Adverum Biotechnologies Announces Completion of Dosing of First Cohort of Patients in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 Gene Therapy for A1AT Deficiency

-- Data Monitoring Committee Recommends Proceeding to the Second Cohort --

-- Company Expects to Report Preliminary Data in the Second Half of 2018 --

MENLO PARK, CA, USA I February 26, 2018 I 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.

About Alpha-1 Antitrypsin (A1AT) Deficiency
A1AT deficiency is an orphan disease impacting approximately 100,000 individuals in the United States. The disease is caused by genetic mutations resulting in very low levels of A1AT. A1AT deficiency is associated with premature emphysema. The market for A1AT deficiency therapy world-wide was estimated at approximately $1.2 billion in 2016. The current standard of care for this disease includes weekly intravenous infusions of an alpha-1 proteinase inhibitor, at an estimated cost of approximately $100,000 annually per patient. The current treatment regimen can result in underdosing and lead to worsening lung function.

About Adverum Biotechnologies, Inc.
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products for ophthalmic diseases and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development. For more information please visit

SOURCE: Adverum Biotechnologies

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