Secarna Pharmaceuticals announces publication of new preclinical data showing strong potential of LNAplus^TM-based ASOs to effectively treat diabetic kidney disease

• Compelling in vivo and in vitro data published in peer-reviewed Journal of the American Society of Nephrology
• Secarna's LNAplus^TM-based ASOs effectively and specifically target cell death-promoting transcription factor C/EBP homologous protein (CHOP), a mediator of diabetes-induced stress conditions in the kidneys
• ASO approach holds potential to be exciting new therapeutic modality in halting progression of diabetic kidney disease
• Pre-clinical testing and planning of clinical trial program will follow next year

Munich/Martinsried, Germany, October 05, 2021 - Secarna Pharmaceuticals GmbH & Co. KG ("Secarna"), a biopharmaceutical company focusing on the discovery and development of next-generation antisense oligonucleotide (ASO) therapies to address challenging or previously undruggable targets via its LNAplus^TM platform, today announced the publication of new preclinical data supporting the use of ASOs in the treatment of diabetic kidney disease. The data were published in the peer-reviewed publication, Journal of the American Society of Nephrology. The article can be found , a summary of the publication can be found .

Researchers selected the cell death-promoting transcription factor C/EBP homologous protein (CHOP) for a target, as it is known to be a key regulator of the endoplasmic reticulum (ER)-stress response. When chronically activated, the ER-stress response can be linked to a variety of diseases, including diabetic kidney disease (DKD). The studies discussed in the publication evaluated the use of ASOs targeting CHOP as a potential new approach to treating DKD.

An ASO targeting CHOP was evaluated both as a monotherapy and in combination with an ACE inhibitor (ACEi), the current standard of care, in both early- and late-stage disease. In a diabetic in vivo model, CHOP-ASOs were shown to reduce renal CHOP expression and reduce markers of DKD at both early and late stages of disease. Adding a CHOP-ASO provided additional benefit compared to treatment with an ACEi alone, particularly at later stages of the disease. This was an important finding as previously published data suggest that use of an ACEi at later timepoints is less effective. CHOP-ASO was also shown to regulate SGLT2 expression, another important target in DKD

In cell culture experiments, glucose-stressed human kidney cells were exposed to CHOP-ASO. The ASO, similar to what was observed in the in vivo testing, suppressed CHOP and thereby protected human renal cells from glucose-induced apoptosis.

Frank Jaschinski, Ph.D., Chief Scientific Officer of Secarna Pharmaceuticals, said: "The promising pre-clinical results seen with our CHOP-ASOs support that inhibiting CHOP may be a new way to effectively treat diabetic kidney disease. We were particularly excited to see that CHOP-ASO treatment has the potential to improve the standard of care in this challenging disease. The data further indicate the potential of CHOP-ASOs for treatment of other diseases driven by maladaptive ER-stress response and show the ability of our LNAplus^TM platform to generate potent ASOs having a favorable safety profile against a variety of diseases."

Alexander Gebauer, MD, Ph.D., CEO of Secarna Pharnaceuticals, said: "Despite advances in treatment, today there still is no therapy that halts or reverses the progression of diabetic kidney disease. The development of a disease-modifying antisense oligonucleotide that targets the cell death-promoting transcription factor C/EBP homologous protein, could be an exciting new approach to treating this painful and ultimately life-threatening disease. I very much look forward to seeing the CHOP-ASOs advance in development."

Secarna expects to conduct additional pre-clinical testing and then to start planning the clinical trial program for the CHOP-ASOs next year.

About Secarna's proprietary drug discovery platform, LNAplus^TM

Secarna's proprietary, customized LNAplus^TM drug discovery platform is being applied to the discovery, testing and selection of antisense oligonucleotides (ASOs) for pre-clinical and clinical development. LNAplus^TM encompasses all aspects of drug discovery and pre-clinical development and has proven to be fast, reliable, scalable and efficient, enabling the discovery of novel antisense-based therapies for challenging or currently undruggable targets.^{The platform includes the powerful proprietary OligofyerTM bioinformatics pipeline, a streamlined, high efficiency screening process, including Secarna's proprietary LNA-Vit(r)oxTM safety test system as well as target-specific functional assays. Secarna's platform and ASOs have been validated by numerous in-house projects as well as in several academic and industry collaborations.}

About Secarna Pharmaceuticals GmbH & Co. KG

Secarna Pharmaceuticals is the leading independent European next generation antisense drug discovery and development company addressing high unmet medical needs in immuno-oncology, immunology, as well as viral, neurodegenerative and cardiometabolic diseases. Secarna's mission is to maximize the performance and output of its proprietary LNAplus^TM antisense oligonucleotide discovery platform, as well as to develop highly specific, safe, and efficacious best-in-class antisense therapies. With over 15 development programs, including both proprietary pipeline projects and partnered programs, Secarna focuses on targets in indications where antisense-based approaches have clear potential benefits over other therapeutic modalities.

Contact

Secarna Pharmaceuticals GmbH & Co. KG

Alexander Gebauer, MD, PhD
CEO

Secarna Pharmaceuticals GmbH & Co. KG
Am Klopferspitz 19
82152 Planegg/Martinsried
Tel.: +49 (0)89 215 46 375

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