Why it matters: Mironid is moving into human testing as several rival ADPKD programmes reach Phase 2 or prepare for pivotal development. The company will need to show that its approach can improve on tolvaptan, the only drug approved to slow the disease.
Zoom in: The Scottish National Investment Bank joined existing investors Roche Venture Fund, Epidarex Capital, Sofinnova Partners, BioGeneration Ventures and the University of Strathclyde in the round.
- CEO Neil Wilkie told Pharmaceutical Technology that the financing should fund the company through the generation of initial clinical data. Mironid’s previous financing, a 2023 Series A extension, brought its total funding at the time to £35 million (€41 million).
How it works: Mironid’s orally available LoAc molecules activate long-form PDE4 enzymes, increasing the breakdown of cAMP. Abnormally high cAMP levels promote cell growth and fluid secretion into the cysts that progressively damage the kidneys in ADPKD.
- Tolvaptan, sold by Otsuka Pharmaceutical as Jinarc in Europe, reduces cAMP indirectly by blocking the vasopressin V2 receptor. It can slow disease progression but commonly causes thirst and increased urination and requires liver monitoring.
Between the lines: Mironid’s financing announcement refers to a lead LoAc candidate but does not name the molecule, describe its clinical trial or provide a development timeline.
- Public records fill in part of that gap. The EU clinical trial registry lists MR-L45 in a first-in-human Phase 1 study sponsored by Mironid and is the company’s only asset currently identifiable in a public clinical registry. The study is recruiting 72 healthy adults at one site in Groningen, in the Netherlands. It began in July and is assessing safety, tolerability and pharmacokinetics.
- However, Mironid has not confirmed that MR-L45 is the lead candidate referenced in the financing announcement. The company told European Biotechnology that it could not answer questions about the trial because it had not yet announced the programme’s next stage.
What is known: Wilkie told FirstWord Pharma that Mironid has completed the GLP toxicology and safety pharmacology studies required before clinical development. He said the company’s LoAc molecules reduced cyst numbers and kidney volume in preclinical models, alongside encouraging safety findings. Mironid has not publicly provided the underlying numerical data or identified which candidate generated those results.
- The most detailed public efficacy data concern MR-L22, a different LoAc compound. In a 2024 European Renal Association abstract, an oral dose of MR-L22 lowered urinary cyclic AMP, or cAMP, by more than 40% in rats.
- MR-L22 also reduced kidney volume, cyst burden and disease markers in an ADPKD mouse model. The researchers reported fewer effects on urine production than with tolvaptan, although they could not establish whether combining the two treatments provided an additional benefit.
The big picture: Mironid is entering behind three differently positioned programmes:
- Farabursen: Novartis acquired Regulus Therapeutics for up to $1.7 billion to obtain the injectable miR-17 inhibitor. It has completed Phase 1b testing in ADPKD patients, and Novartis presented the design of a global Phase 3 trial in May.
- ABBV-CLS-628: Calico’s anti-PAPP-A antibody is being administered intravenously every four weeks in a randomized Phase 2 trial.
- VX-407: Vertex Pharmaceuticals’ oral corrector is in Phase 2 but targets only certain PKD1 variants, representing up to about 10% of the ADPKD population.
What to watch: Mironid could potentially differentiate through oral dosing, broad genetic applicability and fewer side effects than tolvaptan. For now, those advantages remain preclinical hypotheses, while its competitors are already generating data in people with ADPKD.
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