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 …

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.

PTC Therapeutics’ (PTCT) product Risdiplam demonstrated safety and tolerability at all the doses used in the studies on Type 1, 2 and 3 spinal muscular atrophy (SMA). The good news emanated from interim data of the Part 1, open-label studies of FIREFISH and SUNFISH trials, with the oral drug Risdiplam on SMA patients. Risdiplam was dubbed effective,  substantiated by a confirmed increase in motor function. …

Before we sell PTC Therapeutics (PTCT), after the stock had reached and surpassed our latest and highest 2018 target, the firm did the right thing that we expected was good enough to encourage investment in this firm.  The good thing is acquiring Agilis Biotherapeutics – a small firm that succeeded in creating innovative gene therapy programs for rare genetic disorders that affect the central nervous …

Yesterday’s news unveiled that PTC Therapeutics (PTCT) has entered into an agreement to acquire Agilis Biotherapeutics. The transaction was approved by the Boards of both companies. The acquisition Agilis Biotherapeutics is expected to bring to PTC Therapeutics an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system. It is supposed to add to PTC Therapeutics’ pipeline an advanced gene therapy candidate, …

IN THE NEWS PTC THERAPEUTICS  PTC Therapeutics (PTCT) stock, which dipped yesterday, probably on news announcing a public offering of 4,000,000 shares of its common stock at a public offering price of $27.04 per share. Yesterday, in a crazy market that looked as if the bull and bear are boxers knocking each other down every 10 minutes, PTCT lost around $5. The stock seems to rebound in today’s premarket …

The Week in Review #19 Targeting the Root-­Cause of Diseases - Investing in the Future is Currently the Best Way - Stopping aberrant genes from causing diseases has taken time to move from wishful thinking to reality. Through trial and error, various different approaches have been tested over the years, including attempts at genetic engineering... &nbsp . . . This content is for paid subscribers. …

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.

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 …

PTC THERAPEUTICS Why Buying Emflaza? Buying Emflaza, a steroid approved by the FDA for Duchenne muscular dystrophy (DMD) from Marathon Pharmaceuticals invited an intractable headache to PTC Pharmaceuticals’ shareholders and scrutiny by some law makers. The cause of the law makers’ involvement has been the Marathon’s pricing Emflaza at $89,000, but PTCT shareholders had several question marks revolving around the motive for buying this drug …

Prohost Letter #401 SYNOPSIS PART 1 In this Letter CONTENTS Duchenne Muscular Dystrophy (DMD) FDA Compassionate Approval of Eteplirsen! PART 2 In The Letter Issue #402 CONTENTS EYE ON IONIS (IONS) Antisense Proof of Concept and Feasibility EYE ON INVIVA (INVA) AND THERAVANCE BIOPHARMA (TBPH) Sales of The Ellipta Inhalers Started to Pick up Before an expected Boom Not Losing . . . This content …

The media failure to forecast the results of the Presidential election has become the talk of the town and of the failed media itself. The New York Times’ article about this forecast fiasco titled “How Data Failed Us In Calling An Election” was great, showing that forecasting science is still immature to bring accurate results. The article stated that the failure in predicting the winner …

Prohost Letter #396 CELEBRATING MEMORIAL DAY - Wish you celebrated the Memorial Day. Memorial Day’s purpose was remembering the people who died while serving in the country’s armed forces. The Memorial Day was initiated as Decoration Day after the Civil War in the year 1868 when the Grand Army of the Republic - an organization of Union Veterans in Illinois established it to decorate the graves …

EXELIXIS AGAIN They said: Sell EXEL, as no partner is interested in the firm’s FDA approved cabozantinib to date. What really happened: It did not take long for IPSEN to come forward and take the responsibility of marketing cabozantinib abroad and pay $200 million upfront payment to Exelixis. They said: Although the FDA approved Cabometyx (cabozantinib) for advanced renal cell carcinoma (RCC), the drug will …

In the biotech sector, daily stories (news) are not just announced, but mostly edited in ways that project the narrators’ tendencies and wishes rather than the original news. Last week’s headlines comprised positive news from a firm’s drug aimed at treating a chronic progressive disease, muscular dystrophy, which disables children’s by weakening their voluntary and involuntary muscles, while threatening their hearts and lungs and lives. Other headlines …

Prohost Letter #388 TOPICS - 1. AN INDISPENSABLE SHORT INTRODUCTION 2. GENE EDITING 3. GENE THERAPY 4. CANCER IMMUNOTHERAPY 1. GENE EDITING Exon Skipping: Biomarin (BMRN); Sarepta (SRPT); These firms are waiting FDA approvals for Duchenne Muscular Dystrophy (DMD) Gene Editing Technology Competitors: Cellectis S.A. (CLLS); CRISPR; PTC Therapeutics (PTCT) Firms Using Different Approaches For DMD Treatment (see Below) Zink Finger Tech: Sangamo (SGMO); RNAi …

Prohost Letter #387 Topic of the Week DUCHENNE MUSCULAR DYSTROPHY Which Firms Will Get It Right – Deleting DMD Children’s Despair? The topic is important because, first and foremost, Duchenne muscular dystrophy (DMD) disables children. Also because, from no treatment at all, now we have several biotech firms developing various drugs based on different scientific strategies. The new approaches have increased in number and have …

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, …