On June 2, 2016 Onxeo S.A. (Euronext Paris, Nasdaq Copenhagen: ONXEO), an innovative company specializing in the development of orphan oncology therapeutics, reported the first set of positive results from a preclinical pharmacokinetic (PK) study on the bioavailability of an oral formulation of belinostat (Beleodaq) and the next steps in its development plan for the new formulation (Press release, Onxeo, JUN 2, 2016, View Source [SID:1234512969]). Beleodaq is a histone deacetylase inhibitor (HDACi) currently approved in the US for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL).

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The current formulation of belinostat is a lyophilized formulation for intravenous (IV) use. The aim of the company is to develop an oral formulation of belinostat offering strong benefits for patients and physicians in terms of compliance, as well as opening new opportunities for indications for which an oral formulation of belinostat is relevant.

As a key step in the development plan, the company has performed a canine PK bioavailability study to screen two enabling formulation technologies dedicated to improving solubility and assessed two prototypes selected based on dissolution properties, and physical and chemical stability.

Findings from this study showed a good level of bioavailability approaching the theoretical maximum achievable as indicated by the performance of the oral solution. Onxeo will then select the best of the two formulation technologies, to pursue formulation development and obtain a suitable clinical prototype and, in parallel, fine tune the optimal dosing regimen to then start clinical development in selected indications.

Judith Greciet, CEO of Onxeo, commented: "The positive results that we have obtained represent an important milestone towards developing a clinically-ready oral formulation of belinostat, which will provide multiple benefits to patients and physicians in terms of convenience, pain-free administration, and need for assistance from medical staff, especially as the other available PTCL treatments are injectable forms. Finally, it opens up a range of new opportunities in the orphan oncology field and beyond, giving Onxeo the opportunity to extend belinostat’s patent protection, thus reinforcing the interest to evaluate the compound in combination with other drugs for new indications."

Graham Dixon, PhD, Chief Scientific Officer of Onxeo, added: "The preclinical study conducted using the oral formulation of belinostat provides solid proof of achieving a good level of bioavailability and promising preliminary PK data. These data strongly support our next steps to conduct preclinical in vivo efficacy studies to confirm efficacy at achievable exposure levels and clinical trials with oral belinostat. "

On June 02, 2016 Intensity Therapeutics, Inc., a privately held biotechnology company developing proprietary cancer immunotherapy products, reported that the United States Patent and Trademark Office issued the Company US Patent 9,351,997 (Press release, Intensity Therapeutics, JUN 2, 2016, View Source;.pdf [SID:1234512967]).

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"Intensity’s DfuseRxSM platform technology has identified our lead drug, INT230-6. This novel product has shown great promise in regressing tumors and extending life in murine models of severe metastatic cancer via a combination of tumor cell death and immune system activation. Animals with large tumors achieve a complete response and become immunized against re-inoculation of the same cancer," said Intensity’s President and CEO, Lewis H Bender. "This issued US patent allows us to protect the market exclusivity of our unique and potent drug product. As we continue to prosecute additional patent applications in the US and countries around the world, the issuance of patent 9,351,997 increases our confidence that the Company’s unique cancer treatment technology can be secured."

About INT230-6
INT230-6 is a novel, anti-cancer drug product able to disperse through tumors and diffuse into cancer cells. The product was identified from Intensity’s DfuseRxSM platform technology. Using preclinical in vivo models of severe cancer, INT230-6 treatment results in substantial improvement in overall survival compared to standard therapies. The product can completely clear large tumors in animal models. Complete responders have long-term, durable protection even after multiple re-inoculations of the cancer. INT230-6 administration has shown an increased recruitment of immune cells to the tumor micro-environment.

On June 2, 2016 Mylan N.V. (NASDAQ, TASE: MYL) reported the U.S. launch of Azacitidine for Injection, 100 mg/vial, which is a generic version of Celgene’s Vidaza Injection, 100 mg/vial. Mylan received final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA) for this product (Press release, Mylan, JUN 2, 2016, View Source [SID:1234512966]). Azacitidine for Injection is a nucleoside metabolic inhibitor indicated for the treatment of the five French-American-British (FAB) subtypes of myelodysplastic syndrome, a blood cell disorder that can occur as a result of cancer treatments or can progress to leukemia.

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Mylan
Azacitidine for Injection, 100 mg/vial, had U.S. sales of approximately $236.3 million for the 12 months ending March 31, 2016, according to IMS Health.
Mylan’s launch of this product adds to the company’s portfolio of more than 150 injectable products available to patients in the U.S. across a broad array of therapeutic categories. Azacitidine for Injection is also a part of a growing U.S. portfolio of more than 20 oncology medications that includes treatments for breast, lung, colorectal, ovarian and hematologic cancers.
Currently, Mylan has 254 ANDAs pending FDA approval representing $108.3 billion in annual brand sales, according to IMS Health. Forty-three of these pending ANDAs are potential first-to-file opportunities, representing $37.2 billion in annual brand sales, for the 12 months ending December 31, 2015, according to IMS Health.

On June 2, 2016 MiNA Therapeutics, the pioneer in RNA activation therapeutics, reported the initiation and first patient treated in the OUTREACH Phase I clinical study of their lead program, MTL-CEBPA, in severe liver cancer (Press release, MiNA Therapeutics, JUN 2, 2016, View Source [SID:1234512962]).

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The study is the first-in-human trial of a small activating RNA (saRNA) and is designed to assess the safety and tolerability of MTL-CEBPA, an saRNA restoring the expression of CCAAT/enhancer binding protein alpha (C/EBP-a). C/EBP-a plays an important role in normal liver function and the benefits of increasing its expression have been demonstrated in multiple pre-clinical models of disease.

"Initiation of the Phase I study is an important achievement in our mission to improve patients’ lives with our groundbreaking class of medicines known as small activating RNAs," said Robert Habib, CEO of MiNA Therapeutics. "There is increasing excitement about the possibility of using RNA to induce therapeutic protein production. We believe our unique approach, here applied to the upregulation of C/EBP-a protein, may provide to patients significant benefits over conventional medicines."

"MTL-CEBPA has shown great promise in pre-clinical studies in liver disease models," commented Dr. Debashis Sarker, Principal Investigator of the National Institute for Health Research Biomedical Research Centre at Guy’s and St. Thomas’ and King’s College London, and chief investigator of the study. "We are looking forward to evaluating this highly innovative therapy in the upcoming Phase I trial. We hope MTL-CEBPA could represent an important new treatment option for patients with advanced liver cancer."

About the OUTREACH Study

OUTREACH is a first-in-human Phase I clinical study in patients with severe liver cancer. The multi-centre Phase I study will assess the safety and tolerability of MTL-CEBPA in patients with advanced primary or metastatic liver cancer who are ineligible or resistant to standard therapies. The study consists of a dose-escalation followed by a dose expansion. MTL-CEBPA will initially be administered as an intravenous infusion once weekly for three weeks followed by one week of rest. To learn more about the OUTREACH clinical study, please visit our listing at clinicaltrials.gov.

About MTL-CEBPA

MTL-CEBPA consists of a double stranded RNA formulated into a SMARTICLES liposomal nanoparticle and is designed to activate the CEBPA gene. The CEBPA gene encodes for the CCAAT/enhancer binding protein alpha (C/EBP-a), a transcription factor that acts as a master regulator of cell lineage determination and differentiation in several tissues including liver, myeloid cells and adipose tissue. In the liver, C/EBP-a plays an important role in normal hepatocyte function and response to injury. By restoring C/EBP-a expression to normal levels, MTL-CEBPA has been demonstrated to attenuate or reverse liver disease in a range of pre-clinical studies including models of liver cancer, liver cirrhosis, non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). In the future MiNA Therapeutics expects to initiate clinical trials of MTL-CEBPA in a number of diseases beyond liver cancer.

On June 2, 2016 Oxford BioMedica plc ("Oxford BioMedica" or "the Group") (LSE: OXB), a leading gene and cell therapy group, today announces that it has entered into a Research & Development Collaboration with Green Cross LabCell ("GCLC"), a subsidiary of Green Cross Holdings, a leading South Korean biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines (Press release, Oxford BioMedica, JUN 2, 2016, View Source [SID:1234512959]). The collaboration will focus on identifying and developing gene modified natural killer (NK) cell-based therapeutics for treatment of life-threatening diseases such as cancer.

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This collaboration brings together two clinically validated platform technologies with the aim of generating a pipeline of novel and differentiated cell-based products in areas of serious unmet medical need. Oxford BioMedica is contributing its clinically tested LentiVector gene delivery platform for the efficient modification of immune cells, as well as its expertise in GMP bioprocessing, clinical development and regulatory affairs in ex vivo cell & gene therapy. GCLC is contributing its clinically tested platform for production of highly potent and activated NK cells.

During the initial Research Collaboration, Oxford BioMedica and GCLC (the "Parties") will collaborate to identify potential NK cell-based product candidates, modified using lentiviral vectors. The collaboration will focus on allogeneic CAR-NK cell-based products. Under the terms of the agreement, Oxford BioMedica and GCLC will share the costs associated with the Research Collaboration equally. Oxford BioMedica’s contribution to the Research Collaboration will be funded from its existing Discovery resources and budget. The Parties will agree which product candidates from the Research Collaboration will be taken forward into the Development Collaboration stage of the programme on a product-by-product basis.

After nearly a decade of research on NK cells, GCLC has gathered a substantial body of evidence that their proprietary manufacturing platform for activated NK cells derived from peripheral blood mononuclear cells (PBMCs), such as MG4101, has potential to act as the basis for a pipeline of CAR-NK and other gene-modified NK cell products. By forming a partnership with GCLC, Oxford BioMedica gains access to one of the leading, clinically validated NK cell platforms in the industry and access to a highly skilled and experienced R&D partner in Korea, one of the most dynamic biotechnology markets in Asia.

Commenting on the new partnership, John Dawson, CEO of Oxford BioMedica, said: "We are very excited to form a key partnership with Green Cross LabCell, who have the industry-leading production platform for NK cell therapeutics for cancer. Over the years Green Cross LabCell has built a significant depth of scientific, technical and industrial expertise in NK cells that is difficult to find elsewhere.

"We are therefore delighted to be collaborating with Green Cross LabCell in a new partnership which brings our LentiVector delivery platform and expertise to the relationship and which builds on our existing partnerships with other companies including Novartis, Sanofi, GSK and Immune Design. We believe that our collaboration will lead to the generation of a rich pipeline of novel gene-modified NK cell therapeutics for both companies."

Bok-Soo Park, Chief Executive Officer of Green Cross LabCell, commented "To work with Oxford BioMedica as our partner for the development and future commercialization of gene-modified NK cell products is exciting, given their world-leading expertise in the clinical development and bioprocessing of lentiviral vector based products. This agreement underscores our commitment to maximise the value of our technologies and accelerate their progress to the market through partnering with industry leaders. We chose to work with Oxford BioMedica as we believe they are best positioned to accelerate development of these programs and are fully committed to their success. We look forward to working with the team at Oxford BioMedica to bring desperately needed new treatment options to cancer to patients all around the world."