We reiterate that undeniably outstanding news about gene therapy is taking time to penetrate investors’ ears. They are still falling victim to bad rules of thumb and critics’ magnifications of the negative impact of some stocks’ dilution when companies with superior programs try to finance through a public offering. The stocks’ punishments following financing is not a rule to apply to all firms, especially those …

A complete response letter (CRL) sent by The Division of Metabolism and Endocrinology Products of the United States’ FDA to Ionis Pharmaceuticals (IONS) and its affiliate Akcea Therapeutics (AKCA) regarding Waylivra™ (volanesorsen) carried like usual bad news. The FDA has decided against approving Waylivra, the drug designed to treat the lipid disorder known as familial chylomicronemia syndrome (FCS). Familial chylomicronemia syndrome is an ultra-rare, devastating hereditary …

Before we sell PTC Therapeutics (PTCT), after the stock had reached and surpassed our latest and highest 2018 target, the firm did the right thing that we expected was good enough to encourage investment in this firm.  The good thing is acquiring Agilis Biotherapeutics – a small firm that succeeded in creating innovative gene therapy programs for rare genetic disorders that affect the central nervous …

Portola (PTLA) has two important approved breakthrough products and an investigational product having promising results on very difficult-to-treat cancers. Instead of soaring following the approval of its two drugs and the good results coming from the investigational third drug, Portola’s stock took several dives, which raised the eyebrows of many investors and motivated more shareholders to sell PTLA. Let us see whether the firm deserves …

When the Goal is Reaching into the Future, Pessimists Have No Place on the Ship The revolution in the biotech sector has proven to be moving fast towards reaching goals that were impossible to imagine a couple of years ago that they could be attainable. This is not true only regarding lay investors, or desperate patients, but also regarding the most professional clinicians, researchers and …

Celyad (CYAD) is an immuno-oncology firm, which created CAR T CELL treatments for cancers, including  solid tumors. Celyad announced its consolidated financial results for the six-month period ending 30 June 2018. Celyad’s report is endorsed by Europe. Dr. Christian Homsy, Celyad’s CEO started by expressing the firm’s great enthusiasm towards the progress it has made in the first half of 2018, which include a significant advancement of …

WE ARE LIVING AN HISTORIC BIOLOGICAL REVOLUTION. Unfortunately, the undeniably outstanding news has not yet penetrated the ears of many investors. They are still falling victim to the stock market traders’ games and to critics’ and bloggers’ magnifications or fabrications of negative news. Many investors end up selling their thoughtfully chosen highly scientific biotech firms to regret later on what they have done when their …

Vertex Pharmaceuticals (VRTX) announced that the Pharmaceutical Benefits Advisory Committee’s (PBAC) in Australia recommendation the listing of Orkambi® (lumacaftor/ivacaftor) on the Pharmaceutical Benefits Scheme (PBS). The recommendation is for cystic fibrosis people ages six and over who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Many thousands of patients worldwide are already receiving lumacaftor/ivacaftor in countries where it is reimbursed …

The FDA Approves Regeneron’s Product EyLea® (aflibercept) sBLA in Wet Age-Related Macular Degeneration Regeneron (REGN) has two good news. The first is from one of its approved products, EYLEA® announcing that the U.S. Food and Drug Administration(FDA) has approved a supplemental Biologics License Application (sBLA) for EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD). The sBLA was based on second-year data from the …

Aerie Pharmaceuticals (AERI) announced results from a double-masked, randomized, placebo-controlled study designed to evaluate the effect of its glaucoma product Rhopressa® (netarsudil ophthalmic solution) 0.02% on aqueous humor dynamics in patients with open-angle glaucoma or ocular hypertension. This study is the first performed on glaucoma patients to confirm that Rhopressa® lowers intraocular pressure (IOP) primarily through trabecular meshwork outflow facility.  In the trials, the enrolled patients …

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 …