Some useful infoPhase II Clinical TrialOnce the initial safety...

Some useful info

Phase II Clinical Trial

Once the initial safety of the study drug has been confirmed in Phase I trials, Phase II trials are performed on larger groups (100-300) and are designed to assess how well the drug works, as well as to continue Phase I safety assessments in a larger group of volunteers and patients. When the development process for a new drug fails, this usually occurs during Phase II trials when the drug is discovered not to work as planned, or to have toxic effects.

Phase II studies are sometimes divided into Phase IIA and Phase IIB.

-Phase IIA is specifically designed to assess dosing requirements (how much drug should be given).
-Phase IIB is specifically designed to study efficacy (how well the drug works at the prescribed dose(s)).

Some trials combine Phase I and Phase II, and test both efficacy and toxicity.

Due to the nature of ATL1103, the IGF-1 level should be detected in Phase 2A stage. So I would assume that an overlapping trial for efficacy will be built into the trial design. So it is possile that Phase 2A and 2B could run concurrently on the same subjects. The efficacy could show at the end of Phase 2A (reduction in growth hormone level).
This will give an early indication of how Phase 2B will progress as well.

That is why the data from Phase 2a for Antisense drug trial can be used by Pharma to evaluate the possible success and commercialisation.

This is also why Mipomersen was licensed after Phase 2a stage with a deal of $325m plus up to $1.5b milestone.