Longeveron Inc (NASDAQ: LGVN) has edged nearer to the Nasdaq’s US$1.00 listing floor after its latest clinical setback. Shares tumbled 59 per cent on Thursday when the firm disclosed that its lead stem cell candidate failed to hit the main target in a mid-stage study for a rare heart defect in infants.
The trial tested the treatment, given during surgery, in 40 babies born with an underdeveloped left side of the heart. It did not improve the pumping strength of the right side after 12 months compared with surgery alone. Some secondary signs looked a little better, such as fewer deaths and fewer major heart events in the treated group, but these did not reach statistical strength. The therapy remained safe, with no new concerns.
The failure stung even more because the FDA had already told Longeveron that the trial’s main measure of how well the right side of the heart pumped was not a good way to prove the treatment worked. This study was unlikely to win approval no matter what the results showed.
The sharp fall comes only weeks after a 1-for-10 reverse split meant to lift the share price and protect the Nasdaq listing. That step has so far done little lasting good. The drop hurts most because the infant heart condition was Longeveron’s most advanced programme. Work on Alzheimer’s, age-related frailty and another childhood heart problem sits further behind. The company now plans to review options to try and protect shareholders. As of the end of June, Longeveron held just over US$10 million in cash, enough to fund operations only into Q4 against a backdrop of ongoing losses and limited resources on the balance sheet.
Other public firms that currently lead in stem cell approaches include Mesoblast Ltd (NASDAQ: MESO) (FRA: LWB1), which already has one approved product for a serious transplant complication; Capricor Therapeutics Inc (NASDAQ: CAPR) (FRA: 4LN2), advancing cell treatments for muscle and heart issues; and Fate Therapeutics Inc (NASDAQ: FATE) (FRA: F6T), developing immune cell treatments from stem cells. By contrast, Mesoblast’s approved stem cell product for a serious transplant complication brought in US$115 million in revenue last year, showing just how far Longeveron still has to go.
Stem cell research itself is still very limited. Doctors have long used certain stem cells to rebuild blood systems after cancer treatment. Most other applications stay experimental. Early trials in conditions such as Parkinson’s show cells can survive and may ease symptoms for some patients. Larger studies are needed, however, to prove lasting benefit and manage risks such as immune reactions. Progress continues, but widely available therapies beyond a few specialised uses remain scarce.
Read more: Breath Diagnostics advances pre-op pneumonia screening with FDA breakthrough designation
Follow Rowan Dunne on LinkedIn
rowan@mugglehead.com
