BioMarin Pharmaceutical Inc. · NASDAQ:BMRN
BioMarin Pharmaceutical — Technical Presentation (2021)
11 slides from BioMarin Pharmaceutical's technical presentation, 2021
Slide 1: Study 165-305: Interim Safety and Efficacy of Pegvaliase in Japanese Adults with Phenylketonuria
STUDY 165-305: INTERIM SAFETY AND EFFICACY OF PEGVALIASE IN JAPANESE ADULTS WITH PHENYLKETONURIA Kuwahara M1, Ishige M2, Ito T3, Hamazaki T4, Rowell R1, Taguchi T1, Shintaku H4 1BioMarin Pharmaceutical Japan K.K., Tokyo, Japan 2Department of Pediatrics, School of Medicine, Nihon University, Tokyo, J
Slide 2: COI Disclosure
日本先天代謝異常学会 ＣＯ Ｉ 開示 筆頭発表者名： 桑原 光弘 Employees: Kuwahara M, Rowell R, Taguchi T Clinical trial investigators: Ishige M, Ito T, Hamazaki T, Shintaku H （BioMarin）
Slide 3: Background
3 Background • Phenylketonuria (PKU) is caused by deficiency of the phenylalanine hydroxylase enzyme, resulting in abnormally high concentrations of phenylalanine (Phe) in the blood and brain. High Phe levels are associated with significant negative effects on neurocognitive function in adults with
Slide 4: Methods
Methods • Figure 1 provides an overview of the study design • Participants were instructed to maintain a stable diet throughout Part 1 of the study, assessed using data from 3-day diet diaries; however, modifications to a participant’s diet could be made if blood Phe levels were confirmed to be <30
Slide 5: Results
Results Table 1. Baseline demographics and characteristics Safety Efficacy Evaluable Characteristic Population Population (N=12) (N=11) Age, years Mean (SD) 29.4 (8.1) 29.4 (8.5) Female, n (%) a 4 (33.3) 4 (36.4) • A total of 12 patients (safety population) Weight, kg were enrolled into the study an
Slide 6: Exposure
Exposure • All 12 participants completed ≥4 weeks induction followed by ≥5 weeks of titration with 10 mg/day; the mean (min, max) time to first 20 mg dose was 14.0 (9.3, 29.1) weeks • Pegvaliase exposure by dose is shown in Table 2 Table 2. Pegvaliase exposure by dose category (N=12) <10 mg/day ≥10-
Slide 7: Blood Phe
Blood Phe Figure 2. Mean change in blood Phe levels over time from naïve baseline • At week 52 the mean (SD) blood Phe and percentage change from baseline was 448.3 (458.8) µmol/L and -56.4 (42.3)%, respectively • By 52 weeks the following proportion of participants had achieved the following thresh
Slide 8: Dietary Intake
Dietary Intake Figure 3. Dietary intake up to Week 52 • Three participants experienced blood Phe concentrations <30 µmol/L and were permitted per protocol dietary modifications. By Week 52 they had a reduction in medical food and increase in intact protein 8
Slide 9: Safety Overview
Safety Overview • All 12 participants (100%) experienced 1 or more treatment emergent adverse events (TEAEs), None of the TEAEs had Common Terminology Criteria for Adverse Events (CTCAE) severity Grade ≥3 • The most commonly injection site erythema and injection site swelling (each 83.3%), arthralgi
Slide 10: Safety Events by Treatment Phase
Safety Events by Treatment Phase • The occurrence of AEs was higher Table 3. Adverse events by treatment phase during the Induction/Titration (I/T) phase Induction/Titration Maintenance Overall AE Category as compared to the Maintenance phase (N=12) (N=5) (N=12) (Table 3) Subjects with any AE, n (%)
Slide 11: Conclusions
Conclusions • Analysis of the interim results up to Week 52 support the administration of pegvaliase in Japanese adults with PKU according to an I/T/M dosing regimen • These results demonstrate the efficacy of pegvaliase in reducing blood Phe concentrations with a manageable safety profile, and are
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