CAMBRIDGE, Mass.—Targeted toward discovering a more tolerable treatment for people suffering with diabetic macular edema (DME), SciFluor Life Sciences Inc. has reported top-line results of a Phase 1/2 study of its lead drug, SF0166, an eye drop solution that could one day replace the traditional therapeutic mode of an injection in the back of the eye.
The Phase 1/2 study assessed the safety and preliminary efficacy of SF0166 in 40 evaluable patients with DME, randomized to one of two dose strengths (2.5 percent and 5 percent solutions) self-administered by patients as an eye drop twice a day for 28 days.
This less-intrusive treatment mode would, of course, have appeal to physicians and patients alike.
“The safety and biological activity, clearly demonstrated in this first-in-patient study, supports continued clinical development of SF0166,” says Dr. David Boyer of the Retina-Vitreous Associates Medical Group of Los Angeles. “DME is a devastating condition that often results in vision loss. A safe and effective eye drop treatment for patients living with DME would be a major advance in the fight against this debilitating disease.”
Using a simpler approach to DME treatment “may not only increase compliance, but also allow the opportunity to prevent vision loss by treating DME earlier in the disease pathway,” Boyer adds.
Dr. Omar Amirana, SciFluor’s CEO, says, “We believe SF0166 represents an important breakthrough in the treatment of retinal disease, given its unique mode of action and its administration as an eye drop. We look forward to further advancing SF0166.”
The combination of “safety, efficacy and ease of use will account for a high adoption rate by consumers,” Amirana says. “Given the practicality and ease of an eye drop therapy, we envision potential earlier treatment of this serious condition. We believe that SF0166 represents a major breakthrough in the treatment of retinal disease, given it is an eye drop and not a needle to the eye, with all the ramifications involved.”
“There are over a million Americans suffering from DME, and a similar number suffering from wet-AMD,” he adds. “If successful, SF0166 would represent a major advance in the treatment of these diseases, which today represent in excess of $8 billion in annual revenue.”
Approximately 30 million Americans are living with diabetes and roughly 4 percent of diabetes patients—more than 1 million individuals—experience DME, according to the U.S. Centers for Disease Control.
Treatment options for patients with DME traditionally include anti-VEGF drugs, corticosteroid drugs, and laser surgery. The anti-VEGF drugs are administered by frequent intravitreal injections into the back of the eye. While the biology and pathology of DME have been generally understood, safe and effective topical therapy in the form of an eye drop has remained elusive.
“The next step is to assess SF0166 in one or multiple Phase 2 studies where we will further evaluate safety, dosing and efficacy, as is typically done in Phase 2 studies,” Amirana says. “We are still in an early stage of clinical development.”
The Phase 1/2 primary outcome measure of safety was achieved with no drug-related serious adverse events observed in the study throughout the 28-day course of treatment, as well as during the 28-day follow-up period, researchers report. Ocular adverse events were recorded in the treated eyes of six patients, with all events mild in severity and one considered possibly drug-related.
SF0166 demonstrated biological activity in both patient groups, with 53 percent of the evaluable patients demonstrating a reduction in retinal thickness (RT) and improvements in visual acuity. Durability of RT response to the 28-day course of therapy was observed during the month after discontinuing treatment.
There were no significant decreases in visual acuity in study eyes during treatment or follow-up, and no patient required rescue treatment with an anti-VEGF injection during the treatment phase.
The study was conducted at six sites in the United States and included treatment-naïve patients as well as patients with prior anti-VEGF treatment.
Results of a Phase 1/2 trial in wet acute macular degeneration are expected in the fourth quarter of this year.