Biotechnology company Phylogica (ASX: PYC) is one step closer to finding a treatment for the leading cause of childhood blindness, achieving a major milestone in its human ‘retina in a dish’ study.
The company today reported results from its flagship drug program, in which its proprietary drug delivery technology was used on a 3D model of a retina created from human stem cells.
According to Phylogica, the study achieved greater than 90% effectiveness after a single dose.
Phylogica chief executive officer Dr Rohan Hockings said the success of the study is an important milestone for the company as it materially increases the probability that its flagship drug program will prove effective in human studies.
The results also complement Phylogica’s earlier outcomes in animal models and functional studies in human cells with the targeted disease, retinitis pigmentosa.
Treating childhood blindness
Retinitis pigmentosa is a genetic degenerative eye disease that is considered to be the leading cause of childhood blindness.
It affects between 4,000-8,000 people in the western world and is estimated to be a $1 billion per annum market. There are currently no treatment options for the disease.
“The company is very excited by the result of this study given its implications for our objective of taking a treatment to market,” Dr Hockings said.
In summary, Phylogica’s drug program for treating retinitis pigmentosa has now shown highly effective delivery in animal models with sustained duration of effect; the ability to reverse the disease process in human cells; highly effective outcomes in complex models of the human eye; and a favourable proof of concept toxicity profile.
Phylogica’s next aim is to seek validation of its latest results across multiple patients with different genetic mutations in the same ‘retina in a dish’ models.
This will allow the company to begin investigational new drug enabling studies, including large animal toxicology studies, before progressing to human clinical trials.
By afternoon trade, Phylogica shares were sitting 8.51% higher at $0.051.