PulseSight Therapeutics Initiates Clinical Plan of PST-611 Transferrin Vectorized Therapy for Dry AMD/GA

PulseSight Therapeutics SAS announced it has submitted a clinical trial authorization (CTA) to Agence Nationale de Sécurité du Médicament et des produits de santé (ANSM) for a phase 1 trial (PST-611-CT1) assessing the safety and tolerability of PST-611 in humans.

PST-611-CT1 is a first-in-human single ascending dose trial aiming to confirm the favorable safety profile of the drug and validate the maximal dose for the phase 2 proof-of-concept study, in 6 to 12 dry age-related macular degeneration (AMD)/geographic atrophy (GA) patients. The study PST-611-CT1 is expected to start early Q2 2025 with a readout anticipated by the end of 2025 or early 2026.

PST-611 is a first-in-class non-viral vectorized therapy in clinical development for the treatment of dry AMD/GA, expressing human transferrin, a highly potent iron regulator, to restore normal iron homeostasis.

The PST-611-CT1 phase 1 trial builds on PulseSight’s previous clinical demonstration of the favorable safety profile of its electrotransfection delivery system and plasmid technology, as well as preclinical experiments demonstrating the beneficial effects of PST-611 in reducing oxidative stress and inflammation, and preserving the integrity of the retinal pigment epithelium, with the potential of preventing retinal degeneration and vision loss.

“This first in human study is a significant milestone for the clinical development of PST-611, which we believe holds the potential to become a major new treatment option for patients with dry AMD/GA," Judith Greciet, CEO of PulseSight Therapeutics, said in a company news release. "Our goal is to confirm the safety of our drug candidate to then rapidly move into a phase 2 proof-of-concept study, to demonstrate transferrin’s ability to protect retinal cells from atrophy and preserve vision.”