Sarepta Therapeutics in the NEWS On June 20, 2024, Sarepta Therapeutics (SRPT) announced that the U. S. FDA has approved an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age. Confirming the functional benefits, the FDA granted . . . This …

Sarepta Therapeutics in the NEWS On 12, 22, 2023, Sarepta Therapeutics (SRPT) - a precision genetic medicine for rare diseases, announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows: “ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed . . . This content is for paid …

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 …

Sarepta Therapeutics (SRPT) has news from preliminary Duchenne muscular dystrophy trials. Although the number of recruited patients was only three, the strong positive data excited the media and Wall Street investors who rushed to buy the stock, making it add over $50 in today’s morning trading. The Story At the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from Phase 1/2a …

The Week in Review #22 What Went Right or Wrong in the Biotech Sector During the Departing Week? - A lot happened in the biotech sector during the week, which departed at midnight. Topping the list of the week’s events was the approval of Portola’s (PTLA) product  Andexxa . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The Week in Review #6 A lot has happened in the past week regarding treatments of devastating diseases. One of the accomplishments was the outcome of the revolution in life sciences’ research and capabilities. This is the discovery of a protein that causes the growth of the malignant brain tumor glioblastoma . . . This content is for paid subscribers. Please click here to subscribe or …

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 FDA approval of Sarepta’s (SRPT) Duchenne muscular dystrophy (DMD) product, Exondys 51 (eteplirsen), surprised many analysts. Nonetheless, it doubled SRPT's price after the stock had already multiplied everal folds since the stock had been cremated. Good for those who dared to bet on SRPT in spite of the FDA’s independent advisory committee’s voting against the approval and the FDA hesitation to listen to the …

Pfizer accepted to pay $14 billion in cash to acquire Medivation. It is important for investors to know what Pfizer is after with regard to this acquisition so that investors could sense the current deep-pocketed biopharmaceutical firms’ preferences in selecting their takeover targets. Recognizing the facts behind Pfizer’s decision could also protect investors from being mislead by negative bloggers who, while serving their own agendas, …

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 …

Prohost Letter #394 Part 1 Gilead - Some people sold Gilead’s (GILD) stock on Wednesday, March 23 after news announced on Tuesday that a jury decided that Gilead has infringed on two HCV molecules patents that Ionis (IONS), formerly Isis, and Merck had secured from their research. Ionis CEO said in a press release that his firm and Merck used their expertise during their collaboration in …

Prohost Letter #389 Happy New Year 2016 Wishing You Good Health Peace of Mind Love And happiness  We expect 2016 to be the year that fulfills many of our great expectations about successfully managing intractable and life-threatening malignant and non-malignant disease. We expect the biotech firms to bring us more cures based on the maturation of genetic engineering processes following a lot of trial and …

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 …

About Duchenne muscular Dystrophy treatments, bloggers tried to explain the reasons that led the FDA to decide upon reviewing BioMarin’s (BMRN) Duchenne muscular dystrophy (DMD) drug Drisapersen a few weeks earlier than reviewing Sarepta (SRPT) DMD drug Eteplirsen. The real reasons for the FDA decision remain inside the FDA mind. No outsider will be allowed to know anything related to NDA reviews. All the talk and …

Prohost Letter #384 The Assassination of the Biotech Sector The Market (Part 2) On Friday September 18, 2015, the biotechnology sector occupied the news headlines for two different reasons. The first was the sudden unexplicable and unexpected plummeting of the biotech sector. The stocks were flying sky high until modday and suddenly, all the biotech stocks, without exception, took a dive. The second reason was the triumph of …

Prohost Letter #383 The Market - Recent tails about the market and the biotech sector are true thrillers. By true we mean no-fiction. The accounts lack common sense. Professionals' interpretations of the stocks' and of the market's moves are bizarre and misleading. General investors complain only when the stocks' volatility gets out of hand, yet, do not object to the hype, or interferes against the baseless …

The biotechnology industry is proving to be the most innovative and the most growing among Wall Street industries. Individual biotech companies’ achievements complement each other. Some are evolving the sequencing of genes and genetic analysis, while others are excelling in designing drugs based on the tremendous amount of facts they are getting on root causes of the diseases and the interaction of proteins. The biotech …

but first: Here is some good news from  SAREPTA Yesterday we wrote about BioMarin (BMRN) being granted FDA “Rare Pediatric Disease Designation for its Duchenne potential muscular dystrophy (DMD) drug drisapersen.Today, Sarepta Therapeutics (SRPT) also a developer of RNA-targeted therapeutics has been granted the same for its drug eteplirsen, also a potential treatment for DMD patients who are amenable to skipping exon 51. The Rare …