Vertex Pharmaceuticals in the NEWS Vertex Pharmaceuticals (VRTX) announced advancements across its suzetrigine pain program, which has the potential to be the first new class of medicine for acute and neuropathic pain in more than two decades. Vertex’s product suzetrigine is an oral selective NaV1.8 pain signal inhibitor, which has been granted FDA Fast Track and Breakthrough Therapy Designations in moderate-to-severe . . . This …

Vertex Pharmaceuticals Acquisition of Alpine Immune Sciences We start this posting by answering a question by savvy investors regarding Vertex Pharmaceuticals' (VRTX) decision to acquire Alpine Immune Sciences (

Vertex Pharmaceuticals in the NEWS Vertex Pharmaceuticals (VRTX) announced that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label expansion of KALYDECO ® (ivacaftor) for the treatment of infants with cystic fibrosis (CF) ages 1 month to less than 4 months old who have one of the . . . This content is for …

CRISPR Therapeutics Announcement Today, Feb. 15, 2024, CRISPR Therapeutics announced that members of its senior management team will present at the Citi 2024 Virtual Oncology Leadership Summit on Wednesday, Feb 21, 2024, at 1:00 p.m. ET. A live webcast of the fireside chat will be available on Events & Presentations page in the Investors section of the Company's website. A replay of the webcast will …

Crispr Therapeutics First NEWS As we learned from Vertex Pharmaceuticals (VRTX) in an earlier, posted press release about the European approval, Crispr Therapeutics (CRSP) has also announced the same breakthrough . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

European Commission Grants Conditional Marketing to Vertex Pharmaceuticals CASGEVY Tuesday Feb 13, 2024, Vertex Pharmaceuticals (VRTX) announced that the European Commission has granted conditional marketing authorization to CASGEVY (exagamglogene autotemcel ), a CRISPR/Cas9 gene-edited therapy. CASGEVY is being approved for patients 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso . . . This content is for paid …

Vertex Pharmaceuticals Positive Results for VX-548 Today, Vertex Pharmaceuticals (VRTX) announced positive results from its Phase 3 program for the treatment of moderate-to-severe acute pain. The Phase 3 program included two randomized, double- blind, placebo-controlled, pivotal trials, one following abdominoplasty . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vertex Pharmaceuticals Updates Vertex Pharmaceuticals (VRTX) announced multiple program updates ahead of the upcoming investor meetings in January, including the 42 Annual J.P. Morgan Healthcare Conference, which took place on January 8, 2024. From Vertex Pharmaceuticals Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex said,  “2023 was marked by continued strong performance in the cystic fibrosis business and acceleration of our . . …

Editas Medicine and Vertex Pharmaceuticals in the NEWA Today December 13, 2023, Editas Medicine (EDIT) announced that it entered into an agreement with Vertex Pharmaceuticals (

Vertex Pharmaceuticals in the NEWS Today, December 13, 2023, Vertex  Pharmaceuticals (VRTX) announced positive results from its Phase 2 dose-ranging study of the selective NaV1.8 inhibitor VX-548 in people with painful diabetic peripheral neuropathy (DPN). Treatment with all . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Crispr Therapeutics and Vertex Pharmaceuticals Gene Editing Therapey Will Be Marketed On Nov. 16, 2023, Crispr Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX)

Vertex Pharmaceuticals and CRISPR Therapeutics Announce Authorization of CASGEVY Conditional Approval for Both Sickle Cell Disease and Transfusion Dependent Beta Thalassemia Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) today announced that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) granted them conditional marketing authorization for CASGEVYTM (exagamglogene autotemcel ), a CRISPR/Cas9 gene . . . This content is for paid subscribers. Please …

CRISPR Therapeutics and Vertex Pharmaceuticals NEWS CRISPR Therapeutics (CRSP) announced the completion of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee meeting for exagamglogene autotemcel (exa-cel) for the treatment of Sickle Cell Disease (SCD) in people ages 12 and older with recurrent vaso-occlusive crises (VOCs). Exa . . . This content is for paid subscribers. Please click here to subscribe …

Vertex Pharmaceuticals ORKAMBI Receives Positive CHMP Opinion Today, April 27, Vertex Pharmaceuticals  (VRTX) announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) embraced a positive opinion for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 . . . This content is for paid subscribers. Please click here to subscribe or here to log …

Vertex Pharmaceuticals and CRISPR Therapeutics Completion of BLAs Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced the completion of the . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vertex Pharmaceuticals and Crispr Therapeutics Agreement Aims at Accelerating Vertex's Hypo immune Cell Therapies for Type 1 Diabetes Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics’ gene editing . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Important News from Vertex Vertex Pharmaceuticals (VRTX) is a worldwide biotechnology company that invests in scientific innovation. The firm created transformative medicines for people with serious diseases. Vertex has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease. It has several ongoing clinical and research programs in CF. Beyond CF, Vertex . . . This content is …

January 5, 2023 In Today's Down Market the stocks that are outperforming are as follows: Reata Pharmaceuticals (RETA) bluebird bio Inc (BLUE) And Vertex Pharmaceuticals . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Prohost Comprehensive Letter #451 Shorting Biotech Firms with Solid Fundamentals Unfair shorting of promising stocks seems to have become the norm during the harsh circumstances that not only the United States but several other countries have been living under for more than two years. The bad circumstances in the United States seem to be motivating unfair short-sellers to take advantage of bad news and cause …

Vertex Pharmaceuticals and ViaCyte Inc are in the NEWS Vertex Pharmaceuticals (VRTX) entered into a definitive agreement under which it will acquire ViaCyte, a private biotechnology company focused on delivering novel stem cell-derived cell replacement therapies as a functional cure for type 1 diabetes (T1D), for $320 million in cash. From Vertex Pharmaceuticals and ViaCyte Inc Reshma Kewalramani, M . . . This content is for paid subscribers. …

Vertex Pharmaceuticals VX-880 for Type 1 Diabetes Vertex Pharmaceuticals (VRTX) announced that the FDA has lifted the clinical hold placed on the Phase 1/2 clinical trial of VX-880. VX-880 is an investigational allogeneic stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia . . . This content is for paid subscribers. …

Vertex Pharmaceuticals to Announce New Data on VX-880 Clinical Trial Vertex Pharmaceuticals (VRTX) announced three abstracts, including two oral presentations and one poster, detailing data on type 1 diabetes (T1D) and its Phase 1/2 trial of VX-880, a stem cell-derived, fully differentiated pancreatic islet cell replacement therapy. The Abstracts have been accepted for presentation during the American Diabetes Association 82nd Annual Scientific Sessions Conference held …

A lot is happening while the Stock Market is tanking and is finally being considered a Bear Market. Trading in this Market as it is now is more like gambling rather than trading. We are working hard towards evaluating our picked stocks and finding the possible time they might reach our new targets.   In the meantime, Vertex's stock, which we believe is one of …

Vertex Pharmaceuticals Statistically Significant Results From Vertex VX-548 for Acute Pain Vertex Pharmaceuticals (VRTX) announced positive results from two Phase 2 proof-of-concept (POC) studies that investigated treatment with the selective NaV1.8 inhibitor VX-548 for acute pain following abdominoplasty surgery or bunionectomy surgery. Treatment with an initial dose of 100 mg followed by 50 mg . . . This content is for paid subscribers. Please click here …

Comprehensive Prohost Letter Issues Reality The Best of words in informative texts are the smallest in number and the most truthful in contents. That’s what Prohost believes, especially in the presence of the cruel circumstances our entire planet is currently living. Last Friday, the three indexes of the stock market had their best performances since the 4th quarter of 2020. Why? We don’t know, but …

Some Gains in Biotech Stocks During the War in Europe On Tuesday, Mar 8,2022 the United States’ government decided to ban the importation of Russian oil, liquefied natural gas and coal. This decision was made to further deprive Russia from the resources that enable it to continue with the unwarranted war against Ukraine. This resolution has come at a time when the price of oil …

Vertex Pharmaceuticals Phase 2 Results of VX-147 Vertex Pharmaceuticals (VRTX) announced today that, in a Phase 2 proof-of-concept (POC) study in patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS), VX-147, on top of standard of care, achieved a statistically significant, substantial and clinically meaningful . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Cassava Sciences, Vertex Pharmaceuticals and Gilead Sciences All Have Good News Cassava Sciences & Alzheimer's Disease Cassava Sciences (SAVA) announced the initiation of a second Phase 3 trial of simufilam, its investigational drug for Alzheimer’s disease. This second Phase 3 study is designed to evaluate the safety and efficacy of simufilam over 78 weeks in approximately 1,000 Alzheimer’s . . . This content is for …

Vertex Pharmaceuticals Receives FDA Approval for TRIKAFTA® Vertex Pharmaceuticals (VRTX) announced the U.S. FDA approved the expanded use of TRIKAFTA® to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive . . . This content is for paid subscribers. Please click here to …

Vertex and CRISPR Granted PRIME Designation from the EMA for CTX001 Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (

CRISPR Therapeutics Dr. Emmanuelle Charpentier, Co-Founder CRISPR Therapeutics (CRSP) announced on October 7, 2020 that Dr. Emmanuelle Charpentier has been awarded the 2020 Nobel Prize in Chemistry for her groundbreaking work on the CRISPR/Cas9 system.  Dr. Emmanuelle Charpentier is the co-founder of CRISPR Therapeutics together with Rodger Novak and Shaun Foy. Dr. Charpentier is the Founding, Scientific and Managing . . . This content is …

The Latest on the COVID-19 Vaccines AstraZeneca (AZN): After pausing for more than a month, AZN is expected to resume its Phase 3 US study of its COVID-19 vaccine. The trial was paused after a patient, who received the vaccine, developed an illness suspected to be a serious spinal inflammatory condition known as transverse myelitis. The patient was . . . This content is for …

Crispr Therapeutics and Vertex Pharmaceuticals CTX001 Granted PRIME Designation by the EMA CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (

CRiSPR Therapeutics Rally  For those who wonder why CRiSPR Therapeutics (CRSP) is soaring we remind that they should have been expecting this rally and for it to be followed by its two sisters Intellia Therapeutics (

Illumina Diagnostic Test COVIDSeq to Detect SARS-CoV-2 Illumina (ILMN) was issued an Emergency Use Authorization (EUA) from the FDA for the first COVID-19 diagnostic test utilizing next-generation sequence technology. The authorization was granted for the Illumina COVIDSeq Test for the qualitative detection of SARS-CoV-2 RNA from respiratory specimens collected from individuals suspected . . . This content is for paid subscribers. Please click here to subscribe …

Prohost Letter #440 Coronavirus The Therapeutics Gilead’s Remdesivir Treating COVID-19 patients requires therapeutic products that can deal with moderate and severe persistent symptoms that are unwilling to ease or completely disappear after weeks of isolation, when following the distancing and wearing masks. The first product proven effective and safe to be granted FDA Emergency Use Approval (EUA) happens to be remdesivir from Gilead Sciences (

Vertex and Regeneron Will Survive the Coronavirus Market Vertex We picked Vertex (VRTX) on March 18, 2011 at $44.39. The stock closed at $218 at the end of 2019. It rallied in mid-February reaching $247.78 then the unexpected and unseen novel coronavirus started infecting the world. Our 2020 target is still $280. We decided upon this new target in . . . This content is …

Prohost Letter #439 The Coronavirus Outbreak - COVID-19 The news announced that the coronavirus (now known as COVID-19) outbreak would affect not only people’s health and survival but the world’s economy, Wall Street’s performance, and especially the international giant industrial firms that are committed to China. British AstraZeneca (AZN

CRISPR, Intellia and Editas CRISPR Therapeutics (CRSP) announced revenues of Q4 2019 that beat analysts’ expectations. Based on the good news the stock market’s technicians contributed to boosting the stock’s price enabling the stock to recuperate a large part of its unwarranted recent selloff losses. New investors, who finally heard about the CRISPR gene editing technique and the Companies that specialize in it, seem to …

Vertex Beats Expectations in 2019 Vertex (VRTX) reported fourth-quarter and full-year 2019 financial results yesterday. The results beat analysts’ expectations about the last quarter and the whole year 2019. In the same report the firm’s full-year financial guidance promises a thrilling future for Vertex. The report indicated that the total GAAP and Non . . . This content is for paid subscribers. Please click here to …

A Comprehensive Essay Part 2 In Part 1 of our comprehensive essay series Vertex Pharmaceuticals (VRTX) was the first firm we used to make a case in point to demonstrate successful criteria and strategies towards evaluating biotech firms. In Part 2, Vertex . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

A Comprehensive Essay Part 1 Yes, we have stories to tell about some of our picked stocks which proved to be highly successful after being condemned by erroneous evaluations from unfair analysts. Some of these firms faced problems that are not unusual for drug designing and developing firms. While trying to overcome their obstacles, the firms in question were faced with unfortunate debilitating attacks by …

Coming Soon at Prohost Biotech We have stories to tell about our picked stocks which we believe could deepen the Prohost Biotech readers’ understanding of our strategy regarding the evaluation of the biotech companies. Our strategy has made it possible for us to fairly evaluate both the development-stage firms and the revenues- and incomes-generating firms in the biotech sector.     Most of our stories will …

Prohost Letter #438 End of the Year Issue  2019 Happy New Year 2020 The NASDAQ reached 9000 for the first time in the market history on December 30, 2019. However, on the last trading day of 2019, the Nasdaq Composite Index closed at 8,945.99 after giving back 60.62 points or 0.7%. During the departed year, however, the stock exchange was boosted by the outperformance of …

The Week in Review #48 Holiday Edition ******************** Merry Christmas and Happy Hanukkah! We Wish You a Happy New Year Filled with Excellent Health, Peace of Mind, Prosperity and Happiness What a Year for Biotechnology What happened in the biotech sector toward the end of 2019 is briefed in this Review as we focus on the performance of our picked firms’ stocks’ prices. We will …

Firstly, Forty-Seven and IVERIC bio  We remind that the two condemned stocks Forty-Seven (FTSV) and  IVERIC bio (

The Market May be Plummeting But Prohost Picks are Not Biotech stocks had a strong start, but the gains disappeared in many stocks as the market demonstrated bearishness following a long-lasting rally. Currently, during the strong economic conditions, commentators attribute the reasons for the market’s upside and downside moves to China’s positive, or negative ongoing negotiations rather than to fair correction of overbought or oversold …

Prohost Letter #437 An All Time High Market More Prohost Picks Have Reached the 2019 Targets The stock market performance has reached an all-time high with more of our picks reaching or nearing the Prohost 2019 price targets. There are stories to tell about some devastated stocks which many believed were dead and buried but were surprisingly resurrected, and their stocks are going up. Another …

Vertex Pharmaceuticals and CRISPR Therapeutics Rallied Today Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. A patient with transfusion-dependent beta-thalassemia (TDT) received CTX001 in the first quarter of 2019. Data for this patient reflect nine months of safety and efficacy follow-up. …

The Week in Review #46 The Quarterly Financial Results The quarterly financial results that analysts and investors relied on for years to evaluate biotech firms, including development stage firms, seem to change. Investors have finally realized that development-stage firms have no marketed drugs and no revenues, hence, no incomes and should be evaluated based on the clinical trial results of their pipeline products. They saw …

The Week in Review #44 Quarterly Financial Results What Was Seen and What Was Ignored History and self-experiences enabled investors to expect and swallow biased reactions following the announcements of the biotech sector’s firms’ quarterly results. Investors have long been expecting negative reactions towards small development-stage biotech firms following the announcement of their quarterly financial results. What they did not expect is for the same …

The Week in Review #43 This Past Week After hearing hints about the trade war between China and the United States being avoided the market reversed course from faltering to flying at the speed of light. This trade war replaced all important factors that would usually affect the market performances. Unemployment is the lowest since 1969 and is expected to cause a bullish market, but …

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 …

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.

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 …

AstraZeneca: Farxiga for Heart Failure with Reduced Ejection Fraction  AstraZeneca (AZN) has good news. Many of them are extremely positive. However, the firm’s stock is still struggling to get this pharmaceutical company out of the oversold territory where it has been living since the beginning of the year.    Among the recent good news came the results from a Phase 3 trial using AstraZeneca’s SGLT2 inhibitor …

Regeneron Product REGN-EB3 Saving Lives Regeneron (REGN) announced that an independent monitoring board ended its investigational product REGN-EB3 trial after reviewing interim mortality data from 499 patients. REGN-EB3 was found superior to rival therapy from Mapp Biopharmaceuticals (MAPP) in saving the lives in . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Nektar Therapeutics We believe that Nektar Therapeutics (NKTR) is undervalued. We base this on the firm’s technological capability. Its current pipeline is filled with promising products. Today we learned that the U.S. FDA granted Nektar’s and Bristol-Myers Squibb’s . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vertex and CRISPR Therapeutics While some groups are doubting the gene editing capability of CRISPR gene editing Vertex’s (VRTX) scientists enjoyed what they experienced during their clinical collaboration with CRISPR Therapeutics (

Prohost Letter #431 Genomes, Genes and Gene Therapies In this issue of the Prohost Letter we focus on genomes, genes and gene therapies. National DNA day is April 25th . On this day we commemorate: James Watson, Francis Crick, Maurice Wilkins, Rosalind Franklin and other of their collegues who published papers on the structure of DNA in 1953. It is an opportunity to celebrate the …

Vertex’s Good News Vertex and Kymera Therapeutics Collaboration Last week Vertex Pharmaceuticals (VRTX) and Kymera Therapeutics entered into a four-year strategic research and development collaboration to advance small molecule protein degraders against multiple targets. Kymera is an expert in targeted protein degradation and has its own drug discovery program known as Pegasus™. The collaboration will benefit from this program and from Vertex’s scientific, clinical and regulatory capabilities …

Vertex Pharmaceuticals' Product Kalydeco for CF in Young Children Good news for Vertex (VRTX) product Kalydeco® (ivacaftor) is the first cystic fibrosis (CF) product to be prescribed for children ages six months to less than 12 months. Beneficiary children are those who have at least one mutation in their . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

CRISPR Therapeutics' (CRSP) stock added around $8 today while reporting financial results for the fourth quarter and full year ended December 31, 2018. Here is what Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, has stated,“This past year was truly transformational for CRISPR Therapeutics as we achieved milestones across our key programs in β-thalassemia, sickle cell disease and immuno-oncology. We’re pleased with the progress we’ve made in …

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 …

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 …

The Week in Review #29 PROHOST AGGRESSIVE PORTFOLIO  - The aggressive portfolio hosts biotech firms with solid scientific fundamentals and prominent scientists whose stocks have plummeted following some setbacks. A setback could be a failure of an investigational product to meet the endpoints of its clinical trial or that the small biotech firm has taken long to achieve its goals . . . This content …

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 …

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 …

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 …

Prohost Letter #418 Observing Queer Stories  PORTOLA - Portola (PTLA) has been granted approval of an important factor Xa inhibitor Bevyxxa (betrixa-­ban for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for a life-­threatening VTE . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Expected or not, we believe that the news announcing that the U.S. Food and Drug Administration (FDA) approval Vertex’s (VRTX) cystic fibrosis combo product Symdeko™ (tezacaftor/ivacaftor and ivacaftor) is good news. It is indeed excellent news for the many cystic fibrosis people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis trans-membrane conductance regulator (CFTR) gene or who have at least one mutation that …

Vertex (VRTX) announced the selection of two next-generation correctors, VX-659 and VX-445, to advance into Phase 3 development as part of two different triple combination regimens for people with cystic fibrosis (CF). The decision was based on data from initial Phase 2 trials, including new data from ongoing Phase 2 studies demonstrating mean absolute improvements in percent predicted forced expiratory volume in one second from baseline through four weeks of treatment …

The Week in Review #16 More Exciting Rallies in Prohost Picks - The past week and for several week fascinating outperformances have been seen for many of the Prohost Portfolio stocks. We continue to articulate that the good news is not only in the stocks’ rallies, but also in the outstanding stories behind them . . . This content is for paid subscribers. Please click here …

VERTEX From Europe, Vertex (VRTX) was informed that the European Commission has granted an extension of the Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor). Orkambi is for cystic fibrosis (CF) in people with two copies of the F508del mutation, including children ages 6 through 11 Reimbursement: In countries such as Ireland, reimbursement agreements will lead to rapid access to ORKAMBI. In other countries across the European Union, Vertex will begin a country-by-country reimbursement process. Clinical trial Results …

The Week in Review #9 KNOCKING DOWN THE BIOTECH STOCKS - Knocking down the biotech companies’ stocks during the announcement of the quarterly reports is no news. Most investors in the stock market, especially those who invest in the biotech sector have become familiar with this pattern . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

CRISPR Therapeutics (CRSP) and Massachusetts General Hospital Cancer Center (MGHCC) have entered into a two-year research collaboration and license option agreement to develop Novel T cell therapies for cancer. As part of the collaboration, CRISPR/Cas9 gene editing technology will be utilized to improve upon current T cell therapies in development addressing unmet needs in both hematologic and solid tumors.  This improvement is expected to occur by combining the …

Synopsis Most of the Prohost picked stocks gained momentum and closed higher at the end of the week. Some rallied because that’s what they were supposed to do after the quarterly financial when most biotech stocks are subjected to selloffs, including those that have clearly outperformed in real life. The most outperforming of our picked stocks in July and continue to outperform are Illumina (ILMN) …

Yesterday, Wednesday July 19, Vertex’s (VRTX) stock added over 20% to its value, recording a new all time high. Why? On Tuesday the firm announced a better than technical analysts’ and its own expectations’ results in three trials testing its cystic fibrosis (CF) drugs VX-152, VX-440 and VX-659 combined with two the its cystic fibrosis drugs. The positive data came from Phase 1 and Phase …

Prohost Letter #408 AT A GLANCE - Continued from the Previous Prohost Letter #408 In the Past issue #407, we picked the following firms: NEUROCRINE BIOSCIENCES (NBIX) Treating Tardive Dyskinesia CYTOMX Improving On Cancer Immunotherapy CELYAD SA (CYAD) Bringing Solutions To Immunotherapy Problems Please Read the articles written about these firms in the Issue #407 In This Issue We Add  NEW LINK GENETICS...

Prohost Letter #406 A Special Prohost Issue SYNOPSIS REASONS FOR THE FOLLOWING PROHOST PICKS RALLIES VERTEX Vertex (VRTX): Great news about 2016 revenues & earnings Growth and pipeline. FROM YESTERDAY: Tremendous Revenues Growth. Cystic fibrosis (CF) revenues grew up around 75%. FOR TOMORROW:  Boosting  the  pipeline  value  by Advancing CF novel correctors into Phase 2 trials while Producing and advancing new CF correctors. PROJECTIONS: Continued …

Prohost Letter #405 Synthetic Biology (3) We have already decided upon becoming shareholders the day synthetic biology firm called Synthetic Genomics turns public. We reiterate that firms specialized in synthetic biology are numerous private and publicly traded. Some are promising, others are walking in place and a few have plummeted. With the general underperformance of synthetic biology stocks, many shareholders abandoned ship. Investors felt that …

Vertex (VRTX) entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four research and development programs that the firm believes represent novel approaches to cancer treatment Merck KGaA will license two clinical-stage programs targeting DNA damage and repair, along with two additional novel pre-clinical programs. Upon the agreement Vertex will receive an upfront payment of $230 million, …

Prohost Letter #403 Part 2 Continued From Wednesday Instead of continuing yesterday article about Agenus, we decided to post it all so that the reader can find it in one place. Important changes and additions have been made especially under the headline “Near-­Term Priorities, Possibilities and Catalysts”, which are important to read. Learning More About AGENUS Since its inception, Agenus (AGEN), previously known as Antigenics, …

Pfizer accepted to pay $14 billion in cash to acquire Medivation. It is important for investors to know what Pfizer is after with regard to this acquisition so that investors could sense the current deep-pocketed biopharmaceutical firms’ preferences in selecting their takeover targets. Recognizing the facts behind Pfizer’s decision could also protect investors from being mislead by negative bloggers who, while serving their own agendas, …

GILEAD & GENMAB To Access Genmab’s (GEN) bispecific antibody technology, Gilead (GILD) signed a new agreement with Genmab. The King of HIV treatment objective is to use its exclusive license from Genmab to produce an HIV bispecific monoclonal antibody therapeutic. Gilead is paying around $5 million in upfront payment and up to $277 million in milestones. Genmab will also be entitled to one-digit royalty of …

Prohost Letter #392 February 28, 2016 - How could it be possible for any genius to make intelligent guesses about the market's next moves when highly regarded experts, including Nobel Prize winners in economics, remained quizzical about the recent horrifying performances of publicly traded stocks? Since the beginning of 2016, the stock market has been made to look as if a runaway train with many passengers …

Incyte: A Good Company Facing Irrelevant Investors’ Reaction  Incyte decided to stop an early ongoing trial of a combination treatment ruxolitinib (Incyte) plus regorafenib (Bayer) for colon cancer after observing the combination wasn’t working. Both drugs are approved for different indications and both are doing well in the market. At this moment in the market chaos, this news looked as if a terrible thing that must cause an unwarranted …

Prohost Letter #390 COMPANIES USING CRISPR GENE EDITING TOOLS - The Firms Launched with CRISPR Patents Either Pending or Disputed! What Does This Mean? What are these Firms’ Priorities? But First: A Reminder In the last issue, Prohost #389, we pinpointed the gene editing technologies and techniques and demonstrated the difference between gene editing, which is genetic engineering, i.e., making changes in the genes themselves, and antisense …

Europe Approves Orkambi® The European Commission has granted Marketing Authorization for Orkambi® (lumacaftor/ivacaftor), for cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation. There are approximately 12,000 people with CF ages 12 and older who have two copies of this mutation. Vertex will now begin the country-by-country reimbursement approval process across the European Union (EU). They said: The …

Prohost Letter #386 An Evaluation of Prohost Picks and Some Other Interesting Biotechnology Companies (Part 2) (Continued from Previous Prohost Letter #385) See Page Listening to Top-tier biotech firms’ presentations we could not resist appreciating their accomplishments, which made us dare to predict that the time has come for the self-serving preachers to find it extremely hard to convince shareholders to to sell stocks of …

Prohost Letter #379 Investors Concerns, Excitments and Expectations It is understandable the fact that bullish and bearish analysts have opposite opinions (opposite wishes too) on biotech firms’ pipelines products and clinical trial results. They differ in assessing important news and in applying different criteria in their evaluation in order to establish proof of concept of their personal views. Articles are filling the media with contradicting …

Prohost Letter #378 Part 1 Views and Visions TOP-TIER BIOTECHNOLOGY COMPANIES GILEAD We wrote a lot about Gilead (GILD), doing our best to protect investors against negative gossip that filled the media and aimed at misleading and insulting investors’ intelligence. We never lowered our target based on the perpetrated, fabricated stories, which made many investors lose confidence in Gilead’s strategy and sell their stocks, thus, …

AS WE AND PREDICTED, the FDA Pulmonary-Allergy Drugs Advisory Committee (PADAC), voted in favor of approving Vertex’s (VRTX) drug Orkambi™ (lumacaftor/ivacaftor) for people with Cystic Fibrosis (CF) ages 12 and older who have two copies of the F508del mutation in the CFTR gene. The FDA is expected to make a decision on the approval of Orkambi by July 5, 2015 under the Prescription Drug User …

Prohost Letter #377 PICKING BIOTECH FIRMS Before Investing in Them Raising the bar in designating small drug developing firms as biotechnology firms has occurred several times since 2011. In that year, many technologies were tremendously enhanced, which enabled them to realize superior goals, i.e., produce safer and more effective breakthrough drugs for chronic debilitating and life-threatening diseases. Prohost Portfolios picks, which have outperformed other industries’ …