Mironid

Mironid successfully doses initial cohorts in Phase 1 clinical trial investigating the Company’s first-in-class LoAc® small molecule for patients with ADPKD

Mironid successfully doses initial cohorts in Phase 1 clinical trial investigating the Company’s first-in-class LoAc® small molecule for patients with ADPKD

• Phase 1 study is evaluating the safety, tolerability and pharmacokinetics of Mironid’s lead LoAc® small molecule in healthy volunteers
• Dosing follows Clinical Trial Application approval and marks the transition of Mironid into a clinical stage company

Glasgow, Scotland, October 1, 2026 – Mironid, a biopharmaceutical company developing small molecule therapeutics for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), a life-threatening hereditary kidney disease, today announces it has successfully dosed the initial cohorts in its Phase 1 clinical trial of its first-in-class LoAc® small molecule, marking Mironid’s transition to a clinical-stage company.

Neil Wilkie, CEO of Mironid said: “Dosing the initial cohorts in our Phase 1 study is a defining milestone for Mironid and an important step in the clinical development of LoAc®. It follows years of preclinical research characterising this novel mechanism that targets the underlying biology of ADPKD, a disease where there remains significant need for more effective treatment options. With the trial now progressing, we look forward to reporting initial data in the coming year.”

The Phase 1 clinical trial (EudraCT trial number 2025-524899-40-00) is a dose-escalation study, evaluating the safety, tolerability and pharmacokinetics of Mironid’s lead LoAc® small molecule in healthy volunteers. The initial single ascending dose (SAD) part of the study is designed to enroll up to 48 participants across six sequential cohorts.

Vicente E. Torres, MD, PhD, a Founding Member of the Mayo Clinic Translational Polycystic Kidney Disease (PKD) Center said: “ADPKD remains an area of significant unmet need, and there is a clear need for therapies that act on the underlying drivers of cyst growth rather than its downstream effects. Mironid's approach to modulating cAMP, as well as the strong preclinical data behind this molecule, has been encouraging. Reaching the clinic is an important step, and I look forward to seeing how the programme progresses.”

ADPKD is one of the more prevalent rare diseases and the most common hereditary kidney disorder. The disease is caused predominantly by mutations in the PKD1 or PKD2 gene and is characterized by uncontrolled growth of fluid-filled cysts in the kidney. Mironid’s first-in-class LoAc® small molecules represent the only drug class directly targeting cyclic AMP (cAMP), which is active in all stages of the ADPKD disease process from initiation through to end-stage, stimulating both cell proliferation and fluid secretion. Preclinical data shows significant efficacy and a favourable safety profile across all disease endpoints, including a reduction in cyst number and kidney volume. The ability of cAMP modulators to prevent new cyst formation and arrest the growth of existing cysts is indicative of the potential to offer an effective and durable treatment option with an improved side-effect profile for all ADPKD patients.

-ENDS-

Enquiries:

Mironid
Neil Wilkie, Chief Executive Officer
enquiries@mironid.com

ICR Healthcare
Mary-Jane Elliott / Lindsey Neville / Stephanie Cuthbert / Evi Useh
Mironid@icrhealthcare.com

About Mironid
Mironid is a biopharmaceutical company developing small molecule therapeutics for the treatment of life-threatening hereditary kidney disease. Its lead programme is for Autosomal Dominant Polycystic Kidney Disease (ADPKD), which is characterized by uncontrolled growth of fluid-filled cysts and is the most common hereditary kidney disorder, but has limited treatment options. Mironid’s first-in-class small molecule LoAc® drug candidates directly target the abnormally high kidney cAMP levels that drive cyst formation. The Company is led by an industry-experienced management team and supported by a strong advisory network and blue-chip investors including Roche Venture Fund, Epidarex Capital, Sofinnova Partners and BioGeneration Ventures. The Scottish National Investment Bank, the University of Strathclyde, Scottish Investment Bank and the European Investment Fund (EIF) are also investors in Mironid.