Perth-based biotech company Phylogica (ASX: PYC) has reported positive early stage results from a pilot study of human cells using its proprietary drug delivery technology to create a treatment for blinding genetic eye diseases.
The study delivered an anti-sense oligonucleotide (ASO) drug cargo into human retinal pigment epithelial cells using Phylogica’s cell penetrating peptide (CPP) platform, as part of the company’s flagship program focused on “undruggable genomes” or high-value drug targets which exist inside cells.
It compared the exon skipping rate in human cells achieved by an ASO with and without the aid of CPPs, with results showing the ASO on its own achieved 8% exon skipping while the CPP-ASO combination achieved 100%.
Exon skipping is a measure of efficacy in drug administration.
Higher levels of exon skipping indicate more effective delivery of an ASO by a CPP.
Phylogica’s study is a collaboration between Murdoch University’s Centre for Molecular Medicine and Innovative Therapeutics and the Ocular Tissue Engineering Laboratory at Lions Eye Institute.
The human cell achievement follows a similar milestone reached by Phylogica in June during an animal study using CPP and ASO drug cargoes.
In that study, second-generation CPPs were used to deliver an ASO drug cargo into the vitreous of mice, showing that the delivery method was viable across eight different layers of cells and the drug could be successfully delivered into targeted retinal pigment epithelium cells.
At the time, Phylogica said the animal study represented “achievement of the major pre-clinical milestone” on the drug development pathway and clearly demonstrated the outperformance of the delivery technology.
Both studies have become “critical pieces of data” which inform the prospect of successful translation of pre-clinical results into clinical (human) outcomes and a potential marketed drug.
Phylogica said it would now focus on the delivery of a final pre-clinical technical milestone known as 3D organoid modelling (or ‘retina in a dish’), which combines the structural complexity of a living organism represented in the animal models with the unique elements of human cellular material.
“The benefits of successful studies in 3D organoids are an increased probability of success in the subsequent clinical (human) evaluation of a drug molecule,” the company said.
Inherited retinal diseases are a group of conditions caused by known genetic mistakes in more than 200 human genes, causing loss of vision in million of adults and children worldwide.
Retinitis pigmentosa, macular dystrophy and macular degeneration are among the more common degenerative disorders affecting people of all ages, with different conditions progressing at different rates.
The delivery of large drugs into the cells of inherited retinal diseases is one of the major challenges currently facing the pharmaceutical industry.
Many large drugs on the market are unable to effectively penetrate cells to reach the highest-value drug targets on their own.
Phylogica’s CPP delivery platform has been designed to address this limitation and provide drugs with access to the inside of cells.
The technology can be used to deliver a wide range of drugs to multiple target tissues within the human body, providing opportunities to treat many different diseases.
At mid-afternoon, shares in Phylogica were trading 20% higher at $0.042.