Staff Writer

Angion Biomedica Corp. is working to develop a drug that could be used to salvage marginally functioning kidneys that have been harvested from deceased donors and otherwise might have been discarded, potentially increasing the pool of patients receiving renal transplants.

The company's most advanced product, BB3, currently is undergoing Phase II testing to evaluate whether it can reduce delayed graft function in kidney transplant patients. Preliminary data are expected at the end of 2011. Phase I in normal volunteers and Phase II studies in renal dialysis patients have been successfully completed in preparation for the study in renal transplantation.

If successful, BB3 could help expand the pool of patients receiving kidney transplants, including those on dialysis, Itzhak Goldberg, president and CEO of Angion, told BioWorld Today. Roughly 17,000 to 18,000 kidney transplants are performed each year in the U.S., but many more are waiting for donors and others are on dialysis, representing a huge unmet need, he said.

Most transplants come from deceased donors but the percentage of living donors is increasing.

Founded in 1998, Angion has kept a low profile in that 12-year span as it continued to work on products that were undergoing preclinical research.

Ultimately, Angion would like to study BB3 as a potential treatment for kidney failure, which affects an estimated 700,000 patients a year in the U.S. But first, the company is aiming to show efficacy in the much smaller population of patients undergoing kidney transplants, explained Goldberg, founder of Angion.

The company said Tuesday that BB3 has received both orphan drug and fast-track status from the FDA in the area of kidney transplantation. It is a field where Angion has few potential rivals, and even fewer in the specific area of delayed graft function (DGF), a complication of organ transplantation.

Currently, there is no marketed drug therapy for the prevention or treatment of DGF. Though, most kidney transplant patients take immune suppressing medication such as cyclosporine, tacrolimus or sirolimus to prevent organ rejection.

Among the handful of companies working on new ways to address DGF in renal transplants is Pharming Group NV, of Leiden, the Netherlands, which earlier this year reported the published results from a preclinical study of its recombinant human C1 inhibitor (rhC1INH). The preclinical evidence, published in the American Journal of Pathology, showed that Pharming's rhC1INH may play a role in the prevention of delayed graft function after solid organ transplantation.

According to Pharming's figures, more than 79,000 patients in the U.S. are waiting for an organ transplant (35,000 in the EU). And each month, nearly 3,000 new patients are added to this waiting list. However, only 25,000 solid organs are available and transplanted each year, including kidney, liver, lung and heart transplants.

Quark Pharmaceuticals Inc., of Fremont, Calif., also recently reported that the European Commission has granted orphan status to QPI-1002 (also referred to as I5NP) for the prevention of DGF in kidney transplant patients. The designation was granted under the name of the European sponsor, Verius Ltd., of Cambridge, UK. QPI-1002 is a synthetic siRNA targeting p53 mRNA and is the first synthetic siRNA to be administered systemically to humans.

DGF affects 25 percent to 40 percent of the deceased donor renal transplants in the U.S and Europe, according to Quark, which has completed enrollment for the dose escalation safety portion of a two-part Phase I/II trial for the prevention of DGF in patients undergoing deceased donor kidney transplantation. The second part of that study is expected to begin this year.

Angion also has some preclinical products in its pipeline and expects to make further announcements in the coming months about its plans for BB3. It hopes to be able to take BB3 through proof of concept with its current funds.