PulseSight Therapeutics Submits CTA for Phase I Trial of PST-611

PulseSight Therapeutics SAS, a biotech firm focused on ophthalmology that creates groundbreaking non-viral vector therapies utilizing minimally-invasive delivery methods, has announced it has filed a Clinical Trial Authorization (CTA) with the Agence Nationale de Sécurité du Médicament et des produits de santé (ANSM) for a phase I trial (PST-611-CT1) to evaluate the safety and tolerability of PST-611 in humans.

PST-611-CT1 is a first-in-human trial involving single ascending doses, intended to validate the drug’s favorable safety profile and determine the maximum dose for the phase II proof-of-concept study, in six to twelve patients with dry age-related macular degeneration (AMD) or geographic atrophy (GA). The study PST-611-CT1 is projected to commence in early Q2 2025, with results expected by late 2025 or early 2026.

AMD is the primary reason for central vision impairment among older adults, impacting 200 million individuals globally. The pathogenesis of AMD is intricate and includes the disruption of iron balance, causing an accumulation of free iron that leads to inflammation, oxidative stress, and cellular death. PST-611 is a novel non-viral vector therapy designed for treating dry AMD/GA, which delivers human transferrin, a powerful iron regulator, to reestablish proper iron balance.

The PST-611-CT1 phase I clinical trial expands on PulseSight’s earlier clinical showcase of its electrotransfection delivery system and plasmid technology's positive safety profile, along with preclinical studies illustrating PST-611's advantageous effects in lowering oxidative stress and inflammation, and maintaining the integrity of the retinal pigment epithelium, suggesting its potential in preventing retinal degeneration and vision impairment.