KEEPING AND EYE ON ADVERUM BIOTECHNOLOGIES, INC.

Slowly succumbing to blindness is one of the greatest fears that confronts today’s senior citizens. Treatments for age related macular degeneration are available, but currently they only offer limited efficacy and they require constant, uncomfortable injections directly into the eye. Is there a better way?  

Biotech Analyst Nathaniel Suryabudi explores……

Adverum Biotechnologies is a prominent clinical-stage gene therapy company that is at the forefront of developing innovative treatments for ocular diseases and rare genetic disorders. The company is headquartered in California, United States, and has gained recognition for its pioneering work in the field of gene therapy.

Gene therapy is a rapidly advancing area of medical research that aims to treat diseases by introducing genetic material into a patient’s cells to correct or modify faulty genes. Adverum focuses on leveraging gene therapy to address serious eye-related conditions, particularly those affecting the retina.

The retina is a vital part of the eye that plays a crucial role in vision. Diseases such as wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR), and retinal vein occlusion (RVO) can cause significant damage to the retina, leading to vision loss and impaired quality of life for patients. Adverum is dedicated to developing gene therapies that can provide long-term benefits by targeting the underlying causes of these conditions.

The Pipeline

A key focus of Adverum’s efforts is on wet age-related macular degeneration (wet AMD), a leading cause of blindness globally. Approximately 20 million people in the United States have AMD and with aging populations on the rise (Between 2010 and 2050, the estimated number of people with severe AMD will more than double from 2.1 million to 5.4 million) the incidence of wet AMD is increasing. Current treatment options, such as painful eye injections of anti-VEGF therapies, are administered by retinal specialists every 4-8 weeks. However, these treatments often fall short in effectively preventing blindness in real-world use.

Recognizing the burden and limitations of the current standard of care, Adverum aims to address these challenges with their investigational gene therapy, Ixo-vec. This potential breakthrough therapy is being developed to offer an alternative to the recurring and painful eye injections. Instead, Ixo-vec is designed to provide an in-office, single-administration intravitreal (IVT) therapy.

The innovative aspect of Ixo-vec lies in its potential to extend the treatment benefit from weeks to years. By delivering a therapeutic gene through a single IVT administration, Adverum aims to provide long-lasting and sustained effects. This approach has the potential to significantly reduce the treatment burden for patients, eliminating the need for frequent visits and injections.

The convenience and extended treatment duration offered by Ixo-vec could have a transformative impact on the lives of patients with wet AMD. It may improve treatment adherence and reduce the risk of vision loss associated with missed treatments. Moreover, by targeting the underlying cause of the disease, Ixo-vec has the potential to achieve functional cures, restoring vision and preventing further progression of wet AMD.

Adverum’s pursuit of developing Ixo-vec as a gene therapy for wet AMD reflects their commitment to advancing the field of ocular gene therapies. By leveraging the power of gene therapy to provide long-term benefits and minimize treatment burdens, Adverum aspires to make a substantial difference in the lives of patients affected by wet AMD and other ocular diseases.

It’s important to note that while Adverum’s investigational gene therapy shows promise, it is still undergoing clinical trials  (currently stage 2). The ultimate success and approval of Ixo-vec will depend on the results of these studies and subsequent regulatory processes. Initial results have shown promise, but there are not guarantees in biotech. 

Competition and The Market

The potential of gene therapy in treating various eye diseases has garnered significant interest from major pharmaceutical companies, including Novartis, Biogen, and Johnson & Johnson. This groundbreaking approach has also attracted the attention of smaller biotechnology firms such as Regenxbio, which are actively testing gene therapies for common forms of vision loss. With the aim of revolutionizing treatment options, these companies are exploring the potential of gene therapies to address the unmet medical needs of patients suffering from debilitating ocular conditions.

Major pharmaceutical companies, such as Novartis, Biogen, and Johnson & Johnson, recognize the immense potential of gene therapy in transforming the treatment landscape for eye diseases. These companies have made significant investments and formed strategic partnerships to advance research and development efforts in this field.

Novartis, a global pharmaceutical powerhouse, has been actively involved in the development of gene therapies for ocular diseases. Their efforts include investigating treatments for retinitis pigmentosa, an inherited condition that causes progressive vision loss. Novartis has also initiated clinical trials for gene therapies targeting other eye conditions, such as X-linked retinitis pigmentosa. Norvatis’s leading medicine, Beovu, also known as brolucizumab, is a medication that has been used for the treatment of certain eye conditions. However, it is believed to cause serious eye injuries, including vision loss and even blindness.

Biogen, renowned for its expertise in neuroscience, has expanded its focus to include gene therapies for ocular diseases. The company is investigating treatments for common forms of vision loss, including geographic atrophy associated with age-related macular degeneration (AMD). Biogen’s leading drug is BYOOVIZ, which is FDA approved. 

Johnson & Johnson, a diversified healthcare company, has also entered the field of gene therapy for ocular diseases. The company aims to develop innovative treatments that address significant unmet medical needs in ophthalmology, including gene therapies for inherited retinal diseases. The acquisition of rights to Hemera Biosciences’ investigational gene therapy, HMR59, by Janssen Pharmaceuticals (a subsidiary of Johnson & Johnson), reflects the company’s interest in expanding its presence in the age-related macular degeneration (AMD) market. By acquiring the rights to HMR59 (phase 2), Johnson & Johnson aims to expand its portfolio in ophthalmology and position itself as a potential competitor in the AMD market.

In addition to the major pharmaceutical players, smaller biotechnology companies are actively involved in advancing gene therapy for vision loss. Regenxbio, a notable player in the field, is conducting clinical trials for gene therapies targeting common forms of vision loss, such as wet AMD and diabetic retinopathy.

Regenxbio’s investigational therapies utilize adeno-associated viruses (AAVs) as vectors to deliver therapeutic genes into retinal cells. Their innovative approach has shown promise in preclinical and early clinical studies, raising hopes for potential breakthrough treatments in the future. ABBV-RGX-314 is an investigational gene therapy being developed by AbbVie in collaboration with REGENXBIO. It aims to provide a one-time subretinal treatment for retinal diseases by utilizing a NAV® AAV8 vector that carries a gene encoding for a monoclonal antibody fragment. ABBV-RGX-314 is currently in Phase 2 of clinical development.

All of this means that Adverum faces substantial competition in the race to cure age related blindness. But it also validates the impressive commercial potential that this line of treatment represents.

In 2021, the market size of global age-related macular degeneration reached a noteworthy valuation of USD 9.84 billion. Looking ahead, it is projected to exhibit a steady growth trajectory with a compound annual growth rate (CAGR) of 6.9% from 2022 to 2030. This growth rate would mean that the market should double to almost $20 billion annually in just one decade. This forecast indicates a promising outlook for the age-related macular degeneration market, suggesting sustained expansion over the next decade. With Adverum currently valued at just over $200 million, this means that any effective treatment at all could easily triple or quadruple the stock price. 

Financial Standing and Conclusion

Adverum, a company with an exceptional financial standing, demonstrated robust cash reserves and financial stability. As of March 31, 2023, their cash, cash equivalents, and short-term investments amounted to $164.3 million, indicating a strong financial position. Although this figure decreased from $185.6 million reported on December 31, 2022, the cash balance remains highly significant. Moreover, Adverum confidently projects that their cash position as of March 31, 2023 will be sufficient to fund their operations well into the year 2025. This assurance of extended financial sustainability reflects Adverum’s excellent financial position and their ability to effectively manage resources.

In light of Adverum’s strong financial standing and the promising growth prospects of the global age-related macular degeneration market, the company presents itself as an attractive candidate for inclusion in one’s investment portfolio. With a robust cash position and the ability to fund operations into 2025, Adverum demonstrates a solid foundation for long-term success. Furthermore, the projected growth rate of the age-related macular degeneration market provides a favorable market environment for Adverum’s innovative therapies and treatments. Considering these factors, Adverum’s potential for delivering value to shareholders makes it a compelling choice for those seeking to diversify and capitalize on the opportunities in the healthcare sector.