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APHENITY Study

APHENITY Study: From Identification to Confirmation

Two-part design: Identify responders, then confirm efficacy1,2

SEPHIENCE was evaluated in a two-part, global, double-blind, randomized, placebo-controlled study in patients with PKU. The primary endpoint was assessed by the mean change in blood Phe levels from baseline to Week 6 in the SEPHIENCE-treated group compared to the placebo group.

Description1,2

A 2-week open-label period identified participants aged ≥2 years with a ≥15% blood Phe reduction from baseline for Part 2.

SEPHIENCE

2 weeks

Washout Period

2 weeks

Baseline Characteristics2

Age <18 Years:

64%

Classical PKU:

23%

BH4 non-responsive:

36%

Response with SEPHIENCE1,2

73 percent

of patients had a ≥15% reduction in blood Phe

66 percent

of patients had a ≥30% reduction in blood Phe

In Part 2, 110 patients from Part 1 were randomized, with 109 meeting the threshold of a ≥15% reduction in blood Phe. However, 11 of these patients—who had a ≥15% but <30% reduction—were excluded from the primary analysis set (N=98).2

Description2

A 6-week study with dose escalation (‍20, 40, 60 mg‍/‍kg‍/‍day) assessed SEPHIENCE’s efficacy and safety against placebo.