The longer the experiments continue (post 7 days administration), the higher the outperformance, which could be much greater 'potentially' than the 400% already achieved.
What does the company need to achieve in order for their delivery platform to be successful? We know that a 30-60 x increase in cargo is required in order to correct a disease process occurring within a cell, however, it would also depend on the particular disease in question, so that percentage would most likely differ depending on the disease target one is going after.
The graph highlights a 33% increase already, just after 7 days post administration at a single dose. That's very impressive, so early on. Longer time course studies are currently underway. That's something I'm looking forward to.
Delivering a sufficient amount of cargo inside animal cells in a safe manner would be the major pre-clinical milestone in the development of a platform in this space.
The company highlighted...
"PYC’s CPP’s (including the CPP described above as demonstrating the 400% outperformance in the efficacy studies in the eye) demonstrated minimal toxicity at doses of 40mg/kg (the maximum dose allowed under the study)."
PYC were able to deliver the ASO cargo in three different segments of the eye, at a single dose of 1.6 micrograms per eye at 7 days post-administration (a dose that is substantially lower than competitive approaches). They were able to achieve successful exon skipping by the ASO in the cell's nucleus. The higher the doses without compromising safety, the higher the percentage of exon skipping as the CPP-ASO has gotten to its target. In other words, more exon skipping, means more drug has been delivered to the target. There was no toxicity observed even at a dose of 30mg/kg and very little toxicity even at 40mg/kg in a separate mouse model. Whereas the clinical benchmark peptide demonstrated toxicity when administered at the lower dose of 20mg/kg. Toxicity was observed for PepK and Pip6a at 10mg/kg and the animal ethics protocol for the study prevented dosing at higher levels with these CPPs.
"Therapeutic in vivo outcomes are the primary reference point for Pharma partners assessing whether pre-clinical (animal) outcomes will translate into clinical (human) outcomes"
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