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Alkahest’s oral small molecule drug aims to report on a Phase IIa study for age-related macular degeneration

Alkahest Inc., a clinical stage biopharmaceutical company based in San Carlos, California, has announced that an experimental drug, AKST4290, in a Phase IIa study for AMD, showed that  83% of patients maintained or improved their visual acuity, and 21% of patients gained at least 15 letters in BCVA. The open-label Hungarian trial enrolled 29 patients to receive twice-daily 400 mg oral AKST4290 and, to date, results reported an mean improvement of 7 letters in best corrected visual acuity (BCVA) from a baseline to six weeks in treatment-naïve patients.


The clinical trial states that the AKST4290 molecule will evaluate the therapeutic effects and safety of 800 mg administered daily over a 6-week dosing period in newly diagnosed patients with wet age-related macular degeneration (wAMD).  The NCT identifier, NCT03558061, is logged in and a study report should be available within 12-months from the completion of the trial.  Similarly, the European Union Clinical Trials Register (EUCTR) will also report the results of the trial should be available also within 12-months of study completion.  According to the company, AKST4290 is a highly selective and potent small molecule antagonist of CCR3 stating the drug as “an orally administered CCR3 inhibitor that blocks the action of eotaxin, an immunomodulatory protein that increases as humans age and with specific age-related diseases. By targeting eotaxin and its downstream effects, AKST4290 may reduce the hallmark inflammation and neovascularization of wet AMD while also acting more broadly to reduce inflammation associated with many other age-related diseases”.


Commenting on the clinical trial results, Dr. Michael Stewart, Chair, Department of Ophthalmology at the Mayo Clinic, Jacksonville stated: “These early data for AKST4290 are extremely promising, showing an impressive increase in visual acuity even over a short 6-week period of treatment. The potential for gaining meaningful visual improvement with an oral agent in neovascular AMD represents a major step forward for patients. Based on the data, it is clear these encouraging results warrant further study.”