Vertex Posts Positive Phase II Data on Kidney Disease Drug
Vertex Pharmaceuticals VRTX announced positive data from the phase IIb AMPLIFIED study, which evaluated its investigational drug, inaxaplin, for the treatment of patients with APOL1-mediated kidney disease ("AMKD") and modest proteinuria and in people with AMKD and type II diabetes.
AMKD is a rapidly progressing kidney disease caused by inherited variants in the APOL1 gene. It damages kidney cells, leading to proteinuria and declining kidney function. In severe cases, the disease can progress to dialysis or a kidney transplant.
Year to date, VRTX's shares have rallied 13.6% compared with the industry’s 8% growth.
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Key Highlights of VRTX’s Phase IIb AMPLIFIED Study Data
The phase IIb AMPLIFIED study enrolled patients across two cohorts. The first cohort included patients with AMKD and modest proteinuria, while the second cohort included patients with AMKD, type II diabetes and proteinuria. Patients received inaxaplin 45 mg once daily for 13 weeks in addition to optimized standard care. The primary endpoint was the percentage change in urine albumin-to-creatinine ratio ("UACR") from baseline. The study also evaluated changes in urine protein-to-creatinine ratio ("UPCR") and the safety of inaxaplin.
In the first cohort, inaxaplin reduced the UACR by 42.7% from baseline at week 13. UPCR, another measure of proteinuria, decreased 44.7% over the same period of time. The results were consistent with earlier phase IIa findings in patients with AMKD and focal segmental glomerulosclerosis.
In the second cohort, patients receiving inaxaplin experienced a 17.3% reduction in UACR and 25.4% decline in UPCR from baseline at week 13.
Inaxaplin was generally well-tolerated in both cohorts, with no serious adverse events reported.
Vertex Completes Inaxaplin’s AMPLITUDE Study Enrollment
Alongside the AMPLIFIED results, Vertex announced that it has completed patient enrollment in the global phase II/III pivotal AMPLITUDE study. The study is evaluating inaxaplin as a 45-mg once-daily oral treatment in patients with AMKD and severe proteinuria. Its primary endpoint is the estimated glomerular filtration rate slope after two years of treatment, a measure designed to assess the impact of treatment on kidney function over time.
Vertex expects to report interim data from the study in early 2027. Positive results could potentially support accelerated approval for the drug in the United States, subject to regulatory review.
VRTX’s Zacks Rank & Stocks to Consider
Vertex currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Precigen PGEN, currently sporting a Zacks Rank #1 (Strong Buy), AC Immune ACIU and Aldeyra Therapeutics ALDX, carrying a Zacks Rank #2 (Buy) each. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Precigen’s 2026 bottom line have improved from a loss of 2 cents to earnings per share of 25 cents. Over the same period, earnings estimates for 2027 have risen from 25 cents to 86 cents. PGEN's shares have gained 88.8% year to date.
Precigen’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 108.96%.
Over the past 60 days, estimates for AC Immune’s 2026 loss per share have narrowed from 84 cents to 60 cents. Over the same period, earnings estimates for 2027 remained unchanged at 17 cents per share. ACIU's shares have lost 13.1% year to date.
AC Immune’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 33.25%.
Over the past 60 days, loss per share estimates for Aldeyra Therapeuticshave narrowed from 43 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 22 cents to 16 cents. ALDX's shares have plunged 73.1% year to date.
Aldeyra Therapeutics’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 29.25%.
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This article originally published on Zacks Investment Research (zacks.com).
