Company: NCEL
Filing Date: 2025-06-09
Form Type: F-4/A
Source: 0001213900-25-052354
Chunk: 329

Company: NewcelX Ltd.
Filing Date: 2025-06-09
Form: F-4/A
Chunk 329
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 promote and regulate wakefulness. NLS believes Quilience may bridge this considerable treatment gap and that the current narcolepsy landscape may provide an opportunity to establish NLS as a leader in this space. Real-World Evidence in Narcolepsy The use of real -worldevidence may improve the quality and efficiency of clinical development and clinical trial design, with the potential to accelerate the development of therapies that may provide meaningful benefits to patients. Real -worldevidence 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