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OK432

Phase 2

Lymphatic Malformations | Small molecule | Other |Smith & Nephew SNATS, Inc.|Last Updated: Nov 23, 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
UNCONTROLLED
Total Trials1
Total Enrollment275
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03427619OK432 (Picibanil) in the Treatment of Lymphatic MalformationsPHASE2 COMPLETED 275Oct 5, 2005Apr 30, 2018Nov 23, 202114 United States
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Study Endpoints
Primary Endpoints
Number of Participants With Clinical Success at 1 to 6 Months Post-Therapy as Assessed by Imaging
1 to 6 Months Post-Therapy

Clinical success was defined as having either a complete (90% 100%) or substantial (60% 89%) reduction in lymphatic malformation (LM) volume after treatment. Response was determined using post treatment imaging studies at approximately 1 to 6 months after completion of treatment

Secondary Endpoints
Number of Participants With Clinical Response 1 to 6 Months Post-Therapy as Assessed by Imaging
1 to 6 Months Post-Therapy
Number of Participants With Investigator-Evaluated Overall Response
1 to 6 Months Post-Therapy
Change From Baseline in Lesion Volume
Baseline and 1 to 6 Months Post-Therapy
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
OK432 (Picibanil)EXPERIMENTALThere is no control in this study. All participants will receive the actual drug -OK432. With each injection they may receive 0.01 to 0.05mg/mL 6-12 weeks apart up to 4 injections total.
Interventions
NameTypeDescription
OK432DRUGOK432 will be injected at dosage of 0.01 to 0.05 mg/mL 6-12 weeks apart up to 4 injections total.
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Eligibility Criteria
Age Range6 Months — 17 Years
SexALL
Healthy VolunteersYes
Study Sites14

Inclusion Criteria: To be eligible to receive OK432 immunotherapy * Patients must be ages 6 months to 17 years * Patients must have a macrocystic Lymphatic Malformation * Patients may have had surgical treatment for their Lymphatic Malformation * Patients must have an imaging study to confirm the ...

Countries:United States
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