Longeveron's drug didn't help reverse aging frailty in a group of seniors, but the small, Florida-based biotech is nevertheless confident the trial has revealed some efficacy signals that could inform a later study with a higher dose.
And "later" is the operative word here: Executives say they are encouraged by some data that suggest an effect at Day 270.
Longeveron is studying Lomecel-B’s effects on 148 patients aged 70 to 85 years old with mild to moderate frailty. The primary goal was an assessment of exercise tolerance and endurance through a six-minute walk test. Additional goals included gait speed, grip strength, risk of falling, depression, sexual function, cognition and others.
The drug did not beat placebo on the six-minute walk test performed at Day 180, failing the main goal of the trial. But Lomecel-B did appear to induce a response—it just took awhile. Some efficacy was seen on a “pre-specified exploratory endpoint” when the six-minute walk test was performed at Day 270.
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On a secondary analysis, Longeveron found a dose-response relationship at Day 180 in patients treated with the therapy. Co-founder and Chief Science Officer Joshua Hare, M.D., said the company is “extremely pleased” with this result.
As for the additional endpoints, which mainly assess physical function, Lomecel-B "did not show significant differences versus placebo at most of the time points for any of the endpoints."
“Based on the results observed at 270 days, we believe that a larger and more extended trial would have a greater likelihood of achieving statistical significance,” Hare said during a second-quarter earnings call held Friday morning.
Lomecel-B is made up of medicinal signaling cells from the bone marrow of adult donors that are culture expanded at a Longeveron cell processing facility.
The company had caught the Biogen train back in June when the FDA approved controversial Alzheimer’s disease therapy Aduhelm. The green light has pushed shares of companies working in the space up, with each little news update typically adding to the trend. See: Eli Lilly.
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Longeveron, which is also developing Lomecel-B for the devastating neurological disorder, gained about 13% to $7.77 in June after the approval. The aging frailty trial failure, however, sent the shares downhill 9% to $4.92 as the markets opened Friday.
CEO Geoff Green provided an update on the Alzheimer's program during the earnings call as well, reviewing results from a phase 1 trial that were issued in April. Lomecel-B was found to be safe and tolerable, and no serious adverse events were observed.
Most notably, patients did not appear to have any imaging abnormalities after treatment. This has been a key concern with Aduhelm, which has been found to cause abnormalities on MRI scans that can signal swelling or bleeding in the brain.
“We view these results with cautious optimism due to the fact that it is a phase one trial with a small sample size,” Green said. But the company will move forward with a larger phase 2 study of Lomecel-B in Alzheimer’s later this year, he added.
It seems that for Lomecel-B, only time will tell.