Hi. Actionaire,We can target specific cells and also deliver our...

Hi. Actionaire,

We can target specific cells and also deliver our drugs effectively. We can deliver the drugs at much lower doses causing a large reduction in the potential for toxicity. About 100 times less.

The in human model is a useful way to determine if a drug candidate is likely to be successful. Not a guarantee but PYC feel it is likely to be a good indicator.

They will still need to run trials for both drugs to prove their value. PKD is significantly behind the retinoblastoma candidate.

Perhaps the biggest factors with PKD is the huge market size it is much bigger than retinoblastoma. Also in the human models there was a suggestion of some return of function.

The biomarkers are a useful way to objectively assess function. From memory one was decreasing size with ultrasound measurement (The cysts in PKD cause the kidneys to be football size and therefore a reduction in size is good). I’m not sure of the other without going back over all the documents but I wonder if it is as simple as eGFR a commonly used clinical indicator of renal function (a routine blood test). These are likely to be quite effective and easy tests to monitor outcomes.

I don’t think it is clear if this will be quicker path to determine efficacy than retinoblastoma (Although I suspect you are right). The head start retinoblastoma has in trial status will probably mean we know about its efficacy in retinoblastoma before PKD.

As to when BP comes knocking who knows. Too many factors some of them not related to our company or our product. But in general my feeling is efficacy in one product is proof of concept. This will cause a big stir in my opinion.

2 reasons one is there is potential for treatment using this platform in additional conditions with a single gene mutation it seems almost formulaic to me. The second is the possibility of companies seeking our delivery platform for their products.

Sorry if there are a couple of details missing or if I haven’t quite answered your question. Just wanted to give you a low level answer in case some of the good posters on here don’t see your message and respond.

Eagle