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Longeveron's Founders Hopeful That With Continued Clinical Trial Success, Lomecel-B May Win Regulatory Approval

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  • LGVN

Longeveron Inc. (NASDAQ: LGVN) is optimistic it could receive regulatory approvals in different countries for its lead regenerative medicine product Lomecel-B (cell-based therapy) after promising results from clinical trials.

The company has conducted (or is conducting) Phase 1 and 2 clinical trials in Aging Frailty, Alzheimer's disease, metabolic syndrome (a substudy in the US frailty trials), acute respiratory distress syndrome (ARDS) and hypoplastic left heart syndrome (HLHS).

Longeveron’s Founders: Mutual Interests that Led to a Biotech Company

The story behind the founding of Longeveron is interesting and occurred as a result of a relationship that developed back in 2013 between a wealthy, prominent real estate developer and philanthropist named Don Soffer, and a visionary cell therapy research scientist and clinician at the University of Miami, Dr. Joshua Hare.

Don Soffer, who is the man largely responsible for inventing the south Florida city of Aventura, had long been a champion of biomedical research focused on understanding the aging process, and learned of fascinating research conducted at the University of Miami led by Dr. Hare. Introduced by mutual friends, Mr. Soffer requested a meeting with Dr. Hare to learn more about the stem cell research in heart failure and frailty. This became the start of a long standing personal and business relationship—Mr. Soffer at the time had been considering potentially funding a start-up company that could be the vehicle to get this therapy to the market.

This all preceded the trend that has emerged with billionaires from silicon valley like Peter Thiel, Jeff Bezos and Larry Ellison investing in or starting anti-aging companies, including the highly prominent venture called Calico, a subsidiary of Google. So after some discussion and evaluation of several proposals, Longeveron began operations in 2014 with a $25 million equity investment, led by Don Soffer.

Real estate developer and philanthropist Don Soffer & cell therapy research scientist and clinician Dr. Joshua Hare

The funds were deployed to assemble a management team, initiate clinical trials, and build a state-of-the-art production facility where cells and other cellular constituents could be produced, packaged, and stored for clinical use. Longeveron has built a world-class research team, and has licensed technologies from the University of Miami.

Longeveron’s in-house scientists are working on platform technologies that are designed to solve key challenges in the field of cell-based therapy such as potency assays required by regulatory authorities and personalized medicine strategies to optimize cell products for various diseases. Armed with its new war chest post its February IPO, Longeveron is interested in developing important partnerships internationally to further its long term goals.

Lomecel-B: Completion of Phase 2b Clinical Study

In April this year, Longeveron announced completion of its Phase 2b clinical study of Lomecel-B infusion for aging frailty subjects in the United States. Lomecel-B is an allogeneic, bone marrow-derived medicinal signaling (MSC) living cell product manufactured in Longeveron's cell-processing facility in Miami.

A total of 149 trial subjects received a single peripheral intravenous infusion of Lomecel-B (25 million, 50 million, 100 million or 200 million cells) or placebo, followed by a 52-week observation period to evaluate safety and efficacy. The study's primary objective was to assess the effect of Lomecel-B treatment on exercise tolerance and endurance via the six-minute walk test (6MWT).

"Completing our Phase 2b clinical study of Lomecel-B Infusion to treat aging frailty marks a major milestone for the company. We are grateful to the exceptional clinical trial sites, our partner, the National Institute on Aging and, of course, the subjects for their participation in this study,” Geoff Green, Chief Executive Officer of Longeveron, said in a statement after the completion of the trials.

“We are committed to developing safe and effective cell therapies for chronic aging-related diseases and life-threatening conditions. We look forward to sharing the trial data later this year," Green said.

Longeveron, through its 7 year history, has developed core competencies in cell production techniques, potency analysis, and biomarker discovery. The core technologies pave the way for future platforms of cutting edge strategies to treat human diseases, and perhaps the aging process itself.

About Aging Frailty

Aging frailty is a life-threatening geriatric condition affecting approximately 15% of Americans older than 65 (or 8.1 million people). Aging frailty patients are vulnerable to poor clinical outcomes than their age-matched peers despite sharing similar comorbidities and demographics.

Aging frailty also is associated with chronic low-level sterile inflammation. Individuals with aging frailty have decreased reserves and a reduced ability to cope with minor illnesses or stressors that generally have a minimal impact, such as an infection or a fall. As a result, the individual is more likely to be hospitalized and may need long-term care or die. Frailty would be a new regulatory indication so further trials, and the potential pathway to a regulatory approval, will be dependent on the strength of the pending clinical data and discussion with regulatory authorities.

Trials In Japan

In addition to the United States, Longeveron is conducting trials in other countries. In June 2020, the company announced that Japan's Pharmaceutical and Medical Devices Agency (PMDA) approved a clinical trial notification application to initiate the Phase 2 clinical study. The company is working with the National Center for Geriatrics and Gerontology (NCGG) to evaluate the safety and efficacy of Lomecel-B infusion to treat aging frailty in Japanese patients.

The planned study is an investigator-initiated, randomized, double-blind, placebo-controlled design and is expected to initiate in the second half of 2021.

Regulatory Approval Process

Dr. Joshua Hare, Longeveron's co-founder and Chief Science Officer told Benzinga that the company is thrilled with the progress and momentum of the research and clinical trials so far.

Aging frailty is one of the most significant drivers for all Longeveron research and trials. Results from the company’s 3rd-quarter could be a game-changer, Hare said.

Thirty-three people took part in Longeveron’s Phase 1 Alzheimer trial, and the company is in the process of completing plans to rapidly initiate a Phase 2 trial, focused on efficacy and biomarker endpoints.

After completing Phase 1 and Phase 2 of some of the trials, Longeveron has been pleased with the safety profile to date, and the steady stream of results recorded so far, Hare said.

"We are hoping to take full advantage of accelerated regulatory pathways that may be available to us due to the 21st Century Cures Act passed by Congress in 2016,” Hare said. “As data from completed trials become available in the next 2 quarters, we have the opportunity to share it with the FDA and the Japanese PMDA and seek guidance on advancing to next steps in the approval process."

In March, the FDA granted expanded access approval for the administration of its investigational cell therapy Lomecel-B to a child with Hypoplastic Left Heart Syndrome (HLHS). The Phase 2 HLHS trial initiated enrollment on time and enrolled its first patient at Lurie Hospital in Chicago in June 2021.

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