Makana News

• Discovery to pave way for better outcomes for xenotransplantation as company continues 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, FL September 4, 2024 — Makana™ Therapeutics, a global leader in the field of xenotransplantation, announced today that the company’s scientists have discovered that pigs contain a fourth gene that when inactivated will greatly reduce the risk of organ rejection in a human recipient.

Makana’s Triple Knockout Pig (“TKO”) is the current foundational genetics used across the field, and is deficient in three genes which drive hyperacute rejection of pig tissue transplanted into humans. In 2012, Makana scientists discovered the first two of the TKO’s genetic pig modifications and, in 2014, discovered the third. Now in 2024, Makana has found a fourth genetic knockout required to address the early rejection seen in clinical application of pig organs transplanted into human recipients.

In recently publicized pig-to-human xenotransplantations, the transplanted organs were rejected by their human recipients within a two-month period. Each of those transplants were approved under “compassionate use” protocols and were not part of a U.S. Food and Drug Administration (FDA) clinical trial. These consistent early losses led Makana to direct its discovery efforts to identify the mechanism responsible for the failures. Every human sample Makana analyzed contained antibodies to the antigen eliminated by Makana’s fourth genetic target.

Joe Tector, MD, PhD, FACS, Makana’s founder and a practicing transplant surgeon at the Miami Transplant Institute, said, “Our research continues to focus on better understanding the mechanisms of organ rejection in xenotransplantation. As a result, we have discovered an additional human immune system response responsible for early xenograft organ failure. We have already created healthy pigs that inactivate the fourth gene to evade this immune response. This is a major breakthrough that will pave the way for clinical success in xenotransplantation.”

Mark Platt, Makana’s Chief Executive Officer, said pigs incorporating this knockout were produced last year as part of its research collaboration with the University of Miami. He added that Makana is preparing to launch its Definitive Preclinical Study in 2025 with plans to conduct a First-in-Human Clinical Trial in kidney xenotransplantation in early 2026 (upon FDA approval).

Specifics of Makana’s research will be detailed in a scientific paper to be published in the coming weeks.

Platt said Makana’s first focus will be in the kidney space. 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,” Platt said. “Our sole focus is to restore an entirely normal life to every patient with organ failure.”

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 scientifically validated 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.

Contact
Mark Leonard
mark@reachthenextlevel.com
847-651-9682
View Business Wire Press Release

• 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, FL — May 1, 2024 — 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 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.

Contact
Mark Leonard
mark@reachthenextlevel.com
847-651-9682
View Business Wire Press Release

MIAMI, FL — June 26, 2023 — Makana™ Therapeutics, a global leader in the field of xenotransplantation, announced today that it has been awarded an Artificial Kidney Phase II Prize from KidneyX to continue development of its genetically engineered donor pigs for use in kidney transplantation. The Kidney Innovation Accelerator or KidneyX is a public-private partnership between the US Department of Health and Human Services (HHS) and the American Society of Nephrology (ASN) to accelerate innovation in the prevention, diagnosis, and treatment of kidney diseases.

Makana is working to solve the organ shortage crisis by making genetically modified pigs for use as organ donors for human recipients. Makana’s TKO pig, or “Triple Knockout” 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.

“Innovation is urgently needed,” said Matt Tector, PhD, Chief Scientific Officer at Makana. “Through this prize competition, KidneyX is seeking to advance a field that has seen little progress in more than 60 years. The current standard of care for renal failure is a kidney transplant, but the supply of organs only addresses a small fraction of the need. Xenotransplantation could potentially save many lives. We are honored to have been chosen as a KidneyX Phase II Prize Winner.”

Makana has demonstrated compelling results in xenotransplantation. “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 Dr. Tector.

The Triple Knockout Pig was discovered in the lab of Joe Tector, MD, PhD, FACS, who is also the founder of Makana, and continues to guide its efforts. Dr. Tector, a practicing transplant surgeon, heads up the xenotransplant program at Miami Transplant Institute (MTI), a collaboration between Jackson Memorial Hospital and the University of Miami Health System.

“The Triple Knockout Pig has fundamentally changed the xenotransplantation field,” said Mark Platt, the company’s Chief Executive Officer. “The organs from this animal have been shown to be an acceptable match to more than 30% of patients waiting for a kidney transplant, and likely 70% of patients can benefit from these organs with available pre-transplant treatment.”

There are 850 million people worldwide who live with kidney diseases, including 37 million Americans. In the United States alone, treatment costs total more than $100 billion a year. Each day, 13 people die while waiting for a kidney transplant, while those on dialysis face a 50 percent mortality rate during the first five years of treatment. Communities of color are disproportionately affected with increased incidence, fewer organs available for transplant, and poorer outcomes overall.

“The TKO pig is widely seen as the preferred genetic profile that will enter clinical trials,” Platt said. “Sucessfully executing our clinical trial will represent one of the greatest medical breakthroughs of our lifetime, and bring hope to millions of people who will otherwise never have the opportunity to receive the life-giving gift of an organ transplant.”

Makana is in talks with the U.S. Food and Drug Administration regarding next steps to conduct the first-ever human clinical trial in kidney transplant.

About KidneyX
The Kidney Innovation Accelerator (KidneyX) is a public-private partnership between the US Department of Health and Human Services (HHS) and the American Society of Nephrology (ASN) to accelerate innovation in the prevention, diagnosis, and treatment of kidney diseases. For more information, please visit www.kidneyx.org.

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.

Contact
Mark Leonard
mark@reachthenextlevel.com
847-651-9682
View Business Wire Press Release

• Company in discussion with FDA regarding first-ever human clinical trial as a solution to help solve the organ donor shortage crisis

MIAMI, FL — January 04, 2023 — Makana™ Therapeutics, a global leader in the field of xenotransplantation, was granted an important European patent that will catalyze xenotransplantation efforts abroad, the company announced today. Makana is working to solve the organ shortage crisis by making genetically modified pigs for use as organ donors for human recipients. The patent was granted on the TKO pig, or “Triple Knockout,” 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. “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.

The Triple Knockout Pig was discovered in the lab of Joe Tector, MD, PhD, FACS, who is also the founder of Makana. “The Triple Knockout Pig has fundamentally changed the xenotransplantation field,” Platt said. “The organs from this animal have been shown to be an acceptable match to 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.”

According to the United States Renal Data System (USRDS), 23,853 kidney transplants were performed in the United States in 2020. At the end of 2020, 75,747 patients were on the waitlist for a kidney transplant. The active waiting list remains substantially larger than the supply of donor kidneys, which presents a continuing challenge.

Makana is in talks with the U.S. Food and Drug Administration regarding next steps to conduct the first-ever human clinical trial in kidney transplant. The trial would be conducted at the University of Miami. Dr. Tector, a practicing transplant surgeon, heads up the xenotransplant program at 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. “Sucessfully executing our clinical trial will change the field of transplantation forever.”

This recent development will add to Makana’s portfolio of intellectual property and will help the company to serve patients in Europe who are in desperate need of a transplant. It is estimated that 75 million people in Europe suffer from chronic kidney disease, according to the European Kidney Health Alliance. Many of those individuals languish on transplant waiting lists.

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.

Contact
Mark Leonard
mark@reachthenextlevel.com
847-651-9682
View Business Wire Press Release

• Company also announces launch of its website: makanatherapeutics.com

MIAMI, FL — April 7, 2022 — Makana™ Therapeutics, a global leader in the field of xenotransplantation, announced today that it has relocated its corporate headquarters to Miami, Florida from Eagan, Minnesota. The move enables closer synergy with the Miami Transplant Institute (MTI), a collaboration between Jackson Memorial Hospital and the University of Miami Health System. The xenotransplant program at MTI is led by Joe Tector, MD, PhD, FACS, who is also the founder of Makana.

Xenotransplantation is the process of transplanting organs or tissues between members of different species. Makana is working to help solve the organ donor shortage through xenotransplantation.

“Makana is making great strides in the development of genetically-modified pigs suitable for xenotransplantation,” said Mark Platt, the company’s President and Chief Executive Officer. “In addition, our unique immunosuppression regimen combined with our proprietary crossmatch assay puts us in a strong position to move our work into the clinic.”

Platt said moving the company’s corporate headquarters to Miami streamlines communication and collaboration with researchers at the xenotransplant program at MTI. “Dr. Tector is an internationally recognized researcher and transplant surgeon whose expertise in xenotransplantation has helped advance the entire field tremendously,” Platt added. “Having Dr. Tector on the Makana team puts us in position to lead the xenotransplantation field into the clinic.”

Makana has demonstrated compelling results in xenotransplantation to date. The Triple Knockout (TKO) pig (which was developed by Makana) removes three key xenoantigens which contribute to antibody-mediated rejection of the transplanted organ. “The TKO pig combined with our advancements in immunosuppression and patient matching has resulted in the longest and most consistent preclinical survival data in the xenotransplantation field,” Platt said. “We are now focusing our efforts to translate that success in the clinic starting with kidney transplantation,” Platt said.

Platt also reported that the company recently launched its corporate website at www.makanatherapeutics.com. “We’re excited to start telling Makana’s story and keep everyone up-to-date on our rapidly advancing clinical developments.”

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.

Contact
Mark Leonard
mark@reachthenextlevel.com
847-651-9682
View Business Wire Press Release