Systemic Lupus Erythematosus Therapeutics – Pipeline Analysis, Clinical Trials and Results, Patents

Systemic lupus erythematosus (SLE) is a chronic disease that causes inflammation of cartilage and blood vessels. Pain and swelling in joints, sensitivity to sun, unexplained fever, joints and muscle pain, mouth ulcers, swollen glands, and others are some common symptoms of the disease. Additionally, kidneys, lungs, heart, and others can also get affected by SLE. The diagnosis of SLE is little difficult as it requires multiple tests and constant analysis. Anticardiolipin antibody test, antinuclear antibody (ANA) test, blood chemistries, complete blood count (CBC), urinalysis, and the erythrocyte sedimentation rate (ESR) are some tests to diagnose the disease.

Companies that are developing therapeutics for SLE have shown positive clinical results in the various phases of drug development. For instance, in March 2018, Ablynx announced topline results of vobarilizumab phase II trials. Treatment-related serious adverse events were reported in 2.0% of all vobarilizumab treated patients as compared to 6.5% in patients administered with placebo. The percentage of patients experiencing a serious infection was also lower in the vobarilizumab arms compared to the placebo arm (2.8% versus 6.5%). Treatment-emergent adverse events that led to study drug discontinuation were reported in 12.4% of all vobarilizumab treated patients.

Pharmaceutical companies are tending to develop various strategies to enhance the therapeutic pipeline of SLE. For instance, Ablynx is developing vobarilizumab for the treatment of vobarilizumab with the help of its proprietary Nanobody technology.

Takeda Pharmaceutical Company Limited, Anthera Pharmaceuticals Inc., Genentech Inc., UCB Biopharma SPRL, Merck & Co. Inc., Ablynx, and GlaxoSmithKline plc are some of the prominent players for developing drugs and therapies for SLE.

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Niemann-Pick Disease Type C NPC Therapeutics Pipeline Analysis, Clinical Trials and Results, Patents

 Niemann-Pick disease is divided into four main types: type A, type B, type C1, and type C2. These types are classified on the basis of genetic cause and signs and symptoms of the disease. NPC is identified as a rare genetic disorder in which the body is unable to transport cholesterol and lipids inside the cells. This causes accumulation of these substances within various tissues of the body. These substances when gets collected in the brain tissues, it affects neurologic and psychiatric function.

The disease shows its first signs in mild-to-late childhood with the insidious onset of ataxia, dementia, and vertical supranuclear gaze palsy (VSGP). The occurrence of NPC can be confirmed by biochemical testing. NPC can also be diagnosed by skin test, in order to assess how the cells, move and store cholesterol. Furthermore, testing for the cells’ ability to modify cholesterol (cholesterol esterification test) has also been used for diagnostic purpose. Recently, blood-based testing for biomarkers (oxysterols, lysosphingolipids, bile acid metabolites) and molecular gene sequencing of NPC1 and NPC2 have replaced these methods.

It has been observed that regulatory bodies are granting designations to the pipeline drugs, in order to pace up the development process of these drugs. For instance, IntraBio Inc.’s IB1001 was granted Rare Pediatric Disease Designation by the USFDA in April 2018 and Orphan Drug Designation by USFDA and EMA in February 2018 and April 2017, respectively. Similarly, Orphazyme A/S’s arimoclomol has been granted Orphan Drug Designation (in January 2015), Fast-Track Designation (in June 2016), and Rare Pediatric Disease Designation (in January 2018) by the USFDA.

Some of the key players involved in the development of NPC therapeutics are Orphazyme A/S, IntraBio Inc., CTD Holdings Inc., Mallinckrodt plc, SOM INNOVATION BIOTECH SL, Neurotrope Inc., Actelion Pharmaceuticals Ltd., Okklo Life Sciences BV, Amicus Therapeutics Inc., Oraxion Therapeutics Inc., and COI Pharmaceuticals Inc.

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Wnt Signaling Pathway Inhibitors - Pipeline Analysis, Clinical Trials & Results

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.

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.

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.

It has been observed that many pharmaceutical companies are leveraging new and improved technologies for the development of Wnt Signaling Pathway inhibitors. These technologies play an important role in the drug development thereby helping the companies develop more specific and effective targeted therapies. For instance, the focus of Samumed's technology platform is on the ability to modulate the Wnt signaling pathway in selected tissues with small molecules. 

The company has discovered various new biological processes related to the Wnt signaling pathway, identified novel biological targets, and developed small-molecule drugs that modulate Wnt activity. In addition, JW Pharmaceutical CORPORATION is engaged in the development of novel therapeutics using high-throughput screening technology and chemical library. The company specializes in identifying small molecule drugs that modulate signaling pathways involved in cell proliferation and differentiation.

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Akt/Protein Kinase B (PKB) Inhibitors - Analysis by Stages, Drug Class, Company Profile, & Discovery Stage Insights

Akt/Protein Kinase B (PKB) Inhibitors - Pipeline Analysis, Clinical Trials & Results, Patents, Designations, Collaborations, and Other Developments published by Pharma Proff Akt/PKB inhibitors therapeutics currently exhibits a proliferating pipeline with 23 therapeutic candidates. 

Akt plays a critical role in the regulation of cell’s apoptotic pathway and it is being studied extensively for targeting cancerous cells. Akt inhibitors are therapeutics that obstruct the Akt pathways or Akt enzyme, which play a key role in proliferation of solid tumors or cancer cells. Akt are composed of various AGC kinases present in the cells that have hydrophobic motif at the c-terminus of their catalytic core.

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; less risk of systemic infections; and has inexpensive nature. Akt inhibitors through oral route of administration have shown positive results in the clinical studies.

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The companies who are developing Akt/PKB inhibitors for the treatment of various chronic diseases have shown positive clinical results in the various phases of drug development. For instance, in June 2018, F. Hoffman-La Roche Ltd. announced the interim results of Phase II study (LOTUS) for the drug candidate, ipatasertib, that showed improvement in patients with triple negative breast cancer (TNBC) toward overall survival (OS). This result was presented in the 2018 ASCO Annual Meeting, held in the U.S. The Phase II LOTUS study showed an increase in OS up to 23.1 months (median) in interventional study, as compared to 18.4 months (median) in placebo study.

  

Some of the key players involved in the development of Akt/PKB inhibitors include F. Hoffman-La-Roche Ltd, Eli Lilly & Company, Merck & Co. Inc., Novartis AG, Denovo Biopharma LLC, Diffusion Pharmaceuticals Inc., Astex Pharmaceuticals, ArQule Inc., Cotinga Pharmaceuticals Inc., and Oneness Biotech Co. Ltd.

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