A phase 2b study of Longeveron’s stem cell therapy in a pediatric heart disease has missed its primary endpoint, prompting the cash-strapped biotech to “review all options” for the business.
The Elpis II trial studied laromestrocel in 40 infants with hypoplastic left heart syndrome. Patients received laromestrocel as an adjunct to stage 2 palliative surgery, reflecting Longeveron’s belief that the investigational stem cell therapy could address the underlying cause of right ventricular failure in infants with the congenital heart birth defect. A control group received no drug during the surgery.
At Month 12, the change from baseline in right ventricular ejection fraction was statistically no larger in the treatment arm than in the control group, causing the study to miss its primary endpoint. Longeveron planned to seek FDA approval and land a partnership if the data were positive.
The company will continue to analyze exploratory clinical endpoints and plans to discuss a potential path forward with the FDA. As-treated analyses showed no patients died in the 12 months after receiving the study drug, compared to one person in the control group.
There were 12 major adverse cardiovascular events (MACEs) in the treatment group, versus 19 in the control cohort. However, the MACE analysis fell short of statistical significance. Similarly, a composite endpoint of all-cause mortality and the duration of inpatient hospitalization missed the bar for statistical significance. No treatment-emergent adverse events were ruled to be related to laromestrocel.
Longeveron’s stock fell 60% to $2.70 in after-hours trading Wednesday in the wake of the readout.
Following the fail, Longeveron outlined plans to “implement cash conservation measures to optimize cost containment.” In parallel, the biotech will engage an investment bank to serve as a strategic advisor as it reviews “all options with the goal of maximizing shareholder value.”
The company ended June with $10.1 million in cash and cash equivalents, a sum it predicted would fund operations into the fourth quarter. Longeveron extended its cash runway beyond the Elpis II readout in March through a private placement that provided $15 million upfront, with the chance of a further $15 million tied to the phase 2b data.
Having bet its future on Elpis II, Longeveron now plans to seek funding to advance laromestrocel in longevity and aging-related frailty. The therapeutic area was one of four fields on Longeveron’s roadmap for 2026. A phase 2b trial, which ended in 2021, in the indication missed its six-month primary endpoint, although the company pointed to longer-term outcomes to make the case for laromestrocel.
The biotech has navigated its recent money pressures amid turnover at the top of its financial team. Lisa Locklear stepped down as chief financial officer in July. Longeveron appointed Marie Washburn as its next CFO, only to replace her with Nirav Jhaveri the following month. Washburn returned to her role as corporate controller after Jhaveri’s appointment.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.