Theriva Biologics announces topline data from Phase 1 trial of VCN-01

Theriva announced topline data from the investigator sponsored Phase 1 trial conducted by collaborators at Sant Joan de Deu-Barcelona Children’s Hospital. The Phase 1 trial was designed to evaluate the safety and tolerability of two intravitreal injections of Theriva’s investigational oncolytic adenovirus VCN-01 in patients with intraocular retinoblastoma that is refractory to chemotherapy or radiotherapy, and for whom enucleation was the only recommended treatment. Key Takeaways: Patients received two intravitreal injections of VCN-01, 14 days apart, at a dose of either 2 x 109 vp/eye or 2 x 1010 vp/eye. The data for 9 evaluable patients were reviewed by the study Monitoring Committee who agreed that the trial had a positive outcome: Safety: VCN-01 was well tolerated after intravitreal administration at the 2 doses and the most frequently reported treatment-related adverse events were Grade 1 or 2. There were no dose limiting toxicities and no ocular or systemic toxicities equal to or greater than Grade 3 during the evaluation period. Some degree of ocular inflammation and associated turbidity was observed after VCN-01 injection. Inflammation was managed, and vitreous haze improved in some cases, by local and systemic administration of anti-inflammatory drugs. Antitumor effects: intravitreal VCN-01 demonstrated promising antitumor activity and did not appear to change the retinal function. Four patients presented a response characterized by unequivocal improvement in vitreous seed density. Eye enucleation was avoided in 3 patients to date, one of whom has retained their eye after 4 years of follow-up.