RNA therapy company PYC Therapeutics (ASX:PYC) has updated investors, confirming that it has concentrated on advancing its portfolio of first-in-class drug candidates, each intended to address the root cause of monogenic disorders.
Across its four key programs, PYC reported steady progress in development and clinical preparation. The therapy for polycystic kidney disease completed dosing in early clinical testing among healthy volunteers and moved into the stage where the drug was administered to patients with autosomal dominant polycystic kidney disease. Safety results from the first part of the study were presented at a regional nephrology conference, reinforcing confidence in the program’s direction.
At the same time, the company’s program for Phelan-McDermid syndrome produced encouraging data from non-human primate studies that supported earlier findings from laboratory models derived from patient brain cells. The program is now advancing through toxicology work and remains on track for a regulatory submission that could allow first-in-human trials to begin in early 2027.
Progress was also evident in PYC’s ophthalmology pipeline. Its therapy targeting retinitis pigmentosa type 11 entered an extension phase of clinical testing after results showed sustained vision improvements compared with untreated patients. Preparations are underway for regulatory discussions with the US Food and Drug Administration to confirm the design of a registrational study that could ultimately support a future approval pathway.
Meanwhile, the company’s program for autosomal dominant optic atrophy completed dosing in its early patient cohorts and has already progressed to a repeat-dose study aimed at establishing clinical proof of concept in affected patients.
The financial results reflect the realities of a company investing heavily in research rather than generating commercial revenue. Income for the half year declined slightly to just over $11 million, largely due to fluctuations in incentive income and other sources. Research and development remained the dominant cost, with total expenditure exceeding $34 million for the period. The company recorded a net loss of approximately $22.8 million, an improvement compared with the prior year. Management attributed the reduction in spending primarily to the timing of major clinical and manufacturing milestones as well as a slowdown in new discovery initiatives.
PYC said it finished the reporting period with a strong cash balance of about $120 million, providing a foundation for ongoing clinical work and strategic flexibility.
The period also saw changes in the company’s governance structure, with a new chair and additional non-executive director joining the board while several previous members stepped down. Shortly after the reporting date, the company announced a major capital-raising initiative that could raise significant funds through a combination of an institutional placement and a shareholder entitlement offer.