Developing Alpha-1 Antitrypsin Therapeutics; Public Workshop - Agenda
Center for Biologics Evaluation and Research/FDA
National Heart Lung and Blood Institute/NIH
Alpha-1 Foundation
September 16, 2019 8:00 AM-5:00 PM
Natcher Auditorium, NIH Campus
| 8:00 - 8:30 AM | Breakfast | Sponsored by the Alpha-1 Foundation |
| 8:30 - 8:40 AM | Welcome and Introductory Remarks | Peter Marks, MD, PhD Director FDA/CBER NHLBI Representative Miriam O’Day CEO, Alpha-1 Foundation |
| 8:40 - 9:15 AM | Alpha-1 Antitrypsin Deficiency and Augmentation Therapy | Robert Sandhaus, MD, PhD Professor of Medicine National Jewish Health |
| 9:15 - 9:45 AM | The Alpha-1 Foundation Scientific Infrastructure | Charlie Strange, MD Professor of Pulmonary and Critical Care Medicine Medical University of South Carolina Miriam O’Day |
| 9:45 - 10:15 AM | Update and Summary of Alpha-1 Therapeutics | Ross Pierce, MD Medical Officer Division of Clinical Evaluation & Pharm/Tox Office of Tissues and Advanced Therapies FDA/CBER |
| 10:15 - 10:30 AM | Break | |
| 10:30 - 11:15 AM | New Approaches to Clinical Trials | John Scott, PhD Director, Division of Biostatistics Office of Biostatistics and Epidemiology FDA/CBER |
| 11:15 - 11:30 AM | The Patient Journey | Peggy Iverson Alpha-1 Patient |
| 11:30 - 12:00 PM | Novel Targets for Alpha-1 Antitrypsin | Adam Wanner, MD Professor of Medicine University of Miami School of Medicine |
| 12:00 - 1:00 PM | Lunch | Sponsored by the Alpha-1 Foundation |
| 1:00 - 2:15 PM | Novel therapeutic Approaches that Raise Levels of Alpha-1 Antitrypsin | Terence R. Flotte, MD Professor of Medical Education The University of Massachusetts Medical School Stefan Marciniak, PhD Professor of Respiratory Science Cambridge Institute for Medical Research Mark Brantly, MD Professor of Medicine, Molecular University of Florida College of Medicine Klaus Wagner, MD, PhD Chief Medical Officer Inhibrx Charlotte McKee, MD Vice President, CF Clinical Development Vertex Pharmaceuticals |
| 2:15 - 2:40 PM | Implications for Other Plasma Based Therapies | Senator Rick Santorum Patient Advocate Partner in Plasma Technologies |
| 2:40 - 3:00 PM | Break | |
| 3:00 - 3:30 PM | Therapeutic Approaches to Liver Disease in AATD | James Hamilton, MD, MBA VP, Clinical Development Arrowhead Pharmaceuticals |
| 3:30 - 3:50 PM | Clinical Endpoints and Biomarkers in COPD Trials | Jeanine D’Armiento, MD, PhD Professor of Medicine Columbia University |
| 3:50 - 4:10 PM | CT Densitometry | Ken Chapman, MD Director, Asthma & Airway Centre University Health Network |
| 4:10 - 4:45 PM | Panel Discussion: Current Needs in Alpha-1 Antitrypsin Drug Development | Moderated by Peter Marks |
| 4:45 - 5:00 PM | Summary and conclusions/next steps | Peter Marks |