Adenosine 5-Monophosphate Activated Protein Kinase (AMPK) Modulators Therapeutics Pipeline Analysis 2018

Adenosine 5-monophosphate activated protein kinase (AMPK) plays an important role in regulation of homeostasis. Dysregulation of AMPK can result in many diseases which includes obesity and type 2 diabetes. Several studies have also suggested that AMPK are also associated with the development of neurological diseases and cancer.

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Despite of many advancements in the development of anti-cancer therapies, cancer is still one of the major causes of deaths, globally. AMPK is one of the newest targets for the anti-cancer therapies, which modulates cellular protein and lipid metabolism, affecting the cellular growth and division. The importance of AMPK mediate signalling in various diseases and its complexity suggested that there is an urgent need for the development of additional AMPK modulators that can be used to not only disturb the mechanism of action but also as a drug candidate for therapeutic development. Also, researches have demonstrated that, in-depth knowledge and evidences are required to produce desired physiological outcomes by developing specific targeted therapies with reduced adverse events and better efficacy.

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Esperion Therapeutics Inc. is in the process of developing Bempedoic acid as a small molecule which acts as an AMPK stimulant for the treatment of hypercholesterolemia, dyslipidaemia, and hypertension. Betagenon AB, Boehringer Ingelheim International GmbH, and Poxel SA are some other companies having pipeline AMPK modulators for the management of several diseases.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with detailed analysis of pipeline and clinical trials.
Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology and others.

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Retinitis Pigmentosa (RP) Therapeutics Pipeline to Witness Remarkable Growth due to Increased Number of Pipeline Candidates by Drug Developers

Retinitis pigmentosa (RP) is a group of hereditary disorders that lead to the breakdown and loss of cells in the retina. Mutation in genes encoding the proteins required by retinal cells for their proper functioning, results in occurrence of RP. The disease results in partial vision loss during the earlier stage of the disease, which may lead to complete blindness with the progression of the disease. Some of patient-reported side effects include night visual impairment, loss of the visual field, and trouble in differentiating colors.

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The aim of diagnosing RP is to determine the type of mutation that has occurred in genes and can be detected by performing tests, including electroretinogram (ERG), visual field testing, and genetic testing.

The available treatment options include retinal implant, increased intake of vitamin A palmitate, and acetazolamide medication. Furthermore, Luxturna (Spark Therapeutics Inc.) is the only approved therapy by the U.S. Food and Drug Administration (USFDA) for the treatment of RP.

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According to the research findings, majority of the pipeline drug candidates are being developed for subretinal administration. It has been observed that this route of administration has high bioavailability and ensures direct effect on targeted cells.

Positive Clinical Results are Expected to Drive the RP Therapeutics Pipeline

Companies that are involved in developing therapeutics for RP have shown positive clinical results in various phases of drug development. For instance, in December 2018, Applied Genetic Technologies Corporation announced positive top-line results from the phase I trial of XLRS gene therapy for the treatment of patients with RP. The results of the study exhibited that 2 of 13 adult patients treated at high dose showed some improvements in retinal structure as measured by optical coherence tomography (OCT), an imaging technology that provides a side view of retina’s layers.

Companies are actively seeking designation grants to accelerate the process of development. For instance, HORA – PDE6B of Horama, was granted Orphan Drug Designation by the USFDA for the treatment of RP in July 2017.

Luxturna is the only approved therapy for the treatment of RP. Therefore, with the emergence of late- and mid-stage pipeline products in the market, the overall RP therapeutic market is expected to rise significantly in the upcoming years.

Acucela Inc., HORAMA S.A., GenSight Biologics S.A., Novartis AG, Allergan Inc., Spark Therapeutics Inc., Sanofi-aventis Groupe, Ocugen Inc., Applied Genetic Technologies Corporation, Eyevensys, MeiraGTx Holdings plc, and Biogen Inc. are some of the key players involved in the development of drugs indicated for the treatment of RP.

RP Therapeutics Pipeline Analysis

By Phase
By Molecule Type
By Route of Administration
By Company

The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of RP. Comprehensive insights on the pipeline products have been provided, with special focus on strategic developments of key players, information on drug licensing, designations, financing, and grants, technological advancements, patents, and upcoming conferences. In addition, the report highlights the winning strategies of companies involved in the RP therapeutics development. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in this report. Furthermore, the report contains competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanisms of action, and drug origin with respect to RP.

Wnt Signaling Pathway Inhibitors Pipeline: Do You Really Need It? This Will Help You Decide!

Wnt signaling pathway plays an important role in embryonic development. The signaling pathway regulates cell-to-cell interaction, accelerates trophoblast development, activates blastocyst, accelerate chorion-allantois fusion and implantation. A wide range of diseases, such as different types of cancer and degenerative diseases can be treated by deregulation of components involved in Wnt/β-catenin signaling pathway. Also, researchers have demonstrated that moderate weakening of Wnt signaling can eliminate its carcinogenic potential. Based on the recent researches, Wnt Signaling Pathway inhibitors have also shown potential in cardiovascular disorders.

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According to a new research report “Wnt Signaling Pathway Inhibitors – Pipeline Analysis 2018, Clinical Trials & Results, Patents, Designations, Collaborations, and Other Developments” published by Pharma Proff, Wnt Signaling Pathway Inhibitors therapeutics currently exhibits a proliferating pipeline with 27 therapeutic candidates.

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According to the research findings, majority of the drug candidates in the pipeline are being developed to be administered by the oral route. It has been observed that oral route of medications is convenient; available in delayed or rapid release formulation; provides improved patient’s compliance; and has less risk of systemic infections. Administration of Wnt Signaling Pathway Inhibitors through oral route has shown promising results in the clinical studies. Also, according to the analysis, maximum number of drugs in the pipeline are being developed as small molecules.

Positive Clinical Trial Results are Expected to Drive the Advancements in Wnt Signaling Pathway Inhibitors Pipeline

The companies developing Wnt Signaling Pathway inhibitors for the treatment of cancer and degenerative diseases, have shown positive clinical results in the various phases of drug development. For instance, in October 2018, Samumed LLC, announced the topline data from the phase IIb trial SM04690 in knee osteoarthritis, wherein the treatment with SM04690 showed improvement in function, pain, and global scores of patients.

Some of the key players involved in the development of Wnt signaling pathway inhibitors in the late and mid stage include Samumed LLC, Can-Fite BioPharma Ltd., Tactical Therapeutics Inc., Leap Therapeutics Inc., 2X Oncology Inc., Eisai Co. Ltd., and Novartis AG.

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Therapeutics Pipeline Analysis 2018

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a progressive disorder that primarily affects brain and spinal cord (central nervous system). People with MPS III generally do not display features at birth, but they begin to show signs and symptoms of this disorder during early childhood. Affected children often initially have delayed speech and behaviour problems.

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In later stages of this disorder, people with MPS III may develop seizures and movement disorders. Patients with MPS III are also known to have short stature, joint stiffness, or mild dysostosis multiplex. Affected individuals often experience chronic diarrhoea and recurrent upper respiratory and ear infections. People with MPS III may also have hearing loss and vision problems. MPS III is divided into four types; IIIA, IIIB, IIIC, and IIID, which are distinguished by their genetic cause.

Get the detailed analysis @ https://www.pharmaproff.com/report/mps-iiitherapeutics-pipeline-analysis The drug candidates in MPS III pipeline include, but are not limited to, EGT-101, MPS IIIA (Sanfilippo A) Program and LYS-SAF302. Some companies having drugs in the MPS III pipeline are Esteve Pharmaceuticals S.A., Orchard Therapeutics Limited, Lysogene S.A. among others.

Myelodysplastic Syndromes (MDS) Epidemiology Insights

Myelodysplastic syndromes (MDS) can be described as a group of hematologic malignancies, that occurr due to abnormality in blood-forming cells in the bone marrow. It is a rare blood cancer and can be mild, moderate or severe, in terms of severity.

This syndrome can be classified into three main types: MDS with single lineage dysplasia wherein a single type of blood cell appears abnormal; multilineage dysplasia causing two or more cell types to appear abnormal; and MDS with excess blasts, wherein the patients have a shortage of either red blood cells, white blood cells or platelets. Some prominent risk factors that increase the risk of myelodysplastic syndromes are aging, exposure to industrial chemicals, chemo- or radiation therapy, and smoking.

MDS is an uncommon syndrome with an incidence of 4–5 patients per 100,000 populations. It increases with age and can reach up to 20–50 per 100,000 population, in patients aged over 60 years. According to a 2019 study, the annual incidence of pediatric MDS is approximately 1–4 cases/million, which accounts for less than 5% of childhood hematological malignancies.

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Pipeline Analysis of Hormone Sensitive Breast Cancer Therapeutics

Breast cancer generally occurs when breast cells grow uncontrollably. Mostly, breast cancer development starts in the duct that carries milk to the nipples. Breast cancer generally occurs when breast cells grow uncontrollably. Mostly, breast cancer development starts in the duct that carries milk to the nipples.

Some breast cancer cells develop in milk producing glands. As per the National Institutes of Health (NIH), in the U.S., 14.6% of all new cancer cases diagnosed every year are of breast cancer. Also, as per the American Cancer Society (ACS), 1 in every 5 woman has cancer cell in their breast tumour with several growth promoting protein receptors on their surface. HER2 (human epidermal growth factor receptor 2) gene plays significant role in the development of breast cancer in most of the cases. Alpelisib is one of the key pipeline drug candidates for hormone sensitive breast cancer pipeline.

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Pfizer Inc., and Novartis AG are major players involved in the development of drugs for hormone sensitive breast cancer. Get the Clinical Trials & Results Report of Hormone Sensitive Breast Cancer Therapeutics at https://www.pharmaproff.com/request-sample/1114

Sezary Syndrome Therapeutics Pipeline Analysis 2018

Sezary syndrome, a type of cutaneous T-cell lymphoma, affects Sézary cells present in the skin, lymph nodes and blood. It accounts approximately 3 to 5 percent of cases of cutaneous T-cell lymphoma.

Sézary syndrome is more prevalent in males compared to females. It is characterized by red, severely itchy rash that covers large areas of the body. The common symptoms of Sézary syndrome includes alopecia, lymphadenopathy, palmoplantar keratoderma, ectropion and abnormalities of the fingernails and toenails.

The standard treatments for Sézary syndrome are combination of phototherapy and chemotherapy, and medications including vorinostat and romidepsin. Takeda Pharmaceutical Company Limited is in the process of developing brentuximab vedotin for the treatment of Sézary syndrome.

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