Clinical Study Results | FABHALTA® (iptacopan)

^{In adults with primary IgAN at risk of their disease worsening}
FABHALTA HELPED SLOW KIDNEY FUNCTION DECLINE

FABHALTA was tested in a large clinical study of 477 adults with IgAN

APPLAUSE-IgAN was a phase 3 clinical study in adults with primary IgAN: FABHALTA was compared to placebo (sugar pill).

Who was studied?

Adults with biopsy-proven IgAN who:

Had elevated proteinuria, UPCR ≥1 g/g (UPCR is a test to measure amount of protein leaking into urine collected over 24 hours)
Were on a stable dose of ACEi/ARBs at a maximally tolerated dose with or without other background therapies before and during the study

How was the study done?

The study included adults taking FABHALTA (200 mg twice daily) and patients taking placebo (sugar pill twice daily) for 24 months. An interim analysis was done at 9 months and a final analysis at 24 months.

What was studied?

In the interim analysis at 9 months

Included the first 125 patients taking FABHALTA vs 125 patients taking placebo
The primary goal was to compare the change in UPCR at 9 months to the start of the study (baseline):
- 44% proteinuria reduction with FABHALTA vs 9% with placebo

In the final analysis at 24 months:

Included 238 patients taking FABHALTA and 239 patients taking placebo
The primary goal was to study the rate of kidney function decline over 24 months as measured by eGFR (estimated glomerular filtration rate)

All efficacy analyses were done in participants with eGFR ≥30 mL/min/1.73 m².

Estimated glomerular filtration rate helps show how well your kidneys are functioning

Kidney function decline over time is reflected as a negative value. eGFR can be tracked over time to calculate the average yearly change.

A negative number closer to zero indicates slower decline in kidney function
A negative number further from zero indicates faster decline in kidney function

Proteinuria is a measure used to evaluate your IgAN

One common way to measure proteinuria is a test called urine protein-to-creatinine ratio (UPCR). UPCR helps your health care team monitor how much protein leaks into your urine.

UPCR can be measured in a couple of ways by:

Collecting urine over a 24-hour period, called 24-hour UPCR
Testing a single urine sample from your first urination of the day (called the first morning void, or FMV)

FABHALTA helped preserve kidney function better than placebo

The numbers shown above tell you how many people were included in the study results at each point in time.

There was a 50% slower yearly decline in kidney function with FABHALTA vs placebo over 24 months.

Substantial proteinuria reduction with FABHALTA

Change in proteinuria levels measured by 24-hour UPCR at 9 and 24 months compared to start of the study

^*Percent reduction was calculated by comparing average proteinuria levels at the start of the study to average proteinuria levels at month 9. ^†Percent reduction was calculated by comparing average proteinuria levels at the start of the study to average proteinuria levels at month 24.

Fast and lasting proteinuria reduction with FABHALTA

Proteinuria was also studied by measuring UPCR in first urination of the day, called first morning void (FMV)
Proteinuria reduction was seen as early as week 2 and sustained through month 24.