PYC Therapeutics (ASX:PYC) reports its Investigational New Drug (IND) application for its VP-001 drug has been approved by the US Food and Drug Administration (FDA).
The company says its first drug candidate is being developed for the treatment of blinding eye disease Retinitis Pigmentosa type 11 (RP11), and that the approval from the FDA represents the first potential treatment option to enter human trials for this disease.
PYC notes following the approval of the FDA it has applied for human ethics approval ahead of the planned commencement of enrolling patients in the clinical trial.
“This is very exciting for the RP11 patient community and an outstanding achievement by the PYC team”
Dosing of the first RP11 patient with VP-001 is expected to occur in Q2 2023.
Meanwhile, human studies are also expected to commence with a single ascending dose protocol treating an estimated 10 to 15 RP11 patients with the objective of establishing a safe and well-tolerated dose to progress into multi-dose trials in 2024.
Addressing the beginning of human trials, PYC Therapeutics CEO Dr. Rohan Hockings said: “This is very exciting for the RP11 patient community and an outstanding achievement by the PYC team. The validation of PYC’s platform technology as we move ahead into human trials has implications for the company’s entire pipeline of first-in-class and potentially disease-modifying drugs. The potential for patient-impact now extends well beyond RP11.”
PYC Therapeutics is an ASX-listed clinical-stage biotechnology company creating precision medicines for patients with major unmet needs in genetic disease. The company’s platform combines a novel drug delivery technology with the rapidly RNA therapeutic class to create a pipeline of first-in-class drugs that address the root cause of the targeted disease.
PYC is currently focused on advancing its VP-001 new drug candidate, which represents the first potential treatment for RP11, a blinding eye disease that begins in childhood and ultimately leads to legal blindness in middle age. The disease affects 1 in every 100,000 people and is caused by insufficient expression of the PRPF31 gene in the retina.
VP-001 is a precision therapy that aims to restore the expression of the PRPF31 gene back to levels required for the normal function of the retina utilising PYC’s proprietary drug delivery technology to overcome the major challenge for RNA drugs by ensuring that sufficient drug reaches its target inside the cells affected by RP11.