Makana™ Issued Patent in South Korea for Its Genetically-Modified TKO Pig for Use in Xenotransplantation

• Company in discussions with U.S. Food and Drug Administration regarding first-ever human clinical trial as a solution to help solve the organ donor shortage crisis

MIAMI--(BUSINESS WIRE)-- Makana™ Therapeutics, a global leader in the field of xenotransplantation, was granted an important patent in South Korea that will catalyze xenotransplantation efforts in that country, the company announced today. Makana was granted a similar patent in Europe in 2023.

Makana is working to solve the organ shortage crisis by making genetically-modified pigs for use as organ donors for human recipients. The South Korean patent was granted on Makana’s Triple Knockout pig, or “TKO,” which is a combination of three xenoantigen gene knockouts in the pig that effectively camouflage the cross-species grafts from the human recipient’s immune system.

Makana has demonstrated compelling results in xenotransplantation to date. “Our knockout pigs combined with our advancements in immunosuppression and patient matching have resulted in the longest and most consistent preclinical survival data in the xenotransplantation field,” said Mark Platt, the company’s President and Chief Executive Officer. “We are very pleased that the Triple Knockout Pig, which was discovered in the lab of our founder, Dr. Joe Tector, is being adopted as the ‘gold standard’ of baseline genetics for xenotransplantation.”

On March 21, 2024, Mass General Hospital announced that it had transplanted a genetically modified pig kidney into a patient suffering from End Stage Renal Disease. The procedure was performed under the expanded access pathway or “compassionate use” which allows a single patient to receive an investigational medical product when facing an immediate life-threatening condition. The pig kidney used in the case contained the baseline TKO genetics along with other modifications. The earlier reported “compassionate use” cases of pig heart xenotransplants at the University of Maryland School of Medicine also incorporated the TKO genetics discovered by Makana. While compassionate use authorizations may help a small number of patients, Makana remains focused on pursuing a clinical trial to help the entire population that suffers from organ failure.

Platt added, “The Triple Knockout Pig has fundamentally changed the field and has moved xenotransplantation closer to becoming a clinically available reality. The organs from our TKO Pig have been shown to match more than 30% of patients waiting for a kidney transplant, and likely more than 70% of patients can benefit from these organs with available pre-transplant treatment. Our team is working tirelessly to reach our goal of allowing every patient suffering from organ failure to return to an entirely normal life.”

According to the National Kidney Foundation, there were 786,000 patients in the U.S. living with kidney failure in 2021. In 2022, there were approximately 25,000 kidney transplants performed in the U.S. However there are still an estimated twelve people dying each day in the U.S. while waiting to receive a life-saving kidney transplant. Currently there are more than 93,000 people on the U.S. kidney transplant waitlist. This has been an enduring challenge for organ transplantation, with the waiting list substantially larger than the supply of donor organs.

Makana is in discussions with the U.S. Food and Drug Administration regarding the final steps prior to conducting the first-ever human clinical trial in kidney xenotransplantation. The trial is expected to be conducted at the University of Miami. Dr. Tector, MD, PhD, FACS, a practicing transplant surgeon, heads up the xenotransplant program at the Miami Transplant Institute (MTI), a collaboration between Jackson Memorial Hospital and the University of Miami Health System.

“The TKO pig is widely seen as the preferred genetic profile that will enter clinical trials,” Platt said. “We’ve been diligently driving our work to its clinical stage for more than a decade. We will profoundly improve both the length and quality of life for our patients, beginning with Patient One.”

About Makana Therapeutics

Founded in 2009, Makana Therapeutics is focused on developing swine with reduced xenoantigen expression, making human transplantation of cells, tissues and organs from these animals possible. Makana’s focus on simplified genetics, optimized pig cloning techniques and careful patient selection is expected to streamline product development and result in safer more efficacious products. For more information on Makana, please visit www.makanatherapeutics.com.

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