Business Overview and History

REGENXBIO was formed in 2008 in the State of Delaware and is headquartered in Rockville, Maryland. The company is a clinical-stage biotechnology company that has developed a broad pipeline of gene therapy product candidates using its proprietary adeno-associated virus (AAV) gene delivery platform, the NAV Technology Platform.

In March 2014, REGENXBIO entered into an exclusive license agreement with Novartis Gene Therapies, Inc. (formerly AveXis, Inc.) to grant them an exclusive, worldwide commercial license to the NAV Technology Platform for the treatment of spinal muscular atrophy (SMA) in humans by in vivo gene therapy. This led to the development and 2019 launch of Zolgensma, a licensed product under the agreement, which is the first and only gene therapy approved to treat SMA. REGENXBIO receives royalties on net sales of Zolgensma.

In addition to licensing its NAV Technology Platform to other companies, REGENXBIO has also developed its own pipeline of investigational gene therapy product candidates to address a variety of diseases. The company's lead internal programs include RGX-202 for the treatment of Duchenne muscular dystrophy, RGX-121 for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, and ABBV-RGX-314, which is being developed in collaboration with AbbVie for the treatment of chronic retinal conditions such as wet age-related macular degeneration and diabetic retinopathy.

Despite its progress in developing a broad pipeline of gene therapy candidates, REGENXBIO has incurred cumulative losses since inception and as of December 31, 2023 had generated an accumulated deficit of $705 million. The company's ability to achieve recurring profitability is dependent upon the successful development, approval and commercialization of its product candidates, which is uncertain. REGENXBIO will need to continue to rely on additional financing to achieve its business objectives.

Financial Overview

REGENXBIO's financial performance has been characterized by significant research and development investments as the company advances its diverse pipeline of gene therapy candidates. For the fiscal year ended December 31, 2023, the company reported total revenues of $90.24 million, primarily driven by license and royalty revenue. During this period, REGENXBIO incurred net losses of $263.49 million, reflecting the company's ongoing commitment to clinical development and innovation.

For the nine months ended September 30, 2024, REGENXBIO reported total revenues of $62.11 million, a decrease of $5.92 million compared to the same period in 2023. This decrease was primarily attributable to lower Zolgensma royalties, which decreased from $63.45 million in the first nine months of 2023 to $60.85 million in the first nine months of 2024. The company's research and development expenses decreased by $18.44 million to $158.14 million, while general and administrative expenses decreased by $12.85 million to $56.57 million during the nine-month period. REGENXBIO reported a net loss of $175.92 million for the first nine months of 2024, an improvement from the $200.60 million net loss in the same period of 2023.

In the most recent quarter (Q3 2024), REGENXBIO reported revenue of $24.20 million, representing a 16.3% decrease year-over-year, primarily due to a decrease in Zolgensma royalties. The net loss for the quarter was $59.60 million. Research and development expenses for Q3 2024 were $54 million, compared to $58 million for Q3 2023, a decrease primarily due to reductions in headcount and preclinical activities.

Liquidity

As of September 30, 2024, REGENXBIO had cash, cash equivalents, and marketable securities of $278.60 million, which the company believes will be sufficient to fund its operations into 2026. This liquidity position provides the company with the financial resources necessary to advance its pipeline and pursue its strategic objectives.

The company's cash position decreased from $314 million as of December 31, 2023, primarily driven by cash used to fund operating activities during the 9 months ended September 30, 2024. This decrease was partially offset by $131 million in net proceeds from a public offering in March 2024.

REGENXBIO's financial stability is further supported by its favorable liquidity ratios. As of September 30, 2024, the company reported a debt-to-equity ratio of 0.29, a current ratio of 3.05, and a quick ratio of 3.05. These metrics indicate a strong short-term liquidity position and a manageable level of debt relative to equity.

Pipeline and Key Programs

REGENXBIO's diversified pipeline is anchored by several promising gene therapy candidates targeting a range of indications, including rare genetic disorders and widespread retinal conditions.

RGX-202 for Duchenne Muscular Dystrophy (DMD) REGENXBIO is developing RGX-202 as a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (DMD), a rare and devastating genetic disorder that primarily affects young boys. The company's AFFINITY DUCHENNE study is a Phase I/II clinical trial evaluating the safety, tolerability, and efficacy of RGX-202 in DMD patients aged 1 to 11 years old. In March and May 2024, REGENXBIO reported interim data from the trial, showcasing robust microdystrophin expression and a favorable safety profile. The company plans to initiate the pivotal phase of the AFFINITY DUCHENNE study imminently, making RGX-202 the only next-generation gene therapy in Phase 3 development for this devastating disease.

Interim data from the trial has shown meaningful increases in microdystrophin expression and reductions in serum creatine kinase levels, supporting evidence of clinical improvement. REGENXBIO plans to share a full program update, including pivotal trial design and plans for accelerated approval, as well as initial strength and functional assessment data, in November 2024.

RGX-121 for Mucopolysaccharidosis Type II (MPS II) REGENXBIO is developing RGX-121 as a potential one-time gene therapy for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. In February 2024, the company announced that the pivotal phase of the CAMPSIITE trial evaluating RGX-121 had achieved its primary endpoint, demonstrating a statistically significant reduction in cerebrospinal fluid heparan sulfate levels, a key biomarker of brain disease activity. REGENXBIO has initiated a rolling biologics license application (BLA) submission for RGX-121 using the accelerated approval pathway and expects to complete the filing in the first quarter of 2025. If approved, RGX-121 would become the first gene therapy and one-time treatment for MPS II, addressing the significant unmet need for this progressive and debilitating disorder.

ABBV-RGX-314 for Wet Age-Related Macular Degeneration (wet AMD) and Diabetic Retinopathy (DR) In collaboration with AbbVie, REGENXBIO is developing ABBV-RGX-314 as a potential one-time gene therapy for the treatment of chronic retinal conditions, including wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). The company's ATMOSPHERE and ASCENT pivotal trials, evaluating the subretinal delivery of ABBV-RGX-314 in wet AMD patients, are expected to support global regulatory submissions in the first half of 2026. Additionally, REGENXBIO and AbbVie are planning the Phase 3 clinical program for ABBV-RGX-314 in DR, with an End-of-Phase 2 meeting with the FDA expected in the fourth quarter of 2024. These late-stage programs represent significant commercial opportunities for REGENXBIO and its partner.

ABBV-RGX-314 uses the NAV AAV8 vector to deliver a gene encoding a therapeutic antibody fragment that inhibits vascular endothelial growth factor (VEGF). The product candidate is currently being evaluated in multiple ongoing clinical trials, including two pivotal trials for wet AMD (ATMOSPHERE and ASCENT) and two Phase II trials for wet AMD (AAVIATE) and DR (ALTITUDE) utilizing different delivery methods (subretinal and suprachoroidal, respectively). Positive interim data has been reported from the AAVIATE and ALTITUDE trials, demonstrating that ABBV-RGX-314 has been well-tolerated with no drug-related serious adverse events.

Collaborations and Partnerships

REGENXBIO has strategically leveraged its NAV Technology Platform through various collaborations and partnerships, further validating the versatility and potential of its proprietary technology.

In September 2021, the company entered into a collaboration and license agreement with AbbVie to jointly develop and commercialize ABBV-RGX-314 for the treatment of wet AMD and DR. Under the terms of this agreement, AbbVie is responsible for the majority of development expenses related to ABBV-RGX-314 starting in 2023, and the two companies will share equally in the net profits and losses associated with the commercialization of the product in the United States.

More recently, in January 2025, REGENXBIO announced a strategic partnership with Nippon Shinyaku to develop and commercialize RGX-121 for the treatment of MPS II and RGX-111 for the treatment of MPS I in the United States and Asia. This collaboration further solidifies REGENXBIO's position in the rare disease gene therapy space and provides additional resources to advance these programs.

These partnerships not only supplement REGENXBIO's internal resources but also validate the potential of its technology and product candidates, ultimately benefiting the company's long-term growth and the patients it aims to serve.

Risks and Challenges

As a clinical-stage biotechnology company, REGENXBIO faces a range of risks and challenges inherent to the industry, including:

1. Clinical Development Risks: The successful development and regulatory approval of REGENXBIO's gene therapy candidates are subject to significant uncertainty and may be delayed or ultimately unsuccessful, which could adversely impact the company's financial performance and future prospects.

2. Manufacturing Challenges: The production of gene therapy products requires specialized facilities and expertise, and any issues related to manufacturing scale-up, consistency, or quality control could hamper REGENXBIO's ability to advance its pipeline.

3. Competitive Landscape: REGENXBIO faces competition from other gene therapy companies, as well as traditional therapies, which may develop treatments that are safer, more effective, or more convenient than the company's investigational products.

4. Regulatory Uncertainties: The gene therapy industry is subject to evolving regulatory frameworks, and REGENXBIO must navigate the complex approval process for its product candidates, which could result in delays or setbacks.

5. Intellectual Property Risks: The company's ability to protect its proprietary technology and innovations is crucial, and any challenges to its intellectual property rights could undermine its competitive position.

6. Financing Needs: As a clinical-stage company, REGENXBIO will likely require additional capital to fund its ongoing operations and the advancement of its pipeline, which may not be available on favorable terms or at all.

Despite these risks, REGENXBIO's strong intellectual property position, diverse pipeline, and strategic partnerships position the company as a leader in the gene therapy field, with the potential to deliver transformative treatments for patients in need.

Conclusion

REGENXBIO's pioneering approach to gene therapy has the potential to revolutionize the treatment of a wide range of devastating diseases. With a robust pipeline, including late-stage programs targeting rare genetic disorders and widespread retinal conditions, the company is at the forefront of this rapidly evolving field.

Through its proprietary NAV Technology Platform and strategic collaborations, REGENXBIO is well-positioned to continue its mission of improving lives and delivering on the curative promise of gene therapy. As the company advances its pipeline and navigates the complexities of the regulatory landscape, investors will closely monitor REGENXBIO's progress and the potential impact of its innovative treatments on the lives of patients worldwide.

The company's current cash position of $278.60 million as of September 30, 2024, is expected to fund operations into 2026, providing a solid financial foundation for the advancement of its key programs. This cash runway guidance excludes any potential payments from AbbVie upon development or commercial milestones for the RGX-314 collaboration, as well as the potential monetization of a Priority Review Voucher for RGX-121, which could further extend the company's financial resources.

As REGENXBIO continues to progress its pipeline, with multiple potential catalysts on the horizon, including the completion of the rolling BLA submission for RGX-121 in Q1 2025 and the initiation of a pivotal trial for RGX-202 in Q4 2024, the company remains focused on delivering innovative gene therapies that have the potential to transform the lives of patients with serious genetic diseases and conditions.