SAR444836 / Sanofi, MediciNova - LARVOL DELTA

A Phase 1/2 Open-label Study to Evaluate Safety, Tolerability and Efficacy of SAR444836, an Adeno-associated Viral Vector-mediated Gene Transfer of Human Phenylalanine Hydroxylase in Adult Participants with Classical Phenylketonuria (ASGCT 2026) - P1/2 | "The mainstay of therapy in classical PKU is maintenance of a severe protein and Phe- restricted diet which is difficult to sustain, or treatment with pegvaliase which requires daily subcutaneous administration...Whether a more aggressive or prolonged prophylactic immunosuppression regimen, or a higher dose level of SAR444836 administered would have facilitated improved efficacy are unanswered questions. Figure 1: Cohort 1 (1e13 vg/kg) Phe levels vs timeFigure 2: Cohort 2 (3e13 vg/kg) Phe levels vs time"

Clinical • P1/2 data • Viral vector • Cardiovascular • Gene Therapies • Hematological Disorders • Infectious Disease • Metabolic Disorders • Phenylketonuria • Pneumonia • Pulmonary Embolism • Rare Diseases • Respiratory Diseases • Thrombocytopenia

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria (clinicaltrials.gov) - P1/2 | N=32 | Active, not recruiting | Sponsor: Sanofi | Recruiting ➔ Active, not recruiting | Trial completion date: Apr 2031 ➔ Apr 2030 | Trial primary completion date: Apr 2031 ➔ Apr 2030

Enrollment closed • Trial completion date • Trial primary completion date • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: Sanofi | Trial completion date: Jul 2027 ➔ Apr 2031 | Trial primary completion date: Jul 2027 ➔ Apr 2031

Trial completion date • Trial primary completion date • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

PHASE 1/2 OPEN-LABEL STUDY DESIGN TO EVALUATE SAFETY, TOLERABILITY, AND EFFICACY OF SAR444836, AN AAV-MEDIATED GENE TRANSFER IN PKU PATIENTS (SSIEM 2024) - P1/2 | "Background: Patients with phenylketonuria (PKU) require lifelong treatment with dietary Phenylalanine (Phe) restriction, tetrahydrobiopterin (BH4), and/or PEGylated phenylalanine ammonia lyase (Pegvaliase). Discussion/Conclusion Targeting the unmet need in the treatment of PKU, this innovative trial will investigate whether AAV gene therapy can provide a substantial treatment advantage over existing therapy. NCT number: NCT05972629 Palavras-chave : Phenylketonuria, Safety, Efficacy, Study design, AAV-mediated gene transfer"

Clinical • P1/2 data • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: Sanofi | Not yet recruiting ➔ Recruiting

Enrollment open • Viral vector • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria (clinicaltrials.gov) - P1/2 | N=32 | Not yet recruiting | Sponsor: Sanofi

New P1/2 trial • Viral vector • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases