US FDA Clears Perfuse Therapeutics IND Application For Phase 1/2a Trial Of PER-001 Intravitreal Implant in Patients With Glaucoma

Perfuse Therapeutics, a biopharmaceutical Firm pioneering transformational therapies to treat ischemia-induced ocular diseases, announced that the FDA has approved an Investigational New Drug (IND) application for a phase 1/2a clinical study of the PER-001 Intravitreal Implant in glaucoma patients. The study's first-in-human single-arm, open label phase 1 will assess the safety and tolerability of the PER-001 Intravitreal Implant in patients with advanced glaucoma.

The phase 2a trial is a randomized, sham-controlled, patient-masked study that will assess the safety, tolerability, and pharmacodynamics of two doses of PER-001 in patients with progressive glaucoma. "The start of this Ph1/2a clinical trial is a significant milestone for both Perfuse and the glaucoma community as we progress into the clinic with a novel, IOP-independent glaucoma treatment," said Sevgi Gurkan, MD, CEO and founder of Perfuse Therapeutics.

"There is a significant unmet medical need for a safe and effective treatment of ischemia in the retina, and we intend to evaluate PER-001 Intravitreal Implant in other retinal diseases caused by ischemia, such as diabetic retinopathy, dry age-related macular degeneration, and retinal vein occlusion." PER-001 is a first-in-class small molecule in a proprietary sustained release delivery platform that selectively inhibits the endothelin pathway. Endothelin is the most powerful vasoconstrictor in the human body and an important regulator of vascular tone. Through its receptors expressed in vascular and non-vascular cells, endothelin signalling is also involved in inflammation and cell death. Numerous studies have shown that the endothelin pathway is activated in a variety of ocular diseases, including glaucoma, diabetic retinopathy, age-related macular degeneration, and retinal vein occlusion.

"Numerous lines of evidence point to vascular dysregulation as a driver of glaucomatous disease progression, with excess endothelin levels playing a key role in reducing ocular blood flow." According to Phil Lai, MD, chief medical officer of Perfuse Therapeutics, "preclinical studies of PER-001 in relevant disease models, including the non-human primate model of glaucoma, have demonstrated potent and selective inhibition of endothelin signaling, with resultant increase in optic nerve head blood flow (presented at 2022 ARVO Meeting) as well as neuroprotective effects on the retinal ganglion cells."

"We are very excited to conduct the phase 1/2a trial with the PER-001 Intravitreal Implant." PER-001 is a novel (NCE), first-in-class small molecule endothelin receptor antagonist. The PER-001 Intravitreal Implant is a 4 mm, bio-erodible cylindrical implant, administered into the vitreous cavity of the eye using a single use, 25-gauge applicator and designed to provide a sustained release of PER-001 for 6 months. Perfuse Therapeutics is a clinical-stage biotechnology company pioneering first-in-class therapies to treat ischemia-mediated ocular diseases.