"Had only planned the PDD study as safety and PK information to decide whether a pivotal study would make sense, hence short 14 week study. Did not plan in advance to have 48 week OLE, but now very useful.
"He mentioned that the PDD ADS-COG 70% correlation to AD along with the SIGMAR1 biomarker results clearly stronger than the signal that got Aduhelm approved."
These comments reinforce further the force of the mRNA biomarker findings, something that Missling was not expecting to find (or he would have designed the PDD study differently).
I want to examine the PDD/AD 70 percent correlation remark in context. Missling is not going to be using a potential PDD NDA to promote the approval of the ongoing AD trial, obviously, although it does give a strong signal that the AD trial will also succeed. The fact that it may work in AD the same as PDD doesn't make the PDD case, alone, stronger. Instead, taken together the correlation and the validated biomarker strengthen his argument to the FDA that the mRNA biomarker, correlated for efficacy in both PDD and Rett 001, is very likely to do the same for Alzheimer's and all other CNS diseases that may involve the Sigma1 receptor. Missling is attempting to get the mRNA biomarker accepted as a CNS-wide biomarker that will ease his path in all future CNS NDAs.
Very useful report, Investor. Thanks for posting.