AsiaNet 89264

HEIDELBERG, Germany and CAMBRIDGE, Mass., April 28, 2021 /PRNewswire=KYODO JBN/ --

- 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