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DX-2930

Phase 3

Hereditary Angioedema (HAE) | Small molecule | Other |Takeda Pharmaceutical Company Limited|Last Updated: Jun 8, 2021

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials4
Total Enrollment407
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02741596Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAEPHASE3 COMPLETED 212May 26, 2016Oct 31, 2019Jun 8, 202143 United States, Canada +5
NCT02586805Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAEPHASE3 COMPLETED 125Mar 3, 2016Apr 13, 2017Jun 2, 202141 United States, Canada +5
NCT02093923A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema ParticipantsPHASE1 COMPLETED 38May 14, 2014May 18, 2015May 27, 202114 United States, Italy +1
NCT01923207A Single Increasing Dose Study to Assess Safety and Tolerability of DX-2930 in Healthy SubjectsPHASE1 COMPLETED 32Aug 12, 2013Jan 7, 2014May 17, 20211 United States
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
From start of the study up to follow-up (Day 952)

An adverse event (AE) was any untoward medical occurrence in a clinical trial Participant whether or not it appeared to have a causal relationship with the treatment administered. Treatment-emergent AEs were defined as AEs with onset at the time of or following the first exposure to open-label DX-2930 in this study, or medical conditions present prior to the start of treatment but increasing in severity or relationship at the time of or following the start of treatment.

Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period
From Day 0 to Day 182

HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks was analyzed using a generalized linear model (GLM) for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

Number of Participants With Serious Adverse Events (SAE) and Treatment-Emergent Adverse Events (TEAE)
From Day 1 up to final follow-up (Day 123)

A SAE was any adverse experience occurring at any dose that resulted in any of the following outcomes: Death, Life-threatening experience, required inpatient hospitalization or prolongation of existing hospitalization. Resulted in persistent or significant disability or incapacity. Was a congenital anomaly or birth defect. Was considered to be an important medical event. An AE was considered treatment-emergent if the onset time was after administration of study drug through the Day 120 post-dose final follow-up visit or, in the event that onset time preceded study drug administration, the AE increased in severity during the 120-day post-dose follow-up period.

Proportion of subjects with adverse events
112 days
Secondary Endpoints
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During the Treatment Period
Up to Day 924
Rate of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks Requiring Acute Treatment During the Treatment Period
Up to Day 924
Rate of Moderate or Severe Hereditary Angioedema (HAE) Attacks During the Treatment Period
Up to Day 924
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposePREVENTION
Treatment Arms
ArmTypeDescription
Rollover ParticipantsEXPERIMENTALParticipants who rollover from the DX-2930-03 study will receive 300 milligram (mg) DX-2930 subcutaneous injection at Day 0 followed by second dose following the first HAE attack and then once in every 2 weeks until the end of the treatment period (up to 924 days). A wash-out period of a minimum of 10 days and a maximum of 18 days is required between subsequent administrations.
Non-rollover ParticipantsEXPERIMENTALParticipants who were not participants in DX-2930-03 will receive 300 milligram (mg) DX-2930 subcutaneous injection once in every 2 weeks until the end of the treatment period (up to 924 days).
DX-2930 300 mg every 2 weeksEXPERIMENTAL300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
DX-2930 300 mg every 4 weeksEXPERIMENTAL300 mg DX-2930 administered every 4 weeks by subcutaneous injection
DX-2930 150 mg every 4 weeksEXPERIMENTAL150 mg DX-2930 administered every 4 weeks by subcutaneous injection
PlaceboPLACEBO_COMPARATORPlacebo administered every 2 weeks by subcutaneous injection.
DX-2930, Cohort 1EXPERIMENTALParticipants will receive 30 milligram (mg) dose of DX-2930 subcutaneous (SC) injection once and followed by the second dose after 2 week into the upper arm.
DX-2930, Cohort 2EXPERIMENTALParticipants will receive 100 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm.
DX-2930, Cohort 3EXPERIMENTALParticipants will receive 300 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm.
DX-2930, Cohort 4EXPERIMENTALParticipants will receive 400 mg dose of DX-2930 subcutaneous (SC) injection once and followed by the second dose after 2 week into the upper arm.
DX-2930EXPERIMENTALDX-2930 administered by subcutaneous route
Interventions
NameTypeDescription
DX-2930DRUGParticipants who rollover from the DX-2930-03 study will receive 300 milligram (mg) DX-2930 subcutaneous injection at Day 0 followed by second dose following the first HAE attack and then once in every 2 weeks until the end of the treatment period (up to 924 days). A wash-out period of a minimum of 10 days and a maximum of 18 days is required between subsequent administrations.
DX-2930 - 300mg/2wkDRUG300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
DX-2930 - 300mg/4wkDRUG300 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.
DX-2930 - 150mg/4wkDRUG150 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.
PlaceboDRUGPlacebo administered every 2 weeks by subcutaneous injection.
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites43

Inclusion Criteria: * Male and female HAE participants who are 12 years of age or older at the time of screening * Documented diagnosis of HAE (Type I or II) based on 1. Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying u...

Countries:United StatesCanadaGermanyItalyJordanPuerto RicoUnited Kingdom
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