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Saturday, June 19, 2021 5:13:13 AM
A real, I think not bad, rare diseases reference comparison to $AVXL.
I mentioned Orphazymee not so long ago, which has now become a real illustration of the point I made in my previous post.
Niemann-Pick Disease (NCP) Type C is a Devastating Rare Genetic Disorder, Life-limiting with High Unmet Need. The sort of indication where the FDA presumably operates with a lower bar for approval.
I invested recently before the readout in the company's trial of the same drug (arimoclomol) in ALS, which unfortunately failed to meet its endpoints.
However, the key reason I invested was the NPC PDUFA date for the company's accepted NDA with priority review and Orphan Drug Designation, which was filed on the back of their NCP-002 Phase 2/3 study.
I did that essential based on slide 22 below because POST-HOC, as encouraged by FDA, excluding 3 patients with double functional
null mutations made the results in trial (arimoclomol n=34, placebo + routine care n=16) highly stat sig.
Interestingly Orphazyme for unexplained reasons had become another meme stock driven by the company's US listed ADR going from $5'ish to $56 in an instant, reflecting somewhat onto the main Danish listed stock too.
Of course I sold half of my Orphazyme stock for > 100% gain. The other half I kept because of the NCP results and the presumed lenient and friendly towards rare disease and orphan drug view given the FDA's post hoc suggestion.
Regardless, on the 17th June after trading close in Denmark the FDA sent a CRL to Orphazyme requesting further data meaning another trial.
But of course, I know with Anavex it will be entirely different because 'we got this!'.
I mentioned Orphazymee not so long ago, which has now become a real illustration of the point I made in my previous post.
Niemann-Pick Disease (NCP) Type C is a Devastating Rare Genetic Disorder, Life-limiting with High Unmet Need. The sort of indication where the FDA presumably operates with a lower bar for approval.
I invested recently before the readout in the company's trial of the same drug (arimoclomol) in ALS, which unfortunately failed to meet its endpoints.
However, the key reason I invested was the NPC PDUFA date for the company's accepted NDA with priority review and Orphan Drug Designation, which was filed on the back of their NCP-002 Phase 2/3 study.
I did that essential based on slide 22 below because POST-HOC, as encouraged by FDA, excluding 3 patients with double functional
null mutations made the results in trial (arimoclomol n=34, placebo + routine care n=16) highly stat sig.
Interestingly Orphazyme for unexplained reasons had become another meme stock driven by the company's US listed ADR going from $5'ish to $56 in an instant, reflecting somewhat onto the main Danish listed stock too.
Of course I sold half of my Orphazyme stock for > 100% gain. The other half I kept because of the NCP results and the presumed lenient and friendly towards rare disease and orphan drug view given the FDA's post hoc suggestion.
Regardless, on the 17th June after trading close in Denmark the FDA sent a CRL to Orphazyme requesting further data meaning another trial.
But of course, I know with Anavex it will be entirely different because 'we got this!'.
