Sandor Batkai, Head of Medical Research and Intelligence at Cardior Pharmaceuticals examined how microRNA therapies can treat heart failure. Batkai explained that Cardior Pharmaceuticals is a spin off company of the Hanover Medical School in Germany, established in 2016. Their lead innovation is an antisense microRNA inhibitor targeting microRNA-132, a key regulator of cardiac pathophysiology.
MicroRNA-132 plays a key role in cardiac hypertrophy and remodelling, processes that lead to chronic heart failure. By targeting this molecule, Cardior’s compound works to restore normal protein expression, thereby improving cardiac function.
Batkai stated: “We developed an LNA/DNA mixmer antisense antimere, which is now our lead candidate, its progressing through small animal studies and large animal programmes for cardiac diseases.”
This LNA/DNA mixmer antisense antimere is water-soluble, intravenously administered, and is progressing toward subcutaneous delivery.
Early phases established the compound’s safety, pharmacokinetics, and efficacy in reducing circulating microRNA levels. Large animal models, including pigs, demonstrated the therapeutic’s ability to mitigate cardiac remodelling, with imaging and biomarkers confirming clinically relevant improvements. These findings informed the ongoing phase II study.
Batkai outlined the phase II trial: “We started the phase II trial with 280 patients split across two dose groups, 5 mg/kg and 10 mg/kg, in a Europe-wide multinational phase II study. The patients were administered three injections and the follow up of six months and an additional 6-month safety follow up.” Current results from the study showed no safety concerns so far.
Cardior aims to expand its platform into other forms of heart failure, such as cardiac hypertrophy, and is negotiating with the FDA to secure funding for further trials. Batkai emphasised that non-coding RNAs offer untapped therapeutic potential, expanding the target space beyond traditional gene-protein interactions. This novel modality represents a significant opportunity to address chronic diseases more effectively.
As their phase II study progresses, Cardior is committed to transforming heart failure treatment and improving patient outcomes through innovative RNA-targeting therapeutics.