Perfuse Therapeutics, a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases announced today that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for a Phase 1/2a clinical study of PER-001 Intravitreal Implant in patients with glaucoma.

The first-in human single-arm, open label, Phase 1 portion of the study will evaluate the safety and tolerability of PER-001 Intravitreal Implant in patients with advanced glaucoma. The Phase 2a trial, is a patient masked, randomized, sham-controlled study that will evaluate the safety, tolerability, and pharmacodynamics of two doses of PER-001 in patients with progressing glaucoma.

"The initiation of this Ph1/2a clinical trial is a significant milestone for both Perfuse and the glaucoma community as we advance into the clinic with a novel, IOP-independent treatment for glaucoma" said Sevgi Gurkan, MD, CEO and Founder of Perfuse Therapeutics. "The absence of a safe and effective treatment of ischemia in the retina represents a significant unmet medical need and we plan to evaluate PER-001 Intravitreal Implant in other retinal diseases driven by ischemia including diabetic retinopathy, dry age-related macular degeneration and retinal vein occlusion." 

PER-001 is a first-in-class small molecule in a sustained release proprietary delivery platform that selectively targets inhibition of the endothelin pathway. Endothelin is the most potent vasoconstrictor in the human body and is a key regulator of vascular tone. Endothelin signaling is also involved in inflammation and cell death through its receptors expressed in vascular and non-vascular cells. Many studies have demonstrated that the endothelin pathway is upregulated in a wide range of ocular diseases, including glaucoma, diabetic retinopathy, age-related macular degeneration and retinal vein occlusion.

"Multiple lines of evidence implicate vascular dysregulation as a driver of glaucomatous disease progression and that excess endothelin levels play a key role in reducing ocular blood flow. 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", said Phil Lai, MD, Chief Medical Officer of Perfuse Therapeutics. "We are excited to evaluate the PER-001 Intravitreal Implant in the Phase 1/2a trial."