Considerations for Novel Endpoint Development in AL Amyloidosis

Following the first meeting held in October 2020, specialized working groups in the areas of cardiac, hematological, renal, and other organ systems were formed. Additional working groups reviewed health-related quality of life measures and statistical approaches to analysis of clinical trial data. Each working group was comprised of leading physicians, patient representatives, statisticians, and representatives from the FDA, MHRA, and pharmaceutical companies. Each working group prioritized organ-specific endpoints for use in AL amyloidosis clinical trials. In January 2021, all members of the working groups reconvened and shared their findings and recommendations.

Hematology Working Group

• Angela Dispenzieri, MD, Consultant; Division of Hematology; Serene M. and Frances C. Durling Professor of Medicine and of Laboratory Medicine and Pathology; Hematology Research Chair – Mayo Clinic College of Medicine
• Shaji Kumar, MD, Mark and Judy Mullins Professor of Hematological Malignancies Consultant, Division of Hematology Professor of Medicine
• Ashutosh Wechalekar, MBBS, MD, FRCP, FRCPath, DM, Professor of Medicine and Haematology, Hon. Consultant, National Amyloidosis Centre, University College London (Royal Free Campus); Dept of Haematology, Royal Free Hospital; Dept of Haematology, University College Hospital, London
• Nicole Gormley, MD, Division Director for the Division of Hematologic Malignancies II, CDER, US Food and Drug Administration
• Beatriz Flores, MD, Expert Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK
• Brendan Weiss, MD, Senior Director Multiple Myeloma Strategy Leader, Janssen Research and Development
• Liang Xiu, PhD, Director, Biostatistics, Janssen Pharmaceuticals
• James Signorovitch, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
• Mackenzie Boedicker, Founder, President, and Chairman of the Board, Mackenzie’s Mission

Cardiology Working Group

• Mathew S. Maurer, MD, Medical Director of The HCM Center at New York-Presbyterian Hospital/Columbia University Medical Center
• Claudio Rapezzi, MD Professor of Cardiology, University of Ferrara, Italy
• Marianna Fontana, MD, PhD, Professor of Cardiology, Hon. Consultant Cardiologist Deputy Clinical and Academic Lead for the National Amyloidosis Centre, UCL Cardiac CMR Service, University College London (Royal Free Campus)
• Ronald Witteles, MD Professor of Medicine (Cardiovascular Medicine, Co-Director, Stanford Amyloid Center, Co-Director, Stanford Multidisciplinary Sarcoidosis Program Director, Internal Medicine Residency Training Program, Stanford University School of Medicine
• Giampaolo Merlini, MD, Director of the Center for Research and Treatment of Systemic Amyloidosis, and of the Biotechnology Research Laboratories, Scientific Institute Policlinico San Matteo, University of Pavia, Italy
• Preston Dunnmon, MD, MBA, FACP, FACC, Cardiologist, Medical Team Leader (acting) in the Division of Cardiology and Nephrology, CDER, US Food and Drug Administration
• Krishna Prasad, MD, Senior Medical Assessor Medicines and Healthcare Products Regulatory Agency (MHRA), UK
• Cristina Quarta, MD, PhD, Director, Clinical Development Sciences, Global Medical Lead for Cael-101 and AG10 programs, Metabolic Diseases Therapeutic Area, Alexion
• James Signorovitch, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
• Isabelle Lousada, CEO and President, Amyloidosis Research Consortium

Renal Working Group

• Nelson Leung, MD, Consultant, Department of Nephrology, Professor of Medicine, Mayo Clinic College of Medicine
• Andrea Harvasi, MD, Assistant Professor of Medicine Boston University Medical Center Department of Medicine/Renal Section/Amyloidosis Center
• Julian Gillmore, MBBS, MD, PhD, FRCP, FRCPath, Professor of Medicine & Honorary Consultant Nephrologist Head, Centre for Amyloidosis & Acute Phase Proteins Research Lead, National Amyloidosis Centre, UCL Division of Medicine
• Raymond Comenzo, MD, Director, Transfusion Services; Director, John C. Davis Myeloma and Amyloid Program; Professor, Tufts University School of Medicine
• Kimberly Smith, MD, MS, Clinical Team Leader, Division of Cardiology and Nephrology (DCN), CDER, US Food and Drug Administration
• Krishna Prasad, MD, Senior Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK
• Yolanda Barbachano, PhD, Senior Statistician, Medicines and Healthcare products Regulatory Agency (MHRA), UK
• Spencer Guthrie, MBA, MPH , CEO, Attralus, Inc
• James Signorovitch, PhD, Managing Principal, Analysis Group, Inc., Health Economics and Outcomes Research, Epidemiology and Biostatistics
• Dena Heath, Northern California Support Group and Amyloidosis Research Consortium

Nerve, GI and Liver Working Group

• Michelle Mauermann, Consultant, Department of Neurology, Professor of Neurology, Mayo Clinic Rochester
• John Clarke, MD, Director, Esophageal Program, Stanford University School of Medicine, Stanford University
• William Litchy, MD, Consultant in Neurology, Department of Neurology, Mayo Clinic Rochester
• Morie A Gertz, MD, M.A.C.P., Consultant, Division of Hematology, Roland Seidler Jr. Professor Department of Medicine, College of Medicine, Mayo Distinguished Clinician
• Laura Obici, MD, Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy
• Melanie Blank, MD, Division of Clinical Evaluation and Pharmacology/Toxicology in the Office of Tissues and Advanced Therapies, CBER, US Food and Drug Administration
• Michelle Campbell, PhD, Senior Clinical Analyst, Division of Neurology Products (DNP),CDER, US Food and Drug Administration
• Laura Jawidzik, MD, Clinical Team Lead, Division of Neurology 1, CDER, US Food and Drug Administration
• Matthew Kowalik, MD, Acting Team Leader, Division of Gastroenterology, CDER, US Food and Drug Administration
• Sarrit Kovacs, PhD, Clinical Reviewer, Division of Gastroenterology, CDER, US Food and Drug Administration
• Douglas Faller, MD, PhD, Executive Medical Director, Takeda
• James Signorovitch, PhD, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
• Yolanda Barbachano, Senior Statistician, Medicines and Healthcare Products Regulatory Agency (MHRA), UK
• Kathy Koontz, Patient Representative

HQRoL Working Group

• Vaishali Sanchorawala, MD, Professor of Medicine, Director, Amyloidosis Center, Boston University School of Medicine and Boston Medical Center
• Anita D’Souza, MD, Associate Professor of Medicine, Scientific Director, CIBMTR® Medical College of Wisconsin, Froedtert Clinical Cancer Center
• Elektra Papadopolous, MD, Acting Director Division of Clinical Outcome Assessment, Office of New Drugs, CDER, US Food and Drug Administration
• Michelle Campbell, PhD, Senior Clinical Analyst, Division of Neurology Products (DNP), CDER, US Food and Drug Administration
• Beatriz Flores, MD, Expert Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK
• Tiffany Quock, PhD, Senior Medical Director, Prothena Biosciences Inc
• Avery Rizio, PhD – Scientist QualityMetric Incorporated, LLC
• James Signorovitch, PhD, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
• Paula Schmitt, Executive Director, Amyloidosis Support Groups
• Michelle White, PhD, Vice President and Senior Scientist, QualityMetric

Statistical Working Group

• James Signorovitch, PhD, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
• Preston Dunnmon, MD, MBA, FACP, FACC – Cardiologist, Medical Team Leader (acting) in the Division of Cardiology and Nephrology, CDER, US Food and Drug Administration
• Mark Rothmann, PhD, Deputy Division Director, Division of Biometrics II, CDER, US Food and Drug Administration
• Jialu Zhang, PhD, Division of Biometrics II, CDER, US Food and Drug Administration
• David Brown, PhD, Expert Statistician, Medicines and Healthcare Products Regulatory Agency (MHRA), UK
• Yolanda Barbachano, Senior Statistician, Medicines and Healthcare Products Regulatory Agency (MHRA), UK
• Liang Xiu, PhD, Director, Biostatistics, Janssen Pharmaceuticals