Company: NCEL
Filing Date: 2025-05-16
Form Type: 20-F
Source: 0001213900-25-044868
Chunk: 168

Company: NewcelX Ltd.
Filing Date: 2025-05-16
Form: 20-F
Item: Item 4
Chunk 168
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 decades of highly promising off-label
use and compassionate use of mazindol in patients with narcolepsy.

Quilience Label Expansion

Following our current
focus on the development of Quilience for narcolepsy in adults, and if approved for marketing, we intend to seek a label expansion
for the treatment of narcolepsy in pediatric patients, which may require additional nonclinical and clinical studies. We are also
aiming to develop Quilience for the treatment of IH, a rare and chronic hypersomnia disorder for which there is currently no
effective or approved treatments available. Its hallmark symptom is chronic EDS and a craving to sleep during the day, regardless of
how many hours slept at night, which results in such persons taking daytime naps that are usually long and not refreshing.
Individuals with IH struggle to wake, despite setting multiple alarms and may have difficulty rising from bed, called sleep inertia.
Sleep inertia also includes feelings of grogginess upon waking and can result in impaired alertness and interfere with the ability
to perform mental or physical tasks. Similar to narcolepsy, people with IH may also suffer from hallucinations and sleep paralysis
when going to bed or upon waking.

The active molecule in Quilience
was also prescribed under compassionate use for the treatment of IH, providing positive real-world evidence of its benefit in improving
EDS specifically in patients with IH. We have received orphan drug designation from both the FDA and the European Commission for the treatment
of IH and this designation is expected to provide us initially with 7 years and 10 years of market exclusivity in the United States and
Europe, respectively. Additionally, the recently granted patent for a proprietary modified-release formulation, provides patent protection
through 2037 in the United States.

Quilience Development Program

We conducted a Phase 2 randomized,
double-blind, placebo controlled clinical trial in adult patients with narcolepsy in the third quarter of 2021 and concluded the trial
in the third quarter of 2022. The primary endpoint of the study was the change from baseline in EDS, as measured by the Epworth Sleepiness
Scale (ESS). The key secondary endpoint was the change from baseline in the weekly number of cataplexy attacks in the subset of patients
with cataplexy. The trial design is shown below:

The trial was conducted in
21 clinics in the U. S. and enrolled 67 patients with both narcolepsy Type 1 and Type 2, who received treatment