Adverum Biotechnologies Reports Recent Business Progress and Fourth Quarter 2019 Financial Results
-- Cohort 3 dosing complete and cohort 4 screening underway in OPTIC Phase 1 trial of ADVM-022 in wet AMD --
-- New OPTIC data to be presented in
-- Data from all four cohorts of OPTIC planned in 2020 --
-- Company to host conference call today at
“2020 is poised to be an exciting year of execution as we rapidly advance our development efforts for our lead programs ADVM-022 for wet AMD and for diabetic retinopathy,” said
- Completed patient dosing in cohort 3 (n=9, three-fold lower dose compared to cohort 1, 2 x 10^11 vg/eye) of the OPTIC Phase 1 clinical trial of ADVM-022 in wet age-related macular degeneration (wet AMD). Patients are being screened in cohort 4 (n=9, dose 6 x 10^11).
- In cohorts 3 and 4, patients are receiving prophylactic topical steroid eye drops for a total of 6 weeks instead of prophylactic oral steroids for 13 days used in cohorts 1 and 2.
- At the Angiogenesis, Exudation, and Degeneration 2020 Annual Meeting in
February 2020, presented new data from OPTIC cohorts 1 and 2. Results demonstrated that in treatment-experienced patients previously requiring frequent anti-VEGF injections to maintain vision, ADVM-022 demonstrated a robust efficacy signal and evidence of a dose response:
- Cohort 1 (6 x 10^11 vg/eye): 6 of 6 patients remained rescue-injection-free at a median follow up of 50 weeks, with 3 patients at 52 weeks.
- Cohort 2 (three-fold lower dose 2 x 10^11 vg/eye): 4 of 6 patients remained rescue injection free at 24 weeks.
- In both cohorts combined, 10 of 12 (83%) patients remained rescue injection free. For these patients:
- Vision was generally maintained as demonstrated by stable mean best corrected visual acuity (BCVA) compared to baseline.
- Retinal anatomy improvements were achieved and maintained as demonstrated by mean central subfield thickness (CST) compared to baseline.
- ADVM-022 continued to demonstrate a favorable safety profile and be well tolerated.
- Raised approximately
$140.8 millionin net proceeds from an underwritten public offering in February 2020
- Moved to the company’s new
Redwood Citycorporate headquarters in January 2020, which includes approximately 81,000 square feet of office, laboratory, and manufacturing space to advance the development of Adverum’s novel gene therapies
Angela Thedingaas chief technology officer. Ms. Thedingapreviously served as Adverum’s vice president, program management, and has deep experience transitioning and expanding viral vector gene therapy manufacturing and analytical capabilities from academic to commercial scale, which will be important as ADVM-022 advances in development toward potential commercialization.
Second Quarter of 2020:
- Present new clinical data from the OPTIC trial in
- Submit an investigational new drug application for ADVM-022 in diabetic retinopathy, a key VEGF-driven cause of vision loss among working-age adults
Second Half of 2020:
- Present clinical data from cohorts 1-4 of the OPTIC trial
- Begin enrolling patients in a planned Phase 1/2 clinical trial for ADVM-022 in diabetic retinopathy
Financial Results for the Three Months Ended
- Cash, cash equivalents and short-term investments were
$166.0 millionas of December 31, 2019, of which $25.8 millionin net proceeds was raised through an at-the-market program during this quarter, compared to $162.0 millionat September 30, 2019and $205.1 millionas of December 31, 2018. Adverum expects this year-end cash position, together with approximately $140.8 millionin net proceeds raised in February 2020, to fund operations into 2022.
- Research and development expenses were
$11.4 millionfor the three months ended December 31, 2019, compared to $11.6 millionfor the same period in 2018.
- General and administrative expenses were
$8.3 millionfor the three months ended December 31, 2019, compared to $5.2 millionfor the same period in 2018. General and administrative expenses increased primarily due to higher consultant and professional service expenses and increased facilities costs related to the company’s new facility, partially offset by lower stock-based compensation expenses.
- Net loss was
$18.9 million, or $0.29per basic and diluted share, for the three months ended December 31, 2019, compared to $15.7 million, or $0.25per basic and diluted share, for the same period in 2018.
- Shares of common stock outstanding were 79.7 million as of
February 28, 2020.
Conference Call Information
Individuals can participate in today’s conference call at
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 advance ADVM-022 in 2020, all other statements under the caption “Future Plans”, Adverum’s expectations that its current cash will fund operations into 2022, and the potential benefits of ADVM-022, 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 achieve any of these in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations 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 risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022; obtaining regulatory approval for gene therapy product candidates; enrolling patients in clinical trials; reliance on third parties for conducting the OPTIC trial and vector production; and ability to fund operations through completion of the OPTIC trial and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverum’s Form 10-K filed with the
Investor and Media Inquiries:
|Consolidated Balance Sheets|
|Cash and cash equivalents||$||65,897||$||154,949|
|Prepaid expenses and other current assets||9,835||3,675|
|Total current assets||175,870||208,754|
|Operating lease right-of-use asset||20,963||-|
|Property and equipment, net||24,884||3,586|
|Deposit and other long-term assets||11||156|
|Liabilities and stockholders' equity|
|Accrued expenses and other current liabilities||11,271||8,784|
|Lease liability, current portion||4,034||-|
|Deferred rent, current portion||-||228|
|Total current liabilities||19,408||10,719|
|Deferred rent, net of current portion||-||1,366|
|Lease liability, net of current portion||28,214||-|
|Other noncurrent liabilities||148||243|
|Additional paid-in capital||560,704||522,503|
|Accumulated other comprehensive loss||(725||)||(799||)|
|Total stockholders' equity||174,957||201,167|
|Total liabilities and stockholders' equity||$||222,727||$||213,495|
|Consolidated Statements of Operations and Comprehensive Loss|
|(In thousands except per share data)|
|Three Months Ended
|Collaboration and license revenue||$||-||$||70||$||250||$||1,612|
|Research and development||11,374||11,642||40,419||50,133|
|General and administrative||8,279||5,187||28,376||24,560|
|Impairment of goodwill and intangible asset||-||-||-||5,000|
|Total operating expenses||19,653||16,829||68,795||79,693|
|Other income, net||728||1,100||4,059||4,204|
|Net loss before income taxes||(18,925||)||(15,659||)||(64,486||)||(73,877||)|
|Income tax benefit||-||-||-||1,250|
|Other comprehensive income|
|Net unrealized gain on marketable securities||6||39||33||168|
|Foreign currency translation adjustment||14||17||41||(4||)|
|Net loss per share — basic and diluted||$||(0.29||)||$||(0.25||)||$||(1.01||)||$||(1.18||)|
|Weighted-average common shares outstanding - basic and diluted||65,104||62,915||64,102||61,375|
Source: Adverum Biotechnologies, Inc.