Adverum Biotechnologies Reports Fourth Quarter 2017 Financial Results and Provides Corporate Update
“We enter 2018 with significant momentum following a year of critical execution to transform Adverum into a clinical-stage company,” said
February 2018, Adverum completed the dosing and evaluation of patients (n=2) in the first cohort of the ADVANCE Phase 1/2 trial for ADVM-043 in alpha-1 antitrypsin (A1AT) deficiency. Based on a review of the preliminary safety information, the independent data monitoring committee (DMC) recommended proceeding to the second cohort of patients, which is open for enrollment. Adverum has initiated patient enrollment in the second intermediate-dose cohort, which will receive an intermediate dose of ~5E12 vg/kg (equivalent to ~4E14 total vg based on an 80-kg patient). The primary endpoint is safety and tolerability, and secondary endpoints include changes in plasma concentrations of both total and M-specific A1AT levels. Additional information about this clinical trial can be found at ClinicalTrials.gov under trial identifier number NCT02168686.
February 2018, Adverum raised $69.0 millionin gross proceeds from an underwritten public offering of its common stock.
January 2018, Adverumand Editas Medicine, Inc.announced the extension of the companies’ collaboration agreement to explore the delivery of genome editing medicines to treat up to five inherited retinal diseases.
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
- Report 12-month efficacy data in non-human primates in the first half of 2018.
- Complete 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 IND-enabling preclinical studies.
- Submit an IND Application to the
FDAin the second half of 2018.
- Adverum plans to attend the following upcoming conferences:
° Cowen 38th Annual
Health Care Conferencein Bostonon March 12, 2018at 1:30 pm ET
° Cowen 17th Annual Life Sciences Winter Meeting in
Colorado, March 20-23, 2018
° ARM Cell & Gene Therapy Investor Day in
New York, April 17, 2018
° ARVO 2018 Annual Meeting in
Honolulu, April 29-May 3, 2018
• Poster titled “Therapeutic potential and safety of sequential intravitreal dosing to the contralateral eye of novel AAV vectors in non-human primates” on
May 3, 2018, 8:15 – 10:00 am HST
• Poster titled “Long-term functional delivery of the human L-opsin cDNA via intravitreal administration of an AAV vector in Mongolian gerbils” on
May 3, 2018, 8:15 - 10:00 am HST
° 2nd Annual Gene Therapy for Rare Disorders 2018 Meeting in
Boston, April 30-May 2, 2018
° ASGCT 21st Annual Meeting in
Chicago, May 16-19, 2018
Financial Results for the Three Months Ended
- Cash, cash equivalents and marketable securities were $190.5 million as of
December 31, 2017, compared to $186.6 millionas of September 30, 2017and $222.2 millionas of December 31, 2016. The year-end cash position, added with approximately $64 million in net proceeds raised in February 2018, is expected to fund the three lead gene therapy programs through 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 $0.5 million for the three months ended December 31, 2017, compared to $0.5 million for the same period in 2016.
- Research and development expenses were $12.0 million for the three months ended December 31, 2017, compared to
$7.9 millionfor the same period in 2016. This increase was due to an overall increase in research and development activities for the Company’s gene therapy programs, primarily for material production costs for the ADVANCE clinical trial for ADVM-043.
- General and administrative expenses were $4.0 million for the three months ended December 31, 2017, compared to $4.8 million for the same period in 2016. This decrease was primarily due to lower legal fees.
- Net loss attributable to common stockholders was $14.8 million, or
$0.32per basic and diluted share, for the three months ended December 31, 2017, compared to $22.4 million, or $0.54per basic and diluted share, for the same period in 2016.
- Shares of common stock outstanding were 62.2 million as of
February 28, 2018.
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 matters that are not historical facts 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, potential opportunities, expectations, projections, goals, objectives, milestones, strategies, product pipeline, financial condition and results of operations, the sufficiency of its cash, cash equivalents and marketable securities, as well as the advancement of, and anticipated development and regulatory milestones and plans related to Adverum’s product candidates and preclinical and clinical studies, and the commercial potential of its product candidates, all of which are based on certain assumptions made by Adverum on current conditions, expected future developments and other factors Adverum believes are appropriate in the circumstances. Adverum may not consummate any plans or product or clinical development goals in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations or projections disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risk that Adverum’s resources will not be sufficient for Adverum to conduct or continue planned development programs and planned clinical trials, the risk of a delay in the enrollment of patients in Adverum’s clinical studies or in the manufacturing of products to be used in such clinical studies, the risk that Adverum will not be able to successfully develop or commercialize any of its product candidates and the risk that Adverum will be delayed in receiving or fail to receive required regulatory approvals. Risks and uncertainties facing Adverum are described more fully in Adverum’s periodic reports filed with the
|ADVERUM BIOTECHNOLOGIES, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|December 31,||December 31,|
|Cash, cash equivalents and marketable securities||$||70,519||$||222,170|
|Receivable from collaborative partner||-||886|
|Prepaid expenses and other current assets||3,256||2,218|
|Total current assets||193,741||225,274|
|Property and equipment, net||3,024||4,169|
|Deposits and other long-term assets||140||140|
|LIABILITIES AND STOCKHOLDERS' EQUITY|
|Accounts payable and accrued liabilities||$||8,695||$||7,950|
|Current portion of deferred rent||129||96|
|Current portion of deferred revenue||1,850||1,850|
|Total current liabilities||10,674||9,896|
|Deferred rent, less current portion||222||352|
|Deferred revenue, less current portion||5,250||7,099|
|Deferred tax liability||1,250||1,250|
|Other non-current liabilities||481||386|
|Total liabilities and stockholders’ equity||$||201,905||$||234,583|
|ADVERUM BIOTECHNOLOGIES, INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(In thousands, except per share amounts)|
|Three Months Ended December 31,||Year Ended December 31,|
|Collaboration and license revenue||$||461||$||488||$||1,849||$||1,455|
|Research and development||12,014||7,898||39,839||31,670|
|General and administrative||4,042||4,777||20,857||24,355|
|Impairment of goodwill and intangible assets||-||11,200||-||60,714|
|Total operating expenses||16,056||23,875||60,696||116,739|
|Other income (expense), net||806||218||2,700||762|
|Net loss before income tax benefit||(14,789||)||(23,169||)||(56,147||)||(114,522||)|
|Income tax benefit||-||775||-||775|
|Net loss attributable to|
|Net loss per share attributable to common|
|stockholders, basic and diluted||$||(0.32||)||$||(0.54||)||$||(1.29||)||$||(3.14||)|
|Weighted-average common shares outstanding,|
|outstanding, basic and diluted||46,069||41,758||43,661||36,246|
Contact for Adverum:
Leone PattersonChief Financial Officer 650-665-7222 email@example.com
Source: Adverum Biotechnologies, Inc.