Precision RNA therapeutics for genetic diseases, lead program VP-001 for retinitis pigmentosa
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Outcome quality (positive/negative readouts) reflects the science, not execution — shown above but excluded from the score.
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These changes were automatically detected by comparing ClinicalTrials.gov records over time. A slipped date means the trial's expected completion moved later; pulled forward means it moved earlier.
| Event | Type | Confidence | Impact | Date | Status |
|---|---|---|---|---|---|
| VP-001 Phase 1/2 RP11 further data Continued patient dosing and follow-up data in retinitis pigmentosa type 11 | data readout | Expected | arrow_upwardHigh | 30 June 2026 | Upcoming |
| PYC-003 PKD Phase 1b MAD trial data readoutopen_in_new Phase 1b Multiple Ascending Dose clinical trial data for PYC-003 in polycystic kidney disease patients expected to be reported, evaluating safety, tolerability, and efficacy endpoints including urinary PC1 protein levels, total kidney volume, and glomerular filtration rate. [Source: PKD - Initiation of Multiple Dose Clinical Trial, 2026-05-06] | data readout | Expected | arrow_upwardHigh | 31 Dec 2027 | Upcoming |
| PHASE1/PHASE2 data readout — A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-open_in_new Primary completion expected for NCT06852963. Indication: {"Retinitis Pigmentosa 11","Retinal Degeneration","Retinal Disease","Eye Diseases Hereditary","Retinal Dystrophies"} | data readout | Speculative | removeMed | 8 Mar 2028 | Upcoming |
| PKD - Initiation of Multiple Dose Clinical Trialopen_in_new PYC Therapeutics has initiated a Phase 1b Multiple Ascending Dose (MAD) clinical trial for PYC-003 in polycystic kidney disease patients, with the first patient dosed. The trial will evaluate safety, tolerability, and efficacy endpoints including urinary PC1 protein levels, total kidney volume, and glomerular filtration rate, with data expected in CY27. | other | Confirmed | removeMed | 6 May 2026 | Completed |
| PHASE1/PHASE2 data readout — Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosoopen_in_new SUNDEW and MYRTLE Phase 1 studies with PYC-001 for ADOA demonstrated no treatment-related serious adverse events across all dose cohorts (3-60 mcg), with mostly mild and procedure-related adverse events, and encouraging improvements in visual acuity in ADOA patients. | data readout | Speculative |
| removeMed |
| 3 May 2026 |
| Completed |
| VP-001 EMA — Orphan Drug Designation Granted for RP11 by EMAopen_in_new The European Medicines Agency (EMA) has granted Orphan Drug Designation to VP-001, PYC Therapeutics' investigational RNA therapy candidate for the rare blinding eye disease Retinitis Pigmentosa type 11. The designation provides benefits including 10 years of European market exclusivity upon approval, scientific advice, and reduced regulatory review fees. | regulatory decision | Confirmed | removeMed | 26 Apr 2026 | Completed |
| VP-001 FDA — Regulatory Alignment on Registrational Trial Requirementsopen_in_new PYC Therapeutics held a Type D meeting with the FDA to align on the registrational study design intended to support a New Drug Application (NDA) for VP-001 in RP11, reaching agreement on the primary endpoint, key secondary endpoints, inclusion of a sham control arm, and incorporation of natural history study data. The FDA also highlighted the availability of the Special Protocol Assessment procedure to further confirm trial details. | regulatory decision | Confirmed | removeMed | 15 Mar 2026 | Completed |