Biotech Values

[biotech jim]

Sage used Captisol with their phase 2 PPD compound that did really well. Does that mean Ganaxolone will also meet p2 endpoints, also given they are identical compounds with the same MOA?

Ganaxolone, a non-natural steroid, has a 3beta methyl group, whereas SAGE-547 (allopregnanolone), an endogenous 5alpha reduced steroid metabolite of progesterone, has a hydrogen hanging in the 3beta position. Both have 3alpha hydroxyl groups.

The methyl group in the 3beta position restricts movement of the steroid A and B ring backbone, while the hydrogen there allows for the boat and chair configuration flexibility

Bottom line is they are both ion channel modulators with closely related but not identical structures. Note also that a few chemical catalogs and other sources have the ganaxolone structure incorrect. (I discovered the brain enzymes responsible for the 3alpha reduction, FWIW.)

As to their MOAs, that is a different question.