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BPS804

Phase 2

Hypophosphatasia | Small molecule | Other |Ultragenyx Pharmaceutical Inc.|Last Updated: Sep 16, 2022

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment8
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01406977Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)PHASE2 COMPLETED 8 — — Jul 1, 2011Sep 1, 2012Sep 16, 20221 Germany
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Study Endpoints
Primary Endpoints
The number (percent) of patients experiencing adverse events or serious adverse events
141 days following initial investigational product administration
Change from baseline in primary serological bone biomarkers
141 days following initial investigational product administration
Secondary Endpoints
Characterization of the pharmacokinetic profile of BPS804: area under the plasma concentration-time curve (AUC)
1, 29 and 141 days following initial investigational product administration
Characterization of the pharmacokinetic profile of BPS804: observed maximum plasma concentration following drug administration (Cmax)
1, 15 and 29 days following initial investigational product administration
Characterization of the pharmacokinetic profile of BPS804: time to reach the maximum concentration (Tmax)
1, 15 and 29 days following initial investigational product administration
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BPS804 dose escalationEXPERIMENTALBPS804 IV Setrusumab given in escalating doses from 5mg/Kg to 20mg/Kg
Interventions
NameTypeDescription
BPS804DRUG -
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Male and female patients 18 to 60 years of age in good health (other than pre-established clinical diagnosis of HPP) as determined by past medical history, physical examination, vital signs, electrocardiogram, and laboratory tests at screening. * Previously established clinica...

Countries:Germany
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