Alnylam (ALN) is probably experiencing a stock selloff following the announcement of successful results from Pfizer’s (PFE) drug tafamidis.    Indeed, results from Phase 3 ATT-ACT clinical trial demonstrate that tafamidis succeeded in treating Transthyretin Cardiomyopathy, a subtype of TTR amyloidosis caused by the destabilization of the protein transthyretin. Tafamidis demonstrated a statistically significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months. The drug was …

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 …

A False Reason for Unwarranted Selling of CRISPR/Cas9 Gene Editing Shares     CRISPRs are bacterial immune systems that contain many defense enzymes such as the Cas9 “molecular scissors,” which scientists have engineered as a powerful DNA-targeting gene-editing tool and created companies specialized in CRISPR gene editing tools. The outstanding revolutionary news, which is wrongly interpreted by Wall Street, which led to a recent selling of the shares of CRISPR gene …

The Week in Review #20 THE MARKET - My friend asked: “Are  you  scared?” “Of  what?” I asked “The  market?” The  market? No,” I  said.  We are afraid of those who are rendering the market nervous and  temporarily confused, as we observed while monitoring its volatility.  The market has never been chronically confused . . . This content is for paid subscribers. Please click here to …

Exelixis: Delightful Overseas Cabozantinib News Ipsen (EPA:IPN) announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA) provided a positive opinion for Exelixis’ (EXEL) product Cabometyx® (cabozantinib) 20, 40, 60 mg regarding the first-line treatment of adults with intermediate- or poor- risk advanced renal cell carcinoma (aRCC). The CHMP positive opinion will be reviewed by the European Commission (EC), which has the …

Regeneron (REGN) and Alnylam (ALNY) announced a collaborative agreement aimed at identifying RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis (NASH) and potentially other related diseases. The agreement is not the first between the two firms, but it would not have occurred if not for Regeneron’s identification of an important and unique therapeutic target that influences the liver debilitating and life-threatening nonviral hepatitis diseases, which affect thousands …

In spite of their successes, Immuno-oncology products, which we still like to call immunotherapy for several reasons, are a work in progress. The most two highlighted approaches that enable the immune system to clear cancers are the checkpoint inhibition and the chimeric antigen receptor (CAR T) genetically engineered immune system T cells therapies. Therapeutic vaccines had some successes, but the outcome was inferior to the two approaches that have …

Arena Pharmaceuticals (ARNA) announced positive Phase 2 results from the OASIS trial for etrasimod in ulcerative colitis (UC). The Drug: Etrasimod is a selective sphingosine 1-phosphate (S1P) receptor modulator. Patients receiving the 2 mg dose of the drug achieved statistically significant improvements versus placebo in the primary, all secondary, and clinical remission endpoints. Relative to placebo: there was a statistically significant improvement in a 3-component (stool frequency, rectal bleeding, and findings on endoscopy) Mayo Clinic …

What Do We Make of the Following Good News? HERON THERAPEUTICS Will HTX-011 Help in the War Against Opioids?  Heron Therapeutics (HRTX), a commercial-stage biotechnology company announced positive results from its completed Phase 3 studies of the investigational agent HTX-011 in people undergoing bunionectomy and hernia repair. HTX-011 is described as the first and only long-acting local anesthetic to demonstrate in Phase 3 studies significantly reduced pain …

Exelixis (EXEL) completed the submission of a supplemental New Drug Application (sNDA) to the FDA for Cabometyx® (cabozantinib) tablets for previously treated patients with advanced hepatocellular carcinoma (HCC). The sNDA submission is based on results from the CELESTIAL randomized pivotal phase 3 trial of Cabometyx in patients with advanced HCC who received prior sorafenib. Some History: On October 16, 2017, Exelixis announced that the independent data …

Prohost Letter #419 RE-­EVALUATION ONLY - The Prohost Portfolio comprises biotechnology and biopharmaceutical companies, that we believe have solid scientific fundamentals and dedicated managements. All the firms in the portfolio, except a few, have proprietary technologies, which enable the firms’ scientists to design in house safer and more effective new molecular entity therapeutic molecules . . . This content is for paid subscribers. Please click here …

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. …

Extremely important news has come from Montreal, Canada carrying the announcement by Theratechnologies (TSX:TH) and its partner TaiMed Biologics (TaiMed) that the FDA has granted approval of Trogarzo™ (ibalizumab- uiyk) Injection. The approval is for Trogarzo to be used with other ARTs for HIV-1 infection in heavily treated adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen. Trogarzo™ is a humanized monoclonal antibody created for multidrug resistant …

Regeneron’s (REGN) pipeline products have still a lot of good news to announce. The latest is about its product Dupixant (dupilumab) which has already been FDA approved for atopic dermatitis. Now, there are reasons to believe that this drug could be also approved as an add-on maintenance for people with moderate to severe Asthma. The news has come from both Regeneron and its partner Sanofi (SNY), stating that …

On March 4, 2018, Aimmune Therapeutics (AIMT) – a biopharmaceutical company developing treatments for potentially life-threatening food allergies confirmed the superior news that brings hope to children and adults who are susceptible to peanut allergic reactions. The results from the firm’s pivotal Phase 3 PALISADE trial of its product AR101 for the treatment of peanut allergy were presented at the 2018 American Academy of Allergy, Asthma & Immunology–World Allergy Organization Joint Congress …

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.

FERTILITY Fertility is what guarantees the presence of humans on the planet Earth. The reproductive system in men and women are the engines of fertility that creates living beings. These important engines get disturbed in some people, which represents a problem that needs repair. Life sciences arts have done a good job towards overcoming the causes that would prevent a couple from bearing children. Still …

The Food and Drug Administration (FDA) approved AstraZeneca (AZN) Checkpoint inhibitor drug Imfinzi® (durvalumab) for patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy (CRT). Explaining the importance of this approval, Scott J. Antonia, M.D., Ph.D., Chair of the Thoracic Oncology Department at the H. Lee Moffitt Cancer Center and Research Institute in Tampa, Florida and investigator …

Kite, a Gilead Company, and Sangamo Therapeutics Announce Collaboration to Develop Next-Generation Engineered Cell Therapies for Cancer Kite, a Gilead (GILD) company, and Sangamo Therapeutics (SGMO) announced a worldwide collaboration agreement for using Sangamo’s zinc finger nuclease (ZFN) technology platform for the development of next-generation ex vivo cell therapies in oncology. Kite will use Sangamo’s ZFN technology to modify genes to develop next-generation cell therapies for autologous and allogeneic use in treating …