Small-Molecule Therapeutic for Stabilizing HIV-1 Envelope Proteins to Increase Immune Response

Small-molecule inhibitor that prolongs exposure of HIV-1 to broadly neutralizing antibodies to suppress viral entry and infection
Problem:
The human immunodeficiency virus (HIV-1) infects human cells through binding of its envelope proteins to surface-expressed CD4 and CCR5 receptors. Broadly neutralizing antibodies can recognize the native, functional shape of envelope to suppress infection. However, interactions between the envelope proteins and CD4 receptors trigger conformational changes of envelope. The host immune system cannot recognize these conformations of HIV-1 envelope and fail to suppress viral entry and infection.
Solution:
The therapeutic is a chemical analogue of a small-molecule viral entry inhibitor, BMS-806. These chemicals stabilize the HIV-1 envelope protein in its native, functional shape, and thus, blocking any conformational changes. This allows broadly neutralizing antibodies to bind and neutralize the HIV-1 spike proteins. The inhibitory effect of the therapeutic is reversible upon removal.
Technology:
Inhibition of HIV infection is based on a benzoyl ring. The therapeutics were modified with either azide or diazirine groups at the opposite side of BMS-806 to stabilize the interaction between the benzoyl group and HIV-1 envelope protein. The therapeutic also depends on an intact binding cavity within HIV-1 envelope proteins, suggesting they inhibit viral infection by blocking this site.
Advantages:

  • Long-acting and stabilize HIV-1 envelope protein in its native, functional shape for at least 21 days after a single application
  • Maintain or enhance binding of most broadly neutralizing antibodies to native, functional shape of HIV-1 envelope protein

Stage of Development:

  • Preclinical Discovery





The percent of drug-bound HIV-1 Env protein as a function of time. Existing viral entry inhibitors, BMS-806 and BMS-529 are compared to analogues . Two of the six analogues remain bound to Env protein after three weeks. In addition, nearly all analogues perform better than BMS-806 or BMS-529.
Intellectual Property:

Reference Media:

Desired Partnerships:

  • License
  • Co-development

Docket #20-9154

Patent Information: