UNITY Biotechnology, Inc. (UBX) has announced that following a Type C interaction with the U.S. Food and Drug Administration (FDA), a pivotal study for UBX1325 (foselutoclax) in diabetic macular edema (DME) is expected to be a non-inferiority trial. The trial will compare UBX1325 to an approved anti-VEGF agent like aflibercept, with the primary endpoint focusing on best-corrected visual acuity (BCVA). This regulatory guidance shapes the future development path for UBX1325, a novel senolytic therapeutic aimed at improving long-term visual outcomes for DME patients.
Regulatory Path for UBX1325 in DME
The FDA's feedback indicates that regulatory approval will hinge on demonstrating non-inferiority in BCVA, as measured by the ETDRS scale, with a non-inferiority margin of 4 letters. This benchmark sets a clear target for UNITY as it prepares for a potential pivotal study. Anirvan Ghosh, Ph.D., chief executive officer of UNITY, stated that the Phase 2b ASPIRE study's readouts would provide a definitive dataset to inform the design of this pivotal study.
ASPIRE Phase 2b Study and Upcoming Data
The Phase 2b ASPIRE study (NCT06011798) is a multi-center, randomized, double-masked, active-controlled trial evaluating the safety and efficacy of UBX1325 in a head-to-head comparison with aflibercept. UNITY anticipates topline 24-week primary endpoint data in the first quarter of 2025 and 36-week data in the second quarter of 2025. These results are expected to play a crucial role in shaping the design of the pivotal trial and the future of UBX1325's development.
UBX1325: A Novel Approach to DME Treatment
UBX1325 (foselutoclax) is designed as a novel and durable therapeutic option for DME, acting via a senolytic mechanism of action. It aims to address the shortcomings of current standard of care, such as high treatment burden and sub-optimal response to treatment. By selectively eliminating or modulating senescent cells, UBX1325 has the potential to improve long-term visual outcomes for DME patients.
Financial Position
As of September 30, 2024, UNITY's cash, cash equivalents, and marketable securities totaled $29.0 million, compared to $43.2 million as of December 31, 2023. The company believes that its current financial resources are sufficient to fund operations into the third quarter of 2025. The net loss for the three months ended September 30, 2024, was $6.5 million, compared to $14.8 million for the same period in 2023. Research and development expenses decreased by $1.8 million, primarily due to reduced personnel costs and the completion of Phase 2 studies of UBX1325 in DME and AMD.
