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

DNA

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.

FREE DAILY NEWSLETTER

Like this story? Subscribe to FierceBiotech!

Biopharma is a fast-growing world where big ideas come along every day. Our subscribers rely on FierceBiotech as their must-read source for the latest news, analysis and data in the world of biotech and pharma R&D. Sign up today to get biotech news and updates delivered to your inbox and read on the go.

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%. 

Suggested Articles

By employing heart rate signals, physical activity and sleep quality, common Fitbit trackers may be able to predict the spread of the flu.

Nanox has raised $26 million to help fuel the development and commercialization of its Star Trek-inspired digital X-ray bed.

Oncology is clearly a major medical and societal issue, but one that sees too much focus from biopharmas at the expense of other killers.