Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a
clinical stage biotechnology company developing cellular therapies
for chronic aging-related and life-threatening conditions, today
announced the final clinical results from its Phase I clinical
study of Lomecel-B in Hypoplastic Left Heart Syndrome (HLHS), a
rare and life-threatening congenital heart disease. Lomecel-B is an
allogeneic, bone marrow-derived medicinal signaling cell (MSC)
therapy product manufactured under current good manufacturing
practice (cGMP) in Longeveron’s cell processing facility in Miami.
The Phase I, open-label single arm study was
designed to assess safety and tolerability of intramyocardial
injection of Lomecel-B administered to 10 infants with HLHS during
Stage 2 bidirectional cavopulmonary anastomosis (BDCPA, or “Glenn
procedure”) surgeries. HLHS is a rare congenital heart defect that
affects approximately 1,000 babies per year in the U.S. Babies with
HLHS are born with an underdeveloped left ventricle, which impairs
the heart’s ability to pump blood throughout the body. HLHS is
fatal without surgical intervention, in which 3 surgical procedures
must be performed to allow the right ventricle to be configured to
pump blood to the body. Even with this surgery, HLHS is still
associated with a very high mortality rate and need for heart
transplantation. The trial was partially funded by a grant from the
Maryland Stem Cell Research Fund (MSCRF).
The primary safety endpoint was the incidence of
the following treatment-emergent Serious Adverse Events (TE-SAEs):
i) major adverse cardiac events (MACE), including
sustained/symptomatic ventricular tachycardia requiring
intervention with inotropic support, aggravation of heart failure,
myocardial infarction, unplanned cardiovascular operation for
cardiac tamponade, and death through one-year post-treatment; and
ii) infections during the first month post-treatment.
Intramyocardial injection of Lomecel-B at 2.5 × 106 cells/kg of
body weight was well-tolerated, with no MACE, and no infections
reported that were considered to be related to investigational
treatment.
Secondary endpoints were measured per protocol
to gain insight into Lomecel-B’s potential effect on clinical
outcomes and heart function. These secondary endpoint results
should be viewed with caution due to the lack of a control arm for
comparison, meaning we cannot conclude whether these secondary
outcomes were surgery-related, Lomecel-B-related, or both.
- 100% of infants treated were alive
and transplant-free one-year after injection. Patients have now
been followed for two-to-3.5 years and remain alive and transplant
free. This exceeds historical control results, which estimate only
78% survival free of transplantation following the Glenn procedure.
(1)
- Heart function and structure,
including tricuspid regurgitation fraction (the fraction of blood
going backwards in the right ventricle), right ventricle ejection
fraction (RVEF), RV volume and chamber size did not change
significantly from baseline at six and 12 months after Lomecel-B
injection.
- The children’s growth matched
trends in published literature, suggesting normal development of
the children. (2)
- Through one-year follow-up, one
patient had an ascending aortic obstruction requiring angioplasty,
and four other patients required re-hospitalization, none
considered related to Lomecel-B injection.
“We are gratified by the very promising finding
in this study that 100% of the participating children had a two to
four year transplant-free survival which compares very favorably to
published mortality rates of up to 22% in HLHS patients after the
Glenn procedure,” said Dr. Sunjay Kaushal, Principal Investigator
of the study. “We are highly motivated to continue our studies of
Lomecel-B cell therapy for children with HLHS. If we determine in
the future that Lomecel-B reduces the need for heart
transplantation in these children, this will represent a major
advance in the field, and have a substantial impact on these
infants and their families.”
“HLHS is a rare but life-threatening, congenital
heart defect for which we currently have limited treatment options
and poor outcomes. We are delighted to have been able to support
this clinical trial in Maryland and advance this important work
with Dr. Sunjay Kaushal and Longeveron”, said Dr. Amritha
Jaishankar, Executive Director of the Maryland Stem Cell Research
Fund. “We are committed to Accelerating Cures, and hope that
the results from the HLHS Lomecel-B cell therapy study that our
grant helped fund will bring us one step closer to helping these
babies and their families."
Phase 2 Trial (ELPIS II)
Longeveron is pleased that its research program
has advanced into a blinded, controlled trial that is evaluating
safety and effectiveness of Lomecel-B as an intervention for HLHS.
In this regard, the HLHS program has transitioned into a Phase 2
trial titled: Evaluation of
Lomecel-B™ Injection in
Patients
with Hypoplastic Left Heart
Syndrome: A Phase
IIb Clinical Trial. (ELPIS II)
ELPIS II is being funded by a grant from the National Institute of
Health’s National Heart, Lung, and Blood Institute (NHLBI; Grant
number 1UG3HL148318), in collaboration with Longeveron, and is led
by Principal Investigator Sunjay Kaushal, MD, PhD, Division Head,
Cardiovascular-Thoracic Surgery, Ann and Robert H. Lurie Children’s
Hospital of Chicago. With a target enrollment of 38 infants, the
trial began enrollment in July, 2021, and it is anticipated that up
to seven children’s hospitals will be participating, all in major
metropolitan centers located throughout the U.S. For more
information regarding the trial design and location of clinical
sites please visit www.clinicaltrial.gov (NCT04925024), and
www.elpistrial.org hosted by the University of Texas Health
Sciences Center which serves as the data coordinating center.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology
company developing cellular therapies for specific aging-related
and life-threatening conditions. The Company’s lead investigational
product is the LOMECEL-B™ cell-based therapy product (“Lomecel-B”),
which is derived from culture-expanded medicinal signaling cells
(MSCs) that are sourced from bone marrow of young, healthy adult
donors. Longeveron believes that by using the same cells that
promote tissue repair, organ maintenance, and immune system
function, it can develop safe and effective therapies for some of
the most difficult disorders associated with the aging process and
other medical disorders. Longeveron is currently sponsoring Phase 1
and 2 clinical trials in the following indications: Aging Frailty,
Alzheimer’s disease, the Metabolic Syndrome, Acute Respiratory
Distress Syndrome (ARDS), and hypoplastic left heart syndrome
(HLHS). The Company’s mission is to advance Lomecel-B and other
cell-based product candidates into pivotal Phase 3 trials, with the
goal of achieving regulatory approvals, subsequent
commercialization and broad use by the healthcare community.
Additional information about the Company is available at
www.longeveron.com.
References
- Son JS, James A, Fan C-PS, Mertens
L, McCrindle BW, Manlhiot C, Friedberg MK. Prognostic value of
serial echocardiography in hypoplastic left heart syndrome.
Circulation: Cardiovascular Imaging. 2018; 11(7):e006983.
- Burch, P. T., E. Gerstenberger, C.
Ravishankar, D. A. Hehir, R. R. Davies, S. D. Colan, L. A. Sleeper,
et al. 2014. “Longitudinal Assessment of Growth in Hypoplastic Left
Heart Syndrome: Results From the Single Ventricle Reconstruction
Trial.” Journal of the American Heart Association: Cardiovascular
and Cerebrovascular Disease. doi:10.1161/JAHA.114.000079.
Cautionary Note Regarding
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Research referred to in this press release is
being supported by the National Heart, Lung, and Blood Institute of
the National Institutes of Health under Award Number UG3HL148318.
The content is solely the responsibility of the authors and does
not necessarily represent the official views of the National
Institutes of Health.
Contact:Brendan PayneStern Investor
RelationsTel: (212) 362-1200Email: Brendan.payne@sternir.com
Source: Longeveron IncSource: LGVN
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