Company: NCEL
Filing Date: 2025-06-09
Form Type: F-4/A
Source: 0001213900-25-052354
Chunk: 341

Company: NewcelX Ltd.
Filing Date: 2025-06-09
Form: F-4/A
Chunk 341
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 granted the following major markets: Hong Kong, Japan, South Korea, the U.S., Europe & Canada. 176 Compassionate Use Objectives in CDH 1 in Europe While “compassionate use” originally was understood to imply that the drug product is supplied for free, there is a growing understanding with policymakers, payers and patient organizations, that paid -forCUPs are justifiable. In Europe and other regions of the world (excluding the United States), it is recognized that CUP funding can provide an early source of biopharma revenues and incentivize the increased availability of potentially transformative treatments, especially for rare disorders and orphan diseases. Given the extensive therapeutic experience with mazindol, including off -labelin narcolepsy patients, NLS believes that there is ample precedence to justify a funded CUP in selected European countries for a defined group of patients. NLS intends to conduct either or both a cohort program and a named patient program, or NPP, each of which is highlighted below in a variety of European countries. Although NLS has yet to determine which countries, NLS will initiate CUPs in first, NLS’s current plan is to target the following countries in the following priorities (priority in each wave has yet to be determined): •First Wave: United Kingdom, Netherlands, Belgium, France, Italy and Switzerland; •Second Wave: Latin America, the Czech Republic, Denmark and Spain; and •Third Wave: China, Germany, Austria, Japan, Sweden and Taiwan. In addition to the potential benefit of generating revenues before Quilience market authorization from the regulatory agencies, the collection of data within the CUP is a key potential benefit for the overall Quilience evidence generation strategy, as NLS believes that a CUP program for Quilience would: •complement the clinical data package for regulatory submissions; •support primary regulatory approvals with longer -termfollow up effectiveness and safety data and patient lived experience evidence; •support label expansions in broader populations than included in the Quilience late state trials (e.g., in juvenile narcolepsy); •potentially accelerate regulatory approval in China if NLS was to seek regulatory approval for Quilience; •enhance the evidence package for market access and pricing and reimbursement; •provide qualitative live -experiencedata and information on the unmet needs from the patient (and caregiver) perspective that may contextualize the net therapeutic benefits offered by Quilience within the narcolepsy management paradigm; •bridge patients from the end of Phase 2