Good summary from yesterday’s presentation.
By Traderpete at the swamp
My HC Wainwright Presentation Notes:
Starting a pivotal study in Fragile X where we have very good pre-clinical date which is being submitted for publication.
Received Fast Track plus Orphan designation for Rett Syndrome as well as the pediatric voucher for priority review.
We are able to show compelling human data, available in three different indications; Rett Syndrome, Parkinson’s Dementia as well as Alzheimer’s disease.
We also have enough cash for 36 months which is sufficient to reach these milestones.
Catalysts;
Complete data on Rett U.S. Study
Complete data on Parkinson’s Dementia Study
Top Line data for Avatar Study (Adult Rett Syndrome)
Top Line Data for Phase One A3-71
Top Line Data for Excellence Study, (pediatric Rett Syndrome)
Top Line Data for A2-73 Alzheimer’s Disease
Additional Rare disease not yet disclosed
Focusing on activation of the body’s own defense mechanism to provide support to avoid or reduce the stress which can be part of these diseases.
Sigma One activation is beneficial for downstream activities such as;
Inflammation Reduction
Restoration of Autophagy
Reduction of Abeta and Tau
Rett Syndrome Trial:
Safety profile the same for placebo and active arm
No Adverse events.
All patients continued Study with no drop outs.
All patients continued in the extension study which was recently extended from twelve weeks to an additional six months to now 36 weeks by the FDA
Alzheimer’s disease Phase 2a; A2-73
Those with sufficient, 30 to 50 mg doses benefitted more than lesser
doses of 10 to 30 mg over 148 weeks.
Parkinson’s Dementia Phase2;
The cognitive features, Episodic Memory, Attention, Language Visual Spacial Skills,
Executive Function all improved with A2-73 in the Double Blinded, Placebo Controlled Study.
The Episodic Memory Endpoint correlates highly with ADAS Cog which is the Primary Endpoint of our Alzheimer’s Disease Study.
There was a dose response in this Placebo Controlled study where the Placebo Arm declined while the Medium 30mg dose stopped decline and the 50mg dose Improved Cognition.
This high correlation data point provides a comfort level for being replicated in the Phase2b/3 Alzheimer’s Study.
By Traderpete at the swamp
My HC Wainwright Presentation Notes:
Starting a pivotal study in Fragile X where we have very good pre-clinical date which is being submitted for publication.
Received Fast Track plus Orphan designation for Rett Syndrome as well as the pediatric voucher for priority review.
We are able to show compelling human data, available in three different indications; Rett Syndrome, Parkinson’s Dementia as well as Alzheimer’s disease.
We also have enough cash for 36 months which is sufficient to reach these milestones.
Catalysts;
Complete data on Rett U.S. Study
Complete data on Parkinson’s Dementia Study
Top Line data for Avatar Study (Adult Rett Syndrome)
Top Line Data for Phase One A3-71
Top Line Data for Excellence Study, (pediatric Rett Syndrome)
Top Line Data for A2-73 Alzheimer’s Disease
Additional Rare disease not yet disclosed
Focusing on activation of the body’s own defense mechanism to provide support to avoid or reduce the stress which can be part of these diseases.
Sigma One activation is beneficial for downstream activities such as;
Inflammation Reduction
Restoration of Autophagy
Reduction of Abeta and Tau
Rett Syndrome Trial:
Safety profile the same for placebo and active arm
No Adverse events.
All patients continued Study with no drop outs.
All patients continued in the extension study which was recently extended from twelve weeks to an additional six months to now 36 weeks by the FDA
Alzheimer’s disease Phase 2a; A2-73
Those with sufficient, 30 to 50 mg doses benefitted more than lesser
doses of 10 to 30 mg over 148 weeks.
Parkinson’s Dementia Phase2;
The cognitive features, Episodic Memory, Attention, Language Visual Spacial Skills,
Executive Function all improved with A2-73 in the Double Blinded, Placebo Controlled Study.
The Episodic Memory Endpoint correlates highly with ADAS Cog which is the Primary Endpoint of our Alzheimer’s Disease Study.
There was a dose response in this Placebo Controlled study where the Placebo Arm declined while the Medium 30mg dose stopped decline and the 50mg dose Improved Cognition.
This high correlation data point provides a comfort level for being replicated in the Phase2b/3 Alzheimer’s Study.
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