Financial Status - Adverum held approximately $174 million in cash, cash equivalents, and short-term investments as of June 2024, which is expected to fund operations into late 2025[8] Ixo-vec Clinical Development (Wet AMD) - Ixo-vec demonstrates a potential best-in-class product profile for wet AMD treatment, aiming for a one-time IVT injection to turn the eye into an anti-VEGF biofactory[10] - In the LUNA trial, 76% of patients at the 6E10 dose remained injection-free at 26 weeks, and 88% preferred Ixo-vec to prior injections[10] - The LUNA Phase 2 study results show a 90% to 95% reduction in anti-VEGF treatment burden across both the 6E10 and 2E11 Ixo-vec doses, aligning with OPTIC study results[70] - In the LUNA trial, 76% to 83% of patients were injection-free across both Ixo-vec doses, consistent with the OPTIC trial[73] - Among patients with ≤ 6 prior injections, 90% were injection-free at Week 26 in the LUNA study[78] - 100% of patients previously receiving Faricimab (Vabysmo) remained injection-free across both Ixo-vec doses in the LUNA study[81] - Both Ixo-vec doses in the LUNA study maintained visual and anatomic outcomes through 26 weeks[84] - Local corticosteroid prophylaxis was effective in minimizing inflammation, with 91% of participants having no or minimal inflammation at any study visit through Week 26[105] Market Opportunity - The global wet AMD market is large and growing, with approximately 1.5 million patients in the U S and 20 million worldwide, and about 200,000 new cases annually[14] - The global sales for wet AMD are projected to be around $9 billion by 2025[17] - A 1% market share for a gene therapy like Ixo-vec could translate to approximately $260 million to $440 million in sales, assuming a 3-5 year benefit[18]

Financial Performance - As of March 31, 2025, the company had an accumulated deficit of $1.1 billion and has not generated positive cash flow or net income since inception [76]. - The operating loss for the three months ended March 31, 2025, was $48.2 million, an increase of $19.0 million from the operating loss of $29.2 million in the same period of 2024 [89]. - Net loss for the three months ended March 31, 2025, was $47.0 million, compared to a net loss of $27.1 million for the same period in 2024, reflecting an increase of $19.9 million [89]. - The company expects to continue incurring net losses and operating cash outflows for at least the next several years as it develops its product candidates [97]. - General and administrative expenses increased by $5.7 million to $19.5 million for the three months ended March 31, 2025, from $13.8 million in the same period of 2024 [92]. - Other income, net decreased by $0.9 million to $1.2 million for the three months ended March 31, 2025, from $2.1 million for the same period in 2024 [93]. Cash Flow and Funding - Cash and cash equivalents decreased to $83.1 million as of March 31, 2025, down from $125.7 million as of December 31, 2024 [94]. - Net cash used in operating activities was $42.8 million for the three months ended March 31, 2025, compared to $23.2 million for the same period in 2024 [105]. - The company plans to raise substantial additional funding to finance operations through various means, including public or private equity or debt financings [98]. - For the three months ended March 31, 2024, the net cash used in operating activities was $23.2 million, primarily due to a net loss of $27.1 million from ongoing research and development activities [107]. - The net cash provided by financing activities for the three months ended March 31, 2024 was primarily $119.7 million from the issuance of common stock and Pre-Funded Warrants in Private Placements [109]. - Additionally, $0.1 million was generated from the exercise of stock options during the same period [109]. Research and Development - The company announced top-line 52-week results from the LUNA Phase 2 clinical trial, showing a reduction in annualized anti-VEGF injection rates of 88% for the 6E10 dose and 92% for the 2E11 dose [72]. - Nearly 50% of patients treated with Ixo-vec were injection-free through four years following treatment in the OPTIC trial [71]. - The company plans to initiate the AQUARIUS Phase 3 trial in the second half of 2025, following the initiation of the ARTEMIS trial in February 2025 [73]. - The LUNA trial enrolled 60 subjects, randomized between two doses, and assessed visual and anatomic outcomes at 52 weeks [72]. - The FDA granted Ixo-vec Fast Track designation in September 2018 and Regenerative Medicine Advanced Therapy designation in August 2024 [74]. - Research and development expenses include personnel-related costs, stock-based compensation, and external contract research expenses [81]. - Research and development expenses increased by $13.3 million to $28.7 million for the three months ended March 31, 2025, compared to $15.4 million for the same period in 2024 [90]. Operational Status - The company has not generated any revenue from product sales to date, relying on research and collaboration arrangements for revenue [80]. - The company currently has no operational clinical or commercial manufacturing facilities, relying on third-party contractors for clinical manufacturing [78].

Financial Performance - Cash, cash equivalents, and short-term investments were $125.7 million as of December 31, 2024, down from $153.2 million as of September 30, 2024, and up from $96.5 million as of December 31, 2023[4] - Net loss for the three months ended December 31, 2024, was $40.9 million, or $1.96 per share, compared to a net loss of $24.5 million, or $2.42 per share, for the same period in 2023[8] - Total stockholders' equity increased to $70.7 million as of December 31, 2024, compared to $67.2 million as of December 31, 2023[12] Expenses - Research and development expenses increased to $24.1 million for the three months ended December 31, 2024, compared to $15.3 million for the same period in 2023[8] - General and administrative expenses rose to $18.5 million for the three months ended December 31, 2024, compared to $11.7 million for the same period in 2023[8] Clinical Trials - The ARTEMIS trial, the first registrational intravitreal gene therapy trial for wet AMD, will evaluate Ixo-vec in approximately 284 patients[3] - The primary endpoint of the ARTEMIS trial is the mean change from baseline of best corrected visual acuity (BCVA) at one year with a non-inferiority margin of -4.5 letters[3] - The company plans to initiate the global AQUARIUS Phase 3 trial in the second half of 2025[5] - Results from the Phase 2 LUNA trial support the selection of the 6E10 dose, demonstrating potential best-in-class efficacy and favorable long-term safety[5] Regulatory Designations - Ixo-vec has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA for the treatment of wet AMD[6]

Core Insights - Adverum Biotechnologies reported strong progress in its gene therapy pipeline, particularly with Ixo-vec for wet AMD, and initiated its first pivotal trial, ARTEMIS [2][4][5] - The company highlighted the potential of Ixo-vec to provide long-term vision preservation with a single injection, reducing the need for frequent anti-VEGF treatments [2][7][10] Financial Performance - For the three months ended March 31, 2025, Adverum reported a net loss of $47.0 million, or $2.25 per share, compared to a net loss of $27.1 million, or $1.65 per share for the same period in 2024 [9][15] - Research and development expenses increased to $28.7 million from $15.4 million year-over-year, driven by higher clinical trial costs and material production [9][15] - General and administrative expenses rose to $19.5 million from $13.8 million, attributed to higher facility and professional service costs [9][15] Pipeline Highlights - The ARTEMIS trial is the first registrational intravitreal gene therapy trial for wet AMD, enrolling approximately 284 patients [5][6] - New data presented at ARVO 2025 demonstrated the first human cell-level mapping of Ixo-vec transduction and aflibercept mRNA expression, supporting its potential for lifelong vision preservation [3][4] - The company plans to initiate the global AQUARIUS Phase 3 trial in the second half of 2025 and present two-year follow-up data from the LUNA study in Q4 2025 [9][10] Product Overview - Ixo-vec (ixoberogene soroparvovec) is designed for a one-time intravitreal injection, aiming to provide long-term efficacy and improve patient compliance compared to current treatment paradigms [7][10] - The product utilizes a proprietary AAV.7m8 vector, which has shown a five-fold increase in transduction efficiency compared to natural AAV2 [9]

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud and unlawful business practices involving Adverum Biotechnologies, Inc. following the company's announcement of the need to restate its financial statements due to accounting errors [1][3]. Group 1: Investigation Details - Pomerantz LLP is representing investors of Adverum and is looking into claims of securities fraud or other unlawful practices by the company and its officers or directors [1]. - The investigation was prompted by Adverum's disclosure that certain previously issued financial statements should no longer be relied upon due to non-cash errors related to tenant improvement allowances [3]. Group 2: Financial Impact - On March 31, 2025, Adverum announced it would restate its financial statements for the years ended December 31, 2022 and 2023, as well as unaudited quarterly financial information for 2023 and 2024 [3]. - Following the announcement, Adverum's stock price dropped by $0.54 per share, representing a decline of 12.36%, closing at $3.83 per share on April 1, 2025 [3]. Group 3: Company Background - Pomerantz LLP is recognized as a leading firm in corporate, securities, and antitrust class litigation, with a history of recovering significant damages for victims of securities fraud and corporate misconduct [4].

Core Points - Adverum Biotechnologies granted inducement awards to seven new employees, including non-qualified stock options for 32,620 shares and restricted stock units (RSUs) for 16,310 shares under its 2017 Inducement Plan [1][2] - The stock options have an exercise price of $3.33 per share, equal to the closing price on the grant date, and vest over four years [2] - The RSUs vest over three years, with one-third vesting on each anniversary of the applicable vesting commencement date [2] Company Overview - Adverum Biotechnologies is a clinical-stage company focused on gene therapy to preserve sight in prevalent ocular diseases, aiming to develop functional cures to restore vision and prevent blindness [3] - The company utilizes its proprietary intravitreal (IVT) platform to create durable, single-administration therapies, reducing the need for frequent ocular injections [3] - Adverum is evaluating its gene therapy candidate, ixoberogene soroparvovec (Ixo-vec), as a one-time IVT injection for patients with neovascular or wet age-related macular degeneration [3]

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Adverum Biotechnologies, Inc. following the company's announcement of the need to restate its financial statements due to accounting errors [1][3]. Group 1: Investigation Details - Pomerantz LLP is representing investors of Adverum and is looking into claims of securities fraud or other unlawful practices by the company and its officers/directors [1]. - Investors are encouraged to contact Pomerantz LLP for more information regarding the class action [2]. Group 2: Financial Disclosure - On March 31, 2025, Adverum disclosed that certain previously issued financial statements should no longer be relied upon due to non-cash errors related to tenant improvement allowances for an operating lease in North Carolina [3]. - The company announced it will restate its financial statements for the years ended December 31, 2022, and 2023, as well as for the quarterly periods in 2023 and 2024 [3]. - Following this announcement, Adverum's stock price dropped by $0.54, or 12.36%, closing at $3.83 per share on April 1, 2025 [3].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Adverum Biotechnologies, Inc. following the company's announcement of the need to restate its financial statements due to accounting errors [1][3]. Group 1: Investigation Details - Pomerantz LLP is representing investors of Adverum and is looking into claims of securities fraud or other unlawful practices by the company and its officers or directors [1]. - Investors are encouraged to contact Pomerantz LLP for more information regarding the class action [2]. Group 2: Financial Disclosure - On March 31, 2025, Adverum disclosed that certain previously issued financial statements should no longer be relied upon due to non-cash errors related to tenant improvement allowances for an operating lease in North Carolina [3]. - The company announced it will restate its financial statements for the years ended December 31, 2022, and 2023, as well as for the quarterly periods in 2023 and 2024 [3]. - Following this announcement, Adverum's stock price dropped by $0.54 per share, a decline of 12.36%, closing at $3.83 per share on April 1, 2025 [3].

Core Viewpoint - The Schall Law Firm is investigating Adverum Biotechnologies, Inc. for potential violations of securities laws related to misleading financial statements and disclosures [1][2]. Group 1: Investigation Details - The investigation centers on whether Adverum made false or misleading statements and failed to disclose critical information to investors [2]. - Adverum announced in an SEC filing on March 31, 2025, that certain previously issued financial statements should no longer be relied upon due to non-cash errors in accounting for tenant improvement allowances related to an operating lease in North Carolina [2]. - The company plans to restate its financial statements for the years ended December 31, 2022, and 2023, as well as unaudited quarterly financial information for 2023 and 2024 [2]. - Following this announcement, Adverum's shares dropped by over 12% on April 1, 2025 [2]. Group 2: Shareholder Rights - The Schall Law Firm encourages shareholders who suffered losses to participate in the investigation and offers free consultations regarding their rights [3]. - The firm specializes in securities class action lawsuits and shareholder rights litigation, representing investors globally [3].

Core Viewpoint - Adverum Biotechnologies is advancing its gene therapy candidate Ixo-vec for the treatment of wet age-related macular degeneration (AMD), with promising clinical trial results and a strong pipeline of upcoming studies aimed at redefining treatment standards for patients [2][3][6]. Financial Results - As of December 31, 2024, Adverum reported cash, cash equivalents, and short-term investments of $125.7 million, a decrease from $153.2 million as of September 30, 2024, but an increase from $96.5 million as of December 31, 2023 [8]. - Research and development expenses for Q4 2024 were $24.1 million, up from $15.3 million in Q4 2023, attributed to higher material production and preparation for Phase 3 trials [8]. - General and administrative expenses rose to $18.5 million in Q4 2024 from $11.7 million in Q4 2023, driven by increased consultant fees and a non-cash tenant improvement allowance [8]. - The net loss for Q4 2024 was $40.9 million, or $1.96 per share, compared to a net loss of $24.5 million, or $2.42 per share, for the same period in 2023 [8][11]. Pipeline Highlights - The ARTEMIS trial, the first registrational intravitreal gene therapy trial for wet AMD, has been initiated, evaluating Ixo-vec against aflibercept in approximately 284 patients [3]. - The primary endpoint of ARTEMIS is the mean change from baseline in best corrected visual acuity (BCVA) at one year, with a non-inferiority margin of -4.5 letters [3]. - Long-term data from the LUNA trial support the selected 6E10 dose for the pivotal program, showing potential best-in-class efficacy and favorable long-term safety [3][4]. Upcoming Milestones - The global AQUARIUS Phase 3 trial is expected to be initiated in the second half of 2025, with the presentation of two-year long-term follow-up data from the LUNA trial planned for Q4 2025 [8][9]. Product Overview - Ixo-vec, a gene therapy product candidate, is designed for a one-time intravitreal injection to treat wet AMD, aiming to provide long-term efficacy and reduce the burden of frequent anti-VEGF injections [6][7]. - The therapy has received Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA, as well as PRIME designation from the EMA [6]. Company Overview - Adverum Biotechnologies is focused on establishing gene therapy as a new standard of care for prevalent ocular diseases, with aspirations to develop functional cures to restore vision and prevent blindness [7].