PTC Therapeutics Risdiplam Results for SMA PTC Therapeutics (PTCT) has positive results from part 2 of the SUNFISH trial evaluating the safety and efficacy of the product risdiplam. The results demonstrate that the trial has met its primary endpoint of change from baseline after 1 year of treatment with risdiplam compared to placebo. The change was measured by the Motor . . . This content …

Reata Pharmaceuticals CARDINAL Study Met Primary and Key Secondary Endpoints Reata Pharmaceuticals (RETA) announced that the Phase 3 portion of the CARDINAL study of bardoxolone methyl (bardoxolone) in patients with chronic kidney disease (CKD) caused by Alport syndrome met its primary and key secondary endpoints.   This news is surely music in the ears of families with members . . . This content is for paid …

Exelixis and Ipsen Received Approval from Health Canada for CABOMETYX® From Exelixis (EXEL) we learned that its partner, Ipsen Biopharmaceuticals Canada, has received approval from Health Canada of CABOMETYX® (cabozantinib) tablets for the treatment of hepatocellular . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Exelixis and Daiichi Sankyo Partner to Prevent and Treat Diabetic Nephropathy Exelixis (EXEL) and Daiichi Sankyo (

Karyopharm Therapeutics Third Quarter Financial Results Generated Revenues For the first time since its establishment Karyopharm Therapeutics (KPTI) financial results report revenues from a strong launch of its first approved product. Indeed, the firm’s third-quarter 2019 financial results are the first quarter in which Karyopharm generated revenues. In early July the U.S. FDA granted the company’s product Xpovio® (selinexor . . . This content is for …

IVERIC bio Stock Outperforms IVERIC bio (ISEE) has good news its stock is responding to. A randomized, controlled Phase 2b clinical trial data seem to confirm that the IVERIC bio product Zimura® (avacincaptad pegol), a complement factor C5 inhibitor, has met its prespecified primary endpoint in reducing the rate of geographic atrophy (GA) growth in . . . This content is for paid subscribers. Please …

Seattle Genetics Announced Positive Results for HER2CLIMB Trial Seattle Genetics (SGEN) announced positive topline results from the HER2CLIMB trial evaluating tucatinib in HER2-positive breast cancer.  The trial compared tucatinib in combination with trastuzumab and capecitabine to trastuzumab and capecitabine

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 (

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 …

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.

Enanta Positive Results of EPD-305 for NASH Enanta (ENTA) has positive results from the ARGON-1 Phase 2 study evaluating the safety, tolerability pharmacokinetics and efficacy of its product EPD-305 for the treatment of non-alcoholic steatohepatitis (NASH).   The results met the primary endpoint of ALT reduction at week 12; in the . . . This content is for paid subscribers. Please click here to subscribe or here to …

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.

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.

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 …

Vertex Pharmaceuticals Is Acquiring Semma Therapeutics Vertex Pharmaceutical (VRTX) entered into a definitive agreement to buy a private company, Semma Therapeutics. Semma uses stem cell-derived human islets as a potential cure for type 1 diabetes. Semma’s major scientific advances include: an ability to produce large quantities of functional human pancreatic beta cells that could . . . This content is for paid subscribers. Please click here to subscribe …

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.