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REC-994

Phase 2

Cerebral Cavernous Malformation | Small molecule | Other |Recursion Pharmaceuticals, Inc.|Last Updated: Aug 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment62
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05085561The Symptomatic Cerebral Cavernous Malformation Trial of REC-994PHASE2 COMPLETED 62Mar 17, 2022Jun 30, 2025Aug 6, 202515 United States
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Study Endpoints
Primary Endpoints
Incidence and severity of adverse events (AEs)
Up to 24 months
Secondary Endpoints
Change in patient reported outcomes (Cerebral Cavernous Malformation Health Index)
Up to 24 months
Change in patient reported outcomes (Modified Rankin Scale)
Up to 24 months
Change in patient reported outcomes (SymptoMScreen Score)
Up to 24 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
REC-994 200 mgACTIVE_COMPARATORREC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet)
REC-994 400 mgACTIVE_COMPARATORREC-994 400 mg po QD (2 200 mg REC-994 tablets)
PlaceboPLACEBO_COMPARATORMatching Placebo po QD (2 matching placebo tablets)
Interventions
NameTypeDescription
REC-994DRUGREC-994 200 mg tablets
PlaceboDRUGPlacebo Tablets
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites15

Inclusion criteria: 1. 18 years of age or older with anatomic CCM lesions demonstrated by brain MRI 2. Have symptomatic CCM 3. Have provided written informed consent to participate in the study 4. Have not participated in a clinical trial utilizing an investigational agent within 28 days or within ...

Countries:United States
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