Tredenwater...this is what I was talking to you about in regards to the OLE from the P2b/3 study...see the information underlined in bold below.
[This was posted by Hosai...who copied it from FB]
Another recap of the annual shareholder meeting:
Hi, I got permission from a friends take to release his notes. Cheers.
They are calling themselves a regulatory stage CNS company.
Patients are peeling off of Trofinetide, and patients on Blarcamesine, through compassionate care schemes, are continuing on the drug. FDA gave them feedback on RETT to run another phase 3 trial because it would be unfair to Acadia (Trofinetide) who had a drug approved on a successful phase 3 trial if drug was approved despite missing statistical significance on endpoints. They are at a RETT conference concurrent with the ASM, to present a new trial (12 week), in which they will enroll 2x patients (~150) on a 1:1 ratio.
Saw a picture of the pill. It’s a clear capsule with branding on the coating.
They have not met with fda on Alzheimer’s because of the missed ADL, but now with the new draft guidance and bio-markers, and black box warning and failure of MAB uptake, feel much more confident to talk with them. When asked if European approval would be first and FDA second, he answered not necessarily because though they started with EMA, FDA is much quicker.
When asked about long-term efficacy, he said they have good data and RWE of AD patients on drug for 146 weeks. They have not seen any of the ATTENTION data on the OLE. When asked if FDA will accept aBeta as a biomarker using blood plasma, he said many companies have now adopted this method. On brain atrophy data, he used the following language (attenuated, stopped, delayed). He did not give any ground on timeline for EMA submission or peer review, but held to by ‘end of year’ though he conceded this was playing it safe, which I interpret as meaning it may happen earlier. We saw some new slides, one in which they do a much better job illustrating their drug vs the MAB’s, comparing their downstream approach to the upstream approach of activating the sigma-1 receptor, emphasizing Autophagy. In my opinion, focusing on Autophagy, is smart because it gets lots of headlines in health news as it relates to intermittent fasting and other trending health fads regarding cell health.
They have completed part A of Schizophrenia trial which was a dosing study and have now started part B. When asked about delays with PD/PDD, he answered strongly that major advances were being made in PD biomarkers, and testing doesn’t just come off the shelf at ‘Amazon’ as he put it, they are being developed, and thus they are waiting because they feel they can run a more effective trial with them. He used the 2b/3 AD trial as an example, suggesting at the time they initiated that trial, the biomarkers they were testing for were not at all common then, but are now common in most AD trials. His comments seem to be pointing toward the new alpha-synuclein biomarker discovered by the Michael J Fox foundation last year.
In a question asked about partnering versus buyout, he first responded by saying that a buyout is an extreme form of partnership and at the other end of the spectrum was doing everything themselves. He indicated he was open to all options, with the criteria that it would be whatever was in the best long term interest of shareholders.
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