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Page 1: AsiaTides -Peptides and Oligonucleotides 2015

Media Parters: Premier Publication: Organized by:

Where Global Oligonucleotide and Peptide Leaders Connect to Share Groundbreaking Science and Form Successful Business Collaborations

March 3-5, 2015 • Hyatt Regency Osaka • Osaka, Japan

IBC’s 7th Annual

Keynote Presentations

Transfer of exRNAs in Cancer Metastasis

Takahiro Ochiya, Ph.D. Chief, Division of Molecular and Cellular Medicine, National Cancer Center Research Institute, Japan

Amylin and its Analog: Potential Diagnostic Test and Therapeutic Drug for Alzheimer's Disease

Wendy Wei Qiao Qiu, M.D., Ph.D. Associate Professor, Departments of Psychiatry and Pharmacology, Alzheimer's Disease Center, Boston University School of Medicine, USA

n Accelerate Your Discovery and Development Programs by Applying Successful Strategies from 18+ Case Study and New Data Presentations from Arrowhead, Isis, Roche, Takeda and other Industry Leaders from not only Japan, but also USA, Europe and Asia

n Ensure a Successful Market Launch by Utilizing Manufacturing and Analytical Strategies from Global Experts from 8 different countries, including Avecia, Agilent, Bachem, Ajinomoto and more

n Gain Faster Therapeutic Approval by Hearing Guidance from Two Former FDA Officials for Overcoming the Latest Regulatory Requirements and Challenges

n Highlight Your Expertise in Untapped Marketsby Forging Partnerships with Key Decision Makers and Academic Leaders from Across the Globe

Register Early and Save Up to USD300! www.IBCLifeSciences.com/AsiaTIDES

You Asked and We Listened – AsiaTIDES is Coming to Osaka in 2015!

オリゴヌクレオチドとペプチドのグローバルリーダー達が集い、革新的な科学研究結果を共有し、事業コラボレーションを成功させる場です

IBC主催 第7回 Asia TIDES オリゴヌクレオチドとペプチド® 研究、技術および製品開発2015年3月3 -5日大阪、ハイアット・リージェンシー大阪

Page 2: AsiaTides -Peptides and Oligonucleotides 2015

Dear Colleagues,

Coming to Osaka in 2015, IBC’s 7th Annual AsiaTIDES is the preeminent conference in Asia to gain the latest development updates on preclinical and clinical peptides and oligonucleotides. Only at AsiaTIDES can you connect with global leaders who share their novel strategies and lessons learned to help accelerate your oligonucleotide and peptide therapeutics to market.

Your registration at the 2015 event in Osaka gives you exclusive access to the most up-to-date reports available only at AsiaTIDES from not only Japanese peptide and oligonucleotide innovators, but also their Asian and U.S. collaborators – providing you with crucial knowledge that you can immediately apply to your development portfolio. Additionally, our 2015 program brings together more thought-leaders and more new data than ever before to help you effectively meet regulatory expectations and overcome complex product and manufacturing challenges.

So, register early and save up to USD300 and be sure to log on to www.IBCLifeSciences.com/AsiaTIDES for program announcements and updates.

I look forward to seeing you in March 2015 at AsiaTIDES in Osaka: The premier meeting to accelerate your oligonucleotide and peptide discovery and development programs towards commercial success.

Sincerely,

Mark A. DeSorbo Conference Producer

2 ToRegister,Call:(+65)65082401•Fax:(+65)65082407•E-mail:[email protected]

Advance Your Oligonucleotide and Peptide Therapeutics Towards Commercial Success

MainConference•Plenary Keynote Presentations Tuesday, March 3, 2015

8:00 Registration and Coffee/Tea 9:10 Chairman’s Remarks

Mark Graham, Executive Director, Cardiovascular Disease Research, Antisense Drug Discovery, Isis Pharmaceuticals Inc.

9:15 Transfer of exRNAs in Cancer Metastasis The existence of circulating miRNAs and exosomes in the blood of cancer patients has raised the possibility that disease-specific miRNAs and exosomes may serve as a novel diagnostic marker. Moreover, exosomes secreted from tumor cells contribute micromanaging tumor microenvironment. We and other groups reported that tumor-derived exosomes mediated tumor angiogenesis and thus facilitated tumor metastasis for distant organs. Here we will discuss importance of transfer of extracellular microRNAs (exRNAs) and their contribution to tumor development and metastasis.Takahiro Ochiya, Ph.D., Chief, Division of Molecular and Cellular Medicine, National Cancer Center Research Institute, Japan

9:45 CASE STUDY / NEW DATA Amylin and its Analog: Potential Diagnostic Test and Therapeutic Drug for Alzheimer's Disease Amylin is a naturally occurring peptide with important functions in the brain, including regulating glucose metabolism. Using Alzheimer's disease (AD) mouse models, we show here the evidence that chronic peripheral treatment with amylin or its analog, pramlintide, reduced the amyloid burden and lowered the concentrations of amyloid-beta peptides (Aβ) in the brain when compared with saline treatment. Further, behavioral tests showed that learning and memory in these mice were improved by the treatment. Despite the fact that amylin and Aβ share a similar secondary structure, our study indicates the therapeutic potential of amylin type peptides for treating Alzheimer’s. Wendy Wei Qiao Qiu, M.D., Ph.D., Associate Professor, Departments of Psychiatry and Pharmacology, Alzheimer's Disease Center, Boston University School of Medicine, USA

関係各位

2015年大阪にて開催されるIBC主催の第7回Asia TIDESは、アジにおける最高峰のカンファレンスであり、オリゴヌクレオチドとペプチド治療の臨床前研究と臨床研究における最新開発情報を取得できます。皆様のオリゴヌクレオチドとペプチド治療研究の商業化を加速するため、全く新しい戦略や教訓を共有して下さるグローバルリーダー達と知り合えるのはAsia TIDESだけです。

2015年の大阪でのイベントに登録すると、日本のオリゴヌクレオチド革新者のみならず、アジアやアメリカにいる彼らの共同研究者より、Asia TIDESでしか入手できない最新レポートを入手でき、開発ポートフォリオにすぐに適用可能な重要な知識を得ることができます。さらに、2015年のプログラムでは、今までよりも多くのオピニオンリーダーとデータを一堂に集め、効率よく規制・基準を満たし、複雑な製品製造過程の課題を克服できるようにサポートします。

早めに登録すると、最大300米ドルオフとなります。プログラム詳細情報や最新情報に関しては、www.IBCLifeSciences.com/AsiaTIDES にログオンして下さい。

それでは、大阪で2015年3月にAsia TIDESで皆様とお会いできることを楽しみにしております。このプレミア会議に出席することで、皆様のオリゴヌクレオチドとペプチド研究および開発プログラムの商業化成功を促進できると確信しております。

敬具

マーク・A・デソーボ

カンファレンス・プロデューサー

10:15 Grand Opening of the Poster and Exhibit Hall with Refreshments Sponsored by:

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Oligonucleotide-BasedTherapeuticsinPreclinical and Clinical Development

Peptide-BasedTherapeuticsinPreclinicalandClinical Development

MainConference•ConcurrentTracks Tuesday, March 3, 2015

Sponsored by:

11:00 Chairman’s RemarksDavid L. Lewis, Ph.D., Chief Scientific Officer, Arrowhead Research Corp., USA

11:15 NEW DATA N-acetyl Galactosamine Conjugation Enhances Potency of Second-Generation Antisense Targeting Hepatocyte Expressed GenesSecond-generation antisense oligonucleotide (ASO) mRNA/protein potency was increased 5-10 fold versus parent following N-acetyl galactosamine (GalNAc3) conjugation of human apolipoprotein C-III, apolipoprotein(a) and angiopoeitin protein-like3 ASOs in transgenic mice. GalNAc3 ASO conjugation should significantly enhance the therapeutic index, reduce cost, and support monthly dosing regimens for hepatocyte mRNA targets.Mark Graham, Executive Director, Cardiovascular Disease Research, Antisense Drug Discovery, Isis Pharmaceuticals Inc., USA

11:45 Optimization of Phosphorothioate Antisense DNA by Controlling the Phosphorous ChiralityThis presentation demonstrates that the P-chirality affects nearly all the critical properties of the antisense drugs, from binding affinity to target RNA to metabolic stability and RNase H activity.Takeshi Wada, Ph.D., Department of Medicinal and Life Science, Faculty of Pharmaceutical Sciences, Tokyo University of Science, and Department of Medical Genome Sciences, Graduate School of Frontier Sciences, The University of Tokyo, Japan

12:15 Luncheon in Poster and Exhibit Hall 1:25 Chairman’s Remarks

Dr. Troels Koch, VP & CTO, Roche Innovation Center Copenhagen, Denmark

1:30 Using Dynamic Polyconjugate (DPC) Delivery Technology in RNAi-Based Therapeutics for the Treatment of Liver DiseaseWe have developed a platform technology called Dynamic Polyconjugate (DPC) for targeted delivery of RNAi trigger molecules. Key to this technology is a reversibly masked endosomolytic polymer. The masking chemistry prevents the polymer from interacting with blood components and non-targeted tissues, but is hydrolyzed in the acidic environment of endosomes allowing activation of the polymer and release of the RNAi-trigger to the cytoplasm of the targeted cell. Attachment of N-acetyl galactosamine moieties to the DPC enables delivery specifically to liver hepatocytes via the asialoglycoprotein receptor. We are currently using this technology in therapeutics designed for the treatment of chronic hepatitis B virus infection and alpha-1 antitrypsin deficiency-associated liver disease.David L. Lewis, Ph.D., Chief Scientific Officer, Arrowhead Research Corp., USA

2:00 Nucleic Acid Ligands with Protein-like Side Chains and Their Application in Diagnostics and TherapeuticsThis presentation discusses a new class of nucleic acid ligands with protein-like side chains, called slow off-rate modified aptamers (SOMAmers). Their broader chemical diversity results in higher success rate of SELEX, expanded range of accessible epitopes, and enhancement in nuclease resistance. These features are well-suited for large-scale proteomics, biomarker discovery and development of new diagnostic and therapeutic agents. Nebojsa Janjic, PhD, Chief Science Officer, SomaLogic, Inc., USA

2:30 NEW DATA Gene Silencing Oligos: The Next Generation in Gene SilencingThis presentation discusses a unique therapeutic oligonucleotide modality. Gene silencing oligonucleotides (GSOs) are composed of two single stranded oligonucleotides directed against the same target mRNA connected at their 5’ ends by a linker. GSOs show greater activity, potency and duration when compared to antisense oligonucleotides (ASOs) directed against the same target. In addition, GSOs mitigate the immune response seen with second generation ASOs and show no obvious signs of organ toxicity after repeat dosing. Significant work went into the SAR of GSOs demonstrates specific unique structural properties and provide opportunities for additional optimization of the drug-liker properties of GSOs. The GSOs represent a significant opportunity in exploding field of oligonucleotide therapeutics. The presentation also includes exploration of the structure/activity relationship and work with the GSOs in various preclinical models.Walter Strapps, Ph.D., Executive Director of RNA Therapeutics, Idera Pharmaceuticals, USA

11:00 Chairman’s RemarksJörg Vollmer, Ph.D., Chief Executive Officer, Nexigen GmbH, Germany

11:15 CASE STUDY / NEW DATA Design, Optimization and Early Development of Novel Peptide Drug ConjugatesAbstract not available; please check www.IBCLife Sciences.com/AsiaTIDES for updates.Yvonne M. Angell, Ph.D., Director of Peptide and Protein Chemistry, Ipsen Bioscience, Inc., USA

11:45 CASE STUDY Development of a Novel Peptide Immunotherapeutic for Prostate CancerWe are developing a second generation immunotherapeutic peptide vaccine for various cancers. The peptide was designed to activate critical components of the cellular immune system in an antigen-specific manner. In a Phase I prostate cancer study, both a robust immune response and correlates of improved overall and progression-free survival were obtained. The same peptide gave encouraging results from a Phase II breast cancer study.Eric von Hofe, Ph.D., President, Antigen Express Inc., USA

12:15 Luncheon in Poster and Exhibit Hall 1:25 Chairman’s Remarks

Eric von Hofe, Ph.D., President, Antigen Express Inc., USA

1:30 PeptiDream; PDPS and the Discovery & Development of Constrained Peptide TherapeuticsPeptiDream’s proprietary Peptide Discovery Platform System (PDPS) has proven highly effective at identifying constrained lead peptide candidates, in which such hits exhibit excellent therapeutic properties directly from selections themselves. In addition, advanced screening systems have been established to allow for the rapid assessment of those hits without the need for laborious chemical synthesis, significantly shortening the time from selections to the identification of hit constrained peptides that exhibit the desired therapeutic candidate profile. Patrick C. Reid, Chief Scientific Officer, PeptiDream Inc., Japan and USA

2:00 CASE STUDY / NEW DATA Targeting Key Signaling Pathways of Cancer Stem Cells with Intracellular Peptide TherapeuticsOne of the challenges in today’s treatment of cancer is tumor recurrence and spread caused by the existence of subpopulations within a tumor with distinct tumor-initiating powers, the cancer stem cells. Peptide therapeutics targeting intracellular protein-protein interactions are an attractive alternative to conventional therapies to inactivate signaling pathways in cancer stem cells. Nexigen’s NexiTides target such pathways and have powerful in vitro anti-cancer stemness activity and are effective in xenografted and orthotopic models of tumor growth and metastases.Jörg Vollmer, Ph.D., Chief Executive Officer, Nexigen GmbH, Germany

2:30 CASE STUDY / NEW DATA High Affinity Bicyclic Peptides: Application to Payloads in OncologyThe Bicycle technology is based on repertoires of peptides displayed on the surface of bacteriophages which can be modified with organochemical scaffolds to create a diverse array of constrained peptides. These repertoires have been extensively used for iterative selections to identify high affinity binding peptides for a wide array of targets, including receptors, interleukins and proteases. The bicycle peptides show antibody-like properties such as low to sub-nanomolar affinities and exquisite selectivity, but in a 100-fold smaller, chemically synthesized format. We showed that these small entities extravasate and penetrate tumors much more rapidly and efficiently than antibodies, thus delivering high concentrations of payloads in a very short time. These features allow efficient tumor killing while drastically minimizing systemic toxin exposure. Bicycle Therapeutics will present in vivo POC data from its pre-clinical programs demonstrating the power of its technology to deliver cytotoxic payloads to tumor cells.Christophe Bonny, Ph.D., Chief Scientific Officer, Bicycle Therapeutics, United Kingdom

Visitwww.IBCLifeSciences.com/AsiaTIDESforup-to-dateinformationonthisevent 3

3:00 Networking Reception in Poster and Exhibit Hall

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MainConference•ConcurrentTracks Tuesday, March 3, 2015 (continued)

4 ToRegister,Call:(+65)65082401•Fax:(+65)65082407•E-mail:[email protected]

Oligonucleotide-BasedTherapeuticsinPreclinical and Clinical Development (continued)

Peptide-BasedTherapeuticsinPreclinicalandClinical Development (continued)

Novel LNA and siRNA Developments 3:40 Chairman’s Remarks

Patrick Y. Lu, Ph.D., President & CEO, Sirnaomics, Inc., USA

Featured Presentation

3:45 NEW DATA LNA Therapeutics: A Technology PerspectiveThe field of LNA and RNA therapeutics is undergoing rapid development. New trends in the field influence future developments and further progress will be linked to a reformulation of older concepts. It will be shown that, in general, the way forward will benefit from a fresh view of the technology space. It will be illustrated how the recent progresses in the field has enabled renewed interest in the technology and in the deal space.Dr. Troels Koch, VP & CTO, Roche Innovation Center Copenhagen, Denmark

4:15 CASE STUDY / NEW DATA Advancing Novel siRNA Therapeutic Products in Both China and USASirnaomics has developed an enriched product pipeline including siRNA therapeutic STP705 (Cotsiranib®) for Hypertrophic Scar prevention and reduction, STP702 for “Resistent-Proof ” influenza therapeutics, and STP909 for HPV and cervical cancer treatment. The company is pushing two siRNA therapeutic programs into clinical study in China and USA with support of manufacturing capability, technical strength and financial resources. Patrick Y. Lu, Ph.D., President & CEO, Sirnaomics, Inc., USA

4:45 NEW DATA Locked Nucleic Acid: Enabling RNA Therapeutics RNA therapeutics have over the past decade developed from concept, clinic to market. However, the development has been hampered by a too simplistic approach to the underlying drug discovery principles resulting in unnecessary attrition rates during development. The lessons learned have resulted in new concepts to conduct drug discovery leading to increases in the productivity of RNA therapeutics. Locked Nucleic Acids are recognized as a preferred chemistry for RNA therapeutics and this will be presented in the context of lesson learned and LNA drug discovery case stories.Dr. Bo Rode Hansen, Vice President, Drug Discovery & Alliance, Roche Innovation Center Copenhagen, Denmark

5:15 NEW DATA DT01: A First-in-Class DNA Repair Inhibitor Against Advanced Cancers, From Concept to ClinicEnhanced DNA repair activity in tumors confers resistance to treatment. A first-in-class oligonucleotide therapeutics that mimics DNA double-strand breaks (named “Dbait”) has been developed. It acts by jamming DNA damage signaling that disorganizes DNA repair system and thereby inhibits DNA repair. This presentation will describe its mechanism of action and preclinical proofs of concept, animal toxicology and pharmacokinetics data, and preliminary data in the first-in-human trial of DT01 - 1st drug candidate of Dbait.Professor Jian-Sheng Sun, CEO, DNA Therapeutics SA, France

5:45 End of Day One

3:40 Chairman’s RemarksPatrick C. Reid, Chief Scientific Officer, PeptiDream Inc., Japan and USA

3:45 Kisspeptin and its Agonist Analogs: From Discovery to Clinical StudiesAbstract Not Available. Please visit www.IBCLife Sciences.com/AsiaTIDES for updates.Hisanori Matsui, Principal Scientist (Pharmacology), Extra Value Generation & General Medicine Drug Discovery Unit, Pharmaceutical Research Division, Takeda Pharmaceutical Company, Ltd., Japan

4:15 FRONTIER: A Phase 3, Pre-Hospital Peptide Therapeutic Trial for Acute Ischemic StrokeAbstract Not Available: Please check www.IBCLifeSciences.com/AsiaTIDES for updates.Dave Garman, Ph.D., Technology Officer, NoNo Inc., Canada

4:45 Late-Breaking PresentationIkuo Fujii, Ph.D., Professor Graduate School of Science, Osaka Prefecture University

5:15 NEW DATA Cryptides and Their Accumulative Signaling Mechanisms for Novel Therapeutic Targets Many fragmented peptides are produced during maturation and degradation processes of endogenous proteins but their physiological and/or pathological roles are not well elucidated yet. We discovered novel bioactive peptides derived from various mitochondrial proteins that efficiently activated neutrophils and named such functional peptides hidden in protein structures “cryptides.” Here I will discuss about such cryptides and their signaling mechanisms for possible therapeutic targets.Hidehito Mukai, Ph.D., Principal Investigator, Laboratory of Peptide Science and Associate Professor, Graduate School of Bio-Science, Nagahama Institute of Bio-Science and Technology, Japan

5:45 End of Day One

5:15 Networking Reception in Exhibit and Poster Hall Co-sponsored by:

Conference Language

TheconferencewillbeconductedinEnglishwithouttranslation.

About the Organizers

This event is brought to you by the organizers of the TIDES and EuroTIDES conferences. IBC Life Sciences, an Informa business, is your connection to the life sciences industry. To see all of the in-depth content IBC Life Sciences has to offer, visit www.IBCLifeSciences.com.

Making Hotel ReservationsSpecial room rates have been contracted with Hyatt Regency Osaka Hotel for IBC’s delegation. To take advantage of this special rate, please visit the conference website at www.IBCLifeSciences.com/AsiaTIDES and select the Hotel Info Link. More information on how to reserve your room is available there.

Travel/Visa InformationPLEASE NOTE: Visas are required for some nationalities to travel to Japan for this conference. Please contact your travel agent and/or the Japanese Consulate/Embassy in your country for exact details and visa application procedures as soon as possible. Visa processing times can vary.

Call for Posters Limited space is available for poster presentations at this event. If you have new results/data on topics relevant to this conference, we encourage you to submit a poster abstract for consideration. To present a poster, complete the conference registration form and submit poster title and one page poster abstract online at www.IBCLifeSciences.com/AsiaTIDES by January 30, 2015. See registration form for poster fees.

Page 5: AsiaTides -Peptides and Oligonucleotides 2015

Main Conference Wednesday, March 4, 2015

Visitwww.IBCLifeSciences.com/AsiaTIDESforup-to-dateinformationonthisevent 5

8:00 Networking Coffee and Tea

DDS (Drug Delivery Systems and Strategies for Peptides and Oligonucleotides)

8:55 Chairman’s RemarksBruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

9:00 NEW DATA Lipid Nanoparticles for Therapeutic Delivery of miRNA-Targeting OligonucleotidesmiRNA dysregulation plays a critical role in tumor development and resistance to therapy. miR mimics and antimiR oligos have potential therapeutic applications. A series of lipid nanoparticle formulations have been developed for therapeutic delivery of these oligonucleotide agents in leukemia and in lung cancer. We have shown in animal models that miR-29b mimics can inhibit xenograft tumor growth and extend the survival of leukemia engrafted mice. Lipid nanoparticles loaded with antimiR-21 have been shown to effectively modulate miR-21 targets in vitro and in vivo, and to inhibit tumor growth in a xenograft model. Lipid nanoparticle-based delivery of miR-modulating oligos has great promise for clinical translation for cancer therapy.Robert J. Lee, Ph.D., Professor of Pharmaceutics, Division of Pharmaceutics, OSU College of Pharmacy, The Ohio State University, USA

9:30 CASE STUDY Intranasal Drug Delivery: Drug Development ConsiderationsIntranasal route of administration is attractive for small molecules, peptides, oligonucleotides and even proteins. The nasal epithelium has a large surface and is highly vascularized which can result in rapid absorption and drug onset. The nasal route represents a non-invasive means of delivery and is amenable to peptide drugs as it avoids gastric degradation and hepatic first-pass metabolism. This presentation will go through drug product characterization and highlight aspects which are unique to nasal drugs. The challenges with nonclinical dose administration, determination of the maximum tolerated dose, and methods for pharmacokinetic evaluation will be discussed using case examples. Bruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

10:00 Networking Refreshment Break

10:40 Chairman’s Opening RemarksBruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

10:45 CASE STUDY / NEW DATA Block Copolymer-Based Nanosystems for siRNA and Oligonucleotide DeliveryThis presentation features supramolecular nanosystems assembled from charged block copolymers for siRNA and oligonucleotide delivery. Focus will be on the systemic delivery systems aiming to target intractable cancer, including pancreatic cancer. Tumor penetrability of nanosystems will be a critical point to be discussed particularly in this presentation.Kazunori Kataoka, Ph.D., Professor, Department of Bioengineering, School of Engineering, University of Tokyo

11:15 NEW DATA Probing Structural Requirement of Triantennary N-Acetylgalactosamine for Hepatic Targeting of Antisense Oligonucleotides A summary of our effort to identify simpler GalNAc clusters with specific emphasis on lowering molecular weight and ease of synthesis for Gapmer antisense therapeutics will be presented.Thazha Prakash, Ph.D., Senior Research Fellow, Isis Pharmaceuticals, USA

11:45 Late-Breaking Presentation

MainConference•ConcurrentTracks

Manufacturing and Analytical Strategies for Oligonucleotides

Sponsored by:

2:00 Rapid Synthesis of Oligonucleotides for Research and Preclinical StudiesAbstract not available; please check www.IBCLifeSciences.com/AsiaTIDES for updatesRowshon Alam, Group Leader, SSOU, Nitto Denko Avecia, Inc., USA and JapanTatsuya Konishi, Process Development, Group Leader, Nitto Denko Avecia, Inc., USA and Japan

2:30 First Manufacturing Results from the Next Generation OligoProcess SynthesizerDr. Huseyin Aygün, Ph.D. CSO, BioSpring GmbH, Germany

3:00 Networking Refreshment Break

Manufacturing and Analytical Strategies for Peptides

2:00 Large Scale Manufacturing of Synthetic PeptidesLatest development in large scale manufacturing of synthetic peptides will be presented. The talk will consider both chemical processes as well as engineering aspects of large scale SPPS and downstream manufacturing. A case study will be discussed.Daniel Samson, Ph.D., Director API Manufacturing, Bachem, Switzerland

2:30 Characterization of Degradation Impurities in API or Formulated drugs: Example of a beta-Aspartic Isomer Identification by Complementary Analytical MethodsADeep characterization of degradation impurities is a challenge in the analytical laboratory. In this real-life case study, we supported a customer for the characterization of a drug product. An unexpected degradation impurity was detected and identified, with the help of orthogonal HPLC methods, small scale purification, Edman sequencing, and mass spectrometry sequencing.Didier Monnaie, Analytical Development Group Leader, or David Cosquer, Mass Spectrometry Specialist, Analytical Development, Lonza, Belgium

3:00 Networking Refreshment Break

Featured Presentation: Manufacturing and Analytical Strategies for Peptides and Oligos

1:30 Purification of Synthetic Oligodeoxynucleotides and Peptides Using a Readily Scalable Catching by Polymerization ApproachSynthetic oligonucleotides and peptides have received increasing attention for drug discovery but their purification is very expensive. To lower the purification costs, we have developed an innovative catching by polymerization approach where a simple polymerizable group is attached to either the failure sequences or the full-length sequences but not to both and purification is achieved by simple washing and extraction process.Durga P. Pokharel, Ph.D., Post-Doctoral Research Fellow, Michigan Technological University, USA

12:15 Networking Luncheon in Poster & Exhibit Hall 1:25 Chairwoman’s Remarks Paula Lorence, VP Business Development & Project Management, Nitto Denko Avecia, USA

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MainConference•ConcurrentTracks Thursday, March 5, 2015

6 ToRegister,Call:(+65)65082401•Fax:(+65)65082407•E-mail:[email protected]

8:00 Networking Coffee and Tea

Regulatory Considerations for Peptides and Oligonucleotides

8:55 Chairman’s RemarksDavid T. Lin, Ph.D., Senior Consultant, Biologics Consulting Group, Inc., USA

9:00 US Regulatory Development and Review Perspectives for New and Generic Peptide DrugsThis session will provide a regulatory overview of the requirements for new and generic peptide drugs. The presentation will include a discussion of the review process in FDA for peptide products with respect to a new peptide as compared to a generic peptide product. Regulatory requirements and expectations between new and generic peptide products are similar so differences will be discussed. To understand these regulatory requirements case studies will be presented that outline development strategies for new dosage forms and delivery systems.David Lin, Ph.D., MBA, Senior Consultant, Biologics Consulting Group, Inc. (former acting Division Director, Office of New Drug Chemistry, CDER, U.S. FDA), USADuu-Gong Wu, Ph.D., Director, Regulatory Consulting/Senior Consultant, PPD, USA (former Deputy Division Director, Office of New Drug Chemistry, CDER, U.S. FDA, USA)

10:00 Update on the Regulatory Expectations for the Quality of Oligonucleotide ProductsThe complex and diverse nature of oligonucleotides currently in development is posing unprecedented challenges in meeting the regulatory requirements for drug quality. This presentation provides a historical overview and evolution of the US, European and other regulatory agencies’ review practices of oligonucleotide products. Also highlighted are emerging trends in the industry to meet current expectations for the characterization and quality control of oligonucleotide drug candidates from preclinical to late stage clinical development.G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA

10:30 Oligonucleotide Therapeutics One Step CloserOligonucleotide therapeutics continues to advance toward approval and yet there still remain some critical questions. The early challenges have been met and now to obtain more approvals it will be necessary to address new issues. Identifying these issues and their solutions is the key to future regulatory approvals.Art Levin, Ph.D., Executive Vice President, Research and Development, Avidity NanoMedicines, USA

11:00 AsiaTIDES Closes and Post-Conference Workshops Begin

Main Conference Wednesday, March 4, 2015 (continued)

Manufacturing and Analytical Strategies for Oligonucleotides

Manufacturing and Analytical Strategies for Peptides

3:30 Late-Breaking Presentation from AgilentAbstract not available; please check www.IBCLifeSciences.com/AsiaTIDES for updates

4:00 AJIPHASE®; Novel & Practical Solution Phase Oligonucleotides Synthesis for Large Scale ManufacturingAJINOMOTO’s original liquid-phase technology AJIPHASE® initially developed for peptide manufacturing has been successfully applied to oligonucleotide synthesis.This presentation will describe the novel and practical liquid-phase approach for synthesis or manufacturing of two types of oligonucleotides, DNA/RNA-type oligonucleotides and Morpholino oligonucleotides. Several examples of liquid-phase oligonucleotide synthesis had been reported in the literatures; however, most of them were fit only for short length oligos in terms of yield and quality. Here, we developed ‘one-pot’ elongation cycle and optimized several factors of the reaction condition to obtain highly pure oligonucleotide in a large scale.Satoshi Katayama and Daisuke Takahashi, Research Institute for Bioscience Products & Fine Chemicals, Ajinomoto Co. Inc., Japan

4:30 You Have Developed a Process – Now What? Key Elements & KPIs to Consider When Setting Up Successful Technology Transfers at Different Stages of the Product LifecyclePre-tech transfer activities are critical in ensuring success of the tech transfer process and clear, specific communication is essential before, during, and after the tech transfer process. You must have the appropriate skill sets and competencies in place with all of your upstream / downstream partners so that you have understood any constraints and agreed on the scope of the work and acceptable parameters months before the tech transfer actually begins. Jayant Aphale, Ph.D., MBA, Senior Vice President, Technical Operations, Sarepta Therapeutics, USA

5:00 Forced Degradation of Phosphorothioate OligonucleotidesForced degradation studies are an important component of drug development. Forced degradation studies may be used to determine the inherent stabilities of drug substances and drug products and to develop an understanding of degradation pathways and degradation products. The main degradation pathways, the structures of degradation products and the inherent stabilities of synthetic phosphorothioate oligonucleotides exposed to a variety of physical and chemical insults will be presented. Daniel Capaldi Ph.D., Vice President, Analytical and Process Development, Isis Pharmaceuticals, Inc., USA

5:30 Close of Day 2

3:30 Quantifying Sequence and Structural Features of Protein–RNA InteractionsWe quantified the contribution of both sequence- and structure-based features as indicators of RNA-binding propensity using a machine-learning approach. Several novel and modified features enhanced the accuracy of residue-level RNA-binding propensity beyond what has been reported previously. We constructed a web server called aaRNA that implements the proposed method and demonstrate its use in identifying putative RNA binding sites.Singling Li, Ph.D., Specially Appointed Researcher, Systems Immunology Laboratory, Immunology Frontier Research Center (IFReC), Osaka University, Japan

4:00 Methods for Peptide Thioester PreparationChemical ligation technique is widely used for protein synthesis. The ligation methods, such as thioester method and native chemical ligation, have been developed based on the use of peptide thioesters as building blocks. We have developed methods for preparation of the peptide thioesters based on an intramolecular N to S acyl shift reaction at a thiol containing residue of peptides.Turo Kawakami, Ph.D., Associate Professor, Institute for Protein Research, Osaka University, Japan

4:30 Late-Breaking Presentation 5:00 Molecular Hiving Technology as a Strategy for the

Manufacturing of PeptidesMolecular Hiving Technology™ is innovative, hydrophobic tagged Liquid Phase Peptide Synthesis (LPPS) that integrates the key advantages of both LPPS and Solid Phase Peptide Synthesis (SPPS). Sekisui Medical has been developing this technology since 2010. Recent progress has allowed Sekisui to carry out a synthesis with a substantial increase in crude purity and reduction in consumables. This has resulted in faster lead times, a more efficient downstream process and considerable reduction in costs. Examples of this will be given in the presentation. This presentation will also include case studies (all unpublished data) for the synthesis of synthetically challenging oligopeptides (e.g. C-terminal modified cyclized). All candidates were synthesized obtaining a high yield and crude purity, followed by an excellent final purity after the purification process.Barry O’Connor, Research Scientist, Sekisui Medical Co Ltd., Japan

5:30 Close of Day 2

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Gold SponsorDriven by Excellence, Guided by Experience. When you partner with Avecia, (www.Avecia.com) you will be working with the leading oligonucleotide CMO. Avecia offers the most extensive process validation experience in the oligo industry. As a member of the Nitto Denko Corporation (www.nitto.com), Avecia is committed to

the future of the oligonucleotide market. We offer our customers oligo manufacturing capacity in two separate facilities (MA and OH). Our Cincinnati facility also provides expanded pre-clinical oligo services (OliGROW), as well as small molecule expertise and production. Our aim is to leverage our wealth of experience, to ensure we exceed our customer’s expectations. Avecia has the stability and vision required for the long-term success of your oligo program.

Silver SponsorsAgilent’s Nucleic Acid Solutions Division offers industry leading experience to efficiently advance your lead oligo candidates from

clinic to market with a common goal of patient health and safety. With Agilent, you always have peace of mind by partnering with a company that has the financial resources, stability and vision required for long-term success. Contact us at: [email protected] and find out why the world’s most revolutionary biotech companies and their Big Pharma partners are choosing Agilent Technologies to develop and commercialize their oligo APIs.

Established in 1981, ChemGenes, an ISO9001 certified company, has consistently provided the highest quality Phosphoramidites and Solid Supports in the market and

continues to lead the industry in oligonucleotide reagent manufacturing. Our Massachusetts facility is setup for Bulk Therapeutic Grade phosphoramidite production for GMP grade oligonucleotide manufacturing. Additionally, ChemGenes carries the widest variety of modified phosphoramidites and supports currently used in oligonucleotide synthesis including Microarray Technology, Oligonucleotide Therapeutics, Oligonucleotide Based Probes and other areas of Nucleic Acid research. ChemGenes remains devoted to providing you with invaluable customer service and comprehensive technical support.

Bronze SponsorsBachem specializes in the manufacture of peptides and complex small molecules as APIs as well as innovative biochemicals for

research purposes. We offer technical consultancy, comprehensive regulatory affairs support, and dedicated project management from drug development through commercial scale cGMP production. Headquartered in Bubendorf, Switzerland with affiliates in Europe and the U.S.

GeneDesign offers contract manufacturing of various therapeutics and diagnostics oligonucleotide including

PEGylation, peptide conjugation and ligand conjugation from screening use to hundreds gram GMP manufacturing with sterilized condition. With our widely experience and challenge to the new technology area, we can contribute to your successful oligo therapeutics development. We have launched new miRNA Inhibitor called “S-TuD” and miRNA mimic as a research tools and started novel liquid phase oligonucleotide synthesis.

SponsorsandExhibitors

Visitwww.IBCLifeSciences.com/AsiaTIDESforup-to-dateinformationonthisevent 7

For more than 16 years, BioSpring has been providing manufacturing and analytical services for the oligonucleotide market. We manufacture an entire range of unmodified and

modified oligonucleotides from mg to multi-kg quantities for the use in therapeutic (GMP) and diagnostic applications (ISO 13485 certified), molecular biology, and research and development. In addition, we have a dedicated analytical team and employ state of the art techniques for fully testing and characterizing oligonucleotides and oligonucleotide containing products. For additional information, contact us via email: [email protected].

Session Sponsors

Spotlight Presentation Sponsor

Exhibitors (As of October 16, 2014)

NetworkingReceptionCo-Sponsor

Literature Sponsor Refreshment Break Sponsor

Would you like to reach the AsiaTIDES audience?• Meet face-to-face with top-notch researchers and executives from

pharmaceutical and biotechnology companies

• Showcase your latest technology to our targeted audience of key decision-makers

• Build invaluable relationships and form new partnerships that give you a competitive advantage in the US, Europe and Asia-Pacific

• Gain international exposure through our specialized marketing campaign

• Network with international players in the industry

Forsponsorshipandexhibitingopportunities,contact: Sherry Johnson: [email protected]•Tel:+1(508)614-1451

Post-ConferenceWorkshops Thursday, March 5, 2015

Workshop #1: Impurities in Oligonucleotides:Sources, Analysis and Control

Workshop Leaders: Thomas Rupp, Thomas Rupp Consulting, Germany G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA

With the market approval of three oligonucleotide drugs and the advancement of a myriad of oligonucleotide programs to late stage development, there is an increased interest on the topic of impurities in synthetic oligonucleotides. Due to their inherent complexity, synthetic polymers such as peptides and oligonucleotides are specifically excluded from the scope of the ICH Q3A impurities guideline. This workshop will present current regulatory strategies for the control of impurities through discussions of the sources, methods of analysis and control. 11:00 Welcome and Opening Remarks

Thomas Rupp and G. Susan Srivatsa 11:15 Practical Considerations for the Implementation of Quality

Criteria for Amidite Starting Materials (TBD) 12:00 Networking Lunch 1:15 Impurities Formed During Synthesis and

Downstream ProcessingThomas Rupp, Thomas Rupp Consulting, Germany

2:00 Application of Risk Assessment in Development of Testing Strategies for Non-Oligonucleotide ImpuritiesEmma Wright, Nitto Denko, USA

2:45 CMC Considerations for the Control of Impurities in OligonucleotidesG. Susan Srivatsa, Ph.D., ElixinPharma, USA

3:30 Safety Considerations for the Control of Impurities in OligonucleotidesMike Templin, SNBL, USA

4:15 General Discussion and Close of Workshop

Workshop #2:Current Practices in Peptide Therapeutics

This course will cover the main topics of manufacturing, formulation and regulatory strategies for peptide therapeutics.

11:00 Evolution of Peptide API Specifications from IND to NDARobert Hagopian, Director of Business Development, PolyPeptide Group, USA

11:30 Scale-Up Manufacturing Case Study: Considerations of Transition from Solid-Phase to Solution-Phase SynthesisBruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

12:00 Networking Lunch 1:15 Development of a Stable Lyophilized

Peptide FormulationDave Garman, Ph.D., Technology Officer, NoNo Inc., Canada

1:45 Regulatory Trends and Strategy for Peptide Products Duu-Gong Wu, Ph.D., Director of Regulatory Consulting/Senior Consultant, Global Regulatory Consulting, PPD, USA

2:00 General Discussion and Close of Workshop

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Venue & Hotel InformationHyatt Regency Osaka

1-13-11 Nanko-Kita, Suminoe-KuOsaka, Japan, 559-0034

Tel: +81 6 6612 1234 • Fax: +81 6 6614 7800 Email: [email protected]

PaymentPayment must be received 10 business days prior to the event. To take advantage of discounts with an expiry cut-off date, registration and payment must be received by the cut-off date.• Payments by USD bank draft or cheque should be made in

favour of “IBC Asia (S) Pte Ltd” and mailed to:IBC Asia (S) Pte Ltd c/o Informa Regional Business Services111 Somerset Road, TripleOne Somerset #10-06, Singapore 238164Attn: The Accounts Receivable Team

• Payment by telegraphic transfer in USD must be made to:IBC Asia (S) Pte LtdA/C No.: 260-457866-178 (USD)The Hongkong and Shanghai Banking Corporation Limited, 21Collyer Quay, HSBC Building, Singapore 049320Bank Swift Code: HSBCSGSGBank Code: 7232

• Payment by Credit Card (AMEX, VISA or MASTERCARD)Please provide your Card Number, Name of Cardholder, Expiry Date and Signature and send it by fax to +65 6508 2407.

Important NotePlease quote the name of the delegate, event title and invoice number on the advice when remitting payment. Bank charges are to be deducted from participating organisations’ own accounts. Please fax your payment details (copy of remittance advice, cheque or draft to +65 6508 2407). Attendance will only be permitted upon receipt of full payment. Participants wishing to register at the door are responsible to ensure all details are as published. IBC assumes no further liability or obligation, beyond the refund of the paid registration fee, in the event of postponement or cancellation by IBC.

Registration Substitutions/CancellationsShould you be unable to attend, a substitute delegate is welcome at no extra charge. Cancellations must be received in writing at least 10 business days before the start of the event, to receive a refund less 10% processing fee per registration. The company regrets that no refund will be made available for cancellation notifications received less than 10 business days before the event. IBC reserves the right to cancel or alter the content and timing of the program for reasons beyond its control and will NOT be held accountable for any costs incurred by the participants. Speakers are subject to change without prior notification.

Data ProtectionThe personal information entered during your registration/order, or provided by you, will be held on a database and may be shared with companies in the Informa Group in the UK and internationally. Occasionally, your details may be obtained from or shared with external companies who wish to communicate with you offers related to your business activities. If you do not wish your details to be used for this purpose, please contact our Database Department at Email: [email protected], Tel: +65 6508 2400 or Fax: +65 6508 2408.

Please tick: o I enclose my Cheque/Draft payable to IBC Asia (S) Pte Ltd o I am paying by bank transfer (copy attached) o Payment by Credit Card: o Amex o Visa o Mastercard

Credit Card Payments: The best way to pay by credit card is through our secure on-line registration process, simply log on to the website at www.IBCLIfeSciences.com/AsiaTIDES and click “Register Now”. If you would prefer to pay over the phone please complete the contact name and details and someone from our Customer Services Team will call within 24 hours to take payment. As we treat your credit card information in the strictest confidence, please do not send payment details by email.

Credit Card Contact: ____________________________________ Department: _______________________________________

Direct phone number: __________________________________ Email:_______________________________________________

Name: Dr/Mr/Ms __________________________________________________ E-Mail _____________________________________________________________

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Name & Title of Approving Manager _______________________________ Name & Title of Training Manager _________________________________

Industry Rates On or before Dec. 19, 2014

On or before Jan. 23, 2015

On or before Feb. 13, 2015

Standard Rate After Feb. 13, 2015

o Main Conference + Workshop* USD1999 USD2099 USD2199 USD2299

o Main Conference + Workshop* Group Rate (3+ from same company) USD1799 USD1889 USD1979 USD2069

o Main Conference Only USD1699 USD1799 USD1899 USD1999

o Main Conference Only Group Rate (3+ from same company) USD1529 USD1619 USD1709 USD1799

*Select one Workshop: o Workshop #1: Impurities in Oligonucleotides: Sources, Analysis and Control o Workshop #2: Current Practices in Peptide Therapeutics

Academia/Government Discounts – 40% Savings: Academic and government employees are eligible for over 40% savings compared to the industry fees. Rate extended to full-time employees of government, universities and university-affiliated hospitals who have NO affiliation to a for profit entity. See website for details.

Present a Poster o USD125 - Commercial o FREE - Academic

Please tell us your primary area of interest: o Oligonucleotides o Peptides

Multiple Bookings Discount pricing is applicable to groups of 3 or more delegates from the same organisation registering for the same event, at the same time. Fee stated is the discounted price PER DELEGATE. Only one discount applies; either the early bird rate OR special rate OR group rate. All fees stated include luncheons, refreshments and complete set of documentation. It does not include the cost of accommodation and travel. Registration fees are subject to the prevailing government tax.

If undelivered, please return to:

111 Somerset Road TripleOne Somerset #10-06, Singapore 238164 Tel: +65 6508 2400 Fax: + 65 6508 2408

RESERVE YOUR PLACE TODAY!

This label contains your priority booking code. To expedite registration, please do not remove label. If you have already received a copy of this brochure, we apologise. For reasons of confidentiality, your full particulars were not available to IBC

Asia (S) Pte Ltd for deduplication prior to mail drop.

Save Up To USD300 with the Early Bird Rates!

5 Easy Ways To Register Mail the attached registration form with your cheque to IBC Asia (S) Pte Ltd c/o Informa Regional Business Services 111 Somerset Road, TripleOne Somerset #10-06, Singapore 238164

PHONE: +65 6508 2401

FAX: + 65 6508 2407

E- MAIL: [email protected]

WEB: www.IBCLifeSciences.com/AsiaTIDES

See grid below for group discounts for companies registering

3 or more attendees

AsiaTIDES – Oligonucleotide and Peptide® Research, Technology and Product Development

P46261

REGISTERNOW!FAXBACKTO(65)[email protected]