Adverum Biotechnologies has concluded dosing patients in a Phase I/II clinical trial of ADVM-043 to treat patients with alpha-1 antitrypsin (A1AT) deficiency.

The company has also completed an evaluation of patients in the first cohort of the multi-centre, open-label, dose-escalation ADVANCE trial.

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Up to 20 patients will be enrolled across in four dosing cohorts of up to five patients each across five centres in the US.

The first cohort of the ADVANCE trial has received an intravenous (IV) low-dose of ADVM-043 of ~1E12vg/kg.

The study’s primary endpoint is to achieve safety and tolerability, and the secondary endpoints include changes in plasma concentrations of both total and M-specific A1AT levels.

Preliminary data from the trial is expected to be available in the second half of this year.

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“We are eager to assess ADVM-043’s safety and tolerability, as well as protein expression in patients.”

Adverum Biotechnologies president and CEO Amber Salzman said: “We are pleased by the physician and patient interest in the ADVANCE trial and we are eager to assess ADVM-043’s safety and tolerability, as well as protein expression in patients.

“We continue to work closely with the Alpha 1 Foundation and the ADVANCE trial continues on track and we expect to report preliminary data in the second half of 2018.”

Adverum also noted that on the basis of a review of the preliminary safety data of the ADVANCE trial, the independent data monitoring committee (DMC) has recommended the company advance to the second cohort of patients with A1AT deficiency.

Enrolment for the second cohort is currently taking place.

The second cohort will receive an IV intermediate dose of ~5E12vg/kg, while the third cohort will receive an IV high-dose of ~1.5E13vg/kg.

Adverum also plans to open a potential fourth cohort to analyse an intrapleural (IP) delivery of ADVM-043.

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