Bausch + Lomb and Novaliq Announce Publication of Pivotal Phase 3 Data on NOV03 (Perfluorohexyloctane) in Ophthalmology
NOV03 is Being Investigated to Treat the Signs and Symptoms of Dry Eye Disease Associated with Meibomian Gland Dysfunction
NOV03 PDUFA Action Date is June 28, 2023
VAUGHAN, Ontario, and HEIDELBERG, Germany, Jan. 4, 2023 – Bausch + Lomb Corporation (NYSE/TSX: BLCO) (“Bausch + Lomb”), a leading global eye health company dedicated to helping people see better to live better, and Novaliq GmbH, a biopharmaceutical company focusing on first- and best-in class ocular therapeutics, today announced that Ophthalmology, the peer-reviewed journal of the American Academy of Ophthalmology, has published results from the pivotal Phase 3 trial GOBI, which is one of two pivotal Phase 3 trials for NOV03 (perfluorohexyloctane). NOV03 is being investigated to treat the signs and symptoms of dry eye disease (DED) associated with Meibomian gland dysfunction (MGD). The U.S. Food and Drug Administration (FDA) assigned NOV03 a Prescription Drug User Fee Act (PDUFA) action date of June 28, 2023.
“NOV03 was specifically developed to address excess tear evaporation and alleviate the signs and symptoms of dry eye disease associated with Meibomian gland dysfunction,” said Christina Ackermann, president, Ophthalmic Pharmaceuticals, Bausch + Lomb. “These findings reinforce NOV03 as a potential new treatment option that is distinct from anti-inflammatory and immunomodulatory agents. We are excited that the FDA is reviewing the New Drug Application for NOV03, which, if approved, would provide millions of patients with dry eye disease associated with Meibomian gland dysfunction a prescription treatment option.”1
DED is one of the most common ocular surface disorders, with MGD as a major cause of development and progression, affecting approximately nine out of 10 people with DED.2,3 DED due to MGD is caused by a deficient tear film lipid layer that leads to increased tear evaporation.4 There is currently no approved prescription eye drop in the United States indicated for DED associated with MGD. “These data show that the GOBI trial met both co-primary endpoints and all key secondary endpoints, with statistical significance achieved in treating the signs and symptoms of dry eye disease associated with Meibomian gland dysfunction as early as day 15,” said Christian Roesky, Ph.D., CEO, Novaliq. “We are pleased to be able to share these exciting findings in Ophthalmology, and we look forward to our continued collaboration with Bausch + Lomb to hopefully bring NOV03 to market in the United States, and ultimately address an unmet medical need for those suffering with dry eye disease associated with Meibomian gland dysfunction.”
“All of the patients in the study were required to meet specific criteria indicating presence of Meibomian gland dysfunction. The findings of this study reinforce NOV03 as a potential treatment option for this patient population with unmet medical needs,” said Yehia Hashad, M.D., executive vice president, Research & Development and chief medical officer, Bausch + Lomb.
The data from the Phase 3, multicenter, randomized, hypotonic saline-controlled, double masked GOBI study was based on results from 597 subjects aged 18 years and older who were randomized to either receive treatment with NOV03 four times daily or hypotonic saline solution four times daily (n=303 NOV03; n=294 saline).
The two primary endpoints were change from baseline at Week 8 (Day 57 ± 2) in total corneal fluorescein staining (tCFS) and eye dryness Visual Analog Scale (VAS) score. Key secondary endpoints included change from baseline in eye dryness VAS score and tCFS at Week 2 (Day 15 ± 1) and eye burning/stinging VAS score and central corneal fluorescein staining (cCFS) at Week 8. Significant improvements vs. hypotonic saline solution were seen as early as day 15. Data highlights include:
At Week 8, change from baseline in tCFS was statistically significantly greater in the NOV03 arm compared to the control saline group (least-squares [LS] mean treatment difference, -0.97; (95% confidence interval [CI]: -1.40 vs. -0.55) (P<0.001)).
At Week 8, eye dryness VAS score was statistically significantly improved in the NOV03 arm compared to control group (LS mean treatment difference, -7.6; (95% CI: -11.8 vs. -3.4) (P<0.001).
Key secondary endpoints
At Week 2 (day 15), tCFS and eye dryness VAS score were statistically significant compared to saline, with an LS mean treatment difference (95% CI) for change from baseline in tCFS of -0.6 (-0.9, -0.2) (P<0.01) and VAS score of -4.7 (-8.2, -1.2) (P<0.01).
At Week 8, VAS burning/stinging score and cCFS also favored the NOV03 group, with an LS mean treatment difference (95% CI) for change from baseline in VAS burning/stinging score of -5.5 (-9.5, -1.6) (P<0.01) and cCFS of -0.2 (-0.4, -0.1) (P<0.01).
In the study, NOV03 was well tolerated with few subjects experiencing ocular adverse events (AEs) (9.6% NOV03 group, 7.5% control group) or treatment-related ocular AEs (6.3% NOV03 group, 3.1% control group). Most AEs were mild to moderate in severity. The most common AEs (incidence ≥1%) experienced in the NOV03 group were blurred vision, mostly mild and transient (3.0% vs 0.3%), instillation site pain (1.0% vs 1.0%), and eye discharge (1.0% vs. 0.0%). Ocular AEs led to treatment discontinuation in one subject in the NOV03 group (eye irritation) and three subjects in the saline group (conjunctivitis, dry eye, punctate keratitis).
About NOV03 (perfluorohexyloctane) Ophthalmic Solution
NOV03 is an investigational, proprietary, water-free, single-component preservative-free eye drop.5 In 2019, Bausch + Lomb acquired an exclusive license for the commercialization and development of NOV03 in the United States and Canada. Results from the pivotal Phase 2 trial (SEECASE) were published in Cornea in September 2021. Data from the first pivotal Phase 3 trial (GOBI) were presented at the American Society of Cataract and Refractive Surgery (ASCRS) annual meeting in Washington, D.C. on April 24, 2022. Data from the second pivotal Phase 3 trial (MOJAVE) were presented at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in Denver on May 2, 2022. The clinical program for NOV03 concluded with the completion of a multi-center, open-label, single-arm, 12-month safety extension trial (KALAHARI). In September 2022, Bausch + Lomb and NOV03 announced that the U.S. FDA had accepted the NDA filing for NOV03 and assigned a PDUFA action date of June 28, 2023.
National Eye Institute. Dry Eye. Last updated April 8, 2022. Accessed December 21, 2022. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/dry-eye
Leonardi, A., Modugno, R. L., & Salami, E. (2021). Allergy and Dry Eye Disease. Ocular immunology and inflammation, 29(6), 1168-1176. https://doi.org/10.1080/09273948.2020.1841804.
Lemp, MA, Crews, LA, Bron AJ. (2012). Distribution of Aqueous-Deficient and Evaporative Dry Eye in a Clinic-Based Patient Cohort: a retrospective study. Cornea, 31(5), 472-478. https://journals.lww.com/corneajrnl/Abstract/2012/05000/Distribution_of_Aqueous_Deficient_and_Evaporative.2.aspx
Geerling G, Baudouin C, Aragona P, et al. (2017). Emerging strategies for the diagnosis and treatment of meibomian gland dysfunction: Proceedings of the OCEAN group meeting. The Ocular Surface,15(2): 179-192. https://doi.org/10.1016/j.jtos.2017.01.006
In December 2019, Bausch Health acquired the rights from Novaliq GmbH to pursue development and commercialization of NOV03 for DED and combination products based on NOV03 in additional ophthalmic indications in the United States and Canada.