VP-001 Shows Sustained Vision Improvement Up to 18 Months in RP11 Trials
PYC Therapeutics reports sustained vision improvements in patients with Retinitis Pigmentosa type 11 treated with VP-001, showing promising safety and efficacy up to 18 months. The company plans to engage the FDA in early 2026 to finalize pivotal trial design.
- VP-001 shows sustained vision improvement in RP11 patients up to 18 months
- Treated eyes outperform untreated eyes and natural disease progression controls
- No treatment-related serious adverse events reported to date
- PYC to meet FDA in Q1 2026 to align on registrational trial design
- Registrational trial expected to commence in 2026
A Breakthrough in a Rare Genetic Eye Disease
PYC Therapeutics, a clinical-stage biotechnology company focused on precision RNA therapies, has announced encouraging results from its ongoing Phase 1/2 trials of VP-001, an investigational drug targeting Retinitis Pigmentosa type 11 (RP11). This rare, blinding eye disease, caused by a single gene mutation, currently has no approved treatments. VP-001 aims to correct the underlying genetic deficit, offering hope to patients facing progressive vision loss.
Sustained Vision Improvements Demonstrated
Data from the trials reveal that patients receiving VP-001 experienced sustained improvements in both functional vision and retinal sensitivity for up to 18 months after treatment initiation. Notably, the treated eyes consistently outperformed the untreated fellow eyes and a natural history control group across multiple key measures, including low luminance visual acuity and microperimetry sensitivity. These gains suggest that VP-001 may not only halt but potentially reverse aspects of vision decline in RP11.
Safety Profile Supports Further Development
Alongside efficacy, VP-001 has maintained a favourable safety and tolerability profile, with no treatment-related serious adverse events reported to date. This safety record is critical as PYC prepares to advance the drug into registrational trials, which are designed to provide the robust data necessary for regulatory approval.
Regulatory Pathway and Next Steps
PYC is preparing for a Type D meeting with the US Food and Drug Administration (FDA) in the first quarter of 2026 to align on the design of a registrational trial for VP-001. The company expects to initiate this pivotal study later in 2026, marking a significant step toward potentially bringing the first approved treatment for RP11 to market. Updates on this and other programs will be presented at the upcoming Royal Australian and New Zealand College of Ophthalmology meeting.
Looking Ahead
While the patient numbers remain limited and dosing has not been continuous, these early results provide a compelling foundation for further clinical development. If VP-001 continues to demonstrate durable benefits and safety, it could transform the therapeutic landscape for patients with RP11 and other monogenic diseases.
Bottom Line?
VP-001’s promising data set the stage for a pivotal trial that could redefine treatment for a devastating genetic eye disease.
Questions in the middle?
- Will the FDA agree on the proposed registrational trial design in early 2026?
- Can VP-001’s vision improvements be sustained beyond 18 months with continuous dosing?
- How will VP-001’s efficacy compare to emerging therapies in the genetic eye disease space?
