Company: RGNX
Filing Date: 2025-03-13
Form Type: 10-K
Source: 0000950170-25-038770
Chunk: 88

Company: REGENXBIO Inc.
Filing Date: 2025-03-13
Form: 10-K
Item: Item 1
Chunk 88
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 from the FDA, as well as orphan designation and ATMP classification from the EMA for RGX-111.

Clinical Development of RGX‑111 for the Treatment of MPS I

A Phase I/II study evaluating RGX-111 initiated in 2017 and enrollment for that study completed in 2023. The multi-center, open-label, dose escalation trial evaluated the safety, tolerability and pharmacodynamics of RGX-111 delivered via single injection to the CNS to patients with MPS I. Interim results from the study, reported in February 2023, showed RGX-111 to be well tolerated across two dose levels in the eight patients enrolled in the Phase I/II trial as of January 17, 2023 and in the single-patient IND as of December 12, 2021, with no drug-related SAEs. Biomarker and neurodevelopmental assessments indicated an encouraging CNS profile in patients dosed with RGX-111.

In November 2023, future development of RGX-111 was largely halted as a result of a strategic pipeline prioritization and corporate restructuring. Efforts to continue development of RGX-111 are set to be reinitiated following our announcement in January 2025 of a strategic partnership with Nippon Shinyaku to develop and commercialize RGX-111 in the United States and Asia. For more information, refer to "Nippon Shinyaku Partnership for MPS Diseases" below.

Collaborations, Licensing and Company Formation

Collaborations, licensing and company formation are a key part of our commitment to enable the ongoing development of gene therapy treatments. 

AbbVie Eye Care Collaboration

In September 2021, REGENXBIO and AbbVie announced a global strategic partnership to develop and commercialize ABBV-RGX-314, a potential one-time gene therapy for the treatment of wet AMD, DR and other chronic retinal diseases. 

Under the terms of our Collaboration and License Agreement with AbbVie (the AbbVie Collaboration and License Agreement), we received an upfront payment of $370 million. Additionally, we are eligible to receive up to $1.38 billion in additional development, regulatory and commercial milestone payments.

11

In accordance with the AbbVie Collaboration and License Agreement, through December 31, 2022 we were responsible for development expenses related to certain ongoing clinical trials of ABBV-RGX-314 and the remaining ABBV-RGX-314 development expenses were shared with AbbVie. Beginning