Drug Pipeline

FDA approved April 2018 for ADPKD. The only drug specifically approved for PKD. Requires liver monitoring (REMS program). Causes significant polyuria (4–8L/day urine). TEMPO 3:4 enrolled 1,445; REPRISE (NCT02160145) enrolled 1,370 additional patients.

10mg daily for 36 months vs placebo. Primary endpoint: chronic eGFR slope. Sites across Germany, Netherlands, Austria, Spain.

Investigator-initiated European trial: hydrochlorothiazide 25 mg vs placebo on top of tolvaptan. Co-primary focus on eGFR decline and quality of life. 22 sites across the Netherlands, Germany, France, Spain, Belgium and Switzerland. Primary completion estimated July 2030.

Largest PKD trial to date. Prior Phase 2 (TAME-PKD) showed safety and encouraging trend. Up to 2000mg daily.

Phase 1b (NCT05521191, 68 patients, completed) showed htTKV growth halted (0.05% vs 2.58% placebo). Novartis acquired Regulus for $1.7B (closed Jun 2025). Phase 3 registrational trial design presented at ERA 2026 (Abstract #3398); no Phase 3 NCT ID registered yet.

Multicenter across ~100 sites in 11 countries. Primary endpoint: Total Kidney Volume change via MRI at Week 96. Study completion extended to August 2029; the Week 96 TKV readout is expected earlier.

Multiple Phase 1 PK/DDI studies also ongoing.

Prior IMPROVE-PKD study showed beneficial effects over 2 months. Addresses defective polycystin-mediated mechanotransduction. Dosing 4 mg/24h transdermal for 24 months; first patient enrolled May 2026.

With Washington University and Mayo Clinic (NIDDK/NIH). Restricted to overweight/obese ADPKD patients (BMI ≥ 27).

Preclinical data showed cyst growth inhibition in ADPKD models. 180mg daily for 2 years. Start pushed from July to September 2026 per the August 2026 registry record.

Open-label pilot study testing atorvastatin with or without sodium bicarbonate.

Three parallel trials: EMPA-PKD (NCT06391450, Hannover, n=44, Phase 4, active not recruiting), SIDIA (NCT06435858, Graubuenden Switzerland, n=40, recruiting), Colorado Feasibility (NCT05510115, n=50, active not recruiting).

Targets patients unable/unwilling to take tolvaptan. 52-week treatment. 8 sites in Japan.

Precision medicine approach analogous to Vertex's CFTR modulators for cystic fibrosis. Phase 1 (159 participants) fully completed. Phase 2a enrollment closed as of August 2026 with 26 participants; primary completion estimated July 2027.

Study completed. Aimed to improve urine osmolality and reduce excessive urination from tolvaptan.

Novel mechanism. SC/IV multiple ascending dose study. Sites in US, UK, China.

Three-part study: healthy volunteer SAD, then ADPKD patient SAD and MAD. Sites in Australia/New Zealand. Enrollment target raised from 116 to 166 in 2026.

GSK's first disclosed ADPKD programme. Randomised, double-blind, placebo-controlled: Part A in healthy participants, Part B in ADPKD patients, with blood and urine biomarker readouts. 23 sites across the US, Canada, UK, Germany, France, Spain, Belgium and the Netherlands. No longer recruiting as of July 2026.

Phase 1 in healthy volunteers completed June 2022. No Phase 2 registered yet.