Secarna Pharmaceuticals presents data at OTS showcasing unique characteristics and factors with a role in the activity and side effect profile of LNA-modified ASOs – Biotech Investments

Secarna Pharmaceuticals presents data at OTS showcasing unique characteristics and factors with a role in the activity and side effect profile of LNA-modified ASOs

• Comprehensive data sets presented in two poster presentations at the 19^th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS)
• Secarna investigated the immunostimulatory potential of LNA-modified antisense oligonucleotides (ASOs), a potential factor of undesired side effects of treatment, and factors that lead to the different knockdown profiles of ASOs

Munich/Martinsried, Germany, October 25, 2023 – 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 platform, has presented data showing the potential as well as the versatility of Secarna’s LNA-modified ASOs. The data support the ongoing optimization of Secarna’s proprietary Oligofyer™ bioinformatics system, an in-silico program shown to result in a high yield of potent and safe molecules. The two posters were presented this week, at the 19^th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) in Barcelona, Spain.

The poster 201: Characterization of immunostimulatory potential of LNA-modified antisense oligonucleotides was presented by Irene Riera Tur, PhD. Secarna has systematically investigated the immunostimulatory potential of ASOs using different human cell culture systems, as theTLR-9 immunostimulatory signaling, which has the physiological function to rapidly recognize infections and initiate an early immune response, is not comparable in mouse and human. It could be shown that, among others, LNA-modified ASOs actually can induce activation of the human TLR9 pathway and downstream signaling.

Hence, the prediction of immunostimulatory effects of LNA-modified as well as other types of ASOs, requires a multifaceted human in-vitro approach to de-risk highly potent and selective ASOs for further development.

Frank Jaschinski, PhD, Chief Scientific Officer of Secarna Pharmaceuticals commented: “Characterization of the immunostimulatory properties of oligonucleotides is a key step in successful development of oligonucleotide-based therapies. In particular cases it might be beneficial to induce immune activation, but in most cases, it is an undesired side-effect of treatment. It is therefore crucial to reliably detect such immunostimulatory ASOs early in the discovery process.”

The second poster 160: In silico and in vitro investigation of gene targetability and knockdown kinetics by LNA-modified antisense oligonucleotides, was presented by Sven Michel, PhD. These data showed that intrinsic target gene properties, such as gene length and expression level, influence the targetability of a gene and therefore ASO’s potencies. The existence of more than one binding site within the target sequence was shown to have a positive impact on ASO activity. In a second study, knockdown properties of individual ASOs specifically targeting IDO1 were investigated by their different knockdown kinetics and potencies. It could be shown that ASOs with high potency have a faster target knockdown. In addition, no evidence was found that individual uptake and transport kinetics to the molecular target are responsible for the differences in speed of knockdown.

“A comprehensive approach that analyses both multiple target gene characteristics as well as intrinsic ASO features is key for the development of safe and efficacious antisense therapies. These important findings on the therapeutic potential of LNA-ASOs support the continuous progress and innovation generated by our proprietary LNAplus platform”, concluded Frank Jaschinski, Chief Scientific Officer of Secarna Pharmaceuticals. “These data confirm our previous studies and convince us that we are on the right path to fully exploit the potential of ASOs. We are committed to expanding the knowledge in this field and to creating candidates with high activity and reduced side-effects, resulting in safe therapies for patients in need.”

About Secarna’s proprietary drug discovery and development platform, LNAplus
Secarna’s proprietary, customized LNAplus platform is being applied to the discovery, testing and selection of antisense oligonucleotides (ASOs) for pre-clinical and clinical development. LNAplus 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. This platform includes the powerful proprietary Oligofyer™ bioinformatics system, a streamlined, high efficiency screening process, including Secarna’s proprietary LNA-Vit(r)ox™ 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 and immunology, as well as viral, neurodegenerative and cardiometabolic diseases. Secarna’s mission is to maximize the performance and output of its proprietary LNAplus antisense oligonucleotide discovery platform, as well as to develop highly specific, safe, and efficacious best-in-class antisense therapies. With over 20 discovery and 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. www.secarna.com