PER-001 / Perfuse Therap - LARVOL DELTA

Key findings from the diabetic retinopathy Phase 2a trial showed a single intravitreal administration of PER-001 released for six months (PRNewswire) - P2a | N=24 | NCT06003751 | Sponsor: Perfuse Therapeutics, Inc. | "Improved function (contrast sensitivity, visual acuity and visual field). There was a mean of +0.9 dB improvement in low luminance contrast sensitivity in the high-dose group and +0.65 dB in the low-dose group across multiple frequencies measured at week 20. In contrast, a mean of -2.1 dB worsening occurred in the control group over the same period...The low luminance, low contrast visual acuity was better by a mean difference of 5.5 and 5.1 letters from baseline in low- and high-dose groups compared to control measured at week 20. Patients with reduced peripheral vision showed an improvement of +1.8 dB per year with PER-001 treatment compared to +0.01 dB per year in the control group at six months...Perfuse Therapeutics plans to initiate pivotal trials across both indications [glaucoma, diabetic retinopathy] with a larger population of patients over a longer period, starting in the second half of 2025."

New trial • P2a data • Diabetic Retinopathy • Glaucoma

Key findings from the Phase 2a glaucoma trial showed that after single intravitreal administration of PER-001 released for six months, added to existing standard-of-care IOP-reducing therapies (PRNewswire) - P2a | N=36 | NCT05822245 | Sponsor: Perfuse Therapeutics, Inc | "22.2% of low-dose and 37.5% of high-dose patients experienced ≥7 decibel (dB) improvement in a pre-defined retina region of minimal 5 test points compared to 0% in control in six months. This improvement is 8x to 14x better than natural history of disease (2.7%) with currently available treatments. No PER-001 treated patient experienced ≥7 dB loss compared to 12.5% in control in six months. Control rate of ≥7 dB loss observed in this study is consistent with the natural history of disease (13%) with currently available treatments....This initial dataset demonstrated improvement in ocular blood flow, optic nerve structure (OCT RNFL) and function (visual field rate of change) outcomes with PER-001 in glaucoma patients."

P2a data • Glaucoma

Perfuse Therapeutics Announces Oral Presentation on PER-001 Intravitreal Implant for Glaucoma at the Association for Research in Vision and Ophthalmology (ARVO) Meeting (PRNewswire) - "Perfuse Therapeutics, Inc...today announced that an oral presentation on PER-001 intravitreal implant for glaucoma has been accepted for the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2025 being held May 4 to May 8, 2025, in Salt Lake City, Utah....The oral presentation will share data from all three cohorts of patients with glaucoma in the completed Phase 1/2a clinical trial."

P1/2 data • Glaucoma

PER-001, an endothelin antagonist, increased optic nerve head blood flow with structural and functional improvements in patients with glaucoma (ARVO 2025) - "In this study, PER-001 was shown to be safe and well tolerated after a single IVT injection in patients with glaucoma. Furthermore, PER-001 was noted to increase optic nerve head blood flow over the 6 month study and demonstrated improvement in glaucoma functional and structural outcomes compared to the control group.These findings suggest that PER-001 may be a promising IOP-independent neuroprotective and neuroenhancing treatment for patients with glaucoma."

Clinical • Glaucoma • Ophthalmology • EDN1

Perfuse Therapeutics Announces Positive Visual, Structural and Safety Data from a Ph1/2a Trial of PER-001 in Glaucoma Patients at the American Glaucoma Society Meeting (PRNewswire) - P1/2 | N=36 | NCT05822245 | Sponsor: Perfuse Therapeutics, Inc. | "The 24-week data demonstrated that PER-001 was well-tolerated, with only one drug-related adverse event of vitreous floater (mild and transient), no drug-related serious adverse events and no reports of intraocular inflammation. Patients treated with PER-001 showed a mean increase in optic nerve head blood flow of at least 10% from baseline starting at Week 1, which was sustained out to Week 24 and was associated with a mean improvement in visual field mean deviation of 1 dB/yr and a mean increase in retinal nerve fiber layer thickness of 3 microns, as measured by optical coherence tomography (OCT) at Week 24. In contrast, the sham control group revealed a 0.63 dB/yr decline in visual field mean deviation and a decrease of 1.3 microns in OCT RNFL at Week 24."

P1/2 data • Glaucoma

A Study of PER-001 in Participants with Diabetic Retinopathy (clinicaltrials.gov) - P2 | N=24 | Active, not recruiting | Sponsor: Perfuse Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial primary completion date: Nov 2024 ➔ Apr 2025

Enrollment closed • Trial primary completion date • Diabetic Retinopathy • Retinal Disorders

A Study of PER-001 in Participants with Open-Angle Glaucoma (clinicaltrials.gov) - P1/2 | N=36 | Active, not recruiting | Sponsor: Perfuse Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial primary completion date: Aug 2024 ➔ Apr 2025

Enrollment closed • Trial primary completion date • Glaucoma • Ophthalmology

Ocular and systemic safety of PER-001 Intravitreal Implant after single administration in rabbits and monkeys (ARVO 2024) - "The results of GLP toxicity studies in two species demonstrated favorable safety and tolerability profile of PER-001 Intravitreal Implants with negligible systemic exposure after single dose administration."

Clinical • Preclinical • Diabetic Retinopathy • Glaucoma • Ophthalmology • Retinal Disorders • EDN1

PER-001 Ocular Tissue Distribution and Duration of Release Following Intravitreal Administration of PER-001 Sustained Release Intravitreal Implants in Rabbits (ARVO 2024) - "These results demonstrate that intravitreal administration of PER-001 Implants were well tolerated and has the potential to deliver therapeutic levels of PER-001 to the target ocular tissues (retina and RPE/Choroid) for up to 12 weeks in rabbits."

Preclinical • Diabetic Retinopathy • Glaucoma • Ophthalmology • Retinal Disorders

An Endothelin-1 receptor antagonist PER-001 improved maladaptive neovascularization at equal efficacy as aflibercept in an oxygen-induced retinopathy mouse model. (ARVO 2024) - "ET-1 is a key driver of RNP and VEGF signaling in the OIR model, and treatment with PER-001 reduces retinal NV at equal efficacy as aflibercept, making it a promising therapeutic for treatment of underlying RNP in ischemic retinal diseases."

Preclinical • Age-related Macular Degeneration • Diabetic Retinopathy • Glaucoma • Macular Degeneration • Ophthalmology • Retinal Disorders • Retinal Vein Occlusion • EDN1

A Study of PER-001 in Participants With Diabetic Retinopathy (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Perfuse Therapeutics, Inc. | Phase classification: P2a ➔ P2

Phase classification • Diabetic Retinopathy • Retinal Disorders

A Study of PER-001 in Participants With Diabetic Retinopathy (clinicaltrials.gov) - P2a | N=24 | Recruiting | Sponsor: Perfuse Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Diabetic Retinopathy • Retinal Disorders

A Study of PER-001 in Participants With Diabetic Retinopathy (clinicaltrials.gov) - P2a | N=24 | Not yet recruiting | Sponsor: Perfuse Therapeutics, Inc.

New P2a trial • Diabetic Retinopathy • Retinal Disorders

A Study of PER-001 in Participants With Open-Angle Glaucoma (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Perfuse Therapeutics, Inc. | Trial completion date: Jan 2025 ➔ Jan 2026

Trial completion date • Glaucoma • Ophthalmology

A Study of PER-001 in Participants With Open-Angle Glaucoma (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Perfuse Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Glaucoma • Ophthalmology

A Phase 1/2a Study of PER-001 in Participants With Open-Angle Glaucoma (clinicaltrials.gov) - P1/2 | N=36 | Not yet recruiting | Sponsor: Perfuse Therapeutics, Inc.

New P1/2 trial • Glaucoma • Ophthalmology