Tag: Vertex (VRTX)

Why Vertex and CRISPR Rallied Today

Why Vertex and CRISPR Rallied Today

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 Quarterly Financial Results

The Quarterly Financial Results

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 …
What Was Seen and What Was Ignored

What Was Seen and What Was Ignored

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 …
This Past Week

This Past Week

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 is Rebounding. See Also: A Scientifically Valuable Agreement Between CRISPR Therapeutics and KSQ Therapeutics

RegenxBio is Rebounding. See Also: A Scientifically Valuable Agreement Between CRISPR Therapeutics and KSQ Therapeutics

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 …
Why Alder Biopharmaceuticals Stock Gained Over 83%. See Also: Prohost Picked Stocks Outperforming in a Down Market Day

Why Alder Biopharmaceuticals Stock Gained Over 83%. See Also: Prohost Picked Stocks Outperforming in a Down Market Day

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: In Pursuit of Cures

Vertex: In Pursuit of Cures

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 and Vertex Both Have Good News

AstraZeneca and Vertex Both Have Good News

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: Successful Cure for Ebola-Infected People. Read Also: The Midday Outperforming Biotech Stocks

Regeneron: Successful Cure for Ebola-Infected People. Read Also: The Midday Outperforming Biotech Stocks

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.
The Nektar Good News Cannot be Hidden Any Longer

The Nektar Good News Cannot be Hidden Any Longer

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.
Genomes, Genes and Gene Therapies

Genomes, Genes and Gene Therapies

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: Good News and Good Decisions

Vertex: Good News and Good Decisions

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 Has Good News and Exelixis Is Reinforcing Its Outperformance

Vertex Has Good News and Exelixis Is Reinforcing Its Outperformance

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.
Why CRISPR Therapeutics’ stock gained around $8 today

Why CRISPR Therapeutics’ stock gained around $8 today

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 …
The importance of the FDA granting Fast Track Designation to Vertex’s and CRISPR’s Product CTX-001

The importance of the FDA granting Fast Track Designation to Vertex’s and CRISPR’s Product CTX-001

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: Offering proof of leadership in treating Cystic Fibrosis

Vertex: Offering proof of leadership in treating Cystic Fibrosis

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 …
The molecule that enhanced the T Cells cancer treatment efficacy while preventing autoimmune diseases

The molecule that enhanced the T Cells cancer treatment efficacy while preventing autoimmune diseases

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 …
Positive News

Positive News

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...
Following the Stars. Less Science and More Logic

Following the Stars. Less Science and More Logic

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 …