- Filing process for CTA submission will be initiated
- SYN002 is first clinical antiviral drug candidate for ex vivo treatment of CMV in kidneys
COPENHAGEN (March 12th, 2025) – Synklino, a Danish biotechnology company pioneering antiviral therapies, has completed a scientific advice meeting with the UK Medicines and Healthcare Products Regulatory Agency (MHRA) regarding the company’s antiviral drug candidate SYN002. Synklino presented preclinical data for SYN002 along with the proposed Phase 1 study design for a first-in-human (FIH) ex vivo clinical Phase 1 trial to be conducted in the UK.
MHRA agreed that the preclinical safety and efficacy data support the design of the FIH study. Based on the results of this meeting, Synklino will initiate the process of filing a Clinical Trial Application (CTA) with the MHRA.
“We are enthusiastic about receiving endorsement of our proposed path forward in this groundbreaking FIH study designed to remove latent CMV-infected cells prior to transplantation,” said Ian McGowan, MD PhD, Synklino Chief Medical Officer.
The SYN002 antiviral ex vivo therapy is the first drug candidate against CMV to be integrated into an ex vivo kidney reconditioning process using machine perfusion to actively improve organ function before transplant surgery.
The aim of the Phase 1 trial is to generate safety, pharmacokinetic, and preliminary efficacy data in transplant recipients receiving a kidney from a CMV-positive donor. A CMV-infected organ poses a high risk to all transplant recipients and represents a major unmet medical need. By treating the organ prior to transplantation, SYN002 offers the potential to eliminate the risk of CMV infection and reactivation in immunocompromised patients.
“Ex vivo perfusion offers a unique window of opportunity to administer tailored treatments to donated organs. This CMV antiviral study is an extremely relevant clinical innovation that parallels our team’s ongoing research and dedication to bringing more healthy organs to more transplant patients,” said Michael Nicholson, Professor of Transplant Surgery at the University of Cambridge and a Consultant Surgeon at Addenbrooke’s Hospital in the United Kingdom. Professor Nicholson is Director of the NIHR Blood and Transplant Research Unit (BTRU) in Organ Donation and Transplantation. He will serve as the Lead Coordinating Investigator in the SYN002 trial.
Synklino
Synklino is a Danish biotechnology company pioneering transformative therapies to eliminate chronic viral infections. We aim to build a pipeline by harnessing our proprietary target-identification platform to tailor novel antiviral drug candidates. Our first-in-class breakthrough treatment, SYN002, is a therapeutic fusion protein designed to revolutionize the standard of care prophylaxis for CMV by eliminating both active and latent CMV-infected cells in donated organs through ex vivo organ perfusion, offering a preventative solution for immunocompromised transplant recipients.
Background
Cytomegalovirus (CMV) is the main agent involved in infectious complications following transplant surgery, and a major risk for morbidity and increased hospital readmissions. CMV is a chronic viral infection found in more than 60% of humans worldwide. A healthy individual’s immune system confines the virus to an inactive but persistent state; however, in transplant patients with supressed immune systems, CMV emerges from latency and significantly impacts morbidity and mortality. CMV increases transplant costs by up to 50% due to long-term hospitalizations and difficult courses of treatment. CMV-infected organs constitute a key risk for CMV infection and complications in all transplant recipients at the time of transplantation, regardless of the recipient’s CMV status. No currently marketed or pipeline drugs can cure CMV, and current drugs can be associated with the risk of developing resistance to CMV-therapies.
SYN002 is a therapeutic fusion protein, uniquely targeting both latent and lytic CMV-infected cells. SYN002 is expected to be highly efficacious and potent compared to standard of care antiviral therapeutics which only target active (lytic) infection. Given the compound’s unique mechanism of action, SYN002 has the potential to eliminate the risk of CMV infection in immunocompromised transplant recipients who would otherwise receive a CMV-infected organ. In preclinical studies SYN002 has been shown to be fast-acting with full efficacy anticipated within the few hours that the organ is treated ex vivo (outside the body).
Ex vivo organ perfusion is the treatment of an organ after it has been removed from the donor, and prior to transplantation. Ex vivo organ care has evolved from storage on ice into dynamic reconditioning using machine perfusion. Continuous flushing of donor organs with fluids during machine perfusion allows enhanced storage at low temperatures with assessment of critical organ function parameters. Organ perfusion enables improvements of organ function by supplementing blood products or solutions that contain important nutrients, cells, and therapeutics, such as SYN002. Ex vivo machine perfusion increases the number of available transplanted organs and subsequent acceptance for transplantation, ultimately improving the chance for a positive life-saving outcome.
For additional information, please contact:
Synklino
Thomas Kledal, CEO
+45 2012 1656
tnk@synklino.com