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 …

In our yesterday’s article asking about how the FDA advisory committee has to vote regarding Biomarin product Kyndrisa (drisapersen) for Duchenne muscular dystrophy. We got the answer yesterday during the meeting that took a much longer time than committees’ meetings usually take. At the end of the long session that included several patients’ and patients’ relatives’ testimonies, the FDA Advisory Committee was incapable of voting for …

Today, the FDA advisory panel will discuss BioMarin (BMRN) product drisapersen for Duchenne muscular dystrophy based on documents released to the committee by the FDA. The FDA said, “The benefit of drisapersen in exon 51 skip amenable patients is inconclusive at this time. Therefore, the benefit-risk assessments were not made.” Analysts interpreted the FDA statement as a condemnation of the drug, which does not really tell what the FDA meant …

Europe Approves Orkambi® The European Commission has granted Marketing Authorization for Orkambi® (lumacaftor/ivacaftor), for cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation. There are approximately 12,000 people with CF ages 12 and older who have two copies of this mutation. Vertex will now begin the country-by-country reimbursement approval process across the European Union (EU). They said: The …

When we hear about a new approach that promises bringing in hopefulness in patients diagnosed with brain glioma, we try to listen and hope for the best, rather than develop and demonstrate  skepticism. Are we not walking the road of genetic engineering, gene therapy approaches and immunotherapy? Our optimism becomes realistic, especially when a scientific rationale is explained by researchers in scientifically solid firms that …

Food allergies are bad, significant, dangerous and growing in the U.S. and the rest of the world. More than 30 million people in the United States and Europe have a food allergy, and more than five million people in the United States and Europe have peanut allergy, including more than two million children. The prevalence of peanut allergy in children in the United States has increased …

Exelixis (EXEL) received the first of the great news we expected to hear. Today, the U.S. Food and Drug Administration (FDA) approved COTELLIC™ (cobimetinib) a cancer drug discovered by Exelixis in combination with Roche’s drug vemurafenib for BRAF V600E or V600K mutation-positive unresectable or metastatic melanoma. COTELLIC is a selective MEK inhibitor is subject of a worldwide collaboration agreement between Exelixis and Genentech (Roche). Switzerland had …

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 …

To shareholders who were stupefied with an instigated selloff of Incyte’s (INCY) stock today, we say do not get panicky. Nothing is wrong with Incyte, no bad news was announced with any of the firm’s 12 ongoing clinical trials or with its approved drug Jakafi, which demonstrated progressive growth since it was launched.  So, a clever correct move now would be taking advantage and accumulate …

– Shire to Acquire Dyax for $5.9million and some possible extras. – Angioedema market has treatments for attacks, but not prophylactic products for the prevention of HAE attacks. – A primary reason for the acquisition, but not the only one, is getting to Dyax’s prophylactic Hereditary Angioedema (HAE) product. – What will be the outcome of Biocryst’s lead HAE prophylactic drugavoralstat? -Will Biocryst be able …

Prohost Letter #386 An Evaluation of Prohost Picks and Some Other Interesting Biotechnology Companies (Part 2) (Continued from Previous Prohost Letter #385) See Page Listening to Top-tier biotech firms’ presentations we could not resist appreciating their accomplishments, which made us dare to predict that the time has come for the self-serving preachers to find it extremely hard to convince shareholders to to sell stocks of …

Another Durable Botulinum Toxin with Many Advantages REVANCE THERAPEUTICS As per its self- described profile, Revance Therapeutics (RVNC) manufactures and develops novel botulinum toxin products for multiple aesthetic and therapeutic indications. The company is leveraging its proprietary portfolio of botulinum toxin compounds combined with its patented TransMTS® peptide delivery system to address unmet needs in the growing markets. Revance’s proprietary TransMTS technology seems to enable …

POSITIVE TOP-LINE RESULTS FROM A PHASE 2 TRIAL OF OMECAMTIV MECARBIL IN PATIENTS WITH CHRONIC HEART FAILURE Data Showed Statistically Significant Improvements in Several Pre-Specified Measures of Cardiac Function Amgen (AMGN) and Cytokinetics (CYTK): Data from the expansion phase of COSMIC-HF – a Phase 2 trial evaluating omecamtiv mecarbil in patients with chronic heart failure, showed statistically significant improvements in several measures of cardiac function, including systolic …

The FDA issued a warning that hepatitis C treatment Viekira Pak and Technivie can cause serious side effects and serious liver injury, mostly in patients with underlying advanced liver disease. The drugs in question belong to AbbVie (ABBV) with some of their  ingredients made by Enanta (ENTA), both were subjected to selloffs after the news, in addition to Express Script (ESRX) Holding, which  operates as a …

– Exelixis submitted rolling NDA for cabozantinib. – Exelixis plans to complete the submission in the next two months. – Positive Results from the METEOR trial with cabozantinib on advanced renal Cell carcinoma led the FDA to grant the drug breakthrough designation. Based on good results from a Phase 3 from the METEOR pivotal trial, Exelixis (EXEL) initiated submission of its rolling New Drug Application (NDA) to …

Prohost Letter #385 An Evaluation of Prohost Picks and Some Other Interesting Biotechnology Companies The Top-Tier Group The indiscriminate attack on the biotech sector impacted the best biotech firms more than it did the worst. In other words, the attack devastated the undervalued, without correcting the stock price of overvalued, or even valueless, firms. The problem is that those who instigate computer programs selling of …

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 …

Incyte (INCY) and Merck (MRK) announced the expansion of their ongoing clinical collaboration to include a Phase 3 study evaluating the combination of epacadostat,Incyte’s investigational selective IDO1 inhibitor, with Keytruda® (pembrolizumab), Merck’s anti-PD-1 therapy. The trial is for the use of the combination as first-line treatment for advanced or metastatic melanoma. The Phase 3 study, which is expected to begin in the first half of 2016, …

Stories from Yesterday’s Market We are not claiming that the market volatility has subsided and the leadership has shown and is in full control of the chaos. But yesterday biotech stock performance made us feel that investors are regaining their common sense and trying to benefit from the catastrophic plummeting of the member firms stock prices. Today’s trading seems negative for the biotech stocks, but …

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 …