Longeveron Inc.

LOMECEL-B: A product candidate with broad therapeutic potential

Lomecel-B is Longeveron’s lead investigational product candidate that is currently undergoing clinical testing for multiple medical indications. Lomecel-B is a cell-based product therapy that is derived from culturally expanded Medical Signaling Cells (MSCs) that are sourced from the bone marrow of young and healthy adult donors. MSCs are multipotent cells that are able to form into a variety of cells and tissues of the human body. These cells promote tissue repair, maintain organs, and assist in the proper functioning of the immune system. 

Lomecel-B cells have a high potential to address a range of aging-related declines by targeting the processes of aging through multiple methods of action (MOAs). Potential MOAs of Lomecel-B include:

• Release of proteins and other growth factors that reduce inflammation and stimulate a regenerative response from other nearby cells
• Engage in direct cell interactions to establish positive pathways between contacted cells
• Formation of nanotube bridges to allow the exchange of mitochondria and other cellular content between cells
• Release exosomes which contain cargo like RNA, proteins, and other molecules that can be absorbed by other cells for beneficial effects

Longeveron is currently carrying out clinical testing of Lomecel-B on four areas of therapeutic need. 

Lomecel-B for Aging Frailty

Aging frailty is an extreme form of unsuccessful aging where patients exhibit weakness, slowness, fatigue, unintentional weight loss, and low activity. It is a biological process that is distinct from normal aging patterns. Patients with aging frailty are especially at risk of poor outcomes like disability and death because their bodies are less capable of dealing with stressors like sickness and disease. Inflammation is thought to be a major contributor to the physical decline in those with aging frailty, which is why the anti-inflammatory properties of Lomecel-B could be an effective therapy. Around 10% of people aged 65 and above are burdened with aging frailty. However, there are no FDA-approved therapies currently on the market for the condition. 

Lomecel-B for Alzheimer’s Disease 

Alzheimers is a form of dementia that affects approximately 6.2 million Americans age 65 and older and is the nation's 6th leading cause of death. Brain inflammation is a key driver of Alzheimer’s and contributes to structural changes in the brain that help progress the disease. Lomecel-B is a promising treatment for treating Alzheimer's, as it has shown the potential ability to reduce brain inflammation, reduce Alzheimers-related brain damage, improve the function of blood vessel function, and promote regeneration. Lomecel-B for Alzheimer's is currently undergoing a pivotal phase 2 clinical trial. 

Lomecel-B for the Metabolic Syndrome

Metabolic syndrome is a cluster of conditions that occur simultaneously and increase the risk of heart disease, type 2 diabetes, and stroke. These conditions include excess body fat around the waist, abnormal cholesterol levels, increased blood pressure, and heightened blood sugar. Metabolic syndrome is extremely common in the United States, with around 33% of people developing the condition at some point, a figure that continues to increase. Lomecel-B has been pegged as a potential treatment for metabolic syndrome because of its ability to reduce inflammation and improve vascular function.

Lomecel-B for Hypoplastic Left Heart Syndrome (HLHS) 

HLHS is a severe birth defect characterized by a missing or underdeveloped left heart ventricle, resulting in the severely diminished function of the circulatory system. The condition is rare, with approximately 1,000 babies born with it each year in the United States. The currently available treatment for HLHS is a multi-year, three-stage heart reconstruction that leverages the remaining right ventricle to support blood circulation. Without this treatment, there is a 100% mortality rate for babies born with the condition. Although babies that undergo the heart reconstruction option usually live into adulthood, early mortality is very common. Lomecel-B is being tested in combination with heart reconstruction surgery as an enhanced therapeutic option that could potentially improve both short and long-term outcomes for patients. When tested on animals, this combination resulted in a 10% to 15% improvement in right ventricle function. Lomecel-B for HLHS is currently undergoing phase 2 trials. 

Operational overview

Longeveron Inc. has no products approved for commercial sale and therefore has generated no revenues from the sale of its therapeutic products. The company has funded operations through private and public equity financings, grant awards, and fees generated from clinical trial and contract manufacturing services. Longeveron will need to seek additional funding to continue its operation goals, which could come as debt financings, further equity sales, or the formation of collaboration agreements with third parties. 

Longeveron does have one leg up over many other small-scale pharmaceutical companies in that it developing its own manufacturing capabilities. Longeveron’s manufacturing facilities allow the company to efficiently develop its own products, as well as the ability to generate revenue from third parties seeking clinical manufacturing assistance. 

Conclusion

Longeveron’s Lomecel-B certainly has exciting therapeutic potential and seeks to address disease areas with major market opportunities. However, the company’s current reliance on a single product candidate is risky, especially if future medical insights find an issue with treatment by MSCs. Furthermore, the company faces substantial competition in the field of regenerative medicine from other companies like Athersys Inc. and Mesoblast. Although potential hurdles exist, Longeveron and its MSC-based product therapy show excellent promise and have made steady strides toward treating medical conditions that negatively impact human health and lifespan.