Core Viewpoint - Adverum Biotechnologies is advancing its gene therapy candidate Ixo-vec for the treatment of wet age-related macular degeneration (AMD), with significant clinical data updates and a focus on commercialization strategies. Corporate Highlights - Jason Mitchell has been appointed as Chief Commercial Officer, bringing over 20 years of experience in retinal disease, including the launch of SYFOVRE® [3] - The company has $153.2 million in cash and equivalents, expected to fund operations into late 2025 [4][9] - The planned initiation of the pivotal Phase 3 trial is set for the first half of 2025 [4] Upcoming Anticipated Milestones - In Q4 2024, Adverum will present LUNA 52-week data, OPTIC 4-year clinical data, and updates on the Phase 3 trial design [4] - The initiation of the Phase 3 trial is planned for the first half of 2025 [4] Financial Results - For Q3 2024, cash and cash equivalents increased to $153.2 million from $96.5 million at the end of 2023 [4][9] - Research and development expenses were $20.4 million, slightly down from $20.7 million in Q3 2023 [4][10] - General and administrative expenses decreased to $9.8 million from $13.8 million in the same period last year [4][10] - The net loss for Q3 2024 was $27.1 million, or $1.30 per share, compared to a net loss of $32.9 million, or $3.26 per share, in Q3 2023 [4][10] About Wet AMD - Wet AMD is a leading cause of blindness in individuals over 65, with approximately 20 million affected globally [5] - The incidence of new cases is expected to rise significantly as populations age, with AMD projected to impact 288 million people by 2040 [5] About Ixo-vec - Ixo-vec is a gene therapy product candidate designed for a one-time intravitreal injection to treat wet AMD, aiming to reduce the need for frequent anti-VEGF injections [6] - The therapy has received Fast Track and RMAT designations from the FDA, as well as PRIME designation from the European Medicines Agency [6]

Core Insights - Adverum Biotechnologies has appointed Jason L. Mitchell as chief commercial officer, who has over 20 years of experience in the commercial sector, particularly in retinal diseases [1][2] - The company is advancing its gene therapy candidate, ixoberogene soroparvovec (Ixo-vec), which is currently in Phase 2 clinical trials and aims to establish a new standard of care for wet age-related macular degeneration (wet AMD) [1][5] - Adverum plans to announce 52-week data from the LUNA trial and pivotal program details in Q4 2024, with the initiation of the pivotal program expected in the first half of 2025 [1][2] Company Overview - Adverum Biotechnologies is a clinical-stage company focused on developing gene therapies for prevalent ocular diseases, aiming to restore vision and prevent blindness [4][5] - The company utilizes a proprietary intravitreal (IVT) platform to create durable, single-administration therapies that reduce the need for frequent ocular injections [5] Leadership Background - Jason L. Mitchell previously led the successful launch of SYFOVRE® for geographic atrophy and has held significant roles in sales, marketing, and operations at various pharmaceutical companies [2][3] - His expertise is expected to enhance Adverum's strategy for introducing Ixo-vec as a leading gene therapy for wet AMD [2]

Core Viewpoint - Adverum Biotechnologies (ADVM) has been upgraded to a Zacks Rank 1 (Strong Buy) due to an upward trend in earnings estimates, which is a significant factor influencing stock prices [1][2]. Earnings Outlook - The Zacks rating upgrade reflects a positive earnings outlook for Adverum Biotechnologies, which is expected to positively impact its stock price [2]. - For the fiscal year ending December 2024, Adverum is projected to earn -$4.64 per share, representing a 60% change from the previous year's reported number [5]. Earnings Estimate Revisions - Analysts have increased their earnings estimates for Adverum Biotechnologies, with the Zacks Consensus Estimate rising by 8.4% over the past three months [5]. - The correlation between earnings estimate revisions and near-term stock movements is strong, indicating that tracking these revisions can be beneficial for investment decisions [4]. Zacks Rank System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with Zacks Rank 1 stocks historically generating an average annual return of +25% since 1988 [4]. - Adverum's upgrade to Zacks Rank 1 places it in the top 5% of Zacks-covered stocks, suggesting a strong potential for market-beating returns in the near term [7].

Company Overview - Adverum Biotechnologies, Inc. is a clinical-stage company focused on gene therapy for prevalent ocular diseases aiming to establish a new standard of care [2] - The company is developing a one-time, intravitreal injection therapy, ixoberogene soroparvovec (Ixo-vec), for patients with neovascular or wet age-related macular degeneration [2] - Adverum's goal is to create durable therapies that eliminate the need for frequent ocular injections, thereby transforming the treatment landscape for debilitating ocular diseases [2] Upcoming Events - Adverum will present at the H.C. Wainwright 26th Annual Global Investment Conference on September 10th at 3:30 p.m. ET [1] - The company will also participate in Chardan's 8th Annual Genetic Medicines Conference on October 1st at 8:00 a.m. ET [1] - Additionally, Adverum will be featured in the UBS Virtual Ophthalmology Day on October 2nd at 10:30 a.m. ET [1] Access to Information - On-demand webcasts of the presentations will be available under the Events and Presentations section on Adverum's website [1] - Each webcast will be accessible for at least 30 days following the events [1]

Core Viewpoint - Adverum Biotechnologies (ADVM) shows potential for significant upside based on Wall Street analysts' short-term price targets, with a mean estimate of $30 indicating a potential increase of 332.3% from the current price of $6.94 [1] Price Targets and Analyst Consensus - The mean estimate consists of seven short-term price targets with a standard deviation of $11.02, indicating variability among analysts [1] - The lowest price target of $12 suggests a 72.9% increase, while the highest target of $40 implies a surge of 476.4% [1] - A low standard deviation indicates a high degree of agreement among analysts regarding the stock's price movement direction [4] Earnings Estimates and Market Sentiment - Analysts have shown increasing optimism about ADVM's earnings prospects, with a 15.6% increase in the Zacks Consensus Estimate for the current year, supported by six upward revisions and no negative revisions [5] - ADVM holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, suggesting strong potential for near-term upside [5] Caution on Price Targets - While consensus price targets can provide insights, they should not be the sole basis for investment decisions due to historical inaccuracies in predicting actual stock price movements [3][4] - Analysts may set overly optimistic price targets influenced by business relationships, which can lead to inflated expectations [3]

Financial Position - As of June 30, 2024, the company had an accumulated deficit of $963.0 million and expects to incur substantial expenses and continuing losses in the foreseeable future [71]. - The accumulated deficit as of June 30, 2024, was $963.0 million, with cash, cash equivalents, and short-term investments totaling $173.8 million [99]. - The company has not generated positive cash flow or net income from operations since inception and may need substantial additional funding in the future [74]. - The company expects existing cash and cash equivalents to fund operations into late 2025, but may need to raise additional funds sooner due to various risks [99]. Revenue Generation - The company has not generated any revenue from product sales to date, relying instead on research, collaboration, and license arrangements with strategic partners [77]. - License revenue for the six months ended June 30, 2023, was $3.6 million, primarily from a milestone payment, while no license revenue was recorded for the same period in 2024 [89]. Clinical Trials and Development - The LUNA Phase 2 trial of Ixo-vec was fully enrolled with 60 subjects, and interim analysis indicated maintenance of visual and anatomic outcomes [68]. - Ixo-vec has received multiple regulatory designations, including Fast Track designation from the FDA and Priority Medicines designation from the EMA [69]. - The company plans to initiate a Phase 3 clinical trial of Ixo-vec in wet AMD in the first half of 2025 [68]. - Ixo-vec is designed to provide long-term durable therapeutic levels of aflibercept with a single in-office injection, targeting wet AMD [63]. - The company anticipates needing to raise substantial additional capital for the development and commercialization of product candidates, influenced by various factors including clinical trial outcomes and regulatory approvals [101]. - The company may delay or terminate development programs if unable to raise additional funds when needed [101]. Financial Performance - The net loss for the three months ended June 30, 2024, was $18.5 million, a decrease of $13.0 million compared to a net loss of $31.5 million for the same period in 2023 [88]. - The net loss for the six months ended June 30, 2024, was $43.3 million, compared to a net loss of $60.6 million for the same period in 2023 [103][104]. - Research and development expenses decreased by $3.5 million to $17.1 million for the three months ended June 30, 2024, from $20.6 million for the same period in 2023, primarily due to a reduction in facilities expenses [90]. - Research and development expenses for the six months ended June 30, 2024, decreased by $9.2 million to $32.5 million from $41.7 million for the same period in 2023, due to various cost reductions [91]. - General and administrative expenses decreased by $8.7 million to $3.8 million for the three months ended June 30, 2024, from $12.5 million for the same period in 2023, largely driven by a cumulative catch-up adjustment of sublease income [93]. - Other income, net increased by $0.8 million to $2.4 million for the three months ended June 30, 2024, from $1.6 million for the same period in 2023, primarily due to higher average invested balances [95]. Cash Flow and Financing - Net cash used in operating activities for the six months ended June 30, 2024, was $43.1 million, a decrease from $45.7 million in the same period of 2023 [102][103][104]. - Net cash provided by financing activities for the six months ended June 30, 2024, was $119.8 million, significantly higher than $0.2 million in the same period of 2023 [102][106][107]. - The company reported a net cash increase of $52.3 million for the six months ended June 30, 2024, compared to an increase of $42.9 million in the same period of 2023 [102]. - Net cash used in investing activities for the six months ended June 30, 2024, was $24.4 million, a decrease from $88.6 million provided in the same period of 2023 [102][105]. Operational Aspects - The company currently has no operational clinical or commercial manufacturing facilities and relies on third-party vendors for manufacturing [73]. - The company has a significant focus on research and development, with expenses primarily related to personnel, consulting, and clinical study materials [78]. Risk Factors - The company faces risks related to the emergence of competing technologies and adverse market developments [101]. Internal Controls - The evaluation of disclosure controls and procedures concluded that they were effective as of June 30, 2024 [110][111]. - There were no changes in internal control over financial reporting that materially affected the company during the most recent fiscal quarter [113].

```markdown [Overview and Key Highlights](index=1&type=section&id=Overview%20and%20Key%20Highlights) Adverum announced Q2 2024 results, selecting Ixo-vec's 6E10 dose for Phase 3 trials and reporting **$173.8 million** in cash - The 6E10 dose of Ixo-vec has been selected for Phase 3 pivotal trials following promising interim results[1](index=1&type=chunk) - The LUNA 26-week interim analysis demonstrated a potential best-in-class product profile for Ixo-vec, with similar efficacy and a more favorable safety profile compared to the earlier OPTIC study[1](index=1&type=chunk)[2](index=2&type=chunk) - The company holds **$173.8 million** in cash, cash equivalents, and short-term investments, which is expected to fund operations into late 2025[1](index=1&type=chunk)[8](index=8&type=chunk) [Ixo-vec Program Highlights](index=1&type=section&id=Ixo-vec%20Program%20Highlights) Ixo-vec achieved key milestones, including positive LUNA Phase 2 data leading to 6E10 dose selection for Phase 3, and FDA RMAT designation LUNA 26-Week Interim Analysis Highlights (6E10 dose) | Metric | Result | | :--- | :--- | | Injection-Free Patients | **76%** | | Safety Profile | Improved inflammatory profile compared to OPTIC | | Patient Preference (difluprednate-alone regimen) | **100%** would prefer Ixo-vec over prior treatments | - The U.S. FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Ixo-vec for treating wet AMD, recognizing its transformative potential and offering benefits like intensive FDA guidance and potential priority review[4](index=4&type=chunk) [Corporate Updates and Outlook](index=2&type=section&id=Corporate%20Updates%20and%20Outlook) Adverum strengthened leadership with new appointments and outlined a roadmap for upcoming milestones, including Phase 3 trial initiation in H1 2025 - In June 2024, Dr. Rabia Gurses Ozden was appointed Chief Medical Officer, and Dr. Szilárd Kiss joined the Board of Directors[5](index=5&type=chunk)[6](index=6&type=chunk) Upcoming Anticipated Milestones | Timeline | Milestone | | :--- | :--- | | 2H 2024 | Continued FDA and EMA regulatory interactions | | 4Q 2024 | LUNA 9-month analysis clinical data | | 4Q 2024 | Phase 3 pivotal trial design update | | 1Q 2025 | LUNA 52-week analysis clinical data | | H1 2025 | Planned initiation of Phase 3 trial | [Financial Results](index=3&type=section&id=Financial%20Results) Adverum reported a Q2 2024 net loss of **$18.5 million**, reduced from Q2 2023 due to decreased expenses, with cash increasing to **$173.8 million** [Key Financial Metrics (Q2 2024)](index=3&type=section&id=Key%20Financial%20Metrics%20(Q2%202024)) In Q2 2024, Adverum's cash and investments grew to **$173.8 million**, with R&D and G&A expenses decreasing, improving net loss per share - Cash, cash equivalents, and short-term investments stood at **$173.8 million** as of June 30, 2024, compared to **$96.5 million** at the end of 2023[8](index=8&type=chunk) Operating Expenses (Three months ended June 30) | Expense Category | 2024 ($ millions) | 2023 ($ millions) | | :--- | :--- | :--- | | Research & Development | $17.1 | $20.6 | | General & Administrative | $3.8 | $12.5 | Net Loss Summary (Three months ended June 30) | Metric | 2024 ($ millions) | 2023 ($ millions) | | :--- | :--- | :--- | | Net Loss | $18.5 | $31.5 | | Net Loss per Share | $0.89 | $3.13 | [Financial Statements](index=5&type=section&id=Financial%20Statements) The report includes selected data from the Consolidated Balance Sheet and Statements of Operations, providing a snapshot of financial position Selected Consolidated Balance Sheet Data | Metric | June 30, 2024 (Unaudited, $ thousands) | Dec 31, 2023 ($ thousands) | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | $173,827 | $96,526 | | Total assets | $253,803 | $173,010 | | Total stockholders' equity | $168,029 | $83,469 | Condensed Consolidated Statements of Operations (Three months ended June 30) | Metric | 2024 (Unaudited, $ thousands) | 2023 ($ thousands) | | :--- | :--- | :--- | | Total operating expenses | $20,882 | $33,065 | | Operating loss | $(20,882) | $(33,065) | | Net loss | $(18,476) | $(31,510) | [Company and Product Information](index=3&type=section&id=Company%20and%20Product%20Information) This section provides background on wet AMD, the target indication for Ixo-vec, detailing the disease, technology, and regulatory designations [About Wet Age-Related Macular Degeneration (AMD)](index=3&type=section&id=About%20Wet%20Age-Related%20Macular%20Degeneration%20(AMD)) Wet AMD is a leading cause of blindness globally, affecting **20 million** people, with current treatments posing a burden gene therapy aims to alleviate - Wet AMD is a leading cause of blindness in people over 65, with about **20 million** individuals affected worldwide[10](index=10&type=chunk) The standard treatment requires frequent, lifelong injections into the eye [About Ixo-vec in Wet AMD](index=3&type=section&id=About%20Ixo-vec%20in%20Wet%20AMD) Ixo-vec is Adverum's clinical-stage gene therapy for wet AMD, a one-time IVT injection for long-term efficacy, with multiple expedited regulatory designations - Ixo-vec is a one-time intravitreal (IVT) injection gene therapy designed to deliver long-term efficacy by carrying an aflibercept coding sequence via a proprietary AAV.7m8 vector capsid[11](index=11&type=chunk) - The therapy has received multiple designations to expedite its development, including Fast Track and RMAT from the FDA, PRIME from the EMA, and the Innovation Passport from the UK's MHRA[11](index=11&type=chunk)[12](index=12&type=chunk) ```

Group 1 - Adverum Biotechnologies is a clinical-stage company focused on gene therapy for prevalent ocular diseases, aiming to establish it as a new standard of care [2] - The company is developing a gene therapy candidate, ixoberogene soroparvovec (Ixo-vec), for a one-time intravitreal injection to treat neovascular or wet age-related macular degeneration [2] - Adverum's proprietary intravitreal platform is designed to provide durable therapies that eliminate the need for frequent ocular injections, thereby transforming the treatment paradigm for ocular diseases [2] Group 2 - Laurent Fischer, M.D., the CEO of Adverum, will participate in a fireside chat at the H.C. Wainwright 4th Annual Ophthalmology Virtual Conference on August 15, 2024 [1] - The webcast of the fireside chat will be accessible on Adverum's website, with a replay available for at least 30 days post-presentation [1]

Group 1 - Adverum Biotechnologies is a clinical-stage company focused on gene therapy for prevalent ocular diseases, aiming to establish it as a new standard of care [2] - The company is developing a gene therapy candidate, ixoberogene soroparvovec (Ixo-vec), for a one-time intravitreal injection to treat neovascular or wet age-related macular degeneration [2] - Adverum's proprietary intravitreal platform is designed to provide durable therapies that eliminate the need for frequent ocular injections, thereby transforming the treatment paradigm for ocular diseases [2] Group 2 - Laurent Fischer, M.D., the CEO of Adverum, will participate in a fireside chat at the H.C. Wainwright 4th Annual Ophthalmology Virtual Conference on August 15, 2024 [1] - The webcast of the fireside chat will be accessible on Adverum's website, with a replay available for at least 30 days post-presentation [1]

REDWOOD CITY, Calif., Aug. 01, 2024 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (NASDAQ:ADVM), a clinical-stage company pioneering the use of gene therapy as a new standard of care for highly prevalent ocular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Ixo-vec, its clinical-stage gene therapy product candidate, for the treatment of wet AMD. "The RMAT designation is based on the clinical data from I ...