Image of a researcher looking through a microscope
Source: Firebrick Pharma
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Biotechnology company PYC Therapeutics Ltd (ASX:PYC) has begun a follow-up study in the United States to assess the efficacy and safety of its drug candidate VP-001 to treat patients with blinding eye disease Retinitis Pigmentosa type 11 (RP11).

The latter condition – which develops in childhood – affects one in 100,000 people, and is caused by mutation in 1 copy of the PRPF31 gene leading to a protein insufficiency in photoreceptor and Retinal Pigment Epithelial (RPE) cells.

VP-001 – as the only drug candidate which has progressed to human trials for treatment of the condition – has been fast tracked by the US Federal Drug Administration (FDA).

The first candidate has been dosed as part of PYC’s multiple ascending dose (MAD) study which follows recent positive results yielded from a single ascending dose (SAD) study of VP-001 in patients with RP11.

Building on this knowledge, the company will dose patients in the MAD trial repeatedly with the two highest doses established as safe and well tolerated in the SAD trial – that is, 30 μg and 75 μg per eye – before assessing them for safety and efficacy endpoints.

Read-outs on these points are due for release before the end of 2024, and are set to support PYC’s design of the registrational trial required to facilitate a New Drug Application for this candidate, which will commence in 2025.

PYC has been trading at 12 cents.

PYC by the numbers
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