PYC Therapeutics (ASX: PYC) Announces Positive VP-001 Clinical Data for RP11
In a significant investor update, PYC Therapeutics Limited (ASX: PYC) has released compelling clinical data for its investigational RNA therapeutic, VP-001. The results demonstrate that patients receiving the treatment achieve sustained PYC Therapeutics VP-001 vision improvement lasting 18 months after treatment initiation. This development positions VP-001 as a potential first-in-class treatment for Retinitis Pigmentosa type 11 (RP11), a blinding childhood eye disease with no currently approved therapies.
The clinical-stage biotechnology company announced in a recent ASX announcement that ongoing Phase 1/2 clinical trials show sustained improvement in vision in the treated eye up to 18 months. Crucially, VP-001 outperforms both the patient’s untreated eye and a natural history control group across all key registrational endpoints.
Sustained Vision Gains Confirmed in Clinical Results
The data revealed that patients demonstrate vision improvement outcomes exceeding expectations across multiple clinical measures. VP-001 treated eyes show sustained gains of 6+ letters through 18 months of follow-up. This contrasts sharply with natural disease progression, which typically results in approximately 2 letters lost per year on Low Luminance Visual Acuity tests.
The clinical data encompasses 14 patients analysed at doses of 30 micrograms or higher through an 18-month follow-up period. Results from both Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) studies, including Open Label Extension (OLE) data, provide long-term safety and efficacy insights that validate the therapeutic approach.
Key clinical achievements include:
- Low Luminance Visual Acuity mean improvement of +6.1 letters in treated eyes versus -2.8 letters in fellow eyes (p = 0.0309).
- 25% of patients achieved gains of 10 letters or more, compared to only 8.3% of fellow eyes, representing a 3.67 times higher likelihood.
- Microperimetry whole grid sensitivity improved by +1.1 dB versus +0.56 dB in fellow eyes.
- The mean number of loci improving by more than 7dB reached 4.6 loci, compared to 2.88 loci in fellow eyes (p = 0.0003).
These results are particularly compelling as they compare treated versus untreated eyes within the same patients, which effectively eliminates individual patient variability and provides robust evidence of the drug’s efficacy.
What makes VP-001 a breakthrough treatment for RP11?
VP-001 is designed to rescue the gene expression deficit caused by the genetic mutation underlying RP11, thereby addressing the root cause of the disease. The drug candidate consistently demonstrates PYC Therapeutics VP-001 vision improvement outcomes that outperform both natural disease progression and untreated fellow eyes across multiple registrational endpoints.
Retinitis Pigmentosa type 11 is a blinding eye disease of childhood caused by a mutation in a single gene, and patients currently have no approved treatment options available. Low Luminance Visual Acuity (LLVA) is a critical measure for these patients, assessing their ability to see in dim lighting, which directly impacts quality of life.
Furthermore, the drug candidate utilises PYC’s proprietary drug delivery platform to enhance the potency of precision medicines within the rapidly growing RNA therapeutic class. This positions VP-001 amongst the most innovative approaches to genetic disease treatment.
How does VP-001 compare to natural disease progression?
The outcomes for treated eyes showed a remarkable divergence from the natural history of RP11. Whilst natural disease progression shows an average decline of approximately 2 letters per year, patients receiving VP-001 maintained sustained gains through 18 months of follow-up, illustrating a durable therapeutic benefit.
A natural history study followed 16 eyes from RP11 patients for at least 52 weeks, establishing a clear baseline against which VP-001’s efficacy could be measured. This comparative analysis provides regulatory authorities with high-quality evidence demonstrating a meaningful clinical benefit.
How safe is VP-001 for vision improvement in RP11 patients?
VP-001 continues to show a favourable safety and tolerability profile. There have been zero treatment-related serious adverse events reported in any patient who has received the drug candidate to date. This safety data, current as of 14 November 2025, covers all patients dosed across the clinical development programme.
The consistent safety profile across different dosing regimens provides confidence that the therapeutic benefit does not come at the expense of patient safety.
What are the registrational endpoints demonstrating efficacy?
PYC Therapeutics structured its clinical trials to measure outcomes across multiple registrational endpoints recognised by regulatory authorities. The PYC Therapeutics VP-001 vision improvement data spans functional vision and retinal sensitivity assessments, providing comprehensive evidence of therapeutic benefit.
Registrational endpoints measured include:
- Low Luminance Visual Acuity (mean change from baseline): Assesses functional vision in dim lighting.
- Low Luminance Visual Acuity (proportion achieving 10+ letter gains): Identifies clinically meaningful responders.
- Microperimetry (whole grid sensitivity): Measures retinal function across the visual field.
- Microperimetry (number of loci gaining ≥7dB): Quantifies improvement in specific retinal locations.
The convergence of positive results across these endpoints strengthens the regulatory case for approval by demonstrating a broad therapeutic benefit.
When will VP-001 begin registrational trials?
PYC Therapeutics is preparing for a Type D meeting with the FDA in Q1 2026 to align on the design for a registrational trial. The trial is expected to commence in 2026 following this regulatory meeting, positioning VP-001 to potentially become the first approved therapy for RP11.
Key development milestones include:
- RANZCO Conference Presentation (14-17 November 2025): The company will present updates on both its RP11 and Autosomal Dominant Optic Atrophy (ADOA) programmes.
- FDA Type D Meeting (Q1 2026): To align on registrational trial design.
- Registrational Trial Initiation (2026): Commencement of the pivotal study.
What is the market opportunity for VP-001?
PYC Therapeutics is focused on the precision medicine market, with VP-001 targeting a well-defined genetic condition with no existing treatments. The company has a market capitalisation of $769.9 million, with 646,977,974 shares on issue and a cash position of $135.1 million as of 27 October 2025.
The company’s proprietary RNA therapeutic platform addresses monogenic diseases—the indication class with the highest probability of success in clinical trials, offering a targeted approach to genetic disorders.
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