MENLO PARK, Calif., Feb. 26, 2018 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq:ADVM), a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases, today announced the completion of dosing and evaluation of patients (n=2) in the first cohort of the ADVANCE Phase 1/2 clinical trial for alpha-1 antitrypsin (A1AT) deficiency. Patients were treated with a single administration of ADVM-043 at a dose of ~1E12 vg/kg (8E13 total vg). Based on a review of the preliminary safety data, the independent data monitoring committee (DMC) has recommended proceeding to the second cohort of patients, which is open for enrollment.

“We are excited to advance to the second cohort of patients with A1AT deficiency in this clinical trial,” said Amber Salzman, Ph.D., president and chief executive officer of Adverum Biotechnologies. “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.”

About the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 for A1AT deficiency
The ADVANCE Phase 1/2 clinical trial is a multi-center, open-label, dose-escalation study of ADVM-043 in patients with A1AT deficiency. The study will include up to 20 patients across up to four dosing cohorts of up to 5 patients each. The first cohort (n=2) received an intravenous (IV) low dose of ADVM-043 of ~1E12 vg/kg (8E13 total vg). The second cohort will receive an IV intermediate dose of ~5E12 vg/kg (4E14 total vg) and the third cohort will receive an IV high dose of ~1.5E13 vg/kg (1.2E15 total vg). A potential fourth cohort may be opened to evaluate an intrapleural (IP) delivery of ADVM-043.

The study will be conducted at 5 leading centers in the United States. The primary endpoint is safety and tolerability and secondary endpoints include changes in plasma concentrations of both total and M-specific A1AT levels. Adverum expects to report preliminary data from this trial in the second half of 2018.

Additional information about this clinical trial can be found at under trial identifier number NCT02168686.

About ADVM-043
ADVM-043 (AAVrh.10-A1AT) is a gene therapy candidate designed as a potential single-administration treatment to induce stable, long-term A1AT protein expression. In a preclinical proof-of-concept study, ADVM-043 demonstrated robust protein expression above therapeutic levels in mice following either intravenous (IV) or intrapleural (IP) administration. In another study in non-human primates, evidence of stable long-term expression of hA1AT mRNA was observed out to one year following IP administration of ADVM-043.


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