While we enjoy reporting positive news, we do occasionally have to report bad news, and today is one of those days. There has been some disappointing news from UNITY Biotechnology with the release of its Phase 2 senolytic trial of its candidate drug UBX0101.
SOUTH SAN FRANCISCO, Calif., Aug. 17, 2020 (GLOBE NEWSWIRE) — UNITY Biotechnology, Inc. (“UNITY”) [NASDAQ:UBX], a biotechnology company developing therapeutics to extend healthspan by slowing, halting or reversing diseases of aging, today announced the 12-week results from the Phase 2 study of UBX0101, a p53/MDM2 interaction inhibitor, in patients with moderate-to-severe painful osteoarthritis (OA) of the knee.
There was no statistically significant difference between any arm of UBX0101 and placebo at the 12-week endpoint for change from baseline in WOMAC-A, an established measurement of pain in OA. Given these results, UNITY does not anticipate progressing UBX0101 into pivotal studies and will narrow the company’s near-term focus to its ongoing ophthalmologic and neurologic disease programs.
A bit of perspective on these results
This is indeed disappointing news, but let’s put this into perspective. The pathway that UNITY used is only one of a number of known pro-survival pathways that senescent cells use to evade apoptosis. Due to the heterogeneity of senescent cell populations, it may be the case that the senescent cells in the knee joints of the trial participants were using other pathways to survive and that the ones using p53/MDM2 were either not present or insufficiently present to produce a significant effect.
This in no way invalidates the use of p53/MDM2 as a target pathway; rather, it highlights that we need better biomarkers of senescence in order to better understand what distribution of pathways the senescent cells in a given tissue are using in order to choose the ones most likely to elicit positive results.
It may also be the case that senescent cells do not play a significant role in the development and progression of osteoarthritis.
There are a number of reasons why this particular trial may have failed, and UNITY plans to continue developing and testing its other senolytic drugs, which target other pathways.
Science, particularly that relating to medicine and the biology of aging, is very hard, and there will almost certainly be many setbacks, failures, and bumps in the road before we have our first successes in humans. This news should remind us that we still have a long road ahead of us to end age-related diseases, but it is absolutely a goal worth striving for no matter how hard the challenge is or how many failures it takes before we get it right.