Longeveron (LGVN) Announces Positive Results of Phase I Clinical Study of Lomecel-B Cell Therapy for Alzheimer’s Disease

Longeveron Inc. (NASDAQ: LGVN) announced today the final results of its Phase I clinical study evaluating the safety and efficacy of intravenous (i.v.) administration of Lomecel-B, an allogeneic bone marrow-derived medicinal signaling cell (MSC) product, in subjects with mild Alzheimer’s disease. Preliminary results were previously reported in the Company’s S-1/A Registration Statement as part of Longeveron’s successful Initial Public Offering in the first quarter of 2021. The study met its primary safety endpoint, which paves the way for future trials in subjects with Alzheimer’s disease. Importantly, several pre-specified secondary efficacy endpoints and biomarker results support potential benefit from Lomecel-B. The complete trial results are currently being prepared for publication in a peer-reviewed journal, and will be posted on the Company’s website in the future. Longeveron also indicated they are on track to commence a Phase 2 study of Lomecel-B in Alzheimer’s disease in the second half of 2021.

The phase 1 trial, funded in part by an Alzheimer’s Association Part the Cloud Challenge on Neuroinflammation grant, used a randomized, placebo-controlled double-blind design testing single i.v. 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 were followed for 52 weeks post-infusion.

Key findings from new and previously disclosed data:

• 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. At 13 weeks after infusion, the low-dose Lomecel-B group MMSE score was higher (better) compared to placebo (difference of 2.69 ± 1.39 points; p=0.0182; 2-sided 95% CI 0.51 – 4.97);
• 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 Disease (QOL-AD) compared to placebo (difference of 3.85 ± 1.943 points; p=0.0444; 2-sided 95% CI 0.13 – 9.12);
• 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 placebo (difference of 6.95 ± 3.46 points; p=0.0118; 95% CI 1.99 – 13.94);
• Biomarkers:
  • Subjects receiving Lomecel-B had significantly increased serum levels of several pro-vascular biomarkers (VEGF, IL4, and IL-6) relative to the placebo group post-infusion.
  • There was a significant increase in D-dimer in the high-dose, but not low-dose, Lomecel-B arm versus placebo;
  • Subjects receiving Lomecel-B had significantly increased serum levels of several anti-inflammatory cytokines (sIL-2Rα, IL-4, IL-10 and IL-12) relative to the placebo group post-infusion;
  • There were no significant changes in the Lomecel-B arms versus the change in placebo for any of neuronal-related biomarkers examined;
  • MRIs showed a significant increase in change in left hippocampus volume in the high-dose Lomecel-B arm versus the change in placebo group at Week 13 (p=0.0311). By Week 26, the difference was no longer significant. The low dose Lomecel-B arm showed no significant difference versus placebo.

Geoff Green, Chief Executive Officer of Longeveron, stated, “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 second half of this year.”

“I am excited about the potential for this new approach to the treatment of Alzheimer’s disease. The use of Lomecel-B cells approaches therapy in a unique and novel way. We are treating the underlying problem of inflammation and possibly stimulating the brain to try to repair itself. These positive results, which includes improved patient reported outcomes, encourages us to continue to look at this approach to the treatment of this devastating degenerative brain disease,” said Barry Baumel, MD, of the University of Miami Miller School of Medicine and principal investigator on the study.

“The study results provide support for our hypothesis that Lomecel-B can potentially reduce Alzheimer’s disease associated brain inflammation, improve the function of blood vessels in the brain, reduce brain damage due to Alzheimer’s disease progression, and promote regenerative responses,” said Anthony Oliva, PhD, of Longeveron. Gary Small, MD, of Hackensack Meridian Health, a member of Longeveron’s AD Program Steering Committee, stated, “The results are encouraging that Lomecel-B has potential to eventually become a safe and effective treatment for AD.”

Regarding the MRI findings, Susan Bookheimer, PhD, of the UCLA School of Medicine, and also a member of Longeveron’s AD Program Steering Committee, commented that "while preliminary, the MRI results are very promising in suggesting that Lomecel-B may reduce brain damage due to Alzheimer’s disease progression, and promote regenerative responses."

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