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VK2809 - Viking Therapeutics

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The Science VK2809 is a thyroid hormone receptor agonist that possesses selectivity for liver tissue, it is currently being developed by Viking Therapeutics for the treatment of metabolic disorders, including non-alcoholic steatohepatitis (NASH). Non-alcoholic fatty liver disease (NAFLD) is a condition in which excess fat is stored in your liver, it is an emerging global health problem and is a risk factor for Type 2 Diabetes and Cardiovascular Disease. Nonalcoholic steatohepatitis (NASH) is an advanced form of NAFLD in which you have inflammation and cell damage in the liver (Hepatitis), it is a predisposing factor for the development of cirrhosis and liver cancer. The development of NASH is very complicated, as seen in the figure above. Environmental, metabolic and genetic factors contribute to the development and progression of NASH by affecting diverse organs such as the liver, the intestine, and adipose tissue. In particular, excess caloric intake increases fre
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Ruxolitinib - Incyte Corporation

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The Science Ruxolitinib is a Janus-associated Kinase inhibitor with selectivity for JAK1 and JAK2 proteins, these are proteins that regulate the number of blood cells the body makes.  It has been developed and marketed by Incyte Corporation for the treatment of Myelofibrosis (Bone Marrow cancer) and a condition called Polycythemia Vera (uncontrolled production of blood cells). However it is currently undergoing investigation for the treatment of graft versus host disease (GVHD) in patients following bone marrow transplant. GVHD accounts for almost a third of deaths associated with bone marrow transplants. For patients with GVHD, there is almost no standard treatment, with currents treatments showing limited activity and response rates of 20-40%. There is a clear unmet need for a novel active treatment for GVHD. Patients with cancers that affect the blood or lymph nodes will often have bone marrow transplants as part of their treatment. Malignant cells need to be removed and d
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Rucaparib - Clovis Oncology

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The Science Rucaparib or Rubraca® is a PARP inhibitor currently being investigated for use as an anti-cancer agent by Clovis Oncology. It has been approved for the maintenance of Ovarian cancer, however it is also currently being investigated in three Phase III trials for the treatment of Ovarian, Prostate and Bladder cancers. Exploratory studies in other tumour types are also underway. Rucaparib is a potent inhibitor of PARPs or Poly (ADP-Ribose) Polymerase proteins, PARPs play a pivotal role in repairing DNA damage and maintaining genomic stability in cells. Cells can have certain genomic mutations such as BRCA1/2 or mutations in genes used for repairing DNA double strand breaks which would otherwise cause cell death. Tumours with these mutations must rely on alternative methods to repair DNA damage. PARP enzymes (particularly PARP1/2) play a critical role in repairing the DNA in cells that have these mutations. Inhibition of these PARP enzymes causes a build up of sing
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Andexxa - Portola Pharmaceuticals

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The Science Intravenous andexanet alfa, coagulation factor Xa (recombinant), inactivated-zhzo or Andexxa® is a first-in-class modified factor Xa protein that has been developed by Portola Pharmaceuticals as an antidote to reverse anticoagulant effects of direct or indirect factor Xa inhibitors.  In May 2018, Andexxa received FDA approval in the USA for use in patients that have been treated with rivaroxaban and apixaban, two factor Xa inhibitors. It is currently undergoing regulatory review for use in the EU, approval is expected in February 2019. Direct or Indirect factor Xa inhibitors are powerful anticoagulants for the prevention and treatment of thromboembolism and stroke prevention in atrial fibrillation. These agents have been shown to be more effective than vitamin K antagonists such as warfarin but nonetheless the risk of complications is still high and a reversing agent would be beneficial.  Andexxa is a first in class therapy for reversing the anticoagulat
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Tab Cel - Atara Biotherapeutics

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The Science Tab Cel, Tabelecleucel or allogeneic EBV-specific cytotoxic T lymphocytes ATL129 is a therapy that is currently being investigated by Atara Biotherapeutics for patients with EBV+ PTLD After Failure of Rituximab.  EBV or Epstein-Barr Virus is a human gamma herpes virus, more commonly know as the virus to cause Glandular Fever. This virus asymptomatically infects over 95% of adults by age 30. Infection typically occurs in childhood and is often difficult to distinguish from a cold or flu. The virus establishes a latent infection with B-memory cells and lies dormant in the body in very low numbers.  Patients with malignant bone marrow disorders and other disorders of the immune system often are treated by allogenic stem cell transplantation. This involves high doses of chemo/radiotherapy to destroy the patients immune system followed by a replacement of bone marrow stem cells. As the patients immune system is compromised during the procedure, latent EBV infection
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AMG 420 - Amgen

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In my previous post about BB2121, I noted that AMG 420 seemed more effective scientifically, and more practical from a cost point of view as compared to BB2121. I decided to explore that a bit further. The Science AMG 420 is an anti-BCMA x anti-CD3 BiTE (Bi-specific T cell engager) antibody construct currently being investigated as a treatment for multiple myeloma by Amgen.  Multiple myeloma is a cancer that originates in plasma cells, a type of white blood cell. It can impact much of the body but especially target the bone marrow where plasma cells are made. Multiple myeloma cells produce a protein called B-cell maturation antigen (BCMA) in excess. The immune system usually can recognise abnormal cells, such as cancer cells, and uses specific immune cells, such as T-cells, to destroy them. However, some tumour cells develop mechanisms to evade detection by the immune system. AMG 420 is an immunotherapy, meaning that it does not directly target the tumour but aims
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Liso-Cel

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Liso-Cel The Science JCAR017, Lisocabtagen maraleucel or Liso-Cel for short is a CAR T-cell therapy currently being developed by Juno Therapeutics, in collaboration with Celgene. The therapy has been granted blockbuster drug status to possibly treat certain patients with aggressive B-cell non-Hodgkin’s lymphoma (NHL). NHL is a collective name for a group of closely related blood cancers that develop in the lymphatic system. Clear fluid called lymph flows through the lymphatic vessels and contains infection-fighting white blood cells known as lymphocytes. In non-Hodgkin lymphoma, the affected lymphocytes start to multiply in an abnormal way and begin to collect in certain parts of the lymphatic system, such as the lymph nodes (glands). In B-cell lymphomas, it is B-cells that multiply uncontrollably and loss normal function. B-cells function in the humoral immunity component of the adaptive immune system by secreting antibodies, presenting antigens and secreting cytokines.
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