Genetic eye problems affect over two million people worldwide and often lead to blindness among the working-age population. Though there’s a lot of optimistic research into potential treatments, none exists right now. That’s what the Western Australian biotech company PYC Therapeutics (ASX: PYC) is attempting to change. 

After successfully treating rabbits and monkeys suffering from retinal diseases through its investigational drug VP-0013, PYC has received FDA’s blessing to move on to the next stage: human trials. 

Elated, PYC’s CEO, Dr Rohan Hockings, shared, “This is very exciting for the RP11 patient community and an outstanding achievement by the PYC team. The validation of PYC’s platform technology as we move ahead into human trials has implications for the Company’s entire pipeline of first-in-class and potentially disease-modifying drugs. The potential for patient-impact now extends well beyond RP11.”

Affecting 1 in 100,000 people, Retinitis Pigmentosa type 11 (RP11) is a genetic disorder that affects the eyes, specifically the retina, which is part of the eye that senses light and helps us see. It results from mutations in a gene called PRPF31, important for the production of a protein that looks after the retina.

People with RP11 typically experience progressive vision loss, starting with difficulty seeing in low light and eventually leading to tunnel vision and potentially complete blindness. It typically starts when you’re a child and then worsens as you age. 

Even though there’s an addressable market of over a billion, the Company reports that there are no available treatments for RP11.

But now that the US Food and Drug Administration (FDA) has approved PYC’s Investigational New Drug (IND) application for its RP11 drug candidate, things might look different.

PYC Therapeutics determinedly aims to create solutions for patients with genetic disorders through RNA therapies. In the case of VP-001, the drug utilises precision therapy and works by delivering a gene called PRPF31 to the cells in the eye that need it. This medicine is designed to make sure that enough of the medicine gets to the right place in the cells, which can be tricky with this type of medicine.

Besides PYC’s precision therapy, institutions are also studying stem cell therapy to come up with potential treatments for RP11.

In H1 FY23, PYC reported a revenue fall of 43% to $3.4 million. Its losses increased over 150% to $16.2 million, with money spent on R&D increasing twofold. If all goes well—with the FDA approval and human trials—the Company could consider this money spent well.

Human studies will commence with a single ascending dose protocol treating an estimated 10 to 15 RP11 patients. It aims to establish a safe and well-tolerated dose—as it did with the animals—to progress into multi-dose trials next year.

The Company plans to begin dosing RP11 patients with this drug candidate in 2Q 2023. But before it begins, PYC has applied for human ethics approval to be able to enrol patients in the clinical trial.