UNITY Biotechnology, Inc. announced the release of positive data from its long-term follow-up on the phase 2 BEHOLD study for UBX1325 in patients diagnosed with diabetic macular edema (DME).
Give me a rundown of the company first.
UNITY Biotechnology is a San Francisco-based biotechnology company developing therapeutics for senescent cells to slow, halt, or reverse age-related diseases.
Through this, the company intends to provide transformative benefits for age-related ophthalmologic diseases.
Now UBX1325.
UBX1325 is a potent small-molecule inhibitor of Bcl-xL, part of the Bcl-2 family of apoptosis-regulating proteins. It is designed to hinder protein functions that senescent cells (cells that age and permanently stop dividing but do not die) rely on for survival.
The investigational compound is currently being studied for DME, age-related macular degeneration (AMD), and diabetic retinopathy (DR).
Talk about this trial now.
As a prospective, multi-center, randomized, double-masked, sham-controlled study, the phase 2 BEHOLD trial (NCT04857996) is assessing the safety, tolerability, and efficacy of a single 10 µg intravitreal injection of UBX1325 in DME patients.
And the patients?
The proof-of-concept study enrolled 65 patients who were treated with anti-vascular endothelial growth factor (VEGF) for at least 6 months prior to the start of the trial and had a visual acuity deficit (73 ETDRS letters or worse) and residual retinal fluid (center subfield thickness [CST] ≥300 microns).
Of note: all patients were given the option to continue into a 48-week, long-term extension study following the study’s initial 24 weeks; 50 patients completed the extension.
Any previous clinical data on it?
Previously reported 24-week data in the phase 2 study on UBX1325 indicates that a single injection of the compound could lead to a statistically significant and clinically meaningful improvement in the mean best-corrected visual acuity (BCVA) at 24 weeks (when compared to sham injection).
What’s the latest?
New 48-week data found that UBX1325 demonstrated a favorable safety and tolerability profile, with no reports of intraocular inflammation, endophthalmitis, retinal artery occlusion, or vasculitis.
Give me some numbers.
For BCVA, the mean change among patients treated with UBX1325 was +6.2 ETDRS letters from baseline to 48 weeks (p = 0.0037)—a difference of +5.6 ETDRS letters compared to sham.
Further, UBX1325 showed a +7.6 ETDRS letter advantage over sham (p = 0.0007), based on BCVA change from baseline to last observation prior to anti-VEGF rescue or end of patients’ study participation.
An estimated 53% of UBX1325 patients also went 48 weeks with no need for anti-VEGF rescue treatment (22% reported for sham).
What else?
UBX1325 patients showed a CST mean change of -16.6 microns (baseline to 40 weeks), indicating a -56.3 microns improvement over sham (p = 0.0479); at 48 weeks, the mean was 13.7 microns—a -37.9-micron improvement over sham.
Significance?
UBX1325 is currently the only treatment candidate in development with a therapeutic approach for targeting senescent cells to potentially modify age-related diseases.