Shares in Eledon Pharmaceuticals Inc. fell nearly in half to $2.06 on Nov. 7 after its Phase 2 trial to prevent organ transplant rejection after a kidney transplant failed to meet its primary endpoint.
The Irvine-based clinical-stage biotech company’s lead drug candidate, tegoprubart, did not demonstrate statistically superior kidney function compared to the standard of care, tacrolimus.
Company officials, however, remain confident that it can get approval.
“We met the composite endpoint,” Chief Executive Dr. David-Alexandre “DA” Gros told the Business Journal. “If we were to meet that composite endpoint in a Phase 3 study, then that would support approval of the drug.”
The study, which included 127 participants, showed that participants experienced fewer side effects, such as hypertension and heart failure, with tegoprubart than with tacrolimus.
Even with the disappointing results, Eledon a few days later was able to raise $53.6 million in a public offering, allowing the company to launch a Phase 3 study in the second half of 2026 and ultimately obtain FDA approval.
The company issued 30.3 million shares and pre-funded warrants at $1.65 each. At press time, the shares traded at $1.44 and a $108 million market cap (Nasdaq: ELDN).
The company’s goal is for tegoprubart to eventually be the lead drug for all transplants, regardless of what organ, according to Gros.
It has active trials in two other areas of research, including islet cell transplantation, a procedure that helps restore insulin production in patients with Type 1 Diabetes, and xenotransplantation, the transfer of animal organs or tissues into humans.
Fewer Side Effects
Despite not meeting its primary endpoint, the company said that tegoprubart was found to be non-inferior to tacrolimus. It achieved 22% compared to 17% in an efficacy failure composite endpoint comprising death, graft loss and acute rejection.
“Where we really showed was on the safety,” Gros said. “Our drug appeared much safer and better tolerated overall than tacrolimus.”
One of the main complaints physicians have about tacrolimus, Gros said, is that it can cause patients to develop new onset diabetes.
In Eledon’s Phase 2 Bestow trial, one in six patients on tacrolimus developed diabetes within one year, compared to one in 47 patients receiving tegoprubart.
Additionally, about 25% of the tacrolimus group reported tremors versus just 1.6% in the tegoprubart group.
There hasn’t been much advancement in the case of transplant care, according to Gros.
“We treat transplant patients with the same medicines that we were using 30 years ago,” he said.
The current standard of care, tacrolimus, was approved in the U.S. in 1994 as a more effective alternative to cyclosporine for preventing acute rejection after kidney transplantation.
“The ultimate goal is to replace tacrolimus and do what tacrolimus did to cyclosporine,” Gros said.
$50M to Fund Islet Cell Transplant Work
The company’s recent $50 million raise will also help finance its concurrent islet cell transplant work, Gros said.
Last week, Eledon announced preliminary data from Type 1 diabetes patients in a Phase 1/2 trial who received an islet cell transplant and were treated with tegoprubart.
All six patients achieved and maintained insulin independence after one or two Islet transplants, according to the company.
Gros said that they’re aiming to enroll three more patients in the fourth quarter of this year.
“In a year from now, we could be in registrational track studies in both islet cell transplantation as well as kidney transplantation,” he said.
