Antisense oligonucleotide candidate derived from Secarna Pharmaceuticals' LNAplus^TM platform has entered pre-clinical safety trials for the treatment of elevated blood lipids

• Secarna's partner Lipigon has selected an LNAplus^TM ASO candidate targeting ANGPTL4, called Lipisense, which has shown unique efficacy in reducing lipid triglycerides in experimental disease models and is now entering pre-clinical safety development
• High levels of plasma lipids triglycerides are associated with a plethora of lipid-related diseases with yet unmet clinical treatment needs

Munich/Martinsried, Germany, January 19, 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 that the company's partner Lipigon Pharmaceuticals AB has selected a candidate drug (Lipisense) to treat elevated levels of plasma lipid triglycerides. The candidate is being developed for patients suffering from severe hypertriglyceridemia as well as the rare disease Familial Chylomicronemia Syndrome (FCS), diseases that may lead to painful and potentially life-threatening syndrome acute pancreatitis.

Lipisense, an antisense oligonucleotide (ASO) which has been developed with Secarna's proprietary LNAplus^TM platform, is a first-in-class treatment with a novel mechanism of action central for the regulation of plasma triglycerides and other plasma lipids. The drug candidate hinders the cellular production of ANGPTL4, a protein that has a direct role in lipid metabolism by blocking the clearance of triglycerides. The protein has been associated with tumour progression and has shown a strong genetical validation and correlation with harmful plasma lipids and the associated diseases. By hindering the production of ANGPTL4 in the liver by specifically targeting its protein-coding RNA, Lipisense has great potential for the treatment of lipid-related diseases.

The LNAplus^TM ASO-based drug candidate has shown strong efficacy in experimental disease models and a good safety profile in preliminary safety studies. Lipigon will now continue preclinical development with IND enabling studies; CMC activities have commenced.

Jonas Renz, Managing Director and Co-founder of Secarna Pharmaceuticals, said: "We are excited for our partners at Lipigon to have identified an ASO-based candidate drug derived from our proprietary LNAplus^TM plattform as a first-in-class drug for the treatment of lipid-related diseases. We congratulate the team on entering into pre-clinical safety trials, as together we aim to provide a new line of treatment for patients with severe life-threatening diseases."

Stefan K. Nilsson, CEO and Co-founder of Lipigon, said: "Choosing a candidate drug in our front runner project Lipisense is a major step for Lipigon. The unique mechanism of action opens the possibility for an attractive clinical development path where we can address both smaller indications, which do not require extensive clinical studies, and larger indications, which are more suitable for big pharma development. We are looking forward to continuing the preclinical safety development of Lipisense."

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

For discovering, testing and selecting antisense oligonucleotides (ASOs) for pre-clinical and clinical development, Secarna employs its proprietary, customized LNAplus^TM drug discovery platform. LNAplus^TM encompasses all aspects of drug discovery and pre-clinical development and has proven to be fast, reliable, scalable, efficient and to provide for a uniquely integrated workflow, enabling the discovery of novel antisense-based therapies for challenging or currently undruggable targets.^{The platform includes the powerful proprietary OligofyerTM bioinformatics and LNA Vit(r)oxTM screening system for best-in-class high volume screening, and 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 next generation antisense oligonucleotide (ASO) company addressing high unmet medical needs in the areas of immuno-oncology, immunology, as well as viral, neurodegenerative and cardiometabolic diseases. Secarna's mission is to maximize the performance and output of its proprietary ^{antisense oligonucleotide discovery platform, as well as to develop highly specific, safe, and efficacious best-in-class antisense therapies for challenging or currently undruggable targets. With 13 in-house development programs and 10 partnered programs focusing on targets in indications where antisense-based approaches have clear benefits over other therapeutic modalities, Secarna is the leading European antisense drug discovery and development company. .}

About Lipigon Pharmaceuticals

Lipigon are lipid biology experts focusing on developing novel therapeutics for patients with lipid- related disorders. The company is a spin-off from Umeå University, Sweden, based on five decades of lipid research. Our primary focus is orphan lipid disorders and in addition to the ANGPTL antisense program, Lipigon's pipeline, including a project for gene therapy treatment of lipodystrophy. This program is partnered with Combigene AB (publ). .

Contact

Jonas Renz
Managing Director and Co-founder

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

For media enquiries:

Anne Hennecke/Vera Lang
MC Services AG

Tel.: +49 (0)211.52 92 52 22