PTC Therapeutics Inc On March 20, 2025, PTC Therapeutics, Inc. (PTCT) shared new data from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU . . . This content is for paid …

PTC Therapeutics in the NEWS Today, December 2, 2024, PTC Therapeutics (PTCT) announced the signing of an exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG (

PTC Therapeutics Announcement Today, November 13, 2024, PTC Therapeutics, Inc. (PTCT) announced that the U.S. FDA accelerated approval of the first gene therapy for the treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. This is the first-ever gene therapy to be approved in the United States that is directly administered to the brain. From PTC . . . This content is for paid subscribers. Please click here …

Eye on Reata Pharmaceuticals Recently, Reata Pharmaceuticals (RETA) announced a setback of its chronic kidney disease product known as bardoxolone. The failure of this drug occurred after Reata's product Skyclaris was FDA approved for the treatment of Friedrich's ataxia (FA) - a rare genetic disease that causes difficulty walking, loss of sensation in the legs and arms . . . This content is for paid …

Arcturus Therapeutics Holdings News Arcturus Therapeutics Holdings (ARCT) shared topline data from an ongoing Phase 1/2/3 trial evaluating ARCT-154, Arcturus’ self-amplifying mRNA vaccine candidate against COVID-19. The ongoing Phase 1/2/3 registrational study, sponsored by Arcturus’ collaborator Vinbiocare Biotechnology Joint Stock Company – a member of Vingroup Joint Stock Company, enrolled over . . . This content is for paid subscribers. Please click here to subscribe or here to log …

PTC Therapeutics Approvals and Achievements PTC Therapeutics (PTCT) has achieved a lot recently. The latest is the August approval from the U.S. FDA of its product Evrysdi (risdiplam), the first orally administered at-home treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older.   Evrysdi has been granted approvals in Brazil, Chile and Ukraine as . . . This content is …

Halozyme and PTC Therapeutics: Near-Term Game-Changing PDUFAS Some of the Prohost Portfolio selected companies are having near term PDUFAS which we believe will have a positive outcome. Indeed the FDA approvals are expected to tremendously boost these firms' values. Today we present two selected Biotech firms from the Prohost Portfolio.   Halozyme Halozyme (HALO) has two upcoming important

Ionis Pharmaceuticals Ionis Pharmaceuticals (IONS) announces a private placement of $507.5 million principal amount of 0.125% convertible senior notes due 2024. To read this news go to the original  content to download multimedia by clicking here.  During the announcement of 2019 third-quarter financial results the conference call started citing the firm's improved revenues and income. Revenues have increased by more than 50% to nearly $630 million and …

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 …

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 …