The Medicines and Healthcare Products Regulatory Agency has issued a green light allowing Celixir to carry out a potentially pivotal Phase IIb human clinical trial with its stem cell therapy Heartcel.

The adult heart failure trial will be conducted at Imperial College London’s Royal Brompton, UK, and is expected to complete in 2020, which means that the therapy - Celixir’s lead investigational cardiac regenerative medicine - could be on the market in 2021.

Heartcel is a tissue-engineered medicine comprising allogeneic (off-the-shelf) immunomodulatory progenitor (iMP) cells. iMP cells are a novel cell type engineered to reduce heart scarring and regenerate the heart in patients with heart failure, and are currently delivered during coronary artery bypass graft.

The therapy has already shown strong promise in an earlier Phase II trail, in which Heartcel met all endpoints with statistical and clinically significant results; including 100 percent MACE-free survival (major adverse cardiac event); 30 percent average improvement in left ventricular ejection fraction; 40 percent reduction in left ventricle scar size; and 50 percent improved quality of life.

At the four-year follow-up, MACE-free survival remains 100 percent and imaging analysis evidenced scar reduction and heart regeneration in 70 percent of patients, the firm noted.

“Replicating these results in larger global trials with, could make Heartcel eligible for conditional marketing authorisation in Europe and FDA accelerated approval in the US,” noted the UK biotech, which was formerly known as Cell Therapy.

“Heart failure is still a major cause of death affecting millions of patients worldwide. Current therapies do not reduce chronic myocardial (heart) scarring, diminish disease progression or prolong life. Thus, the initial Heartcel trial results are very exciting, showing the first evidence in humans of disease reversal, scar reduction and heart regeneration,” said the firm’s chief medical officer Professor Stephen Westaby.

“We are assembling a world-class team at Imperial College, in the US and in Asia to accelerate patients’ access to this potential breakthrough in treatment of heart failure.”

Heart failure currently affects over 25 million adults worldwide. Up to 45 percent of patients in hospital with heart failure die within one year of admission, highlighting the urgent need for new treatment strategies.