Celyad (CYAD) is still a clinical-stage biopharmaceutical company developing advanced or improved CAR-T cell-based therapies. Today, Celyad announced that the first patient has received an injection in Phase 1 of the alloSHRINK clinical trial, evaluating its non-gene edited allogeneic CAR-T therapy known as CYAD-101. In this trial, FOLFOX chemotherapy is used simultaneously with CYAD-101 on patients with unresectable metastatic colorectal cancer (mCRC). According to Dr. Christian Homsy, …

We hope you all took advantage of the Thanksgiving holiday to rest and relax. We did. In Today’s NEWS RegenxBio Offers Rocket Pharmaceuticals exclusive, worldwide, rights to adenoviral vector NAV AAV9 for the development of treatments for Danon Disease RegenxBio (RGNX) announced that it awarded an exclusive, worldwide, license agreement to Rocket Pharmaceuticals (RCKT) so it can develop gene therapy for Danon disease. The exclusive license …

This is just to inform that Europe has approved Cabometyx® (cabozantinib) tablets as a monotherapy for hepatocellular carcinoma (HCC). As you know, Cabometyx belongs to Exelixis (EXEL). Today Exelixis announced that its partner, Ipsen, received approval of the drug, from the European Commission (EC), to be used in adults who have HCC and have previously been treated with sorafenib. This approval allows for the marketing of Cabometyx in this indication in …

The Week in Review, which we will post soon, is going to provide news that cancer patients, their parents, their oncologists and investors are anxiously waiting to hear.   The news is about studies with products demonstrating huge improvements in immunotherapy efficacy in addition to preventing checkpoint inhibitors such as: Opdivo, Keytruda and others, from causing adverse effects, including autoimmune reactions that are serious and …

Yesterday, the Stock Market skyrocketed and with it a significant number of Prohost Picks that we keep reminding ourselves are undervalued. The outperformance unveiled the interred strength of firms such as: Illumina (ILMN), Amgen (AMGN) and Vertex (VRTX), the gene therapy firms: RegenxBio (RGNX) and Spark (ONCE), and the gene editing firms: Crispr (CRSP), Intellia (NTLA) and Editas (EDIT), as well as others in the Prohost Portfolio. The rebounding in the …

Exelixis (EXEL) provided an update that testified to its solid fundamentals, the growth of its approved products’ sales, the professionalism and seriousness of its management. Revenues: Exelixis’ total quarter revenues were $225.4 million vs. $152.5 million for the comparable period in 2017. Its net product sales revenues were $162.9 million for the quarter compared to $96.4 million for the comparable period in 2017, which represents a 69 percent increase year-over-year. …

Celyad (CYAD) announced an exclusive agreement with Horizon Discovery Group plc, for the use of its shRNA technology to generate Celyad’s second non-gene-edited allogeneic platform. The encouragement came from data emanating from preclinical studies demonstrating the versatility of the shRNA platform in the allogeneic setting. The data is expected to be presented at the upcoming 2018 Society for Immunotherapy of Cancer (SITC) annual meeting in Washington, D.C., …

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 …