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.

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 …

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. …

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 …

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 …

Novartis Has More Promising Data from Their Phase 3 Gene Therapy Trial with AveXis AveXis Inc., a Novartis (NVS) company, announced positive interim data from its Phase 3 STR1VE trial of Zolgensma® (onasemnogene abeparvovec-xio (AVXS-101) gene therapy in spinal muscular atrophy (SMA) Type 1. About Spinal Muscular Atrophy (SMA . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The United States FDA granted Breakthrough Therapy Designation to AMT-061. The European Medicines Agency gave it access to the Priority Medicine (PRIME) regulatory initiative. Gene therapy firm, uniQure (QURE), announced updated clinical data in patients treated in the ongoing Phase 2b study of AMT-061; an investigational AAV5-based gene therapy containing a patent-protected  FIX9-Padua (Factor 9) for the treatment of patients with severe and moderately severe hemophilia B. The announced data, which …

Now You See it, Now You Don’t Deliberate Omissions of Real Facts Real facts might be displayed or concealed depending on the news announcers’ or bloggers’ self-interests. The following contains cases of deliberate or unintentional omission of facts that led to the decline of fundamentally sound biotech and biopharmaceutical firms in bullish market during the departed trading week . . . This content is for paid subscribers. Please …

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 …

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 …

Novartis’ (NVS) financials results exceeded analysts’ expectations. The firm’s research institutions’ strategy, well-designed structure, plans for innovation and accurate execution led to a wealthy and competitive pipeline with more than 200 projects in clinical development. Novartis is flying into the future finding its way to more effective treatments and possible cures. As a matter of fact, Novartis is at the end of the road towards …

Prohost Letter #422 SUMMARY OF THE PAST ISSUE ARTICLE - REVISITING PROMISING FIRMS (PART1) - The firms we tackled in the past issue comprised: 1. Firms we liked and added to our portfolio. 2. Firms we posted their good news, but did not select... 

Prohost Letter #421 PRIVATE  LESSONS - Stock prices of publicly-­traded biotech companies oscillate for the same reasons other industries’ stock prices swing all day long. The fluctuations are the outcome of investors’ and daily traders’ unrelenting buying and selling . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The Week in Review #21 Part 2 THE  MARKET (2) - Our remarks in the past Week in Review included our confidence that the attempts by negative investors to drive the market crazy will not deprive the market from its innate capability to sense and react to the authentic factors it usually reacts to . . . This content is for paid subscribers. Please click here to …

NOVARTIS TO ACQUIRE AVEXIS A few months ago, Novartis (NVS) CEO agreed to cash out of the company’s consumer health joint venture with GlaxoSmithKline (GSK), which brought in cash intended to further improve and expand Novartis’ pipeline. Here we are. Yesterday, Novartis announced its intention to acquire the gene therapy firm AveXis (AVXS) at $8.7 billion, paying $218 for each share of this firm. In a conference call on Monday, …

Prohost Letter #416 Part 2 2017 - A YEAR OF BREAKTHROUGH TECHNOLOGIES, BREAKTHROUGH PRODUCTS, IMPROVEMENTS ON PROMISING BREAKTHROUGH TREATMENTS, DECIPHERING IMPORTANT LIFE SCIENCE MYSTERIES, PINPOINTING BIOLOGICAL PATHWAYS FOR TREATMENT RESISTANCE, MIRACLE CURES, AND… A LOT OF DISTORTED NEGATIVE INTERPRETATIONS - Prioritizing our choices of breakthroughs has been a dilemma . . . This content is for paid subscribers. Please click here to subscribe or here to log …

Prohost Letter #416 Part 1 The Year In Review - 2017 Unheard of Programs - AN AWESOME YEAR for the biotech sector regarding its accomplishments. Many of the accomplished programs were unheard of regarding the degree of their positive impact on medical practice. Unfortunately, many of these accomplishments, which included the making of new discoveries, the creation of novel advanced technologies and the design of …

Prohost Letter #415 New Promising Technologies PROTAC - A Technology That Offers a Superior Class of Drugs PROTAC is an approach that created a new class of drugs described as superior and far less toxic than targeted drugs known to block disease-­causing proteins. The technology belongs to a private Company called Arvinas . . . This content is for paid subscribers. Please click here to subscribe …