Company: CSCIF
Filing Date: 2025-04-09
Form Type: 20-F
Source: 0001641172-25-003456
Chunk: 53

Company: COSCIENS Biopharma Inc.
Filing Date: 2025-04-09
Form: 20-F
Item: Item 5
Chunk 53
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 an expanded population (approx.
300-3,000 volunteers who have the disease or condition) at geographically dispersed test sites. The sponsor must submit to the FDA a
clinical plan, or “protocol”, accompanied by the approval of the institutions participating in the trials, prior to commencement
of each clinical trial. The FDA may order the temporary or permanent discontinuation of a clinical trial at any time.

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In the case of product candidates for cancer, the
initial human testing may be done in patients with the disease rather than in healthy volunteers. Because these patients are already
afflicted with the target disease, such studies may provide results traditionally obtained in Phase 2 studies. Accordingly, these studies
are often referred to as “ Phase 1/2” studies as they combine two phases. Even if patients participate in initial human testing
and a Phase 1/2 study is carried out, the sponsor is still responsible for obtaining all the data usually obtained in both Phase 1 and
Phase 2 studies.

The sponsor must submit to the FDA the results of
the preclinical and clinical testing, together with, among other things, detailed information on the manufacture and composition of the
product, in the form of a New Drug Application (an “ NDA”) or, in the case of a biologic, a Biologics License Applications
(a “BLA”). In a process that can take a year or more, the FDA reviews this application and, when and if it decides
that adequate data are available to show that the new compound is both safe and effective for a particular indication and that other
applicable requirements have been met, approves the drug or biologic for marketing. The amount of time taken for this approval process
is a function of a number of variables, including the quality of the submission and studies presented and the potential contribution
that the compound will make in improving the treatment of the disease in question.

FDA provides incentives, such as orphan drug
designation or pediatric exclusivity. Orphan-drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or
biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer
than 200,000 people in the U. S., or that affect more than 200,000 people but are not expected to recover the costs of developing and
marketing a treatment drug. The designation provides the sponsor with a seven-year period of U. S. marketing exclusivity if