Interest in antisense oligonucleotides (ASO) therapeutics has...

Interest in antisense oligonucleotides (ASO) therapeutics has been on the rise in 2018.

In April, Biogen struck a $1 bn deal with leading ASO company, Ionis, to license additional assets from Ionis’ ASO pipeline. Biogen has already had major success with commercialization of Ionis’ lead ASO product, Spinraza. The identity of the assets licensed by Biogen wasn’t revealed, other than that the therapy areas involved were broad - dementia, neuromuscular diseases, movement disorders, ophthalmology, diseases of the inner ear, and neuropsychiatry. Biogen said the deal could potentially validate about 50 targets across a wide range of diseases.

Roche has also been active in the ASO field during 2018. In July the company struck a potential $1bn + deal with Pure Tech Health to develop oral antisense oligonucleotide therapies. In September it was announced that Roche would soon commence a large pivotal trial of its ASO drug, RG6042, in Huntington’s disease, following success in a 46 patient trial of the drug which was led by Ionis. Then in October, Roche signed a new collaboration with Ionis, worth up to $760 m, for the option to license another Ionis ASO drug for dry age-related macular degeneration (AMD) on completion of a Phase 2 trial of the drug.

Also in October, FDA approval was granted to another ASO drug, Tegsedi. The Akcea Therapeutics/Ionis drug is indicated for a rare disease polyneuropathy.

In an article published just last month, titled Have antisense oligonucleotides hit their stride? , it was noted that “the oligonucleotide field is full of fresh momentum, and cash.” A major driver of interest is said to be the potential speed of ASO development compared with other classes of drugs. As stated in the article, “seemingly everyone in the field agrees the potential for rapid drug development is what makes the technology so powerful.”

As Phylogica moves forward into the ASO space with its flagship program offering targeted, intracellular delivery of ASOs, it will need compelling data to make its cell penetrating delivery technology stand out from the competition. In July this year it was announced that Ionis had entered a research collaboration with SRI International (formerly Stanford Research Institute) to combine SRI's proprietary FOX Three Molecular Guidance cellular targeting system (MGS) with Ionis' antisense drug-discovery platform. It is said that SRI has already demonstrated that its “unique protein domain” technology can deliver more than a dozen different types of payloads - including enzymes, antibodies, DNA, liposomes and nanoparticles - to targeted, intracellular molecular-target locations. SRI Biosciences currently has a library of more than 30 characterized MGSs targeted to a range of cell types including multiple solid tumour targets, lymphoma, dendritic cells, and cardiomyocytes.