Company: CSCIF
Filing Date: 2025-04-09
Form Type: 20-F
Source: 0001641172-25-003456
Chunk: 52

Company: COSCIENS Biopharma Inc.
Filing Date: 2025-04-09
Form: 20-F
Item: Item 5
Chunk 52
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 on animals for toxicity. Multiple
species are used to gather basic information on the safety and efficacy of the compound being investigated and/or researched. The FDA
regulates preclinical studies under a series of regulations called the current GLP regulations as well as regulatory requirements found
in Part 21 subchapter D of the Code of Federal Regulations. If the sponsor violates these regulations, the FDA may require that the sponsor
replicates those studies or can subject the sponsor to enforcement actions or penalties as described further below. The sponsor then
submits to the FDA an IND application based on the results from the initial testing that include the drug’s composition and manufacturing,
along with a plan for testing the drug on humans. The FDA reviews the IND to ensure that the proposed studies (clinical trials) do not
place human subjects at unreasonable risk of harm. FDA also verifies that there are adequate informed consent and human subject protections
in place.

After a sponsor submits an IND application, it must
wait thirty (30) days before starting a clinical trial to allow the FDA time to review the prospective study. If the FDA finds a problem,
it can order a clinical hold to delay an investigation, or interrupt a clinical trial if problems occur during the study. After the IND
application is in effect, a sponsor may commence human clinical trials. The sponsor typically conducts human clinical trials in three
sequential phases, but the phases may overlap. In Phase 1 trials, the sponsor tests the product in a small number of patients or healthy
volunteers (typically 20-80 healthy volunteers), primarily for safety at one or more doses. The goal in this phase is to determine what
the drug’s most frequent side effects are and, often, how the drug is metabolized and excreted. Phase 2 studies begin if Phase
1 studies do not reveal unacceptable toxicity. In Phase 2, in addition to safety, the sponsor evaluates the efficacy of the product in
a patient population somewhat larger than Phase 1 trials. The number of subjects in Phase 2 studies typically ranges from a few dozen
to about 300. This phase aims to obtain preliminary data on whether a drug works in people who have a certain disease or condition. At
the end of Phase 2, the FDA and sponsor try to come to an agreement on how large-scale studies in Phase 3 should be done.

Phase 3 studies begin if evidence of effectiveness
is shown in Phase 2. Phase 3 trials typically involve additional testing for safety and clinical efficacy in