Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) on Tuesday reported data from its Phase 2b AMPLIFIED trial evaluating inaxaplin in individuals with APOL1-mediated kidney disease (AMKD).
The study tested a daily 45 mg dose alongside optimized standard care across two distinct patient groups with either modest proteinuria or co-existing type 2 diabetes.
Strong Proteinuria Reductions In Modest Proteinuria Cohort
In the first group of 23 participants with modest proteinuria, 22 evaluable patients achieved a 42.7% mean decrease in urine albumin-to-creatinine ratio (UACR) from baseline after 13 weeks.
This cohort also showed a 44.7% reduction in urine protein-to-creatinine ratio (UPCR). Most participants in this group had no prior diagnosis of focal segmental glomerulosclerosis (FSGS).
These findings align closely with prior Phase 2a trial outcomes in AMKD patients with FSGS, where inaxaplin generated a 43.4% UACR reduction and a 47.6% UPCR decline at Week 13.
Key Results For Type 2 Diabetes Group
The second cohort enrolled 18 patients with AMKD, type 2 diabetes, and proteinuria. Among 17 evaluable participants in this group, treatment led to a 17.3% UACR drop at Week 13 compared to baseline.
Additionally, this cohort demonstrated a 25.4% decline in UPCR over the same treatment period.
Favorable Safety Profile And Future Milestones
Inaxaplin displayed a favorable safety and tolerability profile across both patient groups. Researchers observed no serious adverse events linked to the treatment, and all reported adverse events remained mild or moderate.
Headache served as the most frequent side effect, affecting 7.3% of participants. Five patients experienced temporary, symptom-free elevated transaminase levels that fully resolved.
Vertex has completed participant enrollment for its Phase 2/3 AMPLITUDE study, which evaluates inaxaplin in individuals with severe proteinuria lacking other kidney disease causes.
The company expects to share interim analysis results in early 2027 after participants complete 48 weeks of treatment. Positive trial data from this interim evaluation could support a potential accelerated approval application in the U.S.
Analyst View
William Blair sees inaxaplin as underappreciated, and looks to AMPLITUDE data in early 2027 as the next key catalyst for the program.
“Beyond AMKD, with a potentially transformative growth profile in new product launches anchored by Vertex’s CF juggernaut, we continue to view the company as a core mid- to large-cap growth story in the biotech space…,” wrote analyst Myles Minter.
William Blair reiterates the Outperform rating on Vertex.
VRTX Price Action: Vertex Pharmaceuticals shares were up 2.04% at $521.14 at the time of publication on Tuesday, according to Benzinga Pro data.
Photo by Piotr Swat via Shutterstock
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