Novartis stops short of buying Avrobio

Novartis really does love lentiviral, but maybe not as much as some had hoped. The Swiss group today bought Avrobio’s cystinosis gene therapy candidate AVR-RD-04 for $87.5m, preferring this to acquiring Avrobio outright, as had been rumoured in February. A takeout would not have broken the bank. Earlier this year some sellside analysts suggested a $200m price, but Avrobio’s stock has come off since then and the group was worth a mere $35m on Friday. Perhaps Novartis was always only interested in AVR-RD-04, an autologous ex vivo modified haematopoietic stem cell project designed to increase cystinosin, which is defective in cystinosis. The asset is in an investigator-sponsored phase 1/2 trial, from which data on six patients were presented last week at the ASGCT meeting. Avrobio had planned to start its own trial this year; this will now become Novartis’s responsibility. For Avrobio, which only had enough cash to last until the first quarter of next year, this is a reprieve, since it will now be able to keep going until the end of 2024. Still, with the early nature of its pipeline, this only looks like a temporary solution unless the group can drum up interest in its other assets.

Avrobio's pipeline
Project Disease Status
AVR-RD-04 Cystinosis  Ph1/2 single-arm study*; project being acquired by Novartis
AVR-RD-02 Gaucher disease Ph1/2 Guard1 in type 1; phase 2/3 in type 3 not yet recruiting 
AVR-RD-05 Hunter syndrome (MPS II) Ph1/2 investigator-sponsored trial initiated Apr 2023
AVR-RD-03 Pompe disease Preclinical
*Investigator sponsored; Source: Evaluate Pharma &

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