Longeveron Inc. (NASDAQ: LGVN), a clinical-stage biopharma company developing cellular therapies for aging-related illnesses, chronic disorders, and other specific life-threatening conditions, announced the publication of results from a Phase 1 trial testing Lomecel-B on patients with mild Alzheimer’s disease (AD) in Alzheimer’s & Dementia® : The Journal of the Alzheimer’s Association.
The trial met its primary endpoint, demonstrating that Lomecel-B was well tolerated in this patient population. In addition, the data provided indications supporting further exploration of Lomecel-B, particularly the therapeutic potential to slow cognitive decline and improve quality of life in patients with AD.
“We are pleased and encouraged by the publication of our study in this high-impact journal,” said Geoff Green, CEO of Longeveron. “The Lomecel-B study was funded by two competitive Part the Cloud Neuroinflammation Challenge grants awarded to Longeveron from the Alzheimer’s Association, for which we are extremely grateful.”
The article, titled “Results and Insights from a Phase 1 Clinical Trial of Lomecel-B for Alzheimer’s disease,” details the study findings on Longeveron’s lead investigational product, Lomecel-B, an allogeneic bone marrow-derived medicinal signaling cell (MSC) product, given to patients with mild Alzheimer’s disease.
“What makes this study unique is the use of a cellular therapy that may potentially target multiple pathological features of Alzheimer’s disease simultaneously,” said Dr. Mark Brody, lead author of the manuscript. “Not only did the study meet its primary endpoint of safety, but it also suggests the potential that Lomecel-B could have in treating patients with Alzheimer’s disease. We found data suggesting that Lomecel-B could have effects ranging from increasing blood vessel health to reducing inflammation, which could yield promising results.”
The double-blinded, randomized, placebo-controlled trial was conducted on thirty-three patients at four clinical sites. Each patient received a single intravenous infusion of low- or high-dose Lomecel-B, or a placebo. The findings from this study are being tested in a new larger Phase 2 study, which commenced in December 2021, and has already treated the first patient and continues to enroll others. The new trial is double-blinded, randomized, and placebo-controlled, and is designed to evaluate single versus multiple doses of Lomecel-B for mild Alzheimer’s disease.
The major findings of the Phase 1 study as reported in Alzheimer’s & Dementia are as follows:
- The primary endpoint — safety — was met. The Lomecel-B infusion was well-tolerated, and there were no treatment-related safety concerns. Importantly, patients receiving Lomecel-B were not reported to develop a serious side-effect called amyloid-related imaging abnormalities (ARIA) as assessed by magnetic resonance imaging (MRI). Other classes of Alzheimer’s drugs being tested have been reported to cause ARIA.
- Other exploratory findings included:
- The Mini Mental State Exam (MMSE), used to evaluate cognitive status, showed a slower decline in the low-dose Lomecel-B group versus the placebo group.
- The Quality of Life in Alzheimer Disease (QOL-AD), used to evaluate patient well-being, showed a significantly higher (better) score in the low-dose Lomecel-B group versus the placebo group at six months after the single treatment.
- Brain imaging by magnetic resonance imaging (MRI) showed an increase in left hippocampus volume three months after a Lomecel-B infusion in the high-dose Lomecel-B arm versus placebo. The hippocampus is a brain region critical for memory formation, which normally undergoes rapid neuron formation known as neurogenesis. This process is damaged in Alzheimer’s disease, leading patients to have reductions in the size of their hippocampus.
“We are encouraged by these results,” said Dr. Anthony Oliva, Senior Scientist at Longeveron, and principal investigator on the grants. “With a single dose of Lomecel-B, we observed several lines of preliminary evidence supporting multiple mechanisms of action of Lomecel-B, and most importantly, the data supports the potential of Lomecel-B as disease modifying for Alzheimer’s.”
The results of this study could have important implications for combatting this devastating disease that is also taking a significant toll on healthcare systems. According to a recent report by the Alzheimer’s Association, an estimated 6.5 million Americans age sixty-five and older are living with dementia due to Alzheimer’s disease. Seventy-three percent are age seventy-five or older. Of the total U.S. population, about 1in 9 people (11.3%) age sixty-five and older has dementia due to Alzheimer’s disease.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions. The Company’s lead investigational product is the LOMECEL-B™ cell-based therapy product (“Lomecel-B”), which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young, healthy adult donors. Longeveron believes that by using the same cells that promote tissue repair, organ maintenance, and immune system function, it can develop safe and effective therapies for some of the most difficult disorders associated with the aging process and other medical disorders. Longeveron is currently sponsoring Phase 1 and 2 clinical trials in the following indications: Aging Frailty, Alzheimer’s disease, and hypoplastic left heart syndrome (HLHS). The Company’s mission is to advance Lomecel-B and other cell-based product candidates into pivotal Phase 3 trials, with the goal of achieving regulatory approvals, subsequent commercialization and broad use by the healthcare community.