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SAR444836

Phase 1

Phenylketonuria | Small molecule | Rare Disease |Sanofi|Last Updated: Sep 23, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05972629A 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 PhenylketonuriaPHASE1 ACTIVE NOT_RECRUITING 32 — — Aug 7, 2023Apr 2, 2030Sep 23, 202511 United States, Argentina +3
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Study Endpoints
Primary Endpoints
Incidence of treatment-emergent adverse events (TEAEs)
From Baseline to Week 260
Secondary Endpoints
Proportion of participants with sustained plasma level of Phe<360 μmol/L for ≥4 weeks without dietary Phe restriction at Week 24, 48, and Week 96 or Early Termination Visit following SAR444836 administration
From Baseline to Week 96
Change from baseline in plasma level of Phe at Week 24, 48, and Week 96 or Early Termination Visit following SAR444836 administration
From Baseline to Week 96
Change from baseline in dietary protein intake at Week 24, 48, and Week 96 or Early Termination Visit following SAR444836 administration
From Baseline to Week 96
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
SAR444836EXPERIMENTALParticipants will receive a single dose of SAR444836 on Day 1
Interventions
NameTypeDescription
SAR444836DRUGInfusion pump, intravenous infusion (IV)
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: * Adult males, and females of non-childbearing potential, 18-65 years of age at the time of informed consent. * Participants must have uncontrolled classical PKU due to PAH deficiency (despite Phe-restricted dietary management or Palynziq) in the judgement of the Investigator. *...

Countries:United StatesArgentinaBrazilIsraelTurkey (Türkiye)
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Competitive Landscape -Phenylketonuria 12 trials
CompanyTickerTrialsLead PhaseDrugs
BioMarin Pharmaceutical Inc.BMRN5PHASE3Pegvaliase, BMN 307
PTC Therapeutics, Inc.PTCT2PHASE3PTC923, Sepiapterin
Sanofi SA Sponsored ADRSNY1PHASE1SAR444836
Agios Pharmaceuticals, Inc.AGIO1PHASE1AG-181
Illumina, Inc.ILMN1 — Undisclosed
Q32 Bio IncQTTB1 — HMI-102
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05972629primaryCompletionDate: changed
LOWMay 24, 2026NCT05972629studyFirstPostDate: changed