Wednesday, 9 August 2017

Hemophilia Therapeutics Pipeline Analysis 2017 - Designation and Other Developments

The hemophilia therapeutics pipeline is expected to grow in future due to rise in prevalence of genetic variation. Hemophilia which is symptomatized by excessive bleeding in brains, joints, nose and mouth. Some of the drivers for the growth of the hemophilia therapeutics pipeline include increasing incidence of hemophilia, less number of drugs present for treatment of hemophilia.


Hemophilia is the disease, mostly inherited, in which people have little or no clotting factor. Clotting factor is required for normal blood clotting. These proteins work with platelets to promote the clotting of the blood. There are two types of hemophilia. Hemophilia A, in which clotting factor VIII is missing or present in very low level. Hemophilia B, in which clotting factor IX is missing or present in very low levels.

Many institutes or associations and companies are collaborating for developing therapeutics for hemophilia. Wyeth Pharmaceuticals is in research collaboration and license agreement with Nautilus Biotech SA for development of recombinant Factor IX proteins for treatment of hemophilia. Spark therapeutics is developing a late gene therapy in collaboration with Pfizer, Inc. Baxter International Inc. is collaborating with Chatham Therapeutics, LLC for the development and commercialization of treatment of haemophilia A.


Some of the key players having a pipeline of hemophilia therapeutics include Shire Plc, Biogen, Pfizer Inc., Novo Nordisk A/S, CSL Behring, F. Hoffmann-La Roche Ltd., Bayer AG, Biotest AG, Kedrion Biopharma Inc., and Octapharma AG.

Myocardial Infarction Therapeutics Pipeline Analysis 2017 - Collaboration, and Other Developments

The myocardial infarction therapeutics pipeline is expected to increase in future due to rise in prevalence of myocardial infarction. Myocardial infarction is also commonly known as heart attack which is symptomatized by decrease in blood supply to the heart muscle due to coronary artery disease. According to American Heart Association, the mortality rate due to cardiovascular disease is higher than other disease. According to British Heart Foundation, in 2015, around 150,155 deaths, occurred due to cardiovascular disease in the U.K. Some of the drivers for the growth of the myocardial infarction therapeutics pipeline include increasing incidence of coronary artery disease, change in lifestyle, lack of exercise, increase in incidence of blood pressure and obesity. According to World Health Organization (WHO), there has been an increase in incidence of heart disease in smokers as compared to non-smokers.


The myocardial infarction is a state of blockage of flow of oxygen-rich blood to a section of heart muscle, leading to the death of muscle. Myocardial infarction most often occurs in case of coronary artery disease in which a waxy substance, known as plaque gets build up inside the coronary artery, decreasing the oxygen-rich blood supply to the heart. This condition is also known as atherosclerosis due to which area of plaque break open inside a coronary artery, leading to the formation of clot. This clot gets bigger with time and leads to the blockage of coronary artery. The heart failure is a condition in which heart cannot pump enough blood to meet the body need. In various cases, a discomfort occurs in the center or left side of the chest which can last for few minutes which goes away and comes back. The upper body discomfort and shortness of breath also occurs in case of cardiac disorders.

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Some of the key players having a pipeline of myocardial infarction therapeutics include Pfizer Inc., Novartis N.V., Bristol-Myers Squibb Company, Daiichi Sankyo Company Limited, Boehringer Ingelheim GmbH, AstraZeneca, Apotex Inc., Par Pharmaceutical Companies, Inc., Sandoz, Mylan N.V.

Chronic Myelocytic Leukemia Therapeutics Pipeline Analysis - Clinical Trials & Results, Patent and Developments


Chronicmyelocytic leukemia therapeutic pipeline analysis is expected to grow rapidly due to the increasing prevalence of the disease, globally. Some of the main factors driving the pipeline analysis for chronic myelocytic leukemia include increasing aging population and rising prevalence of chronic myelocytic leukemia, and increasing health awareness. Chronic myelocytic leukemia have high incidence rate in men and is the second most common type of cancer occurring in male adults. Women, above 50 years of age are at high risk to develop chronic myelocytic leukemia.


Chronic myeloid leukemia is the tumour that occurs in blood cells and bone marrow, which is the soft parts inside bones where blood cells are produced. Chronic myelocytic leukemia usually occurs in middle-aged or older adults. It occurs due to the swapping of DNA between chromosome 9 nine and chromosome 22. The resultant abnormal chromosome is known as Philadelphia chromosome. This swapping forms a new gene, BCR-ABL which is the type of protein, tyrosine kinase. This protein causes chronic myelocytic leukemia cells to grow and reproduce uncontrollable manner. One of the main cause of chronic myelocytic leukemia is exposure to high radiation. The symptoms of chronic myelocytic leukemia include stroke, dizziness, change in vision, swollen glands, high fever, bruises, weight loss, pain in bones, short of breath and night sweats. Chronic myeloid leukemia can be diagnosed by performing a bone marrow test, complete blood count, FISH test and ultrasound. The treatment for chronic myeloid leukemia includes drug therapy and stem cell transplant.

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Some of the companies having a pipeline of chronic myelocytic leukemia therapeutics include Bristol Myers Squibb Company, ARIAD Pharmaceuticals, Inc., Novartis AG, Pfizer Inc., Teva Pharmaceutical Industries Ltd., Hospira, Prism Pharma, Incyte Corp., Otsuka Pharmaceutical Co., Ltd., Bio-Path Holdings, Hybrigenics Corporation, Fusion Pharma LLC.

Tuesday, 8 August 2017

Fabry Disease Therapeutics Pipeline Analysis 2017

Fabry disease is a type of inherited genetic disease caused due to abnormal deposition of a fatty substance, called globotriaosylcera-mide, in blood vessel walls throughout the body. This further leads to development of other complications, which result in severe morbidities and can also be fatal, in some cases. Anticipated growth of therapeutic pipeline of fabry disease can be attributed to the rareness of the disease, as it is usually found in males at an estimated ratio of one in 40,000 to 60,000 males. Although, the disorder occurs in females as well, its prevalence is still unknown. This disease can be cured using gene therapy and cell therapy, which are still in the development phase. With increasing support from various funding agencies such as National Organization of Rare Disorders (NORD), new and innovative therapies are being developed to cure the problem. Therefore, the therapeutic pipeline of Fabry disease is increasing.


In November 2016, FGK Clinical Research GmbH0 started Phase I, open-label, multi-center study to evaluate pharmacokinetics, pharmacodynamics, and safety of Moss-aGal in patients suffering from fabry disease. This study was completed in February 2017. Moss-aGal is a recombinant human alpha-galactosidase that is produced in moss. It is expected to be the first of its kind enzyme replacement therapy (ERT) for patients with the genetic lysosomal storage disorder, which exhibits optimized N-glycosylation patterns of the protein alpha-galactosidase A. In July 2016, Ozmosis Research Inc. started a Phase I clinical study on autologous stem cell transplantation of cluster of 34 positive (CD34+) cells engineered to express Alpha-galactosidase-A in patients with fabry disease. This study is expected to be completed in July 2022. In June 2016, Protalix Biotherapeutics Inc., started a Phase III trial for testing safety and efficacy of PRX-102, as compared to Agalsidase beta on renal function in patients with fabry disease previously treated with Agalsidase beta. PRX 102 demonstrated improved circulatory half-life, with better enzyme activity in target organs affected by fabry disease.

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Some of the companies having a pipeline of fabry disease therapeutics include Greenovation Biotech GmbH, GlaxoSmithKline Plc, Protalix Biotherapeutics, Inc., Amicus Therapeutics, Inc, Shire Plc, Genzyme Corporation, Ozmosis Research Inc.

Myasthenia Gravis Therapeutics Pipeline Analysis 2017 - Clinical Trials & Results and Developments

Myasthenia gravis is a neuromuscular disease that leads to varying degrees of skeletal muscle weakness. It is an autoimmune disease resulting from the antibodies blocking or destroying nicotinic acetylcholine receptors at the junction between the nerve and muscle, thus preventing nerve impulses rom triggering muscle contractions. Growth is anticipated in the myasthenia gravis therapeuticspipeline on account of increasing participation of organizations, such as National Organization for Rare Disorder, that support research and development activities in field. The unbearable complications such as difficulty in vision, swallowing, weakness in upper arms and legs and impaired speech are also contributing to the growth of pipeline therapeutics of myasthenia gravis.


In August 2005, Debiopharm S.A. entered into a collaboration with DeveloGen AG for development of PTR-262, a novel peptide drug for the treatment of myasthenia gravis (MG). PTR-262 is derived from the myasthenogenic epitopes of the acetylcholine receptor (AChR) alpha-subunit, which specifically arrests the autoimmune destruction of AChR. Under the terms of the agreement, DeveloGen AG would receive milestone payments and, upon commercialisation of the product, royalties based on revenues of Debiopharm S.A., from worldwide sales. Debiopharm S.A. is an independent company, specialising in oncology, endocrinology and niche products, whereas, DeveloGen AG is a biopharmaceutical company, which mainly focuses on development of novel treatments for metabolic disorders. In March, 2016, Penn centre for innovation had developed therapeutic vaccine for myasthenia gravis. The advantages of vaccine are long lasting benefits, potentially permanent protection, prevention as well as reversal of chronic myasthenia gravis, potent safe and rapid acting.

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Some of the companies having a pipeline of myasthenia gravis therapeutics include UCB Pharma, Ltd. Novartis AG, Grifols Therapeutics Inc., Bristol-Myers Squibb Company, Catalyst Pharmaceuticals, Inc., DeveloGen AG, Debiopharm S.A.

Wednesday, 2 August 2017

Chronic Kidney Disease Therapeutics Pipeline Analysis 2017 - Designation, Collaboration, and Other Developments

The therapeutics pipeline of chronic kidney disease has been increasing on account of rising prevalence of diabetes and high blood pressure in patients. The diabetes and blood pressure are the main causes of chronic kidney disease, which is also related to cardiovascular disease. Due to altered lifestyle, the chances of incidence of high blood pressure and diabetes is on a rise. Currently, the prevalence of chronic kidney disease is around 14% in Australia, as studied by Oxford Academic Journal of Nephrology Dialysis Transplantation. According to, National Institute of Diabetes and Digestive and Kidney Disease, the number of kidney failure cases among the Americans stood at more than 661,000, out of which 468,000 patients are undergoing dialysis and 193,000 patients are living with functional kidney transplant. The increasing awareness of healthcare issues also contributes towards the of development of therapeutics pipeline for chronic kidney disease.


Chronic kidney disease is the condition that can lead to the damage of the kidneys. Some of the main symptoms of chronic kidney disease are, weight loss, insomnia, tiredness, blood in urine, feeling sick, muscle cramps shortness of breath.

Many companies are conducting clinical trials for the development of therapeutics for chronic kidney disease. Astellas Pharma, Inc., is in the process of introducing a drug by the name ASP1517, which is in Phase III stage of its clinical development. ASP1517 is an orally administered small molecule which is used for the treatment of anaemia associated with chronic kidney disease. AstraZeneca Plc is also introducing a drug named, Dapagliflozin, which is currently in its Phase III stage of clnical development. Dapagliflozin is a sodium-glucose cotransporter II inhibitor.



Some of the companies having a pipeline of chronic kidney disease therapeutics include Astellas Pharma Inc., AstraZeneca Plc, Proteon Therapeutics, Inc., RegenMed (Cayman) Ltd, Gilead Sciences, Inc., GlaxoSmithKline Plc, Relypsa, Inc., Chugai Pharmaceutical, Inc., Kissei Pharmaceutical Co., Ltd, Boryung Pharmaceutical Co., Ltd.

Cushing's Syndrome Therapeutics Pipeline Analysis 2017 - Collaboration and Other Developments


The expending growth of pipeline for Cushing’s syndrome therapeutics is attributed to the increase in prevalence of inflammatory disorders. For the treatment of various inflammatory disorders patients use corticosteroids, the long-term use of corticosteroids can lead to Cushing’s syndrome. In many diseases such as asthma and rheumatoid arthritis, corticosteroids are being used, for a lifelong period but at low doses. However, when the disease fails to respond to lower doses of corticosteroids, the dose needs to be increased. The pipeline for Cushing’s syndrome is majorly driven by factors such as, increasing healthcare awareness in patients and the occurrence of abnormal growth of cells in pituitary gland which can further lead to the formation of tumour.



Cushing’s syndrome is a condition that occurs when a person is exposed to the hormone cortisol at a very high level, for a longer period of time, this is sometimes called as hypercortisolism. It mostly affects obese people at the aged between 20 to 50 years, who have type 2 diabetes and high blood pressure. The symptoms of the Cushing’s syndrome, include, round face, upper body obesity, deposition of fat around neck and relatively lean arms and legs. In some cases, skin of the patients become fragile, heals poorly, bruises easily and purple or pink marks appear on the abdomen, buttocks, thigh and breast. Bones become weakened and spinal and column fracture can occur when person is doing any routine activity like bending, lifting etc. Other symptoms include severe fatigue, increased thirst and urination, irritability, anxiety, depression and weak muscles.


In April 2012, Strongbridge Biopharma Plc was in the process of collaboration with Moulder Center Drug Discovery for the development of new therapies of cortisol modulation for Cushing syndrome therapeutics. In July 2014, Bristol-Myers Squibb Company and Ono Pharmaceutical Co., Ltd. announced that the companies are undergoing a strategic immuno-oncology collaboration in Japan, South Korea and Taiwan, which includes the development and commercialization of immune mediated endocrinopathies. In September 2008, Corcept Therapeutics and Eli Lilly and Company announced that the companies would continue the collaboration for testing the effectiveness of GRII receptor antagonist in rat models of olanzapine induced weight gain.

Some of the companies having a pipeline of Cushing’s syndrome therapeutics include Corcept Therapeutics, Millendo Therapeutics, Inc., Strongbridge Biopharma Plc, Novartis AG. Ono Pharmaceutical Co., Ltd. Bristol-Myers Squibb Company.