Akouos IPO Presentation Deck
AK-antiVEGF Overview
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AK-antiVEGF
Prevalence
Indication
Delivery Method
Mechanism of Action
Progress and Status
AK-antiVEGF encodes a secreted inhibitor of vascular endothelial growth factor (VEGF) to treat
vestibular schwannomas
Estimated 200,000 cases in the US and EU
AKOUOS
Treatment of sporadic vestibular schwannoma, a common intracranial tumor
• Current standard of care consists of surgical removal and/or radiation, both of which typically
result in lost hearing and can be associated with significant morbidity
Uses AAVAnc80 as a delivery vehicle to achieve local, sustained anti-VEGF protein at tumor site
Administer product candidate directly into the inner ear
Systemic anti-VEGF has been shown to reduce tumor volume and improve hearing in some
patients with vestibular schwannoma
• Local delivery may avoid the systemic side effects of high dose intravenous VEGF inhibitor
infusion and could remove or reduce the need for other interventions
Preclinical data in wild-type mice confirming anti-VEGF protein expression and tolerability
• Completed dosing for a pilot non-human primate tolerability study and preparing to request a
pre-IND meeting with FDA in 2H:20
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