Adverum Biotechnologies Reports First Quarter 2018 Financial Results and Provides Corporate Update
“Our plans and timelines for our three lead gene therapy programs remain on track and we have the resources to execute,” said
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, 2018at 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
FDAin the second half of 2018.
- Adverum plans to attend and present data on ADVM-022 at the ASGCT 21st Annual Meeting in
Chicago, May 16-19, 2018
Poster Title: AAV.7m8-aflibercept Provides Long-term Protection in a Non-human Primate Model of
Wet Macular Degeneration Over One Year Post Intravitreal Vector Administration(#554)
Thursday, May 17, 2018, 5:15-7:15 pm CT
Location: Hilton Chicago, Stevens Salon C & D
Financial Results for the Three Months Ended
- Cash, cash equivalents and marketable securities were $247.0 million as of
March 31, 2018, compared to $190.5 millionas of December 31, 2017. In February 2018, Adverum raised $64.5 millionin net proceeds from a public offering of common stock. This quarter-end cash position is expected to fund the three lead gene therapy programs through at least the end of 2019, including preliminary clinical data for at least two of these programs, and through the initial stage of scaling up manufacturing capabilities.
- Revenues, consisting of revenue from collaborative research, were $0.2 million for the three months ended March 31, 2018, compared to $0.5 million for the same period in 2017.
- Research and development expenses were $12.8 million for the three months ended March 31, 2018, compared to
$9.1 millionfor the same period in 2017. This increase was due to an overall increase in research and development activities, including compensation and benefits, and material production costs for ADVM-022 and ADVM-053 as these programs advance toward Phase 1/2 clinical trials.
- General and administrative expenses were $5.4 million for the three months ended March 31, 2018, compared to $8.0 million for the same period in 2017. This decrease was primarily due to charges during the three months ended
March 31, 2017, including termination costs associated with the company’s master services agreement with Cornelland the litigation settlement expense.
- Net loss attributable to common stockholders was $17.2 million, or
$0.30per basic and diluted share, for the three months ended March 31, 2018, compared to $16.1 million, or $0.38per basic and diluted share, for the same period in 2017.
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with
Statements contained in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding Adverum’s plans to report preliminary data from the ADVANCE study in the second half of 2018, plans to submit two INDs to the
|ADVERUM BIOTECHNOLOGIES, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|March 31,||December 31,|
|Cash and cash equivalents||$||152,716||$||70,519|
|Prepaid expenses and other current assets||2,161||3,256|
|Total current assets||249,198||193,741|
|Property and equipment, net||2,820||3,024|
|Deposits and other long-term assets||140||140|
|LIABILITIES AND STOCKHOLDERS' EQUITY|
|Accounts payable and accrued liabilities||$||7,890||$||8,695|
|Current portion of deferred rent||138||129|
|Current portion of deferred revenue||1,246||1,850|
|Total current liabilities||9,274||10,674|
|Deferred rent, less current portion||187||222|
|Deferred revenue, less current portion||-||5,250|
|Deferred tax liability||1,250||1,250|
|Other non-current liabilities||404||481|
|Total liabilities and stockholders’ equity||$||257,158||$||201,905|
|ADVERUM BIOTECHNOLOGIES, INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(In thousands, except per share amounts)|
|Three Months Ended March 31,|
|Collaboration and license revenue||$||216||$||462|
|Research and development||12,794||9,061|
|General and administrative||5,368||7,989|
|Total operating expenses||18,162||17,050|
|Other income (expense), net||746||489|
|Net loss attributable to|
|Net loss per share attributable to common|
|stockholders, basic and diluted||$||(0.30||)||$||(0.38||)|
|Weighted-average common shares outstanding,|
|basic and diluted||57,420||42,144|
Contact for Adverum:
Leone PattersonInterim President and Chief Executive Officer 650-665-7222 email@example.com
Source: Adverum Biotechnologies, Inc.