Novel Endpoints and Analyses

Advancing Drug Development in AL Amyloidosis: Novel Endpoints and Analyses in Multisystemic Rare Disease Trials


  The virtual Amyloidosis Forum Meeting was held on October 15th, 2020. This page contains a full recording of the meeting, agenda and brief bios on moderators, speakers and panelists.


Watch the Meeting



Forum Two Agenda




Preston Dunnmon FDA

Preston Dunnmon, MD, MBA, FACP, FACC
Cardiologist, Medical Team Leader (acting) in the Division of Cardiology and Nephrology
US Food and Drug Administration
Dr. Dunnmon is the acting Medical Team Leader in the Division of Cardiology and Nephrology as part of the Office of New Drugs (OND), Office of Drug Evaluation I (ODEI), Division of Cardiovascular and Renal Products (DCRP) within the Center for Drug Evaluation and Research (CDER), to serve as the CDER Public Private Partnership (PPP) Liaison to ARC.


Spencer Guthrie

Spencer Guthrie, MBA, MPH
CEO, Attralus
Spencer Guthrie brings significant expertise with over 23 years in drug development and commercialization experience across large and small biotech. He has dedicated more than 12 years of his career to amyloid-related diseases, including AL and ATTR amyloidosis, Alzheimer’s and Parkinson’s disease, and has authored over 30 scientific publications in amyloidosis.



Dianne Finkelstein

Dianne Finkelstein, PhD
Professor, Department of Biostatistics, Harvard T.H. Chan School of Public Health
Dr. Finkelstein is the director of Biostatistics at the Massachusetts General Hospital Cancer Center. Dr. Finkelstein’s research is focused on the development of semi-parametric methodology for analyzing the effect of covariates on failure time distributions that arise in Cancer and Acquired Immune Deficiency Syndrome (AIDS) epidemiological studies and clinical trials. Dr. Finkelstein’s research has also focused on the development of methods for analyzing longitudinal and survival outcomes, and on animal carcinogenicity experiments. She has also developed along with David Schoenfeld a method of combining endpoints in clinical trials which is favored by the Food and Drug Administration.



Beatriz Flores, MD
Expert Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK



Emil Kakkis

Emil D. Kakkis, MD, PhD
CEO, President and Founder, Ultragenyx Pharmaceutical Inc.
Dr. Kakkis leads a team developing and commercializing multiple rare and ultra-rare disease treatments. Over the last 25 years, Dr. Kakkis is best known for his work developing novel treatments for rare diseases and for advocating on policy issues by founding and supporting the EveryLife Foundation for Rare Diseases. Dr. Kakkis went on to found Ultragenyx in 2010 to focus on developing more therapeutics for rare and ultra-rare diseases. Since its founding, Ultragenyx has worked on developing treatments for twenty different genetic diseases and has now received four approvals.


Mathew Maurer

Mathew S. Maurer, MD
Cardiologist and Medical Director, The HCM Center at NewYork-Presbyterian Hospital/Columbia University Medical Center
Dr. Maurer directs the Clinical Cardiovascular Research Laboratory for the Elderly (CCRLE) at the Allen Hospital of NewYork-Presbyterian Hospital. Dr. Maurer is a member of the Advanced Cardiac Care Center at NewYork-Presbyterian/Columbia. He has extensive expertise in amyloidosis and has served as a principle investigator in clinical trials for hereditary


Krishna Prasad, MD

Krishna Prasad, MD
Senior Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK
Dr. Prasad is a senior medical assessor at MHRA with management responsibility for Cardiovascular- diabetes, anti-infective agents, and oncology therapy areas. His additional roles include Cardiology consultancy at St. Thomas’ hospital, London. He has worked for MHRA, the UK regulatory agency since 2002. Dr Prasad’s areas of special interests include heart failure, sudden death, cardiomyopathies and arrhythmias. He is a member of the Cardiovascular working party since 2008, assuming responsibility for coordinating European guidance on heart failure, lipid modifying agents as well as pediatric guidance in these areas.



Jan Regnstroem, MD, PhD
Senior Scientific Officer, Scientific Advice Office, European Medicines Agency 



Mark RothmannMark Rothmann, Ph.D.
Deputy Division Director, Division of Biometrics II, Center for Drug Evaluation and Research, Food and Drug Administration
Dr. Rothmann earned his Ph. D. in Statistics at the University of Iowa in 1990. He then spent the next nine years as a professor at various universities before coming to the FDA in 1999. At the FDA, he has since been involved in the review on Oncology, Hematology, and Metabolism and Endocrinology products and has been a Deputy Division Director or Acting Director since 2016. He has done research in several areas involving the design and analysis of clinical trials.


David Schoenfeld, PhD

David Schoenfeld, PhD
Professor, Department of Biostatistics, Harvard T.H. Chan School of Public Health
Dr. Schoenfeld is a Biostatistician at Massachusetts General Hospital. He is a Professor Emeritus of Medicine at Harvard Medical School, and was formerly a Professor in the Department of Biostatistics at the Harvard School of Public Health. He has developed the first omnibus goodness of fit test for the proportional hazards regression model, a model that is used extensively in clinical trials which have survival or time to progression as an endpoint. He has also developed along with Dianne Finkelstein a method of combining endpoints in clinical trials which is favored by the Food and Drug Administration. He also has done research in Bayesian methods for evaluating historically controlled trials and small pediatric trials.


PK Tandon, PhD

PK Tandon, PhD
Vice President, Biostatistics and Clinical Trial Methodology,Boston Pharmaceuticals Cambridge, MA
Dr. Tandon provides strategic direction and leadership in the clinical development of products and in the design, analysis, and reporting of late stage clinical trials.


Ashutosh Wechalekar, MD

Ashutosh Wechalekar, MD
Senior lecturer/honorary consultant hematologis,t University College London and the Royal Free London NHS Foundation Trust
He trained in hematology in the UK and Canada. His academic and clinical interests are focused on amyloidosis and multiple myeloma. In particular, he is interested in characterization and treatment of systemic AL amyloidosis as well as multiple myeloma, including clinical trials of new and novel agents in these disorders. He has published widely in these areas.


Jialu Zhang

Jialu Zhang
 Jialu Zhang is a mathematical statistician in FDA with over 10 years of experience in reviewing clinical studies. She has worked in multiple divisions including cardiology/nephrology, psychiatry, and neurology. She is currently the statistical team leader supporting the Division of Cardiology and Nephrology in CDER.