Strategic decision

The goal of this study is to assess the safety and therapeutic potential of a single dose of avoralstat in adult participants with DME.

Study BCX4161-111 is an open-label, single ascending dose study with 24 weeks follow-up in participants with DME. Three cohorts with a minimum of 3 participants enrolled in each cohort with 3 dose levels of avoralstat (low, medium, high) are planned.

Key Inclusion Criteria:

1. Male or female aged 18 years or older
2. Glycated hemoglobin A (HbA1c) < 10% at screening
3. Diagnosis of DME with center involvement requiring medical treatment within 6 months of screening and decreased visual acuity attributable to DME
4. Mild-to-severe non-proliferative diabetic retinopathy (NPDR) with DRSS < 61 in the study eye at screening
5. BCVA between 35 and 80 ETDRS letters (20/25 to 20/200 Snellen equivalent), inclusive, in the study eye at screening
6. CST measured by Heidelberg OCT between 325 and 600 μm in the study eye at screening

Key Exclusion Criteria:

1. Participants who have previously received more than 3 anti-VEGF injections
2. Any history of retinal surgery or other surgical intervention for diabetic ocular complications or anticipated need for use of laser photocoagulation course of any of the procedures listed in the criteria in the study eye during the study period
3. Current medically untreated diabetes mellitus or previous medically untreated diabetes mellitus with initiation of oral or injectable anti-diabetic medication ≤ 3 months prior to screening
4. Active intraocular or periocular infection or active intraocular inflammation in the study eye
5. Intraocular surgery ≤ 3 months prior to screening or anticipated need for ocular surgery in the study eye during the study period