PYC Therapeutics (ASX: $PYC) has commenced a Multiple Ascending Dose (MAD) study to assess the safety and efficacy of its investigational drug candidate, VP-001, designed for patients with the blinding eye disease Retinitis Pigmentosa type 11 (RP11). The study follows positive results in a Single Ascending Dose (SAD) study and aims to evaluate the safety and efficacy profile of the drug candidate.
The MAD study marks a significant milestone in our efforts to address the unmet medical needs of RP11 patients. We are encouraged by the progress of our investigational drug candidate, VP-001, and are committed to advancing the development program to support a New Drug Application. The safety and efficacy read-outs from the MAD study are crucial in informing the design of the registrational trial, which, if successful, could lead to the first approved therapy for RP11, addressing a major unmet need in this patient population.
PYC Therapeutics has initiated a Multiple Ascending Dose (MAD) study to evaluate the safety and efficacy of its investigational drug candidate, VP-001, for patients with Retinitis Pigmentosa type 11 (RP11). The study aims to assess the safety and efficacy profile of VP-001 in patients with confirmed PRPF31 mutation-associated retinal dystrophy. The company anticipates safety and efficacy read-outs from the MAD study before the end of the year, which will inform the design of a registrational trial expected to commence in 2025. If successful, VP-001 could become the first approved therapy for RP11, addressing a significant unmet need in this patient population. PYC's ambitions include advancing the development program and leveraging the fast track status granted by the FDA to bring precision therapies to patients with genetic diseases.