BioMarin Pharmaceutical Inc. · NASDAQ:BMRN

BioMarin Pharmaceutical — Technical Presentation (2023)

11 slides from BioMarin Pharmaceutical's technical presentation, 2023

  1. Slide 1: The Long-Term Safety and Efficacy of Pegvaliase 60 mg/day in Adults with Phenylketonuria

    THE LONG-TERM SAFETY AND EFFICACY OF PEGVALIASE 60 MG/DAY IN ADULTS WITH PHENYLKETONURIA Longo N1, Zori R2, Northrup H3, Sanchez-Valle A4, Sacharow S5, Jones S6, Lee L6, Bulloch-Whitehall K6, Lounsbury D6 1Department of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City,

  2. Slide 2: COI Disclosure

    日本先天代謝異常学会 ＣＯ Ｉ 開示 Presenter: Longo N Employees: Jones S, Lee L, Bulloch-Whitehall K, Lounsbury D Clinical trial investigators: Longo N, Zori R, Northrup H, Sanchez-Valle A BioMarin Pharmaceutical Inc. provided funding for the study, data analysis, writing, editing, and presentation production （BioM

  3. Slide 3: Phenylketonuria (PKU) and Uncontrolled Blood Phe

    Phenylketonuria (PKU) and Uncontrolled Blood Phe • PKU is caused by deficiency in the phenylalanine hydroxylase (PAH) enzyme, resulting in phenylalanine (Phe) accumulation in the blood and brain • High concentrations of Phe are neurotoxic with significant neurological, neurocognitive, neuropsychiatr

  4. Slide 4: Pegvaliase is an Enzyme Substitution Therapy to Reduce Blood Phe in PKU Patients Independent of PAH Activity

    Pegvaliase is an Enzyme Substitution Therapy to Reduce Blood Phe in PKU Patients Independent of PAH Activity Phenylalanine ammonia lyase (PAL) Pegvaliase • Converts Phe to trans-cinnamic • PEGylated (polyethylene glycol) acid and ammonia • Enzyme substitution therapy PEGylation • Masks antibody-bind

  5. Slide 5: US Label Recommended Dosing Schedule

    US Label Recommended Dosing Schedule Dosing to 60 mg/day should be considered in patients who have not responded with 40 mg/day after 16 weeks Table 1. Recommended Dosing Schedule Figure 3. Maximum dose US Duration commercial dispensing data1 Treatment Dosage* (weeks) Other Induction 4 2.5 mg once w

  6. Slide 6: Use of Pegvaliase Up to 60 mg/day Was Approved in US from the Results of the Phase 3 PRISM Trials

    Use of Pegvaliase Up to 60 mg/day Dose Was Approved in US from the Results of the Phase 3 PRISM Trials Figure 4. Study design for PRISM-1, PRISM-2, and 165-304 PRISM-1 PRISM-2 165-304 Open-label, parallel-group phase 3 clinical trial to Four-part phase 3 clinical trial of pegvaliase Open-label exten

  7. Slide 7: Baseline Characteristics - Similarities and Key Differences

    Baseline Characteristics - Similarities and Key Differences Baseline characteristics of 60 mg/day stable dose reflect the overall population; marginal differences observed in weight and % male Table 2. Baseline characteristics Stable 60 mg Dose Cohort Other PRISM Participants Number of subjects 51 2

  8. Slide 8: Blood Phe Reductions After Titration to 60 mg in the First 24 Weeks and Through Long-term Follow-up

    Blood Phe Reductions After Titration to 60 mg in the First 24 Weeks (Figure 5) and Through the End of Long-term Follow-up (Figure 6) Figure 6. Percentage of participants achieving Figure 5. Mean (SE) plot of blood Phe in the first a meaningful blood Phe reduction after titration 24 weeks after dose

  9. Slide 9: Impact of Dose Titration on Antibody Titers

    9 Impact of Dose Titration on Antibody Titers • Antibody titers remained stable and did not increase over long-term treatment or after dose increases Figure 7. Antibody titers in participants titrated from 20 to 40 mg/day and 40 to 60 mg/day 9 | © 2022 BioMarin Global Medical Affairs

  10. Slide 10: Rates of the Most Common Adverse Events in ≥60 mg Cohort Mirrors Lower Maintenance Doses

    Rates of the 3 Most Common Adverse Events in ≥60 mg Cohort Mirrors Lower Maintenance Doses Figure 8. Rates of 3 Most Common Adverse Events in ≥60 mg Cohort 25 • Maintenance is defined as when participants reached 20 stable dose for 8 weeks Event rate per person-year • Hypersensitivity events 15 incl

  11. Slide 11: Conclusions

    Conclusions • The unique immune response to pegvaliase requires dose individualization – Demographics of ≥60 mg dose cohort are similar to the larger phase 3 population • Participants requiring exposure to higher doses achieved guideline- recommended thresholds – The maximum labeled dose of pegvalia

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