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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