Amyloidosis Research Consortium (ARC), a 501(c)(3) nonprofit organization focused on accelerating the development, approval, and access to new and innovative treatments for amyloidosis. In 2019, ARC entered into a Public Private Partnership (PPP) with the US Food and Drug Administration (FDA) to identify and bridge the scientific gaps in drug discovery and development for the treatment of AL Amyloidosis. Amyloidosis is the term for a group of rare, progressive and fatal misfolded protein diseases. AL amyloidosis a rare and multi-systemic disease in which there is no approved FDA therapy. AL amyloidosis is a disease that disproportionately afflicts older adults and has a poor prognosis when diagnosed in an advanced stage. All the emerging therapies are aimed at treating the underlying plasma cell dyscrasia are most advantageous when administered early in the course of the disease. As part of the PPP, ARC and FDA are proposing to host the second in a series of cross- stakeholder scientific meetings (with involvement from patients, academia, industry, and regulatory agencies) entitled The Amyloidosis Forum. Each of these meetings is focused on identifying the knowledge gaps, barriers, and strategically addressing these to advance drug development in this challenging disease. The next public meeting will be held on 12th November, 2020, entitled: Amyloidosis Forum Meeting 2: Advancing Drug Development in AL Amyloidosis: Capitalizing Natural History and Assessing Endpoints: Forum Objective 1: Natural History and Endpoint Development: The morning sessions will provide an in depth look at the importance of natural history from academia, industry and regulatory perspectives. There will be presentations on current sources of natural history from academia, industry (placebo data) and other sources, as well as discussion about how to leverage existing datasets to identify meaningful endpoints, disease specific measurements, and ultimately accelerate research. Forum Objective 2: Endpoint Development: Given the complex multi-systemic nature of AL amyloidosis, endpoint choice is challenging. The second part of the forum will focus on endpoint development; traditional designs versus novel designs. such as hierarchical or multimodal endpoints, with case studies from industry and FDA. This meeting will seek to define innovative approaches toward endpoint development including considerations for a multi-domain responder index (MDRI).
AL amyloidosis is the most common type of systemic amyloidosis, with no FDA approved treatments. The Amyloidosis Forum was created through a Public Private Partnership between The Amyloidosis Research Consortium and FDA to bring all the stakeholders in the AL community together to discuss the challenges, address the obstacles, and find pathways towards accelerating drug development in AL amyloidosis. This meeting will focus on natural history and endpoint development.