Aerie Pharmaceuticals announced the topline results of its pilot phase 2 study of netarsudil ophthalmic solution in a Japanese-American population. The study was designed in accordance with the requirements of Japan’s PMDA (Pharmaceuticals and Medical Devices Agency) to support the potential regulatory submission of netarsudil ophthalmic solution in Japan. Netarsudil ophthalmic solution 0.02% is known by the name Rhopressa in the United States, where it is currently marketed.
This pilot study was initially designed as a larger phase 2 trial to be conducted in the United States, enrolling Japanese subjects and Japanese-American subjects that are within second generation. Due to scarcity of qualified subjects in the United States, the enrollment of this study was limited to approximately 40 subjects across three study arms.
The primary objectives of the study were to evaluate (1) the ocular hypotensive activity of two different dose concentrations of netarsudil ophthalmic solution (0.02% and 0.04%) relative to placebo over a 28-day period, for a total of three arms all dosed in the evening, and (2) the ocular and systemic safety of netarsudil ophthalmic solution relative to placebo over that period. The ranges of unmedicated baseline IOP at 8am in the study were greater than or equal to 15 mmHg to less than 35 mmHg for subjects with open-angle glaucoma, and greater than or equal to 22 mmHg to less than 35 mmHg for subjects with ocular hypertension.
The results demonstrated that netarsudil ophthalmic solution 0.02% lowered IOP in mean diurnal IOP by a range of 5.0 to 5.3 mmHg for subjects with an average baseline IOP of 18.3 mmHg. The netarsudil ophthalmic solution 0.04% arm lowered IOP in mean diurnal IOP by a range of 5.2 mmHg to 6.6 mmHg for subjects with average baseline IOP of 20.2 mmHg. The placebo arm lowered IOP in mean diurnal IOP by a range of 2.0 to 2.5 mmHg for subjects with an average baseline pressure of 19.6 mmHg. Both netarsudil arms showed higher levels of IOP reduction as compared to placebo to a statistically significant degree at Day 28. The safety findings were consistent with previous netarsudil trials.
Aerie expects to initiate a phase 2 clinical trial in Japan in the first quarter of 2019 structured, as agreed with the PMDA, consistently with this pilot study with the addition of a 0.01% concentration of netarsudil.
“We are delighted to see that netarsudil reduced pressures in these lower-baseline subjects at consistent levels to what was demonstrated in our previous phase 3 trials in the United States. These pilot study results, while representing a relatively low number of subjects, may hold great promise for the Japanese glaucoma market. Studies in Japan have shown that Japanese glaucoma patients experience IOPs that are generally lower than those experienced in the United States and Europe, essentially what is known as low-tension glaucoma. Also, the netarsudil IOP-reducing performance in this study compares favorably to that of a twice-daily Rho kinase inhibitor currently marketed in Japan,” said Vicente Anido, Jr., PhD, Chairman and Chief Executive Officer at Aerie.
“The Japanese glaucoma market is one of the largest in the world at approximately $1 billion annually, and we believe there continues to be substantial unmet need in this very important market for Aerie,” Dr. Anido said in a news release. “With that, we are pleased to announce the opening of our Japan branch office in Tokyo, and the addition of two well-respected industry leaders to our Tokyo team. Yasuhide Fukushima joins as our Head of Strategy and Professional Affairs in Japan; he previously held a related position in Alcon Japan, Inc. Kenji Aso, MD, PhD, joins as our Head of Clinical; he previously held a related position in Japan Bayer Yakuhin, Ltd. Both are reporting to Theresa Heah, MD, M.B.A., Aerie’s Vice President of Clinical Research, Medical and Professional Affairs. We are fortunate to have such exceptional talent leading the way as we execute our global strategy.”