REGENXBIO Announces Q3 2024 Financial Results and Operational Updates

REGENXBIO Inc., a clinical-stage biotechnology firm based in Rockville, Maryland, has announced significant progress and financial results for the third quarter ending September 30, 2024. The company is advancing its gene therapy treatments, particularly for Duchenne Muscular Dystrophy, MPS II, and retinal diseases.

The Phase I/II AFFINITY DUCHENNE® trial of RGX-202, a gene therapy for Duchenne Muscular Dystrophy, has reached a pivotal stage with the final patient dosed in the 4-11 age group and the first patient dosed in the 1-3 age group. This trial aims to deliver a novel microdystrophin gene to improve patient outcomes. REGENXBIO is the only gene therapy sponsor enrolling patients under four years old in the U.S. A clinical trials application for RGX-202 has also been approved by Health Canada, with plans to initiate sites there in the first half of 2025. The company is set to provide a comprehensive update on the program, including initial data on strength and functional assessments, later this month.

In the realm of retinal diseases, ABBV-RGX-314, developed in collaboration with AbbVie, is showing promising results for both wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). Interim data from the Phase II ALTITUDE® trial for DR has led to an accelerated End-of-Phase II meeting with the FDA, expected this quarter, with a pivotal trial anticipated to begin in the first half of 2025. Additionally, REGENXBIO is enrolling a new cohort in the ALTITUDE trial to assess treatment for diabetic macular edema (DME), a complication of DR affecting millions globally.

For wet AMD, the ABBV-RGX-314 therapy has shown positive results in the Phase II fellow eye sub-study. Patients treated with this gene therapy demonstrated a significant reduction in anti-VEGF treatment burden while maintaining vision and eye anatomy at nine months post-administration. This supports the potential of ABBV-RGX-314 as a one-time treatment for bilateral wet AMD.

Another significant development is the rolling Biologics License Application (BLA) submission for RGX-121, a gene therapy for MPS II, which began in the third quarter of 2024 and is expected to be completed by the first quarter of 2025. If approved, RGX-121 could become the first gene therapy treatment available for MPS II, potentially earning a Priority Review Voucher in 2025.

Financially, REGENXBIO reported cash, cash equivalents, and marketable securities of $278.6 million as of September 30, 2024, a decrease from $314.1 million at the end of 2023. This decline was mainly due to operational expenditures, offset by proceeds from a public offering of common stock and pre-funded warrants in March 2024. The company's revenue for the third quarter of 2024 was $24.2 million, down from $28.9 million in the same period of 2023, primarily due to decreased royalty revenues from Zolgensma. Research and development expenses also decreased to $54.4 million from $58.2 million, and general and administrative expenses fell to $19.4 million from $23.1 million in the same periods.

REGENXBIO's net loss for the third quarter was $59.6 million, or $1.17 per share, compared to a net loss of $61.9 million, or $1.41 per share, in the third quarter of 2023. Looking ahead, the company expects its current cash reserves to fund operations into 2026, excluding potential milestone payments or product commercialization revenues.

REGENXBIO continues to make strides in its gene therapy advancements, with pivotal trials and regulatory submissions on the horizon, aiming to transform treatments for serious diseases.