HighTide Therapeutics presents HTD1801 results for polycystic kidney disease at ASN Kidney Week 2026

USA—HighTide Therapeutics has presented preclinical findings at ASN Kidney Week 2026 in Denver, US, on October 22, showing that HTD1801 reduced kidney cyst growth in a human organoid model of autosomal dominant polycystic kidney disease (ADPKD).

The findings, presented at the 59th Annual Meeting of the American Society of Nephrology (ASN), highlight the drug candidate’s potential to target key mechanisms involved in cyst formation, inflammation and kidney damage.

Study findings

ADPKD is the most common inherited kidney disorder.

It causes fluid-filled cysts to develop and enlarge in the kidneys, with approximately half of affected patients developing kidney failure by age 60.

Although treatments can slow disease progression in some patients, safety concerns and eligibility restrictions leave significant gaps in care.

HighTide Therapeutics is developing HTD1801, a multifunctional drug candidate that targets the AMPK and NLRP3 pathways.

By activating AMPK, the compound may suppress cyclic adenosine monophosphate (cAMP)-driven cyst growth.

Its inhibition of NLRP3, a component of the inflammatory response, may also help reduce kidney inflammation and fibrosis.

Researchers evaluated HTD1801 in human nephron organoids derived from induced pluripotent stem cells and carrying PKD1 mutations.

They used forskolin to induce cyst expansion and compared the results with those obtained using metformin and tolvaptan.

HTD1801 reduced the median cystic area fraction from 20.40% in the model control group to 10.70%.

The proportion of organoids with a low cyst burden, defined as less than 20%, increased from 46.9% to 97.3%.

Meanwhile, the proportion with a high cyst burden of at least 30% fell from 8.2% to zero.

Metformin reduced the median cystic area fraction to 13.12%.

Tolvaptan, an established treatment for selected patients with ADPKD, did not change cyst burden in this model, which the researchers reported was unresponsive to the drug.

Implications for drug development

The findings extend previous research into HTD1801’s potential to address kidney impairment associated with metabolic abnormalities.

The company is investigating whether its dual activity against metabolic and inflammatory pathways could offer benefits across different kidney disease settings.

However, the latest results come from a preclinical organoid model and do not establish whether HTD1801 can slow disease progression or preserve kidney function in people with ADPKD.

Further studies will be needed to assess its safety, efficacy and potential role in clinical treatment.

HighTide Therapeutics Chief Medical Officer Dr Filip Surmont said progressive cyst growth can lead to declining kidney function and kidney failure, underscoring the need for treatments that directly address cyst expansion.

He said the results provide additional evidence supporting HTD1801’s development for ADPKD. The company plans to continue research to validate the findings and assess the candidate’s potential in kidney diseases.

The poster, titled HTD1801 Suppresses Cyst Expansion in PKD1-Mutant Nephron Organoids: Evidence for AMPK-NLRP3 Axis Efficacy in ADPKD, was presented on October 22, 2026, from 10 a.m. to noon Mountain Daylight Time.

It was listed as presentation TH-PO0204 in the ADPKD and Cystic Kidney Disease session.

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