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High dose ANAVEX2-73

Phase 3

Rett Syndrome | Small molecule | Rare Disease |Anavex Life Sciences Corp.|Last Updated: Aug 12, 2024

Target and mechanism

ModalitySmall molecule

Also known as ANAVEX2-73, ANAVEX2-73 Oral

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment64

FDA Designations

FAST_TRACKORPHAN_DRUGRARE_PEDIATRIC_DISEASE

Clinical trial landscape

High dose ANAVEX2-73 · 8 trials · 5 indications

Phase 3 1Phase 2 7
NCT03941444ANAVEX2-73 Study in Patients With Rett SyndromeRett Syndrome
COMPLETED33 Analytics
PHASE3COMPLETED
ANAVEX2-73 Study in Patients With Rett Syndrome
Rett SyndromeUnlock trial analytics

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
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
96 weeks

To continue assessing the safety and tolerability of ANAVEX2-73

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

Cognitive Drug Research (CDR) Computerized Assessment System Continuity of Attention
14 weeks

Change from Baseline to End of Treatment in Continuity of Attention as measured by Cognitive Drug Research (CDR) Computerized Assessment System Continuity of Attention test

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
14 weeks

Assess the safety and tolerability of ANAVEX2-73 compared to placebo

ADAS-Cog (Alzheimer Disease Assessment Scale-Cognition)
48 weeks

Reduction in cognitive decline assessed from baseline over 48 weeks with ANAVEX2-73 compared to placebo using the Alzheimer Disease Assessment Scale-Cognition (ADAS-Cog)

ADCS-ADL (Activities of Daily Living)
48 weeks

Reduction in decline of the ability to perform daily activities assessed from baseline over 48 weeks with ANAVEX2-73 compared to placebo using the Activities of Daily Living Scale (ADCS-ADL)

To determine maximum tolerated dose of Anavex2-73.
36 Days

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
ActiveEXPERIMENTALANAVEX2-73
ANAVEX2-73 ActiveEXPERIMENTALOral capsules
High dose ANAVEX2-73EXPERIMENTALHigh dose ANAVEX2-73
Mid dose ANAVEX2-73EXPERIMENTALMid dose ANAVEX2-73
Placebo oral capsulePLACEBO_COMPARATORPlacebo oral capsule
ANAVEX2-73 Oral as assigned in ANAVEX2-73-002EXPERIMENTAL -
Anavex2-73 oral then the Anavex2-73 intravenous formulationEXPERIMENTALParticipants first receive Anavex2-73 hard gelatin capsule each morning with a light breakfast for 11 days. After a washout period of 11 days they then receive the Anavex2-73 intravenous formulation each morning with a light breakfast for 11 days.
Anavex2-73 intravenous then the Anavex2-73 oral formulationEXPERIMENTALParticipants first receive Anavex2-73 intravenous formulation each morning with a light breakfast for 11 days. After a washout period of 11 days then they receive the Anavex2-73 hard gelatin capsule each morning with a light breakfast for 11 days.
Anavex2-73 30 mg oral formulationEXPERIMENTALParticipants will receive the 30 mg Anavex2-73 hard gelatin capsule orally once daily for 52 weeks.
Anavex2-73 50 mg oral formulationEXPERIMENTALParticipants will receive the 50 mg Anavex2-73 hard gelatin capsule orally once daily for 52 weeks.

Interventions

NameTypeDescription
ANAVEX2-73DRUGLiquid oral solution
PlaceboDRUGLiquid oral solution
High dose ANAVEX2-73DRUGActive oral capsule
Mid dose ANAVEX2-73DRUGActive oral capsule
Placebo oral capsuleDRUGPlacebo oral capsule
ANAVEX2-73 OralDRUG -
ANAVEX2-73 IntravenousDRUG -
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Eligibility Criteria

Age Range18 Years to 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 KingdomCanadaGermanyNetherlandsSpainUnited States
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Competitive Landscape -Acute Respiratory Distress Syndrome 15 trials (matched to "Rett Syndrome")

Top 2 of 6 competitors

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Frequently asked questions about High dose ANAVEX2-73

What is high dose ANAVEX2-73?

High dose ANAVEX2-73 is an investigational small molecule developed by Anavex Life Sciences Corp. (ticker AVXL) for neurological conditions. It is being studied in Alzheimer disease, Parkinson disease dementia, and Rett syndrome. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.

What is high dose ANAVEX2-73 used for in Alzheimer disease?

High dose ANAVEX2-73 is being studied for the treatment of Alzheimer disease, including early Alzheimer disease. It is also being evaluated in Parkinson disease dementia and Rett syndrome. It remains investigational and has not been approved for any indication.

Who makes high dose ANAVEX2-73?

High dose ANAVEX2-73 is developed by Anavex Life Sciences Corp., which trades under the ticker AVXL. The company is conducting clinical development of the drug across multiple neurological indications, including Alzheimer disease, Parkinson disease dementia, and Rett syndrome.

What phase is high dose ANAVEX2-73 in?

High dose ANAVEX2-73 is in Phase 2 development. It is investigational and has not been approved by the FDA. The clinical program includes completed Phase 2 and Phase 3 trials in Alzheimer disease, Parkinson disease dementia, and Rett syndrome.

What clinical trials is high dose ANAVEX2-73 in?

High dose ANAVEX2-73 has been evaluated in completed trials including NCT04575259, an open-label extension in Parkinson disease dementia; NCT04314934, an open-label extension of a Phase 2b/3 Alzheimer disease study; NCT03941444 in Rett syndrome; and NCT03790709 in early Alzheimer disease. Total enrollment across these studies was 809 participants.

Is high dose ANAVEX2-73 the same as ANAVEX2-73?

Yes. High dose ANAVEX2-73 refers to the same investigational compound as ANAVEX2-73, also referred to as ANAVEX2-73 Oral. These names describe the same small molecule developed by Anavex Life Sciences Corp. for neurological indications.