Just a reminder of some of the potential benefits of this second clinical pipeline drug:
- PYC- 001 for ADOA is addressing a patient population size roughly 3 x that of VP-001 for RP11.
- Both VP-001 and PYC-001 have been granted US orphan drug designation, but PYC-001 in ADOA has also received Rare Paediatric Disease designation. This affords the possibility of receiving a Priority Review Voucher at time of marketing approval. Such vouchers can be sold and the current market value is US$100m+.
- As PYC-001 increases cellular bioenergetics, it has potential to treat other eye diseases of bioenergetic deficiency. These include glaucoma, geographic atrophy and Leber hereditary optic neuropathy (LHON). Further indications would be able to move straight through to Phase 2. Clinicians have already expressed interest in testing PYC-001 in these conditions.
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