EX-99.1 2 d90613dex991.htm EX-99.1 EX-99.1

Exhibit 99.1

 

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ENPP1 clinical development strategy and illustrative goals for trials in adult and children patient populations Adults FIH Cohort 3 LATE STAGE STUDIES Dose: 1.8 mg/kg; n:3 Pending regulatory discussions and approval Cohort 2 Phase 1/2 DSMB Dose Phase 1/2 Extension – 48 weeks | Remain on Drug Dose: 0.6 mg/kg; n:3 Extension Selection Cohort 1 DSMB Phase 1/2 Extension Dose: 0.2 mg/kg; n:3 Placebo controlled Expansion Cohort • Confirmed clinical and genetic diagnosis Adults, Phase 2/3 – 2:1 Tx:Pbo Eligibility Criteria • Age 18 65 years • Safety and tolerability of INZ 701 Primary Objective(s) • Establish dosing regimen for future clinical development ENDPOINT MEASUREMENT • Plasma PPi Additional Objectives • Other disease relevant biomarkers PPi Blood biochemistry Planned Doses • 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg; twice weekly subcutaneous Calcification High resolution radiography Phase 1 Duration • 7 weeks duration per subject; staggered recruitment per cohort (DSMB) Pain Pain Scores Children Infants – 0 2 years Pediatric – 3 18 years Infants, Phase 2/3 Open Label – 48 weeks Pending regulatory discussions and approval Pending regulatory discussions and approval ENDPOINT MEASUREMENT ENDPOINT MEASUREMENT PPi Blood biochemistry PPi Blood Calcification High resolution radiography Skeletal Abnormalities RGI C, RSS Survival Alive at 6 months Growth Rate Pediatric, Phase 2/3 Open Label – 48 weeks 23 DSMB = Data Safety Monitoring Board


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ABCC6 clinical development strategy and illustrative goals for trials in adult and children patient populations Adults FIH Cohort 3 LATE STAGE STUDIES Dose: 1.8 mg/kg; n:3 Pending regulatory discussions and approval Cohort 2 Phase 1/2 DSMB Phase 1/2 Extension – 48 weeks | Remain on Drug Dose: 0.6 mg/kg; n:3 Extension selection Cohort 1 DSMB Phase 1/2 Extension Dose: 0.2 mg/kg; n:3 Dose Placebo controlled Expansion Cohort • Confirmed clinical and genetic diagnosis Adults, Phase 2/3 – 1:1 Tx:Pbo – 48 weeks Eligibility Criteria • Age 18 65 years • Safety and tolerability of INZ 701 Primary Objective(s) • Establish dosing regimen for future clinical development • ENDPOINT MEASUREMENT Plasma PPi Additional Objectives • Other disease relevant biomarkers PPi Blood biochemistry Planned Doses • 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg; twice weekly subcutaneous Vascular calcification progression High resolution radiography Phase 1 Duration • 7 weeks duration per subject; staggered recruitment per cohort (DSMB) Maintain or slow vision loss Children Infants – 0 2 years Pending regulatory discussions and approval ENDPOINT MEASUREMENT PPi Blood biochemistry Calcification High resolution radiography Infants, Phase 2/3 Open Label – 48 weeks Survival Alive at 6 months 25 DSMB = Data Safety Monitoring Board