On September 18, 2017 Formula Pharmaceuticals, Inc. ("Formula") reported the opening of the first clinical trial for allogeneic Cytokine Induced Killer (C.I.K.) cell-based Chimeric Antigen Receptor (CAR) cancer immunotherapy (Press release, Formula Pharmaceuticals, SEP 18, 2017, View Source [SID1234520545]).

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This open-label, dose-escalating clinical Phase 1/2a trial will evaluate the safety, pharmacokinetics (PK), and antitumor activity of modified allogeneic C.I.K. cells administered to pediatric and adult patients with relapsed or refractory acute lymphoblastic leukemia (ALL). This clinical trial, sponsored by Fondazione Matilde Tettamanti and supported in part by Formula in accordance with the Italian/EU regulations on investigator-initiated trials, will be conducted at the MBBM Foundation/San Gerardo Hospital in Monza, Italy, and the Azienda Ospedaliera Papa Giovanni XXIII in Bergamo, Italy, both of which are affiliated with the University of Milan-Bicocca.

"We are very proud to have led the C.I.K. CAR program from the bench to the clinic and look forward to demonstrating the therapeutic potential of this platform for patients with persistent unmet need," commented Prof. Andrea Biondi, Director of the Pediatric and Pediatric Hematologic Oncology Clinic at the University of Milan-Bicocca, MBBM Foundation/San Gerardo Hospital, and Scientific Director at the Fondazione M. Tettamanti in Monza, Italy. "We look forward to expanding our close collaboration with Formula into additional hematologic cancer applications."

"This clinical trial is the first to evaluate non-virally transfected, allogeneic C.I.K. CAR in patients with cancer and is designed to demonstrate important clinical proof of concept for our entire platform," said Maurits Geerlings, MD, president and CEO of Formula. "We are honored to support Fondazione M. Tettamanti in reaching this important milestone."

Formula’s product pipeline is based on a proprietary non-viral, allogeneic C.I.K. CAR technology platform, which has the potential to overcome practical, therapeutic and commercial limitations related to existing CAR-T approaches that involve autologous blood, viral transfection and restricted immune effector functionality. Formula’s C.I.K. CAR technology activates T cell and Natural Killer cell functionality within one effector cell population, sourced from healthy donor or cord blood. With this approach, the Company is developing off-the-shelf CAR immunotherapies for various hematologic and solid tumor indications.

On September 18, 2017 UCL Business spinout company, Autolus Limited, a clinical-stage biopharmaceutical company focused on the development and commercialisation of next-generation engineered T-cell therapies, reported completion of the first-dose cohort of its phase I/II study of its novel, dual-targeted Chimeric Antigen Receptor (CAR) in patients with relapsed/refractory multiple myeloma (Press release, UCLB, SEP 18, 2017, http://www.uclb.com/news-and-events/news-post/autolus-announces-first-dose-cohort-completed-in-april-study-of-auto2-a-phase-iii-study-in-patients-with-multiple-myeloma [SID1234520543]).

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AUTO2 is a chimeric antigen receptor T-cell (CAR-T cell) therapy that targets both B-cell maturation antigen (BCMA) and transmembrane activator and calcium modulator and cyclophilin ligand interactor (TACI). The APRIL Study is a dose-escalation phase I/II study in which cohorts of patients receive ascending doses of AUTO2 to determine the maximum tolerated dose and establish a recommended dose. The second part of the study is an expansion phase where patients receive AUTO2 to further evaluate the safety, tolerability and clinical activity at this recommended dose.

Dr Jesus G. Berdeja, Director of Myeloma Research & Senior Investigator, Hematologic Malignancies, Sarah Cannon Research Institute said:
"BCMA CAR-T cell therapies have shown considerable promise in early clinical studies. A dual-targeted approach may minimise the risk for antigen negative escape and extend CAR-T treatment to patients with low density of BCMA antigen on the surface their cancer cells."

Dr Christian Itin, Autolus’ CEO added:
"Breaking the defence mechanisms of cancers against T-cells is key to unlocking the curative potential of CAR-T cell therapies. AUTO2 is a first example of Autolus’ approach to specifically re-programme the patient’s own T-cells to minimise the risk of the cancer cells escaping treatment. With the start of the APRIL study we have transitioned to a clinical stage company; an important step on our path to build a fully integrated autologous CAR-T cell company with a portfolio of differentiated therapies for the treatment of patients with cancer."

On September 18, 2017 RedHill Biopharma Ltd. (NASDAQ:RDHL) (Tel-Aviv Stock Exchange:RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on late clinical-stage development and commercialization of proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, reported that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a new patent covering the use of two of RedHill’s Phase II-stage proprietary investigational compounds, YELIVA and MESUPRON in combination with a known antibiotic (Press release, RedHill Biopharma, SEP 18, 2017, View Source [SID1234520542]).

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Upon issuance, on top of existing intellectual property protection covering the individual compounds, the new patent will provide RedHill with intellectual property protection covering its combination for the potential treatment of cancer, prevention of cancer recurrence or progression and inhibition of growth and proliferation of cancer cells.

Danielle Abramson, Ph.D., RedHill’s VP of Intellectual Property and Research, stated: "We are very pleased with the allowance of this key patent, covering the combination of our proprietary, first-in-class, orally-administered Phase II-stage drug candidates, YELIVA and MESUPRON, both of which are new chemical entities, with a known antibiotic. As part of our oncology program, RedHill is currently evaluating the new combination therapy across several oncology indications with limited treatment options where strong unmet medical need exists."

On September 18, 2017 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" and "the Company"), a biopharmaceutical company focused on the development of new cancer therapies, reported that the U.S. Food and Drug Administration ("FDA") has allowed an additional Investigational New Drug Application ("IND") to study its lead drug candidate VAL-083 as a potential treatment for ovarian cancer (Press release, DelMar Pharmaceuticals, SEP 18, 2017, View Source [SID1234520541]).

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"The opening of this new IND to study VAL-083 in ovarian cancer marks a major milestone for our Company as we continue to investigate this agent as an important potential therapy for the treatment of multiple cancers," said Jeffrey Bacha, DelMar’s president and chief executive officer.

VAL-083 is a first-in-class, DNA targeting agent that demonstrated clinical activity against a range of tumor-types in prior clinical trials sponsored by the U. S. National Cancer Institute ("NCI"). Published results from NCI studies include recommendations for further study of VAL-083 in advanced clinical trials for ovarian cancer and other gynecologic malignancies.

DelMar’s clinical trial will be a multi-center, Phase 1/2 Study of VAL-083 in patients with Recurrent Platinum Resistant Ovarian Cancer ("VAL-083 REPROVe Trial"). DelMar’s research demonstrates that VAL-083’s unique mechanism of action has the potential to overcome chemo-resistance to platinum-based chemotherapy in ovarian, lung and other solid tumors.

Ovarian cancer remains the leading cause of death among women with gynecological cancers and the fifth most frequent cause of cancer deaths in women overall. The American Cancer Society estimates that in 2017, approximately 22,440 women in the US will be diagnosed with ovarian cancer and approximately 14,080 will die from their disease. The majority of these deaths were patients whose tumors had become resistant to platinum-based chemotherapy regimens. Currently, there are no high-efficacy therapeutic options for platinum-resistant ovarian cancer, leaving these cancer patients with very poor prognosis. According to published literature, the overall response rate ("ORR") to second line therapy is in the 10-15% range and overall survival is approximately 12-months.

"The development of new treatments to overcome platinum resistance represents the largest unmet medical need in the treatment of ovarian cancer," stated Dr. Bradley J. Monk, MD, principal investigator of the VAL-083 REPROVe Trial and director of the Division of Gynecologic Oncology Research at Arizona Oncology. "Based on DelMar’s recent presentation of pre-clinical data demonstrating activity of VAL-083 against platinum-resistant ovarian cancer, we are enthusiastic about exploring the drug’s potential in this important clinical setting."

About the VAL-083 REPROVe Trial

The VAL-083 REPROVe Trial is an open label, multi-center, Phase 1/2 clinical trial to evaluate the safety and efficacy of VAL-083 in patients with recurrent adenocarcinoma of the ovary, who have been previously treated with a minimum of two courses of platinum-based chemotherapy, and whose cancer has recurred within six months of the most recent platinum-based chemotherapy. Patients enrolled in the trial will receive VAL-083 intravenously once per week for 16 weeks or until disease progression.

VAL-083 activity against platinum-resistant ovarian cancer will be measured based on ORR using the Response Evaluation Criteria in Solid Tumors ("RECIST") version 1.1 criteria as well as response duration, progression free survival, and a measurement of CA-125 biomarker levels in the blood. The study’s primary endpoint is to demonstrate an ORR benefit compared to historical control of 12-15%.

Twenty-four patients will be enrolled under the first phase of the VAL-083 REPROVe Trial with top line results expected within 18-24 months from trial initiation of patient treatment. DelMar plans to request a meeting with the FDA following the completion of Phase 1. If successful, DelMar expects that data from Phase 1 will lead to a Phase 2 expansion study. If Phase 2 is successful, and subject to FDA feedback, DelMar may be positioned to file an application for accelerated approval or, alternatively, to advance VAL-083 to a pivotal Phase 3 trial.

"Our planned work in ovarian cancer will be complementary to our ongoing Phase 2 and 3 clinical trials with VAL-083 as a potential treatment for refractory and MGMT-unmethylated glioblastoma multiforme ("GBM"). Based on our research, we believe that the REPROVe Trial has the potential to provide significantly improved outcomes for patients suffering from platinum-resistant ovarian cancer," added Mr. Bacha. Subject to availability of funding, DelMar anticipates opening the VAL-083 REPROVe Trial for patient enrollment in early 2018.

Further details on the VAL-083 REPROVe Trial and other VAL-083 clinical trials can be found at View Source

About VAL-083

VAL-083 (dianhydrogalactitol) is a "first-in-class", DNA-targeting agent that introduces interstrand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cancer cell death. VAL-083 has demonstrated clinical activity against a range of cancers including GBM in historical clinical trials sponsored by the U.S. National Cancer Institutes.

VAL-083 has been granted an orphan drug designation by the U.S. FDA Office of Orphan Products for the treatment of glioma, medulloblastoma and ovarian cancer, and in Europe for the treatment of malignant gliomas. VAL-083 is currently being studied in multiple clinical trials as a potential new treatment for GBM, the most common and aggressive form of brain cancer.

DelMar has demonstrated that VAL-083’s mechanism of action is distinct from multiple chemotherapies widely used in the treatment of cancer and that this unique mechanism may offer opportunities to overcome treatment resistance thereby offering new treatment options to cancer patients. Further details regarding these studies can be found at View Source