PYC reports initial clinical trial results for its RNA therapy targeting retinitis pigmentosa

Key Takeaways

• PYC Therapeutics reported initial results from a phase 1 trial of an RNA therapy knows as VP-001 for people with retinitis pigmentosa 11 (RP11), which is caused by mutations in the PRPF31 gene.
• Based on the results, the company has filed a trial protocol amendment with the FDA to deliver a higher dose of the therapy to a group of patients with less advanced disease.

PYC Therapeutics, an Australia-based developer of RNA therapies, reported initial results from its Phase 1 PLATYPUS clinical trial in the US for an RNA therapy known as VP-001 for people with retinitis pigmentosa 11 (RP11), which is caused by mutations in the PRPF31 gene. In people with RP11, one copy of their PRPF31 gene is normal and producing a relatively normal level of protein while the other PRPF31 copy is mutated and not producing sufficient protein. The overall reduced level of PRPF31 protein for RP11 patients leads to retinal degeneration and vision loss.

PYC’s emerging therapy is designed to modify RNA, the genetic messages that cells read to make the proteins which are critical to the health and function of all the cells in the body. By modifying RNA, protein expression can be boosted or reduced, depending on the therapeutic need. VP-001 is a tiny piece of synthetic genetic material designed to decrease the amount of RNA expressed by a gene called CNOT3, which in turn boosts levels of PRPF31. 

A report delivered by Fred Chen, MBBS, PhD, associate professor, Lions Eye Institute, at the 2024 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), in Seattle, discussed initial results of the trial. A total of nine patients — three patients in three dosing groups (3, 10, and 30 μg) — have been dosed thus far. No serious adverse events were observed. One patient in the 30 μg group had a clinically meaningful improvement in retinal sensitivity as measured by microperimetry, a test that captures changes in retinal sensitivity at several locations in the central region of the retina. PYC has filed a trial protocol amendment with the FDA to deliver a dose of 75 μg to a fourth group of patients with less advanced disease.

For more information on this trial, visit https://clinicaltrials.gov/study/NCT05902962 or contact Ora Inc by calling 1-510-423-2680 or emailing VP001@oraclinical.com.

Edited by Miriam Kaplan, PhD

Source:  Foundation Fighting Blindness, Eye on the Cure research news, May 16, 2024; see source article