Enrollment completed for first Phase 3 study of perfluorohexyloctane

Bausch + Lomb is kicking off the first of two Phase 3 studies evaluating perfluorohexyloctane (NOV03, Novaliq) as a first-in-class investigational drug with a novel mechanism of action to treat the signs and symptoms of dry eye disease (DED) associated with meibomian gland dysfunction (MGD) with the enrollment of 599 participants.

Additionally, in the SEECASE Phase 2 clinical study, perfluorohexyloctane met its primary efficacy endpoint of statistically significant improvement of total corneal fluorescein staining over control at eight weeks.

Perfluorohexyloctane also showed statistically significant improvement of certain symptoms, such as severity and frequency of dryness and burning/stinging of the eyes, over the entire duration of the Phase 2 study.¹

“We are committed to addressing the unmet needs of patients, and believe NOV03, if approved, may be a first-in-class treatment option for the millions of patients who suffer from Dry eye disease associated with Meibomian gland dysfunction,” Joseph C. Papa, chairman and CEO, Bausch Health, said in a statement. “This continued progress in our Phase 3 program and the published results of the SEECASE study are both exciting milestones in our development journey for perfluorohexyloctane.”

SEECASE was a prospective, multicenter, randomly assigned, double-masked, saline-controlled clinical study that evaluated perfluorohexyloctane at two dosing regimens.

The data published was based on results from 336 patients age 18 years or older who were randomized to one of four treatment groups: perfluorohexyloctane four times daily (QID), perfluorohexyloctane twice daily (BID), saline BID or saline QID (a 2:2:1:1 ratio).

The study met its primary efficacy endpoint of statistically significant improvement of total corneal fluorescein staining (tCFS) over control at eight weeks. Data showed a change from baseline of tCFS over control, for both dosing regimens (QID and BID, P < 0.001 and P = 0.009, respectively).

Effects on tCFS and symptoms started at two weeks after start of treatment and were maintained over the study duration. The effects were dosing schedule dependent. Perfluorohexyloctane was well tolerated with instillation site reactions below 3% in both treatment regimens.¹

The SEECASE study was conducted at 12 ophthalmology practices in the United States with patients who had a history of DED in both eyes and if one eye (the same eye) met the inclusion criteria at screening and at randomization time.

Several symptom assessments such as eye dryness score measured using visual analog scale (VAS), VAS for other symptoms such as burning/stinging, itching, blurred vision, and sensitivity to light, and ocular surface disease index (OSDI) were evaluated to assess the effect of perfluorohexyloctane on DED symptomatology.¹

Patients treated with perfluorohexyloctane also experienced statistically significant improvement of certain DED symptoms over the entire duration of the Phase 2 study. Changes from baseline were statistically significant compared with those of the control group at week eight [P < 0.001 (QID) and P = 0.002 (BID)].

Other symptoms evaluated included burning/stinging, sticky feeling, foreign body sensation, itching, blurred vision, sensitivity to light and pain. 

The following treatment emergent adverse events (TEAEs) reported by more than 2% of subjects were blurred vision in the perfluorohexyloctane QID group, eye irritation in the perfluorohexyloctane BID group and eye pain in the saline group.

Overall the number of patients reporting at least one TEAE was similar between the treatment groups.¹

"The results demonstrated in the SEECASE study support perfluorohexyloctane as a potential new treatment option for patients with DED associated with MGD, and we look forward to learning the results from both of the Phase 3 studies,” Christian Roesky, PhD and CEO of Novaliq GmbH, said in a statement.

In 2019, Bausch Health and Bausch + Lomb acquired an exclusive license for the commercialization and development of perfluorohexyloctane in the United States and Canada.