Tag: Novartis (NVS)

Regenxbio: Promising Results from RGX-121 Gene Therapy for Mucopolysaccharidosis Type II (MPS II)

Regenxbio: Promising Results from RGX-121 Gene Therapy for Mucopolysaccharidosis Type II (MPS II)

Regenxbio Results from RGX-121 for MPSII Regenxbio (RGNX) announced interim data from Phase I/II trial of RGX-121 for Mucopolysaccharidosis Type II (MPS II). RGX-121 is designed to deliver the gene that encodes iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) using the AAV9 vector. About Mucopolysaccharidosis Type . . . This content is for paid subscribers. Please click here to subscribe or here to log in.
Novartis to Acquire The Medicines Company at $85 a Share

Novartis to Acquire The Medicines Company at $85 a Share

Novartis to Acquire The Medicines Company Novartis (NVS) decided to buy The Medicines Company (MDCO) at $6.8 million. The Medicines Company’s shareholders will be getting $85 per share in cash  – a 45% premium to its unaffected share price and a 57% premium to its unaffected one-week average price. The Transaction is expected to be completed in the first quarter of 2020 The Medicines Company …
PTC Therapeutics’ Product Risdiplam Met Trial Endpoints for the Treatment of Spinal Muscular Atrophy

PTC Therapeutics’ Product Risdiplam Met Trial Endpoints for the Treatment of Spinal Muscular Atrophy

PTC Therapeutics Risdiplam Results for SMA PTC Therapeutics (PTCT) has positive results from part 2 of the SUNFISH trial evaluating the safety and efficacy of the product risdiplam. The results demonstrate that the trial has met its primary endpoint of change from baseline after 1 year of treatment with risdiplam compared to placebo. The change was measured by the Motor . . . This content …
When Negative Reactions to Biotechnology News Mislead. The Case of Regenxbio.

When Negative Reactions to Biotechnology News Mislead. The Case of Regenxbio.

Regenxbio Misleading Selloff A selloff of Regenxbio (RGNX) stock, following the FDA’s partial hold on the intrathecal administration of AVXS-101, is unwarranted and totally misleading.   Why? Because the selloff of RGNX gave the impression that the gene therapy Zolgensma, using the adeno-associated virus (AAV) vector , developed by Regenxbio, has failed, which is not true. The partial hold was put only on the product …
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 …
What Are We Experiencing Now?

What Are We Experiencing Now?

Prohost Letter #436 What Are We Experiencing Now? The bizarre market underperformance progressing rather than regressing and the good economy and the employment gains are the least utilized factors after being the most influential on the stock market performance. We are witnessing the most bullish analysts being confused, hesitating to give their followers a crystal clear explanation about the market’s skepticism, anger and volatility. The …
The Value of the Biotech Companies Continued

The Value of the Biotech Companies Continued

Prohost Letter #435 The Value of the Biotech Companies A Continuation of Prohost Letter #434 A Recap The biotech near and long-term values, we insist, must be based on the firms’ scientific capabilities, products’ potentials and financial results. In the past months the stocks evaluations by bloggers, and some assessors, have been tailored to mislead investors into serving the purposes of the manipulators’ weird agendas. …
About RegenxBio’s Agreement with Clearside Biomedical and More

About RegenxBio’s Agreement with Clearside Biomedical and More

Diseases of the Back of the Eye Diseases of the back of the eye, such as: age-related wet macular degeneration, diabetic retinopathy and diabetic macular edema, are currently managed with injections of anti-vascular endothelial growth factor (anti-VEGF) drugs into the gel-like substance (the vitreous humor) that fills the eye between the lens and the retina. The currently used age-related wet macular degeneration injections are: Lucentis, which belongs …
Gene Therapy and Other Treatments That Work at the Genetic Level

Gene Therapy and Other Treatments That Work at the Genetic Level

Gene Therapy Stocks Experiencing Selloffs  Every investor in the biotech sector must have experienced selloffs of the stocks from companies specialized in gene therapy, gene editing, antisense, RNAi and mRNA therapies. Recently, bluebird bio’s (BLUE) stock was devastated. The selloffs occurred for many reasons, most of . . . This content is for paid subscribers. Please click here to subscribe or here to log in.
The Bizarre Market

The Bizarre Market

The Week in Review #41 We Are Still Witnessing … The bizarre market performance that started in early 2019: In the past couple of months the great enthusiasm and response of the market to the undeniably strong economic indicators and the lowest unemployment figures turned, for mysterious reasons, to a perturbed-looking volatile market beast. The biotech stocks have given back most of their deserved gains …
The Value of the Biotech Companies

The Value of the Biotech Companies

Prohost Letter #434 Special Issue The Value of the Biotech Companies With a strong economy and an unemployment rate reaching the lowest percentage in decades we are witnessing strange, if not bizarre, market performance since the beginning of 2019. From the early signs of economic strength the stock market reacted explicitly to positive, economic news. Later, the enthusiastic market turned dispirited and volatile. The biotech …
Rhythm Pharmaceuticals Has Good News. See Also: The Novartis News Story

Rhythm Pharmaceuticals Has Good News. See Also: The Novartis News Story

Rhythm Pharmaceuticals From Rhythm Pharmaceuticals (RYTM) we learned that positive, top-line results from two pivotal Phase 3 clinical trials evaluating setmelanotide, for pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities, have met their primary endpoints and all key secondary endpoints. The product demonstrated a statistically significant and clinically meaningful effect on weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC and LEPR deficiency …
Gilead Sciences Activities Are Exceeding Its All Time High. Celgene Demonstrates How Valuable It Could be for Bristol-Myers Squibb.

Gilead Sciences Activities Are Exceeding Its All Time High. Celgene Demonstrates How Valuable It Could be for Bristol-Myers Squibb.

Gilead Sciences Activities There is no doubt that Gilead Sciences (GILD) is doing whatever it takes to expand, improve and upgrade its pipeline products. For the last couple of months, each and every week, we heard news about collaborations with some companies and investment in others as well as licensing products from large, medium-sized and small firms. In less than ten . . . This content is for …
Novartis: Psoriatic Arthritis Patients on Cosentyx Had No Radiologic Progression After 2 Years of Treatment

Novartis: Psoriatic Arthritis Patients on Cosentyx Had No Radiologic Progression After 2 Years of Treatment

Good News about Novartis’ Cosentyx® for Psoriatic Arthritis Novartis (NVS) has good news for Cosentyx®. Data emerging out 0f the FUTURE 5 clinical trial on patients with psoriatic arthritis (PsA) showed that there was no radiographic progression in around 90% of PsA patients who were treated with Cosentyx® (secukinumab) 300 mg, over 2 years. These data are being presented at the Annual European Congress of Rheumatology (EULAR) on 12-15, June, in …
bluebird bio Announces a European Conditional Approval of Its Gene Therapy ZYNTEGLO™ for Transfusion-Dependent β-Thalassemia  

bluebird bio Announces a European Conditional Approval of Its Gene Therapy ZYNTEGLO™ for Transfusion-Dependent β-Thalassemia  

Zynteglo™ from bluebird bio Gets European Conditional Approval European marketing authorization for Zynteglo follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program. Zynteglo is bluebird bio’s first gene therapy to gain regulatory approval. Gene therapy is, indeed, moving towards the clinic to provide cures; to save people from an early death from …