Vertex Pharmaceuticals Inc - VRTX STOCK NEWS

Vertex Pharmaceuticals announced a definitive agreement to acquire ViaCyte for $320 million in cash. This acquisition aims to enhance Vertex's efforts in developing VX-880, a stem cell-derived therapy for type 1 diabetes (T1D), which has shown promising results in clinical trials. The integration of ViaCyte's technologies, stem cell lines, and manufacturing capabilities is expected to accelerate Vertex’s T1D programs. The deal is anticipated to close later this year, pending standard regulatory conditions.

Vertex Pharmaceuticals has announced that the FDA has lifted the clinical hold on its Phase 1/2 trial for VX-880, a stem cell therapy aimed at treating type 1 diabetes (T1D) in patients with impaired hypoglycemic awareness. The trial will resume screening and enrollment at multiple U.S. sites. Currently, three patients have been dosed, with further evaluation planned for five more in the next trial phase. VX-880 aims to restore insulin-producing islet cell function, addressing a crucial need in T1D treatment where existing options are limited.

Vertex Pharmaceuticals and CRISPR Therapeutics unveiled promising new data on exa-cel for treating transfusion-dependent beta thalassemia and severe sickle cell disease. The study involved 75 patients, with up to 37.2 months of follow-up. Key findings include:

• 42 out of 44 patients with beta thalassemia were transfusion-free post-treatment.
• All 31 patients with sickle cell disease experienced cessation of vaso-occlusive crises.

The safety profile aligns with myeloablative conditioning and stem cell transplant protocols.

Vertex Pharmaceuticals presented five abstracts on cystic fibrosis (CF) treatments at the ECFS 45th European Cystic Fibrosis Conference from June 8-11, 2022, in Rotterdam. Key findings from a study involving over 16,000 CF patients treated with TRIKAFTA showed significant improvements in lung function and a 77% reduction in pulmonary exacerbations. Additionally, early initiation of KALYDECO demonstrated better lung function preservation in children aged 6-10. These results underline the importance of early and continuous CFTR modulator therapy.

Vertex Pharmaceuticals (NASDAQ: VRTX) will participate in a fireside chat at the Goldman Sachs 43rd Annual Global Healthcare Conference on June 15, 2022, at 1:20 p.m. PT. A live webcast will be available on Vertex's website in the 'Investors' section. Vertex is a biotechnology company focused on creating transformative medicines, especially for cystic fibrosis (CF), and has an extensive pipeline targeting diseases like sickle cell disease and type 1 diabetes. The company has received recognition for its workplace culture and innovation.

Vertex Pharmaceuticals has received Breakthrough Therapy Designation from the FDA for inaxaplin (VX-147) targeting APOL1-mediated focal segmental glomerulosclerosis (FSGS). Additionally, the European Medicines Agency awarded PRIME designation for inaxaplin to treat APOL1-mediated chronic kidney disease (AMKD). Inaxaplin is the first therapy aimed at the underlying cause of AMKD. Vertex now holds nine Breakthrough Therapy Designations in the U.S., including three non-oncological PRIME designations.

Vertex Pharmaceuticals announced significant findings from its ongoing Phase 1/2 trial of VX-880, a stem cell-derived therapy for Type 1 Diabetes (T1D). Data revealed that Patient 1 achieved a remarkable increase in blood glucose time-in-range from 40.1% to 99.9% after 270 days and became insulin-independent. Patient 2 experienced a rise from 35.9% to 51.9% and reduced insulin use by 30% in 150 days. VX-880 showed good tolerability with mainly mild adverse events, suggesting promising potential for glycemic control and less reliance on exogenous insulin.

Vertex Pharmaceuticals and CRISPR Therapeutics announced additional abstracts for the 2022 European Hematology Association (EHA) Congress. The highlight is the late-breaking abstract on the efficacy and safety of exa-cel for beta-thalassemia and sickle cell disease, scheduled for oral presentation on June 12. Three real-world economics abstracts will also be presented on June 10, detailing the economic burden of these diseases in the U.S. Exa-cel is being investigated as a one-time therapy for these patients.

Vertex Pharmaceuticals (NASDAQ: VRTX) announced that three abstracts related to its Phase 1/2 trial of VX-880, a stem cell-derived therapy for type 1 diabetes (T1D), have been accepted for presentation at the American Diabetes Association's 82nd Annual Scientific Sessions from June 3-7, 2022, in New Orleans. One significant study shows VX-880 can enhance insulin production and glucose control in T1D patients. Another abstract highlights ongoing challenges in achieving glycemic control despite advanced diabetes technologies, underscoring the need for innovative T1D treatments.