REDWOOD CITY, Calif. - Adverum Biotechnologies, Inc. (NASDAQ:ADVM), a clinical-stage gene therapy company, announced preliminary data from its Phase 2 LUNA trial for wet age-related macular degeneration (AMD (NASDAQ:AMD)), suggesting potential treatment benefits. The findings were presented at the Macula Society's 47th Annual Meeting.
Wet AMD, a leading cause of blindness in those over 65, currently requires frequent anti-VEGF injections. Adverum's gene therapy candidate, ixoberogene soroparvovec (Ixo-vec), aims to reduce this treatment burden. Early results indicate that Ixo-vec may maintain visual acuity and anatomic outcomes with significantly fewer injections.
The LUNA trial, which is ongoing, has enrolled 60 patients across two dosage cohorts. Preliminary efficacy data showed a 90-94% reduction in annualized anti-VEGF injection rates and a 68-85% injection-free rate at 26 weeks post-treatment. Safety data suggested well-tolerated outcomes with manageable inflammation when present.
Adverum's approach includes a prophylactic regimen combining Ozurdex and difluprednate, which has shown over 90% of patients with no or minimal inflammation. The company plans to present a 26-week interim analysis in mid-2024 and aims to initiate a Phase 3 trial in the first half of 2025.
This gene therapy leverages a proprietary vector capsid, AAV.7m8, to deliver a one-time intravitreal injection, potentially offering lasting management of wet AMD. Ixo-vec has received Fast Track designation from the FDA and PRIME designation from the EMA for the treatment of wet AMD.
Adverum's President and CEO, Laurent Fischer, M.D., noted the therapy's potential to provide a best-in-class efficacy profile, particularly in hard-to-treat patients. Principal Investigator Arshad M. Khanani, M.D., M.A., echoed the sentiment, highlighting the significant reduction in treatment burden observed.
The company continues to explore gene therapy as a standard of care for ocular diseases, with the broader goal of establishing functional cures to restore vision and prevent blindness.
The report is based on a press release statement from Adverum Biotechnologies.
InvestingPro Insights
As Adverum Biotechnologies forges ahead in its quest to revolutionize treatment for wet age-related macular degeneration (AMD), the financial metrics and market sentiment surrounding the company paint a detailed picture of its current standing. Adverum's market capitalization stands at a modest $199.02 million, reflecting its status as a clinical-stage entity focused on the development of novel gene therapies. Despite the company's promising scientific advancements, it operates with a negative P/E ratio of -1.76, which adjusts slightly to -1.63 when looking at the last twelve months as of Q3 2023. This indicates that investors are not valuing the company based on earnings at this stage, which is not uncommon for biotech firms in the development phase.
Investors have recognized the value in Adverum's progress and potential, as evidenced by the significant returns over the past year, with a 198.48% price total return. This optimism is further supported by the impressive short-term gains, with a 38.73% return over the last week alone. With a revenue of $3.6 million over the last twelve months as of Q3 2023, the company is still in the early stages of commercializing its technology.
Among the InvestingPro Tips, two particularly stand out for Adverum Biotechnologies. The company holds more cash than debt on its balance sheet, which is a reassuring sign for investors concerned about financial stability. Furthermore, the firm's liquid assets exceed its short-term obligations, suggesting a healthy liquidity position that could support ongoing clinical trials and research activities.
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