Perfuse Therapeutics, Inc.

SAN FRANCISCO, Aug. 3, 2023 /PRNewswire/ -- Perfuse Therapeutics, a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases is pleased to announce the initiation of enrollment into the Ph2a clinical trial of PER-001 intravitreal implant in Diabetic Retinopathy. The Phase 2a trial is a patient masked, randomized, sham-controlled study that will evaluate the safety and tolerability of two doses of PER-001 Intravitreal Implant in patients with Diabetic Retinopathy. The Company is also evaluating PER-001 intravitreal implant in a Ph1/2a clinical trial in patients with Glaucoma. "The initiation of this trial is a significant milestone for patients, the retina community and Perfuse, as we continue to advance in the clinic with a potentially disease-modifying treatment for diabetic retinopathy. Rapid advancement into multiple Ph2a studies is a testament to the skill and dedication of our team and underscores the broad potential of our intravitreal sustained release PER-001 implant in all retinal diseases driven by ischemia", said Sevgi Gurkan, M.D., Founder and CEO of Perfuse. 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 neuroretinal cells. Many studies have demonstrated that the endothelin pathway is upregulated in a wide range of ocular diseases, including diabetic retinopathy, glaucoma, age-related macular degeneration, and retinal vein occlusion. "Pathologic endothelin signaling in diabetes leads to microvascular complications and ischemia in many tissues, including the retina. Current therapies for diabetic retinopathy do not address the underlying ischemic vascular disease and the absence of a safe and effective treatment of ischemia in the retina represents a significant unmet medical need. In multiple preclinical models, treatment with PER-001 has demonstrated potent and selective inhibition of endothelin signaling, leading to improved retinal blood flow and hypoxia as well as reduction in neovascularization." said Phil Lai, M.D., Chief Medical Officer of Perfuse. About 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. About Perfuse Therapeutics, Inc. Perfuse Therapeutics is a clinical-stage biotechnology company pioneering first-in-class therapies to treat ischemia-mediated ocular diseases. Enabled by its proprietary molecules and sustained release drug delivery platform, the company is developing a robust pipeline of novel assets against validated targets. Headquartered in San Francisco, California and with R&D facilities in North Carolina, Perfuse Therapeutics has established a strong team committed to advancing disease modifying therapies to patients with ocular diseases around the world. More information on Perfuse can be found at and on LinkedIn. Investor Relations and Media Contact Please contact us for more information or questions at – [email protected].com SOURCE Perfuse Therapeutics

SAN FRANCISCO, July 20, 2023 /PRNewswire/ -- Perfuse Therapeutics, a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases is pleased to announce the successful clearance of Phase 1 safety for the low dose PER-001 intravitreal implant. This significant milestone allows the initiation of enrollment to the Ph2a portion of the Phase 1/2a clinical study of PER-001 intravitreal implant in patients with glaucoma. The low-dose cohort in the first-in human, single-arm, open label, Phase 1 portion of the study evaluating the safety and tolerability of PER-001 intravitreal implant in patients with advanced glaucoma is fully enrolled. To date, the low-dose PER-001 intravitreal implant has been found to be safe and well-tolerated allowing the advancement to the Ph2a portion of this study. 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 starting with the low-dose. 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 that the safety data supports evaluation of the low dose PER-001 intravitreal implant in the Phase 2a portion of the trial in patients with progressing glaucoma." "The initiation of the Phase 2a clinical trial is a significant milestone for both Perfuse and the glaucoma community as we continue to advance in 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." About 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. About Perfuse Therapeutics, Inc. Perfuse Therapeutics is a clinical-stage biotechnology company pioneering first-in-class therapies to treat ischemia-mediated ocular diseases. Enabled by its proprietary molecules and sustained release drug delivery platform, the company is developing a robust pipeline of novel assets against validated targets. Headquartered in San Francisco, California and with R&D facilities in North Carolina, Perfuse Therapeutics has established a strong team committed to advancing disease modifying therapies to patients with ocular diseases around the world. More information on Perfuse can be found at and on LinkedIn. Investor Relations and Media Contact – [email protected]com SOURCE Perfuse Therapeutics

This milestone marks transition of Perfuse Therapeutics to a clinical-stage company PER-001 is a first-in-class small molecule in a sustained release proprietary delivery platform that selectively targets inhibition of the endothelin pathway. (Credit: Ion Fet on Unsplash) 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.” Source: Company Press Release