Partnership to develop drugs for eye disease

Drug development company PYC Therapeutics has entered into a commercial collaboration with Lions Eye Institute (LEI), a not-for-profit organisation, to develop drugs for a leading cause of blinding eye disease. The commercial partnership will take the form of a special purpose vehicle, Vision Pharma, in which LEI and PYC have equity interests based on their contributions of IP and funding.

The Vision Pharma lead program combines PYC’s delivery technology with LEI’s novel RNA therapies, both of which originated in Perth, to create a competitively differentiated drug to treat a leading cause of childhood blindness: retinitis pigmentosa. Vision Pharma intends to commence investigational new drug (IND)-enabling studies for this lead program in the first half of 2020; successful IND-enabling studies will culminate in approval for testing in humans.

“The formation of Vision Pharma in WA is the culmination of combining decades of research into splice therapy (led by Sue Fletcher, co-inventor), cell-penetrating peptides (through PYC), data from the Australian Inherited Retinal Diseases Registry (established in the 1980s) and more recently developed local expertise in stem cell retinal disease modelling (LEI),” said Dr Fred K Chen, co-inventor of the lead drug and Head of LEI’s Ocular Tissue Engineering Laboratory.

The development will enable a potential drug to be delivered from discovery right through to the clinic, all driven from Perth. Indeed, Vision Pharma’s lead drug candidate has already shown preliminary evidence that it can reverse the effects of retinitis pigmentosa in human cells.

The below image illustrates the effectiveness of combining PYC’s delivery technology (a cell-penetrating peptide, or CPP) with Vision Pharma’s molecule (an antisense oligonucleotide, or ASO). Healthy retinal cells express the red cilia, whilst diseased cells do not.

For a larger image, click here.

The treatment significantly increases the expression of red cilia by the diseased patient cells, a key functional readout for drugs intending to rescue the target form of retinitis pigmentosa. The image shows that joining Vision Pharma’s ASO to PYC’s CPP increases both the amount of cells expressing cilia and the length of these cilia above the naked ASO.

“LEI is proud to see one of its research developments now entering preclinical trials to treat the commonest form of retinitis pigmentosa, a previously untreatable blinding condition,” said LEI Managing Director Bill Morgan. “Fred Chen and Prof Sue Fletcher have been working on their genetic therapy and are partnering with PYC, using their transporter protein to deliver this novel therapy directly to the sick retinal cells. We are very excited about the possibility of being able to cure this form of blindness.”

PYC Chairman Alan Tribe added, “I am excited by the prospects for this new partnership. It brings together the PYC drug delivery system and an elegant drug to treat the leading cause of childhood blindness. Importantly it also brings together world-class expertise in drug design, delivery and development, as well as the treatment of eye diseases. The team that has been created will be capable of achieving much.”

Top image credit: ©stock.adobe.com/au/Yuganov Konstantin

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