Listed clinical-stage biotech PYC Therapeutics has received major backing from the US Food and Drug Administration for its drug treating a progressive and irreversible blinding eye disease.
The Rohan Hockings-led company told the market on Friday it had received a so-called orphan drug designation for its first-in-class drug candidate — known as PYC-001 — designed to address the root cause of autosomal dominant optic atrophy (ADOA).
According the company, ADOA affects about one in every 35,000 people and represents a market size of about $2 billion a year.
PYC has seen its share price surge 140 per cent in the past year to 12¢, valuing the company nearly $600 million, but Mr Hockings is not yet satisfied.
“In the first instance, it’s disappointing because if you look at our peers in the US, companies at the same stage of development have a market cap about 10 times ours,” he told The West Australian.
“But . . . it’s a reflection of a fact that we’re making some serious progress here and we’re now in the window where the rubber meets the road.
“People are just starting to get the sense there’s a company from Perth, WA that looks like it is not just competing but, differentiating itself on the global stage.”
Benefits of the FDA designation includes tax credits, grants, waived clinical trial fees, as well as seven years of market exclusivity upon drug approval.
“It’s a really nice piece of additional weight behind the program because the FDA look quite carefully at the merits of the drug candidate for the need,” Mr Hockings said.
“But it’s not a huge surprise in the sense that we knew it was a rare disease population and we were always intending on applying for and receving the designation.”
PYC is set to commence clinical trials for PYC-001 in the third quarter of this year.
Additionally, PYC is also conducting a clinical trial in retinitis pigmentosa (RP11) — another degenerative eye disease which begins in childhood and leads to legal blindness by middle age — using its drug VP-001, which received a fast track status from the FDA last August.
The company is also aiming to develop a drug aimed at treating polycystic kidney disease, with a clinical trial scheduled in early 2025.
Mr Hockings reaffirmed its plans to file a new drug application to the FDA in 2027, with another two applications the following year.
Applications to the Therapeutic Goods Administration will follow shortly after.