With just over $10 million in cash available to fuel operations through the end of the year, Longeveron is undertaking a strategic review after the failure of a cardiovascular study.
Longeveron’s investigational stem cell therapy failed to improve blood flow in the heart during a mid-stage study of a rare congenital heart disorder, prompting the Miami biotech to “review all options” to maximize value for shareholders.
The biotech’s shares tumbled 57% to $2.86 in pre-market trading Thursday morning, despite the CEO’s assurance that the therapy laromestrocel will carry on in other indications—particularly longevity. The heart condition study will inform future development plans, CEO Stephen Willard said in a Wednesday statement.
Longeveron evaluated laromestrocel in the Phase 2b ELPIS II study, which enrolled 40 infants with hypoplastic left heart syndrome (HLHS), a rare and critical congenital defect in which the left side of the heart is malformed or underdeveloped.
HLHS disrupts normal blood flow through the heart, leading to difficulty breathing, a weak pulse and a bluish skin color. Patients typically require surgery soon after birth. About one in 3,846 infants in the U.S. are born with HLHS.
In ELPIS II, participants were randomly assigned to receive a single dose of laromestrocel to the heart, administered during surgery, or undergo standard-of-care surgery alone. Results showed that the stem cell candidate failed to significantly improve right ventricular ejection fraction at 12 months as compared with controls, which was the trial’s primary endpoint.
Longeveron however touted initial exploratory analyses, which found that over a 12-month period, none of the patients who were given laromestrocel died. In contrast, there was one death in the control group. The biotech also noted that one of 17 patients treated with the stem cell therapy needed transplantation over a follow-up window that lasted up to five years, as opposed to two out of 21 participants in the control arm.
Major adverse cardiovascular events, such as heart attack or stroke, also occurred 31% less frequently in the laromestrocel group—a finding that was not statistically significant.
As for safety, 94% of laromestrocel-treated patients developed treatment-emergent adverse events, versus 100% of control counterparts. The events were deemed serious in 65% of the patients in the stem cell group compared to 71% of the control arm. None of the side effects were deemed related to the study treatment by investigators. Longeveron did not report the adverse events that were observed.
Longeveron continues to evaluate data from the ELPIS II study and plans to bring its findings to the FDA, which according to the biotech is open to discussing the trial’s outcomes to potentially identify a path forward for laromestrocel.
The biotech touts laromestrocel as a “pipeline in a product,” with studies underway in pediatric dilated cardiomyopathy, Alzheimer’s disease and age-related frailty.
Left in a precarious spot by ELPIS II’s failure, Longeveron will conduct a strategic review with a goal of maximizing value for shareholders. As part of this effort, the biotech plans to “implement cash conservation measures to optimize cost containment,” the company said on Wednesday. BioSpace has reached out to Longeveron for more details on this savings push.
As of June 30, Longeveron had $10.1 million in cash and equivalents, enough to keep the company going into the fourth quarter of this year, according to a Q2 report last month.
The company intends to pursue additional funding opportunities to continue studying laromestrocel in longevity and age-related frailty, according to the release.
