UNITY Biotechnology is focusing on UBX1325 (foselutoclax) as a potential treatment for diabetic macular edema (DME). The company believes UBX1325's novel mechanism of action could improve long-term visual outcomes for DME patients, especially those with inadequate responses to anti-VEGF standard of care.
UBX1325: Targeting Senescent Cells in DME
UBX1325 is designed to act via a senolytic mechanism, potentially addressing the shortcomings of current DME treatments, such as high treatment burden and suboptimal responses. Anirvan Ghosh, Ph.D., chief executive officer of UNITY, stated that DME represents a large underserved market due to these limitations.
Regulatory Pathway and Clinical Trial Design
Following a Type C interaction with the FDA, UNITY anticipates that a pivotal study for UBX1325 would need to be a non-inferiority trial, comparing it to an approved anti-VEGF agent like aflibercept. The primary endpoint for regulatory approval is expected to be best-corrected visual acuity (BCVA), assessed using the ETDRS scale, with a non-inferiority margin of 4 letters.
The Phase 2b ASPIRE study (NCT06011798) is a multi-center, randomized, double-masked, active-controlled study evaluating the safety and efficacy of UBX1325 in a head-to-head comparison with aflibercept. Topline 24-week primary endpoint data is expected in the first quarter of 2025, with 36-week data following in the second quarter of 2025.
Financial Status
As of September 30, 2024, UNITY's cash, cash equivalents, and marketable securities totaled $29.0 million. The company believes these funds are sufficient to support operations into the third quarter of 2025.
For the three months ended September 30, 2024, UNITY reported a net loss of $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 the Phase 2 BEHOLD and ENVISION studies of UBX1325 in DME and AMD patients, respectively.
