Speed to First-in-Human Trials for Biopharmaceuticals: The Risks and Benefits of Short-Cuts During Discovery and Early Development

June 23, 2010
11:30 – 1:00 pm EST

Strategies for selection of lead biopharmaceuticals with enhanced manufacturability and development of production cell lines that can be used throughout development improve process economics. However, the pressure to initiate first-in-human trials drives companies to implement discovery, cell line development, and platform manufacturing approaches that enable faster entry into the clinic. These sub-optimal approaches force companies to introduce new cell lines or processes later in development and incur risks of comparability failure, timeline impact, or other delays in the program. The recent FDA Phase I guidance appears to reduce early development and manufacturing requirements, but the impact of following a sub-optimal path in early development may have a significant impact on later development, scalability, comparability, and process economics, which must be considered when deciding how to initiate a new development program.

Learning Objectives:

• Manufacturing considerations during final lead identification: is it worth the time? What are the pitfalls of neglecting this concern during discovery?
• Production cell line development is one of the most time-consuming critical path CMC activities: what is the risk of starting production with a sub-optimal cell line?
• Proprietary technologies or automated solutions for high throughput, fast, robust cell line development.
• Platform processes versus product-specific process development: when to rely on the platform and when to consider your products specific requirements
• New regulatory guidance for phase 1 trials in the US-does the new guidance really reduce sponsor responsibility?

Who Should Attend:
Program managers, financial decision makers, senior management, regulatory, process development scientists, analytical.

About the Instructor
 Susan Dana Jones, Ph.D., Vice President and Senior Consultant, BioProcess Technology Consultants
Susan Dana Jones, Ph.D., has more than 20 years of discovery, product development, and strategic planning experience in biotechnology. She co-founded two biotechnology companies and has managed discovery and product development programs in multiple organizations and for multiple disease areas. She currently manages several outsourced biopharmaceutical process development and manufacturing programs for client products, assists and advises clients and vendors regarding applicable cGMP regulatory guidelines, performs regulatory compliance audits, prepares CMC sections of client regulatory submissions to FDA, EMEA, and other competent authorities, and performs due diligence of manufacturing and analytical operations for investors or clients considering acquiring products in development. In addition, Dr. Jones serves as a guest lecturer at Northeastern University, as a member of the Board of Directors of Virginia-based start up company Gene Solutions, and as a member of the Editorial Advisory Board of BioProces- International. Prior to joining BioProcess Technology Consultants, Dr. Jones was Senior Vice President of Corporate Development at Serenex, Inc, where she worked with other senior managers to complete a $15M Series B financing. She was previously Vice President of Product Development at Waratah Pharmaceuticals, where her responsibilities included management of outsourced product development and manufacturing activities for a combination protein and peptide therapeutic. Before Waratah, Dr. Jones held development and management positions of increasing responsibility at Peptimed Inc., Virus Research Institute (now CelldexImmunotherapeutics), IntraImmune Therapies Inc., and Dyax Corp. Dr. Jones received her Bachelor's degree from Harvard University, her Ph.D. from the University of California, San Francisco, and performed post-doctoral research at the Dana-Farber Cancer Institute of Harvard Medical School.