바이오스펙테이터

by Sungmin Kim

Signed an ASO drug discovery deal for rare diseases and immuno-oncology targets, respectively

Vanda Pharmaceuticals and OliPass Corporation announced on the 29th that they have entered into a research and development collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on OliPass' proprietary modified peptide nucleic acids.

The collaboration will focus on editing and modifying gene expression using ASOs in disease states where the expression of genes is either altered or the sequence of the expressed genes can be altered for therapeutic benefit. OliPass' unique Olipass Peptide Nucleic Acids (OPNA) technology provides the delivery platform to enable these gene expression modifications.

OliPass will receive a down payment of $3 million from Vanda, and will receive research funding for joint research and development. If the two companies achieve the agreed short-term R&D milestones, Vanda will pay Olipass an option exercise technology fee. After that, a separate license agreement for the new drug target is in progress. Details were not disclosed.

Vanda has already identified two ASO targets that have been validated in cell lines that model two undisclosed disease targets, one rare orphan and the other applicable to a broad set of immuno-oncological conditions. Vanda's partnership with OliPass to enhance the existing ASOs with OliPass' unique OPNA chemistry is the next step to take these preclinical findings to in vivo and clinical testing.

OPNAs are selectively modified with cationic moieties that enhance both stability and cell permeability of peptide nucleic acids (PNA) while maintaining very high binding affinities to target nucleic acids. Therapeutic efficacy for OPNAs has been observed with dosages as low as 10 ng/kg in animal models, showing effects at dosages many orders of magnitude lower than previously achievable with existing ASO technologies. Furthermore, OPNAs have the capacity to bind to pre-mRNA in nucleus and enable targeting pathogenic variants and conditions that were previously untreatable.

"We are excited to have a partner to enhance our antisense oligonucleotide program platform aimed at the treatment of disorders with well-understood genetic mechanisms and for which there is a high unmet medical need," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board. "Olipass' delivery platform holds the promise of efficient delivery of our ASO's in pursuit of the development of precision therapeutics."

"Vanda shares a vision with us on the potential for anti-sense oligonucleotides to open up a broad range of therapeutic options to patients," said Dr. Shin Chung, CEO of OliPass. "We look forward to working together to enhance the therapeutic profile of these molecules and playing an integral role in bringing them into the clinical setting."

OliPass is pursuing a two-track business model strategy of jointly researching RNA therapeutic candidates for specific gene 'target X' through partnerships while confirming clinical efficacy by developing new drugs on its own.