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Longeveron Inc. (NASDAQ: LGVN)
Longeveron Inc. (NASDAQ: LGVN) is a clinical-stage biotechnology company developing regenerative medicines to address unmet medical needs for specific aging-related and life-threatening conditions. The Company's research and therapies are aimed at improving the outcome of infants born with a life-threatening heart condition, as well as improving the healthspan for the aging population – the number of years a person is expected to live in relatively good health, free of chronic disease and disabilities of aging, with function and ability to perform activities of daily living.
Longeveron is involved in clinical trials in the following indications: Hypoplastic left heart syndrome (HLHS), Alzheimer's disease, and Aging-related Frailty.
The Company's philosophy revolves around the idea that regenerative medicine may hold the potential to improve certain rare medical conditions and contribute to healthy aging. While there has been a remarkable rise in life expectancy over the last century due to medical and public health advancements, this increase in longevity has not been paralleled by the number of years a person is expected to live in relatively good health, free of chronic disease and disabilities of aging.
Longeveron's lead investigational product is Lomecel-B™, an allogeneic Medicinal Signaling Cell therapy product isolated from the bone marrow of young, healthy adult donors. As humans age, they experience a decrease in immune system function, a decline in blood vessel functioning, chronic inflammation, and other issues. Clinical data has suggested that Lomecel-B™ may address these conditions through multiple mechanisms of action (MOA) that simultaneously target key aging-related processes.
The Company is headquartered in Miami, Florida.
Lomecel-B™
Lomecel-B™ is being evaluated in multiple clinical trials for aging-related chronic diseases and other life-threatening conditions under U.S. FDA-approved Investigational New Drug applications. Lomecel-B™ has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas.
The drug is made from special living cells called Medicinal Signaling Cells (MSCs) that are isolated from fresh bone marrow tissue that has been donated by adult donors aged 18 to 45. Once the MSCs have been isolated from the fresh bone marrow through a careful selection process, the cells are culture-expanded (allowed to replicate under controlled laboratory conditions) into the billions using specialized techniques and processes. After a specific number of expansion cycles, called "passages," the cells are harvested, separated into specific doses (e.g., 50 million cells), and cryopreserved until future use.
These cells have been shown to have characteristics that allow them to be transplanted from a donor to host without triggering a harmful immune response in the recipient, and they can be administered on an outpatient basis in as little as 40 minutes after thawing. Because of these characteristics, Lomecel-B™ is considered an "off-the-shelf" product.
In some trials, such as for Alzheimer's disease and Aging-related Frailty, Lomecel-B™ is administered via peripheral intravenous infusion, while, in the Company's HLHS trial, Lomecel-B™ is administered via direct injection into the heart tissue.
Market Opportunity
Longeveron estimates the potential market size for Lomecel-B™ in the treatment of HLHS to be up to $1 billion annually, globally.
U.S. patients suffering from Aging-related Frailty are estimated using U.S. Census Bureau statistics to be approximately 8.1 million. That population potentially represents a market for Lomecel-B™ of between $4 billion and $8 billion globally per year, according to Company estimates.
Additionally, the Alzheimer's Association puts the number of Americans with that disease at 5.1 million, highlighting another potentially addressable market for Lomecel-B™, that's worth $5 billion to $10 billion annually.
Management Team
Wa'el Hashad is CEO of Longeveron. He has more than 35 years of experience in the pharmaceutical and biotech industries. He has launched several successful brands in the U.S. and worldwide markets. Prior to joining Longeveron, he was president and CEO of Avanir Pharmaceuticals. Before Avanir, he was the chief commercial officer of Seres Therapeutics. He also has held senior leadership positions at Amgen, Boehringer Ingelheim, and Eli Lilly and Company. He holds a bachelor's degree in pharmacy from Cairo University and an MBA from the University of Akron.
Joshua M. Hare, M.D., FACC, FAHA, is Co-Founder, Chief Science Officer and Chairman of Longeveron. He is a double board-certified cardiologist and is the founding director of the Interdisciplinary Stem Cell Institute at the University of Miami's Miller School of Medicine. He is a recipient of the Paul Beeson Physician Faculty Scholar in Aging Research Award and is an elected member of the American Association of Physicians and The American Society for Clinical Investigation. He is also an elected Fellow of the American Heart Association. He received a bachelor's degree from the University of Pennsylvania and his M.D. from The Johns Hopkins University School of Medicine.
Lisa Locklear is CFO at Longeveron. She previously served as the senior vice president and CFO for Avanir Pharmaceuticals. Prior to Avanir, she held senior financial roles at GSN Games, CoreLogic, Ingram Micro, the Walt Disney Company, and Price Waterhouse, with assignments in Paris and London. She holds a bachelor's degree in plant science from the University of California, Davis, and an MBA from the University of California, Irvine. She is a licensed CPA (inactive) and is a member of the American Institute of Certified Public Accountants, the California Society of CPAs, and Financial Executives International.
Dr. Nataliya Agafonova, M.D., is the Chief Medical Officer at Longeveron. She previously served as clinical development lead, senior medical director, and product development chair at Otsuka Pharmaceuticals. Before that, she was the clinical development lead and senior medical director at Bristol-Myers Squibb. She previously held senior leadership positions at Ardea Bioscience, Biogen, Amgen, and Genzyme Corporation. She earned an M.D. from the Ukrainian National Medical University and completed her internal medicine residency at Kharkov State University Hospital in Ukraine.
Forward-Looking Statements
Certain statements in this corporate profile that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management's current expectations, assumptions, and estimates of future operations, 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. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, statements regarding the offer and sale of securities, the terms of the offering, about the ability of Longeveron's clinical trials to demonstrate safety and efficacy of the Company's product candidates, and other positive results; the timing and focus of the Company's ongoing and future preclinical studies and clinical trials and the reporting of data from those studies and trials; the size of the market opportunity for the Company's product candidates, including its estimates of the number of patients who suffer from the diseases being targeted; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of the Company's product candidates; the Company's ability to obtain and maintain regulatory approval of its product candidates in the U.S., Japan and other jurisdictions; the Company's plans relating to the further development of its product candidates, including additional disease states or indications it may pursue; the Company's plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and its ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and the Company's ability to attract and retain such personnel; the Company's estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Company's need to raise additional capital, and the difficulties it may face in obtaining access to capital, and the dilutive impact it may have on its investors; the Company's financial performance and ability to continue as a going concern, and the period over which it estimates its existing cash and cash equivalents will be sufficient to fund its future operating expenses and capital expenditure requirements. Additionally, Longeveron makes no assurance that any public offering of its securities as described herein will occur on the timelines, in the manner or on the terms anticipated due to numerous factors. 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 Securities and Exchange Commission, including Longeveron's Annual Report on Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission on March 14, 2023 and its Quarterly Report on Form 10-Q for the second quarter of 2023 filed with the SEC on August 11, 2023. The forward-looking statements contained in this corporate profile are made as of the date of this corporate profile, 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.
Investor Contact
Mike Moyer
LifeSci Advisors
Tel: 617-308-4306
Email: mmoyer@lifesciadvisors.com
Date prepared: August 31, 2023
Investment Considerations
- In June 2023, Longeveron filed with the SEC to offer subscription rights worth up to $30 million of shares of Class A common stock to stockholders and holders of warrants. The rights offering, which became effective on August 17, 2023, is being made through a distribution of five tradable subscription rights (listed on The NASDAQ Capital Market under the ticker symbol LGVNR) to purchase shares of Class A common stock for each share of common stock and warrant to purchase common stock owned as of the record date of August 18, 2023, at a $3.00 subscription price per share. The subscription period opened on August 22, 2023, and will continue until September 21, 2023, unless extended at the Company's sole discretion.
- During Q2 2023, the Company announced children in its ELPIS I Trial of Lomecel-B™ for HLHS had 100% survival up to five years of age after receiving the drug, as compared with an approximate 80% survival rate in historical trials. Lomecel-B™ for HLHS has previously received Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).
- In August 2023, the Company announced the Phase 2 ELPIS II trial, funded by the National Heart, Lung, and Blood Institute (NHLBI) and using the Company's Lomecel-B™ asset in Hypoplastic Left Heart Syndrome (HLHS), has surpassed the 50% enrollment threshold. Additionally, the trial has activated its eighth clinical site location – one additional site beyond the seven originally planned – in order to try to expedite the completion of the trial enrollment.
- The Company expects to share top-line results from its Phase 2a CLEAR MIND trial of Lomecel-B™ for the treatment of Alzheimer's disease in early October 2023.
- Longeveron is conducting a Phase 2 study of Lomecel-B in patients with Aging-related Frailty in conjunction with Japan's National Center for Geriatrics & Gerontology and Tokyo's Juntendo University Hospital.
Longeveron Inc. (NASDAQ: LGVN)
