Benzinga | Jun 28, 2021 02:00PM EDT

Longeveron, Armed with Positive Results of Phase I Clinical Study of Lomecel-B Cell Therapy for Alzheimer's Disease, Presenting at AAIC & Headed to Phase 2 Trials

Longeveron CEO Geoff Green presented at the Benzinga Global Small Cap Conference held on May 13-14, 2021. In addition to sharing the company's corporate outlook, Green participated in a panel entitled "Investing in Longevity Science: The New Drugs & Therapies Designed to Prolong Your Lifespan."

Longeveron Inc. (NASDAQ:LGVN), a clinical-stage biotechnology company developing cellular therapies for chronic, aging-related and life-threatening conditions, has successfully met its primary goals in advancing its Lomecel-B Alzheimer's disease research program.

The company reports its Phase 1 study has met the primary safety endpoint, meaning Longeveron can now move ahead with future trials evaluating the safety and efficacy of intravenous administration of Lomecel-B, an allogeneic bone marrow-derived medicinal signaling cell product, in subjects with mild Alzheimer's disease. Longeveron is now on track to begin its Phase 2 study in the 2nd half of 2021.

"We are pleased and encouraged by the results of this study, which indicate preliminary safety and potential efficacy of Lomecel-B in mild Alzheimer's disease, and we look forward to initiating our Phase 2 trial in the 2nd half of this year," Longeveron CEO Geoff Green said.

The Phase 1 trial used a randomized, placebo-controlled double-blind design testing single IV infusion of Lomecel-B 20 million cells ("low-dose"; (n=15)), Lomecel-B 100 million cells ("high-dose"; n=10)) or placebo (n=8). Subjects in the trial were followed for 52 weeks post-infusion.

Key findings included:

* Lomecel-B infusion was well-tolerated in this trial with no treatment-related serious adverse events observed throughout the 1-year follow-up, including no indications of amyloid-related imaging abnormalities (ARIA) as assessed by magnetic resonance imaging (MRI).

* The average Mini-Mental State Exam (MMSE) score, which is a measure of cognitive function, declined more slowly in the low-dose Lomecel-B group compared to the placebo group, with statistically significant differences at 13 and 39 weeks compared to placebo.

* At 26 weeks post-infusion, patients in the low-dose Lomecel-B arm showed a significantly higher (better) average score on the Quality of Life in Alzheimer's disease (QOL-AD) compared to the placebo.

* At 26 weeks post-infusion, the Alzheimer's Disease Cooperative Study Activities of Daily Living (ADCS-ADL), a measure of competence in basic and instrumental activities of daily living, was significantly higher (better) in the low-dose Lomecel-B group compared to the placebo.

Nearly 6 million people in the United States and 30 million globally suffer from Alzheimer's disease. More alarmingly, those numbers are expected to double by 2050. The Food and Drug Administration (FDA) has recently approved the first new medication for Alzheimer's in almost 20 years, Biogen Inc.'s (NASDAQ:BIIB) aducanumab, a monoclonal antibody that is supposed to reduce amyloid proteins from the brain, which is involved in the progression of Alzheimer's. By contrast, Lomecel-B targets neuroinflammation, among other possible mechanisms of action, which can cause neuronal cell death in key areas of the brain that atrophy with Alzheimer's disease. The Alzheimer's Association also agreed with this approach because they awarded Longeveron a total of $4M to fund the Phase 1 clinical study through two Part the Cloud Challenge on Neuroinflammation Grants. Lomecel-B is an allogeneic, bone marrow-derived medicinal signaling cell product manufactured under current good manufacturing practice regulations in Longeveron's cell-processing facility in Miami.

"Like Biogen's recent data showing a slowing of cognitive decline in mild Alzheimer's patients, Longeveron's Phase 1 study also demonstrated a slowing in cognitive decline, albeit in a much smaller trial. However, that's in part what makes this so exciting because we did observe statistically significant differences versus placebo in a small sample size and now we want to confirm these findings in a larger, phase 2 study" said Green. "Fortunately, our study results show no indication of brain edema, which is a problematic side effect that has been observed with monoclonal antibodies, so the safety profile for Lomecel-B continues to look very clean, and further testing is warranted in our opinion."

The company will present its study data at the Alzheimer's Association International Conference in Denver in July 2021.

For more information on Longeveron and its research, please visit www.longeveron.com.