Alkahest announced that its phase 2a trial of AKST4290, an orally-administered small molecule CCR3 inhibitor, met the primary endpoint of achieving an increase in best corrected visual acuity (BCVA) in patients with refractory wet or neovascular age-related macular degeneration (nAMD). AKST4290 was also found to be safe and well-tolerated, meeting the secondary endpoint of the trial.
The results were presented in an oral presentation at the 2019 American Society of Retina Specialists Annual Meeting (ASRS) on July 27, 2019 in Chicago by Michael Stewart, MD, Chair, Department of Ophthalmology at the Mayo Clinic, Jacksonville.
“We are pleased to announce the positive data from this study of AKST4290 in anti-VEGF refractory patients, confirming the safety, tolerability, and improvement in BCVA that was observed in a previously reported phase 2 trial in patients with treatment-naïve nAMD,” Karoly Nikolich, chief executive officer of Alkahest, said in a company news release. “These trials present early, but intriguing data on the efficacy and safety of AKST4290 as an oral treatment for nAMD that warrant continued study.”
In this patient population who are no longer responding to treatment with anti-VEGF injections, twice-daily administration of AKST4290 for 6 weeks was safe and well tolerated, with 72% of patients showing maintenance or improvement in BCVA. The mean change across 6 weeks of treatment in this refractory population was an increase of +2.0 letters gained according to the ETDRS system. 8% of subjects gained ten or more letters. AKST4290 was administered as a monotherapy without concomitant use of anti-VEGF therapy. There were no serious adverse events or adverse events leading to discontinuation of treatment.
“Currently, intravitreal injections of anti-VEGF drugs constitute the standard of care in nAMD. Though efficacious, these treatments are not only burdensome and expensive for patients and the healthcare system; some patients cease to respond to ongoing treatment, risking further vision loss,” Dr. Stewart said. “The results of AKST4290-202 combined with the results of the AKST4290-201 treatment-naïve study shared earlier this year present an early but exciting potential oral alternative to intravitreal injections and I look forward to further studies.”