Theriva Biologics Phase 1 data: VCN-01 well-tolerated in retinoblastoma; eye preserved in 3 of 9 patients

Theriva Biologics, Inc.

VCN-01 intravitreal injection at 2E+10 vp/eye was well-tolerated; no retinal toxicity or DLTs; main AE Grade 1-2 uveitis.
Four of nine patients had improvement in vitreous seed density; 3 avoided enucleation with follow-up up to 49 months.
No Grade >=3 ocular or systemic toxicities; selective replication in tumor cells confirmed by immunohistochemistry.
RP2D defined as 2 doses of 2E+10 vp/eye; VCN-01 genomes in aqueous humor correlated with vitreous seed persistence.

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