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REDWOOD CITY, Calif. - Adverum Biotechnologies, Inc. (NASDAQ:ADVM) announced today that the U.S. Food and Drug Administration (FDA) has awarded its gene therapy product candidate, Ixo-vec, with the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of wet age-related macular degeneration (wet AMD (NASDAQ:AMD)). This status is meant to expedite the development and review of promising medicines that address severe or life-threatening conditions, offering benefits such as FDA guidance and potential for priority review.
Wet AMD is a leading cause of blindness in individuals over 65, affecting approximately 20 million people globally. As populations age, these numbers are expected to rise, with estimates reaching 288 million affected by AMD by 2040. Ixo-vec is designed to reduce or eliminate the need for frequent eye injections that constitute the current standard of care for wet AMD, by providing a one-time intravitreal (IVT) gene therapy.
The RMAT designation was granted based on preliminary clinical evidence suggesting Ixo-vec could meet an unmet medical need for this condition. It is currently under evaluation in the Phase 2 LUNA clinical trial and the Phase 1 OPTIC extension study.
In addition to RMAT, Ixo-vec has received Fast Track designation from the FDA, PRIME designation from the European Medicines Agency (EMA), and the Innovation Passport from the UK's Medicines and Healthcare products Regulatory Agency for wet AMD treatment.
Adverum's proprietary vector capsid, AAV.7m8, carries an aflibercept coding sequence, which is administered via a one-time IVT injection in the physician's office, aiming to deliver long-term efficacy and optimize patient compliance.
This latest FDA designation recognizes the potential of Ixo-vec as a transformative gene therapy for patients with wet AMD. Adverum anticipates sharing updates from regulatory discussions in the fourth quarter of this year. The company is committed to pioneering gene therapy as a standard of care for ocular diseases, focusing on developing functional cures to restore vision and prevent blindness. The information reported is based on a press release statement from Adverum Biotechnologies.
In other recent news, Adverum Biotechnologies has made significant strides in its gene therapy treatments with its leading candidate, Ixo-vec, designed to reduce the treatment burden for patients with wet age-related macular degeneration (AMD). Clinical results have shown a promising reduction in treatment burden and a high injection-free rate. Oppenheimer initiated coverage on Adverum with an Outperform rating, reinforcing the gene therapy's commercial potential.
Adverum has also seen changes in its leadership team, with the appointment of Dr. Rabia Gurses Ozden as the new Chief Medical Officer and the addition of Dr. Szilárd Kiss to its Board of Directors. Both bring extensive experience in ophthalmology and gene therapy, expected to be instrumental in the development of Ixo-vec.
RBC Capital Markets, however, has lowered the company's share price target from $3.00 to $2.00 in response to Adverum's fourth quarter and full-year 2023 financial report. The firm maintains a cautious stance due to Adverum's past clinical challenges. Despite these developments, RBC Capital is closely monitoring the situation, awaiting further evidence to alleviate the risks associated with Adverum's clinical developments. These are the recent developments from Adverum Biotechnologies.
InvestingPro Insights
Amidst the promising news of Adverum Biotechnologies' (NASDAQ:ADVM) gene therapy candidate, Ixo-vec, receiving the Regenerative Medicine Advanced Therapy (RMAT) designation, the company's financial health and market performance provide additional context for investors. An InvestingPro Tip highlights that Adverum holds more cash than debt on its balance sheet, which is a positive indicator of the company's financial stability. This could be particularly relevant as the company continues to invest in the development of its gene therapy product.
However, it is important to note that Adverum is not currently profitable, as evidenced by a negative P/E ratio of -0.79 and an adjusted P/E ratio for the last twelve months as of Q1 2024 at -1.37. Additionally, the company has been rapidly burning through cash, which is a critical consideration for investors given the high costs associated with clinical trials and the path to commercialization for Ixo-vec.
Investors should also be aware of the company's recent market performance. Adverum's stock has taken a significant hit over the last week, with a one-week price total return of -8.28%. This could reflect market reactions to broader industry trends or company-specific events. For those interested in exploring further, there are additional InvestingPro Tips available on the platform, which can be accessed at https://www.investing.com/pro/ADVM.
As Adverum Biotechnologies continues to make strides in the field of gene therapy for ocular diseases, keeping an eye on both the scientific advancements and the financial metrics will be crucial for informed investment decisions.
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