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Ann: CEO letter and Operational Update, page-16

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Akki

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18/01/19

23:02

Post #: 37161351
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Originally posted by andrewk65 ↑

Originally posted by TonyIndo
The company has already shown successful delivery of Cre into kidney cells in-vitro, which has validated the technology in vitro.

In the announcement dated the 31/10/18 this was highlighted...

‘"Can our CPPs deliver a therapeutic cargo to a tissue/cell affected by a disease process in a manner that enables the correction of that disease’?

We will answer this question for multiple cargoes and in multiple tissues in therapeutic read-outs (both in vivo and ex vivo) in 2019."

The liver was one of the tissue/cells highlighted on page 5 in the matrix diagram and has already shown in vivo validation. And if I'm not mistaken, that was with the first generation CPPs - FPP001?

https://phylogica.com/wp-content/uploads/2017/11/171127_Company-Presentation-AGM-2017.pdf

Yesterday's update...

"We are greatly encouraged by the in vitro (test tube) performance of our ‘second generation’ CPPs which include candidates capable of delivering 25-150 times as much cargo into a target cell when compared to the cargo alone."

and...

"Given the extent of the outperformance of our second generation CPPs in vitro, this bodes well for our imminent therapeutic in vivo read-outs."

and..

"1H2019 represents an opportunity to show that our ‘second generation’ CPPs yield meaningful in vivo read-outs, demonstrating our ability to correct human disease processes in animal models of that disease. Success in these models will propel our platform into the clinic.

Importantly, the ‘second generation’ CPP seen in Figure 2 is very attractive in terms of its ‘druglike’ properties (toxicity, manufacturability etc.) indicating an improved probability of success innavigating the IND approval process in the event that therapeutic in vivo efficacy is established."

Tony

thanks Tony. Im not sure if im missing something, just trying to join the dots. I know we got cre into the kidney cells and i know our matrix shows quite a bit of in vitro and in vivo success already. The thing i dont clearly understand is last i heared our lead fpp was not particularly cell specific and current (or last priority at least) was to scan for more cell specific fpp. That led me to believe that indications like kidney or heart cell penetration were good but dont as yet indicate the fpp is specific, it just means it has proven to penetrate these cells well, amongst others. The issue still being if given by iv will penetrate pretty much everything it contacts before it gets to liver etc? Results so far at least in vitro are all on specific isolated cell types aren't they? Its all good progress but i would love to understand exactly where we are at and where we need to be at.

Ie is this all just interesting science if we can't give by iv and have systemic cell specific penetration, or is there also a use for fpp if it has to be isolated and deliberately applied to target cells?

Expand

I think most ASO’s absorbtion through the liver & kidney but that’s also where they find toxicity and halted them over the years ?

Initially i thought when they where going after ASO’s they might be finding an alternative to the liver which would open a big window. But maybe not ?

Last edited by Akki: 18/01/19

PYC Price at posting: 2.8¢ Sentiment: None Disclosure: Held
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