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.

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.

Atypical Hemolytic Uremic Syndrome (aHUS) Therapeutics – Pipeline Analysis 2018

Atypical hemolytic uremic syndrome (aHUS), also known as non-diarrhea – associated hemolytic uremic syndrome, is a rare disease characterized by hemolytic anemia, thrombocytopenia, acute kidney failure and uremia.

The signs and symptoms of aHUS include formation of tiny blood clots in various small blood vessels of the body. These clots reduce or prevent proper blood flow to various organs of the body, especially the kidneys. aHUS is a complex disorder; and certain genetic, environmental and immunologic factors plays significant role in its development.

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Company lile ChemoCentryx Inc. is in the process of developing avacopan, an orally-administered drug candidate, that inhibits the complement C5a receptor for the treatment of aHUS. Omeros Corporation, Greenovation Biotech GmbH, and Laboratoris Sanifit S.L. are some other companies developing therapeutic agents for aHUS.

Aurora Kinase Inhibitor Therapeutics Pipeline Analysis

Aurora kinase are proteins that function as mitotic regulators and are aberrantly expressed in cancerous cells. Aurora kinase inhibitors target these aberrantly expressed regulators, provides genetic stability and prevents tumorigenesis.

Several studies have demonstrated overexpression and amplification of aurora kinase in various haematological and solid cancers. Aurora kinase inhibitors when combined with other anti-cancer agents gives more positive results in variety of cancers. Better evaluation of aurora kinase inhibitors is required which raises the need of biomarkers, effective and novel therapeutic agents. Also, aurora kinase inhibitors selectivity targets cancer cells rather than healthy tissues, providing potential opportunities for more effective drug development with reduced adverse effects.

AstraZeneca PLC is in the process of developing AZD-2811 as a small molecule which acts as an aurora B inhibitor for the treatment of solid tumors. Some of the other companies having pipeline of aurora kinase inhibitors include AbbVie Inc., Merck & Co., Inc., and CASI Pharmaceuticals 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.

Hospital Acquired Pneumonia (HAP) Therapeutics

Hospital acquired pneumonia (HAP) is a lung infection that occurs during hospital stay. HAP can spread via health care workers, who may pass germs from their hands or clothes from one patient to another.

This is why hand-washing, wearing gowns, and using other safety measures is so important in hospitals and other healthcare settings. It has been observed that HAP may develop at least 48 hours after hospital admission.

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There are several risk factors associated with HAP including alcohol consumption, major surgery, weak immune system, chronic lung disease, and swallowing problems.

The disease can be treated with antibiotics and can be controlled by taking hygiene prevention during a hospital stay. Arsanis Inc. is in the process of developing ASN100 as a monoclonal antibody for the treatment of HAP. Some of the companies involved in HAP pipeline are Allecra Therapeutics GmbH and Nabriva Therapeutics plc among others.

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