The first trial will be a small Phase 1 to confirm safety at anticipated therapeutic levels. It should be followed immediately by testing using sick subjects. All of this can be done at the same clinical site under the same combined Phase 1/2a trial. The Phase 1 confirms in humans the PK/PD profile shown in preclinical tox, then Phase 2 demonstrates efficacy.
The alternative would be to run the same short Phase 1 escalation with healthy subjects, then immediately file for a Phase 2/3a combined. This second option would delay efficacy testing in humans by at least 6 months. The former, combined Phase 1/2a protocol would only require approval by the test site IRB and safety committees.
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