New partnership for clinical development of therapeutics for retinitis pigmentosa

Lions Eye Institute (LEI) and PYC Therapeutics have entered into a commercial collaboration agreement 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 Pty Ltd.

The Vision Pharma lead program combines PYC’s best-in-class delivery technology with LEIs novel RNA therapies 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.

This development will enable a potential drug to be delivered from discovery right through to the clinic, all driven from Perth, WA. “We look forward to the clinical development of therapeutics for Retinitis Pigmentosa and other degenerative retinal diseases” commented Professor Sue Fletcher, Chief of Research and Development at PYC Therapeutics and global leader in RNA Therapeutics.

Through this collaboration Vision Pharma’s lead drug candidate has already shown preliminary evidence that it can reverse the effects of Retinitis Pigmentosa in human cells. Figure 1 (attached) 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. The treatment significantly increases the expression of these red cilia by the diseased patient cells, a key functional readout for drugs intending to rescue our target form of Retinitis Pigmentosa. The photographs attached clearly show 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.

On the potential of this exciting collaboration, LEI Managing Director Bill Morgan said “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. Prof 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.”

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