Earlier this month the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for CTX001 for sickle cell disease (SCD). CTX001 is in clinical trials conducted by CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX). It is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to …

Amgen Higher Dividends For those who invest in top-tier biotech firms for dividend payments, in addition to the firms’ continued growth, Amgen (AMGN) announced last Friday that its Board of Directors declared a $1.45 per share dividend for the first quarter of 2019. The dividend will be paid on March 8, 2019 to all stockholders of record as of the close of business on February 15, 2019. This represents …

Prohost Letter #425 When Common Sense Fails to Explain the Market Performance It is not unusual for the stock market to be in a selloff mood for a few days. However, in the last couple of months, the market unexpectedly plunged deep into the ocean of the unknown. The exaggerated volatility was nerve-wracking for professional analysts. This is especially true for candid analysts who base …

Vertex Pharmaceuticals’ triple combination, which added the corrector VX-659 to the tezacaftor and ivacaftor combination, resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis (CF). Data from Vertex’s (VRTX) Phase 3 study in people with one F508del mutation and one minimal function mutation resulted in a mean absolute …

Yesterday, the Stock Market skyrocketed and with it a significant number of Prohost Picks that we keep reminding ourselves are undervalued. The outperformance unveiled the interred strength of firms such as: Illumina (ILMN), Amgen (AMGN) and Vertex (VRTX), the gene therapy firms: RegenxBio (RGNX) and Spark (ONCE), and the gene editing firms: Crispr (CRSP), Intellia (NTLA) and Editas (EDIT), as well as others in the Prohost Portfolio. The rebounding in the …

The Week in Review #32 POSITIVE NEWS PROTEOSTASIS THERAPEUTICS Cystic Fibrosis Two days ago, a clinical-­stage small firm called Proteostasis Therapeutics (PTI) announced positive preliminary results from ongoing Phase 1 cystic fibrosis studies. The trial involved the firm’s proprietary combination therapy doublet, PTI-­808 + PTI-­801...

Prohost Letter #424 Following the Stars Less Science and More Logic We reiterate that while a human star is born every once in a while on Earth, several stars born daily in research labs may be designated every other day by the FDA and other health regulatory agencies around the world. These agencies call these potential stars’ breakthrough drugs, but not all the molecules designated …

Portola Pharmaceuticals Appoints Scott Garland as President and Chief Executive Officer, Effective October 8, 2018. Portola (PTLA) appointed a new president, Mr. Scott Garland, who was Relypsa’s president and previously chief commercial officer and responsible for the integration and growth of the U.S. operations after Relypsa was acquired by Vifor Pharma Group in 2016. Prior to Relypsa, Mr. Garland was executive vice president and chief commercial officer of Exelixis, Inc., …

CRISPR Gene Editing Beyond Bringing Cures to disease-causing genes, CRISPR gene editing technology can change the way diseases are being treated and treat diseases that have yet to find a treatment. CRISPR gene editing can play a key role in solving a lot of problems that prevent breakthrough treatments and approaches from reaching their ultimate goals. A best example is chimeric antigen receptor (CAR T) Cell immunotherapy approach to …

Playing buy and sell games with the CRISPR gene editing stocks is sometimes based on scientific articles that meant to caution about possible problems or by patents that were given to some Universities and denied it to others and vice versa. Different Universities back different CRISPR gene-editing firms, which constituted a reason for the volatility of the gene-editing stocks. Finally, CRISPR Therapeutics (CRSP), Intellia Therapeutics …

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 …

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 …

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 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 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 …

CRISPR Therapeutics, Intellia Therapeutics, and Caribou Biosciences, Inc., announced the news about the granted U.S. Patent No. 10,000,772 (“the ‘772 patent”) by the U.S. Patent and Trademark Office (USPTO). This patent covers methods of using optimized guide RNA formats (including single guide and dual guide formats) in certain environments, including eukaryotic cells (such as human, animal and plant cells). The optimized formats modify the part of …

Slashing of the CRISPR gene editing companies’ stocks, including CRISPR Therapeutics (CRSP), Editas (EDIT) and Intellia (NTLA) is caused by two studies published in Nature Medicine, which revealed that cells whose genes are edited by CRISPR/ Cas9 could develop tumors in treated patients. Nobody could blame those who sold the CRSPR gene editing stocks’ because, although many scientists believe that the cited cause can be overcome and others stated that …

CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX) announced that The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease pending the resolution of certain questions that will be provided by the FDA as part of its review of the IND. The IND was submitted to the FDA in April to support the planned initiation of a Phase …

SPARK THERAPEUTICS, SANGAMO THERAPEUTICS AND SALK INSTITUTE CURING HEMOPHILIA?   Hemophilia is an inherited bleeding disorder that causes abnormal or exaggerated bleeding and poor blood clotting. Hemophilia A and Hemophilia B are inherited in an X-linked recessive genetic pattern, so males are commonly affected while females are usually carriers of the disease. Hemophilia A is caused by clotting Factor VIII deficiency and hemophilia B (Christmas disease) is caused by Factor IX deficiency. A …

A couple of days following the news that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on Amgen’s (AMGN) product Prolia® (denosumab) for the treatment of bone loss associated with long-term systemic glucocorticoid therapy, the same Committee recommended a label variation for KYPROLIS® (carfilzomib) with an addition that includes the final overall survival (OS) data from the Phase 3 ASPIRE trial. In the ASPIRE trial, KYPROLIS …