PROTALIX BIOTHERAPEUTICS, INC. (PLX)

[Spideyboy]

Hi Kronberg, aplogies the late reply, been travelling a bit, and currently in China. Though in Shanghai where the virus isn't really an issue.

On the PSP, it's a good question. I just did a little catch-up on the literature on this, and it seems that for both the FDA and EMA, they have regulations in place which require new drugs to have a PSP unless not relevant to a pediatric population (unsafe or ineffective in children, lack benefit for children or are for diseases and conditions that only affect the adult population).

However things get a little more vague when we dig deeper.

From what I can see, the FDA I would say encourages the PSP to be looked into after Adult Phase II data if there is a good safety profile. But in reality it seems it happens much later if at all. Where in a lot of pediatric studies are 'deferred' until after adult data. Link below concerns FDA/US standpoint.
https://www.europeanpharmaceuticalreview.com/news/81402/drug-testing-children/

Thus it would appear to me that both the FDA and PLX and Chiesi are happy not to defer until after adult data. The primary contributor to this decision would be a good safety profile as you mentioned, though the efficacy data will I would feel definitely contribute highly to the FDA going through with this decision considering Fabryzyme is already on the US market for the pediatric population.

Things look largely similar with regards the EMA and their PIP (Pediatric Investigation Plan). https://www.ema.europa.eu/en/human-regulatory/research-development/paediatric-medicines/paediatric-clinical-trials

In a nutshell, I take this as good news, as the FDA must be happy with all the safety data to date and that should include quite a few samples and maybe even all of the Bridge safety data. This doesn't directly indicate that the efficacy is good, but as mentioned I would expect this to be the case. Also it is a good sign of confidence from PLX & Chiesi that they have not chosen to defer the pediatric study and should also speak to their confidence in both the safety and efficacy.

That news couple with the fact that I was expecting data on Bridge to come out this week or next and then with the 5 presentations coming up this week from today to Thursday from the Dror and KOLs and PRX-102 Clinical Investigators hopefully means something good is close.

Looking forward to this week!