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ANAVEX2-73

Phase 3

Rett Syndrome | Small molecule | Other |Anavex Life Sciences Corp.|Last Updated: Jan 27, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment64
FDA Designations
FAST_TRACKORPHAN_DRUGRARE_PEDIATRIC_DISEASE
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03941444ANAVEX2-73 Study in Patients With Rett SyndromePHASE3 COMPLETED 33May 6, 2019Sep 30, 2021Jan 27, 20227 Australia, United Kingdom
NCT03758924Study of ANAVEX2-73 in Patients With Rett SyndromePHASE2 COMPLETED 31Feb 28, 2019Oct 30, 2020Jan 12, 20218 United States
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Study Endpoints
Primary Endpoints
RSBQ
7 weeks

Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score

Incidence of Adverse Events
7 weeks
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
7 weeks

PK of ANAVEX2-73 and metabolite

Area Under the Curve [AUC] of ANAVEX2-73
7 weeks

PK of ANAVEX2-73 and metabolite

Lipid panel
7 weeks

Significant laboratory findings

Secondary Endpoints
CGI-I
7 weeks
Anxiety, Depression, and Mood Scale (ADAMS)
7 weeks
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
7 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Active armEXPERIMENTALANAVEX2-73 liquid oral solution
Placebo armPLACEBO_COMPARATORPlacebo liquid oral solution
Interventions
NameTypeDescription
ANAVEX2-73DRUGLiquid oral solution
PlaceboDRUGLiquid oral solution
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Eligibility Criteria
Age Range18 Years — 45 Years
SexFEMALE
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: * Aged ≥ 18 years, inclusive. * Diagnosis of classic RTT, according to 2010 criteria (Neul et al., 2010), and a MECP2 mutation. * Current pharmacological treatment regimen, including supplements, has been stable for at least 4 weeks. * If on antiepileptic drugs (AEDs), 1-4 AEDs ...

Countries:AustraliaUnited KingdomUnited States
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