Israeli company nears final hurdle to okay insulin-based preemie therapy
Elgan Pharma uses insulin’s growth factor properties to help the gastrointestinal systems of the tiniest babies develop more quickly and effectively — and get them home sooner
Renee Ghert-Zand is the health reporter and a feature writer for The Times of Israel.
One in 10 babies worldwide is born before the end of 37 weeks of gestation, resulting in 15 million premature births every year. Complications from these births lead to the death of a million of these children by age 5 annually.
Elgan Pharma, a Nazareth-based biotechnology company, aims to save as many of these children as possible by bringing to market an insulin-based oral therapeutic product that will help the gastrointestinal (GI) systems of these “preemies” develop more quickly and effectively.
Critically, the therapy could reduce the incidence of necrotizing enterocolitis, a life-threatening condition with mortality rates as high as 42% in the smallest babies.
Earlier this summer, the company was awarded a grant of 2.36 million euros ($2.5 million) by the European Innovation Council to support the Phase III registrational study of the oral formulation, called ELGN-GI. The clinical study will begin by the end of this year at more than 30 partner centers in Israel, Europe, and the US. There has also been interest from New Zealand and Australia.
The Phase III trial will involve preemies, while a related Phase II trial will involve micro-preemies, or extremely fragile babies born before the 26th week of pregnancy.
Elgan Pharma founder and CEO Miki Olshansky told The Times of Israel her company is well on the way to overcoming American and European regulatory hurdles within the next couple of years.
“If you look at neonatology almost nothing gets approved. It’s very, very difficult… The last time that a drug was developed specifically for neonates was two decades ago. The authorities are very careful with this population,” Olshansky said.
While research to develop ELGN-GI began a decade ago, the initial idea of using insulin to encourage the development of a premature baby’s GI tract goes back to studies done on rats 20 years ago by Prof. Naim Shahadeh of Rambam Medical Center and Prof. Raanan Shamir of Schneider Children’s Medical Center.
“Insulin manages sugar overall in the body, and it also functions as what we call a growth factor. It supports and augments growth, particularly the growth of blood vessels in the gut and elsewhere. The insulin does this because it is not absorbed. It stays in the gut,” explained Prof. Eric Shinwell, director of neonatology at Ziv Hospital in Safed.
The biggest problem for preemies in terms of their survival is their lungs and heart, but they also need functioning intestines to grow and be healthy. However, their intestines are not ready to absorb all the food they need and to excrete what is not needed.
Premature babies are generally too underdeveloped to coordinate sucking, swallowing, and breathing, so they must be fed by a tube introduced through their nose or mouth to reach their digestive tract. This is usually not sufficient, because their immature digestive tracts lead to nutritional malabsorption.
As a result, the babies are put on a central line (parenteral nutrition or PN) through a vein in their chest or their umbilicus so that nutrition can be delivered directly to the bloodstream. This in turn can cause a variety of complications, especially life-threatening infections.
“ELGN-GI is designed to help get preemies off these feeding tubes earlier. If their GI tracts develop more quickly they’ll be able to move on to normal feeding, grow faster, and leave the neonatal intensive care unit and hospital a significant number of days earlier than they would otherwise,” Olshansky said.
Shinwell said that ELGN-GI, an easily dissolvable powder, has advantages because it is made with human recombinant insulin, thereby not introducing a foreign protein.
Olshansky said she expects that since her company has already demonstrated to regulators that its formulation causes no harm and that there are no safety concerns, it will be easy to recruit parents to the upcoming final study. The main trial will need 400 infants and the one for micro-preemies will need 60-100.
“There’s no blood tests this time around other than for standard of care. Nobody likes their child being pricked and blood taken for no good reason. And this also means that the workload will be less for the staff and researchers at the trial centers,” she said.
Noting the international team of researchers Elgan Pharma is working with, Shinwell praised the company for attempting to scale up and go commercial with an idea that until now has only been explored in small studies.
“Is this a revolution? No. Is it an addition [to current feeding supplements] that may well reach the stage of being clinically useful? Yes. It’s not there yet. But this is showing promise,” he said.
Separately, Elgan Pharma is developing another insulin-based therapy aimed at preventing retinopathy in preemies. ELGN-EYE are specially formulated eye drops employing insulin as a growth factor to prevent immature ocular blood vessels from becoming damaged and causing visual impairment or even blindness.
The Times of Israel Community.