- Topline results expected in 2nd half 2021; 475 out of a 834 target patients 
randomized in ESSENCE-2
- Enrollment for 12-month safety extension trial (ESSENCE-2 OLE) completed
- Phase 2B/3 Trial ESSENCE-1 Published in Cornea: The Journal of Cornea and 
External Disease

Novaliq, a biopharmaceutical company focusing on first- and best-in-class 
ocular therapeutics based on the unique EyeSol(R) water-free technology, today 
announced that the second Phase 3 clinical trial evaluating CyclASol(R) to 
treat the signs and symptoms of dry eye disease (DED) has randomized more than 
50% out of 834 targeted patients, with top-line results expected in the 2nd 
half of 2021.

The Phase 3 program for CyclASol(R) includes the already completed 
multi-center, randomized, double-masked, vehicle-controlled trial in 328 
patients (ESSENCE-1); the ongoing Phase 3 trial (ESSENCE-2) designed to 
reconfirm efficacy results of the ESSENCE-1 trial; and a multi-center, 
open-label, single-arm 12-month safety extension trial (ESSENCE-2 OLE). Results 
from ESSENCE-2, if positive, will allow for a New Drug Application (NDA) filing 
to the U.S Food and Drug Administration in 2022.

“We have been very successful in maintaining a good recruitment rate in the 
ESSENCE-2 trial through careful selection and support of our clinical sites, 
despite the COVID-19 pandemic environment,” said Sonja Krösser, Ph.D., Vice 
President Clinical Development at Novaliq. “Patients had the opportunity to 
roll-over into the open-label 12-month safety extension trial and we are 
pleased to report that the ESSENCE-2 OLE trial has been completely enrolled 
with a total of 200 participants. Achieving these two enrollment milestones 
indicates that we are on track with the CyclASol(R) development program to file 
our first NDA in 2022.”

CyclASol(R) is a topical, anti-inflammatory and immunomodulating ophthalmic 
solution, containing 0.1% cyclosporine A in EyeSol(R), developed for the 
treatment of DED. The unique water-free drug product is based on the EyeSol(R) 
enhanced ocular bioavailability technology that allows for several fold higher 
corneal penetration of cyclosporine A in comparison to water or oil-based 
formulations [1]. This has led to the differentiated therapeutic profile of 
CyclASol(R) with an early onset of efficacy within 2 weeks and significantly 
improved tolerability. 

The previous Phase 2B/3 clinical trial ESSENCE-1 which evaluated the efficacy, 
safety, and tolerability of CyclASol(R) in patients with DED has been published 
in Cornea: The Journal of Cornea and External Disease [2]. In this study, 
CyclASol(R) demonstrated statistically significant improvements in 
pre-specified endpoints for both signs and symptoms of DED as compared to its 
vehicle after 4 weeks. Consistent with results from an earlier Phase 2 trial 
[3], CyclASol(R) showed clinically meaningful improvements in both corneal and 
conjunctival staining to monitor the ocular surface condition and improvements 
in symptoms of dryness compared with those of its vehicle. Additionally, the 
ESSENCE 1 trial demonstrated that reading speed improves with the corneal 
staining reduction. Safety and tolerability in the trial were excellent with 
outstanding application comfort scores which are usually only reported with 
lubricating eye drops [2].

The ongoing ESSENCE-2 trial is a multicenter, randomized, double-masked, 
vehicle-controlled clinical trial to assess efficacy, safety and tolerability 
of CyclASol(R) for the treatment of signs and symptoms of DED. The trial is 
planned to enroll approximately 834 subjects in about 25 U.S. clinical centers. 
The primary endpoints of the trial are the change from baseline in total 
corneal staining and in eye dryness score at day 29. The trial will again 
include the assessment of reading speed as an objective and quantifiable 
measurement of visual function. The company expects to publish topline data 
from ESSENCE-2 in the 2nd half of 2021.

“We sincerely thank all patients, investigators and their local site personnel 
for participating in this study, as well as the entire research and development 
team in the U.S. and Heidelberg for their dedication and hard work to achieve 
this important milestone, particularly under the additional complexities due to 
the COVID-19 pandemic,” said Christian Roesky, Ph.D., CEO, Novaliq.

About Novaliq 

Novaliq is a biopharmaceutical company focusing on the development and 
commercialization of first- and best-in-class ocular therapeutics based on 
EyeSol(R), the worldwide first water-free eye-drop technology. Novaliq offers 
an industry-leading portfolio addressing today's unmet medical needs of 
millions of patients with eye diseases. 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. 

Sources:
[1] Agarwal P et al. Semifluorinated alkane based systems for enhanced corneal 
penetration of poorly soluble drugs. International Journal of Pharmaceutics 538 
(2018) 119-129

[2] Sheppard JD et al. A Water-free 0.1% Cyclosporine A Solution for Treatment 
of Dry Eye Disease: Results of the Randomized Phase II/III ESSENCE Study. 
Cornea, January 2021, Publish Ahead of Print 
https://doi.org/10.1097/ICO.0000000000002633 
 
[3] Wirta DL et al. A Clinical Phase II Study to Assess Efficacy, Safety, and 
Tolerability of Waterfree Cyclosporine Formulation for Treatment of Dry Eye 
Disease. Ophthalmology, Volume 126 (2019) 793-800

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Source: Novaliq GmbH