Longeveron Announces First Patient Dosed in Phase 2 Clinical Trial of Lomecel-B™ for Aging-Related Frailty in Japan

Rhea-AI Summary

Longeveron Inc. (NASDAQ: LGVN) has commenced its Phase 2 clinical trial for Lomecel-B™ in Japan, targeting Aging-Related Frailty in the elderly population. The trial aims to enroll 45 patients and primarily focuses on assessing the safety of Lomecel-B™. Longeveron anticipates that the data collected might support limited approval under Japan's Act on the Safety of Regenerative Medicine (ASRM). The study builds on positive results from a previous U.S. trial, where patients experienced significant improvements in mobility. The trial will be conducted in collaboration with prominent medical institutions in Japan, addressing a critical need in a country with a significant aging population.

Positive

• First patient successfully treated in Phase 2 trial for Lomecel-B™ in Japan.
• Study aims to support limited approval under Japan's ASRM, expediting access to regenerative therapies.
• Previous U.S. Phase 2b trial showed statistically significant improvements in walking distance post-infusion.

Negative

• None.

-- The study’s primary objective is to evaluate the safety of Lomecel-B™ in Japan’s elderly population --

-- First-in-human clinical trial of Lomecel-B™ in Japan --

-- Study will also assess efficacy endpoints expanding Longeveron’s database of Lomecel-B™’s physiologic effects in individuals of older age --

MIAMI, April 19, 2023 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing regenerative medicines for unmet medical needs, today announced that the first patient has been treated with Lomecel-B™ in its Phase 2 clinical trial in patients with Aging-Related Frailty in Japan. The trial aims to enroll 45 patients and its primary objective is to evaluate safety.

“We are excited to announce the dosing of the first patient in our Japanese Aging-Related Frailty trial,” said Wa’el Hashad, Longeveron’s Chief Executive Officer. “In 2022, Longeveron aligned with the Japan Pharmaceuticals and Medical Devices Agency (PMDA), on the Phase 2 trial design and we hope the data from this trial will provide support for a limited approval under Japan’s Act on the Safety of Regenerative Medicine (ASRM), which allows regenerative medicine products to be administered to patients by approved hospitals or clinics under the practice of medicine. This is the first use of our U.S.-manufactured product in Japan, and we look forward to advancing Lomecel-B™ as a treatment for Aging-Related Frailty in Japan.”

Hidenori Arai, M.D., Ph.D., President of the National Center for Geriatrics and Gerontology, and principal investigator of the Phase 2 trial, commented, “I am pleased to see this clinical trial program advancing, especially given the large unmet need in Japan, where nearly 30% of the population is over the age of 65. I was encouraged by Longeveron’s previously announced Phase 2b Aging-Related Frailty study in the United States, where subjects receiving a single infusion of Lomecel-B™ showed a statistically significant and clinically meaningful improvement in walking distance at 9 months post-infusion compared to placebo. I look forward to building on that data in the Japanese population.”

The Phase 2 clinical trial is a 3-arm, parallel design, randomized (1:1:1), placebo-controlled, double-blind single infusion study of two different dose levels of Lomecel-B™. The trial is expected to enroll 45 patients and has a primary objective of evaluating safety of Lomecel-B™ as a treatment for Aging-Related Frailty. The trial is being conducted in partnership with the National Center for Geriatrics & Gerontology (NCGG;Nagoya) and Juntendo University Hospital (Tokyo).

Japan is considered to be a “super-aged” society with some 36.4 million individuals aged 65 or older in 2021, representing 29.1% of the population. The overall prevalence of aging-related frailty amongst this demographic is estimated to be 7.9%.

About Aging-Related Frailty

Aging-Related Frailty is an age-associated decline in reserve and function across multiple physiologic systems leading to the inability to cope with stressors. It is characterized by mobility impairment, weakness, fatigue, weight loss, slowness, and low activity and puts individuals at higher risk for poor clinical outcomes, such as infections, falls, fractures, hospitalization, or death.

About Longeveron Inc.

Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company’s lead investigational product is Lomecel-B™, an allogeneic medicinal signaling cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. 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. Longeveron is currently advancing Lomecel-B™ through clinical trials in three indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s Disease, and Aging-Related Frailty. Additional information about the Company is available at www.longeveron.com.

About the Act on the Safety of Regenerative Medicine (ASRM)

The ASRM was passed in 2014 in order to provide incentives and accelerated approval pathways for regenerative medicine products in Japan. The act provides pathways for both conditional approval and limited use of products under the practice of medicine.

Forward-Looking Statements

Certain statements in this press release 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 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; the Company’s plans relating to the further development of its product candidates, including additional disease states or indications it may pursue; existing regulations and regulatory developments in the U.S., Japan and other jurisdictions; 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 the period over which it estimates its existing cash and cash equivalents will be sufficient to fund its future operating expenses and capital expenditures requirements. 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, 2021, filed with the SEC on March 11, 2022, and the Company’s Quarterly Reports on Form 10-Q. 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.

Investor Contact:

Stern IR, Inc.
212-698-8700
longeveron@sternir.com

Source: Longeveron Inc

FAQ

What is the primary objective of the Lomecel-B™ trial in Japan?

The primary objective is to evaluate the safety of Lomecel-B™ in patients with Aging-Related Frailty.

How many patients will be enrolled in the Lomecel-B™ Phase 2 trial?

The trial aims to enroll 45 patients.

What previous results support the Lomecel-B™ trial in Japan?

The U.S. Phase 2b trial showed significant improvement in walking distance for patients post-infusion.

What regulatory framework supports the approval of Lomecel-B™ in Japan?

The trial aims for limited approval under Japan's Act on the Safety of Regenerative Medicine (ASRM).

What is the significance of the aging population in Japan for this trial?

Japan has a large aging population, with about 30% over 65, highlighting the critical need for treatments for Aging-Related Frailty.