On January 4, 2019 BerGenBio ASA (OSE: BGBIO), reported that the first patient has been dosed in an investigator-initiated phase II trial of bemcentinib, a selective, potent and orally bio-available AXL inhibitor, in patients with high-risk myelodysplastic syndrome (MDS) who have failed first-line treatment with hypomethylating agents (Press release, BerGenBio, JAN 4, 2019, View Source [SID1234532440]). The trial may also enrol a proportion of patients with acute myeloid leukaemia (AML). The study is being sponsored by GWT-TUD GmbH (a specialist cancer clinical research institution associated with the University of Dresden, Germany) with the support of BerGenBio.

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The trial (BGBIL009 / BERGAMO) aims to confirm the efficacy of bemcentinib monotherapy in patients with high-risk MDS and AML and will enrol up to 43 patients at 8 hospitals in Germany, France, the Netherlands and Italy. The study will allow for the evaluation of potential predictive and pharmacodynamic biomarkers for MDS in bone marrow and blood, including those associated with patient benefit from bemcentinib.

Prof. Uwe Platzbecker, lead investigator of the trial and director of the Medical Clinic and Policlinic 1, Hematology and Cell Therapy at the University Hospital in Leipzig, Germany, commented: "As treatment of MDS and AML has not changed significantly over the past decades, novel therapies are urgently needed. The survival is still dismal, especially in elderly patients who are not eligible for allogeneic stem cell transplantation and who have failed first line treatment with hypomethylating agents. AXL, a member of the Tyro3, AXL, Mer (TAM) receptor family, mediates proliferation and survival of leukemic cells and is upregulated upon cytostatic treatment. Pre-clinical studies with the inhibitor bemcentinib demonstrated in vitro and in mouse models that leukaemic proliferation was blocked by interference with AXL signalling. Hence, AXL represents a promising new target for the patient population investigated in the BERGAMO trial."

Richard Godfrey, Chief Executive Officer of BerGenBio, added: "We are very pleased that Prof. Platzbecker and GWT-TUD are initiating this study, which if positive will add valuable information on bemcentinib’s monotherapy use in a larger leukaemia population and provide support for our broader development plans for bemcentinib in these indications. We look forward to reporting results as the trial progresses."

END

About MDS
Myelodysplastic syndromes (MDS) are stem cell disorders characterised by a decreased ability of the bone marrow to produce normal blood cells and platelets. MDS is associated with increased risk of developing AML and immune dysfunctions are seen in patients both with lower and higher-risk MDS. Drugs that modify immunological responses can improve blood values and prolong survival in some patients. Thus far, however, the only curative treatment for MDS remains stem cell transplantation. Hence, there is an urgent need for novel therapies to treat MDS.

About AXL
AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying aggressive and life-threatening diseases. In cancer, AXL drives tumour survival, treatment resistance and spread, as well as suppressing the body’s immune response to tumours. AXL expression has been established as a negative prognostic factor in many cancers. AXL inhibitors, therefore, have potential value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities.

About Investigator-sponsored trials
Investigator-sponsored clinical trials are clinical trials proposed by front-line patient-facing physicians who act as the regulatory sponsor and are supported by industry in bespoke clinical development partnerships. The industry partner does not assume the role of sponsor according to European or US regulatory guidelines but may offer support in a variety of different ways, such as providing investigational medicinal product at no cost

On January 4, 2019 Lamellar Biomedical Limited (Lamellar), an innovative biotechnology company, pioneering new approaches for the safe and effective transfer of functional nucleic acids, reported that Dr Alec McLean, CEO; Steven Porteous, Head of Clinical and Regulatory and Dr Lynsey Howard, Head of Pre-clinical Development will be attending Biotech Showcase in San Francisco on 7-9 January 2019 (Press release, Lamellar Biomedical, JAN 4, 2019, View Source [SID1234532438]).

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During the event the Lamellar team will conduct multiple business development meetings with potential partners and investors to highlight the potential of its cutting-edge LAMELLASOME technology for the delivery of nucleic acid therapeutics (NAT). These meetings will build on the very positive reaction to its LAMELLASOME platform, when it was introduced at BIO-Europe 2018 in Copenhagen in November 2018.

Alec McLean, CEO of Lamellar Biomedical, said, "We were very pleased with the reaction from potential partners and investors when we introduced our LAMELLASOME platform for the safe and effective delivery of nucleic acid therapeutics at BIO-Europe late last year. We are now looking forward to multiple new and follow up meetings in San Francisco reflecting the explosion in the number of novel nucleic-acid based therapeutics being developed for a broad range of diseases. Two key factors limiting their progress, from preclinical promise to clinical reality, are safe and effective delivery. Based on our work to-date, Lamellar is confident that it is in an unique position to unlock this enormous potential of nucleic acid based therapeutics using its LAMELLASOME technology."

Lamellar believes that its LAMELLASOME technology will play a key role in realising the potential of many of the nucleic acids in development, including mRNAs, siRNAs, miRNAs and plasmids, potentially revolutionising the treatment of numerous rare and intractable diseases.

In October 2018, Lamellar filed a patent detailing that it had demonstrated functional target downregulation by a transfected nucleic acid delivered via its LAMELLASOME technology. Lamellar has also shown that LAMELLASOME formulations have been effective in delivering functional nucleic acids to a range of cell types including macrophages, human pulmonary fibroblasts and human dendritic cells.

Crucially, all transfected cells types have exhibited very high cell viability and maintenance of cell phenotype/function. Lamellar’s LAMELLASOME technology is entirely novel and does not utilise viral vectors or cationic or divalent ion-associated liposomal systems.

Lamellar is also developing its own pipeline of nucleic-acid based therapeutics, the most advanced of which target two areas of unmet clinical need: Idiopathic Pulmonary Fibrosis and Cystic Fibrosis.

Lamellar is currently completing in-vivo models to demonstrate safe and effective pulmonary delivery and transfection of microRNA(s) prior to executing the final proof of concept efficacy study for its Idiopathic Pulmonary Fibrosis microRNA product in Q1 2019. These studies will be designed to show the performance of LAMELLASOME IPF-NA product in-vivo as well as to demonstrate the unique capabilities of LAMELLASOME nucleic acid transfer technology platform.

The Lamellar team will be available to present the proprietary LAMELLASOME technology to any companies attending Biotech Showcase who see enhanced delivery of their novel nucleic-acid based therapeutics as crucial to their future success.

On January 3, 2019 Avalon GloboCare Corp. (NASDAQ: AVCO), a leading global developer of cell-based technologies, reported that the Company and its wholly owned subsidiary, Avactis Biosciences, have entered into a joint venture and exclusive license agreement with Arbele Limited to co-develop next generation, transposon-based Chimeric Antigen Receptor (CAR)-T and CAR-Natural Killer (NK) cellular therapies (Press release, Avalon, JAN 3, 2019, View Source [SID1234609548]). These unique CAR vector constructs are non-virally engineered, possessing multiple therapeutic targets as well as unique "safety-switch" mechanisms. Based on Avalon’s extensive hospital network for cellular therapy, together with the Company’s established GMP bio-production facility in China (Epicon Biotech, Nanjing), this joint venture allows Avalon to accelerate the clinical development of more efficacious and safer CAR-T/CAR-NK therapies, such as those developed by Arbele.

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"We are very excited to establish this joint venture with Arbele Limited to accelerate our clinical programs in cellular therapy," stated David Jin, M.D., Ph.D., CEO and President of Avalon GloboCare Corp. "Arbele’s strong proprietary technology platform in designing and production of non-viral, transposon-engineered, multi-targeted CAR will allow us to generate next-generation, better and safer CAR-T and CAR-NK cellular therapeutics. This joint venture will strengthen our core technological capabilities, enrich our intellectual properties, as well as further establish our leadership in the field of cellular immunotherapy," added Dr. Jin.

On January 3, 2019 Immatics, a leading company in the field of cancer immunotherapy, reported that the United States Patent and Trademark Office has issued the 100th patent to Immatics (Press release, Immatics Biotechnologies, JAN 3, 2019, View Source [SID1234569547]).

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This is a significant achievement for the Company and demonstrates the breadth of Immatics’ innovation power and the quality of its science. Immatics’ strong patent portfolio enables the Company to develop its pipeline of cancer immunotherapies, to protect Immatics’ innovations and to safeguard the Company’s technology investments.

Immatics’ 100th patent (US Patent No 10,143,731) is directed against a peptide target discovered using the Company’s XPRESIDENT technology. The peptides identified by XPRESIDENT are presented by human leucocyte antigen (HLA) receptors on the surface of cells and are central to developing a range of novel powerful immunotherapies.

"Since its inception, Immatics has put a key focus on the development of its Intellectual Property estate, in order to protect our technology innovations in this highly competitive field", said Dr. Rainer Kramer, CBO of Immatics. "Being awarded the 100th issued US patent in the Life Science industry is fantastic, proving that our inventions move Immatics further to the forefront of the highly competitive field of Immunotherapy development."

Immatics’ patenting strategy ensures broad protection of the Company’s products, technologies and methods while still meeting the patentability requirements in the USA in a fast-paced environment.

On January 3, 2020 Kashiv Pharma, LLC, a pharmaceutical research and drug delivery company developing next-generation technologies, innovative medicines for patients with unmet medical needs and drug life-cycle management products, reported its acquisition of the assets of Adello Biologics, LLC, a biotechnology company developing biosimilars of leading global brands in the fields of oncology and immunology (Press release, Kashiv BioSciences, JAN 3, 2019, View Source [SID1234563292]). Kashiv will now be known as Kashiv BioSciences, LLC with its headquarters in Bridgewater, New Jersey plus research and development and manufacturing facilities in Bridgewater and Piscataway, New Jersey, Chicago, Illinois and Ahmedabad, India.

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Established in 2010, Kashiv Pharma focuses on innovative drug delivery systems and specialty drug products for the treatment of various debilitating diseases. The company seeks to partner with established pharmaceutical firms to commercialize products at an appropriate stage of development by offering novel products based on the next generation of advanced drug delivery platforms. Kashiv has created several proprietary technology platforms under the leadership of Dr. Navnit Shah, a world-renowned pharmaceutical scientist, for extended and time-specific oral delivery of small molecules including a novel gastric retention system (GRANDE) and a chronobiological delivery system (KRONOTEC). These advanced drug delivery systems target the drug to its optimal absorption site and at a time when the body needs it the most for maximal therapeutic effect.

Currently based in Piscataway and Chicago, Adello Biologics has been working to develop high-quality, affordable biosimilars for patients worldwide. The company is advancing a pipeline of complex proteins and monoclonal antibodies with lead candidates in oncology and immunology.

As a result of the acquisition, Kashiv BioSciences’ broad business offering includes drug delivery platforms incorporating delayed release technology and gastric retention systems that improve the efficacy and safety of known drugs; a 505(b)(2) pipeline of seven development products targeting unmet clinical needs; biosimilars; prodrugs; and, as part of a development collaboration with Amneal Pharmaceuticals LLC, 10 approved generic products and an additional 13 generic products in advanced stages of development, including several complex generic products. Kashiv BioSciences’ medications will improve the lives of patients suffering from cancer as well as neuromuscular, movement and endocrine disorders, among other debilitating conditions, both in the U.S. and globally.

"The acquisition of Adello will create a fully integrated biosciences company with a strong platform for growth," said Chintu Patel, who will become CEO of Kashiv BioSciences. "The combined breadth of expertise in small and large molecules, peptides, proteins and monoclonal antibodies will enable us to develop products with meaningful clinical differentiation and a compelling value proposition for patients, physicians and payers."