PYC Therapeutics Limited (PYC Therapeutics, the Company, ASX: PYC), founded on October 9, 2001, and headquartered in Nedlands, Australia, is a clinical-stage biotechnology company advancing Ribonucleic Acid therapeutics to address significant unmet medical needs. Utilising its proprietary library of naturally derived cell-penetrating peptides, the Company is overcoming the challenges associated with current genetic medicines. PYC is developing a robust pipeline of novel therapies, including clinical-stage programs for inherited eye diseases and preclinical-stage programs targeting Paediatric Neurodevelopmental Disorders and Polycystic Kidney Disease (PKD). By leveraging cutting-edge science, PYC aims to pioneer a new generation of intracellular biological therapeutics.
Pre-Clinical Milestones and Safety Profile
PYC-003 has demonstrated remarkable pre-clinical success in addressing the underlying cause of Autosomal Dominant PKD, specifically the deficiency of PC1 protein, which is critical for normal kidney function. Through extensive studies conducted on Non-Human Primates and other animal models, PYC-003 has effectively restored PC1 protein levels, offering hope for a potential therapeutic solution to this life-altering condition. Safety evaluations have confirmed that the drug is well-tolerated, with no observable adverse effect level of 12 milligrams per kilogram. This finding provides a strong basis for advancing into human trials. Furthermore, biodistribution studies have highlighted the drug’s precision in targeting cyst-forming cells within polycystic kidneys, achieving significant reductions in disease markers. These findings underline the potential of PYC-003 as a transformative therapy for PKD.
Mechanism of Action and Delivery Precision
The mechanism of action of PYC-003 focuses on restoring PC1 protein levels in renal tubular epithelial cells, directly addressing the genetic basis of PKD. The drug’s design leverages PYC’s proprietary technology, ensuring high tissue specificity and a prolonged half-life for sustained therapeutic effects.
Pre-clinical pharmacokinetic studies have demonstrated that PYC-003 preferentially accumulates in kidney tissues, where it acts on cyst-forming cells, effectively delivering its therapeutic payload. This targeted approach minimises off-target effects while maximising efficacy. Importantly, the pharmacokinetic data suggests that PYC-003 may require dosing intervals of up to two months in humans, providing a convenient and patient-friendly treatment regimen.
Strategic Clinical Pathway
PYC Therapeutics is set to commence first-in-human clinical trials of PYC-003 in early 2025, building on the strong pre-clinical foundation. These trials will initially assess the safety, tolerability, and pharmacokinetics of the drug in both healthy volunteers and PKD patients. The Company’s planned regulatory submission, anticipated in the first quarter of 2025, is supported by the compelling pre-clinical data, including evidence of disease-modifying effects in advanced animal models. The strategic clinical pathway also aligns with accelerated regulatory pathways, such as those offered by the United States Food and Drug Administration for orphan drugs. By leveraging these pathways, the Company aims to expedite the approval process, with early safety and efficacy insights expected by 2026.
Addressing a Significant Unmet Need
PKD remains a condition with significant unmet medical needs, affecting approximately one in every one thousand individuals worldwide. The disease, characterised by the formation of fluid-filled cysts that impair kidney function, leads to end-stage renal failure in the majority of patients, necessitating dialysis or kidney transplantation. Over 95 percent of PKD patients currently lack effective treatment options. PYC-003 represents a groundbreaking approach by addressing the root cause of the disease through its targeted mechanism of action. This first-in-class therapy holds the promise of transforming the treatment landscape for millions of individuals. The potential market for PYC-003 is estimated to exceed ten billion US dollars annually, reflecting the widespread prevalence and severity of PKD.
Strategic Outlook and Investor Engagement
PYC Therapeutics remains committed to advancing its pipeline of Ribonucleic Acid-based therapies, which aim to address rare genetic diseases with significant unmet needs. Beyond PYC-003, the Company is also focused on clinical-stage programs for inherited eye diseases and preclinical-stage programs targeting neurodevelopmental disorders. This diversified pipeline demonstrates PYC’s capability to address a broad spectrum of genetic conditions. To engage stakeholders and highlight its progress, PYC will host an investor webinar on 28 November 2024. This event will provide an opportunity to showcase the latest advancements in the clinical development of PYC-003 and other programs. Investors and other stakeholders are encouraged to participate and gain insights into the transformative potential of PYC’s innovative therapeutic platform.
