Key Takeaways
- Regenxbio submits BLA for RGX-111, aiming for FDA approval in late 2025.
- The company reported a net loss of $51.2 million for Q4 2024, an improvement from the previous year’s loss.
- RGX-121 is in development as a potential first gene therapy for MPS II, while RGX-111 progresses with a partnership in Japan.
Regenxbio (Nasdaq: RGNX), a US-based biotechnology company, has announced the successful submission of its Biologics License Application (BLA) for RGX-111, a treatment for mucopolysaccharidosis II (MPS II). The company is on track for potential accelerated FDA approval, anticipated in the second half of 2025. Following this news, Regenxbio’s shares increased by 2.6%, reaching $6.72.
In its recently released financial results for the fourth quarter of 2024, Regenxbio reported a net loss of $51.2 million, which translates to a basic and diluted loss of $1.01 per share. This indicates a significant improvement from the previous year’s net loss of $62.9 million, or $1.43 per share. The positive momentum in their financial performance reflects ongoing advancements in their pipeline and strategic initiatives.
Regenxbio is also advancing RGX-121, which aims to become the first approved gene therapy and one-time treatment for MPS II, also known as Hurler syndrome. In addition to RGX-111, the company continues progressing its efforts in gene therapy through a recent strategic partnership with Nippon Shinyaku (TYO: 4516) for the development of RGX-111, aimed at treating MPS I. This collaboration strengthens Regenxbio’s position in the gene therapy market and enhances its therapeutic options for various lysosomal storage disorders.
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