Vertex Presents Phase 3 Vanza Triple Data and TRIKAFTA® Long-Term Impact at CF Conference

Vertex Pharmaceuticals Incorporated has announced significant advancements in the treatment of cystic fibrosis (CF). The company will present Phase 3 data on its investigational once-daily triple combination therapy, vanzacaftor/tezacaftor/deutivacaftor, at the North American Cystic Fibrosis Conference (NACFC). This "vanza triple" therapy represents a potential next-in-class treatment aimed at further improving outcomes for CF patients.

Carmen Bozic, M.D., Executive Vice President of Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex, highlighted the transformative impact of TRIKAFTA® since its introduction five years ago. She expressed excitement over the new vanza triple combination, which could achieve even better results than current treatments.

The vanza triple will be presented in an oral session and two posters on September 27. The clinical data from the Phase 3 trials for patients aged six years and older has formed the basis for global regulatory submissions. Claire L. Keating, M.D., Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University, emphasized the importance of the additional reductions in sweat chloride levels seen in these trials, indicating potential significant benefits for CF patients.

The sessions will include:
1. An oral presentation titled "Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Adolescents and Adults with CF: Results from Two Randomized, Active-Controlled Phase 3 Trials" on September 27 from 10:15 a.m. to 12:15 p.m.
2. A poster presentation on the same topic from 1:15 p.m. to 2:15 p.m. on the same day.
3. Another poster presentation titled "Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Children 6 Through 11 Years of Age with Cystic Fibrosis" also on September 27 from 1:15 p.m. to 2:15 p.m.

The vanza triple has been granted Fast Track and Orphan Drug Designations by the U.S. Food and Drug Administration and is under regulatory review in multiple countries, including the U.S., Canada, and the EU. The Prescription Drug User Fee Act (PDUFA) date set by the FDA is January 2, 2025.

Vertex will also present long-term data on TRIKAFTA® from studies involving children aged 2 to 11 years. These data reinforce the sustained improvements in lung function and suggest that early treatment could improve pancreatic function over time. Professor Marcus A. Mall, M.D., from Charité - Universitätsmedizin Berlin, underscored the positive long-term impacts observed in patients, particularly young children.

In addition to the vanza triple data, Vertex will present four posters, three on TRIKAFTA® and one on KALYDECO®. The presentations include:
1. "Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children 2 years and older with cystic fibrosis and at least one F508del allele: 96-week results from an open-label extension study" on September 27 from 12:15 p.m. to 1:15 p.m.
2. "Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children 6 years and older with cystic fibrosis and at least one F508del allele: Results from a 192-week extension study" on the same day from 1:15 p.m. to 2:15 p.m.
3. "Effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and non-F508del CFTR variants: Interim results from a registry-based study" from 12:15 p.m. to 1:15 p.m.
4. "Long-term benefits of early ivacaftor initiation in people with cystic fibrosis (CF)" from 1:15 p.m. to 2:15 p.m.

Cystic fibrosis is a severe genetic disease affecting multiple organs and primarily caused by mutations in the CFTR gene, leading to defective or missing CFTR protein. Worldwide, over 68,000 people across more than 60 countries are receiving Vertex's CF treatments.

Vertex's investigational vanza triple therapy aims to enhance CF treatment by increasing the quantity and functionality of the CFTR protein at the cell surface. The company's existing CF therapy, TRIKAFTA®, combines elexacaftor, tezacaftor, and ivacaftor to improve the function and quantity of CFTR protein, facilitating better mucus clearance from the airways.

Vertex continues to lead in the development of CF treatments, aiming to significantly improve the quality of life and prognosis for patients with this debilitating condition.