The decision by the FDA in rejecting golodirsen, the second DMD drug by Sarepta, suggests the setting of a higher bar for approval of antisense oligonucleotides. The higher bar appears to be related to toxicity and serious adverse events which are additional to the question marks which remain over trial data for Exondys 51 in relation to efficacy. Arguably, the FDA has made the case for PYC.
The setback for Sarepta highlights the legitimacy of the strategy for drug development of ASOs by PYC. The key for the future development of ASOs and RNA therapy is assisted delivery. As Doug Ingram from Sarepta is quoted to have said "the [drugs] ... don't get into cells as efficiently as we would like". Touchè.
All eyes are now on the current program by PYC in inherited retinal diseases. Of course that is an overstatement for any number of reasons: we are yet to identify a drug molecule for IND enabling studies, inherited eye disease is a niche condition with relatively few players and as always more data is needed. Nonetheless, the case has been made for the critical importance of safe and efficacious delivery of ASOs. The billion dollar question is how to solve the delivery challenge.
Perhaps, eyes are on PYC after all.
PYC Price at posting:
3.7¢ Sentiment: Buy Disclosure: Held
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