Vertex Pharmaceuticals Inc - VRTX STOCK NEWS

Vertex Pharmaceuticals (NASDAQ: VRTX) has received FDA approval to extend the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor) for children aged 2 to 5 years with cystic fibrosis (CF) who possess at least one F508del mutation.

This marks a significant milestone, as approximately 900 children will now have access to a treatment targeting the underlying cause of their disease. This approval was backed by a 24-week Phase 3 study involving 75 children, showing a well-tolerated safety profile and improved lung function metrics.

TRIKAFTA, previously authorized for patients aged 6 and older, has transformed CF management and is expected to continue benefiting the CF community globally as Vertex seeks additional approvals in other regions.

Vertex Pharmaceuticals (NASDAQ: VRTX) has announced its participation in the Bank of America Securities 2023 Health Care Conference, scheduled for May 9, 2023, at 9:20 a.m. PT / 12:20 p.m. ET. The conference will feature a management presentation, and a live webcast will be accessible via the company’s website, specifically in the 'Investors' section under 'News and Events'. A replay of the presentation will also be archived on the site.

Vertex is a leading biotechnology company focused on innovative medicines for serious diseases, primarily known for its successful treatments for cystic fibrosis. The company is headquartered in Boston and has a global presence with multiple clinical programs addressing serious conditions such as sickle cell disease and type 1 diabetes. For further information, visit www.vrtx.com.

Vertex Pharmaceuticals (NASDAQ: VRTX) is set to report its Q1 2023 financial results on May 1, 2023, after market close. A conference call will follow at 4:30 p.m. ET, allowing participants to dial in for insights on the earnings announcement. Vertex is renowned for its innovative treatments for cystic fibrosis and has a diverse pipeline of investigational therapies targeting serious diseases such as sickle cell disease and type 1 diabetes. The call will also be available via webcast on the Vertex website, with an archived version accessible later. Founded in 1989, Vertex has consistently earned recognition as a top employer and continues to advance its commitment to transformative medicine.

Vertex Pharmaceuticals and CRISPR Therapeutics have completed the rolling Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) aimed at sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The applications, submitted to the FDA, include requests for Priority Review, potentially expediting the review period to eight months. The filings are supported by ongoing Phase 3 clinical studies and previous data presented at the ASH Annual Meeting in December 2022. Both companies highlight their commitment to the advancement of CRISPR technology in treating genetic diseases.

Vertex Pharmaceuticals has secured a non-exclusive licensing agreement with CRISPR Therapeutics for its CRISPR/Cas9 technology, aiming to enhance the development of hypoimmune cell therapies for Type 1 Diabetes (T1D). Vertex will pay $100 million upfront, with potential additional payments up to $230 million based on future achievements. This collaboration builds on Vertex's existing programs, including VX-880, which has shown clinical proof of concept. Both companies express optimism about accelerating the creation of insulin-producing cells, though challenges and risks remain in clinical validation.

Vertex Pharmaceuticals (Nasdaq: VRTX) announced the publication of Phase 2 study results in the New England Journal of Medicine, evaluating inaxaplin (VX-147) for APOL1-mediated kidney disease (AMKD). The study demonstrated a statistically significant 47.6% mean reduction in proteinuria at 13 weeks compared to baseline, the primary endpoint. Inaxaplin was well tolerated, with common adverse events including headache and back pain. The FDA granted Breakthrough Therapy Designation for inaxaplin, and the ongoing Phase 2/3 trial aims to assess kidney function over two years, with over 100 global sites open for enrollment.

Vertex Pharmaceuticals has received FDA clearance for its Investigational New Drug Application (IND) for VX-264, a new stem cell-derived pancreatic islet cell therapy aimed at treating type 1 diabetes (T1D). This investigational therapy eliminates the need for immunosuppression, potentially expanding its patient base. Vertex plans to initiate a Phase 1/2 clinical trial in mid-2023 to evaluate VX-264's safety and efficacy, following ongoing trials for a similar therapy, VX-880, in Canada and the U.S.

The trial will enroll approximately 17 patients and seeks to understand the therapy's impact on T1D, a condition characterized by the loss of insulin production.