Vertex Presents New Data at the European Cystic Fibrosis Conference Demonstrating Significant Benefits of Treatment with TRIKAFTA®
Vertex said it's making strong progress bringing its CRISPR Therapeutics-partnered gene therapy to patients after a landmark approval for sickle cell disease in December. Chief operating officer Stuart Arbuckle said Monday that more than 25 treatment sites for the therapy, called Casgevy, have been activated worldwide
Vertex Advances Inaxaplin (VX-147) into Phase 3 Portion of Adaptive Phase 2/3 Clinical Trial for the Treatment of APOL1-Mediated Kidney Disease
Lonza and Vertex Pharmaceuticals broke ground for a dedicated manufacturing facility in Portsmouth, NH, marking a milestone in the strategic collaboration between the two companies, which aims to accelerate the development and commercialization of Vertex’s cell therapies for type 1 diabetes (T1D).
BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today presented data on all patients dosed in Parts A and B of its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy, in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). All six patients treated with VX-880 had undetectable fasting C-peptide (endogenous insulin secretion) at baseline, a history of recurrent SHEs in the year prior to treatment and required an average of 34.0 units of insulin per day. Following treatment, all six patients demonstrated endogenous insulin secretion, improved glycemic control as measured by HbA1c, improved time-in-range on continuous glucose monitoring, and reduction or elimination of exogenous insulin use. Patients with greater than 90 days of follow-up had elimination of SHEs in the evaluation period.
According to an SEC filing, Aurinia’s EVP of research Robert Huizinga and CMO Neil Solomons are both walking away by Halloween, and while no explanations are given, details of their severance packages were provided. Huizinga had spent the last nine years at Aurinia after the Isotechnika merger: first as VP, clinical affairs, then as EVP, corporate development before his latest promotion in 2020. Solomons co-founded Aurinia a decade ago and used to be VP of R&D with Vifor Pharma. The Peter Greenleaf-led Aurinia just launched its “Get Uncomfortable” ad campaign, urging lupus nephritis patients to schedule checkups with their doctor.
Vertex Pharmaceuticals VRTX announced that the FDA approved the expanded use of its cystic fibrosis (CF) drug Orkambi (lumacaftor/ivacaftor) in children aged 12 months to less than 24 months.
Vertex Pharmaceuticals has received the FDA go-ahead to continue studies for its diabetes stem cell therapy. The treatment, which Vertex has attempted to describe as a “cure,” had been on hold at the agency since May, when regulators expressed concerns about a lack of information ahead of the dose escalation portions of the study. With the hold lifted, Vertex will begin enrolling patients again at the high dose level.
Teva CEO Kåre Schultz has found his new R&D chief and CMO in Vertex’s ranks. The global generics giant, which has some 3,500 staffers in the R&D group, has named Eric Hughes to the top research spot in the company. He’ll be replacing Hafrun Fridriksdottir, who held the role for close to five years, on Aug. 1.
Patients with severe forms of sickle cell disease or beta-thalassemia lack enough functioning red blood cells to shuttle ample oxygen through their systems. As a result, they may need recurring blood transfusions throughout their entire lives, on top of a wide range of other serious symptoms.