CAMBRIDGE, MA, April 30, 2019 – Neuroptika, a privately held biotechnology company focused on the development of novel regenerative treatments for ophthalmic diseases, today announced positive data from the company's Phase 1 clinical trial to determine the safety, tolerability, and pharmacokinetics of topical NRO-1 in healthy subjects. NRO-1 is a novel therapeutic with the potential to protect and regenerate corneal nerves in ophthalmic diseases.
The Phase 1 clinical trial was a randomized, double-blind, placebo-controlled, single-ascending-dose (SAD) and multiple-ascending dose (MAD) single-center study in 36 healthy volunteers. Both the SAD and MAD study had two cohorts of 18 total subjects. In each cohort of nine subjects, six subjects received NRO-1 and three subjects received placebo. The MAD portion of the study dosed NRO-1 for seven days.
Key results from the NRO-1 SAD and MAD trial:
1. Well tolerated across entire dose range, all patients completed dosing in both the SAD and MAD portions
2. No drug-related adverse events
3. No serious or severe adverse events and no dose response in adverse events
4. No clinical chemistry, hematology or ECG safety concerns
5. Safety profile consistent with preclinical data and supportive of continuing to Phase 2
“NRO-1 is a promising therapeutic candidate for ophthalmic diseases that targets the underlying cause of nerve degeneration in the cornea unlike most dry eye treatments which focus on symptomatic therapy,” shared Tim Min, Chief Executive Officer of Neuroptika. “We are very encouraged by the positive Phase 1 results and look forward to the continued development to bring a novel treatment option to patients suffering from dry eye disease.”
Based on the results from the single and multiple ascending dose study, Neuroptika plans to initiate a Phase 2 clinical trial of NRO-1 in dry eye disease in the second half of 2019. More information is available at www.neuroptika.com.
About Neuroptika
Neuroptika is a clinical stage biotechnology company formed in December 2018 as a spin out of Senju Pharmaceutical Co., Ltd, a privately held pharmaceutical company based in Osaka, Japan. Neuroptika is developing NRO-1, a novel therapeutic that protects and regenerates the corneal nerve. NRO-1 has generated compelling data demonstrating accelerated nerve regeneration which led to the recovery of functional nerves and corneal sensitivity in animal studies. A successful Phase 1 SAD/MAD trial was completed and Phase 2 trial for dry-eye disease will be initiated in 2H 2019. While most dry-eye treatments are focused on treating signs and symptoms, NRO-1 has the potential to become the first disease modifying regenerative treatment. NRO-1’s benefit is also being studied in other indications, including post-LASIK care, glaucoma and Leber's hereditary optic neuropathy. Our mission is to improve patients’ lives by developing novel regenerative treatments for ophthalmic diseases. For more information, please visit www.neuroptika.com.