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Novel inhibitors of Cytomegalovirus

Reference Number TO 02-00402

Keywords

small molecule, first-in-class, CMV, hCMV, solid organ transplantation, SOT, hematopoietic stem cell transplantation, HSCT

Invention Novelty

First-in-class, small-molecule inhibitors targeting cytomegalovirus (CMV) with a novel structure and mechanism of action. The technology is particularly effective against human CMV infections that are resistant to existing antiviral drugs, offering an innovative antiviral solution for hematopoietic stem cell transplantation and solid organ transplant recipients.

Value Proposition

CMV infections are widespread and pose severe risks for immunocompromised individuals, such as transplant recipients and newborns. Current treatments are often limited by significant side effects and drug resistance. This invention addresses these
challenges with:

  • High Potency: EC50 < 10 nM, surpassing Letermovir in efficacy
  • Resistance-Breaking Ability: Effective against Letermovir- and Ganciclovir-resistant strains
  • Targeted Action: Novel mechanism halts DNA capsid formation via the AHP binding site in the terminase complex
  • Selective Activity: Inactive against non-target viruses (murine CMV, herpes simplex, and adenovirus)

These features highlight the potential to meet an unmet medical need in CMV management.

Novel inhibitors of Cytomegalovirus

Mechanism and binding site to inhibit human cytomegalovirus (HCMV) infection (Unpublished) 

Technology Description

  • Novel  Structure: Unique molecular design with a new mechanism of action that inhibits the AHP binding site of the CMV terminase complex
  • Low Toxicity: Favorable ADME/Tox profiles demonstrated in vitro
  • Scalable Production: Cost-effective synthesis for broader commercial viability

Commercial Opportunity

The technology is available for co-development and in-licensing partnerships.

Development Status

Comprehensive in vitro ADME/Tox data and pharmacokinetic data from a mouse model are available.

Patent Situation

PCT application was filed in February 2024 (WO2024170639) and another European priority application was filed in August 2024 (EP24 194 624.3).

Further Reading

Indication proof of concept – Khawaja et al. 2023. Clin Microbiol Infect. 2023 29(1).