First-In-Human Heart Failure Data of Cardior Pharmaceuticals´ Novel miRNA Antisense Therapy Presented at the 17th Global Cardiovascular Clinical Trialists Forum

• Excellent safety and tolerability of first-in-class compound CDR132L
• Novel mode of action confirmed by data on cardiac function and biomarkers

Hanover, Germany, December 8, 2020 - Cardior Pharmaceuticals GmbH, a clinical-stage biotech company focused on the development of non-coding RNA (ncRNA) based therapeutics for patients with cardiovascular diseases, today announced that its Chief Scientific Officer Thomas Thum, MD, PhD, has presented clinical data on the Company´s lead compound CDR131L at the 17th Global Cardiovascular Clinical Trialists Forum (CVCT) on December 7, 2020.

In his talk titled "The promise of novel miRNA antisense therapy in patients with heart failure", he summarized the first-in-human data of Cardior Pharmaceuticals´ novel miRNA compound CDR132L in patients with chronic heart failure. Building on a completely novel mode of action, the compound is an antisense oligonucleotide inhibiting the non-coding microRNA-132 (miR132) that directly regulates adverse cardiac remodeling. Thereby, the compound is supposed to halt or even revert heart failure.

In the Phase Ib trial, CDR132L met all endpoints and showed excellent tolerability and safety at all dose levels during the 120-day study period. No safety signals or unexpected adverse events were observed. Moreover, PK data confirmed strong dose-dependent linearity and specific target engagement. An exploratory analysis of multiple pharmacodynamic parameters, including measurement of NT-proBNP blood levels, showed beneficial effects on top of standard of care. Results were published in the European Heart Journal (doi:10.1093/eurheartj/ehaa898).

“These are the first clinical data of a potential miRNA-based therapy in heart failure,” said Faiez Zannad, MD, PhD, FESC is Professor of Therapeutics at the University of Lorraine in Nancy, France, Scientific Advisor to Cardior Pharmaceuticals and Founder and Chairman of CVCT. “We are excited that the efficacy signals previously observed in large animal models could be translated to a human setting. Provided that future clinical trials confirm these promising results, this approach may be transforming heart failure treatment as it directly addresses the roots of the disease.”

"Our goal is to provide new therapeutic options for heart failure patients by halting or even reversing the disease,” said Thomas Thum, Professor at Hannover Medical School and CSO of Cardior Pharmaceuticals. “The first-in-human data of our lead compound CDR132L are very encouraging and suggest that we have indeed discovered a master switch to control the disease. We are now looking forward to initiating Phase II clinical trials in 2021.”

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About CDR132L

CDR132L is an antisense oligonucleotide developed by Cardior Pharmaceuticals inhibiting the microRNA-132 (miR132), a non-coding microRNA that regulates cardiac hypertrophy and remodeling in cardiomyocytes by targeting well-defined pathways.

miR132 is a regulatory master switch to control cardiac function and a promising, causal therapeutic target in heart failure therapy. Expression of miR132 is increased in various pathological cardiac conditions in both animals and humans, and previous preclinical studies have shown that miR-132 is essential for driving the pathological growth of cardiomyocytes.

In a randomized, double-blind, placebo-controlled, dose-escalating Phase Ib study, the compound showed excellent safety and tolerability as well as promising pharmacokinetic (PK) and pharmacodynamic (PD) properties in patients with stable heart failure (HF) of ischemic origin (NYHA 1-3). The study design combined dose escalation with repeat dosing (day 1 and 28) at 4 dose levels. 28 patients received CDR132L or placebo (5:2 randomized in 4 cohorts) via short-term (15 min.) intravenous infusions as add-on therapy to standard of care.

About Cardior Pharmaceuticals

Cardior Pharmaceuticals is a privately held German biopharmaceutical company pioneering the development of curative and preventive heart failure therapeutics based on non-coding RNAs (ncRNAs). Cardior’s therapeutic approach is using distinctive ncRNA signatures driving the molecular reprogramming that causes maladaptive remodeling and heart failure. Drug candidates developed by Cardior represent first-in-class ncRNA therapeutics and diagnostics for patients with myocardial infarction and various forms of heart failure. Founded in 2016 based on the work of cardiologist Prof. Dr. Dr. Thomas Thum of Hannover Medical School, the Company has raised EUR 15 Mio. from international investors LSP, BioMedPartners, Boehringer Ingelheim Venture Fund (BIVF), Bristol-Myers Squibb (BMS) and High-Tech Gründerfonds (HTGF).

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Tel: +49 511 33 85 99 30

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