Bayer Decision to Advance IONIS-FXI-LRx for Clotting Disorders On October 9, 2019 Ionis Pharmaceuticals (IONS) announced it had been notified by its partner Bayer (

Hepion Pharmaceuticals CRV431 Decreased Liver Fibrosis and Tumor Burden in Experimental Models EDISON, NJ / ACCESSWIRE / October 16, 2019 / Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a biopharmaceutical company focused on the development of therapeutic drugs for the treatment of liver disease . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Reata Pharmaceuticals Omaveloxolone for Friedreich’s Ataxia On October 14, 2019 Reata Pharmaceuticals (RETA) announced the Part 2 portion of its Phase 2 MOXIe trial, with its product omaveloxolone, met its primary endpoint in modifying Friedreich’s ataxia (FA). The largest change occurred in the modified Friedreich’s Ataxia Rating Scale (mFARS) after 48 weeks of treatment.  Patients treated with omaveloxolone 150 mg/day demonstrated a statistically significant, 2.40 point …

RegenxBio Rebounding RegenxBio (RGNX) has the viral vectors which could safely deliver genes into the cells. The firm has improved the adeno-associated viruses making them safe and effective gene delivery vehicles. One of the three currently approved gene therapy products, Zolgensma® for pediatric spinal muscular atrophy (SMA), marketed by Novartis, used RegenxBio’s adeno-associated . . . This content is for paid subscribers. Please click here to …

RegenxBio Interim Data from Phase I/IIa Trial of RGX-314 for Wet AMD RegenxBio (RGNX) announced interim data from the ongoing Phase I/IIa trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). Here are the results as presented by the Company. The results were presented by Jeffrey S. Heier, M.D., Co-President and Director . . . This content is for paid subscribers. Please …

Stock Market Volatility  Stocks can move up and down for various reasons but not all of them have anything to do with the real value of the firms. As a matter of fact, almost all stocks outperform for a while, reverse their course to underperforming, then restart the cycle. The stock market in its entirety does the same thing for reasons that have nothing to …

Nektar Therapeutics Announces Two Studies of NKTR-358 Nektar Therapeutics (NKTR) announced the initiation of two Phase 1b studies of NKTR-358 (LY3471851*), a novel T regulatory (Treg) cell stimulator, one for psoriasis and one for atopic dermatitis. NKTR-358 for Autoimmune and Inflammatory Conditions NKTR-358 is designed to treat autoimmune and inflammatory conditions by correcting the immune system imbalance that results from reduced numbers and impaired function of …

Seattle Genetics Stock Rally Seattle Genetics (SGEN) stock has rallied today. It gained around $10; trading at over $85, nearing the Prohost 2019 $90 target. The reason for investors’ great enthusiasm is Seattle Genetics announcement of a presentation at the European Society for Medical Oncology . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The Medicines Company ORION-10 Study for ASCVD The Medicines Company (MDCO) announced positive topline results from the ORION-10 Phase 3 clinical trial in patients with atherosclerotic cardiovascular disease (ASCVD). The ORION-10 trial conducted with . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vitiligo Vitiligo is a chronic autoimmune disease. The immune system attacks the pigment-producing melanocyte cells causing loss of skin pigmentation. This causes a skin discoloration condition known as vitiligo. The immune system’s attack affects around 0.2% to 2.0% of the world’s population. There are currently no U.S. FDA or European EMA approved products for this condition. Initial vitiligo symptoms start before age 20 and remain …

News from Nektar Nektar Therapeutics (NKTR) announced that new data from patients with metastatic triple-negative breast cancer, who were enrolled in the PIVOT-02 study, will be presented at the upcoming 5th CRI-CIMT-EATI-AACR Cancer Immunotherapy Conference being held from September 25 to September 28, 2019 in Paris, France. The PIVOT-02 study is evaluating NKTR-214 . . . This content is for paid subscribers. Please click here to subscribe …

bluebird bio Has Promising Results from Its Lenti-D Gene Therapy for CALD Good results have come from bluebird bio’s (BLUE) updated results in the clinical development program for its investigational Lenti-D™ gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress in Athens, Greece. bluebird bio's Starbeam Trial . . . This content is for paid subscribers. Please click here to subscribe or here to …

Atara Biotherapeutics ATA188 for MS Atara Biotherapeutics (ATRA) announced the presentation of initial efficacy data as well as updated safety results from its ongoing Phase 1 study of the firm’s product ATA188 for the treatment of a progressive . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Migraine's Really Effective Treatments The years 2018 and 2019 could be called the years of migraine’s really effective treatments. Three Calcitonin-gene-related peptide (CGRP) monoclonal antibody drugs have been approved in 2018 and 2019. They are: Aimovig for Amgen (AMGN . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Moderna Therapeutics Trial Results Moderna Therapeutics (MRNA) trial results mark the first systemic mRNA therapeutic to show the production of a secreted protein in humans.  Moderna’s Product mRNA-1944 successfully encoded for functional antibody (CHKV-24) in humans at all dose levels tested (0.1, 0.3 and 0.6 mg/kg). The antibody level reached is predicted to protect against chikungunya infection achieved within hours. It is projected to be …

Diseases of the Back of the Eye Diseases of the back of the eye, such as: age-related wet macular degeneration, diabetic retinopathy and diabetic macular edema, are currently managed with injections of anti-vascular endothelial growth factor (anti-VEGF) drugs into the gel-like substance (the vitreous humor) that fills the eye between the lens and the retina. The currently used age-related wet macular degeneration injections are: Lucentis, which belongs …

Amgen's Outperforming After being boxed in, for the past couple of weeks, Amgen’s (AMGN) stock finally outperformed. The firm was outperforming silently with regard to its financial results, sciences, technologies, pipeline products and multiple newly approved products drugs.   In the News Amgen's Product AMG-510 Amgen announced new data from its ongoing Phase 1 study evaluating its unique cancer product AMG . . . This content …

Outperforming Biotechs On August 27, 2019 we posted “The Medicines Company Has Agreeable News” under Today’s Highlights.  The Medicines Company We described The Medicines Company (MDCO) as a firm focused on cardiovascular diseases with a goal of halting the progression of atherosclerosis and lowering the cardiovascular risk caused by high levels of LDL Cholesterol (LDL-C). The Medicines Company’s good news emanated from the results of …

Gene Therapy Stocks Experiencing Selloffs  Every investor in the biotech sector must have experienced selloffs of the stocks from companies specialized in gene therapy, gene editing, antisense, RNAi and mRNA therapies. Recently, bluebird bio’s (BLUE) stock was devastated. The selloffs occurred for many reasons, most of . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The Medicines Company The Medicines Company (MDCO) focuses on cardiovascular diseases. Major goals are halting the progression of atherosclerosis and lowering the cardiovascular risk caused by high levels of LDL Cholesterol (LDL-C). The firm announced good news from the results of its first pivotal Phase . . . This content is for paid subscribers. Please click here to subscribe or here to log in.