Hong Kong, October 23, 2026: HighTide Therapeutics, Inc. (02511.HK) presented new preclinical data on its investigational therapy HTD1801 for autosomal dominant polycystic kidney disease (ADPKD) at Kidney Week 2026, the 59th Annual Meeting of the American Society of Nephrology (ASN) in Denver, US. The findings indicate that HTD1801 demonstrates potent inhibitory activity against cyst expansion in ADPKD models.
ADPKD is the most common inherited kidney disorder, with about 50% of patients progressing to kidney failure by age 60. Existing treatments can slow disease progression for some patients, but safety risks and eligibility limitations leave significant unmet medical needs. HighTide Therapeutics said HTD1801 targets the AMPK–NLRP3 axis, activating AMPK to suppress cAMP-driven epithelial cyst proliferation and inhibiting NLRP3 to reduce renal inflammation and fibrotic remodeling.
Preclinical study results
The study evaluated HTD1801’s effect on forskolin-induced cyst expansion in PKD1−/− human iPSC-derived nephron organoids, using metformin and tolvaptan as comparators. HTD1801 reduced the median cystic area fraction to 10.70%, compared with 20.40% in the model control group. The proportion of organoids with low cystic burden (less than 20%) increased from 46.9% in the control group to 97.3% with HTD1801, while those with high cystic burden (30% or more) dropped from 8.2% to 0%. Metformin reduced the median cystic area fraction to 13.12%, while tolvaptan did not affect cyst burden in this model.
Potential for disease modification
HighTide Therapeutics stated that these results expand the potential multidimensional renal benefits of HTD1801, which has previously shown promise in improving metabolic abnormality-associated renal impairment. The company said the findings validate HTD1801’s dual effects of AMPK activation and NLRP3 inhibition in ADPKD pathogenesis and support further clinical investigation as a possible disease-modifying therapy for ADPKD and other chronic kidney diseases.
Presentation and expert comment
The data were presented by Dr. Filip Surmont, Chief Medical Officer of HighTide Therapeutics, in a poster session titled “HTD1801 Suppresses Cyst Expansion in PKD1-Mutant Nephron Organoids: Evidence for AMPK-NLRP3 Axis Efficacy in ADPKD” (Presentation Number: TH-PO0204) on October 22, 2026, at Kidney Week 2026. “ADPKD continues to pose a substantial unmet medical need, as progressive cyst growth can lead to deterioration of kidney function and ultimately kidney failure, underscoring the need for effective therapies that can inhibit cyst growth,” said Dr. Surmont. “The significant suppression of cyst expansion observed with HTD1801 in this study provides new scientific evidence to support its further development in ADPKD and highlights its potential as a disease-modifying therapy.”
Company background
HighTide Therapeutics is a biopharmaceutical company focused on developing multifunctional, multi-targeted therapies for cardiovascular–kidney–metabolic diseases.
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