Company: NCEL
Filing Date: 2025-09-03
Form Type: F-4/A
Source: 0001213900-25-084157
Chunk: 364

Company: NewcelX Ltd.
Filing Date: 2025-09-03
Form: F-4/A
Chunk 364
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devidence is the analysis of real -worlddata, which can originate from sources such as CUPs, and may provide a more complete picture of patient experience to inform patient -focuseddrug development, and support the advancement of randomized, placebo -controlledclinical trials to support a marketing application. While no longer commercially available in the United States or Europe, the active molecule in Quilience, mazindol, was previously widely used off -labeland for several decades for the treatment of narcolepsy. Additionally, it was prescribed under a long -termCUP administered and regulated by ANSM (“L’agence nationale de 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 -basedproducts. 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 -worlddata, financed by the French Health Ministry, and conducted in part by certain members of NLS’s team and members of NLS’s Scientific Advisory Board (prior to their work with NLS), 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 -reportedmeasure of the patients’ recent likelihood of falling asleep in everyday activities and is the same primary outcome measure widely used in other narcolepsy -relatedPhase 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 ±