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Cardiovascular Mesenchymal Cell Therapy Fast Tracked, Soon to Be Patented

Lomecel-B is being evaluated in the phase 2 ELPISII study in children with hypoplastic left heart syndrome.

Longeveron has received an intent-to-grant notice from the European Patent Office for its methods of using vascular biomarkers to monitor the efficacy of its Lomecel-B cell therapy.1 Lomecel-B is being investigated inseveral indications, including hypoplastic left heart syndrome (HLHS), for which the FDA has also just granted fast track designation.2

“We are extremely pleased to receive this notice from the European patent office,” Chris Min, MD, PhD, interim chief executive officer and chief medical officer, Longeveron, said in a statement.1 “This patent will bolster our robust intellectual property portfolio and support our goal of delivering effective cell therapies for a range of aging-related and life-threatening conditions.”

Lomecel-B is an allogeneic, bone marrow-derived, mesenchymal cell therapy. Longeveronis patenting its use vascular endothelial growth factor monitoring to assess response of Lemocel-B in patients with cardiovascular disease.

The therapy previously demonstrated its ability to improve vascular function and reduce endothelial dysfunction in 10 patients with HLHS undergoing stage 2 reconstructive surgery in the phase 1 ELPIS study (NCT03525418), the final data from which were released in September 2021.3 The study met its primary endpoint, with no major adverse cardiac events (MACE). Preliminary efficacy results were positive, with all infants alive and heart-transplant free after follow-up ranging from 2 to 3.5 years after cardiac surgery that included injection with Lomecel-B. These results contrast with the approximately 20% of patients that normally require heart transplants to survive within 1 year of stage 2 reconstructive surgery. A journal manuscript of the trial has been released and will undergo peer review before final publication.

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After the completion of ELPIS, Lomecel-B is now being evaluated in the same indication in the ELPISII phase 2 study (NCT04925024), which plans to enroll 38 participants to receive a 2.5 x 105 cell/kg dose. The primary endpoint is change in right ventricular ejection fraction (RVEF) at 12 months post-treatment and secondary endpoints include changes in right ventricular function and morphology, growth, clinical outcomes, quality of life, and blood biomarkers as well as safety over a 52-week follow-up.

The study is funded by a grant from the National Institute of Health’s National Heart, Lung, and Blood Institute in collaboration with Longeveron, and the principal investigator is Sunjay Kaushal, MD, PhD, Division Head, Cardiovascular-Thoracic Surgery, Ann and Robert H. Lurie Children’s Hospital of Chicago.

“Fast Track Designation represents a significant milestone in our efforts to develop Lomecel-B as a treatment for infants with HLHS,” Min said in another statement.2 “Fast Track Designation underscores the urgent need in HLHS, and we look forward to continuing to work closely with the FDA to bring this potential new therapy to infants as expeditiously as possible.”

In addition to the new fast track designation, Lomecel-B was also granted orphan drug and rare pediatric disease designation in November 2021 in HLHS. The same therapy is being evaluated for multiple other indications, including Alzheimer disease (AD), for which it is in phase 1 (NCT02600130) and 2 (NCT05233774) studies; aging frailty, in phase 1/2 (NCT02982915) and 2b (NCT03169231) studies; aging frailty with metabolic syndrome in a phase 2 study (NCT02587572); and acute respiratory distress syndrome in a phase 1 study (NCT04629105).

REFERENCES
1. Longeveron Receives Intent to Grant Notice from the European Patent Office for Methods to Monitor Efficacy of Lomecel-B™ Cell Therapy Through Levels of Vascular Biomarker. New release. Longeveron. August 25. 2022. https://www.globenewswire.com/news-release/2022/08/25/2504552/0/en/Longeveron-Receives-Intent-to-Grant-Notice-from-the-European-Patent-Office-for-Methods-to-Monitor-Efficacy-of-Lomecel-B-Cell-Therapy-Through-Levels-of-Vascular-Biomarker.html
2. U.S. Food and Drug Administration (FDA) grants fast track designation for Longeveron’sLomecel-B™ product for treatment of hypoplastic left heart syndrome (HLHS) in infants. News release. Longeveron. August 31, 2022. https://www.globenewswire.com/news-release/2022/08/31/2507637/0/en/U-S-Food-and-Drug-Administration-FDA-Grants-Fast-Track-Designation-for-Longeveron-s-Lomecel-B-Product-for-Treatment-of-Hypoplastic-Left-Heart-Syndrome-HLHS-in-Infants.html
3. Longeveron announces final results of phase 1 clinical study of Lomecel-B Injection in hypoplastic left heart syndrome patients. News release. Longeveron. September 9, 2021. https://www.biospace.com/article/releases/longeveron-announces-final-results-of-phase-1-clinical-study-of-lomecel-b-injection-in-hypoplastic-left-heart-syndrome-patients/