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 …

Tanezumab, an investigational humanized monoclonal antibody developed by Pfizer (PFE) and Eli Lilly (LLY), met all three co-primary endpoints in patients with osteoarthritis (OA). The 16-week Phase 3 study, which evaluated subcutaneous administration of tanezumab in patients with OA, demonstrated that the patients who received two doses of tanezumab, separated by eight weeks, experienced a statistically significant improvement in pain, physical function and patients’ overall assessment …

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 …

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 …

Amgen (AMGN) announced that AmgevitaTM, a biosimilar to adalimumab, will launch in markets across Europe beginning on October 16, 2018. According to Amgen’s press release, AmgevitaTM is the first adalimumab biosimilar to be approved by the European Commission (EC). The EC approval is based on analytical, pharmacokinetic and clinical data, including results from two Phase 3 confirmatory studies of the product for moderate-to-severe plaque psoriasis and moderate-to-severe rheumatoid arthritis patients. The …

Exelixis (EXEL) announced its initiation of a phase 3 pivotal trial (COSMIC-311) of Cabometyx (cabozantinib) in patients with radioiodine-refractory differentiated thyroid cancer (DTC) who have progressed after two prior vascular endothelial growth factor receptor (VEGFR)-targeted therapies. The co-primary endpoints for the trial are progression-free (PFS) survival and objective response rate (ORR). Why conducting trial on patients with radioiodine-refractory differentiated thyroid cancer.    Differentiated thyroid tumors make up …

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. …

The U.S. Food and Drug Administration (FDA) has approved Paratek Pharmaceuticals’ (PRTK) product Seysara™ (sarecycline) for inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients 9 years of age and older. Paratek has exclusively licensed U.S. development and commercialization rights of Seysara for acne to Allergan PLC, who has assigned such rights to Almirall SA. Paratek retains development and commercialization rights in the rest of the world. Seysara (sarecycline) …

The Week in Review #31 IN THE DEPARTED WEEK NOVEL MIGRAINE DRUGS ARE APPROVED ELI LILLY AFTER AMGEN AND TEVA In less than five months, the FDA approved three products belonging to four drug developers – all targeting calcitonin gene-related peptide (CGRP) for the prevention of migraine headache. The latest for Eli Lilly (LLY) last week . . . This content is for paid subscribers. …

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 …

The high risk of premature death from cardiovascular disease is believed to be caused by an elevated level of lipoprotein (a) {Lp(a)}. Elevated Lp(a) is described as an independent, hereditary risk factor for cardiovascular disease (CVD). Diet and exercise, or even currently prescribed cholesterol-lowering treatments do not help the devastating outcome of this condition. Around 8 to 10 million people are living with established cardiovascular …

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 …

The Week in Review #30 FROM THE DEPARTED WEEK In the past week, we came across a lot of news announcing research, clinical trial results, collaborations, product approvals and acquisitions. Much of the departed week’s news though, does not represent complete stories, especially the ones related to research. They represented early works in progress offering no concrete results that we can evaluate. Important news for …

While preparing The Week in Review issue from Prohost, our attention  shifted towards historical news about a procedure that improved the heart function in severe heart failure patients. For cardiologists and their patients, the positive results were surprising, stunning and beyond anticipation. Current treatments of severe heart failure are only symptomatic and do not stop the heart muscle deterioration that results in imminent death. Data …

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., …

Exelixis’ (EXEL) partner Ipsen Biopharmaceuticals Canada Inc. received approval of Cabometyx®(cabozantinib) tablets from Health Canada for adults with advanced renal cell carcinoma (RCC) who have received prior vascular endothelial growth factor (VEGF) targeted therapy. Health Canada had granted Cabometyx priority review status, which resulted in the accelerated review of Ipsen’s new drug submission. Explaining the importance of Cabometyx’s additional approval, Michael M. Morrissey, Ph.D., President and Chief Executive Officer …