Amarna Therapeutics, a privately held biotechnology company based in Leiden, The Netherlands, has formalized a strategic agreement with NorthX Biologics to accelerate the development of Nimvec™ AM510, a next-generation gene therapy targeting Type 1 Diabetes. The collaboration aims to advance the therapy toward cGMP manufacturing and first-in-human clinical trials, with Amarna targeting clinical trial initiation in 2027.
Novel Approach to Type 1 Diabetes Treatment
Nimvec™ AM510 represents a potentially transformative approach to treating Type 1 Diabetes by addressing the root cause of the autoimmune disease. The therapy is designed to restore immune tolerance to insulin-producing beta cells by specifically targeting proinsulin, which serves as the primary antigen that triggers the autoimmune attack on beta cells.
"This agreement represents a major advancement in our partnership with NorthX Biologics and a critical step toward bringing Nimvec™ AM510 to patients," said Dr. Henk Streefkerk, CEO of Amarna Therapeutics. "With this investment, we are accelerating our path to clinical trials and moving closer to providing a potential cure for Type 1 Diabetes."
Manufacturing Partnership and Clinical Readiness
The agreement builds on a strategic partnership announced last year, which initiated the transfer of Amarna's research-scale production process for Nimvec™ AM510 to NorthX Biologics. Under the current terms, the collaboration enables the transfer and optimization of Amarna's research-scale production process to NorthX Biologics' advanced development facilities, including a comprehensive analytical development package to ensure the identity, potency, and quality of the vector throughout its development lifecycle.
NorthX Biologics, a leading biopharmaceutical contract development and manufacturing organization (CDMO), brings extensive expertise in producing cell and gene therapies, proteins, vaccines, and other advanced biologics. The company has been manufacturing biologics to GMP standards since 1992 and was designated an innovation hub for advanced therapies and vaccines in 2021.
"We are proud to deepen our collaboration with Amarna and support the progression of Nimvec™ AM510," said Dr. Janet Hoogstraate, CEO of NorthX Biologics. "Our combined expertise will help ensure the highest standards in process development and manufacturing as we advance this promising therapy."
Unique Platform Technology
The development of AM510 is based on Amarna's proprietary Nimvec™ platform, which has demonstrated exceptional promise in preclinical studies. Unlike other gene therapies that induce strong immune responses, limiting the possibility for repeat dosing and efficacy, Nimvec™ does not trigger such immune responses. Instead, it moderates the immune system to induce tolerance, making it an ideal vehicle for the therapeutic approach.
Preclinical data with Nimvec™ AM510 showcases protective effects in delaying the onset of hyperglycemia and preventing the development of Type 1 Diabetes in relevant animal models. This unique mechanism of action positions the therapy as a potential cure rather than a symptomatic treatment.
Addressing Significant Medical Need
Type 1 Diabetes affects millions of patients globally, with rising incidences each year. Despite advancements in therapy, life expectancy remains lower than the general population. The disease occurs when self-reactive T lymphocytes selectively attack and destroy insulin-producing β cells in the pancreas, leaving patients unable to maintain glucose homeostasis.
Currently, Type 1 Diabetes cannot be cured, and glucose homeostasis can only be maintained using daily insulin injections. Secondary complications of current therapy are considerable and can lead to significant morbidity and mortality. The Nimvec™ AM510 approach aims to restore immune tolerance to insulin and potentially cure patients by addressing the underlying autoimmune process.
Future Development Pipeline
The partnership establishes a framework to support the development and manufacturing of future Nimvec™-based therapies addressing other immune-mediated conditions, including autoimmune diseases and chronic inflammatory disorders. This milestone paves the way for subsequent cGMP manufacturing activities to support First-in-Human clinical studies, positioning Amarna to expand its therapeutic pipeline beyond Type 1 Diabetes.
