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NIA Small Business Showcase: Longeveron, LLC

Banner signifying OSBR handbookLife expectancy has increased substantially over the last century. However, this increase has not been paralleled by an increase in health span—the period of living in generally good health. Ironically, longer life expectancy allows chronic diseases to develop, while physical and cognitive functions decline, predisposing older people to disability or dependency. There is thus a pressing need to develop geroscience therapeutics that can improve both health span and life expectancy.

Longeveron written out with Biological Solutions for Aging written underLongeveron’s lead therapeutic candidate is Longeveron mesenchymal stem cells (LMSCs), an allogeneic product derived from bone marrow of young, healthy donors. LMSCs have the potential to be an off-the-shelf geroscience therapeutic candidate that can easily be administered in an outpatient setting in under an hour, with a single treatment’s effects effects potentially lasting up to a year. Under investigational new drug (IND) applications approved by the U.S. Food and Drug Administration (FDA), LMSCs are now undergoing rigorous evaluation in clinical trials for several aging-related conditions.

These indications include aging frailty, Alzheimer’s disease and related dementias (ADRD), and metabolic syndrome (MetS). Aging frailty is a biologically driven geriatric syndrome of multisystem physiological decline that disproportionately increases vulnerability to disease and debility. The prevalence of aging frailty is above 10% in people age 65 and older and is of great concern to geriatricians, who view it as a potentially treatable condition that is not an inevitable consequence of aging. Likewise, ADRD is viewed as a treatable condition, as is MetS, a chonic syndrome that can lead to cardiovascular disease and type 2 diabetes. While there are no FDA-approved therapies for preventing or reversing these chronic age-related disorders, LMSCs, due to their pleitropic mechanisms of action, offer high promise for all of these and other aging-related conditions.

In the United States, an overall frailty prevalence rate of 10% in adults 65 and older translates to more than 3 million people, with another 21 million considered at risk for becoming frail (pre-frail). According to a 2018 report by IQVIA, if the U.S. heart failure market were used as a comparator, given that heart failure has roughly the same annual prevalence as does frailty at the higher end, the estimated market size would be about $20 billion.

More than 5.5 million Americans have AD and the number is expected to rise, barring significant medical breakthroughs. Market analysts typically estimate peak sales revenue for an effective AD therapy at between $2 billion and $5 billion that future peak sales of any one disease-modifying investigational AD therapy could reach $2 billion annually; a positive Phase III trial would immediately bring a company’s value to more than $10 billion.

Despite being relatively young, Longeveron is a clinical-stage company with definitive market advantages. It is already a recognized leader in regenerative medicine and aging research and is conducting one of the most advanced multicenter interventional clinical studies for aging frailty. In addition, Longeveron is one of the few regenerative medicine companies with its own good manufacturing practice (GMP) production facility, allowing the company to preserve and optimize its proprietary process, control inventory, and maintain quality.

Company Milestones

Scientific

  • 2015–present: Filed five U.S. INDs and initiated six clinical trials
    • 2016: Initiated a 30-subject Phase I trial for Alzheimer’s disease
    • 2017: Initiated an 83-subject Phase I/II trial for immunosenescence in frailty subjects receiving the influenza vaccine
    • 2017: Initiated a 150-subject Phase IIb trial for age-related frailty syndrome
    • 2018: Initiated a Phase I trial in metabolic syndrome substudy
    • o 2018: Initiated a 10-subject Phase I trial for hypoplastic left heart syndrome
    • 2020: Initiated a 35-subject trial for acute respiratory distress syndrome (ARDS) due to COVID-19 infection
  • 2017: Published two manuscripts for Phase I/II aging frailty clinical trial results
  • 2017: Received approval from the Bahamian Ministry of Health to sponsor a treatment registry for aging frailty
  • 2019: Completed a Phase I trial for immunosenescence in frailty subjects receiving the influenza vaccine
  • 2019: Completed enrollment in two trials:
    • Phase I trial for Alzheimer’s disease
    • Phase I trial for hypoplastic left heart syndrome
  • 2020: Completed enrollment in a Phase IIb trial for aging frailty syndrome
  • 2020: Treated the first COVID-19–related ARDS patient under an FDA emergency use authorization
  • 2020: Received Japanese Pharmaceuticals and Medical Devices Agency approval to initiate a Phase II trial for aging frailty patients
  • 2020: Treated the 350th subject from a product made in Longeveron’s GMP facility, with no treatment-related adverse events reported
  • 2020–2021: Expects top-line data readouts for five clinical trials
  • 2020: Is initiating a Phase II trial on aging frailty in Japan
  • 2020–2021: Will be advancing multiple programs into later-phase clinical research, depending on data from Phase I and II trials

Business

  • 2015: Received $25 million in Series A financing
  • 2016–2019: Received more than $10 million in nondilutive funding from NIA, the Alzheimer’s Association, and the Maryland Stem Cell Research Fund through TEDCO
  • 2017: Opened a state-of-the-art cell manufacturing facility
  • 2018: Launched a revenue-generating contract manufacturing business unit
  • 2017–present: Is successfully sponsoring an expanded access program in Nassau, Bahamas
  • 2019–2020: Initiated the next round of equity financing to raise up to $25 million to fund later stage trials

Financial Overview

Longeveron is a private company established with initial equity funding of $25 million. It has since received more than $10 million in extramural grant funding from NIA, the Alzheimer’s Association, and the Maryland Stem Cell Research Fund through TEDCO.

Longeveron is raising additional capital to fund the next phases of clinical development in the United States, Asia, and other territories. The target funding amount is up to $25 million.

Intellectual Property

Longeveron’s intellectual property includes exclusive patent license agreements, exclusivity licensing of stem cell technologies, proprietary manufacturing processes, pending patent applications, and INDs that can lead to FDA-provided exclusivity incentives. Longeveron is also actively developing its pipeline of next-generation regenerative medicine products.

Product Development and Regulatory Strategy

Longeveron’s product development work occurs in-house with a team of scientists and production specialists at the company’s GMP facility and research and development laboratories. Biomarker analysis, potency assays, product characterization, quality control, and quality assurance are central to Longeveron’s product development efforts. Adhering to multicountry regulatory requirements for cell therapies, Longeveron works with regulatory agencies to develop products intended for commercial licensing and distribution. In its almost six-year history, the company has successfully filed five U.S. INDs and one Japanese IND, made products for six U.S. trials, filed several emergency use INDs, completed four treatment registry trials, and performed contract manufacturing for multiple clients.

Commercialization Strategy

Longeveron continues to work with regulatory agencies toward advancing its clinical programs to market authorization in the United States and internationally. Subsequent commercialization will involve long-term strategic partnerships, and Longeveron has already drawn the interest of large commercial-stage pharmaceutical companies. The company’s ultimate pathway to commercialization will be based on the best interests of all stakeholders.

Company Details

Longeveron, LLC website

1951 NW 7th Avenue, Suite 520
Miami, FL 33136

Industry: Therapeutics

Management Team:

  • President: Geoff Green, M.B.A., President
  • Co-Founder and Chief Science Officer: Joshua Hare, M.D.
  • Chief Financial Officer: James Clavijo, CPA
  • International Executive Director and General Counsel: Paul Lehr, J.D.
  • Senior Scientist: Anthony Oliva, Ph.D.
  • Senior Director, Manufacturing: Lisa McClain-Moss

Point of Contact:
Geoff Green, M.B.A.
Email Geoff Green
786-493-7687

Conference Selected for Showcase: BIO Digital (June 2020)