Alkahest Presents Positive Results from Phase 2a Open Label Study of AKST4290 in Treatment-Naïve neovascular AMD at Retina World Congress

SAN CARLOS, Calif., March 25, 2019 /PRNewswire/ — Alkahest, Inc., a clinical stage biotechnology company focused on developing transformative therapies to treat age-related diseases, shared results yesterday from its open label study, ALK4290-201. The data were delivered during a podium presentation as the Abstract Winner of the First-time Presentations of Clinical Trials and Late Breakers session at the 2019 Retina World Congress. The study evaluated the therapeutic effect and safety of the small molecule, AKST4290, formerly known as ALK4290, for the treatment of wet or neovascular age-related macular degeneration (nAMD) in treatment-naïve patients.

“Age-related macular degeneration is the leading cause of age-related blindness,” said Karoly Nikolich, Ph.D, co-founder and chief executive officer of Alkahest. “Today, the standard of care for this disease involves burdensome intravitreal injections, which can result in under-treatment of patients over time. Alkahest’s data show the potential for an orally-administered agent to safely increase visual acuity in nAMD, which would be a true medical advance for patients.”

Data demonstrated that AKST4290, a highly selective and potent small molecule antagonist of CCR3, was safe, well-tolerated, and provided a mean increase in Best Corrected Visual Acuity (BCVA) in naïve nAMD trial subjects. Measured in terms of letters gained according to the ETDRS system, 83% of subjects’ eyes had maintenance or improvement of BCVA, with a mean of +7 letters gained. Twenty-one percent of subjects gained > 15 letters. Investigators also observed trends in morphological changes in retinal pigment epithelium (RPE) detachment height in correlation with increases in BCVA, which will be further investigated in future trials. No serious adverse events or discontinuations due to adverse events were seen in the trial, and the drug was well tolerated. 

Dr. Michael Stewart, Chair, Department of Ophthalmology at the Mayo Clinic, Jacksonville commented: “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.”

Alkahest also conducted the AKST4290-202 study, a parallel Phase 2a clinical trial designed to evaluate the therapeutic effects and safety of the same treatment regimen in patients with refractory nAMD no longer responding to anti-VEGF therapy. Data from this trial will be presented later in 2019.

About ALK4290-201

The ALK4290-201 study was a Phase 2a trial that evaluated the therapeutic effects and safety of a six-week, oral treatment regimen of AKST4290 400mg twice per day (800mg total) in newly diagnosed nAMD patients naïve to anti-VEGF treatment. The study included 29 evaluable subjects. The primary endpoint was mean change in best corrected visual acuity (BCVA) measured according to the Early Treatment Diabetic Retinopathy Study (ETDRS) system. Safety was the secondary endpoint, measured by the incidence and severity of adverse events. Exploratory endpoints included therapeutic effect on disease morphology and an exploration of biomarkers, which is ongoing.

About AKST4290

AKST4290 is 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.

About Alkahest

Alkahest is a clinical stage biopharmaceutical company dedicated to treating neurodegenerative and age-related diseases with transformative therapies targeting the aging plasma proteome. The Alkahest pipeline includes multiple therapeutic candidates ranging from selected plasma fractions to protein-targeted interventions which aim to slow the detrimental biological processes of aging. Alkahest is developing novel plasma-based therapies in collaboration with Grifols, a global healthcare company and leading producer of plasma therapies. For further information see www.alkahest.com or follow us on Twitter @AlkahestInc

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Elizabeth Jeffords
Chief Commercial & Strategy Officer
Alkahest, Inc.
ejeffords@alkahest.com

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