February 26-28, 2019
Hilton Tokyo Bay HotelTokyo, JapanTHE ONLY EVENT IN ASIA BRINGING TOGETHER SCIENCE,TECHNOLOGIES AND PARTNERS TO ACCELERATEOLIGONUCLEOTIDE AND PEPTIDE MOLECULES TO MARKETTop Companies Share Strategies to Accelerate Your Therapeutics
The Discovery of Semaglutide

Jesper Lau, Ph.D.

Vice President, Protein & Peptide
Chemistry, Novo Nordisk A/S,
Denmark

New Strategies and Technologies
in Oligonucleotide Therapeutic
Development

Thazha P. Prakash, Ph.D.
Executive Research Fellow,
Ionis Pharmaceuticals Inc.

RNAi Therapeutics Delivered

Muthiah (Mano) Manoharan, Ph.D.
Senior Vice President of Drug Discovery,
Alnylam Pharmaceuticals

Optimizing the Properties of Antisense
Nucleic Acid Therapeutics

Chandra Vargeese, Ph.D.,

Senior Vice President, Head of Drug Discovery,
WAVE Life Sciences

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APPLY THE LATEST INNOVATIONS IN
OLIGONUCLEOTIDE AND PEPTIDE DEVELOPMENTSCIENCE

Accelerate Your Product To Market
Hear case studies, best practices and lessons
learned from global oligonucleotide and peptide
developers currently in preclinical and phase 1/2/3
clinical trials. Ensure product approval by hearing
regulatory guidance and roadmaps to successful
IND/IMPD submissions from industry leaders.

TECHNOLOGY

Evaluate New Technologies And Services
Improve your discovery, clinical, processdevelopment, analytical and manufacturing efforts
by meeting with 20+ global technology leaders in
the exhibit hall. The exhibit hall also features peersubmitted posters that contain new and unpublishedresearch from global scientists working across allphases of oligonucleotide and peptide development.

NETWORKING

Meet Your Next Partner At AsiaTIDES
Connect with 250+ oligonucleotide and peptide
leaders across Asia, Europe and North America
during networking lunches, poster sessions, dinners
and cocktail receptions.

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2

PRE-CONFERENCE WORKSHOPS • Tuesday, February 26, 2019 • 9:00am – 12:45pm
8:00 Registration and CoffeeWorkshop #1:
Accelerating Oligonucleotides to
IND and Beyond
Workshop Overview:
This workshop will address early drug development and CMC ofoligonucleotide therapeutics. A detailed discussion of moving
oligonucleotide therapeutics from discovery to clinical trials will
include a description of strategies for early clinical development,
including GMP synthesis, analytical controls and specifications;nonclinical, CMC, manufacturing and scale-up and the regulatory
framework for preparation of IND-IMPD dossiers. Participants will also
gain a basic understanding of the considerations and requirements fortaking an oligonucleotide therapeutic into first-in-human clinical trials.
Workshop Co-Moderators:
Marc M. Lemaitre, Ph.D., Principal, ML Consult, USA
Thomas Rupp, Owner & Principal, Thomas Rupp Consulting, Germany
Speakers:

CMC Oligos - Real World Case Studies
Marc M. Lemaitre, Ph.D., Principal, ML Consult, USA

Oligo Synthesis Scale-up and Process Related Issues andTroubleshooting
Thomas Rupp, Owner & Principal, Thomas Rupp Consulting, Germany
Additional Workshop Speakers to Be Confirmed

Workshop #2:
Accelerating Peptides to IND: Moving to the Clinic,
CMC and Beyond
Workshop Overview:

This practical, introductory workshop will address early drug development of peptide
therapeutics. A detailed discussion of moving peptide therapeutics from discovery
to clinical trials will include a description of strategies for early clinical development,
including GMP synthesis, analytical controls and specifications; formulation strategies;
pharmacokinetics and toxicology study designs and requirements; and the regulatory
framework for preparation of IND-IMPD dossiers. Participants will gain a basic
understanding of the considerations and requirements for taking a peptide therapeutic
into first-in-human clinical trials.

Who should attend?
Anyone interested in preclinical/clinical development of peptide therapeutics
including scientists in discovery research, manufacturing, project management, drug
development, business development and regulatory affairs.

Workshop Agenda:

9:00 Workshop Co-Moderators’ Opening Remarks
Bruce Morimoto, Ph.D., Vice President, Drug Development-Operations,
Alkahest, USA
Christopher Rhodes, Ph.D., President and CEO, Drug Delivery Experts,
USA
9:15 Challenges in Synthesis of the Peptide API
An overview of solid versus liquid phase peptide manufacturing approaches
will be presented. This will be followed by potential challenges including
aggregation, scale-up, stability, changes in manufacturing early versus latephase, and other related notes.

Robert Hagopian, Director Business Development, PolyPeptide Group,USA
10:00 Analytical Methods, Pre-formulation, and Formulation for

Early Phase Development

The discussion will focus on all aspects of drug product development. We will
start with analytical methods required for drug product, including in processcontrols, QC release methods, and stability, and how they differ from those fordrug substance. In addition, we will discuss preformulation and formulation
development of peptides to support non-clinical research studies, toxicity
studies, and clinical programs. A third topic area to cover will be the selection,
design, and development of delivery systems for peptides.

Christopher Rhodes, Ph.D., President and CEO, Drug Delivery Experts,
USA
10:45 Networking Refreshment Break
11:15 IND-enabling, Nonclinical Safety Studies for Peptides:

Pharmacokinetics, Bioanalysis and Toxicology

Peptides bridge small molecules and biologics, not only in their size, but alsoin specificity and selectivity. These unique properties of peptides require
specialized consideration when designing and executing the safety studies
to support first-in-human clinical trials. This talk will outline and discuss thepharmacokinetics and bioanalysis of peptides and the design of toxicology
studies for peptides.

Bruce Morimoto, Ph.D., Vice President, Drug Development-Operations,
Alkahest, USA
12:00 Similarities and Differences in the Product Development

between a Peptide Drug and a Peptide Biologic in the US

With the advancement of immunotherapies, the development of peptide
therapeutic cancer vaccines and preventive vaccines as biologics has entered
new therapeutic areas. The regulation and the process in FDA governing
the review and approval these products as biologics under PHS Act arequite different relative to other therapeutic peptides regulated as drugs. The
presentation will discuss the similarity and differences in the review process anddata requirements between a peptide drug and a peptide biologic.

Duu-Gong Wu, Ph.D., Senior Director, Regulatory Consulting, PPD(and Former Deputy Division Director of Division of New Drug Chemistry,
CDER, FDA), USA
12:45 Close of Workshop

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MAIN CONFERENCE PLENARY KEYNOTE SESSION • Tuesday, February 26, 20191:55 Chairperson’s Remarks

3:30 Networking Refreshment Break

2:00 RNAi Therapeutics Delivered: Improving

4:00 Recent Progress in Ribosomal in-vitro

Pharmacological Properties of siRNAsThis presentation will focus on key advances in theseareas: 1) LNP mediated delivery and the approval of thefirst RNAi therapeutic; 2) Advances in GAlNAc conjugatesand 3) Chemical modifications for improving thepharmacology of siRNAs.

Muthiah (Mano) Manoharan, Ph.D.,
Senior Vice President of Drug Discovery,
Alnylam Pharmaceuticals, USA
2:30 The Discovery of Semaglutide –A Journey from Ala Scan to Structural
Design of GLP-1 Analogues
Since the early clinical findings of using glucagon like
peptide-1 (GLP-1) to regulate blood glucose levels in thelate 80´s there has been an increasing interest to discoverand develop GLP-1 receptor agonists for treatment oftype 2 diabetes. The increased non-clinical and clinical understanding of themechanism of action of GLP-1 and the parallel technical development havenow brought the scientific community to a very high level of understanding withstructural insight of the interaction of peptides and small molecules with GPCR
class B receptors. The discovery of semaglutide is a fantastic example on howtechnology and biologic understanding have developed in parallel ending with asuperior peptide for treatment of diabetes.

Jesper Lau, Ph.D., Vice President, Protein & Peptide Chemistry, Novo
Nordisk A/S, Denmark
3:00 New Strategies and Technologies in

Oligonucleotide Therapeutic Development
Inability to selectively deliver antisense oligonucleotide(ASO) therapies to ß-cells is a substantial barrier to thedevelopment of treatments for ß-cell specific diseases. We
show that the GLP1 receptor can be used as a targeting
approach for efficient and selective delivery of ASO to pancreatic ß-cells in cells
and in animals.

Expression of Pseudo-natural Peptides
This lecture discusses recent progress in ribosomal invitro expression of pseudo-natural peptides containing
D-amino acid, beta-amino acids, and other exotic aminoacids. This method opens a new opportunity to explore
novel sequence space of pseudo-natural peptides.

Hiroaki Suga, Ph.D., Professor of Chemistry, School of Science, The
University of Tokyo, Japan
4:30 The Oligo Therapeutics Market

Landscape – A 17 Year Retrospective
on Market Trends, Deals, Investments
and Key Clinical Developments and a
Look to the FutureOver the last 17 years the number of oligo therapeutic
programs has grown from roughly 100 programs in 2002 to over 400 in
2018, driven by the emergence of new classes such as siRNA, miRNA, and
CRISPR therapeutics. This presentation will provide an overview of the oligo
therapeutic programs currently in development, as well as, an analysis ofdeal activity and investment trends over the last 17 years. Major market
trends across the biotech and pharma space will be examined in relation to
the current oligo therapeutic market environment.

Alun Garner, Business Development Manager, Nucleic Acid Solutions
Division, Agilent Technologies, Inc., USA
5:00 Close of Day One
6:00 Networking Dinner in Tokyo
Network with fellow AsiaTIDES attendees from around the world byattending this Networking Dinner event at a Tokyo restaurant. Sign up toattend this optional dinner by selecting this dinner option during registration(additional fee required)

Thazha P. Prakash, Ph.D., Executive Research Fellow,
Ionis Pharmaceuticals, USA

MAIN CONFERENCE PLENARY SESSION • Wednesday, February 27, 20198:00 Registration and CoffeemRNA Therapeutics
8:55 Chairperson’s Remarks
Jim Thompson, Ph.D., Head CMC Project Management,
Moderna Therapeutics, USA
9:00 An Introduction to mRNA Therapeutics
Jim Thompson, Ph.D., Head CMC Project Management,
Moderna Therapeutics, USA

10:00 Effective Delivery of Therapeutic Proteins by mRNAIn recent years the field of mRNA therapeutics has expanded significantly.
mRNA drugs can fill critical gaps not met by traditional small moleculedrugs, available biological treatments, and emerging gene therapies. Ourdata supports the ability of sequence-engineered chemically unmodified
mRNA to achieve ample protein expression.

Nigel Horscroft, D.Phil., Area Head, Molecular Therapy, CureVac AG,
Germany
10:30 Networking Refreshment Break and Exhibit/Poster Viewing

9:30 Novel mRNA Immunotherapies
Thanks to its unique characteristics, mRNA may be employed for the potent
treatment of cancer and other diseases. The potential of mRNA-based
therapeutics will be discussed and preliminary clinical data from BioNTech´smRNA immunotherapy programs will be presented.

Robert Jabulowsky, Ph.D., Deputy Head of Project Management,
BioNTech AG, Germany

Sponsored Presentation Opportunities Available
Sponsored presentation opportunities are available for companies
who have an exciting oligonucleotide or peptide technology orapplication that they are interested in sharing duringa presentation in the conference sessions. For more information andpricing, please contact Patrick Shaughnessy at
Tel: +1- 857-262-5325; Email: Patrick.Shaughnessy@KNect365.com

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MAIN CONFERENCE • Wednesday, February 27, 2019 (continued)
OLIGONUCLEOTIDE TRACK
11:10 Chairperson’s Remarks

Optimizing Properties of Oligonucleotides
11:15 From Stereopurity to Precision Medicine: Optimizing theProperties of Antisense Nucleic Acid Therapeutics

Wave Life Sciences has developed proprietary synthetic chemistry andmanufacturing capabilities that we are using to design and produce
stereopure antisense oligonucleotides (ASOs) for patients with serious,
genetically defined disease. In our evaluation of ASOs that promote
RNase H-mediated activity or exon-skipping activity, we have exploredthe relationships among sequence space, chemical modification andbackbone stereochemistry to optimize their pharmacological properties.
We have discovered a relationship between the molecular configuration of
our ASOs and their activity. We will present data from multiple programs
that illustrate how our chemistry platform allows us to optimize ASOs formechanism (e.g., gene knockdown or splicing), properties in vitro and invivo, and distribution to an array of tissues.

Chandra Vargeese, Ph.D., Senior Vice President, Head of Drug
Discovery, WAVE Life Sciences, USA
11:45 The Role of Base Modifications in Folding, Binding andPharmacology of DNA-based Aptamers

Base modifications front-loaded into SELEX libraries have expanded
the range of proteins for which high-affinity aptamers can be selected.
The composition and physico-chemical properties of modified side
chains profoundly influence folding, binding, metabolic stability andpharmacokinetic properties of aptamers, with implications for developmentof new diagnostics and therapeutics.

Nebojsa Janjic, Ph.D., Chief Science Officer, SomaLogic, Inc., USA
12:15 Next Generation Locked Nucleic Acids
Locked Nucleic Acids (LNA) have come a long way from research tools toclinically validated drug candidates. As we are constantly developing thisplatform, potential next generation analogues have been identified, showing
very promising drug properties. Here we will report our latest observationsof backbone modified LNA with a particular focus on thiophosphate
modifications, including stereo-defined internucleoside linkages.

Konrad Bleicher, Ph.D., Expert Scientist, F. Hoffmann-La Roche Ltd.,
Switzerland
12:45 Networking Luncheon with Poster and Exhibit Viewing
1:55 Chairperson’s Remarks
2:00 In Vivo Activity of Authentic and Theoretical Impurities

in RNAi Therapeutics

RNAi therapeutics are being developed using a broad range of nucleic
acid chemistries and structures, including single and double stranded
modalities. RNAi therapeutics, like all drug substances, contain active
ingredient-related impurities. Typical impurity analyses for nucleic acidtherapeutics focus on shorter or longer strand lengths, but actual impurity
profiles are far more complex. Duplex structures confound these analyses.
In vivo data from a large series of identified and theoretical impurities will
be presented.

Bob Brown, Ph.D., CSO and SVP R&D, Dicerna Pharmaceuticals, USA
2:30 Optimizing Delivery of mRNA Therapeutics
Lipid Nanoparticles are the leading technology for delivery of mRNA
therapeutics. This presentation will describe the features of LNP critical for
maximizing the therapeutic index of mRNA products.

Peter Lutwyche, Ph.D., Chief Technology Officer, Genevant Sciences
Corporation, Canada

Sponsored Presentation Opportunities Available
Sponsored presentation opportunities are available for companies
who have an exciting oligonucleotide or peptide technology orapplication that they are interested in sharing duringa presentation in the conference sessions. For more information andpricing, please contact Patrick Shaughnessy at
Tel: +1- 857-262-5325; Email: Patrick.Shaughnessy@KNect365.com

PEPTIDE TRACK
11:10 Chairperson’s Remarks

Peptide Discovery and Peptide Drug Targets
11:15 Plants as Biofactories for Producing Peptide-based

Pharmaceuticals

This presentation will provide an update on our progress using plants
to discover novel cyclic peptides and then using crop plants to produce‘designer’ cyclic peptides with applications in cancer, multiple sclerosis andcardiovascular disease.

David Craik, Ph.D., Professor of Biomolecular Structure, Institute for
Molecular Bioscience, University of Queensland, Australia
11:45 Genetically Encoded Selection of Novel Cyclic andBicyclic Architectures

The talk will describe genetically-encoded (GE) platform for discovery ofmacrocyclic and macrobicyclic peptides synthesized by aqueous late-stage
functionalization of readily-available libraries of peptides displayed onphage. The structure of these chemical post-translational modifications
can be encoded in the genome of phage using silent encoding technology.
The resulting phage displayed libraries of “unnatural” macro(bi)cyclic
peptides could be used to target either proteins or cells and tissues; thelatter targets are difficult to address with DNA/RNA or bead-based libraries.
Expanded chemical space offers value-added properties such as stability
to aggressive protease environment and incorporation of unnatural
chemotypes that are known to increase bioavailability.

Ratmir Derda, Ph.D., Associate Professor, Department of Chemistry,
University of Alberta, Canada
12:15 Intracellular Screening for Selective Antagonists of an

Oncogenic Transcriptional Regulator

Our research focuses on design, intracellular library screening and selection of
peptide antagonists with high affinity and selectivity for inhibiting protein-protein
interactions implicated in disease. Therapeutically relevant targets that include
the oncogenic Activator Protein-1 transcriptional regulator, and β-amyloid /α-synuclein proteins implicated in AD/PD. Target-specificity is further enhancedby expressing off-target proteins during selection with antagonists downsized
and refined using structure-inducing constraints and non-natural sequences.

Jody Mason, Ph.D., Associate Professor, Biochemistry, University ofBath, United Kingdom
12:45 Networking Luncheon with Poster and Exhibit Viewing
1:55 Chairperson’s Remarks
2:00 Inhibition and Degradation of Drug Targets Using

bioPROTAC mRNAs – A Novel Approach with Broad
Therapeutic Potential

To tackle historically intractable targets, we have developed a platform
employing targeted degradation. Specifically, we have engineered fusion
constructs involving two components I) mini-proteins/peptides withhigh-affinity against therapeutic targets linked to II) truncated E3 ligase
receptors. These ‘bioPROTACs’ have proven broadly successful with many
constructs showing robust degradation activity. Currently, we aim to apply
this technology as research tools and therapeutically by pursuing delivery
strategies of bioPROTAC mRNAs.

Jeff Chang, Ph.D., Associate Scientist and Post Doc Fellow, Early
Discovery Pharmacology, MSD Singapore

Peptide Synthesis
2:30 Massively Parallel Synthesis and Lead Optimization of

Peptidomimetics

We have developed a maskless photolithography peptide synthesis andscreening platform that has been applied both for lead discovery andoptimization. Our chemical catalog of over 350 amino acid building blocks
combined with rapid library design and robust workflows, allows synthesisof over 18 million unique linear or cyclic peptides in less than 48 hrs on asingle glass slide. The process is scalable and large libraries of millions
of peptides can be synthesized on demand. We have made incremental
upgrades and exploring end-point detection, real-time kinetic interaction,and cell-based phenotypic assays. Here, we will describe the coretechnology and its application to rapidly and systematically evolve highaffinity, high-specificity binding peptides to protein targets in a reproducible
and digitally controlled process. We will demonstrate some case-examples,and recent process upgrades that enable real-time kinetic analysis forthousands of peptide-protein interactions in parallel.

Jigar Patel, Ph.D., Director, Peptide Technologies, Roche Madison, USA

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MAIN CONFERENCE • Wednesday, February 27, 2019 (continued)
OLIGONUCLEOTIDE TRACKPEPTIDE TRACK

Oligonucleotide CMC Strategies

3:00 A New Scenario for Disulfide Peptide Synthesis Based on

3:00 Presentation Title TBA
Satoshi Inoue, Ph.D., Scientist, Bio-functional Molecular ChemistryGroup, Ajinomoto Co., Inc., Japan
3:30 Networking Refreshment Break with Poster and Exhibit Viewing
4:00 Antisense Oligonucleotide Purification Process:Successes and Challenges During Scale-up

Recently, our first full scale GMP batch for an antisense oligonucleotide
was manufactured in the newly built synthesis suite at Biogen. A fourstep purification process was then carried out in the existing flexible
volume manufacturing facility that, up until this point, has been used for
manufacturing Biogen’s protein-based parenterals. This was our first test
case to demonstrate that Biogen can manufacture ASOs safely, at scale,and achieve high purity and yield. Results are presented on the scalabilityof the ASO process from bench to GMP scale, highlighting successes andchallenges faced with scale-up of the downstream ASO process.

Robert Gronke, Ph.D., Senior Principal Scientist, TechnicalDevelopment, Biogen, USA
4:30 NittoPhase® HL, High Loading Polymeric Solid Support

for Efficient and Cost-Effective Oligonucleotide
Synthesis

When it comes to “Therapeutic Oligonucleotide manufacturing”
performance and quality of the solid support matter. In this presentation,
quality and performance of NittoPhase® HL is discussed and advantagesof this cGMP manufactured solid support in comparison with other solid
supports are discussed.

Mohammad Ahmadian, Ph.D., Senior Director, Kinovate Life
Sciences, Nitto Denko Technical Corporation, USA
5:00 Presentation Title TBA
Intertek Speaker TBA
5:30 Networking Cocktail Reception with Poster and Exhibit Viewing

Npys Chemistry

Chemistry based on 3-nitro-2-pyridinesulfenyl (Npys) group gave us a new
scenario on disulfide peptide synthesis. Two topics will be discussed in thelecture, i.e., new solid- or no-solid-supported Npys-sulfenates (Npys-OR) as
disulfide bond-forming agents and solid-phase-assisted disulfide ligation method.
The latter actualizes intriguing “disulfide-driven cyclic peptides synthesis”.

Yoshio Hayashi, Ph.D., Professor, Department of Medicinal Chemistry,
Tokyo University of Pharmacy and Life Sciences, Japan
3:30 Networking Refreshment Break with Poster and Exhibit Viewing

Optimizing Peptide Properties
4:00 Peptide Drug Discovery Case Study: Appropriate

Screening for ADME Properties and Pseudo Allergic
Reactions at An Early Stage is Key to Success
The key to success for development of peptide therapeutics is to use anappropriate screening funnel to correctly drive the SAR at the early stageof the project. This presentation will address important issues to consider
in peptide development, namely: subcutaneous and plasma metabolism
with in vitro/in vivo correlation to optimize bioavailability; potential reactionsof peptide induced pseudo-allergy; immunogenicity; oligomerization andaggregation tendencies.

Raffaele Ingenito, Ph.D., Senior Scientist, Peptide Chemistry,
IRBM Science Park, Italy
4:30 Beyond Antibodies: Targeting VEGF-A and PD-1 with

Synthetic D-proteins

Small proteins have historically been under-utilized as human therapeutics
because they are rapidly metabolized, have short half-lives and can be
neutralized by anti-drug immune responses. However, proteins composed
entirely of D-amino acids (“D-proteins”) are resistant to proteases, have longer
half-lives and significantly reduced immunogenicity. These properties alsoenable D-proteins to better penetrate tissues and solid tumors. Because
D-proteins are chemically manufactured they can be engineered in ways notpossible using biologic manufacturing. Mirror image phage display technology
has been used to create D-proteins that are potent inhibitors of VEGF-A, an
important therapeutic target for treating cancer and eye diseases. D-protein
inhibitors of PD-1, an important immune-oncology target, have also beendeveloped. Therapies combining VEGF-A and PD-1 inhibition have resulted in
improved clinical outcomes for liver, lung and kidney cancers. This presentation
will focus on the development of D-protein inhibitors to VEGF-A and PD-1.

Dana Ault-Riche, Ph.D., CEO, Reflexion Pharmaceuticals, USA
5:00 Of Peptides and PANDAS: Innovative Preclinical

Assessment Tools for Safety and Efficacy of Protein and
Peptide Therapeutics

Sponsored Presentation Opportunities Available
Sponsored presentation opportunities are available for companies
who have an exciting oligonucleotide or peptide technology orapplication that they are interested in sharing duringa presentation in the conference sessions. For more information andpricing, please contact Patrick Shaughnessy at
Tel: +1- 857-262-5325; Email: Patrick.Shaughnessy@KNect365.com

Media Partner

The FDA recently released a new draft guidance defining the equivalence ofa rDNA peptide product and a synthetic peptide product. The draft guidance
enables generic manufacturers of peptide drugs to file an Abbreviated New
Drug Application (ANDA) for synthetic peptide drug products that refer tolisted drugs of rDNA origin. Since the processes for manufacturing thegeneric and reference drug (RLD) are not equivalent, peptide drugs canbe associated with impurities. Impurities can result from changes in thesequences due to deletions, insertions, integration of incorrect amino-acids
and modifications and also impurities related to the synthetic production.
The FDA draft guidance requires manufacturers to prove that the synthetic
peptide product does not contain impurities that have an increased affinity
for major histocompatibility complexes and potential for engaging immune
response, which may drive undesired anti-drug antibody development. We
have used both immunoinformatics-driven analysis and in vitro validation
assays to perform immunogenicity risk assessment of peptide generics.
This combination of in silico and in vitro tools is referred to the PANDA
assay which can be used to support generic peptide drug equivalency in an
ANDA application. This presentation will provide insight as to the process of
performing the PANDA assay, illustrating the process with two case studies(such as Calcitonin and Teriparatide).

Annie De Groot, M.D., Professor (Research) and Director, Institute for
Immunology and Informatics, University of Rhode Island and Founder,
CEO and CSO, Epivax, Inc., USA
5:30 Networking Cocktail Reception with Poster and Exhibit Viewing

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MAIN CONFERENCE • Thursday, February 28, 20198:40 Chairperson’s Remarks

Regulatory Strategies for Peptides and Oligonucleotides
8:45 Regulatory and Clinical Development Strategy for Peptides in the Era of ICH; Oligonucleotide Products - Comparing theRegulatory Environment in US and China

The content of this presentation includes an illustration of combined regulatory and clinical development strategies for peptides among ICH countries by taking
advantage of new ICH guidelines such as E17 MRCT guideline. It will also compare the regulatory environment for oligonucleotides in the US and China.

Dan Zhang, M.D., Co-Founder & CEO, Fountain Medical Development, China
9:15 US Regulatory Update: Peptide and Device Combination Products and the Lessons Learned from the Approval of

Oligonucleotide Product, Macugen®

For the convenience of dose delivery and patience compliance, the peptide therapeutics often come as combination products with either a prefilled syringe or a disposable
and non-disposable injector device. Frequently, such a peptide combination product brings different challenges for the development and regulatory review that require theregulatory knowledge of both drug/biologic and device. Also, the recent approval of another oligonucleotide product, Mucagen, represents another milestone among the few
approvals in US. The presentation will discuss various regulatory issues related to a device-drug or device biologic combination products and also the lessons learned from the
new approval of Macugen oligo product in US.

Duu-Gong Wu, Ph.D., Senior Director, Regulatory Consulting, PPD (and Former Deputy Division Director of Division of New Drug Chemistry, CDER, FDA), USA
9:45 Understanding FDA’s Draft Guidance for a Generic Product of a Highly Purified Synthetic Peptide that References an

Approved Application for a Recombinant Peptide ProductThis recently published draft FDA guidance permits generic synthetic peptides to referencing a marketed drug product of rDNA origin. The draft guidance will be
discussed with respect to opportunities and challenges.

David T. Lin, Ph.D., Senior Consultant, Biologics Consulting, (Former Chemistry Team Leader and Former Acting Division Director, Division of New Drug
Chemistry, CDER, FDA), USA
10:15 Networking Refreshment Break and Exhibit/Poster Viewing

OLIGONUCLEOTIDE TRACK
10:55 Chairperson’s Remarks

Delivery Strategies
11:00 Emerging RNA Therapeutics: Delivery Approaches for

mRNA, siRNA and Beyond

Delivery is a still key challenge to expand therapeutic application ofoligonucleotide therapeutics such as siRNA, ASO and mRNA. AccuRna
has unique polymer-based delivery platforms for short chain RNA/DNA
and mRNA. In this talk, we will describe how we could apply our delivery
platform to expand the possibility of nucleotide therapeutics.

Shiro Akinaga, Ph.D., Director, Chief Scientific Officer, AccuRna Inc.,
Japan
11:30 siRNA Delivery is Still the Key to Therapeutic SuccessIn the past 2 decades we have seen dramatic progress in the adoption ofRNAi technologies - from understanding the key criteria for siRNA design
to improve function and specificity, to using siRNAs to identify and validate
targets driving disease and now their validation as viable therapeutics
in their own right. However, while there are many tools that allow digital
design of siRNAs that can silence gene targets, many diseases such as
cancer require inhibition of multiple genes in parallel and the genes thatneed to be targeted may change with time and stage of disease. We
therefore need vehicles that can deliver more than one siRNA into the same
cell concomitantly. Modification of these delivery vehicles with ligands
to home to specific cell types in tissues may provide better therapeutic
efficacy with a higher safety margin. This presentation will discuss our
experiences in identification of multiple siRNAs with synergistic effects,
development of delivery vehicles that can carry multiple siRNAs, and earlysteps we are taking in new screening methods to help rapidly identify andvalidate functional targeting ligands. The benefits of our delivery vehicles
for multiple siRNA delivery and the steps needed to drive these innovations
to the clinic will also be discussed.

David M. Evans, Ph.D., Co-founder and CSO, Sirnaomics Inc., USA
12:00 NJA-730, The First Therapeutic Compound from

NapaJen’s Platform Delivery Technology for aGvHD(acute Graft versus Host Disease) Is in Clinical Stage
Our platform technology is targeted delivery of oligonucleotide therapeutic
compounds to antigen presenting cells using SPG (schizophyllan) as adelivery vehicle. We will introduce NapaJen’s platform delivery technology
and report progress in Phase 1 trial of NJA-730, a CD40-targeting siRNAcomplexed with SPG, which shows potent pharmacological activity.

Kenji Arima, Ph.D., Chief Development Officer, NapaJen Pharma, Co.
Ltd., Japan
12:30 Networking Luncheon with Poster and Exhibit Viewing

PEPTIDE TRACK
10:55 Chairperson’s Remarks
El Djouhar Rekaï, Ph.D., Head of Development & Manufacturing
Process, PolyPeptide Group, Belgium

Peptide CMC Strategies
11:00 Introducing Green and Sustainable Solvents in Liquid

Phase Peptide Manufacturing Processes

The challenge for the pharmaceutical industry is to find ways to develop
and manufacture APIs based on environmentally-friendly and efficient
processes. The selection of solvents and excess of reagents, together
with process design including solvent recycling/VOC emission control areemphasized with the objective to minimize the production of wastes/gas
emission while being operationally safe and economical. We will describe
an overview of the PolyPeptide Group approach for setting environmentallyfriendly, safe and cost-effective processes in liquid phase peptide synthesis.
Case studies of successful process development to reach improved green
processes will be highlighted.

El Djouhar Rekaï, Ph.D., Head of Development & Manufacturing
Process, PolyPeptide Group, Belgium
11:30 Peptide Quality Attributes and USP Standards
United States Pharmacopeia (USP) has official monographs for peptides.
To understand and evaluate the current regulatory considerations andexpectations, an expert panel was formed. The Panel after detailed
deliberation, recommended key Quality Attributes for incorporation in USP
standards. Compendial requirements for new monographs are aligned
to the panel recommendation. The presentation will describe the Expert
Panel’s recommendations on the quality attributes with case studies from
USP’s current synthetic peptide program.

Ranjan Chakrabarti, Ph.D., Vice President & Head - Global Biologics
Lab and Standards, United States Pharmacopeia, India
12:00 Cost Efficient Peptide Purification via ZEOsphere DRP

Mixed-Mode Chromatography

Peptide and Oligonucleotides are important API’s for modern pharmaceuticals
and have to be produced on preparative scale were costs are under pressure.
RPC and IEX are well-established but costly chromatographic modes.
ZEOsphere DRP Mixed-Mode materials combines the dual action of strong
IEX groups (acidic or basic) and Reversed Phase ligands on the packing
surface. ZEOsphere DRP in repulsion-attractive mode shows improved
selectivities, leading to substantial lower production costs.

Jurgen Machielse, Business Development Director, Zeochem AG,
Switzerland
12:30 Networking Luncheon with Poster and Exhibit Viewing

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MAIN CONFERENCE • Thursday, February 28, 2019 (continued)
OLIGONUCLEOTIDE TRACK
1:40 Chairperson’s Remarks

Preclinical and Clinical Case Studies
1:45 Anti-FGF2 Aptamer RBM-007 in Phase I/IIa trials for

Wet AMD

RBM-007 is a novel oligonucleotide-based aptamer with potent anti-FGF2
activity. Intravitreal administration of RBM-007 in animals demonstrated
anti-angiogenic and anti-scarring effects, consistent with a therapeutic
effect desired in the treatment of wet (exudative) AMD. We have enteredphase I/IIa clinical trials last summer in the US.

Yoshikazu Nakamura, Ph.D., President and CEO, RIBOMIC Inc., andProfessor Emeritus, The University of Tokyo
2:15 Therapeutic RNA Medicines: The Silence Therapeutics

Experience in Haematology and Rare Diseases

Silence Therapeutics’ pipeline focus on hepatocyte-associated diseases.
Our most advanced asset, SLN124, is a GalNAc-conjugated siRNA targeting
hepatic TMPRSS6. SLN124 reduces serum and tissue iron levels in arodent model for hereditary hemochromatosis type 1 both as monotherapy
and in combination with an oral iron chelator. SLN124 also demonstrates
therapeutically relevant, dose-dependent and long-lasting effects oniron stores, erythropoiesis and anaemia in an animal model for betathalassemia. The first-in-human study is planned to start in 2019 in betathalassemia and myelodysplastic syndrome patients. We actively explore
options for fine tuning of GalNAc-conjugated siRNA design such as siRNA
modification patterns, end stabilisation, linker chemistry, and the number
and location of GalNAc units. Combination of novel design elements areincluded in siRNA conjugates in pre-clinical stage programs with targets in
haematology and rare diseases.

David Horn Solomon, M.D., CEO, Silence Therapeutics, United Kingdom
2:45 TANGO – Targeted-Augmentation of Nuclear Gene

Output – for the Treatment of Genetic Diseases

Stoke is developing antisense-oligonucleotide (ASO) medicines that target
pre-mRNA splicing to increase gene expression for the treatment ofgenetic diseases. Stoke’s technology, TANGO, prevents naturally-occurring,
non-productive splicing events, thereby increasing productive mRNAs andfull-length proteins. We have built a proprietary bioinformatics pipelinethat has identified over 100,000 non-productive splicing events and nearly
3,700 potentially druggable disease-associated genes. ASO screeningof prioritized disease targets has yielded potent and gene-specific upregulation for multiple diseases. One such disease, Dravet syndrome (DS), isa severe childhood epileptic encephalopathy characterized by high seizure
frequency, cognitive and motor impairments, and increased risk of SUDEP(sudden unexplained death in epilepsy). DS is caused by mutations in the
SCN1A gene leading to haploinsufficiency of the voltage-gated sodium
channel alpha subunit (NaV 1.1). We have identified ASOs that significantly
increase SCN1A mRNA and NaV 1.1 protein in vivo and demonstrate robust
efficacy in a mouse model of Dravet Syndrome. These results indicate that
Stoke’s technology could provide the first gene-specific, disease-modifying
approach to restore NaV 1.1 levels to treat Dravet Syndrome. Stoke’s
technology offers a pioneering strategy to treat diseases that result fromreduced expression or insufficient activity of a gene that contains a nonproductive splicing event. As over 50% of genes possess such events, thepotential target opportunity is significant and largely untapped.

Huw Nash, Ph.D., Chief Operating Officer and Chief Business Officer,
Stoke Therapeutics, USA
3:15 Networking Refreshment Break with Poster and Exhibit Viewing
3:45 Latest Clinical Results from an Oligonucleotide in

Development

Osamu Sato, Ph.D., General Manager, Sakigake-Projects; DS-5141
and DS-1647 and Senior Executive Advisor for Head of R&D, Daiichi
Sankyo Co., Ltd., Japan

4:15 Asymmetric siRNA Targeting Fibrotic and Ocular Disorders
OLX10010, an anti-fibrotic cell-penetrating asymmetric siRNA (cp-asiRNA)targeting connective tissue growth factor (CTGF), effectively reduces target
gene expression as well as expression of fibrotic markers in animal model
study. Preclinical as well as clinical study update of OLX10010 in anti-skinscar will be presented. In addition to skin scar, OLX10010 has a potential to
be developed as therapeutics targeting various fibrotic disorders. We will alsopresent preclinical study data of OLX10010 in other fibrotic diseases in lungand eye, such as idiopathic pulmonary fibrosis (IPF) and subretinal fibrosis.

Dong-ki Lee, Ph.D., CEO, OliX Pharmaceuticals, South Korea

PEPTIDE TRACK
1:40 Chairperson’s Remarks
1:45 Linear and Convergent Approaches to Peptide Synthesis- A Comparison from A Manufacturer’s Point of View

Peptides can be synthesized using liquid phase peptide synthesis (LPPS),
solid phase peptide synthesis (SPPS), and combinations thereof, i.e.
fragment condensation or ligation. The shorter peptides used in fragment
condensations or ligations are themselves accessible via the mentioned
synthesis techniques. Fragment condensations are classically carried out
in solution with C-terminus activated chemically or enzymatically. Larger
fragments can also be ligated by native chemical ligation (NCL). On theother hand, state-of-the-art SPPS conditions are suitable for the synthesisof long peptides. All the mentioned approaches have pros and cons; theselection of the synthesis route depends on the peptide sequence, the target
specifications, time-to-market for development, and last but not least the
manufacturer: SPPS, LPPS and hybrid approaches differ significantly in thenumber of unit operations and the complexity of the required equipmenttrains. Manufacturer’s capabilities and platform knowledge play a major rolein route selection. In addition, cost of goods, process efficiency, maximum
output at peak demand, and green chemistry considerations (e.g. solvent
consumption, reaction conditions, waste reduction, replacement of hazardous
chemicals) are frequently discussed in this context. In this presentation linear
vs. convergent approaches for peptide synthesis are compared and pros andcons are discussed from a manufacturer’s point of view.

Daniel Samson, Ph.D., Vice President API SPPS, Bachem AG, Switzerland

Preclinical and Clinical Case Studies
2:15 ESCAPE-NA1 and FRONTIER: Two Complementary Phase

3 Trials to Demonstrate the Safety and Efficacy of NA-1
in Acute Ischemic Stroke

The development of a safe and effective treatment for stroke is one of thegreatest challenges in biomedical sciences. NA-1 is a novel neuroprotective
peptide that acts by disrupting protein interactions with PSD-95 in the brain.
ESCAPE-NA1 is a pivotal Phase 3 underway in 8 countries trial to assess thesafety and efficacy of NA-1 in reducing disability in stroke victims who are
candidates for endovascular thrombectomy. FRONTIER is a Phase 3 trial
in which NA-1 is administered by paramedics in the ambulance to subjects
with suspected stroke within 3 hours of onset.

Dave Garman, Ph.D., Chief Technology Officer, NoNO, Inc., USA
2:45 The Orally Available Clinical Stage All-D-enantiomeric

Peptide PRI-002 Reverses Cognition Deficits andDecelerates Neurodegeneration in Transgenic
Alzheimer’s Mouse Models

PRI-002 proved to eliminate already formed, toxic Aβ oligomers and toreverse cognitive deficits even in old-aged transgenic Alzheimer’s disease(AD) mice with full blown pathology and deficits, even by oral administration.
PRI-002 is fully blood-brain-barrier penetrable and demonstrated successful
target engagement. I will summarize successful in vivo proof-of-concept infour treatment studies in three different transgenic animal models in threedifferent laboratories. I will report first clinical data for PRI-002.

Dieter Willbold, Ph.D., Director, ICS-6 Structural Biochemistry,
Forschungszentrum Jülich, Germany
3:15 Networking Refreshment Break with Poster and Exhibit Viewing
3:45 NRP2945 with Anti-epileptic Activities in Patients with

Drug-resistant Absence Epilepsy

NRP2945 belongs to the family of Neural Regeneration Peptides (NRPs),
which have been shown to be responsible for “re-setting” the impaired or
injured central nervous system back to a status of inhibitory neural circuits
through quick upregulation of synaptic GABA A receptor subunits after
neuronal CCR3/CXCR4 chemokine receptor complex activation. NRP2945
has completed a large clinical phase 1 study with healthy volunteers
and only showed mild injection site reactions when administered as
subcutaneous bolus every 48 hrs. The drug candidate is now tested in asmall proof-of-concept phase 2 study by dosing a patient cohort diagnosed
with drug-resistant typical absence epilepsy to monitor NRP2945’s ability
to downregulate seizure frequencies. It is well known that absence seizures
are originating from dysfunctional thalamo-cortical network activity and
NRP2945’s ability to re-set the inhibitory status of this particular network
favours this stable peptidomimetic to provide significant anti-seizure activities
for this patient population. Results from the clinical trial will be presented.

Frank Sieg, Ph.D., Chief Scientist, CuroNZ Limited, New Zealand

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MAIN CONFERENCE • Thursday, February 28, 2019 (continued)
OLIGONUCLEOTIDE TRACKPEPTIDE TRACK
4:15 Development of a Peptide Vaccine for the Induction of

4:45 MicroRNA-155 Inhibition by Cobomarsen Demonstrates

Epitope-specific Antibodies

Encouraging Activity in Hematological Malignancies

microRNA-155 (miR-155) is an important control point in the regulationof pathways implicated in oncology and inflammatory disease. Its
overexpression has been shown to be an indicator of poor prognosis in avariety of hematological malignancies. Cobomarsen (MRG-106), an inhibitorof miR-155, is currently being evaluated in several potential indications,
including Cutaneous T-cell Lymphoma (CTCL) and Adult T-cell Leukemia /Lymphoma (ATLL). An overview of our latest clinical observations will bepresented.

William S. Marshall, Ph.D., President and Chief Executive Officer,
miRagen Therapeutics, Inc., USA
5:15 Close of Conference

FunPep has developed chimeric peptide vaccines composed of B-cell and T-cell
epitopes. The peptide vaccine elicits high-titered and high-affinity antibodies thatare able to recognize the target protein without co-treatment of any adjuvants.
In this presentation I will introduce our recent progress from the viewpoint ofpreclinical studies.

Hideki Tomioka, Ph.D., Member of the Board and Director, Research &Development, FunPep Co., Ltd., Japan
4:45 Osseotide, A Synthetic, Selective Osteogenic Peptide for

the Treatment of Osteoporosis

Mineralization in mammalian cells is accomplished by concerted regulationof protein-based extracellular matrix (ECM) components, such as noncollagenous proteins and collagen fibrils in hard tissue such as bone andteeth. Synthetic collagen-binding motif (CBM) peptide mimicking osteopontin,
named as Osseotide, has been developed to selectively target osteoblast
differentiation, which is clear contrast to current therapy targeting osteoclast.
The peptide increased osteogenic differentiation while arresting adipogenic
differentiation. In ovariectomized (OVX) mice, estrogen deficiency induced
osteoporosis and increased fat tissue deposition. Significant bone loss by
osteoporosis was restored by the treatment of Osseotide, evident by welldeveloped bone structure and bone formation. In addition, significant decrease
in total fat and subcutaneous fat was observed in the Osseotide treated group,
which is similar extent to that treated by estradiol or PTH. Taken together,
these results suggest that the Osseotide could be an effective therapeutic
agent for osteoporosis due to its selective targeting osteogenic differentiation.

Yoon Jeong Park, Ph.D., Professor, Seoul National University and Chief
Technology Officer, NIBEC, South Korea
5:15 Close of Conference

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ASIATIDES 2019 ADVISORY BOARD
Hiroaki Suga, Ph.D., Professor of Chemistry, School of
Science, University of Tokyo, Japan
Osamu Sato, Ph.D., General Manager, Sakigake-Projects;
DS-5141 and DS-1647 and Senior Executive Advisor for
Head of R&D, Daiichi Sankyo Co., Ltd., Japan
Muthiah (Mano) Manoharan, Ph.D., Senior Vice Presidentof Drug Discovery, Alnylam Pharmaceuticals
James Thompson, Ph.D., Head of CMC ProjectManagement, Moderna Therapeutics, Inc.
Daisuke Takahashi, Ph.D., Senior Principal Researcher,
Bio-functional Molecular Chemistry Group, ResearchInstitute for Bioscience Products & Fine Chemicals,
Ajinomoto. Co., Inc., Japan
Hideaki Sato, President and CEO, Luxna Biotech, Japan
Robert Hagopian, Director Business Development,
PolyPeptide Laboratories, USAGroup Rates
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Dong-ki Lee, Ph.D., Professor, Sungkyunkwan University
and CEO & Founder, OliX Pharmaceuticals, Korea
Shawn Lee, Ph.D., President and CEO, CPC Scientific, USA
and China
Patrick Lu, Ph.D., Founder, President and CEO, Sirnaomics,
Inc., USA
Michael McGinley, Global Technical Manager,
Phenomenex, USA
Bruce Morimoto, Ph.D., Vice President, Drug DevelopmentOperations, Alkahest, USA

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El Djouhar Rekaï, Head of Peptide Products Operation,
PolyPeptide Group, Belgium
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
Dmitry Samarsky, Ph.D., Chief Technology Officer,
Sirnaomics, USA
Mimoun Ayoub, Ph.D., Director and Head of North
American and Emerging Markets, CordenPharma
International, Switzerland

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