Cambridge Healthtech Institute’s Tenth Annual

Optimizing Biologics Formulation Development

A Best Practices Exchange for Resolving the Challenges of Formulating Novel Biologic Drug Products

January 8-9, 2018

 

Cambridge Healthtech Institute’s Tenth Annual Optimizing Biologics Formulation Development is an essential international gathering of analytical and formulation scientists from leading industry companies, offering an exchange of scientific developments and emerging technologies in an environment that encourages discussion with colleagues. For 2018, the conference focuses on a set of best practices for resolving key formulation development challenges. Each talk in the program will be presented by a different industry or academic group to ensure the widest possible range of perspectives.

This important annual congress kicks off the weeklong PepTalk Formulation & Stability pipeline, which also includes separate meetings on Lyophilization and Drying Technologies and Protein Aggregation. By attending, you may also participate in the full set of eight PepTalk pipelines, including three-meeting sets on analytics and impurities, process technologies, protein engineering, antibody therapeutics and more.

Final Agenda

SUNDAY, JANUARY 7

4:00 - 6:00 pm Pre-Conference Registration

MONDAY, JANUARY 8

7:00 am Registration and Morning Coffee

A Product-Centric View of Formulation Development

9:00 Welcome by Conference Organizer

Kent Simmons, Senior Conference Director, Cambridge Healthtech Institute

9:05 Chairperson’s Opening Remarks

Tarik Khan, Ph.D., Group Leader, Late-Stage Pharmaceutical and Processing Development, F. Hoffmann-La Roche Ltd., Switzerland

KEYNOTE PRESENTATION

9:10 An End to End Approach to Formulation Development: Considerations from Cell Line to Drug Product Process Development

Kapil_GuptaKapil Gupta, Ph.D., Associate Director, Protein Pharmaceutical Development, Biogen

Development of high concentration stable formulations requires an end to end approach. Product heterogeneity and process impurities generated from upstream process can impact drug product stability and might not be solved by formulation optimization. An integrated development approach is needed by balancing bioreactor productivity, yield, quality attributes and drug product stability to ensure success. This talk will highlight this concept using some recent case studies.

9:50 Present and Future Trends in Biotherapeutic Device-Mediated Delivery Technologies

Didier_PertuyDidier Pertuy, Vice President, Global Drug Device Integrated Development & Device Strategy, Sanofi, France

Biotherapeutic device-mediated delivery is dominated by self-injectable devices like prefilled syringes, pens and auto-injectors. Next incremental step should be Large Volume Devices driven by dosing frequency reduction and low device-ability profile of new formats. In parallel, emerging mHealth-enabling technology is bringing new opportunities for smarter devices and integrated care solutions. Even though longer-term evolution stays difficult to predict some potential trends could be considered.

10:20 Networking Coffee Break

Co-Formulation and Co-Administration

10:45 The Challenges and Considerations of Protein and Peptide Coformulations

Rebecca_Davis-HarrisonRebecca Davis-Harrison, Ph.D., Research Scientist, BioTechnology Discovery Research, Eli Lilly and Company

The coformulation of two therapeutic peptides and/or proteins into a single dose is an emerging area of biological therapeutics and brings additional complexity to the already complex field of protein formulations. Preserving the physiochemical integrity of each molecule often requires that formulation conditions needed for one be applied simultaneously to the other. Analytical methods must also be able to detect changes in either molecule in this more complex environment.

11:15 Challenges in mAb Combination Drug Products: A CMC Overview

Jiali_DuJiali Du, Ph.D., Scientist, Formulation Sciences, MedImmune

The use of two mAbs in combination to improve treatment outcomes, and increase patient convenience and compliance is receiving increased scientific interest. This talk will provide a CMC outlook on challenges when formulating mAb combinations. Case studies of challenges during in-use stability, development of formulation and presentations, analytical characterization, and fill/finish processes will be discussed.

11:45 Case Study: Clinical In-Use Stability Evaluation: Co-Administration of Two Antibody Therapeutics

Zhen_WuZhen Wu, Ph.D., Senior Scientist, AbbVie

Concomitant IV administration of drugs, if clinically feasible, is a convenient approach for the patients and may offer competitive advantage. In this presentation, co-administration of two antibody therapeutics will be presented as an example. Both drugs were diluted in an IV bag and an in-use study was performed to evaluate dose solution stability. The approach taken to adapt the analytical methods and overcome the analytical challenges will be discussed.

12:15 pm See Stability in Hi-Def with Hunky

Greg Manley, Ph.D., Senior Applications Specialist, Unchained Labs

Developing biologics requires identifying ideal constructs and assessing a wide range of formulation space to ensure stability and minimize aggregation. Assessing ΔG is a powerful approach for the quantitative assessment of conformational stability and aggregation. Unchained Labs automates the tedious and manual task of determining ΔG, allowing for quantitative stability and aggregation assessment throughout biologic development. We'll discuss how automated chemical denaturation can be used with more traditional approaches to assess stability.

12:45 Enjoy Lunch on Your Own

Cell and Gene Therapies

2:00 Chairperson’s Remarks

Arun Alphonse Ignatius, Ph.D., Principal Scientist, Pfizer

2:05 Formulation Development for AAV-Based Gene Therapy Products

Arun_IgnatiusArun Alphonse Ignatius, Ph.D., Principal Scientist, Pfizer

Over the past few years, adeno-associated viruses (AAV) have evolved significantly with few products moving into late stage clinical development, and many others in early trials showing significant promise in the clinic. However, there are significant challenges that remain primarily due to the rapid transition into industry. This presentation will use case studies to showcase some of the challenges and considerations in the formulation development for AAVs.

2:35 Biophysical Characterization of mRNA Loaded Lipid Nanoparticles Formulations

Flaviu_GruiaFlaviu Gruia, Ph.D., Principal Scientist, Drug Product Analytical Development, Moderna Therapeutics

Developing a delivery vehicle capable of transporting the mRNA cargo to its intended target is a key challenge that should be addressed during development of mRNA-based products. Lipid nanoparticles represent a class of non-viral delivery systems that show potential in this space. The presentation will review some analytical development aspects, with specific examples of techniques that are valuable for biophysical characterization of nanoparticle formulations. Selected case studies will be included.

3:05 Transition to BuzZ Sessions

3:15 BuzZ Sessions with Refreshments

Join your peers and colleagues for interactive roundtable discussions.

Protein Formulation Challenges

4:30 Formulation Challenges for Low and High Concentration Proteins

Shiang_GweeShiang Gwee, Scientist, Drug Product Sciences, MacroGenics, Inc.

DART® molecules are bi-specific antibody-based proteins developed for immuno-oncology indications. These molecules are manufactured using conventional antibody platforms, and demonstrate comparable product quality and stability to conventional antibodies. Increased potency and lower dose requirement of certain bi-specific molecules present challenges for intravenous administration in early stage development. Case studies will be presented of approaches for IV administration of low/high concentration protein formulations that highlight these challenges.

5:00 Pulse Proteolysis: A Novel High-Throughput Tool for Formulation Screening

Lavanya_IyerLavanya Iyer, Ph.D., Research Investigator, Bristol-Myers Squibb

Biologics formulation selection is typically based on shelf-life stability data obtained over months. In this work, a novel analytical method called Pulse Proteolysis was used to rank-order formulations, based on resistance to proteolysis. The results demonstrate that formulations could be rank-ordered based on T-zero stability, as measured by pulse proteolysis. The high correlation with storage stability indicates that pulse proteolysis could prove to be a useful tool for formulation screening.

5:30 Characterization and Control of Interfacial Antibody Adsorption and Aggregation

Ian_ShiehIan Shieh, Ph.D., Scientist, Genentech

Exposure to interfaces can accelerate aggregation of antibody therapeutics during manufacture, transportation, and administration. Controlling interfacial antibody adsorption is critical to limiting aggregation. A panel of mAbs was characterized by multiple surface-sensitive techniques to predict their risk of interfacially mediated aggregation. We also measured and visualized antibody coadsorption with surfactant at the air-water interface to understand the protective mechanisms of the surfactants included in antibody formulations.

 Nanotemper Technologies 6: 00 - 7:15 Welcome Reception in the Exhibit Hall with Poster Viewing

7:15 Close of Day

TUESDAY, JANUARY 9

8:00 am Registration and Morning Coffee

Preformulation Studies

8:30 Chairperson’s Remarks

Rebecca Davis-Harrison, Ph.D., Research Scientist, BioTechnology Discovery Research, Eli Lilly and Company

8:35 Non-Ideal Colligative Properties in High Concentration Mab Solutions and Impact to Biopharmaceutical Manufacturing

WIlliam_CallahanWilliam Callahan, Senior Scientist, Amgen

Osmolality measurement is routinely used to monitor physical properties of protein formulations.  In this work, osmolality of high concentration protein solutions was found to either drift to higher than expected values or to be immeasurable due to a failure to freeze in the freezing point depression (FPD) osmometer. This presentation highlights the problem inherent in determining osmolality of high concentration protein solutions and that caution must be taken in interpreting these measurements.

Surfactants and Excipients

9:05 Challenges and Solutions in Polysorbate Degradation

Steven_LaBrenzSteven LaBrenz, Ph.D., Scientific Director, Cell and Developability Sciences, PDMS, Janssen R&D

Polysorbate degradation events in biopharmaceuticals are becoming more understood, moving beyond the original autooxidation work of Donbrow. Understanding degradation processes, relating that knowledge to actual conditions of use and controlling degradation events involving polysorbate are essential to developing a stable protein formulation. When controlling for external effectors that lead to issues in a protein formulation, degradation events involving polysorbate can be controlled.

9:35 Molecular Knowledge and Experience as the Driving Forces Behind Successful Protein Formulation Development

Katherine Bowers, Ph.D., Principal Scientist, Group Leader, Analytical and Formulation Development, FUJIFILM Diosynth Biotechnologies

9:50 Coffee Break in the Exhibit Hall with Poster Viewing

11:00 Poloxamer 188 as an Alternative Surfactant

Tarik_KhanTarik Khan, Ph.D., Group Leader, Late-Stage Pharmaceutical and Processing Development, F. Hoffmann-La Roche Ltd., Switzerland

Polysorbates have long been the workhorse surfactant for stabilizing biologics. However, due to their susceptibility to degradation there is a clear desire to identify alternative surfactants. Poloxamer 188 (P188) is one such stable surfactant that has been successfully utilized in the formulation of biologics. This talk will highlight real formulation/stability data as well as mechanistic characterization of how P188 stabilizes biologics by interfacial activity and solution behavior.

11:30 Prospects for the Identification and Application of Excipient Mixtures and Novel Excipients

Miko_SchleinitzMiko Schleinitz, PhD Student, Biochemical and Chemical Engineering, Technical University, Dortmund, Germany

Classically, novel excipients and excipient mixtures are identified based on heuristic approaches often neglecting synergetic effects and hindering transferability of the results to novel formulations. It is thus desired, to develop a physically-sound method to identify excipient mixtures and novel excipients based on modeling/predicting the intermolecular interactions in solution. This allows for considering the mutual influence of multi-excipient systems and to determine the optimal excipient mixture circumventing cost-intensive screening methods.

12:00 pm Formulation of Hard-to-Stabilize Biopharmaceuticals

Phil_MortonPhil Morton, Ph.D., Science Director, Bioprocess Characterisation, Albumedix

Modern biopharmaceuticals are changing. Increasingly, the industry is shifting towards more complex biological molecules intensifying formulation challenges requiring more advanced excipients. Human albumin is well known to stabilize proteins through a variety of roles, therefore recombinant human albumin is a promising stabilizer for hard-to-formulate biopharmaceuticals. We present data on the use of Albumedix® Recombumin® as a stabilizing agent across several different stress tests showing its versatility as an advanced excipient.

 


12:30 Close of Optimizing Biologics Formulation Development Conference