Inhalon Biopharma, Inc.

Ind-enabling Development for IN-002, an Inhaled Muco-trapping Mab Against Respiratory Syncytial Virus - Project Summary Respiratory Syncytial Virus (RSV) Is the Leading Cause of Viral Death in Infants and Young Children, and Is Also a Major Cause of Respiratory Illness in Immune Compromised Adults and the Elderly. Unfortunately, There Is Currently No Vaccine or Effective Therapy Available for RSV. Synagis, a Monthly Intramuscular Injection of the Monoclonal Antibody (MAB) Palivizumab, Is the Only Fda-approved Intervention, But Can Only Be Used for Prevention and Is Given Only to a Very Small Subset of High-risk Infants. Synagis Is Not Effective at Treating RSV After Infection Has Begun. Thus, for the Tens of Thousands Hospitalized With RSV, Only Supportive Therapy Is Available; the Resulting Morbidity and Mortality Are Substantial, Particularly Among the Immunocompromised. Interestingly, RSV Spreads in the Lung via Shedding of Virus Exclusively Into the Airway; Thus, RSV Must Traverse the Airway Mucus (AM) Before Infecting Other Neighboring Cells, and Remains Restricted to the Airways With Little-to-no Systemic Viremia. This Unique Pathophysiology Makes RSV Difficult to Target by Systemically Dosed Therapies. We Believe an Rsv-specific, Safe and Effective Antiviral Therapy That Can Be Inhaled Directly Into the Respiratory Tract Would Provide a Powerful Option Addressing the Current Gap in Pharmacological Interventions. to Meet This Goal, Inhalon Has Been Advancing IN-002, Developed Using Its Proprietary and Patented “muco-trapping” Mab Technology Platform. IN-002 Is a Potent Anti-f Mab With Picomolar Binding Affinity and Neutralization Potency, Has Minimal Risk of Viral Escape, and Possess Suitable FC N-glycosylation for Trapping RSV in Am. in Turn, Trapped RSV Are Quickly Purged From the Airways via Natural Mucociliary Clearance Mechanisms. We Have Further Formulated IN-002 to Be Stably Nebulized Using a Vibrating Mesh Nebulizer. by Concentrating IN-002 Directly at the Site of Infection, Rather Than Delivering the Mab Systemically, We Expect to Enable Efficacious and Cost-effective Treatment of RSV, With Little Risk of Adverse Side Effects Due to Limited Systemic Adsorption From Pulmonary Delivery. in a Neonatal Lamb Model of RSV Infection, Daily Nebulized Therapy With IN-002 Initiated Even at Near Peak Viral Titers in the Lung Was Able to Reduce Infectious RSV Viral Load in the Lungs and Balf to Almost Non-detectible Levels Within 3 Days. Inhalon Is Currently Actively Engaging in Cell Line Development for IN-002. to Enable Rapid Translation Into the Clinic, We Seek to Complete the Cell Line Development in This Proposal, and Produce Tox Materials Suitable for Ind-enabling Activities Such as Tissue Cross Reactivity Studies, GLP Pulmonary Tox Studies, and GLP Nebulization Characterization Studies. Together, the Proposed Work Will Support Rapid Advancement of IN-002 Into Clinical Testing. Our Work Here With RSV Will Also Help Pave the Way for Improved, Molecularly-targeted, Inhaled Therapies for Other Respiratory Pathogens.