Secarna Pharmaceuticals GmbH & Co. KG (“Secarna”), a biopharmaceutical company focusing on the discovery and development of next-generation antisense oligonucleotides (ASO) therapies to address challenging or previously undruggable targets, today announced that Denali Therapeutics Inc. (“Denali”) has exercised its option to in-license an ASO program generated by Secarna through its proprietary drug discovery and development platform, LNAplus^TM. This program is part of the Research Collaboration and Option Agreement between the two companies and is the first project for which Denali has exercised an option.

The agreement was signed in October 2020 and expanded in October 2021 to include multiple additional discovery and research programs across a range of undisclosed targets and diseases of the CNS. Under the collaboration, Secarna is employing its LNAplus^TM platform to generate ASO candidates against targets relevant for the treatment of neurodegenerative diseases. Denali is then applying its OTV technology to facilitate delivery of these ASOs across the blood-brain barrier (BBB).

“We are delighted that Denali has exercised the first option to take one of our ASO programs forward in development,” said Alexander Gebauer, M.D., Ph.D., CEO of Secarna Pharmaceuticals. “Our collaboration has made great progress in just 16 months, and we have been able to generate high-quality ASO drug candidates with our LNAplus^TM platform. Secarna’s ASOs combined with Denali’s BBB transport delivery technology offer the potential to provide truly novel therapies to people living with a wide range of difficult-to-treat or even currently untreatable neurodegenerative diseases. We look forward to continuing our fruitful collaboration with Denali as we work together to advance all the other projects towards similar decision points.”

With the exercise of this option, Secarna is receiving an option exercise fee and will be eligible to receive development, regulatory and commercial milestone payments as well as tiered royalties.  Denali has secured exclusive rights for the further development and commercialization of this program. Financial details were not disclosed.

In January 2022, Denali announced the first nonhuman primate data with the OTV delivery platform. The data demonstrated that intravenous administration of an ASO enabled by OTV technology elicited superior, broad brain biodistribution of the ASO (particularly in deep brain regions) and target gene knockdown that was superior to intrathecal administration of the same ASO in a larger species. Further, intravenously delivered OTV in human TfR expressing mice provided robust target knockdown of gene expression across all brain cell types, including neurons, microglia, astrocytes, and oligodendrocytes. These data support the potential of the OTV platform to enable superior biodistribution of ASOs across brain regions, provide superior knockdown of target gene expression across all CNS cell types, and enable peripheral dosing, which would be a less invasive option compared to direct injections into the brain or cerebral spinal fluid.

Secarna’s proprietary drug discovery and development platform, LNAplus™
Secarna’s proprietary, customized LNAplus^TM 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 Oligofyer^TM bioinformatics pipeline, a streamlined, high efficiency screening process, including Secarna’s proprietary LNA-Vit(r)ox^TM 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.

Secarna Pharmaceuticals works in the field of immuno-oncology, immunology as well as viral, neurodegenerative and cardiometabolic diseases.
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 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. www.secarna.com

Denali Therapeutics Inc. explores new therapies for neurodegenerative diseases
Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the BBB for neurodegenerative diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the BBB and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San Francisco (www.denalitherapeutics.com).