Although the tests are for the prevention of a life-threatening metabolic disorder, the prenatal animal testing may be offering the potential to treat many human congenital diseases before birth. The research, which comes from Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania, is published in Nature Medicine. It offers proof-of-concept for prenatal use of a sophisticated, low-toxicity …

IMMUNOTHERAPY  The Checkpoint Inhibitors The most revered news was the discovery of the checkpoint proteins on the surface of the immune system cells. The checkpoint proteins’ natural job is to put a break on the immune system cells to prevent them from attacking people’s own body cells. An immune attack on host cells can lead to disabling and life-threatening autoimmune diseases. Long before the scientists …

Why Portola’s Stock Decline Everybody interested in Portola (PTLA) knows by now that this firm has managed to put two FDA-approved products on the market. They are: Bevyxxa® (betrixaban): The first and only once-daily oral Factor Xa inhibitor, for the prevention of deep vein thrombosis (DVT), that subject them to venous thromboembolism (VTE), which could have life-threatening lung embolism. Andexxa®: The first and only antidote for patients treated with rivaroxaban and apixaban when reversal …

VTV Therapeutics’ (VTVT) stock price added over 48% before trading hours today. The news that some believe is behind investors’ enthusiasm is the firm’s announcement that it will deliver two presentations at the 11th clinical trials on Alzheimer’s Disease (CTAD) Conference in Barcelona, Spain. The oral VTV Therapeutics’ (VTVT) presentations are scheduled for October 24-27, 2018. VTV Therapeutics is a clinical-stage biopharmaceutical company focused on …

In two pivotal Phase 3 placebo-controlled trials Regeneron Pharmaceuticals’ (REGN) and Sanofi’s (SNY) drug, Dupixent® (dupilumab), demonstrated a significant reduction in nasal polyp size, nasal congestion’s severity and the need for systemic corticosteroids and/or surgery. Both trials were evaluating Dupixent® (dupilumab) in adults with inadequately-controlled chronic rhinosinusitis with nasal polyps (CRSwNP). They both met met all their primary and secondary endpoints. Dupixent is a monoclonal antibody designed to …

On Saturday, October 6, 2018, Spark Therapeutics (ONCE) presented positive data from a preclinical trial with SPK-3006 for the treatment of Pompe disease in an oral presentation at the 23rd International Congress of the World Muscle Society in Argentina. SPK-3006 is an investigational liver-directed adeno-associated viral (AAV) gene therapy being developed for Pompe disease. Pompe disease is caused by the buildup of a complex sugar, glycogen, in the body’s cells. It is …

Today, Prohost is highlighting news that could be transformative for a clinical-stage immunotherapy firm. The firm with promising news focuses on creating and developing safer and far-reaching Chimeric Antigen Receptor T-cell (CAR T) Therapy.   The firm we are highlighting today is Celyad (CYAD), whose investigational CAR T products are allogeneic, rather than autologous, which makes it easier to use and less expensive than the autologous products. …

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. …

Seattle Genetics (SGEN) and Takeda Pharmaceutical Limited (TKPYY) announced results from Phase 3 ECHELON-2 clinical trial evaluating Adcetris as part of a frontline combination chemotherapy regimen in patients with previously untreated CD30-expressing peripheral T-cell lymphoma (PTCL), known also as mature T-cell lymphoma (MTCL). The data from the trial with Seattle Genetics' conjugated monoclonal antibody Adcetris (brentuximab vedotin) in combination with CHP (cyclophosphamide, doxorubicin, prednisone) versus the control …

Stage III (locally-advanced) NSCLC is commonly divided into three sub-categories: IIIA, IIIB and IIIC, defined by how much the cancer has spread locally and the possibility of surgery. The difference between Stage III and Stage IV disease (cancer (metastasized to distant organs)) is that Stage III is treated now with curative intent. The majority of Stage III NSCLC patients are diagnosed with unresectable tumors. It …

Compugen Again On Sept 10, 2018, we posted in The Week in Review #28 news we found very interesting about Compugen (CGEN). The news announced that Compugen has dosed its first patient in Phase 1 clinical trial with its first-in class immunotherapy antibody product COM701 targeting the firm's in house discovered immune checkpoint protein PVRIG. In The Week in Review article we wrote in the beginning, investors …

Portola Pharmaceuticals Appoints Scott Garland as President and Chief Executive Officer, Effective October 8, 2018. Portola (PTLA) appointed a new president, Mr. Scott Garland, who was Relypsa’s president and previously chief commercial officer and responsible for the integration and growth of the U.S. operations after Relypsa was acquired by Vifor Pharma Group in 2016. Prior to Relypsa, Mr. Garland was executive vice president and chief commercial officer of Exelixis, Inc., …

CRISPR Gene Editing Beyond Bringing Cures to disease-causing genes, CRISPR gene editing technology can change the way diseases are being treated and treat diseases that have yet to find a treatment. CRISPR gene editing can play a key role in solving a lot of problems that prevent breakthrough treatments and approaches from reaching their ultimate goals. A best example is chimeric antigen receptor (CAR T) Cell immunotherapy approach to …

Today, we see are observing an increased interest in AcelRx Pharmaceuticals (ACRX). The increased investors’ activity happened following the announcement that an Advisory Committee meeting will soon review the firm’s New Drug Application (NDA) for Dsuvia (Sufentanil) developed for the management of moderate-to-severe acute pain in medically supervised settings in adult patients. Sufentanil is an opioid analgesic currently marketed for intravenous (IV) and epidural anesthesia …

Playing buy and sell games with the CRISPR gene editing stocks is sometimes based on scientific articles that meant to caution about possible problems or by patents that were given to some Universities and denied it to others and vice versa. Different Universities back different CRISPR gene-editing firms, which constituted a reason for the volatility of the gene-editing stocks. Finally, CRISPR Therapeutics (CRSP), Intellia Therapeutics …

The disease, mucopolysaccharidosis (MPS) type II, also known as the horrible Hunter syndrome is a killer with no specific treatment anywhere, except enzyme replacement therapy (ERT) that is not stopping the complications of the disease or preventing death. Sangamo (SGMO) made a presentation at the Society for the Study of Inborn Errors of Metabolism in Athens, Greece to demonstrate preliminary results, which included the safety …

A complete response letter (CRL) sent by The Division of Metabolism and Endocrinology Products of the United States’ FDA to Ionis Pharmaceuticals (IONS) and its affiliate Akcea Therapeutics (AKCA) regarding Waylivra™ (volanesorsen) carried like usual bad news. The FDA has decided against approving Waylivra, the drug designed to treat the lipid disorder known as familial chylomicronemia syndrome (FCS). Familial chylomicronemia syndrome is an ultra-rare, devastating hereditary …

Portola (PTLA) has two important approved breakthrough products and an investigational product having promising results on very difficult-to-treat cancers. Instead of soaring following the approval of its two drugs and the good results coming from the investigational third drug, Portola’s stock took several dives, which raised the eyebrows of many investors and motivated more shareholders to sell PTLA. Let us see whether the firm deserves …

WE ARE LIVING AN HISTORIC BIOLOGICAL REVOLUTION. Unfortunately, the undeniably outstanding news has not yet penetrated the ears of many investors. They are still falling victim to the stock market traders’ games and to critics’ and bloggers’ magnifications or fabrications of negative news. Many investors end up selling their thoughtfully chosen highly scientific biotech firms to regret later on what they have done when their …

Vertex Pharmaceuticals (VRTX) announced that the Pharmaceutical Benefits Advisory Committee’s (PBAC) in Australia recommendation the listing of Orkambi® (lumacaftor/ivacaftor) on the Pharmaceutical Benefits Scheme (PBS). The recommendation is for cystic fibrosis people ages six and over who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Many thousands of patients worldwide are already receiving lumacaftor/ivacaftor in countries where it is reimbursed …