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During the congress, E-Posters will be accessible to all participants on the congress website 24/7, as well as in the E-poster stations in the congress center.
Preparing your E-Poster
Please review the E-Poster format requirements carefully when preparing your E-Poster. Should your E-Poster not meet the mentioned requirements, it may not be displayed as described above.
E-Poster Submission Deadline
Please prepare and upload your E-Poster no later than March 14, 2026 11.59PM CET. After this date, you will no longer be able to prepare and upload your E-poster and it will not be displayed and accessible on the congress website.
Please follow the instructions below to input your abstract title.
Abstract titles should be brief and reflect the content of the abstract.
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is caused by haploinsufficiency of the PKD1 gene, resulting in reduced levels of Polycystin 1 (PC1) protein. The loss of PC1 results in the development and progressive enlargement of fluid-filled renal cysts. PYC-003 was designed as a disease modifying therapy to address this underlying pathology in ADPKD. It is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a cell-penetrating peptide that binds to and stabilises PKD1 messenger RNA. This leads to an increased production of the healthy copy of PC1 protein. This approach has previously demonstrated efficacy in reducing cystic phenotype in a 3D cyst assay derived from ADPKD patient cells.
This is a three-part Phase 1, first-in-human trial (NCT06714006) comprising of Part A (N=8 per cohort), a double blinded randomised placebo-controlled single ascending dose (SAD) study in healthy volunteers (HVs)and Parts B and C, open-label studies conducted in ADPKD patients- with Part B (N= 6 per cohort) evaluating single ascending doses and Part C (N= 12 per cohort) evaluating multiple ascending doses. ADPKD patients underwent pre-screening to confirm eligibility prior to formal screening. Key pre-screening criteria included an estimated glomerular filtration rate (eGFR) >30mL/min/1.73m2, Mayo imaging classification of C, D and E, and a genetically confirmed PKD1 mutation. The safety, tolerability, pharmacokinetics (PK) and immunogenicity of PYC-003 were assessed across escalating dose cohorts, where Safety Review Committee (SRC) evaluations guided dose escalation.
Across SAD cohorts in Healthy Volunteer and ADPKD patients, SRC-approved safety data indicated that PYC-003 was well tolerated with no treatment related Serious Adverse Events (SAEs) and no abnormal changes in serum or urinary biomarkers. PK outcomes were characterised in healthy volunteers up to 4 mg/kg and in ADPKD patients up to 1.2 mg/kg. Integrated PK/PD modelling predicts that PYC-003 reaches therapeutic concentrations in human kidneys at doses that are safe and well tolerated. Consistent with this, the minimum effective concentration of PYC-003 is 300 nM in ADPKD patient-derived models, which is below the minimum target-tissue concentration predicted by the PK/PD model for 1.2 mg/kg doses administered once every six weeks.
SRC-approved safety data suggest that PYC-003 is well tolerated in HVs and ADPKD patients, with no significant safety concerns identified to date. These early-phase results support the continued clinical development of PYC-003 as a promising therapeutic candidate for the treatment of ADPKD.
