Adverum Biotechnologies(ADVM) - 2025 Q2 - Quarterly Results

Management Remarks The CEO highlights excellent progress and ahead-of-schedule enrollment in the ARTEMIS Phase 3 trial for Ixo-vec, driven by strong interest from retina specialists - Enrollment in the ARTEMIS Phase 3 trial is progressing well ahead of schedule, with patient and specialist interest surpassing projections[3] - A recent survey of nearly 1,000 retina specialists indicated that almost 50% view gene therapy as the most exciting advancement in the wet AMD field, far surpassing other treatment modalities like TKIs[4] - The company is engaged in active and ongoing partnering discussions, supported by the clinical progress of Ixo-vec[3] - Two-year follow-up data from the LUNA Phase 2 study is expected to be presented later in the year, aiming to reinforce the long-term safety and efficacy profile of Ixo-vec[5] Pipeline and Corporate Highlights Adverum provided key updates on its Ixo-vec program, including accelerated ARTEMIS enrollment, LUNA and AQUARIUS study plans, and a $10 million private placement Ixo-vec Program Update ARTEMIS Phase 3 trial enrollment is ahead of schedule, with completion expected in Q1 2026 and topline data in H1 2027, alongside planned AQUARIUS initiation and LUNA data presentation - ARTEMIS Phase 3 trial enrollment is ahead of schedule, with completion expected in Q1 2026 and topline data anticipated in H1 2027[7][8] - The AQUARIUS Phase 3 study is expected to be initiated in Q4 2025, contingent on securing necessary funding[11] - Two-year long-term follow-up data from the LUNA Phase 2 study is planned for presentation in Q4 2025[7][11] Private Placement with Frazier Life Sciences Adverum secured approximately $10 million through a PIPE financing from Frazier Life Sciences, involving common stock and pre-funded warrants, expected to close by August 12, 2025 PIPE Financing Details | Metric | Value | | :--- | :--- | | Investor | Frazier Life Sciences | | Amount | ~$10 million | | Type | Private Investment in Public Equity (PIPE) | | Price per Share | $2.24 | | Price per Pre-funded Warrant | $2.2399 | | Expected Closing Date | August 12, 2025 | Upcoming Anticipated Milestones The company outlined key upcoming milestones including LUNA data presentation, AQUARIUS trial initiation, ARTEMIS enrollment completion, and ARTEMIS topline data announcement through H1 2027 - 4Q 2025: Present Phase 2 LUNA two-year long-term follow-up data[11] - 4Q 2025: Initiate AQUARIUS Phase 3 trial, pending funding availability[11] - 1Q 2026: Complete enrollment in the ARTEMIS Phase 3 trial[11] - 1H 2027: Announce topline data from the ARTEMIS Phase 3 trial[11] Financial Results Adverum reported a net loss of $49.2 million in Q2 2025, widening from $30.5 million due to increased R&D expenses, with cash decreasing to $44.4 million expected to fund operations into Q4 2025 Statement of Operations Analysis Q2 2025 R&D expenses more than doubled to $37.1 million due to the ARTEMIS trial, leading to a net loss of $49.2 million or $2.34 per share Q2 2025 vs. Q2 2024 Statement of Operations (in thousands) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Research and development | $37,125 | $17,097 | | General and administrative | $12,730 | $15,807 | | Total operating expenses | $49,855 | $32,904 | | Net loss | $(49,191) | $(30,498) | | Net loss per share | $(2.34) | $(1.46) | - The increase in R&D expenses was primarily due to higher clinical trial expenses and personnel-related costs associated with the ARTEMIS Phase 3 trial[11] - The decrease in G&A expenses was driven by lower facilities expenses and reduced stock-based compensation[15] Balance Sheet and Cash Position As of June 30, 2025, Adverum held $44.4 million in cash, a significant reduction from $125.7 million at year-end 2024, expected to fund operations into Q4 2025 Selected Balance Sheet Data (in thousands) | Metric | June 30, 2025 | Dec 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | $44,397 | $125,691 | | Total Assets | $96,179 | $179,841 | | Total Stockholders' (Deficit) Equity | $(20,219) | $70,714 | - The company's current cash, cash equivalents, and short-term investments are expected to fund operations into the fourth quarter of 2025[11] Company and Product Overview Adverum Biotechnologies is a clinical-stage company developing single-administration gene therapies for ocular diseases, with lead candidate Ixo-vec for wet AMD receiving key regulatory designations About Ixo-vec in Wet AMD Ixo-vec is Adverum's one-time, in-office intravitreal gene therapy for wet AMD, utilizing a proprietary AAV.7m8 vector for long-term efficacy, and holds multiple significant regulatory designations - Ixo-vec is designed as a one-time, in-office intravitreal injection, differentiating it from gene therapies requiring sub-retinal surgery[12] - The therapy utilizes a proprietary vector (AAV.7m8) to carry an aflibercept coding sequence, aiming for long-term efficacy[12] - Ixo-vec has received multiple significant regulatory designations, including FDA Fast Track, RMAT, EMA PRIME, and a UK Innovation Passport[12] About Adverum Biotechnologies Adverum Biotechnologies is a clinical-stage company focused on establishing gene therapy as a new standard of care for prevalent eye diseases through durable, single-administration treatments - Adverum is a clinical-stage company focused on developing gene therapies for highly prevalent ocular diseases[13] - The company's strategic goal is to create durable, single-administration treatments to eliminate the need for frequent ocular injections[13] Forward-Looking Statements This section provides a legal disclaimer, cautioning that forward-looking statements regarding Ixo-vec's potential, clinical timelines, and financial sufficiency are subject to significant risks and uncertainties - Statements regarding Ixo-vec's potential, clinical milestones, cash runway, and partnering discussions are considered forward-looking[14] - Key risks include uncertainties inherent to novel technology, clinical trial completion, regulatory approvals, potential side effects, and the company's ability to continue as a going concern[16]