A drug delivery platform developed by biotechnology company Phylogica (ASX: PYC) has been shown to be the most effective method of administering anti-sense oligonucleotide (ASO) medications to patients with genetic eye disease.
A competitive evaluation test on animal models using Phylogica’s proprietary cell penetrating platform demonstrated the technology could significantly increase the effectiveness of an administered drug.
The ASO used in the test was found to be effective at a dose of less than 1 microgram per eye when used with the delivery platform, compared to a “naked” dosage (administered without the platform) which was not effective even at a boosted dose of 10 micrograms per eye.
The results showed the platform can turn ASOs into potent drugs at doses which are otherwise ineffective – meaning better drugs with fewer side effects.
“This result from an animal model proves that drugs incorporating [our] delivery technology are effective at substantially lower doses,” the company said.
“This is a major advantage for correcting diseases and avoiding toxic side-effects and an encouraging indicator of clinical success.”
Phylogica said the evaluation test demonstrates the benefit of its delivery technology for the rapidly-growing ASO class of drugs.
ASO drug Spinraza – adminstered by injection to treat spinal muscular atrophy – has already established a A$2.7 billion global market.
Developer Novartis received FDA approval for the drug – dubbed the world’s most expensive – earlier this year.
Delivering more drug to target cells in a non-toxic manner is the rate-limiting step in RNA therapies – an emerging field in which ASOs are a leading class of drug across a range of diseases.
Unlike gene therapy – which provides new DNA to patient cells – RNA therapy modifies or provides ribonucleic acid (RNA) to the same cells.
The therapy has the potential to treat a wide variety of illnesses including cardiovascular disease, hemophilia and cancer.
Phylogica said data from the evaluation tests would provide the “central piece of pre-clinical evidence” that its delivery platform is capable of achieving outcomes which competitive technologies cannot achieve alone.
The increase in effectiveness observed in the test models “complements the decreased toxicity” associated with the type of ASO the company has prioritised for clinical development known as morpholino.
“The improved effectiveness of the drug that incorporates [our] delivery technology along with the decreased toxicity of these molecules gives our approach to RNA therapeutics a dual advantage in the critical ‘therapeutic index’ of a drug (the ability to effectively treat a patient without causing toxic side effects),” Phylogica said.
At midday, shares in Phylogica were up 2.33% to $0.044.