On November 27, 2017 STORM Therapeutics, the leading drug discovery company focused on the discovery of small molecule therapies modulating RNA epigenetics, reported the publication of data in the internationally renowned scientific journal Nature linking an essential RNA-modifying enzyme to acute myeloid leukaemia (AML) (Press release, STORM Therapeutics, NOV 27, 2017, View Source [SID1234561048]).

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The study has found an unexpected new drug target for acute myeloid leukaemia (AML) that could open new avenues to develop effective treatments against this potentially lethal disease. Data show that inhibiting the METTL3 gene destroys human and mouse AML cells without harming nonleukaemic blood cells. The paper, entitled "Promoter-bound METTL3 maintains myeloid leukaemia via m6A-dependent translation control’, goes on to reveal why METTL3 is required for AML cell survival, by deciphering the new mechanism it uses to regulate several other leukaemia genes.

Professor Tony Kouzarides, Founder of STORM Therapeutics and joint project leader from the Gurdon Institute, University of Cambridge, commented: "This is an important milestone in the understanding of RNA epigenetics and its links to disease. These findings highlight the importance of RNA modifying enzymes in cancer, and in particular in leukaemia. New treatments for AML are desperately needed and we have been looking for novel genes that would be good drug targets. We identified the methyl transferase enzyme METTL3 as a highly viable target against AML. Our study will inspire pharmaceutical efforts to find drugs that specifically inhibit METTL3 to treat AML."

STORM has established a pipeline of drug discovery programmes to develop novel, first-in-class drugs for the treatment of specific cancers and other diseases with high unmet medical need. It is focusing on two classes of RNA modifying enzymes, RNA methyltransferases and terminal uridyltransferases (TUTases), and has already advanced two undisclosed targets in drug discovery.

Keith Blundy, CEO of STORM Therapeutics, said: "STORM leads the field of harnessing the power of RNA epigenetics as a new area of important biology. Our ambition is to become a world leading therapeutics company tackling diseases through modulating RNA modifying enzymes. Publication of these data, in such a prestigious journal as Nature, is validation of the world class science on which the Company was founded."

This publication represents results obtained from research conducted in collaboration by: Wellcome Trust Sanger Institute, The Gurdon Institute and Department of Pathology at the University of Cambridge, Cold Spring Harbor Laboratory and STORM Therapeutics.

On November 27, 2017 AstraZeneca reported a strategic joint venture with the Chinese Future Industry Investment Fund (FIIF) to form an equally-owned, stand-alone company in China to discover, develop and commercialise potential new medicines to help meet unmet needs globally, and to bring innovative new medicines to patients in China faster (Press release, AstraZeneca, NOV 27, 2017, View Source [SID1234537458]). FIIF is managed by the SDIC Fund Management Company (SDIC Fund), a private equity management company.

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The new company, Dizal Pharmaceutical, incorporates all scientific and technical capabilities of AstraZeneca’s Innovation Center China (ICC), and holds exclusive rights to develop and commercialise three potential medicines currently in pre-clinical development from AstraZeneca’s pipeline in its main therapy areas of oncology, cardiovascular and metabolic diseases, and respiratory. It is also expected to initiate novel clinical programmes. The FIIF will contribute funding and expertise in establishing strategic partnerships in China.

Dr. Xiaolin Zhang, previously Head of AstraZeneca’s ICC, has been named as Chief Executive Officer of the new company. All staff employed by the ICC have been invited to join the new company.

Pascal Soriot, Chief Executive Officer of AstraZeneca, said: "AstraZeneca has a long-standing and strong commitment to China, which we are reinforcing today with this ground-breaking joint venture. By joining forces with the FIIF, we aim to accelerate the local discovery and development of innovative, affordable medicines for patients in China and around the world."

Guohua Gao, Chairman of SDIC Fund, said: "FIIF is delighted to be collaborating with AstraZeneca to promote the development of innovative medicines. AstraZeneca’s Innovation Center China has an excellent track record of drug discovery, and the synergy created by combining AstraZeneca’s scientific talent and assets with FIIF’s China expertise and funding will help further promote innovation in medical science."

The remit of the FIIF in the pharmaceutical industry is to promote the development and manufacturing of innovative medicines in China through strategic partnerships. The joint venture supports AstraZeneca’s commitment to enhancing China’s research and development capabilities through diversified external partnerships that deliver value to patients in China.

Genprex has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Genprex, 2018, NOV 27, 2017, View Source [SID1234527530]).

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Varian Medical Systems has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Varian Medical Systems, 2017, NOV 27, 2017, View Source [SID1234522258]).

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On November 27, 2017 Medigene AG (FSE: MDG1, Prime Standard, TecDAX) reported that all planned patients have been enrolled in the Company’s ongoing Phase I/II study with dendritic cell (DC) vaccines in acute myeloid leukaemia (AML) (Press release, MediGene, NOV 27, 2017, https://www.medigene.com/investors-media/press-releases/detail/article/patient-recruitment-completed-for-phase-iii-dc-vaccine-trial-in-aml/ [SID1234522264]). The completion of the study, as previously announced, is expected in 2019 after a treatment period of two years for all patients.

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Medigene’s Phase I/II trial (NCT02405338) includes 20 AML patients who show complete remission after standard chemotherapy, but who are not eligible for stem cell transplantation that would reduce the risk of a relapse. All patients will be vaccinated with Medigene’s DC vaccines for two years. The primary objective is to assess safety and feasibility of the active immunotherapy with Medigene’s dendritic cells. Secondary objectives of the study are induction of immune responses, overall survival (OS), progression free survival (PFS), control of minimal residual disease (MRD) and time to progression (TTP).

Dr. Kai Pinkernell, SVP Clinical Affairs and Chief Medical Officer of Medigene AG, comments: "With this study we aim to improve the outcome of the patients and show that vaccination with dendritic cells can help to control AML, a leukemia with normally high relapse rates. We are glad to announce the complete enrollment of our phase I/II trial as projected, which puts us on track for a study end in 2019. We intend to provide preliminary data on certain aspects of the trial at scientific conferences once a large part of the patients has been treated for more than a year."

About Medigene’s DC vaccines: The platform for the development of antigen-tailored DC vaccines is the most advanced of Medigene’s highly innovative and complementary immunotherapy platforms. Currently Medigene evaluates its DC vaccines in a company-sponsored Phase I/II clinical trial in acute myeloid leukaemia (AML).

Dendritic cells (DCs) are the most potent antigen-presenting cells of our immune system. Their task is to take up, process and present antigens on their cell surface, which enables them to activate antigen-specific T cells for maturation and proliferation. This way T cells can recognize and eliminate antigen-bearing tumor cells. Dendritic cells can also induce natural killer cells (NK cells) to attack tumor cells. The team of Medigene Immunotherapies scientists has developed new, fast and effective methods for generating dendritic cells ex-vivo, which are able to activate both T cells and NK cells. The DC vaccines are developed from autologous (patient-derived) precursor cells, isolated from the patient’s blood, and can be loaded with tumor-specific antigens to treat different types of cancer. Medigene’s DC vaccines are in development for the treatment of minimal residual disease or for use in combination therapies.

About acute myeloid leukaemia (AML): Acute myeloid leukaemia is a malignant disease of the hematopoietic system, affecting mainly adults above 60 years of age. In Germany, about 3,600 incidences are registered annually.
AML is caused by uncontrolled growth of dysfunctional hematopoietic precursor cells in the bone marrow. These cells prevent the generation of normal blood cells, causing a drop in erythrocytes and platelets, for example. Typical symptoms of AML include anemia, fever, increased risk of infection, and blood coagulation disorder. AML progresses rapidly and may be fatal within a few weeks if untreated.
AML is treated initially with intensive chemotherapy. Another treatment option is allogeneic hematopoietic stem cell transplantation. Unfortunately, the majority of patients suffer a relapse. Only about 15 – 20% of the patients show long-term remission after conventional chemotherapy. Allogeneic hematopoietic stem cell transplantation is the only treatment option that offers a more positive prognosis.