FDA Provides Positive Feedback to Amarna Therapeutics on AM510 Gene Therapy for Type 1 Diabetes

On 27th August 2024, Amarna Therapeutics, a private biotechnology firm specializing in transformative gene therapies, announced the successful conclusion of an FDA INTERACT meeting on July 31, 2024. This meeting was pivotal for the development of AM510, a novel gene therapy aimed at treating Type 1 Diabetes Mellitus (T1D). The Initial Targeted Engagement for Regulatory Advice (INTERACT) meeting, requested on April 26, 2024, brought together Amarna's teams from Leiden and Seville, regulatory consultants from Veristat, and key members of the company's T1D Scientific Advisory Board. The session provided an opportunity for an in-depth discussion of the FDA's preliminary feedback in a productive virtual format.

During this meeting, Amarna received comprehensive feedback on various aspects of their development program. This feedback included critical insights on the chemistry, manufacturing, and controls (CMC) program, nonclinical program, and safety considerations for the clinical trial design. The FDA's input will play a crucial role in refining Amarna's approach to ensure the safety and efficacy of AM510 for T1D patients who currently depend on insulin therapy. The company anticipates implementing the guidance received and continuing its collaboration with the FDA as it progresses towards an Investigational New Drug (IND) application.

Henk Streefkerk, the CEO & Medical Director of Amarna Therapeutics, remarked, "Our AM510 tolerance induction program for T1D achieved a significant milestone with the FDA INTERACT meeting. The invaluable feedback we received validates our integrated development strategy and provides a clear regulatory pathway. We are eager to advance our preclinical work and aim for a successful pre-IND meeting, with the goal of starting to treat and ultimately cure T1D patients as soon as possible."

The INTERACT meeting featured open and constructive dialogue, establishing a solid foundation for Amarna's ongoing development efforts and future regulatory submissions. The company aims to engage with the FDA again in a pre-IND meeting within the next two years. Amarna values the FDA's input, noting that the INTERACT format has proven to be an effective catalyst for advancing early-stage drug development.

Type 1 Diabetes Mellitus is a severe disease affecting millions worldwide, with increasing incidence rates each year. Despite therapeutic advancements, life expectancy for T1D patients remains lower than that of the general population. The condition is characterized by the autoimmune destruction of insulin-producing β cells in the pancreas by self-reactive T lymphocytes, leading to an inability to maintain glucose homeostasis. Currently, there is no cure for T1D, and patients must rely on daily insulin injections to manage glucose levels. Additionally, the current therapy can result in significant secondary complications, causing notable morbidity and mortality. Amarna aims to address this with Nimvec™ AM510, which seeks to restore immune tolerance to insulin and potentially cure T1D patients.

The development of AM510 leverages Amarna's proprietary Nimvec™ platform, which has shown tremendous promise in preclinical studies. Unlike other gene therapies that provoke a strong immune response, limiting repeat dosing and efficacy, Nimvec™ does not elicit such responses. Instead, it modulates the immune system to induce tolerance, making it well-suited for Amarna's therapeutic approach. Preclinical data for Nimvec™ AM510 demonstrate its protective effects in delaying the onset of hyperglycemia and preventing the development of T1D in relevant animal models.

Amarna Therapeutics is leading the charge in developing groundbreaking gene therapies for both rare and common autoimmune and genetic diseases. The company's Nimvec™ platform is designed to deliver transformative treatments, showing exceptional promise in preclinical studies. Amarna remains dedicated to pioneering therapeutic solutions for Type 1 Diabetes Mellitus and improving patient outcomes.