Cutting Transplant Demand


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Perth, Nov 13: In a groundbreaking study, PYC Therapeutics reveals the success of their investigational drug, PYC-003, in human 3D models derived from patients with end-stage renal failure due to Autosomal Dominant Polycystic Kidney Disease (PKD).
The results showcase the drug’s effectiveness in addressing the root cause of the disease.
PYC-003, the fourth program in the company’s development pipeline, has demonstrated its efficacy in reducing cyst size and frequency in 3D patient-derived cyst models.

Figure 1 illustrates a notable reduction in cyst size and frequency following treatment with PYC-003 in the 3D model. This success has paved the way for PYC to expedite the drug into human trials.

These models considered the ‘gold-standard’ pre-clinical assay, provide a promising outlook for PYC-003 as a potential treatment for PKD.
The drug joins three other first-in-class RNA drug candidates in PYC’s development pipeline, all showing disease-modifying potential as they progress into or through human trials.

Figure 3 illustrates the impact of PKD on the kidney, emphasizing the disease's destructive nature.
PYC-003 not only addresses the disease at its root cause but also overcomes the 'delivery challenge.'
Figure 3 illustrates the impact of PKD on the kidney, emphasizing the disease’s destructive nature. PYC-003 not only addresses the disease at its root cause but also overcomes the ‘delivery challenge.’

Positive Results and Accelerated Pathway to Human Trials

The company plans to submit an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) in H2 2024, aiming for an accelerated pathway through human trials due to the significant unmet patient need in PKD.
PYC-003’s potential to address a monogenic disease, caused by a mutation in one gene, enhances its likelihood of efficacy in clinical trials and reduces the probability of off-target safety issues.

Global Impact of PKD and PYC’s Solution

PKD, affecting one in every 1,000 people worldwide, poses a major global health challenge. With no available drugs targeting the underlying cause, approximately 50 percent of PKD patients progress to end-stage renal failure by age 60.

 Figure 4 showcases the drug's ability to reach target cells affected by PKD, demonstrating the efficacy of PYC's drug delivery platform.
Figure 4 showcases the drug’s ability to reach target cells affected by PKD, demonstrating the efficacy of PYC’s drug delivery platform.

As PYC Therapeutics plans to advance PYC-003 into human trials, the potential breakthrough offers hope for a transformative treatment for PKD, addressing a critical unmet need in the global healthcare landscape.

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