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.

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.

Multiple Phase 1 PK/DDI studies also ongoing.

Prior IMPROVE-PKD study showed beneficial effects over 2 months. Addresses defective polycystin-mediated mechanotransduction.

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

Precision medicine approach analogous to Vertex's CFTR modulators for cystic fibrosis. Phase 1 (159 participants) fully completed.

Preclinical data showed cyst growth inhibition in ADPKD models. 180mg daily for 2 years.

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.

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.

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