NLS Pharmaceutics, a Swiss clinical-stage biopharmaceutical company focused on the discovery and development therapies for patients with rare and complex central nervous system disorders, announces an interim update from its open label extension (OLE) study for Quilience (Mazindol ER) in the treatment of narcolepsy.

The OLE study offers patients completing the 4-week randomized, double blind (DB) Phase 2 trial for Quilience the option to receive the drug candidate for an additional 6 months as monotherapy on an open-label basis. Of the 60 patients who completed the randomized controlled Phase 2 trial, 52 patients (or 87% of completers) elected to roll over into the OLE study.

Excessive Daytime Sleepiness

For patients treated with Quilience in the randomized Phase 2 trial, excessive daytime sleepiness (EDS) based on the Epworth Sleepiness Scale (ESS) improved by an additional 1.8 points in the OLE study by the fourth month of treatment (DB and OLE treatment combined). At that timepoint, the mean ESS score for these patients reached 9.2, with lower scores denoting an improvement in the condition (improved wakefulness).

ESS scores of 10 or below are considered to be typical scores for patients without narcolepsy. As an extension of the 4-week randomized treatment period in the Phase 2 trial, these data indicate that maximum efficacy for EDS with Quilience is reached at approximately 3 months of treatment. Overall, the mean score for these patients declined by approximately 8.7 points from their baseline levels at the start of the randomized Phase 2 trial to month 3 in the OLE.

For patients receiving placebo in the DB Phase 2 trial and rolling over to receive Quilience in the OLE study, EDS scores declined to levels comparable to those treated with Quilience in the randomized trial at week 4. This effect was maintained through month 3 in the OLE study, with EDS scores just above the “normal” range.

Weekly Cataplexy Episodes

For patients diagnosed with cataplexy and treated with Quilience in the randomized Phase 2 trial, the mean number of weekly cataplexy episodes was approximately 8 at the end of the 4-week DB period, down from a baseline level of approximately 17.5 at the beginning of the trial. During the OLE study, mean weekly cataplexy episodes for these patients declined to 2.1, and remained relatively stable in the 2 to 4 range through week 12.

For patients with cataplexy receiving placebo in the DB Phase 2 trial, the mean number of weekly cataplexy episodes was approximately 10.9 at the end of the 4-week double-blind period. During the OLE study, these patients, when treated with Quilience, were able to catch up to previously treated patients, achieving mean weekly cataplexy episodes of 2.7 at 8 weeks of treatment. This favorable effect was maintained for these patients in the 2-5 episodes per week range through week 12.

Notably, there were patients diagnosed with Narcolepsy Type I who achieved zero weekly cataplexy episodes in the OLE study, with some of those maintaining this effect through week 12 and beyond.

“These early open label extension study data demonstrate the potential of Mazindol ER to effectively treat EDS and cataplexy with a once-daily oral medication,” says Thomas Stern, MD, advanced respiratory and sleep medicine, PLLC, Huntersville NC, in a statement, “As an investigator in both trials, I found the treatment to be highly effective in some of my patients, and those patients elected to roll into the OLE study and continued to do well. As a result, I believe that many patients suffering from narcolepsy could benefit from Mazindol ER as a monotherapy or as part of their treatment regimen.”

“These data from our OLE study seem to confirm the positive results from our Phase 2 trial for Quilience in narcolepsy, and we believe they also demonstrate the longer-term efficacy, tolerability, and safety of the treatment,” says George Apostol, chief medical officer of NLS Pharmaceutics, in a statement. “We are looking forward to sharing final OLE results in the first quarter of next year, along with the full data set from our Phase 2 clinical trial at a major sleep congress in 2023. The results from both studies highlight the benefits of Mazindol ER in treating EDS and cataplexy, the two core symptoms of narcolepsy, and we will continue to advance this program to bring Quilience to patients as soon as possible.”

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