PYC Therapeutics Limited (ASX
YC) (PYC or the Company) has received a response from the US
Food and Drug Administration’s (FDA) Office of Orphan Product Development (OOPD) requesting data
using the Company’s lead drug (VP-001) in an animal model of RP/retinal dystrophy due to PRPF31
mutations or in patients with the disease before considering the Company’s request for orphan drug
designation further. PYC submitted an orphan drug designation request for VP-001 to the FDA’s OOPD
on 30 July 2020 and the OOPD responded on 23 October 2020 with the request described above.
The Company will consider whether there is an appropriate animal model available that will provide the
data requested in light of VP-001’s specific mechanism of action and/or whether to defer the provision of
further data in response to the OOPD’s request until the ‘first in human’ efficacy read-outs are available.
The request for further information by the OOPD does not impact on either:
i) the benefits of orphan drug designation should it be granted to VP-001; nor
ii) the development pathway or timeline for VP-001 through to market.
(20min delay)