MENLO PARK, Calif., May 09, 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 reported financial results for the first quarter ended March 31, 2018 and provided a corporate update.
"Our plans and timelines for our three lead gene therapy programs remain on track and we have the resources to execute," said Leone Patterson, interim president and chief executive officer of Adverum Biotechnologies. "At ASGCT next week, we look forward to presenting long-term preclinical efficacy data on ADVM-022 in wet AMD. For ADVANCE, our Phase 1/2 clinical trial of ADVM-043 in alpha-1 antitrypsin deficiency, we plan to report preliminary data in the second half of 2018. Also in the second half of 2018, we plan to submit two Investigational New Drug Applications to the FDA, for ADVM-022 in wet AMD and ADVM-053 in hereditary angioedema, as we prepare to advance these two additional gene therapies into the clinic."
Recent Progress
- In May 2018, Adverum announced long-term preclinical efficacy data on ADVM-022 gene therapy in a non-human primate model of wet age-related macular degeneration (wAMD). After 13 months, a single intravitreal injection of ADVM-022 was found to be safe and statistically significant (p<0.0001) in preventing the development of Grade IV lesions compared to the vehicle control group. The efficacy at 13 months was consistent with earlier-reported data, demonstrating that ADVM-022 induced long-term efficacy that was comparable to aflibercept, an anti-Vascular Endothelial Growth Factor (VEGF) standard-of-care therapy. ADVM-022 was well tolerated, with no serious adverse events. These data will be presented in a poster on May 17, 2018 at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting.
- In late April 2018, Adverum dosed the first patient in Cohort 2 in the ADVANCE Phase 1/2 trial for ADVM-043 in alpha-1 antitrypsin (A1AT) deficiency and continues to enroll patients. Per protocol, patients being treated with standard-of-care weekly IV infusions of A1AT protein are required to wash out for at least two months prior to receiving ADVM-043. The primary endpoint in the ADVANCE trial is safety and tolerability, and secondary endpoints include changes in plasma concentrations of both total and M-specific A1AT levels. Adverum plans to use the preliminary data from the ADVANCE study to inform next steps, including potential further dose escalation. Additional information about this clinical trial can be found at ClinicalTrials.gov under trial identifier number NCT02168686.
2018 Outlook - Planned Pipeline Milestones
ADVM-043 for A1AT Deficiency
- Report preliminary data from the ADVANCE Phase 1/2 clinical trial in the second half of 2018.
ADVM-022 for wAMD
- Complete ongoing Investigational New Drug (IND)-enabling preclinical studies.
- Submit an IND Application to the U.S. Food and Drug Administration (FDA) in the second half of 2018.
ADVM-053 for Hereditary Angioedema (HAE)
- Complete ongoing IND-enabling preclinical studies.
- Submit an IND Application to the FDA in the second half of 2018.
Upcoming Events
Financial Results for the Three Months Ended March 31, 2018
Contact for Adverum: Leone Patterson Interim President and Chief Executive Officer 650-665-7222 [email protected]
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