Drug Pipeline

16 drugs currently in clinical development for PKD/ADPKD. Data sourced from ClinicalTrials.gov.

What do the trial phases mean?

Pipeline Overview

Each row shows a drug's progression through clinical trial phases

Dapagliflozin

Recruiting

Metformin

Recruiting

Farabursen (CYX082)

Not Yet Recruiting

ABBV-CLS-628

Recruiting

Empagliflozin

Recruiting

JMKX003142

Recruiting

Tirzepatide

Recruiting

VX-407

Recruiting

Bempedoic Acid

Not Yet Recruiting

Rotigotine

Not Yet Recruiting

Atorvastatin

By Invitation

Tamibarotene

Active

Probenecid

Completed

AZD1613

Recruiting

PYC-003

Recruiting

AL01211

Completed
Recruiting
Not Yet
Done
Upcoming
Legend:
Recruiting
Not Yet Recruiting
Active
Completed

Dapagliflozin

Phase 3
Recruiting STOP-PKD
University of Cologne — Cologne, Germany
SGLT2 inhibitor — first dedicated Phase 3 in ADPKD
Started: 2025-12
420 participants

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

View on ClinicalTrials.gov

Metformin

Phase 3
Recruiting IMPEDE-PKD
University of Queensland — Brisbane, Australia
AMPK activator — inhibits CFTR (reduces cyst fluid secretion) and indirectly inhibits mTOR pathway
Started: 2022-11
1174 participants

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

View on ClinicalTrials.gov

Farabursen (CYX082)

Phase 3
Not Yet Recruiting Phase 3 Registrational Trial
Novartis (acquired Regulus Therapeutics) — Basel, Switzerland
Anti-miR-17 oligonucleotide — inhibits microRNA-17, restoring polycystin-1/2 protein production to reduce cyst growth. 300mg SC every 2 weeks.
Started: 2025

Phase 1b showed htTKV growth halted (0.05% vs 2.58% placebo). Novartis acquired Regulus for $1.7B (closed Jun 2025). Phase 3 registrational trial planned; data presented at ASN Kidney Week 2025 and WCN 2026.

View on ClinicalTrials.gov

ABBV-CLS-628

Phase 2
Recruiting ANCHOR
Calico Life Sciences / AbbVie — South San Francisco, CA, USA
Undisclosed mechanism; IV infusion every 4 weeks for 92 weeks
Started: 2025-06
240 participants

Multicenter across ~100 sites in 11 countries. Primary endpoint: Total Kidney Volume change via MRI at Week 96.

View on ClinicalTrials.gov

Empagliflozin

Phase 2
Recruiting EMPA-PKD / SIDIA / Colorado Feasibility
Multiple Sponsors — Germany / Switzerland / USA
SGLT2 inhibitor — testing safety in ADPKD with or without tolvaptan co-administration
Started: 2023
134 participants

Three parallel trials: EMPA-PKD (NCT06391450, Hannover, n=44), SIDIA (NCT06435858, Zurich, n=40, recruiting), Colorado Feasibility (NCT05510115, n=50).

View on ClinicalTrials.gov

JMKX003142

Phase 2
Recruiting Phase 2 ADPKD
Jemincare — China
Oral small molecule — mechanism not publicly disclosed. Targets TKV reduction in rapidly progressive ADPKD.
Started: 2025-03
135 participants

Multiple Phase 1 PK/DDI studies also ongoing.

View on ClinicalTrials.gov

Tirzepatide

Phase 2
Recruiting Tirzepatide in Overweight ADPKD
University of Colorado Denver — Denver, CO, USA
GLP-1/GIP dual receptor agonist — targets visceral adiposity, insulin resistance, and kidney inflammation
Started: 2025-03
126 participants

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

View on ClinicalTrials.gov

VX-407

Phase 2
Recruiting AGLOW
Vertex Pharmaceuticals — Boston, MA, USA
Oral small molecule — likely polycystin-1 modulator/corrector for specific PKD1 gene variants
Started: 2025-11
24 participants

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

View on ClinicalTrials.gov

Bempedoic Acid

Phase 2
Not Yet Recruiting BEAT-PKD
University of Vermont (DoD-funded) — Burlington, VT, USA
ACLY inhibitor / AMPK activator — FDA-approved for cholesterol, pro-drug activated in kidney/liver
Started: 2026-07
120 participants

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

View on ClinicalTrials.gov

Rotigotine

Phase 2
Not Yet Recruiting ETERNAL-PKD
University Hospital Rouen — Rouen, France
Dopamine D5 receptor agonist (transdermal patch) — restores endothelial mechanosensitivity and NO release
Started: 2026-03

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

View on ClinicalTrials.gov

Atorvastatin

Phase 2
By Invitation Atorvastatin + Sodium Bicarbonate Pilot
Taipei Medical University — Taipei, Taiwan
Statin combined with acidosis correction to slow CKD progression in ADPKD
Started: 2024
30 participants

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

View on ClinicalTrials.gov

Tamibarotene

Phase 2
Active Phase 2a ADPKD
Rege Nephro Co., Ltd. — Tokyo, Japan
Synthetic retinoid (retinoic acid receptor agonist) — modulates renal cell proliferation/differentiation
Started: 2023-12
70 participants

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

View on ClinicalTrials.gov

Probenecid

Phase 2
Completed Probenecid + Tolvaptan
Mayo Clinic / Otsuka America — Jacksonville, FL, USA
Reduces tolvaptan-induced polyuria — makes tolvaptan therapy more tolerable
Started: 2022-09
36 participants

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

View on ClinicalTrials.gov

AZD1613

Phase 1
Recruiting PIONEER-PKD
AstraZeneca — Cambridge, UK
PAPPA-1 inhibitor — reduces local IGF signaling that drives cyst growth
Started: 2025-11
40 participants

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

View on ClinicalTrials.gov

PYC-003

Phase 1
Recruiting First-in-Human PKD1
PYC Therapeutics — Perth, Australia
Peptide-PMO conjugate antisense therapy targeting PKD1 mutations, delivered IV
Started: 2025-04
116 participants

Three-part study: healthy volunteer SAD, then ADPKD patient SAD and MAD. Sites in Australia/New Zealand.

View on ClinicalTrials.gov

AL01211

Phase 1
Completed First-in-Human Study
AceLink Therapeutics — China
Glucosylceramide synthase (GCS) inhibitor
Started: 2021
69 participants

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

View on ClinicalTrials.gov

Last updated: May 2026. Information sourced from ClinicalTrials.gov and published research.
Trial status may change — always verify with the trial sponsor for the most current information.