Analyst Says Gene Therapy Data Update Offers Insights into Potential Pivotal Endpoints

Avrobio Inc AVRO shares are trading higher after the company announced follow-up data demonstrating a durable treatment effect across key measures out to 36 months from a Phase 1/2 trial of an investigational gene therapy for cystinosis, dubbed AVR-RD-04.

Needham notes that all six adults treated demonstrated increased levels of functional cytinosin, improved cognitive and motor function, and remained off of cysteamine pills.

The analyst also writes that the therapy also increased CNS activity which is typically unheard of in this population. 

Four of the six patients (those with longer follow-up) have seen improvements or stabilization of their Beery-Buktenica Development Test scores that could potentially serve as an endpoint in a registrational trial.

The evolving data for AVR-RD-04 suggests potential registrational strategies using an accelerated approval pathway, the analyst writes. 

Regulatory clarity on a path forward for AVR-RD-04 could provide AvroBio with strategic flexibility.

Price Action: AVRO shares are up 33.1% at $0.77 on the last check Thursday.