Vertex performs a three-stage trial of cystic fibrosis triple therapy
Vertex performs a three-stage trial of cystic fibrosis triple therapy February 02, 2018 Source: Sina Pharmaceutical On February 1st, Vertex, a Boston-based pharmaceutical company, announced that it plans to use VX-659 and VX-445 as components of two different triple therapies, respectively, to advance to the treatment of patients with cystic fibrosis (CF). In the period of study. The decision is based on relevant clinical Phase 2 study data, with the latest data from the ongoing Phase 2 trial showing that with 4-week treatment, VX-659 (400mg QD) or VX-445 (200mg) compared to baseline levels The triple therapy of QD) component in patients with a F508del mutation and a minimal functional mutation (F508del/Min) can be predicted to exceed the percentage of forced expiratory volume (ppFEV1) in more than 13.3% and 13.8%, respectively. The average is definitely improved. CF is a rare, life-threatening hereditary disease affecting approximately 75,000 people in North America, Europe and Australia. CF is caused by a mutation or deletion of the CFTR protein caused by mutation of the CFTR gene. Children must inherit two defective CFTR genes to develop CF, usually a parent's own gene. There are approximately 2,000 known possible mutant genes in the CFTR. Some of these mutations can be determined by genetic testing or genotyping, which usually results in the production of inactive or too little CFTR protein on the cell surface, which in turn leads to CF. Defects or deficiencies in the function of CFTR proteins result in poor salt and water in and out of many organ cells. In the lungs, abnormally large mucus builds up, causing chronic lung infections and progressive lung damage in the patient, which ultimately leads to death. The average time from illness to death is 20 years. The company is currently in discussions with regulatory agencies to finalize the design of the VX-659 and VX-445 clinical Phase III trials. Later, Vertex plans to launch a clinical phase 3 project in the first half of 2018 to evaluate the efficacy of VX-659 in combination with tezacaftor and ivaraftor. In addition, the company plans to launch another phase 3 trial in mid-2018 to evaluate the efficacy of VX-445 and tezacaftor and VX-561 triple therapy once a day. During the first half of 2018, the company will await other research data, including Phase 2 trial data for VX-445 in conjunction with tezacaftor and VX-561. Clinical Phase 2 and Phase 3 trial development strategies will be discussed in the 2017 Financial Results Investor Communications Meeting. Both of these different triple therapies showed good tolerance, with most adverse reactions being mild to moderate in severity. Throughout the study, the rate of discontinuation due to adverse events was low. Dr. Jeffrey Chodakewitz, Senior Vice President and Chief Medical Officer of Vertex, said: "These Phase 2 results support the option to advance the VX-659 or VX-445 triple programme to Phase 3 clinical trials and demonstrate that these regimens can be 90% of CF patients. The group offers significant clinical benefits. We look forward to communicating with regulators and starting the three phases of research in the first half of this year. Our goal is to bring triple therapy to these patients as soon as possible." Dr. Jennifer Taylor-Cousar, Associate Professor of the National Jewish Health Medicine and Pediatrics of Colorado and Chairman of the Vertex Triple Therapy Planning Committee, said: "The trial data from the Triple Therapy demonstrates the rapid progress we have made in the treatment of the nature of CF. All Phase 2 so far. The trial data provide further evidence that the addition of a new generation of therapeutic components to tezacaftor and ivaraftor has the potential to provide greater clinical benefit to patients with two related mutations, which currently do not have a drug to treat. The underlying cause. These data also provide an additional treatment option for patients with at least one f508del mutation suitable for CFTR modulator therapy." (Sina Medical Compilation/David, ed. / Kerr) Article References: Vertex Selects Two Next-Generation Correctors, VX-659 and VX-445, to Advance into Phase 3 Development as Part of Two Different Triple Combination Regimens for People with Cystic Fibrosis Used in organic synthesis to alter the activity and selectivity of Catalysts in the polymerization of organometallics. Biological Buffer 99%,Crystalline Trishydroxymethyl Aminomethane,Dl-Dithiothreitol 99%,3-Hydroxy Phenyl Phosphoryl Propanoic Acid Shandong YingLang Chemical Co.,Ltd , https://www.sdylhgtrade.com