Company: NCEL
Filing Date: 2025-05-16
Form Type: 20-F
Source: 0001213900-25-044868
Chunk: 162

Company: NewcelX Ltd.
Filing Date: 2025-05-16
Form: 20-F
Item: Item 4
Chunk 162
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 sécurité du medicament et des produits de santé”), the national agency in France responsible for
overseeing the safety of medicines and health products, helping to address the unmet needs of patients who did not initially respond,
later failed or were unable to tolerate the approved available treatments, such as modafinil, methylphenidate, sodium oxybate, and amphetamine-based
products. This CUP ran for 17 years, ended in 2016 due to unavailability of the product, and more than 200 patients with narcolepsy who
were refractory to available treatments were prescribed mazindol for the treatment of EDS and/or cataplexy.

A retrospective, multi-center,
observational study of this real-world data, financed by the French Health Ministry, and conducted in part by certain members of our team
and members of our Scientific Advisory Board (prior to their work with us), was performed to evaluate the effectiveness and safety of
mazindol in real-world, clinical practice. A total of 94 patients with narcolepsy, and suffering from cataplexy, including adults and
children, with a mean 30 months of treatment exposure were included in the analysis to evaluate the effectiveness of mazindol on improving
EDS. Mazindol noticeably improved EDS (p<0.0001), as measured by the ESS, a validated patient-reported measure of the patients’
recent likelihood of falling asleep in everyday activities and is the same primary outcome measure widely used in other narcolepsy-related
Phase 3 clinical trials. An analysis was also performed assessing the effectiveness of mazindol in controlling cataplexy in 62 patients
and demonstrated a statistically significant reduction in the frequency of cataplexy episodes (p<0.0001). The ESS score before mazindol
was 18.0 ± 3.1 and decreased to 13.6 ± 5 after mazindol, for a change of -4.2 (p<0.0001). In addition, the change in
weekly cataplexy rate before and after mazindol changed from 4.6 ± 3.1 (before mazindol) to 2.0 ± 2.8 after mazindol (for
a change of -2.7) (p<0.0001).

This retrospective study with
analysis of real-world data, provides positive real-world evidence that the treatment