. Through the Illuminox Alliance, we attempt to advance novel cancer therapies with speed and agility to provide options to patients in need."

On May 29, 2020 Creatv MicroTech, a privately-held biotechnology company reported that it has pioneered a blood test for the universal screening of early stage cancer (Press release, CREATV MICROTECH, MAY 29, 2020, View Source [SID1234558736]). They will present their initial clinical data showing the ability to identify invasive cancer with 87% accuracy. "We are delighted to present results from a training set of 10 different types of cancers," said Dr. Cha-Mei Tang, Chief Executive Officer of Creatv. "The data shows that we obtained 85% sensitivity for all cancers (from 79% of patients in Stage I and increasing to 95% of patients in Stage IV), and also shows 100% specificity when tested against healthy normal controls. This represents a significant step towards pan cancer screening by a routine blood draw with high sensitivity and specificity."

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Creatv’s poster, Circulating Stromal Cells as a Blood Based Biomarker for Screening Invasive Solid Tumors, Abstract ID: 3535, highlights the performance of the LifeTracDxTM blood test. In a completely novel concept, the test analyzed the patient’s immune response to the presence of cancer by isolating stromal cells originating from cancer sites that have migrated into the blood stream. Creatv has shown that a particular subtype of circulating stromal cell, named Cancer Associated Macrophage-Like cells (CAMLs), can be used to identify patients with cancer but are absent in healthy persons. CAMLs are phagocytic myeloid cells derived from the patient’s immunological response to active malignancy that have engorged cancer cells, thereby containing cancer protein markers and cancer DNA.

In a large multi-institutional study, 7.5mL of peripheral blood was taken from 308 cancer patients after a diagnosis of invasive malignancy, [stage I (n=76), stage II (n=73), stage III (n=72), stage IV (n=65) and unstaged non-metastatic (n=22)]. Patients were recruited with lung, pancreas, breast, prostate, esophageal, renal cell, hepatocellular, neuroblastoma, melanoma, and others. To compare specificity of the test, blood was also taken from 39 patients with untreated non-malignant conditions (i.e. benign breast masses, lupus, liver cirrhosis, etc.), and from 76 healthy volunteers. CAMLs were 87% accurate at identifying cancer patients when compared to healthy controls or from patients with non-malignant conditions.

These initial findings have now been granted funding from National Cancer Institute/National Institute of Health (NCI/NIH), Department of Defense and NCI/NIH for validation studies in the screening of 1000 breast patients, 1000 lung patients and 300 prostate patients, respectively.

Posters will be available online at View Source

About LifeTracDxTM Blood Test

Creatv’s liquid biopsy assays (LifeTracDxTM) are commercialized research use only tests designed for analysis of CAMLs and Circulating Tumor Cells (CTCs) from whole blood. LifeTracDxTM tests are applicable for cancer screening, companion diagnostics, prediction of treatment response including immunotherapy, providing prognosis, delivering purified tumor DNA for sequencing, detecting minimal residual disease and early detection of cancer recurrence. LifeTracDxTM tests are currently being implemented in more than 20 clinical trials spanning from basic research to drug development. Creatv’s publications have shown that LifeTracDxTM can be used in an array of solid cancers as an early predictor of patient response to therapy.

On May 29, 2020 NANOBIOTIX (Paris:NANO)(Euronext: NANO – ISIN: FR0011341205 – the "Company"), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported new data from the expansion part of its phase I trial, evaluating the potential of first-in-class NBTXR3 activated by radiation therapy to improve treatment outcomes for elderly patients with locally advanced head and neck cancer ineligible for chemotherapy or intolerant to cetuximab (Press release, Nanobiotix, MAY 29, 2020, View Source [SID1234558735]). The data were published as part of the virtual scientific program at the 2020 annual meeting of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper).

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Study 102: A phase I trial of hafnium oxide nanoparticles activated by radiotherapy in cisplatin-ineligible locally advanced HNSCC patients

Authors: Christophe Le Tourneau, Valentin Calugaru, Edith Borcoman, Victor Moreno, Emiliano Calvo, Xavier Liem, Sebastien Salas, Bernard Doger, Olivier Choussy, Maria Lesnik, Xavier Mirabel, Nathalie Badois, Samar Krhili, Josefin Blomkvist, Nicolas Fakhry, Stéphanie Wong-Hee-Kam, Caroline Hoffmann.

J Clin Oncol 38: 2020 (suppl; abstr 6573). DOI: 10.1200/JCO.2020.38.15_suppl.6573.

Abstract Number: 6573

Study Design

After reporting promising early signs of activity from the dose escalation part of its phase I trial evaluating the safety and feasibility of NBTXR3 activated by radiation therapy in elderly patients with locally advanced head and neck squamous cell carcinoma (HNSCC), the Company launched an expansion cohort to confirm results and observed trends with a larger population treated at the recommended dose. This part of the study is expected to recruit a total of 44 evaluable patients. To date, 40 patients have been recruited, 30 of whom are evaluable for efficacy and are included in the data presented at ASCO (Free ASCO Whitepaper) 2020.

Topline Results

As previously announced, in the dose escalation part of the study NBTXR3 activated by radiation therapy was safe and well tolerated. The recommended phase 2 dose (RP2D) was determined to be 22% of baseline tumor volume. Among 16 evaluable patients, injected lesion complete response rate was 56% and overall objective response rate was 69%.

Regarding the new, expansion part data, analysis of 40 patients dosed showed that NBTXR3 activated by radiation therapy remains safe and well tolerated. In terms of efficacy, for the 30 evaluable patients, investigators observed, at a median time of 5 months after NBTXR3 injection, an overall objective response rate of 83% and an overall complete response rate of 43% and objective response rate of the primary tumor (target lesion) of 83% with a complete response rate of the primary tumor of 60%. The safety profile was consistent with the dose escalation part and the efficacy data improved (i.e. an increase in overall objective response rate from 69% in the dose escalation part to 83% in the dose expansion part).

In the safety population (all treated patients, N=40), three serious adverse events (SAEs) related to NBTXR3 were observed (0.7% of all AEs), comprising one case each of: Grade 4 tumor hemorrhage also related to radiotherapy, Grade 3 mucosal inflammation and Grade 2 swollen tongue also related to the injection procedure. Two SAEs related to the injection procedure were reported (0.5% of all AEs), comprising: two cases of swollen tongue, of which one was Grade 2 and also related to NBTXR3, and one was Grade 4. The radiotherapy-associated safety profile was as expected with the most frequently occurring AEs being stomatitis and skin injury. Three deaths due to AEs related to radiotherapy and other causes were observed. Four other patients died of non-oncologic or non-toxicity-related reasons.

Next Steps in Head and Neck Cancer

The expansion part of the phase I trial will continue to recruit until reaching 44 evaluable patients. In parallel, subject to the FDA’s pending review, the Company intends to globally launch a pivotal phase III trial. In the planned phase III trial, a futility analysis is expected 18 months after the first patient is randomized, and an interim analysis of progression-free survival (PFS) is expected at 24-30 months.

NBTXR3 for the treatment of locally advanced HNSCC patients who are not eligible for platinum-based chemotherapy received Fast Track designation from the FDA in February 2020. Fast Track designation is a process designed to facilitate the development and accelerate the review of drugs for serious conditions that have the potential to address unmet medical needs. The purpose is to expedite the availability of new treatment options for patients.

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 radiation therapy standards of care. The physical mode of action of NBTXR3 makes it applicable across solid tumors.

NBTXR3 is being evaluated in locally advanced head and neck squamous cell carcinoma (HNSCC) of the oral cavity or oropharynx in elderly patients unable to receive chemotherapy or cetuximab with limited therapeutic options. Promising results have been observed in the phase I trial regarding local control. In the United States, the Company has started the regulatory process to commence a phase III clinical trial in locally advanced head and neck cancers.

Nanobiotix is also running an Immuno-Oncology development program. The Company has launched a Phase I clinical trial of NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors in locoregional recurrent (LRR) or recurrent and metastatic (R/M) HNSCC amenable to re-irradiation of the HN and lung or liver metastases (mets) from any primary cancer eligible for anti-PD-1 therapy.

Other ongoing NBTXR3 trials are treating patients with hepatocellular carcinoma (HCC) or liver metastases, locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and pancreatic cancer. The Company has a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center (initially expected to support 9 new clinical trials in the United States) to evaluate NBTXR3 across several cancer types.

On May 29, 2020 Personalis, Inc., (Nasdaq: PSNL) a leader in advanced genomics for cancer, reported that the company will present new data in abstracts to be presented at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Meeting, which will be held May 29-31, 2020 (Press release, Personalis, MAY 29, 2020, View Source [SID1234558734]).

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One abstract showcases data from the ImmunoID NeXT Platform, the first platform to enable comprehensive analysis of both a tumor and its immune microenvironment from a single sample. ImmunoID NeXT can be used to investigate the key tumor- and immune-related areas of cancer biology, consolidating multiple oncology biomarker assays into one and maximizing the biological information that can be generated from a precious tumor specimen.

Another abstract highlights data from the Personalis NeXT Dx Test, one of the first cancer diagnostic platforms to profile approximately 20,000 genes in both the tumor exome and transcriptome, providing a comprehensive genomic testing solution that goes beyond many existing cancer diagnostic panels that focus on a few hundred genes. The Personalis NeXT Dx Test includes advanced analytics to provide a diagnostic report on genetic alterations in clinically-important cancer genes, as well as emerging immunotherapy composite biomarkers of medical importance.

Following are details and links to the abstracts that will be presented at the online meeting.

Abstract Number &
Session Title

Title & Presenter

Date

Location

6557
Head and Neck Cancers

Exome scale liquid biopsy characterization of putative neoantigens and genomic biomarkers pre- and post anti-PD-1 therapy in squamous cell carcinoma of the head and neck.
Presenter: Charles Abbott

May 29, 2020

Online

e15583
Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Validation of an exome and transcriptome based diagnostic platform enabling clinical cancer therapy selection and emerging composite biomarkers for immunotherapy.
Presenter: Sebastian Saldivar