Initial data from a clinical trial investigating Irvine-based drug research company Eledon Pharmaceutical Inc.’s tegoprubart shows promise as a breakthrough cure for Type 1 diabetes.
Two out of three people with Type 1 diabetes treated with tegoprubart, an antibody therapeutic, achieved insulin independence between three and six months post-pancreatic islet transplantation.
“Two of the three were able to go from very difficult to control diabetes to being insulin free, and the third was finally able to get their blood sugars under control in only three days,” Chief Executive Dr. David-Alexandre “DA” Gros told the Business Journal.
The news comes seven months after Massachusetts General Hospital used tegoprubart as a component of a treatment regimen following the first-ever transplant of a kidney from a genetically modified pig to a human being.
The procedure, performed on a 62-year-old man with an “end-stage kidney disease,” represents tegoprubart’s potential in preventing transplant rejection.
“The benefits that we saw originally in our animal experiments are now being replicated in humans,” Gros said.
Eledon last month also completed a financing of up to $85 million, which will go towards R&D and ongoing trials, including its Phase 2 Bestow clinical trial comparing tegoprubart to the standard of care with 120 participants.
Preventing Organ Transplant Rejection
In Type 1 diabetes, the immune system attacks beta cells in the pancreas, causing an individual to lose their ability to produce insulin.
One solution is islet cell transplantation, a minimally invasive procedure to help patients with Type 1 diabetes control their blood sugar by taking insulin-producing pancreatic cells from a deceased organ donor and infusing it into the patient’s liver.
After such transplants, immunosuppressant drugs are needed to prevent the body’s immune system from rejecting the organ.
Unlike traditional immunosuppressants that wipe out white blood cells, or the body’s natural defense system, tegoprubart stops the immune system from attacking itself by making it harder for certain immune cells to “activate,” according to Gros.
“What we’re doing is giving these individuals their islet cells again so they can start making insulin as if they no longer had diabetes,” he said.
Roots in ALS Research
Eledon traces its roots back to the late Orange County fitness exec Augie Nieto.
Nieto, the founder of Life Fitness, dedicated his life to finding a cure for amyotrophic lateral sclerosis (ALS) after being diagnosed with the disease in 2005.
Following his diagnosis, he and his wife Lynne founded a nonprofit called Augie’s Quest to Cure ALS to fund the discovery of a new drug candidate for ALS.
Tegoprubart, formerly known as AT-1501, is the culmination of years of research and experimentation by the ALS Therapy Development Institute with funding from Augie’s Quest.
It was licensed to Anelixis Therapeutics, a biotech company which sponsored the Phase 1 trials of the drug treatment in 2019. A year later, Anelixis was acquired by Eledon, called Novus Therapeutics at the time.
Nieto died in February 2023, but “his presence and memory continues to impact us today,” Gros said.
“His drive and spirit are key parts of what we do,” he said.
While Eledon has shifted its focus to organ transplantation, Gros said the company still “remains committed” to advancing the drug for ALS and is planning another trial once it secures enough funding.
“Our vision is to run a large ALS study that could help us know whether the drug is helping ALS patients or not,” Gros said.
