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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Drug Designations to Boost the Rhabdomyosarcoma (RMS) Therapeutics Development

Rhabdomyosarcoma (RMS) is a type of cancer that usually affects children. Soft tissues or hollow organs such as bladder or uterus are often affected by this disease. There are mainly two types of RMS: embryonal RMS (ERMS) and alveolar RMS (ARMS). Other types include anaplastic RMS and undifferentiated sarcoma. Depending on the type, RMS is likely to affect head and neck area, vagina, urinary bladder, uterus or testes, arms, and legs. There are certain risk factors associated with the disease, such as genetic disorders, including Li-Fraumeni syndrome, neurofibromatosis, Beckwith-Wiedemann syndrome, and Costello syndrome.

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People affected from RMS may face different signs and symptoms, some of which may not be seen until the tumor becomes large. The most common symptoms observed in 50% of the children with this disease include bleeding from nose, throat, vagina, or rectum (depending on the location of tumor); numbness, pain, or tingling if the tumor affects nerve areas; and loosening of eyelids (may indicate a tumor behind the eyes).

The diagnosis of the disease is reliant on features such as tumor size and where it starts. It can be detected by studying complete medical history of the patient, and imaging tests, such as X-ray, magnetic resonance imaging (MRI), computed tomography (CT) scan, bone scan, and others.

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RMS can be treated by surgery, chemotherapy, radiation therapy, and stem-cell transplantation. Moreover, Cosmegen (Ovation Pharmaceuticals Inc.) and VinCRIStine Sulfate (Hospira Inc.) are amongst the drugs approved by the United States Food and Drug Administration (USFDA).

According to the research findings, majority of pipeline drug candidates are being developed for intravenous route of administration. In this route of administration, entire administered dose reaches the systemic circulation immediately, which increases the bioavailability of drug.

Companies that are actively seeking designation grants to accelerate the process of development. For instance, Taiwan Liposome Company Ltd. was granted Pediatric Disease Designation for TLC178 drug by the USFDA in April 2017 for the treatment of RMS. The company also submitted an investigational new drug (IND) application in June 2018 to initiate a phase I/II clinical study of TLC178 to evaluate pediatric RMS patients.

Cosmegen and VinCRIStine Sulfate are approved drugs for the treatment of RMS. Moreover, with the emergence of late- and mid-stage pipeline products in the market, the overall RMS therapeutic market is expected to rise significantly in the upcoming years.

Taiwan Liposome Company Ltd., Ovation Pharmaceuticals Inc., Hospira Inc., F. Hoffmann-La Roche Ltd., Epizyme Inc., and Novartis AG are some of the significant players involved in the development of RMS therapeutics.

RMS 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 RMS. 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 RMS 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 RMS.

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.

p53 Antigen Modulators Therapeutics Pipeline Analysis 2018

p53 is the tumor suppressor transcription factor that activates to various stimulus, including uncontrolled cell proliferation, oncogene over-expression, and DNA damage. p53 antigen modulators helps in preventing cancer development through regulation of cell cycle and apoptosis.

Thus, new opportunities in cancer immunotherapy are expected to develop better drug candidates targeting p53, with complete understanding of interactions between p53 and the immune system, to avoid various adverse events. The major challenge is prevention of tissue damage by selectively modulating p53 activity, under pathophysiological conditions that generate redox stress.Thus, new opportunities in cancer immunotherapy are expected to develop better drug candidates targeting p53, with complete understanding of interactions between p53 and the immune system, to avoid various adverse events.

p53 antigen modulator therapies are being widely studied for the development of various target specific cancer therapies. Dendritic cell-derived vaccines, adenoviral p53 vectors, MDM2 inhibitors and small-molecules to reinstate the DNA binding activity of p53 are some commercial approaches as p53 antigen modulator therapies, for the treatment of various indications. Also, research studies have demonstrated that novel therapeutic strategies are being developed to overcome the challenges related to in-depth knowledge of p53 and associated pathways.

Quark Pharmaceuticals Inc. is developing QPI-1002 as a nuclease-resistant, synthetic double-stranded RNA oligonucleotide designed to temporarily inhibit the expression of the pro-apoptotic gene p53, for the treatment of delayed graft function and acute kidney injury. Some of the companies having a therapeutic pipeline of p53 antigen modulators include Innovation Pharmaceuticals Inc., Aprea Therapeutics, and Actavalon Inc.

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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, licencing, grants, technology and others.

Septicaemia Therapeutics Pipeline Analysis 2018

Septicemia, also known as bacteremia or blood poisoning, is characterized by bacterial blood infection. Septicemia occurs as a result of bacterial infection elsewhere in the body, which enters in the bloodstream.

Some common infections that lead to septicemia are urinary tract infections, lung infections, infections in the abdominal area, and kidney infections. The symptoms associated with this disease are fever, nausea and vomiting, reduced urine volume, shock rapid heart rate, very fast respiration, and chills.

Septicemia can lead to severe complications like sepsis, septic shock, and acute respiratory distress syndrome. ContraFect Corporation is in the process of developing Exebacase as a protein which acts as a cell wall modulator for the treatment of septicemia.

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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 Pontine Glioma Therapeutics

Pontine glioma is a malignant tumour which develops from the brain stem cells. Based on the grade of growth, it is categorized into anaplastic astrocytomas and glioblastoma multiforme (GBM).

Pontine glioma is more prevalent in children than adults. The common signs and symptoms of pontine glioma are squints, swallowing problems, slurred speech, facial weakness, abnormal gait, difficulty in writing and changes in personality and behaviour.

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The only efficient treatment for pontine glioma is radiotherapy; as surgery is not possible due to the tumor’s position in the brain stem. Company like Y-mAbs Therapeutics Inc. is in the process of developing omburtamab for the treatment of pontine glioma.

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