The Week in Review #27 PRIORITY TOPICS: CRISPR GENE EDITING, GENETIC ENGINEERING & ANTISENSE TECHNOLOGY The choice of these topics was dictated by investors who follow the sell side preaching. These negative investors decided to cause a selloff in all kinds of therapeutics that act at the root cause of diseases, i.e., the malfunctioning genes. These therapeutics are what we call the future treatments and cures. Indeed, during …

Regeneron Pharmaceuticals (REGN) and bluebird bio, Inc. (BLUE) announced a collaboration to apply their respective technology platforms to the discovery, development, and commercialization of novel immune cell therapies for cancer. The collaborators will specifically leverage Regeneron’s VelociSuite® platform technologies for the discovery of human antibodies as well as T cell receptors (TCRs) directed against tumor-specific proteins and peptides, and bluebird bio will contribute its field-leading expertise in gene transfer and …

All the buying and selling of Portola (PTLA) will not make any sense to us until the firm fulfills its obligations towards what is required by the authorities, by health care and by private third-party payers.  So, in the reporting of its Q2 financial results for the three months ending June 30, 2018, in addition to providing a corporate update, nothing matters really except knowing where the …

When an investigational gene therapy gets excellent results, the usual reaction towards the developing firm is a celebration. This is especially true when the developing firm is Spark Therapeutics (ONCE), which has alreadyvalidated its gene therapy approach through the approval of its product LuxturnaTM (voretigene neparvovec-rzyl).         In its quarterly financial report, Spark said that 12 participants in Phase I/II trial investigating SPK-8011 for hemophilia A

Exciting are the Q2 2018 reported results by Exelixis’ (EXEL) including the firm’s revenues and earnings, compared to 2017. Impressive were Cabometyx’ (cabozantinib) revenues, in addition to the milestone payments from collaborating companies and ex-USA marketing agent. Inspiring also are the efforts the firm is spending on research and development with the aim of further boosting its revenues. Of the Results Revenues and Incomes – …

ProhostLetter #423 TOMORROW’S THERAPEUTICS TODAY For a more or less fair evaluation of biotechnology and pharmaceutical firms, analysts currently rely for their assessment on criteria that include quarterly financial results, products’ sales revenues, and year over year growth of each and every marketed therapeutic. When it comes to clinical-stage firms, the logic implies that these criteria cannot be relied on for evaluation, as most of …

The Future is Starting Now Recognizing that the future has already started now is important as it makes analysts use different criteria for the fair evaluation of the future growth of biotechnology startups and revenue-generating companies. This is our main topic the Prohost Letter that we are working diligently working on posting at any time now. This is extremely important to know before the hype …

The Committee for Medicinal Products for Human Use (CHMP) announced a Positive Opinion recommending marketing authorization of Alnylam’s (ALNY) RNAi product Onpattro™ (patisiran). The product is designed to treat hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy. The CHMP opinion is based on conclusive positive results from APOLLO Phase 3 study on patients with hATTR amyloidosis. The results were published in The New England Journal …

AbbVie (NYSE: ABBV), in cooperation with Neurocrine Biosciences (NBIX), announced that the United States FDA approved Orilissa™ (elagolix) under priority review for women with moderate to severe endometriosis pain. Orilissa represents the first FDA-approved oral treatment for moderate to severe endometriosis pain in over a decade. The drug is expected to be available in U.S. retail pharmacies in early August 2018. Endometriosis is a condition where …

The good news from Biogen’s (BIIB) Q2 exciting financial results comprised impressive news for Ionis (IONS) from the commercialization of Ionis’ approved product, Spinraza for Spinal muscular atrophy (SMA).  Biogen is also responsible for the commercialization of Ionis’ products Tegsedi™ (inotersen) and Waylivra™ (volanesorsen) – two antisense drugs that Ionis discovered and successfully advanced through Phase 3 studies. In the second quarter of Biogen’s 2018 …

Yesterday’s news unveiled that PTC Therapeutics (PTCT) has entered into an agreement to acquire Agilis Biotherapeutics. The transaction was approved by the Boards of both companies. The acquisition Agilis Biotherapeutics is expected to bring to PTC Therapeutics an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system. It is supposed to add to PTC Therapeutics’ pipeline an advanced gene therapy candidate, …

Novartis’ (NVS) financials results exceeded analysts’ expectations. The firm’s research institutions’ strategy, well-designed structure, plans for innovation and accurate execution led to a wealthy and competitive pipeline with more than 200 projects in clinical development. Novartis is flying into the future finding its way to more effective treatments and possible cures. As a matter of fact, Novartis is at the end of the road towards …

A Miracle-Like Gene-Editing That People will Sooner or Later Recognize. The Wellcome Sanger Institute’s new findings about CRISPR/Cas9 gene editing is intended to lead to further improvement in the safety of the breakthrough CRISPR gene editing procedure before it is used in clinical practice; the information is not intended to condemn it. Unfortunately, innocent investors got panicky when they heard the Wellcome Sanger findings, which …

The BLA Includes Data from More Than 11,000 Patients Amgen (AMGN) and UCB (Euronext Brussels: UCB) resubmitted a Biologics License Application (BLA) to the U.S. FDA for the approval of their osteoporosis investigational monoclonal antibody product Evenity™ (romosozumab). The product is indicated for osteoporosis in postmenopausal women at high risk for fracture.The drug increases bone formation and reduces bone resorption simultaneously to increase bone mineral …

The Week in Review #26 IN THE BIOTECH WORLD - RESEARCH - IMPROVING CRISPR GENE EDITING The problem cited with gene editing is that the editing may stay switched on after it succeeded in reaching its goals. To prevent further unwarranted mutations, scientists from the Bath and Cardiff University have invented a switch that aims at controlling protein expression in cultured cells and mouse embryos with the …

The New England Journal of Medicine (NEJM) published results from the CELESTIAL phase 3 pivotal trial of Exelixis (EXEL) product Cabometyx® (cabozantinib) in previously treated patients with advanced hepatocellular carcinoma (HCC). The data demonstrate that Cabometyx provided a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo. It is important to know that the clinical trial results formed the basis of regulatory …

Finally, we are hearing what we were constantly expecting to hear coming out of Compugen’s (CGEN) technological capability. Yesterday, the U.S. Food and Drug Administration (FDA) has cleared Bayer AG’s investigational new drug (IND) application for BAY 1905254, a first-in-class immuno- oncology therapeutic antibody targeting ILDR2. The target ILDR2 is a novel immune checkpoint protein discovered by Compugen through its predictive computational capabilities, which enable …

The Week in Review #25 When Stocks Plummet for No Relevant Reasons Sometimes investors need to know why some of their stocks plummet when no bad news is broadcast anywhere and when firms announce terrific news promising progressive growth. Yesterday was one of those days where reasons for stocks’ deviant, and probably unwarranted, negative performances must be identified. Let’s have a look at what happened during …

Health Canada Approves PrSYMDEKO™ For People Ages 12 and Older With Certain CFTR Gene Mutations Vertex (VRTX) announced that Health Canada approved SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or who have one copy of the F508del mutation and a second mutation predicted …

The FDA approved Array BioPharma’s (ARRY) products Braftovi™ capsules in combination with Mektovi® tablets for unresectable or metastatic skin cancer melanoma with a BRAF or BRAFV mutation, as detected by an FDA-approved test. BRAFTOVI is not indicated for the treatment of patients with wild-type BRAF melanoma. The products, which represent a new standard of care for BRAF-mutant melanoma are now available to order through select specialty pharmacies in the U.S. market.  The …