Alnylam (ALNY) announced that Sanofi Genzyme declined its opt-in for the development and commercialization of the RNAi product lumasiran, an investigational RNAi therapeutic for the treatment of Primary Hyperoxaluria Type 1 (PH1). Based on this decision, Alnylam intends to rapidly advance lumasiran into a Phase 3 pivotal study in late 2018, and to commercialize lumasiran globally, assuming product approval. Alnylam has also announced that the FDA has granted lumasiran …

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. …

The Week in Review #16 More Exciting Rallies in Prohost Picks - The past week and for several week fascinating outperformances have been seen for many of the Prohost Portfolio stocks. We continue to articulate that the good news is not only in the stocks’ rallies, but also in the outstanding stories behind them . . . This content is for paid subscribers. Please click here …

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 …

The Week in Review #11 A New Era - Faster than the speed of light, the management of diseases is shifting from fast improvement to a total gigantic unprecedented change. Some of the conquering approaches towards providing long-­lasting cures look as if the outcome of magic rather than mere science . . . This content is for paid subscribers. Please click here to subscribe or here to log …

The Week in Review #5 ALNYLAM (ALNY) and Sanofi Genzyme, the specialty care global business unit of Sanofi announced that the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for hereditary ATTR amyloidosis with polyneuropathy, met its primary efficacy endpoint and all secondary endpoints. The primary endpoint for the study was the change from baseline in the modified neuropathy impairment score (mNIS+7) at …

Alnylam Pharmaceutical (ALNY) calls RNAi (RNA interference) a revolution in biology. The FDA’s just granted the firm’s RNAi therapy breakthrough for a disease nobody has ever dreamed of conquering confirms this reality in spite of spending years of efforts and money towards turning the RNAi molecules into therapeutic molecules that can be used safely and effectively. RNAi-related treatments represent a completely new approach to drug …

An approved rheumatoid arthritis drug developed and sold by Sanofi could become a new treatment for aggressive cancers. The news came in a Mount Sinai press release announcing that researchers have discovered that a rheumatoid arthritis drug can block a metabolic pathway that occurs in tumors with a common cancer-causing gene mutation. The discovery offers a new possible therapy for aggressive cancers with few therapeutic …

Prohost Letter #404 Part 2 Synthetic Biology (2) A Brief History DNA is the software of life. This is how Synthetic Genomics firm, which we believe is now is the leading biotech firm for applied synthetic biology. Synthetic Genomics is still a private firm in spite of that thousands of investors are impatiently waiting for it to turn public and it will. So stay tuned. …

Prohost Letter #404 Part 1 HAPPY SPIRITUAL HOLIDAYS HAPPY NEW YEA

Following recent reports of new onset or worsening peripheral neuropathy in patients with ATTR amyloidosis with cardiomyopathy (hATTR-CM) treated with Alnylam’s (ALNY) RNAi –based drug revusiran, The firm requested that the Data Monitoring Committee (DMC) review the reports and the Clinical trial data on an unblinded basis. After reviewing the reports, the DMC advised suspending the dosing of revusiran. However, the DMC stated that It …

Medivation is a Target for Acquisition The Question is: Who Will Acquire Medivation? Sanofi has made a bid to acquire Medivation for $9.3 billion (£6.37B). Sanofi is expected to pay $52.50 per share in cash for Medivation. Why Medivation? Sanofi's chief executive Olivier Brandicourt said, "Thanks to Medivation's excellent prostate cancer treatments, we believe that a merger with Medivation would benefit patients and, at the same time, create value …

Regulus Pharmaceuticals (RGLS) a biopharmaceutical company that discovers, designs and develop treatments targeting microRNAs announced interim results from one of the company’s ongoing Phase 2 studies of its hepatitis C virus (HCV) product RG-101.  The study was designed to evaluate a shortened, four-week treatment regimen containing a subcutaneous administration of 2 mg/kg of RG-101 at Day 1 and Day 29, in combination with 4 weeks of once/daily approved anti-viral …

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 #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 …

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 …

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, …

Prohost Letter #377 PICKING BIOTECH FIRMS Before Investing in Them Raising the bar in designating small drug developing firms as biotechnology firms has occurred several times since 2011. In that year, many technologies were tremendously enhanced, which enabled them to realize superior goals, i.e., produce safer and more effective breakthrough drugs for chronic debilitating and life-threatening diseases. Prohost Portfolios picks, which have outperformed other industries’ …