Validation of Selective Target Engagement

Validation of Selective Target Engagement

Cytosolic Hsp Sparing (Critical De-Risking Data)

Direct comparative studies versus 17-AAG demonstrate:

Marker

17-AAG

Gam-05

Akt degradation

Unchanged

Chk1 degradation

Unchanged

Heat shock response

Not induced

Conclusion: Gam-05 does not inhibit cytosolic Hsp, confirming true compartmental selectivity.

Sensitive Signaling Readout: Src Phosphorylation

  1. Src kinase is highly dependent on cytosolic Hsp for stability.
  2. Even subtle cytosolic Hsp inhibition rapidly alters p-Src
  3. Gam-05 shows no disruption of Src phosphorylation, further confirming absence of off-target cytosolic effects.

This level of mechanistic validation is uncommon at Phase I entry and significantly reduces class-associated risk.