A Bristol Myers-backed biotech wants to treat heart disease with non-coding RNAs. It's one step closer to proof-of-concept

25 Aug 2021
endpts.com
Acquisition
Is it possible to use strands of non-coding RNA, a burgeoning therapeutic area that researchers now believe plays an important part in how similar genes are expressed in different tissues, to treat heart disease? For Cardior Therapeutics, that’s the $76 million question. The Hanover, Germany-based biotech completed its Series B round Wednesday morning with the promise of advancing its lead ncRNA candidate into the next round of clinical trials. Having returned positive Phase Ib results last November, the program is now primed for a Phase II study in post-heart attack patients suffering from heart failure, CEO Claudia Ulbrich told Endpoints News. All Cardior needed was some cash to get it there. The biotech plans to launch the trial in the first half of next year, with a data readout expected by the end of 2024. Amsterdam VC firm Inkef Capital stepped in to lead Wednesday’s round. “The biggest achievement from the Series A was completing the Phase Ib trial last year, the program is already performing in stable heart failure patients,” Ulbrich told Endpoints. “So finally this is now the next inflection step to get a robust data package, proof-of-concept data and to advance the lead program further.” That program, dubbed CDR132L, is at the center of the biotech’s efforts in heart disease — though it has a few other candidates further down the pipeline. By utilizing non-coding RNA, or RNA strands that are not translated into proteins, CDR132L can affect several cardiovascular pathways at once. Specifically, CDR132L functions as an inhibitor on a micro-RNA known as micro-RNA-132, giving it the ability to act as a “master switch” at the cellular level, Ulbrich said. The micro-RNA itself is implicated during cardiac stress, increasing at high rates in such events and causing a plethora of downstream complications. On top of that, microRNA-132 plays a crucial part in how heart cells can change after an injury, Ulbrich said. With the potential ability to inhibit these mechanisms through blocking microRNA-132, the experimental drug aims to slow down scarring and enlargement of the heart, as well as show positive effects on clinically meaningful biomarkers. Ultimately, that will lead to a reduction in heart volume and cardiomyocytes, or the cells responsible for generating contractile force in the heart’s muscles. Cardior is not the only biotech to go after non-coding RNAs, as more than a few companies have turned their attention to the field. Just this year, Swiss biotech Haya Therapeutics pulled in a $20 million seed round back in May, and UK-based Transine Therapeutics got $12.8 million in seed cash in June. While Transine is shooting for CNS and ophthalmologic diseases with a long non-coding RNA approach, Haya is gunning for heart disease as well. But Ulbrich appears to enjoy the competition, pointing to Bristol Myers Squibb’s acquisition of MyoKardia last October as a sign that there’s enough Big Pharma appetite for many cardiovascular players. And she feels the mechanism directed at microRNA-132 is unique enough for Cardior to stand out as ncRNA continues to spur new biotech launches. The $13 billion MyoKardia buyout “clearly shows new modalities are clearly a need for these patients,” Ulbrich said, adding, “this one crucial microRNA drives the pathology in the heart.” In addition to Inkef, new investors such as Fund+, Sunstone, Hadean Ventures and Coparion joined the round. Cardior also saw participation from existing investors including LSP, BioMedPartners, Bristol Myers Squibb and High-Tech Gründerfonds.
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