Adverum delays start of gene therapy PhI/II by one year, blames manufacturing


Adverum Biotechnologies ($ADVM) has delayed the start of a Phase I/II trial of its alpha-1 antitrypsin (A1AT) deficiency gene therapy by 12 months. The company, which was formerly known as Avalanche Biotechnologies, blamed the setback on the need to upgrade the production process and transfer it to a contract manufacturer.

Avalanche acquired the A1AT deficiency gene therapy in the buyout of Annapurna, a deal it struck to reboot its pipeline in the wake of less-than-stellar data from a clinical trial of its wet, age-related macular degeneration asset. Once the deal closed, Avalanche rebranded as Adverum and pinned its near-term hopes on the A1AT gene therapy, ADVM-043.

Adverum planned, as recently as August, to move into Phase I/II by the end of the year, but that deadline is now set to come and go. The new goal is to start enrolling patients in the fourth quarter of next year, more than 12 months after Adverum initially hoped to get the trial underway.

Adverum is attributing the delay to a decision to upgrade the production process and transfer the revised method to a third-party manufacturer. With ADVM-043 having transferred quickly from Weill Cornell Medicine's Ron Crystal, to Annapurna and then to Avalanche, Adverum sees value in hitting pause now.

Specifically, Adverum plans to adapt ADVM-043 to its baculovirus-based AAV production system and purification processes, capabilities Avalanche established through a pact with contract manufacturer Lonza. Once Adverum has brought the production process up to industry standard and transferred to its large-scale production partner, it thinks it will be positioned to scale up output of ADVM-043 as it advances through the clinic and potentially on to the market.

Responsibility for ensuring the process runs smoothly will fall on Amber Salzman, who is taking over as CEO from Paul Cleveland. Salzman led Annapurna to the buyout by Avalanche. Cleveland, the architect of that deal, is taking on the role of executive chairman of the board.

The reshuffle at the top gives Adverum a leader with experience in the hotseat at fledgling biotechs. Since walking away from GlaxoSmithKline ($GSK) after more than 20 years at the company, Salzman has held leadership positions at Alophera Therapeutics, Cardiokine and Venuvics Pharmaceuticals.

Shares in Adverum opened up 5%.