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 ...

Ixoberogene Soroparvovec (Ixo-vec) IVT Gene Therapy for Neovascular AMD: First Time 26-Week Interim Analysis Results from the Phase 2 LUNA Study Charles C. Wykoff, MD, PhD Director of Research, Retina Consultants of Texas Professor of Clinical Ophthalmology, Blanton Eye Institute, Houston Methodist Hospital Paul Hahn, MD, PhD, Eduardo Uchiyama, MD, Sean D. Adrean, MD, Mark R. Barakat, MD, Cameron Javid, MD, Dante J. Pieramici, MD, Bill Tan, PharmD, Adam Turpcu, PhD, Kalliopi Stasi, MD, PhD, Star Seyedkazemi ...

Adverum Biotechnologies, Inc. (NASDAQ:ADVM) 26-Week Interim Analysis of the LUNA Phase 2 Trial Update Call July 17, 2024 8:00 AM ET Company Participants Mike Zanoni - Head of IR Laurent Fischer - President & CEO Charles Wykoff - Director of Research, Retina Consultants of Texas Conference Call Participants Joon Lee - Truist Securities Joseph Thome - TD Cowen Francois Brisebois - Oppenheimer Graig Suvannavejh - Mizuho Securities Luca Issi - RBC Capital Daniil Gataulin - Chardan Operator Hello, ladies and gen ...

Summary of Adverum Biotechnologies, Inc. Conference Call on LUNA Phase 2 Trial Company and Industry Overview - **Company**: Adverum Biotechnologies, Inc. (NASDAQ:ADVM) - **Industry**: Gene therapy for ophthalmic conditions, specifically targeting wet age-related macular degeneration (AMD) Key Points and Arguments 1. **LUNA Phase 2 Trial Overview**: The call discussed the 26-week interim analysis of the LUNA Phase 2 trial for Ixo-vec, a gene therapy product candidate aimed at treating wet AMD, presented at the 2024 ASRS Annual Meeting in Stockholm, Sweden [6][3][4]. 2. **Goals of the LUNA Study**: The primary objectives include selecting the appropriate dose and regimen for pivotal Phase III trials, expected to start in the first half of 2025 [7][49]. 3. **Clinical Activity and Efficacy**: The interim analysis showed a greater than 90% reduction in treatment burden, with 76% of patients remaining injection-free at the 6E10 dose and 83% at the 2E11 dose after six months [9][24]. Visual acuity was maintained, and central subfield thickness (CST) remained stable [9][28]. 4. **Safety Profile**: Ixo-vec demonstrated an improved safety profile compared to previous studies, with no serious adverse events related to the drug. Over 90% of participants experienced no or minimal inflammation [10][36]. The most common adverse event was dose-dependent anterior cellular inflammation, manageable with local corticosteroids [35][34]. 5. **Patient Preference Survey**: A survey indicated that 88% of patients preferred Ixo-vec over prior treatments, with 100% of patients in the 6E10 difluprednate cohort expressing a desire for Ixo-vec therapy in their other eye if affected by wet AMD [12][48]. 6. **Market Potential**: The wet AMD market is large and growing, with a significant need for innovative treatments that reduce the treatment burden associated with current anti-VEGF therapies [13][14]. Ixo-vec's potential for lifelong therapeutic benefit positions it favorably against existing treatments [16]. 7. **Long-term Efficacy**: Data from the OPTIC trial indicated sustained therapeutic levels of Ixo-vec beyond four years, suggesting a long-term solution for wet AMD patients [17][18]. 8. **Regulatory Engagement**: The company is actively engaging with regulatory bodies like the FDA and EMA to facilitate the development and approval of Ixo-vec [49]. Additional Important Content 1. **Challenges in Current Treatments**: The treatment burden of wet AMD is significant, often leading to under-treatment and poor outcomes due to missed visits and limited access [14][15]. 2. **Comparative Analysis**: Ixo-vec showed superior reduction in annualized injections compared to other gene therapy agents, with a higher injection-free rate [45][46]. 3. **Prophylactic Regimens**: The study evaluated various prophylactic regimens to minimize inflammation, leading to protocol amendments that improved safety outcomes [37][38]. 4. **Future Directions**: The company plans to present further data from the LUNA trial in the fourth quarter of 2024 and initiate pivotal trials in 2025 [49]. This summary encapsulates the critical insights from the conference call regarding Adverum Biotechnologies' ongoing efforts in the gene therapy space for treating wet AMD, highlighting the promising results of the LUNA Phase 2 trial and the potential market impact of Ixo-vec.

Core Insights - Adverum Biotechnologies announced promising results from the 26-week interim analysis of the LUNA Phase 2 trial for Ixo-vec, a gene therapy for wet age-related macular degeneration (AMD), indicating a favorable safety and efficacy profile [1][2][6] Efficacy - 76% of patients receiving the 6E10 dose of Ixo-vec were injection-free at 26 weeks, maintaining visual acuity and fluid control [1][7] - Both 6E10 and 2E11 doses showed maintenance of visual and anatomic outcomes, with 83% of patients in the 2E11 cohort also being injection-free [6][7] - The mean annualized anti-VEGF injections were reduced by 90% for the 6E10 group and 95% for the 2E11 group at week 26 [7] Safety - 100% of patients receiving the difluprednate-alone prophylactic regimen had no or minimal inflammation, with no additional corticosteroids needed beyond scheduled prophylaxis [1][9] - Ixo-vec was well tolerated, with no serious adverse events related to the treatment and only mild to moderate adverse events reported [7][9] Patient Preference - A Patient Preference Survey indicated that 88% of patients preferred Ixo-vec over previous anti-VEGF treatments, and 93% would choose to receive Ixo-vec in the other eye if applicable [1][9] Upcoming Milestones - Key upcoming events include the presentation of the 9-month analysis and updates on Phase 3 trial design anticipated in Q4 2024, with the initiation of the Phase 3 trial planned for H1 2025 [10]

Core Viewpoint - Adverum Biotechnologies (ADVM) shows significant upside potential based on Wall Street analysts' price targets, with a mean target of $33.86 indicating a 290.5% upside from the current price of $8.67 [1] Price Targets and Analyst Consensus - The average price target for ADVM ranges from a low of $20 to a high of $60, with a standard deviation of $14.05, suggesting variability in analysts' estimates [1] - A low standard deviation indicates strong agreement among analysts regarding the stock's price movement direction, which can serve as a starting point for further research [4] Earnings Estimates and Market Sentiment - Analysts have shown growing optimism about ADVM's earnings prospects, as evidenced by a 6.6% increase in the Zacks Consensus Estimate over the past month, with 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 estimates, indicating strong potential for upside [5] Caution on Price Targets - While price targets are a popular metric, relying solely on them for investment decisions may not be prudent due to historical inaccuracies in predicting stock price movements [2][3] - Analysts may set overly optimistic price targets influenced by business relationships, which can lead to inflated expectations [3]

Core Viewpoint - Adverum Biotechnologies is advancing gene therapy as a potential new standard of care for wet age-related macular degeneration (wet AMD) and will present interim data from its LUNA Phase 2 study at the ASRS Annual Scientific Meeting [1][2]. Company Overview - Adverum Biotechnologies (NASDAQ:ADVM) is a clinical-stage company focused on developing gene therapies for prevalent ocular diseases, aiming to create functional cures to restore vision and prevent blindness [4]. - The company is leveraging its proprietary intravitreal (IVT) platform to develop durable, single-administration therapies that can be delivered in physicians' offices, reducing the need for frequent ocular injections [4]. Upcoming Events - A webcast will be held on July 17, 2024, at 8:00 a.m. ET to review the interim 26-week data from the LUNA Phase 2 study of ixoberogene soroparvovec (Ixo-vec) [1]. - Dr. Charles C. Wykoff will present the findings during the Wet AMD Symposium at the ASRS Annual Scientific Meeting on the same day at 8:45 a.m. CEST [2]. Presentation Access - The live webcast will be available on the company's website, and a replay will be accessible for at least 30 days following the event [3].

Core Insights - Adverum Biotechnologies has appointed Dr. Rabia Gurses Ozden as Chief Medical Officer and Dr. Szilárd Kiss to its Board of Directors, enhancing its leadership team as it advances its gene therapy candidate Ixo-vec for wet age-related macular degeneration (AMD) [1][2][5] Company Developments - The company is progressing with the LUNA trial for Ixo-vec, which is positioned as a potential best-in-class treatment for wet AMD, aiming to transform the treatment paradigm by offering a long-term solution to preserve sight [2][5] - Dr. Ozden brings over 20 years of experience in ophthalmology drug development, particularly in AAV-based gene therapies, and has previously held leadership roles in other biotech firms [2][3] - Dr. Kiss, a renowned retina specialist, has over 20 years of experience in ocular gene therapy and has been involved in numerous clinical trials, contributing significant expertise to the Board [3][4] Product Focus - Ixo-vec is being developed as a one-time intravitreal injection aimed at treating neovascular or wet AMD, with the goal of reducing the burden of frequent eye injections currently required by standard care [5] - The company aims to leverage its proprietary intravitreal platform to create durable therapies that can be administered in a physician's office, potentially eliminating the need for frequent ocular injections [5]

Core Viewpoint - Adverum Biotechnologies (ADVM) is experiencing significant selling pressure, with a 19.7% decline over the past four weeks, but is now positioned for a potential trend reversal due to being in oversold territory and positive earnings expectations from Wall Street analysts [1]. Group 1: Technical Indicators - The Relative Strength Index (RSI) for ADVM is at 26.99, indicating that the stock is oversold and may be nearing a price reversal [2][4]. - RSI is a momentum oscillator that helps identify oversold conditions, typically when the reading falls below 30, suggesting potential entry opportunities for investors [2]. Group 2: Fundamental Indicators - There is a strong consensus among sell-side analysts that ADVM will report better earnings than previously predicted, leading to a 0% increase in the consensus EPS estimate over the last 30 days [4]. - An upward trend in earnings estimate revisions is generally associated with price appreciation in the near term, indicating a potential rebound for ADVM [4]. - ADVM holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate revisions and EPS surprises, further supporting the stock's turnaround potential [4].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 ___________________________________________________________________ FORM 10-Q ___________________________________________________________________ (Mark One) QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ☒ EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 or TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ☐ EXCHANGE ACT OF 1934 For the transition period from to Com ...