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[In-Person] Nonclinical Safety Studies for IND and NDA Filing for Small Molecules: Nuts, Bolts and Best Practices

Speakers: Joanne Birkebak, Independent Consultant; Dinah Misner, Aligos; Toufan Parman, Sangamo Therapeutics; Ellen McGlinchey, Gilead Sciences; Joel Bercu, Gilead Sciences; Melisa Masuda-Herrera, Gilead Sciences; Alejandra Trejo-Martin, Gilead Sciences; Jessica Hawes Oliphant, FDA
Organizers: Doris Zane, PhD, DABT, Executive Director, Nonclinical Safety, Gilead Sciences
Date: 2024-05-21
Time: 8:45-17:00 Pacific Time
Registration fee: Regular attendees: $295; Academic/Students/Postdocs: $45; Out-of-Pocket: $45; Major Sponsorship: $3000; Vendor Show: $695, Happy Hour Sponsorship: $975; Lunch Sponsorship: $1250; On-site Registration: $500
Location: Crowne Plaza, Foster City, CA (San Francisco Bay Area)
Major Sponsor: (2)Emery Pharma; WuXi AppTec
Vendor show vendors registered to date: (12)Alturas Analytics, Inc.; Ardena; BioAgilytix; BioIVT; Cyprotex; Deciphex; Discovery Life Sciences; Frontage Laboratories, Inc.; Meadowhawk Biolabs; PharmaBlock; Pharmaron; Veloxity Labs
Registration deadline:2024-05-20  (it will close sooner if the seating cap is reached)

About the Topic

This PBSS workshop will cover the different types of toxicology studies conducted to support IND and NDA filings for small molecules, including an overview of the nonclinical modules, safety pharmacology, genetic toxicology, carcinogenicity, reproductive and developmental toxicology, environmental and occupational toxicology and regulatory toxicology. This workshop will cover best practices and example case studies from leaders in the field.

Time (PST) Topic Presenter
8:45 - 9:00 am PBSS Welcome and Introduction Shichang Miao, PhD, President, PBSS; Doris Zane, PhD, DABT, Executive Director, Nonclinical Safety, Gilead Sciences
9:00 - 10:00 am Introduction to Nonclinical Safety: Who We Are and What We Do Joanne Birkebak DVM, DABT, Independent Consultant
10:00 - 11:00 am Safety Pharmacology Studies for Discovery and Development of Small Molecules for INDs/NDAs Dinah Misner, PhD, DABT, DSP, VP, Aligos Therapeutics
11:00 -11:10 am Major Sponsor Presentation Emery Pharma
11:10 -11:30 am Break and Vendor Show -
11:30 am -12:30 pm Genetic Toxicology and Carcinogenicity: What Do We Need and When Do We Need It? Toufan Parman, PhD, DABT, Senior Director of Nonclinical Safety Evaluation, Sangamo Therapeutics
12:30 - 1:30 pm Lunch Sponsor (Pharmaron)
1:30 - 2:30 pm Developmental and Reproductive Toxicology (DART) Studies: Small Molecule Designs, Endpoints, and Case Studies Ellen McGlinchey, PhD, Senior Project Toxicologist (Sr. Research Scientist II), Nonclinical Safety and Pathobiology Gilead Sciences 
2:30 - 3:30 pm Environmental and Occupational Toxicology Joel Bercu, PhD, MPH, DABT, Executive Director; Melissa Masuda-Herrera, MS, DABT, Senior Associate Scientist; Alejandra Trejo-Martin, MS, Senior Associate Scientist, Environmental and Occupational Toxicology, Gilead Sciences
3:30 - 4:00 pm Break and Vendor Show -
4:00 - 5:00 pm Nonclinical Regulatory Perspectives on Small Molecule Drug Development Programs Jessica Hawes Oliphant, PhD, Deputy Director, Division of Systems Biology, FDA
5:00 - 5:30 pm Panel Discussion All Speakers
5:30 - 6:30 pm Happy Hour Sponsor (Veloxity Labs)


About the Speakers

Joanne Birkebak DVM, DABT, Independent Consultant

Joanne Birkebak is currently an independent consultant with over 20 years experience as a nonclinical safety scientist in the pharmaceutical industry.  She has worked in both small and large size pharmaceutical companies including Pharmacia, Pfizer, Schering Plough, Celgene, Ikaria and Gilead where she has supported the development of both small and large molecules for a variety of indications in inflammation, virology and oncology. Joanne earned her Doctorate of Veterinary Medicine from Washington State University and has been a Diplomat of the American Board of Toxicology since 2004.  Joanne is committed to supporting the toxicology community and has served as a councilor for the Northern California Chapter of the Society of Toxicology and in multiple roles for the IQ Consortium including Chair of DruSafe.

Introduction to Nonclinical Safety:  Who We Are and What We Do 

This presentation will introduce you to Nonclinical Safety and its role in the pharmaceutical development of small molecules.  An overview of Nonclinical Safety will be provided as well as a brief introduction to toxicology.  The types and timing of nonclinical studies for the different stages of drug development will be discussed including the typical studies that make up Module 4 of the IND and NDA packages. In addition, the key regulatory guidance documents pertaining to nonclinical safety and the organization of Module 2.6 will be provided.  An example of a generic repeat dose toxicity study design including some of the key concepts that nonclinical studies are intended to address will be presented. The presentation will conclude with a case example highlighting application of the key concepts and how the nonclinical data were used to proceed into the first in human study.

Dinah Misner, PhD, DABT, DSP, Vice President, Aligos Therapeutics

I received my PhD in Biomedical Sciences at UCSD and was a post-doc at the Salk Institute. I have been in the pharmaceutical industry for over 20 years and am currently a Vice President at Aligos Therapeutics. I serve as a toxicology project team representative on both discovery and development teams and am responsible for the design and conduct of in vitro and in vivo toxicology and safety pharmacology studies for treatment of liver diseases, encompassing small molecules as well as oligonucleotides. Lastly, I am a board-certified toxicologist and safety pharmacologist, and am currently the secretary of the Safety Pharmacology Society.

Safety Pharmacology Studies for Discovery and Development of Small Molecules for INDs/NDAs

In this talk, basic principles of safety pharmacology as a discipline and regulatory requirements for safety pharmacology studies to file INDs and NDAs for small molecules will be discussed. Screening strategies during the discovery phase and de-risking of potential issues related to safety pharmacology will be presented. Required studies prior to first-in-human (FIH) dosing will also be presented, along with post-FIH monitoring once the small molecule enters clinical development and ultimately NDA filing and approval. Case studies will be used throughout, and investigative approaches to issues that may arise during either discovery or development phases will also be discussed.

Toufan Parman, PhD, DABT, Senior Director of Nonclinical Safety Evaluation, Sangamo Therapeutics

Toufan Parman, Ph.D., D.A.B.T. is the Senior Director of Nonclinical Safety Evaluations at Sangamo Therapeutics where she has an oversight on development of the company’s therapeutic pipeline providing safety assessment strategies for various projects from early discovery through clinical trials.  Dr. Parman has over 20 years’ experience in preclinical drug development with a successful track record of regulatory submissions and interactions.  She has managed, designed, developed, and conducted efficacy, pharmacology, general and reproductive toxicology, and carcinogenicity studies as well as specialized mechanistic studies in various animal models for a wide variety of investigative therapeutics including but not limited to chemotherapeutic agents, vaccines, contraceptives, monoclonal antibodies, proteins, as well as gene, and cell-based therapies.  She has been the author or co-author of over 30 peer reviewed articles and book chapters.  Dr. Parman has received her PhD from University of Toronto, Canada in Pharmaceutical Sciences specializing on reproductive toxicology and has been certified by the American Board of Toxicology since 2007.

Genetic Toxicology and Carcinogenicity: What Do We Need and When Do We Need It?

Toufan Parman1, and Jon Mirsalis2

1 Sangamo Therapeutics, Brisbane, CA

2 SRI International, Redwood City, CA

The main objective of genetic toxicology and carcinogenicity assessments is to identify a genotoxic or tumorigenic potential of an investigative new drug from which relevant risk in humans may be assessed. This presentation will provide an overview of the regulatory requirements, types of studies and timing in the development process to consider for these assessments.  Relevant case studies will be provided and the use of Weight of Evidence in carcinogenicity risk assessment will be discussed.  The focus of the presentation will be primarily on regulatory requirements for small-molecule drugs. 

Ellen McGlinchey, PhD, Senior Project Toxicologist (Sr. Research Scientist II), Nonclinical Safety and Pathobiology Gilead Sciences, Inc.  

After her doctorate research on drug abuse, Dr. McGlinchey supported pharmaceutical drug development as a Study Director at Charles River Laboratories in Horsham, PA. She specialized in developmental and reproductive toxicology and juvenile toxicology studies for small molecule, biologic, mRNA, and vaccine programs. During those 3 years, she directed over 60 embryo-fetal development, fertility, pre-and post-natal developmental, and juvenile toxicology studies.  Dr. McGlinchey is a Toxicologist within the Nonclinical Safety and Pathobiology department at Gilead Sciences, Inc., based in Foster City, CA. Her primary responsibility is to evaluate the safety of Gilead’s pipeline in nonclinical studies from discovery through development. She additionally navigates cross-functional teams through the drug development process as a nonclinical subteam leader, as well as a key contributor to wider development teams. In the last 5 years at Gilead, Dr. McGlinchey advanced small and large molecule programs to first-in-human trials, as well as late-stage regulatory filings in multiple therapeutic areas including oncology, antiviral, and inflammatory and fibrotic diseases. Dr. McGlinchey is a member of the Society of Toxicology (SOT), Northern California Chapter of SOT, and the American College of Toxicology (ACT) and previously served as an ACT Program Committee member (2020-2022). She was a member of the Society for Birth Defects Research and Prevention (formerly the Teratology Society), the Middle Atlantic Reproductive and Teratology Association, and the Society for Neuroscience.

Developmental and Reproductive Toxicology (DART) Studies: Small Molecule Designs, Endpoints, and Case Studies

Historical chemical-related tragedies on fetal development emphasized the need to characterize and to reduce the risk of embryo or fetal harm during drug development. To both characterize and minimize the risk of embryo or fetal exposure during pregnancy, developmental and reproductive toxicology (DART) studies are conducted in two animal species before enrollment of women-of-child-bearing-potential (WOCBP) in clinical trials. 

Fertility and early embryonic development, embryo-fetal development, and pre- and post-natal development toxicology studies are the 3 DART studies that characterize potential drug toxicity during the entire reproductive life cycle. These studies evaluate the potential effects of a drug on male and female fertility, the ability to produce offspring, and the growth and maturation of future generations, respectively.

This talk will provide an overview of development and reproductive toxicology for small molecules. It will provide information on the appropriate regulatory guidelines, study designs, and scientific endpoints of the 3 DART studies, and theoretical case studies of toxicity including pharmaceutical label implications.

Joel Bercu, PhD MPH DABT: Introduction and Occupational Toxicology 15 min

Alejandra Trejo-Martin MS:  Toxicological Assessment of Impurities 15 min

Melisa Masuda-Herrera MS DABT:  Environmental Toxicology 15 min

Dr. Joel Bercu PhD, MPH, DABT is an Exec. Director in the Nonclinical Safety and Pathobiology group at Gilead Science and has over 20 years of public health / toxicology experience in pharmaceuticals. His mission while at these positions is to protect the safety of staff, patients, and the environment.  He leads the Environmental and Occupational Toxicology (EOT) group at Gilead.  The EOT group provides expert toxicological documentation for occupational health categorizations / occupational exposure limits, permitted / acceptable daily exposures for cleaning validation, environmental risk assessments, pharmaceutical impurity assessments (including mutagenic / carcinogenic impurities), QSAR assessments of impurities for ICH M7 compliance, deviations, leachables and extractables and excipients.  The EOT group is also responsible for monitoring and reviewing toxicology tests including ecotoxicology, mutagenicity testing for impurities, and worker safety testing.  Prior to joining Gilead Sciences, he has worked at Eli Lilly and Amgen.  He is a member of the Society of Toxicology and the Risk Assessment, Occupational and Public Health (where he served as President), and Medical Devices specialty sections.  He has had several external committees such as chairing the IQ/Drusafe Impurities Working Group and on the board of directors for the Extractables Leachables Safety Information Exchange (ELSIE).  He received his BS from Texas A&M University, MPH from University of Texas – Houston School of Public Health, PhD from Indiana University, and is a Diplomate of the American Board of Toxicology (DABT).  

Alejandra Trejo-Martin is a Senior Associate Scientist in the Environmental and Occupational Toxicology group at Gilead Sciences, where she authors QSAR assessments of impurities to support ICH M7 compliance, documentation for occupational health categorizations / occupational exposure limits, and permitted acceptable daily exposures for cleaning validation. She also monitors and reviews toxicology tests including ecotoxicology, mutagenicity testing for impurities, and worker safety testing. Her background includes extensive experience in medicinal chemistry, in Gilead she worked in HIV and HCV programs, and in prior companies in a variety of targets in the areas of inflammation, cardiovascular and virology diseases. She has a Master of Business Administration MBA from Golden Gate University and a BS in Chemical Pharmaceutical Biologist from the Universidad Nacional de Mexico.

Melisa Masuda-Herrera MS is a Research Scientist in the Environmental and Occupational Toxicology group within Nonclinical Safety & Pathobiology at Gilead. Her responsibilities include authoring health-based risk assessments to support product quality and occupational toxicology programs, monitoring and reviewing worker safety studies and environmental risk assessment studies, and providing toxicology support to the Environmental Health and Safety group for global hazard communication. She has 10+ years of experience in the pharmaceutical industry. Melisa received her Bachelor of Science degree at UC Berkeley in Molecular Toxicology and her Master of Science degree at UC Santa Cruz in Environmental Toxicology. She is a Diplomate of the American Board of Toxicology (DABT).

Occupational Toxicology, Product Quality, and Environmental

In addition to understanding the patient safety of drugs, there are other elements of toxicological testing specifically for small molecules that are important to INDs and NDAs.  While there is no FDA requirement for occupational testing of pharmaceuticals, this is a common practice amongst pharmaceutical companies to ensure the safety of those handling the drug substance, or its intermediates.  The industry as a whole has moved away from the traditional “6-pack” of toxicology studies to assays that reduce or eliminate animal testing.  As part of the quality guidelines (ICH Q3A-D and M7) there is an expectation to understand the toxicological impact of pharmaceutical impurities.  Tests are conducted to understand the potential for mutagenicity and qualification of impurities.  Finally, if predicted environmental concentrations exceed certain thresholds then certain environmental testing is required.  The environmental studies are then used to build an Environmental Assessment (USFDA) or Environmental Risk Assessment (EMA).  This session will discuss toxicology studies used for occupational toxicology, product quality and environmental purposes, and the sections where the data is submitted.  Also it will discuss how such data is used in manufacturing or in a risk assessment.

Jessica Hawes Oliphant, Ph.D. Deputy Director, Division of Systems Biology, National Center for Toxicological Research (NCTR), U.S. Food and Drug Administration (FDA)

Dr. Hawes Oliphant has been with FDA since 2010, where she’s served as Deputy Director for the Division of Systems Biology at FDA’s National Center for Toxicological Research (NCTR) for the past 4 years.  Dr. Hawes Oliphant has 10 years of experience conducting Pharmacology/Toxicology drug reviews and risk assessments for Investigative New Drugs (INDs) and market New Drug Applications (NDAs) for the Office of New Drugs (OND) at the FDA’s Center for Drug Evaluation and Research (CDER).  During her tenure at FDA, Dr. Hawes Oliphant has worked alongside diverse regulators and research scientists on regulatory projects for a myriad of therapeutics, health indications, FDA-regulated products, and safety concerns. 

Nonclinical Regulatory Perspectives on Small Molecule Drug Development Programs

Regulatory expectations for nonclinical studies to support clinical dosing at various stages of the IND lifecycle will be discussed.  Expectations for nonclinical drug development programs to support NDA filing for market approval will also be covered.  Discussion of ICH, FDA, and EMA guidances pertinent to multiple types of therapeutic indications will be incorporated.  Case examples will be provided.

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