A Study of Avoralstat In Participants With Diabetic Macular Edema

A Phase 1b Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Suprachoroidal Injection of Avoralstat In Participants With Diabetic Macular Edema

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 Overview

• Status: Terminated
• Conditions: Diabetic Macular Edema (DME)
• Intervention / Treatment: Drug: avoralstat

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia
      • Investigative Site 1
    • Sydney, New South Wales, Australia
      • Investigative Site 2
    • Sydney, New South Wales, Australia
      • Investigative Site 3
  • South Australia
    • Adelaide, South Australia, Australia
      • Investigative Site 4
  • Victoria
    • Melbourne, Victoria, Australia
      • Investigative Site 5
• United States
  • California
    • Sacramento, California, United States, 95825
      • Investigative Site 6
  • Texas
    • Katy, Texas, United States, 77494
      • Investigative Site 7
    • The Woodlands, Texas, United States, 77384
      • Investigative Site 8

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single dose suprachoroidal injection of avoralstat	Drug: avoralstat; BCX4161 for suprachoroidal injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The safety and tolerability of avoralstat in participants with DME. Incidence and severity of ocular (study eye) and systemic treatment emergent adverse events (TEAEs).	From screening through EOS (Week 24)

Secondary Outcome Measures

Outcome Measure	Time Frame
Therapeutic potential of avoralstat in participants with DME. Trends in central subfield thickness (CST) as assessed by spectral domain optical coherence tomography (SD-OCT).	From baseline through Weeks 12 and 24
Therapeutic potential of avoralstat in participants with DME. Trends in Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA).	From baseline through Weeks 12 and 24

Collaborators and Investigators

• Sponsor: BioCryst Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): October 2, 2025
• Primary Completion (Actual): April 17, 2026
• Study Completion (Actual): April 17, 2026

Study Registration Dates

• First Submitted: September 26, 2025
• First Submitted That Met QC Criteria: November 13, 2025
• First Posted (Actual): November 14, 2025

Study Record Updates

• Last Update Posted (Actual): May 11, 2026
• Last Update Submitted That Met QC Criteria: May 6, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: safety; pharmacokinetics; single ascending dose; suprachoroidal injection
• Additional Relevant MeSH Terms: avoralstat
• Other Study ID Numbers: BCX4161-111

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes