BOSTON, MA, Nov. 05, 2020 (GLOBE NEWSWIRE) -- XenoTherapeutics, a clinical-stage cell and gene therapy company engineering immune-compatible cells, tissues, and organs, has completed the first of two patient cohorts in the company’s Phase I clinical trial of Xeno-Skin™, a first-in-human, live-cell xenotransplant for the treatment of severe and extensive burn wounds. As a result, the U.S. Food and Drug Administration (FDA) has agreed to permit accelerated patient enrollment; patients may now enroll in the trial’s second cohort simultaneously. This first-in-human trial, conducted in partnership with the Massachusetts General Hospital, is expected to be completed by the end of 2020. More information is available at clinicaltrials.gov.
"As an engineer and scientist, I’m trained to be skeptical and rely heavily on real data, and to prioritize facts above what we hope to see as a company. In this case, the evidence speaks for itself – there's no doubt this works. Combine these results with our understanding of essential regulatory requirements specifically related to xenotransplantation, and our demonstrated capability in clinical grade manufacturing of xenotransplantation products, this seemingly small step has significant positive implications for patient care, for our field, and where we’re headed,” said XenoTherapeutics Corporation CEO Paul Holzer. “This affirms my enthusiasm for where our discoveries will go from here – very big things are coming soon.”
Surgeries to implant Xeno-Skin™ were performed under the care of Dr. Jeremy Goverman (Principal Investigator) and Dr. John Schulz at the Massachusetts General Hospital (MGH) Sumner Redstone Burn Center. All patients who participated in the trial received surgical transplants of both live skin from a clinical-grade, genetically engineered porcine donor and skin from a human deceased donor, today’s current clinical standard of care, in a side-by-side comparison.
No adverse events or safety issues have been observed or reported to date, following careful evaluation of all patient data by the trial’s independent Safety Review Committee. Visible signs of efficacy were also promising; in all patients, Xeno-Skin™ and the human skin comparator were indistinguishable at the time of autografting.
“The field of burn care requires a multidisciplinary approach to treat the most complex injuries, and we are constantly seeking new options to improve patient recovery time,” said Jeremy Goverman, MD. “This trial demonstrates a novel way to treat complicated burns, and we are encouraged by the results thus far; additionally, this trial represents a step forward for the field of xenotransplantation.”
Especially notable, post-operative RT-PCR evaluation of all patient samples showed no evidence of detection of the porcine endogenous retrovirus. Historically, this concern has posed a significant hurdle to the widespread clinical use of porcine cells, tissues, or organs in human recipients. The data from this trial align with decades of evidence that zoonotic disease poses a low risk of infection to human patients, a view further supported by numerous other experts in the field.
“There have been many debates as to whether zoonotic disease transmission is indeed still an issue. In 2020, there is still no in-vivo evidence – even long-term – of transmission or infectivity. However, regardless of opinion whether zoonoses is of any continued significance, testing will remain a requirement by regulatory agencies,” said Professor Linda Scobie of Glasgow Caledonian University, who also serves as an Associate Editor of the Journal of Xenotransplantation. Professor Scobie is widely regarded as a leading expert on this topic and presented on safety regulations in xenotransplantation, including XenoTherapeutics’ Phase I trial, at The Transplantation Society 2020 Virtual Congress.
The US-FDA cleared XenoTherapeutics to conduct this first-in-human, investigational clinical trial to evaluate the safety and tolerability of a novel, live-cell xenotransplant sourced from a clinical-grade, genetically optimized porcine donor. This “is a great step forward for the xenotransplantation community and hopefully paves the way for smoother regulatory processes for future xenotransplantation products,” said Professor Scobie.
Clearance of this pivotal clinical trial was based on a comprehensive body of evidence gathered from a series of XenoTherapeutics’ preclinical studies performed under independently audited, GLP-compliant conditions (Journal of Xenotransplantation: Immune Response; Journal of Burn Care and Research: Clinical Efficacy; Journal of Burn Care and Research: Cryopreservation), which confirmed findings from prior academic, peer-reviewed research. For this work, XenoTherapeutics received the American Burn Association’s 2019 Burke/Yannas Bioengineering Award, named in honor of Dr. John F. Burke and Dr. Ioannis V. Yannas, the pioneers of Integra, a widely used artificial skin in the clinic today. Recently, the U.S. Patent and Trademark Office (USPTO) awarded XenoTherapeutics Patent No. 10,799,614, granting the company an exclusive methods patent related to essential regulatory criteria necessary to procure clinical-grade cells, tissues, and organs for transplantation from non-human donor animals.
About XenoTherapeutics
XenoTherapeutics is a 501(c)(3) Massachusetts private scientific research and development foundation founded to further advance the science of xenotransplantation through education, research, development, and clinical testing towards an eventual practical therapeutic use for the public benefit. XenoTherapeutics Foundation collaborates with XenoTherapeutics Corporation, a clinical-stage cell and gene therapy company engineering immune-compatible cells, tissues, and organs. XenoTherapeutics has pioneered the clinical advancement of xenotransplantation by initiating human trials for the first-in-human, live-cell xenotransplantation surgery with Xeno-Skin™. XenoTherapeutics and their collaborators support and rigorously adhere to all applicable laws, regulations, and policies governing the ethical treatment of animals, including the Animal Welfare Act and Public Health Service Policy of Humane Care and Use of Laboratory Animals. More information is available at www.xenotherapeutics.org.
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