Adverum Biotechnologies(ADVM) - 2024 Q1 - Quarterly Results

Executive Summary & Q1 2024 Highlights The company reports Q1 2024 results, highlighting a strong cash position and upcoming LUNA trial data presentation - Adverum Biotechnologies announced Q1 2024 financial results and pipeline highlights, with the Phase 2 LUNA interim analysis to be presented at ASRS on July 17, 2024[1] - CEO Laurent Fischer highlighted Ixo-vec's potentially best-in-class product profile and the upcoming LUNA trial data presentation as a basis for FDA end-of-Phase 2 meeting, aiming to confirm optimal dose(s) for pivotal studies and shift the wet AMD treatment paradigm[1] Key Financial and Operational Highlights | Metric | Value | | :----- | :---- | | Cash, cash equivalents and short-term investments | $193.3 million | | Funding Horizon | Into late 2025 | Ixo-vec Program Updates The company provides updates on its Ixo-vec program, including trial data, presentation plans, and future milestones LUNA Phase 2 Interim Analysis Presentation The 26-week interim analysis from the LUNA Phase 2 study will be presented at the ASRS meeting in July 2024 - The 26-week landmark interim analysis from the LUNA Phase 2 study of Ixo-vec for wet AMD will be presented at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting from July 17-20, 2024[2] Preliminary LUNA Trial Data (Efficacy & Safety) Preliminary LUNA trial data shows positive efficacy and safety, with significant reductions in anti-VEGF injections - Positive preliminary efficacy and safety data from the LUNA Phase 2 trial of Ixo-vec in wet AMD patients were announced in February 2024[3] - Ixo-vec was generally well tolerated, with intraocular inflammation responsive to local corticosteroids and no Ixo-vec related serious adverse events, episcleritis, vasculitis, retinitis, choroiditis, vascular occlusion or hypotony reported[4] LUNA Trial Efficacy Data (through 26 weeks) | Dose | Mean Reduction in Annualized Anti-VEGF Injections | Patients Free of Supplemental Injections | | :--- | :------------------------------------------------ | :--------------------------------------- | | 2E11 | 94% | 85% | | 6E10 | 90% | 68% | Anticipated Milestones Key upcoming milestones include regulatory interactions, data presentations, and the initiation of a Phase 3 trial - Key anticipated milestones include continued regulatory interactions and data presentations leading to a Phase 3 trial initiation in H1 2025[5] Anticipated Milestones Timeline | Milestone | Timing | | :-------- | :----- | | Continued FDA and EMA regulatory interactions | 2024 | | Presentation of landmark LUNA 26-week interim analysis at ASRS | July 2024 | | Ixo-vec Phase 3 program and regulatory update | 4Q 2024 | | Presentation of landmark LUNA 52-week analysis | 1Q 2025 | | Initiation of Phase 3 trial | H1 2025 | Financial Results for Q1 2024 The company reports a strengthened cash position, reduced operating expenses, and a lower net loss for Q1 2024 Key Financial Highlights The company improved its cash position, extended its operational runway, and reduced both expenses and net loss per share - The company expects its cash, cash equivalents and short-term investments to fund operations into late 2025[7] Cash and Investment Position | Metric | March 31, 2024 | December 31, 2023 | Change | | :----- | :------------- | :---------------- | :----- | | Cash, cash equivalents and short-term investments | $193.3 million | $96.5 million | +$96.8 million | Q1 2024 vs Q1 2023 Expense and Loss Comparison | Metric | Q1 2024 ($M) | Q1 2023 ($M) | Change ($M) | Change (%) | | :----- | :----------- | :----------- | :---------- | :--------- | | Research and development expenses | 15.4 | 21.1 | (5.7) | -27.0% | | General and administrative expenses | 11.4 | 12.8 | (1.4) | -10.9% | | Net Loss | 24.8 | 29.1 | (4.3) | -14.8% | | Net Loss per share (basic and diluted) | (1.50) | (2.90) | 1.40 | -48.3% | Condensed Consolidated Balance Sheet Data The balance sheet reflects a significant increase in cash, total assets, and stockholders' equity as of March 31, 2024 Condensed Consolidated Balance Sheet Data (in thousands) | Metric | March 31, 2024 (Unaudited) | December 31, 2023 | | :----------------------------------------------- | :------------------------- | :---------------- | | Cash and cash equivalents, and marketable securities | $193,328 | $96,526 | | Total assets | $267,828 | $173,010 | | Total current liabilities | $21,549 | $24,914 | | Total liabilities | $85,540 | $89,541 | | Total stockholders' equity | $182,288 | $83,469 | Condensed Consolidated Statements of Operations The Q1 2024 statement of operations shows lower operating expenses and a reduced net loss compared to the prior year Condensed Consolidated Statements of Operations (in thousands, except per share data) | Metric | Three months ended March 31, 2024 (Unaudited) | Three months ended March 31, 2023 | | :----------------------------------------------- | :------------------------------------ | :-------------------------------- | | License revenue | $— | $3,600 | | Research and development expenses | $15,410 | $21,059 | | General and administrative expenses | $11,429 | $12,780 | | Total operating expenses | $26,839 | $33,839 | | Operating loss | $(26,839) | $(30,239) | | Other income, net | $2,052 | $1,200 | | Net loss | $(24,787) | $(29,056) | | Net loss per share — basic and diluted | $(1.50) | $(2.90) | | Weighted-average common shares outstanding | 16,479 | 10,030 | Disease and Product Information This section details Wet AMD, the current standard of care, and the Ixo-vec gene therapy candidate About Wet Age-Related Macular Degeneration (Wet AMD) Wet AMD is a leading cause of blindness, with current treatments requiring frequent injections - Wet AMD is a VEGF-driven advanced form of AMD, affecting approximately 10% of AMD patients and is a leading cause of blindness in people over 65, with an estimated 20 million individuals worldwide living with this condition[8] - The current standard of care involves frequent, life-long anti-VEGF injections, while IVT gene therapy aims to provide stable therapeutic levels of anti-VEGF to control macular fluid, potentially preserving vision and reducing injections for the patient's life[8] About Ixo-vec in Wet AMD Ixo-vec is a one-time IVT gene therapy candidate designed to reduce treatment burden for Wet AMD patients - Ixo-vec (ixoberogene soroparvovec) is a clinical-stage gene therapy product candidate for wet AMD, designed as a one-time intravitreal (IVT) injection using a proprietary AAV.7m8 vector carrying an aflibercept coding sequence[9] - Ixo-vec aims to deliver long-term efficacy, reduce the burden of frequent anti-VEGF injections, optimize patient compliance, and improve vision outcomes for wet AMD patients[9] - Ixo-vec has received Fast Track designation from the U.S. FDA, PRIME designation from the European Medicines Agency, and the Innovation Passport from the UK's MHRA for wet AMD treatment[9] Company Information This section provides an overview of Adverum Biotechnologies and details on a recent inducement grant About Adverum Biotechnologies Adverum is a clinical-stage company developing gene therapies for prevalent ocular diseases - Adverum Biotechnologies is a clinical-stage company pioneering gene therapy for highly prevalent ocular diseases, aiming to develop functional cures to restore vision and prevent blindness[10] - The company leverages its proprietary intravitreal (IVT) platform to develop durable, single-administration therapies, eliminating the need for frequent ocular injections[10] Inducement Grant A stock option grant was issued to a new non-executive employee as an inducement to employment - On May 6, 2024, Adverum granted a new non-executive employee a stock option to purchase 21,000 shares of common stock at an exercise price of $10.66 per share, vesting over four years, as an inducement to employment[6] Legal & Contact Information This section includes forward-looking statements, risk disclosures, and company contact details Forward-looking Statements The report contains forward-looking statements subject to risks and uncertainties detailed in SEC filings - Statements in the press release regarding future events or results are 'forward-looking statements' under the Private Securities Litigation Reform Act of 1995, covering topics like cash runway, Ixo-vec's potential, and trial timelines[11] - Actual results could differ materially due to various risks and uncertainties, including market conditions and those detailed in Adverum's SEC filings, such as the Annual Report on Form 10-K and subsequent 10-Q reports[11] Corporate, Investor and Media Inquiries Contact information is provided for corporate, investor, and media inquiries - Contact information for corporate, investor, and media inquiries is provided via email: ir@adverum.com[12]