Novaliq submits New Drug Application seeking approval for first-of-a-kind Dry Eye Disease Treatment CyclASol®
Heidelberg, Germany, and Cambridge, MA, USA, August 09, 2022 – Novaliq, a biopharmaceutical company focusing on first- and best-in-class ocular therapeutics based on the unique EyeSol® water-free technology, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval for CyclASol® (cyclosporine ophthalmic solution), a proposed novel treatment for the signs and symptoms of dry eye disease (DED).
CyclASol® has demonstrated in two pivotal studies fast onset of therapeutic effect in the indication, clinical meaningful improvement of ocular surface damage, and excellent tolerability. Results from a 12-month long-term study confirmed that the effects are maintained, and even improved for most sign and symptom endpoints.
“This is the first submission of a novel product category of water-free topical drug therapies utilizing EyeSol® as a drug carrier”, said Christian Roesky, Ph.D., CEO, Novaliq. “CyclASol® is a first-of-a-kind drug therapy and aims to expand treatment success for patients with dry eye disease and their eye care professionals. If approved by the FDA, CyclASol® addresses important unmet medical needs in DED through its ocular surface healing effect combined with high comfort of administration.”
Dry eye is one of the most common ocular surface disorders, with approximately 18 million Americans diagnosed with DED.1,2 Inflammation and immunologic processes play a key role in the pathology of the disease.
A compromised ocular surface secondary to DED may also compromise refractive measurements before keratorefractive and phacorefractive surgeries and adversely impact expected visual outcomes after these surgeries.3,4 The impact of the corneal surface damage secondary to DED on visual function is an underestimated aspect of the disease. Multiple guidelines recommend treatment of the corneal surface damage prior to ocular procedures. A high unmet need remains for better tolerated drugs with an early onset of therapeutic effect, which are compelling to be used and prescribed.5,6
“We are very proud to see another product rapidly moving to the market, which marks yet another important inflection point and milestone in Novaliq’s growth trajectory”, said Dr. Mathias Hothum, board member and managing director of dievini. “We are currently evaluating the commercialization strategies which includes talking to interested parties.”
CyclASol® is a first-of-a-kind topical treatment of cyclosporine, a potent anti-inflammatory and selective immunomodulatory drug. Whilst not water-soluble, cyclosporine is soluble in the EyeSol® excipient perfluorobutylpentane allowing for its improved bioavailability and better efficacy on the target tissue. The product contains no oils, no surfactants and is preservative-free due to the novel carrier. This provides additional clinical benefits for patients, such as improved tolerability and decreased visual disturbances.
The NDA is supported by safety and efficacy results in over 1,000 patients with DED from a Phase 2 dose finding study, the Phase 2b/3 ESSENCE-1 study, the Phase 3 ESSENCE-2 study and its open label extension study.7,8
CyclASol® has demonstrated in two independent adequate and well-controlled, multicenter studies (ESSENCE-1 and ESSENCE-2) clinically meaningful and statistically significant improvements in the indication.
Effects on the ocular surface include a statistically significant reduction in total corneal fluorescein staining (tCFS) score favoring CyclASol® in both studies at Days 15 and 29. Up to 71.6% of patients responded within four weeks with a clinically meaningful improvement of ≥ 3 grades in total corneal staining. This proportion of responders was significantly higher compared to vehicle-treated patients in both studies. Responders showed also statistically significant improvements in a variety of symptoms compared to non-responders at day 29. The ASCRS guidelines recognize corneal staining as the single most important clinical sign of DED as it indicates the level of epithelial damage and visual impairment, and if left undertreated, DED can become chronic and more difficult to treat.3
Effect on tear production: In both studies, compared to vehicle at the end of treatment, there was a statistically significant (p<0.05) higher percentage of patients with increases of 10 mm from baseline in Schirmer’s tear test score at Day 85 and Day 29, respectively, confirming a known effect of the active ingredient cyclosporine. Meeting this endpoint in two independent studies is clinically meaningful on its own and considered to demonstrate efficacy for the treatment of signs and symptoms of DED.
Head-to-head data versus Restasis™ from the phase 2 study suggest that CyclASol® has a stronger and faster therapeutic effect on the ocular surface.8
Maintenance of effect results from the long-term study CYS-005 confirmed that the effect of CyclASol® was maintained, and even improved for most endpoints, over the 52-week treatment period.
Safety and Tolerability: Tolerability of CyclASol® was shown by high drop comfort patient ratings in both studies. The most common adverse reaction observed was instillation site reactions, which was reported in 8.1% of patients in the pooled studies. These were in all but one case mild. The only other adverse reaction reported in > 2% of the patients was visual acuity reduced (2.7%).
Novaliq is a biopharmaceutical company focusing on the development and commercialization of first- and best-in-class ocular therapeutics based on EyeSol®, the worldwide first water-free technology. EyeSol® is Novaliq’s proprietary water-free technology using ultrapure semifluorinated alkanes (SFAs) that are physically, chemically, and physiologically inert with excellent biocompatibility and a very good safety profile. Novaliq offers an industry-leading portfolio addressing today’s unmet medical needs of millions of patients with eye diseases.
In July 2022 submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) was announced seeking approval for NOV03 (perfluorohexyloctane), for the proposed indication of treating the signs and symptoms of dry eye disease (DED) associated with Meibomian gland dysfunction (MGD). In addition to CyclASol®, the company continues to progress multiple additional pipeline opportunities based on its validated EyeSol® platform, both in ophthalmology and adjacent indications like dermatology.
Novaliq GmbH is headquartered in Heidelberg, Germany and Novaliq Inc. has an office in Cambridge, MA, USA. The long-term shareholder is dievini Hopp BioTech holding GmbH & Co. KG, an active investor in Life and Health Sciences companies. More on www.novaliq.com.
- Leonardi A, Modugno RL, & Salami E. Allergy and Dry Eye Disease. Ocular immunology and inflammation. 2021; 29:1168–1176
- 2020 Dry Eye Products Market Report: A global Analysis for 2019 to 2025. Market Scope
- Starr CE, Gupta PK, Farid M, Beckman KA, Chan CC, Yeu E, Gomes JAP, Ayers BD, Berdahl JP, Holland EJ, Kim T, Mah FS (the ASCRS Cornea Clinical Committee). An algorithm for the preoperative diagnosis and treatment of ocular surface disorders. J Cataract Refract Surg. 2019; 45:669–684
- Donaldson K, Parkhurst G, Saenz B, Whitley W, Williamson B, Hovanesian J. Call to action: treating dry eye disease and setting the foundation for successful surgery. J Cataract Refract Surg. 2022; 48:623–629
- White DE, Zhao Y, Ogundele A, Fulcher N, Acs A, Moore-Schiltz L, Karpecki PM. Real-World Treatment Patterns Of Cyclosporine Ophthalmic Emulsion And Lifitegrast Ophthalmic Solution Among Patients With Dry Eye. Clin Ophthalmol. 2019; 13:2285-2292
- Dunn JD, Karpecki PM, Meske ME, Reissman D. Evolving knowledge of the unmet needs in dry eye disease. Am J Manag Care. 2021; 27:S23-32
- Sheppard JD, Wirta DL, McLaurin E, Boehmer BE, Ciolino CB, Meides AS, Schlüter T, Ousler GW, Usner D, Krösser S. A Water-free 0.1% Cyclosporine A Solution for Treatment of Dry Eye Disease: Results of the Randomized Phase II/III ESSENCE Study. Cornea. 2021; 40:1290-1297
- Wirta DL, Torkildsen GL, Moreira HR, Lonsdale JD, Ciolino JB, Jentsch G, Beckert M, Ousler GM, Steven P, Krösser S. A Clinical Phase II Study to Assess Efficacy, Safety, and Tolerability of Waterfree Cyclosporine Formulation for Treatment of Dry Eye Disease. Ophthalmology. 2019; 126:793-800
Bausch + Lomb and Novaliq Announce FDA Approval of MIEBOTM (Perfluorohexyloctane Ophthalmic Solution) for the Treatment of the Signs and Symptoms of Dry Eye Disease
Novaliq announces publication of second pivotal phase 3 trial data on CyclASol® 0.1% (Cyclosporine Ophthalmic Solution) in JAMA Ophthalmology
NOV03 Consistently Met Primary Endpoints for Signs and Symptoms of Dry Eye Disease Associated with Meibomian Gland Dysfunction
Bausch + Lomb and Novaliq Announce Publication of Second Pivotal Phase 3 Data on NOV03 (Perfluorohexyloctane) in American Journal of Ophthalmology NOV03 Consistently Met Primary Endpoints for Signs and Symptoms of Dry Eye Disease Associated with Meibomian Gland Dysfunction NOV03 PDUFA Action Date is June 28, 2023VAUGHAN, Ontario, and HEIDELBERG, Germany, March 22, 2023 – Bausch + Lomb Corporation (NYSE/TSX: BLCO) (“Bausch + Lomb”), a leading global eye health company dedicated to helping people see better to live better, today announced that American Journal of Ophthalmology has published results from MOJAVE, the second pivotal Phase 3 trial for NOV03 (perfluorohexyloctane). NOV03 is being investigated to treat the signs and symptoms of dry eye disease (DED) associated with Meibomian gland dysfunction (MGD). Results from the first pivotal Phase 3 trial, GOBI, were published earlier this year in Ophthalmology. The U.S. Food and Drug Administration (FDA) assigned NOV03 a Prescription Drug User Fee Act (PDUFA) action date of June 28, 2023.