Press release
Vertex Announces Publication in New England Journal of Medicine of Results from Phase 2 Study of Inaxaplin (VX-147)
BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in the New England Journal of Medicine (NEJM) of
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":" BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in the New England Journal of Medicine (NEJM) of results from preclinical studies and a Phase 2 study evaluating the efficacy and safety of inaxaplin (VX-147) on top of standard-of-care in people with focal segmental glomerulosclerosis (FSGS) and two APOL1 variants, a severe, rapidly progressive form of chronic kidney disease also known as APOL1-mediated kidney disease (AMKD). AMKD is caused by variants of the APOL1 gene and affects approximately 100,000 people in the U.S. and Europe. Inaxaplin is an APOL1 inhibitor aimed at treating the underlying cause of AMKD.\n\nThe manuscript presents results from the Phase 2 study of inaxaplin, demonstrating a statistically significant and clinically meaningful mean reduction in proteinuria of 47.6% (95% CI: 60.0%, 31.3%) at 13 weeks compared to baseline, which was the primary endpoint of the study. Reduction in proteinuria was observed early and continued throughout the 13-week treatment period. Results were consistent regardless of baseline proteinuria or background standard-of-care therapy. Inaxaplin was generally well tolerated in the study. The most common adverse events (occurring in >15% of subjects) were headache, back pain and nausea. These results provide the first clinical evidence that an oral small molecule APOL1 inhibitor can decrease proteinuria in patients with AMKD.\n\n“Inaxaplin has the potential to be a breakthrough for people living with AMKD by addressing the underlying cause of this devastating disease,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We look forward to exploring the full potential of this molecule in the ongoing Phase 2/3 pivotal trial and bringing this potential therapy to patients who are waiting.”\n\n“The results from the Phase 2 inaxaplin study show real promise for patients suffering from AMKD,” said Glenn Chertow, M.D., M.P.H., Professor of Medicine, Stanford University School of Medicine and Chair of Vertex’s APOL1 Program Steering Committee. “APOL1-mediated kidney disease can progress swiftly to kidney failure, and the potential for inaxaplin to precisely target the underlying cause of AMKD should bring hope and excitement to persons living with AM...