Full Press Release Details
Cell-Based Therapies
Longeveron Announces
Positive Results of Phase I Clinical Study of Lomecel-B Cell Therapy for Alzheimer's Disease
Study meets primary safety endpoint; positive
secondary efficacy assessments support potential benefit from Lomecel-B
Decline in cognitive function slower in patients
who received low-dose Lomecel-B as compared with placebo
Quality of life metrics improved with Lomecel-B
Complete results being prepared for publication.
On track to initiate Phase 2 study in the second
Miami, Florida-April 13, 2021-
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
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
"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."
About Alzheimer's Disease
Fifty million people worldwide currently have
dementia, which is a progressive decline in cognitive functioning that can interfere with daily activities, and eventually lead to a loss
of independence and death over the course of years. The most common form of dementia is Alzheimer's disease, which is characterized
by pathological hallmarks or beta amyloid deposits (also called "plaques") and neurofibrillary tangles in the brain. Some
early signs of Alzheimer's disease may include: memory loss; confusion about time and places; impaired problem solving and planning
abilities; difficulty with speaking, writing clearly and completing familiar tasks; impaired ability to understand visual images and spatial
relationships; and mood or personality changes. In the US alone, approximately 5.4 million people suffer from Alzheimer's disease,
and this number is projected to increase to 14 million by 2050 according to the Alzheimer's Association, a leading non-profit organization
for dementia research. The cost of care for these patients is already stressing healthcare systems, not to mention the enormous toll it
takes on families and care-givers.
There is thus an urgent need for finding effective
treatments for Alzheimer's disease and related dementias. However, there are currently no approved medications that can slow-down,
stop, or reverse the progression of Alzheimer's disease. Only 5 medications have received FDA approval for Alzheimer's disease,
and none significantly alter the course of disease progression. Three of these (donepezil, galantamine and rivastigmine) fall into a class
of drugs called "cholinesterase inhibitors," and another, memantine, is in a class of drugs called "NMDA receptor antagonists."
All four of these work by altering the communication between the cells of the brain (these are called neurons). The fifth medication is
a combination of two of these drugs (donepezil and memantine). With the exception of these above 5 drugs (which do not appear to ameliorate
disease progression), we have witnessed decades of failed attempts to develop small molecule drugs for treating Alzheimer's disease.
Potential of Lomecel-B for Treating Alzheimer's
Medicinal Signaling Cells (MSCs), as the main
ingredient of Lomecel-B, have numerous mechanisms of action that may potentially treat the complex pathology associated with Alzheimer's
disease. Beyond the hallmarks of beta amyloid deposits (plaques) and neurofibrillary tangles, Alzheimer's disease is also characterized
by inflammation in the brain (referred to as "neuroinflammation"), poor functioning of the blood vessels of the brain ("neurovasculature
dysfunction"), and degeneration of brain cells ("neurodegeneration"), among other features. The properties of MSCs can
potentially treat all of these aspects of Alzheimer's disease pathology, and preclinical studies support this conclusion. Using
mouse models of Alzheimer's disease, MSCs were shown to be able to decrease inflammation in the brain, promote break-down and clearance
of A (the protein component of beta amyloid), decrease the protein that causes neurofibrillary tangles, promote new nerve cell formation
("neurogenesis"), and improve cognitive/behavioral performance.
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, the Metabolic Syndrome, Acute Respiratory Distress Syndrome (ARDS),
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. Additional information about the Company is available at www.longeveron.com.
Forward-Looking Statements
Certain statements in this press release that
are not historical facts are forward-looking statements that reflect management's current expectations, assumptions, and estimates of
future performance and economic conditions, and involve risks and uncertainties that could cause actual results to differ materially from
those anticipated by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology
such as "believe," "expects," "may," "looks to," "will," "should," "plan,"
"intend," "on condition," "target," "see," "potential," "estimates," "preliminary,"
or "anticipates" or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events,
circumstances, or effects. Moreover, forward-looking statements in this release include, but are not limited to, statements about the
ability of our clinical trials to demonstrate safety and efficacy of our product candidates, and other positive results; the timing and
focus of our ongoing and future preclinical studies and clinical trials; the size of the market opportunity for our product candidates,
the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates; our ability to obtain and maintain
regulatory approval of our product candidates, our plans and ability to obtain or protect intellectual property rights, including extensions
of existing patent terms where available and our ability to avoid infringing the intellectual property rights of others. Further information
relating to factors that may impact the Company's results and forward-looking statements are disclosed in the Company's filings with the
SEC. The forward-looking statements contained in this press release are made as of the date of this press release, and the Company disclaims
any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new
information, future events, or otherwise.
Crescendo Communications, LLC