-- Long-term durability beyond 15 months from single IVT injection with zero rescue injections in Cohort 1 --
-- Well tolerated across all Cohorts; encouraging early safety data from Cohort 4 --
-- OPTIC enrollment complete; planning to start pivotal trial in wet AMD mid-2021 --
-- Company to host conference call and webcast with Key Opinion Leader
Dr. Arshad Khananitoday at 1:30 pm PT / 4:30 pm ET --
REDWOOD CITY, Calif., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced positive new interim data from Cohorts 1-4 of the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their wet age-related macular degeneration (AMD). The company also reported recent business progress and financial results for the second quarter ended June 30, 2020.
For the first time, interim data from all four cohorts of the OPTIC trial (July 23, 2020 cutoff date), including preliminary safety data from Cohort 4, are presented. These data further demonstrate the transformative potential of ADVM-022 to greatly reduce the treatment burden for patients with wet AMD:
OPTIC Phase 1 Clinical Trial Data:
|Results Following a Single ADVM-022 Dose:||
||Cohort 3||Cohort 4|
|Median follow-up visit (weeks)||
6 x 10^11 vg/eye
2 x 10^11 vg/eye
2 x 10^11 vg/eye
6 x 10^11 vg/eye
Prophylactic steroid regimen
||13-day oral||13-day oral||6-week eye drops||6-week eye drops|
|Number of patients requiring anti-VEGF rescue injections||0/6 patients||3/6 patients||2/9 patients||0/9 patients|
|Total anti-VEGF rescue injections||0 injections||13 injections||5 injections||0 injections|
|Mean annualized anti-VEGF injection frequency before and after ADVM-0225||
|Reduction in mean annualized anti-VEGF injection frequency after ADVM-0225||100%||87%||N/A|
|Follow-up BCVA1and CRT2:|
|All Patients||All Patients||Rescue Free Patients
||Rescue Free Patients
|BCVA mean change from baseline (letters)||-3.2||-2.0||0||+4.0||+6.4||N/A|
|CRT mean change from baseline (mm)||-21.0 mm||-24.8 mm||-8.3 mm||-118.6 mm||-152.7 mm||N/A|
1 Best corrected visual acuity (BCVA)
2 Central retinal thickness (CRT)
3 All patients from Cohort 1 (n=6)
4 All patients from Cohort 2 (n=6) and Cohort 3 (n=9)
5 Annualized rate (Before) = (number of IVTs in 12 months prior to ADVM-022) / (days from the first IVT in the past 12 months to ADVM-022 / 365.25)
Annualized rate (After) = (number of aflibercept IVTs since ADVM-022) / (days from ADVM-022 to the last study follow-up / 365.25)
6 Fluorescein angiography of posterior pole
Arshad M. Khanani, M.D., M.A., managing partner and director of clinical research, Sierra Eye Associates, clinical associate professor of ophthalmology, University of Nevada, and top enrolling investigator in the OPTIC trial said, "In my experience the sustained anatomical treatment response following a single intravitreal injection of ADVM-022 is unprecedented, which is remarkable considering that the patients enrolled in OPTIC were difficult-to-treat and had previously required frequent injections to maintain their vision. The safety profile to date shows that ADVM-022 is well tolerated, and that the prophylactic steroid eye drop regimen has been effective at limiting early ocular inflammation. I believe that ADVM-022 has the potential to be a transformational gene therapy improving real-world outcomes for patients living with wet AMD."
Aaron Osborne, MBBS, chief medical officer of Adverum, added, "We have completed dosing patients in OPTIC, with 30 patients being followed up across four cohorts, and we look forward to presenting additional data from these cohorts by the end of this year. With the exciting data presented to date from OPTIC highlighting the potential of ADVM-022 to dramatically reduce treatment burden for patients with wet AMD, we plan to seek U.S. and international regulatory authorities' input as we progress towards initiating a pivotal clinical trial in mid-2021."
Laurent Fischer, M.D., chief executive officer of Adverum stated, "With these impressive OPTIC data, we are now poised to move our potentially transformative therapy into pivotal trials. ADVM-022, a novel gene therapy providing continuous delivery of aflibercept, has the potential to be a "one and done" IVT injection that would dramatically reduce the treatment burden for the millions of patients with wet AMD and DME worldwide. As we continue to see positive data from ongoing trials, we are preparing to accelerate our development and future commercial launch plans for ADVM-022. We continue to add industry-leading talent to our team and are beginning to expand our business operations and capabilities, including clinical, regulatory, manufacturing, and pre-commercial functions. It's an exciting time at Adverum, and I'm humbled by the dedication of the retina specialists, their patients and our employees for their tireless efforts to help patients with severe ocular diseases during a pandemic."
INFINITY Phase 2 Clinical Trial of ADVM-022 in Diabetic Macular Edema
ADVM-022 in wet AMD
ADVM-022 in DME
To date, Adverum has experienced limited impact from COVID-19 on its operations and ongoing clinical programs, including the OPTIC and INFINITY clinical trials. The company is continuing to monitor and attempt to address or limit the potential impacts of COVID-19 on its employees and operations, patient safety, patient enrollment, continued participation of patients already enrolled in the company's clinical studies, protocol compliance, data quality, and overall study integrity.
Financial Results for the Three Months Ended June 30, 2020
Conference Call and Webcast Information
Adverum will host a conference call and webcast today with Key Opinion Leader Dr. Arshad Khanani at 1:30 pm PT / 4:30 pm ET to present new data from the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD and provide an update on recent business progress. The live webcast will be accessible under Events and Presentations in the Investors section of the company's website. To participate in the conference call dial 1-855-327-6837 (domestic) or 1-631-891-4304 (international) and refer to the "Adverum Biotechnologies' Conference Call." It is recommended call participants dial in 15 minutes in advance. The archived webcast and slide presentation will be available on the Adverum website following the call and will be available for 30 days.
About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMD
This multi-center, open-label, Phase 1, dose-ranging trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. Patients received a high dose (6 x 10^11 vg/eye) of ADVM-022 in Cohort 1 (n=6) and Cohort 4 (n=9) and patients received a low dose (2 x 10^11 vg/eye) of ADVM-022 in Cohort 2 (n=6) and Cohort 3 (n=9). Patients in Cohorts 3 and 4 received six weeks of prophylactic steroid eye drops rather than 13 days of prophylactic oral steroids which were used in Cohorts 1 and 2. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as the need for anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.
About Adverum Biotechnologies
Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visit www.adverum.com.
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: the potential for ADVM-022 in treating patients with wet AMD and DME; Adverum's expectations that it will present additional data from all four cohorts of the OPTIC Phase 1 trial for ADVM-022 in wet AMD by the end of this year; Adverum's expectations as to its plans to advance ADVM-022 in wet AMD by initiating a pivotal trial mid-2021 and in DME by continuing to enroll patients in the INFINITY trial, including without limitation its expected enrollment numbers for the INFINITY trial; Adverum's expectations that it will present data from the INFINITY trial in the second half of 2021; Adverum's expectations that it will accelerate its development, manufacturing, and commercial launch plans for ADVM-022; and Adverum's expectations that its current cash position will fund its operations into 2022. All of these statements 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 preliminary or interim results of clinical trials to change as the clinical trial continues or in connection with the preparation and analysis of final 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 and INFINITY trials and vector production; the effects of the COVID-19 pandemic on the company's operations and on the company's ongoing clinical trials; and ability to fund operations through completion of the OPTIC and INFINITY trials and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverum's Form 10-Q filed with the SEC on August 10, 2020 under the heading "Risk Factors." All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Cherilyn Cecchini, M.D.
|Adverum Biotechnologies, Inc.|
|Consolidated Balance Sheets|
|June 20||December 31,|
|Cash and cash equivalents||$||27,359||$||65,897|
|Prepaid expenses and other current assets||2,542||9,835|
|Total current assets||282,681||175,870|
|Operating lease right-of-use assets||20,011||20,963|
|Property and equipment, net||27,466||24,884|
|Deposit and other long-term assets||19||11|
|Liabilities and stockholders' equity|
|Accrued expenses and other current liabilities||6,666||11,271|
|Lease liability, current portion||4,221||4,034|
|Total current liabilities||21,254||19,408|
|Lease liability, net of current portion||27,258||28,214|
|Other noncurrent liabilities||101||148|
|Additional paid-in capital||720,288||560,704|
|Accumulated other comprehensive loss||(598||)||(725||)|
|Total stockholders' equity||282,563||174,957|
|Total liabilities and stockholders' equity||$||331,176||$||222,727|
|(1) Derived from Adverum's annual audited consolidated financial statements.|
|Adverum Biotechnologies, Inc.|
|Consolidated Statements of Operations|
|(In thousands except per share data)|
|Three Months Ended
|Six Months Ended
|Research and development||$||19,177||$||8,970||$||33,928||$||19,101|
|General and administrative||10,598||7,132||19,638||12,708|
|Total operating expenses||29,775||16,102||53,566||31,809|
|Other income, net||575||1,148||1,460||2,366|
|Net loss per share — basic and diluted||$||0.36||$||(0.23||)||$||(0.68||)||$||(0.46||)|
|Weighted-average common shares outstanding - basic and diluted||80,229||63,740||77,010||63,429|
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