I have viewed ProQR as PYC’s closest competitor. Both were small companies focused on antisense therapies for rare eye disease. The key differences have been in the antisense technologies used - PYC uses PPMOs while ProQR has been using naked antisense (ASOs) - and the fact that ProQR was a few years ahead of PYC.
ProQR’s troubles have been caused by its lead candidate, sepofarsen, failing to show efficacy in a pivotal trial. One of the challenges with antisense therapies is achieving efficacy at a dose that is also safe.The advantage of PPMOs over ASOs is that greater efficacy can be achieved at a lower dose.
The following is form a PYC announcement (3 September 2019)
‘PYC’ has successfully completed a competitive evaluation of a drug that includes our delivery technology compared to a ‘naked’ Anti-Sense Oligonucleotide (ASO) that does not.
The drug that includes our delivery technology is effective at a dose of less than 1 microgram per eye in a mouse and the drug that does not include our delivery technology is not effective at either 1 microgram or 10 micrograms per eye. The results show that PYC’s delivery platform can make ASOs into potent drugs at doses that are otherwise ineffective – meaning better drugs with fewer side effects.
Delivering more drug to the target in a non-toxic manner is the rate-limiting step in the burgeoning field of RNA therapeutics2 – an emerging field in which ASOs are a leading class of drug across a range of diseases. This data provides the central piece of pre-clinical evidence that our delivery platform is capable of achieving outcomes that competitive technologies alone cannot.
ProQR’s pivotal trial used a dose of 80 µg, after a 160 µg loading dose. The second injection was given after 3 months and then at 6 monthly intervals.
Despite ProQR’s “pivot”, it remains a competitor. A key focus area now will be CNS, like PYC. A different antisense approach will be used and ProQR has Eli Lilly on board as partner.
Also, the rare eye disease portfolio may make a comeback as ProQR is seeking a partner to take it forward. The question is, will there be any takers?
PYC Price at posting:
6.6¢ Sentiment: Hold Disclosure: Held
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