Oral Medication Associated With Improved Acuity in Some AMD Patients

Elderly woman drinks a pill, glass of water, close-up
Elderly woman drinks a pill, glass of water, close-up
A tablet may help treatment-naïve patients regain lost visual acuity in 6 weeks, according to a report.

An oral tablet, AKST4290, is a safe option, with mild or moderate adverse events, and improved best corrected visual acuity (BCVA) scores in treatment-naïve patients with neovascular age-related macular degeneration (nAMD) patients at 6 weeks, according to a study published in Retina.

The prospective, multicenter, open-label Phase 2a pilot clinical trial (Clinicaltrials.gov Identifier: 03558061) looked at 30 patients newly diagnosed with nAMD who self-administered AKST4290 (400 mg) orally twice daily for 6 weeks in 2018. They underwent weekly exams that checked for safety, BCVA measurements, and exploratory morphological assessments. Primary endpoint was mean change in BCVA from baseline to end of treatment. Secondary endpoint was safety. Exploratory endpoints were possible changes in macular morphology.

Researchers found that mean BCVA improved by +7.0 letters (95% CI, 2.2–11.7). A total of 24 patients (82.8%) had stable or improved BCVA and 6 (20.7%) gained at least 15 letters. No severe or serious adverse events (SAEs) were reported, but 14 (46.7%) patients reported mild or moderate adverse events (AE). AE’s included visual acuity losses; the most common nonocular AEs were arthralgia (5 [16.7%] patients), back pain (3 [10.0%] patients), and cystitis and headache (2 [6.7%] patients each). 

“Treatment of nAMD with AKST4290 warrants further investigation in randomized, placebo-controlled trials,” the researchers explain.

The study’s main limitations were its open-label design, limited duration of treatment, small sample size, no control group, and limited geographical study area.

“As a result, no definitive assessment of the therapeutic effects of AKST4290 in nAMD patients can be made based on this preliminary study,” researchers report. “However, the observed BCVA gains warrant larger placebo-controlled trials of longer duration to fully characterize the safety and efficacy of this novel therapeutic candidate.”

Disclosure: This research was supported by Alkahest, Inc. Please see the original reference for a full list of disclosures.


Stewart MW, Garg S, McCaskill E, et al. Safety and therapeutic effects of orally administered AKST4290 in newly diagnosed neovascular age-related macular degeneration. Retina. Published online February 15, 2022. doi:10.1097/IAE.0000000000003446