Ann: Trading Halt, page-49

I contacted Rohan yesterday to try to better understand the clinical significance of one of the items in the presentation.

Some points he made which I think are worth sharing:

1. Apoptosis is the key.
2. The ATP itself (Figure 2) is an interim read-out (hence the greater focus on apoptosis as the ultimate read-out).
3. Anything that stops the Retinal Ganglion Cells from undergoing apoptosis is clinically meaningful. It is the cell death (apoptosis) of the RGCs that causes blindness in ADOA so every additional RGC that remains alive confers better vision for ADOA patients.
4. He views the results as “very impressive”.

From the presentation

Rescue from apoptosis is the primary non-clinical predictor of efficacy of this drug candidate in clinical development....

PYC’s ADOA drug candidate has now proven its ability to prevent apoptotic cell death (see Figure 3) in models derived from patients with ADOA, reducing apoptosis to levels close to those observed in healthy individuals....

'If the cell survival and functional benefits that we see in patient-derived models treated with this drug candidate are replicated in the clinic, this has the potential to change the lives of ADOA patients in a very meaningful way’ (Sue Fletcher).