– Phase 3 NEAR-1 and NEAR-2 clinical trials met primary and key secondary endpoints
– Presbyopia is the loss of ability to focus on near objects as a result of the natural aging process and affects more than 120 million people in the U.S.
– Data from these trials (N=613) will be the basis for regulatory submission in the U.S. in the second half of 2022
Ponte Vedra, FL, April 21, 2022 – Orasis Pharmaceuticals, an emerging ophthalmic pharmaceutical company focused on developing a unique eye drop to improve near vision for people with presbyopia, today announced that the Phase 3 NEAR-1 and NEAR-2 clinical trials, which evaluated the efficacy and safety of CSF-1, its novel eye drop candidate, met their primary and key secondary endpoints. Additional details of these trials will be presented at future medical meetings and will serve as the basis for the New Drug Application submission to the U.S. Food and Drug Administration (FDA) in the second half of 2022.
In both trials, CSF-1 met its primary and key secondary endpoints on Day 8, achieving statistically significant 3-line or more gain in distance-corrected near visual acuity (DCNVA), and no loss of 1-line or more in distance visual acuity. Pooled across the two studies, 40% and 50% of participants demonstrated these gains 1-hour post-dose 1 and 1-hour post-dose 2 respectively (P<0.0001). CSF-1 also achieved statistically significant 3-line improvement at all measured time points on Days 1 and 15. On Day 15, participants achieved statistically significant 3-line or more improvement in DCNVA as early as 20 minutes and up to 8 hours post-dose 1. In addition, CSF-1 demonstrated an excellent tolerability and safety profile, with comparable redness and comfort versus vehicle, validating the preservative-free presentation and proprietary formulation of CSF-1. The most common treatment-related adverse events of headache and instillation site pain occurred in only 6.8% and 5.8% of participants, respectively. Of all CSF-1 participants, only 2.6% reported moderate treatment-related adverse events. All other adverse events were mild.
These results were achieved with a minimum effective dose of pilocarpine hydrochloride at 0.4%, which is less than one-third the concentration of the commercially available treatment. With a proprietary vehicle, CSF-1 is formulated preservative-free and provides for dosing flexibility with a comfort and safety profile that does not compromise distance or night vision.
“Currently, optometry delivers 85% of all comprehensive eye care exams, making a difference in the vision of so many patients,” said Paul Karpecki, O.D., FAAO. “New and existing presbyopia patients will come into an optometry practice that now offers presbyopia drops, as well as contact lenses and spectacles – all playing a role in the management of the patients looking for options.”
“These statistically significant and clinically meaningful results are promising as eye care providers are eager to find alternate treatment options to improve quality-of-life for their patients with presbyopia, many of whom rely solely on reading glasses, which can be cumbersome,” said Edward Holland, M.D., Professor of Ophthalmology, University of Cincinnati and Director of Cornea, Cincinnati Eye Institute. “Based on the efficacy and excellent tolerability demonstrated with such a minimum effective dose, I would see CSF-1 as a clear place to start many presbyopia patients.”
“We are extremely pleased with these positive results, which mark a significant milestone for CSF-1 and Orasis, and they position us well to be the next product to launch in this exciting category,” said Elad Kedar, Chief Executive Officer of Orasis Pharmaceuticals. “The potential of CSF-1 to provide a first-line treatment option for people living with presbyopia is promising and we look forward to working with regulatory authorities to advance CSF-1 toward commercialization.”
To provide strategic options for CSF-1, Orasis plans to initiate a safety study with the option to extend long-term.
About the Studies
The Phase 3, multi-center, double-masked, parallel-group NEAR-1 and NEAR-2 clinical studies enrolled 613 participants aged 45-64 years in the U.S. with presbyopia to further evaluate the efficacy and safety of CSF-1. Participants were randomized in a one-to-one ratio of vehicle (placebo) to CSF-1 (pilocarpine hydrochloride 0.4%). CSF-1 was administered twice-daily over two weeks with four study visits: screening, Days 1 (baseline), 8 and 15. The primary endpoints were defined as the percentage of participants with a 3-line or more gain in mesopic, distance-corrected near visual acuity (DCNVA), 1-hour post-dose 1 on Day 8. Key secondary endpoints were defined as the percentage of participants with a 3-line or more gain in DCNVA and no loss of 1-line or more in distance visual acuity at 2-hours post-dose 1, 1-hour post-dose 2 and 2-hours post-dose 2 on Day 8.
CSF-1 is a novel corrective eye drop candidate being investigated for the treatment of presbyopia. CSF-1 is a proprietary, preservative-free formulation of low-dose pilocarpine and multi-faceted vehicle designed to achieve an optimal balance between efficacy, safety and comfort. CSF-1 improves near visual acuity through pupil modulation, resulting in a “pinhole effect” and an increase in the depth of field, thus increasing the ability to focus on near objects.
Presbyopia is the loss of ability to focus on near objects as a result of the natural aging process. It occurs mostly after the age of 40 when the crystalline lens of the eye gradually stiffens and loses flexibility. There are almost two billion people globally and more than 120 million people in the U.S. living with presbyopia. People with presbyopia experience blurred vision when performing daily tasks that require near visual acuity, such as reading a book, a restaurant menu, or messages on a smartphone. Presbyopia cannot be prevented or reversed, and it continues to progress gradually. Many existing treatment options can be either cumbersome or invasive, presenting a significant unmet need for quality-of-life improvement for people with presbyopia.
About Orasis Pharmaceuticals
Orasis Pharmaceuticals is developing CSF-1, a corrective eye drop for the treatment of presbyopia as an alternative to reading glasses. By repurposing existing and well-studied molecules, CSF-1 is designed to be effective, safe, comfortable, and easy-to-use. Orasis is led by a collaborative team of industry executives and eye care specialists with a broad range of experiences in research, development, and commercialization of pharmaceutical drugs, as well as finance and business development. Orasis is funded by a diverse group of sophisticated and experienced life science and healthcare investors including the ophthalmology focused venture capital fund Visionary Ventures, Sequoia Capital, SBI (Japan) Innovation Fund, Bluestem Capital, LifeSci Venture Partners, Maverick Ventures Israel, and other private investors. Orasis has offices in the U.S. and Israel. For more information, visit www.orasis-pharma.com and connect with us on LinkedIn: LinkedIn.