Perth biotech company PYC Therapeutics has hit another major milestone, gaining approval to move its first-in-class PYC-001 drug to human trials.
The Nedlands-based company today told the market it had received approval for PYC-001, an intravitreal injection developed for patients with a blinding eye disease called autosomal dominant optic atrophy.
The approval means the company will now move to a single ascending dose study, which aims to find the safe dose range and look for any side effects.
SAD studies involve a very small initial dose, gradually increasing to test for side effects.
The Rohan Hocking-led company said trials would take place in Australia at two unspecified sites.
The development comes after the company in May received FDA orphan drug designation for PYC-001, a designation given to drugs designed to treat rare diseases which come with a host of benefits.
Those benefits include tax credits for qualified clinical trials, exemptions from some regulatory fees, and the potential for seven years of market exclusivity post-approval.
The disease it aims to treat, ADOA, is a progressive and irreversible blinding eye disease affecting approximately one in every 35,000 people.
The hereditary disease is caused by a mutation in the OPA1 gene and leads to cell stress and death in the retina.
PYC-001 aims to restore the OPA1 gene back to levels required for the normal function of the retina.
There is currently no treatment for ADOA, however PYC-001 has shown promising results in the lab, and in non-human primate trials.
The company in March tapped investors for $75 million to progress trials of PYC-001, among three other drug candidates.
PYC was also recently named a newcoming to Business News’ BN30.
In January, PYC announced a collaboration with Google to develop and host an artificial intelligence platform for precision medicines.
Under the project, PYC’s proprietary precision medicine data sets would be used and designed to predict the delivery of precision medicine to specific cells of the human body.
Mr Hocking was a 2022 40under40 award winner in the medium-sized business category, recognising the work he’d done to build the company.
