Biotechnology company Phylogica (ASX: PYC) has unveiled favourable lab results in relation to its time-course studies in animals, with superior performance to current benchmarks officially confirmed.
The most recent animal model results also complement the single time point results published last month.
The company announced that today’s results differentiated its platform on both efficacy and toxicity with a “significantly improved safety profile” compared to current alternatives.
As part of a study designed to assess the effectiveness of its Cell Penetrating Peptide (CPP) platform to deliver drugs into the eye, Phylogica said that recently completed animal studies demonstrated that one of Phylogica’s CPPs delivers “substantially more drug cargo inside its target cell than the current CPP benchmark” for clinical development.
The biotech company declared “this result is significant given that the rate-limiting step for the development of delivery technologies is the amount of cargo that can be safely delivered inside a cell,” according to a company statement.
Furthermore, Phylogica’s second-generation CPPs were 400% more effective than the gold standard CPP at achieving exon skipping – the desired effect of a drug cargo – within the retina in animal studies at day seven post-administration.
In addition, the magnitude of outperformance of Phylogica’s CPP “is expanding the longer the experiments continue”.
Eyeing up solid results
The results published this morning were achieved as part of Phylogica’s “flagship program” directed towards delivering a promising class of drug cargo known as an Anti Sense Oligonucleotide (ASO) into the retina of mice.
ASOs are short, synthetic, single-stranded oligodeoxynucleotides that can alter RNA and reduce, restore, or modify protein expression through several distinct mechanisms.
Their potential influence on treating neurological disorders was described as “the next frontier” by scientists amidst a growing number of ASO-mediated therapies obtaining FDA approval in the US, with the aim of treating muscular dystrophy and spinal muscular atrophy.
The innovation was first discovered to influence RNA processing and modulate protein expression over two decades ago but developmental progress has been slow due to “inadequate target engagement, insufficient biological activity, and off-target toxic effects”, according to research published in Nature Reviews Neurology.
For Phylogica, its focus is to commercialise its intracellular drug delivery platform and screening its peptide libraries to identify drug cargoes for development against a wide range of disease targets.
The company’s platform of proprietary cell-penetrating peptides has been validated across multiple animal models for the ability to deliver a diverse range of drug cargoes into cells.
So far, developmental progress has been robust and multilateral with Phylogica setting up varied collaborations with several pharmaceutical companies including Roche, Medimmune, Pfizer, Janssen and Genentech.
One of the more intriguing pathways in which to develop and commercialise its platform has been animal models and drug delivery into the eye.
The retina is a tissue at the back of the eye which can be affected by a variety of blinding diseases with Phylogica electing to prioritise treating inherited retinal diseases for the clinical development of its platform.
In a bid to discuss its time-course results and their relevance to the future direction of the company, Phylogica announced that it would be hosting a special event for shareholders at the Harry Perkins Institute in Perth tomorrow.
This news helped Phylogica shares to rise almost 4% in morning trade to $0.028.