Angion Biomedica Corp. plans to soon begin enrolling a phase III study of BB3, an organ-protecting regenerative treatment it is testing in kidney transplant recipients with delayed graft function (DGF), a situation in which the organ fails to function properly, pushing patients onto dialysis after surgery.

Top-line data from the trial, called GIFT (Graft Improvement Following Transplantation), are expected in first quarter 2017. Positive results could validate the program's value as the company seeks to move a step closer to becoming a key player in a much bigger space, the market for acute kidney injury prevention.

DGF generally hits patients who have received suboptimal kidneys from donors who have died, or about 20 percent to 40 percent of transplant recipients. Despite the drawbacks, cadaveric kidneys offer an important option for the growing number of people waiting for a new kidney – a list that stood at 101,666 Americans on Thursday afternoon, according to the U.S. Organ Procurement and Transplantation Network. The longer a DGF patient requires dialysis, the greater the risk of the new kidney having diminished function and the lower the odds the organ will be viable.

"The waiting list for kidney transplants is long, and many waitlisted patients die on dialysis," said Hamid Rabb, a professor of medicine and medical director of Johns Hopkins University's kidney transplant program. "We are reluctant to use many marginal kidneys for our patients because of the increased risk of delayed graft function and reduced long-term graft survival," he said.

Angion president and CEO Itzhak Goldberg told BioWorld Today that "the hope is that if you can take some of the kidneys that are being discarded today because they're not good kidneys and turn them into better kidneys, that can potentially help a lot of patients that are sitting on the waiting list for transplantation, I think the impact can be quite significant."

Angion's most notable company in the DGF space is Alexion Pharmaceuticals Inc., which presented preliminary data from a phase II study of Soliris (eculizumab) to prevent delayed graft function in renal transplant patients at the 2015 American Transplant Congress earlier this month. But so far, no drugs are approved for DGF and no other competitors have reached phase III. Due to demand for a therapy, the FDA has granted both Soliris and BB3 orphan designations in the space. BB3 also has a fast track designation from the agency for renal transplantation.

The data on BB3, which the Uniondale, N.Y.-based company owns outright, has so far been encouraging. An interim analysis of a double-blind, placebo-controlled phase II trial of BB3 administered 24 hours after renal transplantation met its primary and secondary endpoints, improving urine output and lowering serum creatinine levels. It also reduced the duration of dialysis and shortened patients' hospital stays, the company said. The drug was well tolerated and no treatment-related serious adverse events were reported. Angion is submitting the data for presentation at the American Society of Nephrology 2015 annual meeting.

With those data in hand, a feat made possible in large part due to its receipt of more than $55 million in grants from the NIH, the company filed for an IPO in April last year, something that it plans to go ahead with this fall, Goldberg said. Meanwhile, it's in the middle of raising a series B crossover round prior to that. (See BioWorld Today, April 16, 2014.)

The company's broad strategy is to expand outward into the treatment of acute and chronic organ dysfunction overall. In line with that mission, it has filed an FDA investigational new drug application, seeking permission to conduct a multicenter phase II study of the drug to prevent acute kidney injury (AKI) following cardiac surgery, an event that's often associated with morbidity and mortality, especially among elderly patients. Enrollment in the AKI trial is projected to start in the second half of 2015, with a readout expected in the fourth quarter of 2016.