Nonclinical Program Considerations for Peptide Safety Assessment (S02)

Symposium Chair(s)

• Tim Hummer, PhD, DABT

  Senior Director
  Cardiovascular, Renal, and Metabolism Safety
  AstraZeneca
  Gaithersburg, Maryland, United States

• Peter Korytko, PhD, MBA

  CEO
  Preclinical GPS
  Bainbridge Island, Washington, United States

Symposium Speaker(s)

• Mayur Mitra, PhD, DABT

  Distinguished Scientist
  Genentech, Inc.
  South San Francisco, California, United States

• Patricia Brundage, PhD

  Lead Interdisciplinary Scientist / Pharmacologist
  US FDA

• Wei Wang, PhD, DABT

  Director
  Nonclinical Safety Assessment
  Eli Lilly and Company, Inc.
  Indianapolis, Indiana, United States

• Thomas Kjaergaard Andreassen, PhD

  Toxicologist, Principal Scientist
  Toxicology and Development Projects
  Novo Nordisk
  Bagsvaerd, Hovedstaden, Denmark

• Ed Dere, PhD, DABT

  Senior Principal Scientist
  Safety Assessment
  Genentech, Inc.
  South San Francisco, California, United States

Educational Support Provided by: Preclinical GPS

Nonclinical development programs for therapeutic peptides often fall somewhere between that of small molecule and biologics programs, with aspects of both ICH M3(R2) and ICHS6 (R1) being applicable. Peptides can range from simple synthetic polypeptides with natural amino acids to more complex molecules with non-natural amino acids and/or conjugated moieties. The types of toxicology studies needed often differ depending on the type of peptide modification. Because of this complexity, current guidance documents often lack sufficient advice for peptide development. In 2020, FDA codified the definition of a protein as any alpha amino acid polymer with a specific, defined sequence greater than 40 amino acids. For these molecules, a Biologics License Application regulatory route is used. For polypeptides of < 40 amino acids, regardless of manufacturing method, a New Drug Application regulatory route is used. Although this provides clarity for US marketing applications, this definition is not necessarily followed by other global regulatory agencies. The EFPIA peptide safety working group has conducted an industry-wide survey to capture the standard approaches companies are using for the development of therapeutic peptides and recommendations on what guidance documents could be updated to provide additional advice. In this symposium, results of the survey will be presented in an effort to harmonize toxicology programs implemented across pharmaceutical companies and the expectations from regulators. Case studies will be presented to describe successful approaches that have been taken for various peptide programs, with an emphasis on adhering to 3Rs principles. Additionally, general considerations for peptide development based on current FDA policy will be presented.

Presentations:

• 9:00 AM - 9:25 AM CT

  Peptide Safety Assessment: Current Regulatory Expectations
  

  Symposium Speaker: Mayur Mitra, PhD, DABT – Genentech, Inc.
• 9:25 AM - 9:50 AM CT

  Nonclinical Considerations for Development of Peptides: A CDER Perspective
  

  Symposium Speaker: Patricia Brundage, PhD – US FDA
• 9:50 AM - 10:15 AM CT

  EFPIA Peptide Safety Working Group Survey Results
  

  Symposium Speaker: Wei Wang, PhD, DABT – Eli Lilly and Company, Inc.
• 10:15 AM - 10:45 AM CT

  Break
• 10:45 AM - 11:15 AM CT

  Case Studies of Peptide Safety Assessment
  

  Symposium Speaker: Thomas Kjaergaard Andreassen, PhD – Novo Nordisk
• 11:15 AM - 11:45 AM CT

  Regulatory and Nonclinical Safety Considerations for Intravitreal Peptide Development
  

  Symposium Speaker: Ed Dere, PhD, DABT – Genentech, Inc.
• 11:45 AM - 12:00 PM CT

  Panel Discussion