Ann: New Drug For Major Kidney Disease Effective In Human Models, page-54

Wasn’t Alnylam Pharma the company that Rohan suggested was kind of a “model” for how PYC could grow/develop?

Alnylam is the example Rohan has used multiple times now when explaining how genetic medicines targeting single gene mutations have a much higher chance of clinical success than other drug modalities.

He has used the Alnylam presentation slide below which highlights that Alnylam’s drugs, on entering Phase 1, already have a 64.3% probability of cumulative clinical success compared with 10.3% probability for other industry biomarker-driven programs. Alnylam now has a number of FDA approved drugs and has a market cap of US$21bn.



Regulus was mentioned as it is also developing a (MicroRNA) therapy for PKD and so it has “set up the path to market”. That therapy is currently in Phase 1. More information on that program can be found in the corporate presentation on the Regulus website.

The cell penetrating peptide PYC will use in the PKD program is different from the one being used in their two eye programs. This CPP has demonstrated excellent broad and deep distribution within the kidney, “nailing the delivery challenge” according to Rohan.
Apparently clinicians in the US who have seen the results have been very impressed.

As TB has pointed out, a particular attraction of this program is the ability to fairly quickly and easily see during human trials whether the drug is having an impact. As the drug is designed to increase the inadequate production of the protein Polycystin 1 (PC1), which leads to PKD, urine testing of PC1 is a simple way of checking whether the drug has indeed increased protein levels. And as the volume of the kidneys dramatically expand with the disease, drug effect in reducing kidney volume can be easily measured through imaging.

Rohan’s excitement was palpable and it is his clear intent to drive this asset through clinic to market as fast as is humanly possible. Manufacturing is already happening and further testing in the non-human primate model is slated for H1 next year. The NHP model will also provide data on liver distribution. In this instance, some drug distribution in the liver is preferred, as PKD can also cause cysts in the liver. The ambitious plan is to have the IND submitted and PYC-003 in the clinic by the end of next year.

There was a question about IP and the answer was that two patent applications, one for composition of matter and the other for RNA sequence, have already been filed.

There was also a question regarding recruitment challenges. Rohan said he’s not anticipating recruitment challenges because clinicians are already excited and on board with the program.

When asked about potential for partnering, Rohan said that there really hasn’t been time yet for any proper discussions but he suspects that there will be a huge appetite by both larger biotechs and by pharma. The plan is for PYC to drive the asset further forward until the “terms are more in favour of PYC”. The steel in his reply made clear that he is fully aware of the commercial opportunity of this PKD asset and it will not be given away too early for a pittance.