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SerpinPC

Phase 1

Hemophilia a | Small molecule | Hematology |Centessa Pharmaceuticals plc|Last Updated: Feb 6, 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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment39
FDA Designations
FAST_TRACK
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04073498The Safety and Tolerability of SerpinPC in Healthy Men and in Men with Severe Blood Disorders (haemophilia a and B)PHASE1 COMPLETED 39Aug 14, 2019Sep 26, 2024Feb 6, 20253 Georgia, Moldova +1
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Study Endpoints
Primary Endpoints
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
From Day 1 up to 74 weeks

Number of Participants with One or More Drug Related Adverse Events (AEs) or any Serious AEs

Secondary Endpoints
PK of SerpinPC in plasma after single dose administration - Cmax
From Day 1 up to 26 weeks
PK of SerpinPC in plasma after single dose administration - Tmax
From Day 1 up to 26 weeks
PK of SerpinPC in plasma after single dose administration - kel
From Day 1 up to 26 weeks
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingSINGLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part 1a - Cohort 1EXPERIMENTALA single IV infusion of 0.0003 mg/kg SerpinPC in healthy subjects.
Part 1a - Cohort 2EXPERIMENTALA single IV infusion of 0.001 mg/kg SerpinPC in healthy subjects.
Part 1a - Cohort 3EXPERIMENTALA single IV infusion of 0.003 mg/kg SerpinPC in healthy subjects.
Part 1a - Cohort 4EXPERIMENTALA single IV infusion of 0.01 mg/kg SerpinPC in healthy subjects.
Part 1a - Cohort 5EXPERIMENTALA single SC dose of 0.03 mg/kg SerpinPC and a single SC dose of placebo in healthy subjects.
Part 1b - Cohort 6EXPERIMENTALA single SC dose of 0.1 mg/kg SerpinPC and a single SC dose of placebo in patients.
Part 1b - Cohort 7EXPERIMENTALA single SC dose of 0.3 mg/kg SerpinPC and a single SC dose of placebo in patients.
Part 1b - Cohort 8EXPERIMENTALA single SC dose of 0.6 mg/kg SerpinPC and a single SC dose of placebo in patients.
Part 1b - Cohort 9EXPERIMENTALTwo single SC doses of 0.6 mg/kg SerpinPC in patients.
Part 2EXPERIMENTALUp to 3 SC doses may be selected for Part 2 and each patient will be assigned a single SerpinPC dose level (and placebo). The dose for Part 2 will be determined from ongoing review of the Part 1 data. The dose in Part will not exceed 1.2 mg/kg, or the highest dose deemed safe from Part 1b.
Part 3EXPERIMENTALA single flat SC dose of SerpinPC will be administered every 4 weeks for 48 weeks for patients who have completed Week 24 of Part 2. The dose level will be chosen after reviewing Part 2 data and will not exceed maximum dose level in Part 2.
Part 4EXPERIMENTALSC doses of 1.2 mg/kg SerpinPC will be administered every 2 weeks for 24 weeks for patients who have completed Week 48 of Part 3.
Part 5EXPERIMENTALSC doses of 1.2 mg/kg SerpinPC will be administered every 2 weeks for 52 weeks for patients who have completed Week 24 of Part 4.
Part 6EXPERIMENTALA single flat SC dose of 60mg SerpinPC will be administered every 2 weeks for 52 weeks for patients who have completed Week 52 of Part 5.
Interventions
NameTypeDescription
SerpinPCDRUGAdministered as IV infusion (over 30 minutes) or SC injection (abdomen) according to cohort dosing instructions.
PlaceboDRUGMatching placebo administered by SC injection according to cohort dosing instructions.
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Eligibility Criteria
Age Range18 Years — 55 Years
SexMALE
Healthy VolunteersYes
Study Sites3

Inclusion Criteria: Part 1a (Healthy Subjects) 1. Males age ≥18 years and ≤55 years. 2. Capable of giving written informed consent to participate after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or delegate. 3. Willingness ...

Countries:GeorgiaMoldovaUnited Kingdom
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