Eledon Pharmaceuticals’ experimental immunosuppressant helped two patients with type 1 diabetes go insulin-free following an islet cell transplant, the company announced Tuesday.
The company’s stock
$ELDN
rose about 12% on Tuesday morning after Eledon announced the
data
and an $85 million
offering
.
It said the investigator-initiated trial, which is being conducted by researchers at UChicago Medicine Transplant Institute, could have produced the first results showing that two patients became insulin-independent after undergoing treatment with an anti-CD40L monoclonal antibody and without taking tacrolimus, which is the current standard of care to prevent transplant rejections.
Tacrolimus, a type of immunosuppressant, comes with a laundry list of toxicity issues, including kidney and beta cell toxicity, post-transplant new-onset diabetes, hypertension, brain fog and tremors, according to Eledon CEO David-Alexandre Gros.
“We’re trying to get people off insulin, and yet, right when we’re giving them new islet cells, we’re giving them a drug that is going to harm,” Gros told
Endpoints News
. “We’re looking to develop a new generation of immunomodulator, one that will be both more effective as well as safer than the current standard of care.”
Gros pointed to Vertex’s work in stem cell-derived islet cell therapy — the company announced over the summer that three of its patients had gone insulin-free
using VX-880
— but patients also have to be on chronic immunosuppressive therapy. Vertex is working on VX-264, which is designed to treat patients without immunosuppressants, but Gros thinks there will be a place for Eledon’s drug, called tegoprubart, if VX-264 fails.
“What we’re hoping to do is to unlock this market if tegoprubart can provide as good or better protection but without all of the side effects,” Gros said. “And so far, the data is very encouraging.”
In the study, patients received islet transplants combined with induction therapy — mycophenolate mofetil plus tegoprubart — every three weeks by an IV infusion. The company said treatment with tegoprubart was “generally well-tolerated” in all subjects, with no unexpected adverse events, graft rejection or hypoglycemic episodes. A third patient decreased their insulin use by more than 60% three days after the transplant procedure.
Along with achieving insulin independence at approximately three months and six months post-transplant, the first two patients also recorded normal HbA1c levels. All three patients saw improved islet engraftment — engraftment was three to five times higher than three comparable subjects who received only tacrolimus — and Eledon said this suggests that tegoprubart is “less toxic” to transplanted islets and led to improved graft survival and function.
Back in 2020, Eledon bought tegoprubart through its acquisition of Anelixis, though the ALS Therapy Development Institute had discovered the drug. Eledon first ran a Phase 2 trial with the drug in ALS patients before pivoting to transplant rejection. It still has plans to develop the drug in ALS.
The company is continuing the trial in islet cell transplant, which is designed for up to nine patients, and it has three ongoing trials in kidney transplantation.
Editor’s note: This story has been updated with the name of the UChicago Medicine Transplant Institute.
