On July 8, 2019 AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, reported new clinical data from its ongoing glioblastoma Phase 2 clinical trial, investigating AIVITA’s platform immunotherapy targeting cancer stem cells (Press release, AIVITA Biomedical, JUL 8, 2019, View Source [SID1234537413]). Blood plasma biomarker analyses have identified predictive markers of efficacy in seven of eight treated subjects, a sample that represents 15% of the total clinical trial size.

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The findings were made by analyzing data collected from AIVITA’s ongoing Phase 2 clinical study in glioblastoma and data collected from completed Phase 2 clinical trials in melanoma which had demonstrated 72% 2-year survival, and 54% 5-year survival. In both the current and past trials, blood was collected from subjects at one week after dose administration. Principal component analysis, an advanced statistical tool used in making predictive models, was performed to analyze the immune responses in all patients from both trials. The majority of glioblastoma patients had the same cytotoxic and immunoglobulin responses that were associated with survival in the previous melanoma trials.

"The data indicate that all mechanistic pathways with anti-tumoral effects are being activated in the surviving melanoma patients and seven out of eight of the treated glioblastoma patients, in particular the cytotoxic Th1-Th17 response and an immunoglobulin Th2 response," said Dr. Gabriel Nistor, AIVITA’s Chief Science Officer. "It just makes sense; this is how the immune system fights cancer."

The findings add to AIVITA’s understanding of the mechanism of action behind its next-generation cancer stem cell targeting immunotherapy and could offer physicians an early indication of how patients will respond to treatment.

"Predictive markers of efficacy, an outstanding patient recruitment rate and a manufacturing success rate of over 95%; this is shaping up to be a highly successful clinical trial in a field that desperately needs a win," said Dr. Hans S. Keirstead, AIVITA’s Chief Executive Officer.

AIVITA is currently conducting three clinical studies investigating its platform immunotherapy in patients with ovarian cancer, glioblastoma and melanoma. AIVITA uses 100% of proceeds from the sale of its ROOT of SKIN skincare line to support the treatment of women with ovarian cancer.

CLINICAL TRIAL DETAIL

OVARIAN CANCER

AIVITA’s ovarian Phase 2 double-blind study is active and enrolling approximately 99 patients who are being randomized in a 2:1 ratio to receive either the autologous cancer stem cell-targeting immunotherapy or autologous monocytes as a comparator.

Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient monocytes were obtained, (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), and (5) who have completed primary therapy. The trial is not open to patients with recurrent ovarian cancer.

For additional information about AIVITA’s AVOVA-1 trial patients can visit: www.clinicaltrials.gov/ct2/show/NCT02033616

GLIOBLASTOMA

AIVITA’s glioblastoma Phase 2 single-arm study is active and is enrolling approximately 55 patients to receive the cancer stem cell-targeting immunotherapy.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC). The trial is not open to patients with recurrent glioblastoma.

For additional information about AIVITA’s AV-GBM-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03400917

MELANOMA

AIVITA’s melanoma Phase 1B open-label, single-arm study will establish the safety of administering anti-PD1 monoclonal antibodies in combination with AIVITA’s cancer stem cell-targeting immunotherapy in patients with measurable metastatic melanoma. The study will also track efficacy of the treatment for the estimated 14 to 20 patients. This trial is not yet open for enrollment.

Patients eligible for treatment will be those (1) for whom a cell line has been established, (2) who have undergone leukapheresis from which sufficient monocytes were obtained, (3) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), (4) who have either never received treatment for metastatic melanoma or were previously treated with enzymatic inhibitors of the BRAF/MEK pathway because of BRAF600E/K mutations and (5) are about to initiate anti-PD1 monotherapy.

For additional information about AIVITA’s AV-MEL-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03743298

On July 8, 2019 Targovax ASA (OSE: TRVX), a clinical stage biotechnology company developing oncolytic viruses to target hard-to-treat solid tumors, reported that clinical responses were observed in 3 out of 9 patients in part 1 of the ONCOS-102 and Keytruda combination trial in anti-PD1 checkpoint inhibitor (CPI) refractory advanced melanoma, corresponding to an overall response rate (ORR) of 33% (Press release, Targovax, JUL 8, 2019, View Source [SID1234537412]).

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In this trial, ONCOS-102 immune activation is being tested in patients with advanced, unresectable melanoma who have had disease progression on treatment with anti-PD1 CPI. This is a particularly challenging patient population, with few treatment alternatives available. The nine patients in part 1 of the trial were given three intra-tumoral ONCOS-102 injections during the first week, followed by re-challenge with the anti-PD1 CPI Keytruda. The primary and secondary endpoints of the trial are to assess safety, immune activation and clinical responses of ONCOS-102 and Keytruda combination treatment. The main scientific aim is to test the hypothesis that ONCOS-102 can immune activate anti-PD1 resistant patients to respond to re-challenge with an anti-PD1 CPI.

Part 1 safety data shows that the sequential ONCOS-102 and Keytruda treatment regimen is well tolerated. The efficacy is also very encouraging, with three of nine patients demonstrating a clinical response (33% ORR); one patient had a complete response (CR) and two patients had partial responses (PR) according to RECIST1.1 and irRECIST assessment criteria. Although the patient number is small, these results compare favorably to reports from other explorative immunotherapy trials in this hard-to-treat population. Importantly, the data also confirms the hypothesis that ONCOS-102 is able to immune activate treatment resistant tumors to respond to re-challenge with an anti-PD-1 CPI.

ONCOS-102 was also seen to induce profound innate and adaptive immune activation. By week three, prior to starting Keytruda treatment, systemic increases in pro-inflammatory cytokines were observed in all nine patients (IL-6, TNFα and/or IFNγ), demonstrating potent systemic immune activation in response to the intra-tumoral ONCOS-102 injections. At the tumor level, increased infiltration of CD8+ T-cells were found in eight out of nine patients and the relative level of activated CD8+ T-cells (GrzB+) increased in all nine patients. Examples of increased T-cell infiltration into lesions not injected with ONCOS-102 were also observed. In addition, T-cells recognizing specific tumor antigens were found in circulation in four patients (MAGE-A1 and/or NY-ESO-1). These data suggest that the initial innate immune activation is translated into systemic anti-tumor immune responses.

Part 2 of the trial is currently enrolling patients, where safety and efficacy of a more intensive treatment regimen of twelve ONCOS-102 injections will be evaluated. The trial is running at Memorial Sloan Kettering Cancer Centre in New York, Fox Chase Cancer Centre, Philadelphia, and the University of Maryland, Baltimore.

Dr. Alexander Shoushtari, Principal Investigator, Memorial Sloan Kettering Cancer Centre, New York said: "It is encouraging to see clinical responses in this hard-to-treat population of advanced melanoma. Earlier this year, we decided to expand the trial to test a more intensified schedule of ONCOS-102, and it will be interesting to see whether this regimen can generate more and deeper clinical responses."

Dr. Magnus Jäderberg, CMO of Targovax, said: "We are very pleased to confirm our hypothesis that ONCOS-102 has the potential to immune activate checkpoint inhibitor resistant patients to respond to PD-1 blockade with Keytruda. It is promising to see this level of clinical responses after only three ONCOS-102 injections, including a complete response, which is rare in this heavily pre-treated patient population. Based on our experience so far, we speculate that patients will benefit from receiving more ONCOS-102 injections over a longer period of time and we will follow with interest the effect of the intensified dosing regimen in the second patient cohort of the trial."

CONTACT:

For further information, please contact:
Renate Birkeli, Investor Relations
Phone: +47-922-61-624
Email: [email protected]

Media and IR enquires:
Andreas Tinglum – Corporate Communications (Norway)
Phone: +47-9300-1773
Email: [email protected]

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On July 8, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported that it will report second quarter financial results after the Nasdaq market closes on Monday, July 29, 2019 (Press release, Neurocrine Biosciences, JUL 8, 2019, View Source [SID1234537411]). Neurocrine will then host a conference call and webcast to discuss its financial results and provide a Company update that day at 1:30 p.m. Pacific Time (4:30 p.m. Eastern Time).

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Participants can access the live conference call by dialing 866-342-8591 (US) or 203-518-9713 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine’s website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

On July 8, 2019 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported the publication of a study in BMC Biotechnology, a Springer Nature journal, describing and evaluating the development of the SWIFF-CAR, a CAR construct with an embedded on/off-switch, which enables tight control of the CAR surface presentation and subsequent cytolytic functions using a small molecule drug (Press release, Cellectis, JUL 8, 2019, View Source [SID1234537410]). The reversible control of these engineered T-cells represents a promising approach to further mitigate the potential toxicities that are associated with CAR T-cell administration in clinical settings and to improve the process of CAR T-cell production for specific target antigens.

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"Following Cellectis’ previously developed CubiCAR system, an all-in-one CAR architecture with an embedded multi-functional tag for purification, detection and elimination of CAR T-cells, we knew that we needed an additional ‘on/off switch’ to direct T-cells to either be active or inert, without necessarily killing them," said Dr. Alexandre Juillerat, Ph.D., Project Leader and U.S. Laboratory Head, Cellectis. "The SWIFF-CAR system does precisely that, representing a major breakthrough in our ongoing efforts to develop innovative applications to treat a range of diseases, including cancer and autoimmune diseases."

"While suicide gene systems have proven to be an effective way to eliminate transduced CAR T-cells, they also potentially terminate CAR T-cell treatment altogether," added Dr. Philippe Duchateau, Ph.D., Chief Scientific Officer, Cellectis. "The capability to manipulate CAR surface presentation improves the safety of CAR-T therapies and enhances our ability to circumvent hurdles associated with manufacturing. The SWIFF-CAR system addresses both of these factors and is an important next step for Cellectis in treating deadly illnesses that affect patients globally."

Alexandre Juillerat, Ph.D. Project Leader and Senior Scientist, Cellectis

Dr. Alexandre Juillerat, Ph.D., graduated in Chemistry from the University of Lausanne, Switzerland. After receiving his Ph.D. in 2006 in protein engineering from the École Polytechnique Fédérale de Lausanne (EPFL, Switzerland), he moved to the laboratory of Structural Immunology at the Institut Pasteur in Paris, France. In 2010, he joined the R&D department of Cellectis in Paris, France, working on the development and implementation of sequence specific designer nucleases including the transcription activator-like effector nucleases (TALEN). He then joined the Cellectis facility based in New York, NY, USA, leading projects associated with the development of the T-cell chimeric antigen receptor (CAR) technology.

Modulation of Chimeric Antigen Receptor surface expression by a small molecule switch

Alexandre Juillerat1, Diane Tkach1, Brian W. Busser1, Sonal Temburni1, Julien Valton1, Aymeric Duclert2, Laurent Poirot2, Stéphane Depil2 and Philippe Duchateau2

1Cellectis Inc, 430E 29th street, NYC, NY 10016, USA
2Cellectis, 8 rue de la croix Jarry, 75013 Paris, France

On July 8, 2019 OncoSenX, Inc., a late preclinical-stage company developing therapeutics to kill cancer cells based on their genetics, reported it has raised $3 million in pre-seed funding to advance its pipeline (Press release, OncoSenX, JUL 8, 2019, View Source [SID1234537409]).

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"These funds will allow us to accelerate the preclinical research necessary for us to begin phase 1 clinical development"

"These funds will allow us to accelerate the preclinical research necessary for us to begin phase 1 clinical development," said Matthew Scholz, chief executive officer of OncoSenX. "We believe our non-viral gene therapy for solid tumors represents the first in a new class of cancer therapeutics. The OncoSenX team is diligently working to bring this new approach into the clinic for the benefit of a global oncology community clearly in need of new options."

OncoSenX is developing a highly selective tumor-killing platform with two main components: a proprietary lipid nanoparticle (LNP) for cellular delivery and a highly selective DNA payload. The LNP is designed to deliver its non-integrating DNA payload to solid tumors, while an engineered promoter drives expression of a potent, inducible death protein only in the target cell population. The goal is to precisely target cell populations based on their genetic activity without harming nearby cells. The platform can be effectively programmed to implement logic gates (IF/OR/AND) to provide selectivity to any target cell based on its genetics.

"Our preclinical studies suggest the OncoSenX approach has the potential to precisely kill cancer cells based on the mutations they harbor," said John Lewis, Ph.D., chief science officer of OncoSenX. "If substantiated in the clinic, the platform could deliver reduced toxicity and improved tolerability over conventional chemotherapy, with the potential for superior targeting over biologics or even CAR-T therapy."

OncoSenX is currently preparing for its regulatory interactions and will then perform pivotal toxicology studies in anticipation of filing to begin human trials.