Programme 2019

September 9-10, 2019; Boston, MA

7:40 AM - 8:40 AM

Registration & Refreshments

8:40 AM - 8:50 AM

8:50 AM - 9:25 AM - Keynote

Small Molecules

Understanding Developability Assessment of Small Molecules

Sudhakar Garad, Global Head of Chemical and Pharmaceutical Profiling, Novartis Institutes for BioMedical Research

  • Why solubility, stability and delivery is imp during lead optimization and its impact of selection of dev candidates
  • Impact of strong interface/collaboration with chemist and pharmacokinetics during NCE selection
  • Speed to clinic with right biopharm properties

9:30 AM - 10:05 AM - Case Studies

Small Molecules

Current Trends in Technology and Regulatory Landscapes for Developing and Bringing Generics and Innovative Medicines to Market

Rosario LoBrutto, Head of Scientific Affairs, Sandoz

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Biologics

Ultrasmall Targeted Nanoparticles with Engineered Antibody Fragments for Imaging of HER2-overexpressing Breast Cancer

Marcello Marelli, Senior Scientist Antibody Discovery and Protein Engineering, AstraZeneca

  • Construction and efficient assembly of silica-based ultrasmall nanoparticle with anti-Her2 targeting antibodies
  • Targeted nanoparticles effectively localize to Her2 expressing tumors in vivo
  • Nanoparticles show low liver accumulation and high tumor to background ratios in vivo
  • Targeted nanoparticles have the potential for use in staging, risk stratification, treatment planning and development of next generation therapeutics.

Device Development

Biopharm Combination product development: Container Closure selections/criteria and Injection force modelling

Aarti Gidh, Technology Development Team Lead, GSK

The Combination product development presentation will include 2 case studies:
  • Cross-functional selection criteria and assessment tools applied to propose new selections and justify changes of container closure platforms.
  • Viscosity, injection force and delivery time modeling study for different needle gauge and autoinjector spring combinations.

10:10 AM - 10:45 AM - Solution Spotlights

Small Molecules

Unlocking Full Formulation Performance through Advanced Excipients

Dr. Kathryn Hewlett, Application Development & Innovation Scientist , DuPont Nutrition & Biosciences

The talk will cover several emerging formulation trends and how excipient suppliers are responding including,
  • Low solubility drug formulations
  • Continuous manufacturing processes
  • Taste masking for improved patient compliance

Biologics

Hard-to-Stabilize Proteins and Peptides: Can Recombinant Human Albumin be an Effective Approach?

Eleonora Cerasoli, Ph.D., Science Manager, Technology Group, Albumedix

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Device Development

A QbD Approach Towards Manufacturing of Parenteral Packaging Components with First Line USA

Eugene Polini, Technical Key Account Manager, Datwyler Pharma Packaging, Inc.

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10:45 AM - 11:35 AM

iSolve & Networking Break

11:35 AM - 12:10 PM - Case Studies

Small Molecules

Solubility Strategies at the Interface of Drug Discovery and Development

Dr. Philippe Lienard, CMC Pre-Development Science Leader, Sanofi

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Biologics

Machine Learning Models for Predicting P-gp and BCRP Mediated Efflux

Istvan Enyedy, Principal Scientist, Biogen

The Design of therapeutic agents that penetrate the blood-brain barrier is challenging mainly due to the promiscuity of efflux transporters. The structures of P-gp and BCRP have been published allowing us to build machine learning models that combine the transporter structural information with traditional ligand-based descriptors. The performance of these models will be presented.

Device Development

Incorporating Behaviour Design into Digital Health Tools

Paul Upham, Head, Smart Devices, Genentech

When the intended uses of a digital health solution include things like: remote monitoring of the subject; helping to improve medical adherence or persistence; increasing engagement; and clinical decision support, it is likely that the design of that solution would benefit from incorporating principles rooted in behavior neuroscience – especially related to how to encourage behavior change and the adoption of new or modified behaviors. Historically, the med-tech and pharmaceuticals industry has developed and launch digital tools with many of these intended uses, but were unsuccessful in achieving meaningful, sustained results. The consumer technology world, however, has demonstrated that the careful application of behavior design to the user experiences in digital products can significantly change human behavior in ways that achieve meaningful, sustained value. This presentation will provide examples of failures and success and introduce key aspects of behavior design that can be incorporated into the device design and development process.

12:15 PM - 12:50 PM - Solution Spotlights

Small Molecules

Continuous Melt Granulation of Thermally Labile Drug

Tony Listro, Vice President of Technical and Quality , Foster Delivery Science

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Technology & Innovation

Bridging the Drug Substance to Drug Product Continuum for Poorly Soluble Molecules

David Lyon, Senior Fellow, Research, Lonza Pharma & Biotech

  • Bioavailability enhancement technology selection
  • Speed to First-in-human studies
  • Integrated drug substance and drug product services offering
  • Bioavailability enhancement case studies

Device Development

The Sorrel Wearable Drug Delivery Platform for Your Primary Container

Dr. Andrei Yosef, CEO, Sorrel Medical

Sorrel Medical’s CEO, Andrei Yosef PhD, presents a unique drug delivery platform that integrates primary containers of choice, expediting time to clinical trials and commercial launch with a pre-filled and pre-loaded wearable drug delivery platform. Sorrel’s vial-based devices can open up new pathways for pharmaceutical and biotech companies to utilize wearable devices as a commercially viable, clinic ready option.

12:50 PM - 1:50 PM

Networking Lunch

Lunch & Learn Round Table with W.L. Gore

Silicone-Free Option for Delivery of Sensitive Biologics in Bare Glass Pre-Filled Syringes

To ensure functionality of pre-filled syringe systems, the pharmaceutical industry has been utilizing silicone to ensure system functionality and performance. However, silicone poses many challenges not only to the drug product, but also to the manufacturing process. This talk will focus on some of the latest insights to highlight the challenges that silicone can introduce to the newer, more complex and sensitive biologics like aggregation, precipitation or immunogenicity issues.  In this forum, we will discuss the GORE ImproJect Syringe Plunger, a tested and validated commercial product, and its ability to enable bare glass (non-siliconized) syringes for a silicone-free delivery option.

Moderated by: 

Russ Hornung, Business Development- Drug Delivery and Packaging, W.L. Gore & Associates

1:50 PM - 2:25 PM - Case Studies

Small Molecules

Building a Chemical Space Map for in- silico Prediction of Supersaturation by Mesoporous Silica

Suman Luthra, Senior Principal Scientist, Pfizer

  • Mesoporous silica has received increasing attention for stabilizing API in amorphous phase and yielding enhancement of apparent solubility relative to crystalline form.
  • The objective of our research is to elucidate molecular descriptors of drug like compounds which are most important for in- silico prediction of supersaturation by mesoporous silica.
  • Preliminary data on 12 test compounds demonstrates good correlation of total hydrogen bond formers and Tm/Tg with high probability of a solubility enhancing formulation with mesoporous silica.
  • This technology has shown promise to be used in quick evaluation of amorphous formulations in preclinical PK studies

Biologics

Quest for Quality Drug – Use of First Principle Based Biophysical Assays to select Manufacturable Molecule

Sathya Venkataramani, Associate Director, Janssen Biotherapeutics

According to Trends in Clinical Success, only ~10% of drugs that enter Phase I are destined to get FDA approval. One of the potential factors that has commonly impacted the success rate is poorly behaved molecules. To save cost and to expedite development that will enable higher success rate, it is undoubtedly critical to characterize the intrinsic properties of molecules early on. This talk is designed to highlight few high throughput assays and first principle based biophysical tools to select molecules that are risk free from manufacturing challenges.

Device Development

Harnessing the Power of the Digital Exhaust to Improve Patient Outcomes

Graham Jones, Director of Innovation, Novartis

Clinical decision-making using diagnostic, prognostic, and monitoring biomarkers requires coordination between the healthcare and technology industries and careful design of longitudinal efficacy studies. Community health providers and pharmacies are poised to play a pivotal point‑of‑care role by helping capture prodromal diagnostic data and incorporating biomarker monitoring into wellness programs.  A model for the pharmaceutical in­dustry to embrace these patient engagement opportunities is outlined together with recent advances in connected care and digital enabled treatment solutions.

2:30 PM - 3:05 PM - Solution Spotlights

Small Molecules

How do I quickly develop first-in-human (FIH) dosage forms and avoid CMC delays while achieving proof-of-concept (POC)?

Kieran Crowley, Executive Director and Head of Drug Development Solutions North America, Quotient Sciences

  • Choosing the appropriate dosage form for first-in-human/Phase I trials – fit for purpose, fit for phase?
  • Compounding or GMP manufacturing – how to balance cost, time and dose flexibility?
  • Challenges with poorly soluble molecules in early development – what technologies can we deploy?
  • Bridging to robust and scalable Phase II drug products – how to avoid losing time in development?
  • Integrating pharmaceutical sciences and clinical testing to de-risk and accelerate drug product development

Biologics

Freeze Drying Proteins Formulation: A Journey from Challenge to Opportunity

Clara Ip, International Sales Executive, Biopharma Group

The presentation will describe a case study about the process development of a lyophilised protein product. The different phases from formulation to cycle development and technology transfer will be covered outlining challenges and commercial benefits.

Device Development

High Volume, High Viscosity, Cartridges or even Multi-Container and Vial Applications? The Swiss Wearable Injector Platform to make sense of it all – From Clinical Trials to Lifecycle Management

Tom Mayer, Business Development Manager, Sonceboz SA

  • Device platforms should be patient centric but also offer maximum flexibility to Pharma companies with respect to use-case, drug volume and container.
  • This presentation highlights a highly modular and flexible platform capable of serving multiple use cases without trade-offs.

3:05 PM - 3:55 PM

iSolve & Networking Break

3:55 PM - 4:30 PM - Case Studies

Small Molecules

Flexibility in Pharmaceutical Manufacturing

Yash Kapoor, Principal Scientist, Merck

Although flexibility in manufacturing is not a novel concept, its definition is amorphous, and the financial benefits are ambiguous. What specific types of flexibility are most valuable in pharmaceutical manufacturing: facility, process technology or regulatory flexibility; product, operation or capacity flexibility? How much additional resource should a firm be willing to spend upfront to gain flexibility down the road? And how do emerging technologies compare to traditional technologies across the multiple dimensions of flexibility? In this talk, the answers to these questions will be pursued, and the argument will be made that a premium must be paid upfront to obtain flexible manufacturing down the road, in order to best respond to our volatile, uncertain, complex and ambiguous (VUCA) world.

Biologics

Nanoemulsion-Based Therapies: Antimicrobial, Anti-inflammatory and Drug Delivery Properties

Susan M. Ciotti, Director of Formulation Development, Ann Arbor MI, Adjunct Professor, Pharmaceutical Sciences, University of Michigan, Ann Arbor MI, BlueWillow Biologics

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Device Development

Intracutaneous Microneedle Patch Delivery System for the Treatment of Acute Migraine

Yi Ao, Associate Director, Zosano Pharma

  • Overview of Zosano’s intracutaneous microneedle patch delivery technology
  • Evaluating the stability and delivery of zolmitriptan intracutaneous microneedle patch system
  • Clinical assessment on efficacy and safety of zolmitriptan intracutaneous microneedle patch system for the treatment of acute migraine

4:30 PM - 5:05 PM - Keynote

Biologics

RNA Nanotechnology for Drug Delivery and Formulation

Peixuan Guo, Sylvan G. Frank Endowed Chair in Pharmaceutics and Drug Delivery Systems, The Ohio State University

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5:05 PM - 5:50 PM - Panel Discussion

Preparing for Patient-Centric Drug Development Regulations

Diane Harper, Director Regulatory Affairs-CMC, Merck

Galen Shi, Senior Engineering Advisor, Eli Lilly

Graham Jones, Director of Innovation, Novartis

Paul Upham, Head, Smart Devices, Genentech

  • What do the recent FDA Guidelines on Patient-Focused Drug Development (PFDD) mean to us?
  • Why do we need more patient focussed thinking and design of drug products, medical devices and combination products?
  • How will become more patient focussed shake up the industry and make us more competitive

5:50 PM - 6:00 PM

6:00 PM - 6:10 PM

Poster Presentation Award

DDF Poster 2019 Winner Announcement and Award Presentation

6:10 PM - 7:10 PM

Evening Drinks Reception

8:00 AM - 8:45 AM

Registration & Refreshments

8:45 AM - 8:50 AM

8:50 AM - 9:25 AM - Keynote

Biologics

Formulation Development Gap Assessment for An In-licensed Program: A Case Study

Nazila Miller, Director, Drug Product Development , Takeda

Complex due diligence activities often overshadow potential scientific challenges associated with an in-licensed program.  To successfully transition a product in-house, a cross-functional team of subject matter experts, in addition to the due diligence team members, should be engaged in a timely manner to identify development gaps, and assess the time and resources required for a successful product transition and an ultimate launch.  This presentation highlights a formulation-specific case study and the challenges that are scientifically being remedied.

9:30 AM - 10:05 AM - Case Studies

Small Molecules

Formulation Challenges and Strategies for Supporting Toxicology and First in Human Studies

Chris Ho, Senior Director, Head of Early Formulation Development, PDS, U.S., Sanofi Genzyme

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Biologics

Evaluating the Impact of Polysorbate 20 Hydrolysis During the Life-time of Protein Formulation: A De-risking Case Study

Keethkumar Jain, Senior Scientist Drug Product Development , Takeda

Polysorbates are commonly used in biotherapeutic formulations to protect against interfacial stresses encountered during handling, manufacturing, and transportation processes. They are known to degrade by hydrolysis and contribute to potential protein instability. This presentation discusses a formulation-specific understanding of polysorbate 20 hydrolysis, evaluates the effect on protein stability in pharmaceutically relevant storage and shipping conditions, and presents a de-risking strategy to support inclusion of polysorbate 20 in the current formulation.

Device Development

Applying ICH Q12 to Combination Products

Diane Harper, Director Regulatory Affairs-CMC, Merck

With the anticipation of ICH Q12 guideline on Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management proceeding to Step 4 in late 2019, combination product sponsors have an opportunity to level set expectations on CMC life cycle management as well as to improve the CMC submission strategy and dossier. Perspectives on how ICH Q12 can be implemented for drug-device combination products will be discussed with a focus on the device constituent. Additionally, current thinking on the use of Established Conditions to create a performance-based device constituent control strategy will be shared.

10:10 AM - 10:45 AM - Solution Spotlights

Small Molecules

Furosemide- How to make a leading drug better and its use wider

Pieter Muntendam, President and CEO, SQ Innovation AG

  • Introducing subcutaneous infusion of a novel formulation of Furosemide
  • A new chapter for one of the most important cardiovascular drugs of our time
  • Reducing the volume of administration for improved cost-efficiency
 
Novel formulations are required for subcutaneous delivery.  Furosemide is poorly soluble at neutral pH. Furosemide Injection USP, the product which has been used for over 50 years for intravenous administration, has a pH of around 9.0.  This is too alkaline for subcutaneous delivery and may case a stinging/burning sensation and skin irritation.  A novel higher concentration formulation with a neutral pH is under development by SQ Innovation  The higher concentration allows for use of standard 3 mL cartridges and the smaller patch pumps that were originally developed for insulin delivery.

Technology & Innovation

Oral Thin Films – Tailoring Therapy Needs

Dr. Marco Emgenbroich, Corporate Vice President Head of Pharmaceutical Development I R&D , LTS Lohmann

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Device Development

A New Micro-Pump for Parkinson’s Disease from Sensile Medical

Dr. Gerhard Mayer, Vice President, Business Development , Sensile Medical AG

  • A new pump on the market for continuous PD Therapy
  • Variable, programmable dosing, while extremely accurate
  • Platform approach with a variety of Pump Configurations

10:45 AM - 11:35 AM

iSolve & Networking Break

11:35 AM - 12:10 PM - Case Studies

Small Molecules

Oral Controlled-Release Liquid for a Low Potency Drug

Alfred Liang, Senior Director, Pharmaceutical Sciences, Ferring

  • Overview of current oral controlled-release liquid technologies
  • Challenges of developing a palatable controlled-release liquid for high dose, low potency drugs
  • Examples of viable delivery options

Biologics

mRNA delivery: Lipid Nanoparticles and Beyond

Puneet Tyagi, Senior Scientist Dosage Form Design & Development, AstraZeneca

mRNA is an exciting new class of drug that has the potential to play a role in gene therapy. With several advantages over DNA, including the lack of any requirement for nuclear localization or transcription and the nearly negligible possibility of genomic integration of the delivered sequence, mRNA holds tremendous potential to cure diseases. However, the labile nature of mRNA is an important limitation to its in vivo application. Thus, its paramount to develop mRNA delivery strategies for its success as a therapeutic. In this presentation, drug delivery platforms will be discussed, with focus on commercial application and viability.

Device Development

Modelling Autoinjector Injection Time Distribution

Jean-René Authelin, Global Head of Pharmaceutical Engineering, Sanofi

Autoinjectors are more and more often required for the subcute administration of biologic drugs. However developing an autoinjector with the right injection time is often very challenging, especially for highly concentrated and viscous protein solutions. In this talk we will how understanding the physics may help in predicting injection times distribution and designing the right device, by taking into account variability in  drug and components.

12:15 PM - 12:50 PM - Solution Spotlights

Small Molecules

Unleashing the Potential of Poorly Water Soluble Drugs with the Next Generation Nano Technology

Andrea Cusack, Managing Director & Chief Business Officer, Leon-nanodrugs GmbH

  • SMART nanoparticle™ technology
  • Oral & parenteral case studies
  • Easy scale up & low complexity
  • Bringing improved products to market

Technology & Innovation

Transmucosal Delivery – Using the Mucofilm® Technology to Bypass the First Pass Effect

Dr. Ahmad Ghoniem, Pharmacist - Business Development Manager , tesa Labtec North America

The oral mucosa seems to be the ideal location for delivering large APIs systemically and still avoiding the first pass effect. Certain challenges arise when developing such a dosage form. The product has to adhere on a wet and high mobile surface, needs to resist enzymatic degradation and increased saliva flow and has to simple disappear after a few minutes or a few hours. Challenges that can be overcome to develop the next generation of large molecule application systems.

Device Development

enFuseTM, a true Wearable Technology Changing the way Drugs are Delivered

Michael Hooven, President and CEO, Enable Injections, Inc.

The current treatment for many diseases requires patients to spend hours each week receiving IV infusions at a healthcare facility. But what would happen if the common IV formulation paradigm could shift to subcutaneous formulations? Could patients get their time and their lives back? Would adherence improve, and therefore improve patient outcomes? The enFuseTM platform, an on-body drug delivery system, is a unique true wearable technology being developed for patient self-administration of high-volume drugs from 5 - 50mL. The system is designed with novel features to provide comfort, convenience, and simplicity for the patient. Some of these unique features will be discussed in terms of their impact on patients, drug development, and the overall healthcare landscape.

12:50 PM - 1:50 PM

Networking Lunch

1:50 PM - 2:25 PM - Case Studies

Small Molecules

Inner Ear Drug Delivery: a case study on the Inner Ear Delivery of Neurotrophin-3

Qi-Ying Hu, Director of Formulation and Drug Delivery, Decibel Therapeutics

  • Overview of the inner ear drug delivery
  • A case study on the inner ear delivery of NT3, including (1) The electrophysiological response after dosing NT3 in a noise induced hearing loss mouse model; (2) The NT3 disposition in the perilymph; (3) The NT3 distribution in the cochlear tissue determined by radio-imaging methods; (4) The pharmacodynamic response in cochlea upon the inner ear dosing of NT3.
  • Future perspectives

Biologics

Challenges and Impact of Formulation on Exposure to Drive PKPD in CNS Drug Discovery

Patrick Trapa, Principal Investigator, DMPK, Biogen

  • Early discovery compound characteristics often make high exposure for PKPD necessary and difficult to achieve
  • Driving CNS exposure is even more challenging due to the blood brain barrier
  • Dosing strategies to address this challenge are complicated and yield mixed results

Device Development

Compatibility between Drug Formulation and Syringe / Autoinjector Performance

Galen Shi, Senior Engineering Advisor, Eli Lilly

  • Formulation matrix can impact silicone oil lubricity of syringe and PFS/AI functionality upon stability 
  • Accelerated stability data for glide force can predict refrigerated shelf life performance
  • Important to select right excipients to avoid negative impact on device performance stability
  • Syringe friction change is caused by dynamic interactions of formulation excipients with silicone oil, water and glass

2:30 PM - 3:05 PM - Solution Spotlights

Small Molecules

Functional Polyamino acids: a Versatile & Biocompatible Drug Delivery Toolbox

Vicent J. Nebot, Chief Technical Officer, Polypeptide Therapeutic Solutions S.L.

Disruptive delivery technologies are paving their way to the treatment of severe diseases with multitude of nanomedicines performing as great propositions at the proof of concept level, clinical developments or marketed products. Polyamino acid (PAA)-based therapies are emerging as promising treatments for a number of neurodegenerative, inflammatory, infectious or oncologic pathologies. Major features of PAAs includes superior performance in the synthesis of polymer-based therapeutics in terms of cargo loading capacity, multifunctional and architecturally versatile polymer backbone, biodegradability and the adaptability of these nanomedicines to bypass biological barriers with a non-toxic and safe biodistribution profile. Despite most of the novel delivery technologies are disruptive enough to make the clinical translation a major challenge from the regulatory and fabrication standpoint, a deep insight into the chemistry of PAAs-based nanomedicines enables a robust and reliable approach to guarantee the appropriate manufacturability into GMP environment.

Biologics

What's in your formulation?

John Proctor, Vice President Marketing , Halo Labs

  • Impact of refractive index on sub-visible particle measurement techniques
  • Measuring 96 samples in 96 minutes
  • Analyzing insoluble protein aggregates in most formulations with only 25uL
  • Fluidics-free sub-visible particle counting with the HORIZON® system

Device Development

Solution Spotlight (Session TBC)

3:05 PM - 3:35 PM

Networking Break

3:35 PM - 4:10 PM - Case Studies

Small Molecules

Quantifying the Value of Flexibility from Continuous Manufacturing of Drug Products

Robert Meyer, Head of Innovation Technology Group, Pharmaceutical Commercialization, Merck

The benefits derived from Continuous Manufacturing of drug products span the product development life cycle, from rapid experimentation and facile scale up, to site tech transfer, flexible batch sizes and automated digital manufacturing. Despite the broad benefits, efforts to quantify the value of continuous manufacturing have been difficult, as much of the value is realized when flexibility is used in reaction to a changing landscape - a situation not often included in business case analyses. We attempt to quantify the value of flexibility obtained through continuous manufacturing by a novel method of analysis, backed by data obtained from Merck’s first product manufactured by this route.

Biologics

Application of Dynamic Mechanical Analyzer (DMA) to Determine the Glass Transition Temperature of Frozen Protein Formulations

Younghoon Kim, Scientist- Biologics Drug Product Development and Manufacturing Group, Sanofi

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Small Molecules

Lyospheres: Enabling Flexibility for Lyophilized Products

Brian K. Meyer, Principal Scientist New Technologies, Vaccine Drug Product Development, Bioprocess Research and Development, Merck

Currently, pharmaceutical products that are labile are lyophilized (freeze dried) to maintain their stability and potency.  Traditional lyophilized products require the liquid product to be filled into the final container prior to freeze drying, limiting the drug product to a specific formulation, target potency, and volume.  Lyospheres, which are small, freeze-dried beads that contain the active material and excipients, are dispensed into vials and potentially allow for a  more innovative and flexible approach for achieving final drug product images.  Lyospheres are first generated from a bulk drug substance batch using “straight through processing” which is independent of the bulk potency.  Following the drying process, the lyospheres are tested for potency and based on their result, titrated into the final drug product vial, minimizing overages.  In the case of vaccines, individual lyospheres may be generated from a variety of vaccine bulk drug substances and combined into a single image to form a multi-vaccine product.  This could allow new combinations of vaccine products and enable the ability to “mix and match” products.  In addition, by changing the amount of lyospheres per vial, the supply chain could have flexibility to have both single- and multi-dose images.  Another benefit of lyospheres is with regards to potentially improving thermal stability profiles for products.  By generating individual lyospheres for different components, an “optimized” formulation could be developed for each component without compromising stability of other components.  In conclusion, lyosphere technology represents a potential flexible way of making lyophilized drug products in the future.

4:15 PM - 4:50 PM - Keynote

Small Molecules

CMC Strategies to Accelerate Proof-of-Concept for Oral Drugs in the Biotech Industry

Jatin Patel, Executive Director-CMC, Constellation Pharmaceuticals

In recent years innovation in the pharmaceutical industry has been fuelled by small and mid-size biotech companies which have the benefit of cutting-edge science, nimble organizational processes and increased capital to fund research with a clear focus.  More recently, regulatory processes like breakthrough designation have provided additional impetus to bringing new medicines forward rapidly.   Despite these catalysts, some biotech’s face significant hurdles as they transition from discovery to development.  This is especially true for small biotech’s that use a virtual model where most if not all the development work is outsourced and there is significant reliance on external partners.  In this approach, one of the most common gaps is in the areas of pharmaceutical development that includes preformulation, formulation, drug delivery as well as CMC regulatory strategies. This could lead to conservative or erroneous strategies for early development studies and a negative impact on expediting transition to registrational studies. This presentation will outline several key areas of CMC focus for the discovery to development transition to establish POC and rapid transition to registrational studies.

 

Case studies highlighting smart risk taking and adoption of phase-appropriate development that enables rapid human assessment, speed to POC and transition to registrational studies will be covered.

4:50 PM - 5:00 PM