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SAFETY AND THERAPEUTIC EFFECTS OF ORALLY ADMINISTERED AKST4290 IN NEWLY DIAGNOSED NEOVASCULAR AGE-RELATED MACULAR
Michael W Stewart1, Seema Garg2, Erin M Newman2
1Department of Ophthalmology, Mayo Clinic, Jacksonville, Florida.
Purpose:
To evaluate the safety and therapeutic effects of orally administered AKST4290 (formerly BI 144807 and ALK4290) in treatment-naive patients with neovascular age-related macular degeneration.
Methods:
In this prospective, multicenter, open-label Phase 2a pilot clinical study, 30 patients with newly diagnosed neovascular age-related macular degeneration self-administered AKST4290 (400 mg) orally twice daily for 6 weeks. Patients were examined weekly for safety, to measure best-corrected visual acuity (BCVA), and to perform exploratory morphologic assessments. The primary endpoint was the mean change in BCVA from baseline to end of treatment, and the secondary endpoint was safety. Exploratory endpoints investigated potential changes in macular morphology.
Results:
Mean BCVA improved by +7.0 letters (95% CI, 2.2-11.7); 24 patients (82.8%) had stable or improved BCVA, with 6 (20.7%) gaining ≥15 letters. No patients experienced severe or serious adverse events.
Conclusion:
In this 6-week study, AKST4290 treatment was associated with improved BCVA scores in patients with treatment-naive neovascular age-related macular degeneration. All adverse events were mild or moderate in severity and no safety issues were identified. Treatment of neovascular age-related macular degeneration with AKST4290 warrants further investigation in randomized, placebo-controlled trials.
Insights
Oral AKST4290 showed promising results for neovascular age-related macular degeneration. This treatment improved visual acuity and was well-tolerated in treatment-naive patients, warranting further study.
Area of Science:
- Ophthalmology
- Retinal Diseases
Background:
- Neovascular age-related macular degeneration (nAMD) is a leading cause of vision loss.
- Current treatments often require intravitreal injections.
Purpose of the Study:
- To assess the safety and efficacy of orally administered AKST4290 in treatment-naive nAMD patients.
- To evaluate changes in visual acuity and macular morphology.
Main Methods:
- A prospective, multicenter, open-label Phase 2a pilot study.
- 30 treatment-naive nAMD patients received oral AKST4290 (400 mg twice daily) for 6 weeks.
- Primary endpoint: change in best-corrected visual acuity (BCVA); Secondary endpoint: safety.
Main Results:
- Mean BCVA improved by +7.0 letters.
- 82.8% of patients had stable or improved BCVA.
- No severe or serious adverse events were reported; all events were mild to moderate.
Conclusions:
- AKST4290 treatment was associated with improved BCVA in treatment-naive nAMD patients over 6 weeks.
- The oral medication demonstrated a favorable safety profile.
- Further investigation in randomized, placebo-controlled trials is warranted.
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