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AsiaTIDES: Oligonucleotide and Peptide Research, Technology and Product Development

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

February 25 - February 27,--> 2014 · Tokyo, Japan

Agenda

Agenda

Pre-Conference Tutorials - Tuesday, February 25, 2014

8:30
Registration and Coffee/Tea

Tutorial #1

Manufacturing of Oligonucleotide Drugs

Tutorial Leader: Thomas Rupp, Owner, Thomas Rupp Consulting, Germany

The workshop will lead off with a review about US, Canada and European regulatory expectations for the manufacturing of therapeutic oligonucleotides. Additional important topics will cover the importance of incoming raw material controls as well as considerations about upscaling of modified oligonucleotides from bench scale to process scale. As nowadays more and more special amidites are included into therapeutic oligonucleotides this topic will be addressed as well before the workshop concludes with a presentation about oligonucleotide analytics. Each talk will be 30 minutes with an additional 15 minutes for discussion.

9:00
Regulatory Expectations for Oligonucleotides
Kathy Ackley, Ph.D., R.A.C., Senior Director, Chemical Operations, Nitto Avecia, USA

9:45
Analysis and Controls of Raw Materials
Hüseyin Aygün, Ph.D., Chief Scientific Officer, Biospring, Germany

10:30
Networking Refreshment Break

11:00
Special Considerations for Novel Amidites
Yogesh Sanghvi, Ph.D., President, Rasayan Inc., USA

11:45
Large Scale Manufacturing of Oligonucleotides
Thomas Rupp, Owner, Thomas Rupp Consulting, Germany

12:30
Networking Luncheon

1:45
Podium & Audience Discussion

2:30
Close of Workshop

Tutorial #2

The Challenge of Oligonucleotide In Vivo Delivery and Currently Proposed Drug Delivery Systems (DDS) and Approaches for It

Tutorial Organizers:
Laura Sepp-Lorenzino, Ph.D., Executive Director, RNA Therapeutics, Merck, USA Dmitry Samarsky, Ph.D., Executive Vice President, Technology Development, RiboBio, China

8:30
Registration

9:00
Introduction
Dmitry Samarsky, Ph.D., Executive Vice President, Technology Development, RiboBio, China

9:10
Oligonucleotide Chemistry: Bio-Distribution, Targeting and Uptake Challenges
Sterghios Moschos, Ph.D., Associate Professor, Industrial Biotechnology, University of Westminster, UK

9:50
Conjugation Strategies to Deliver RNAi Molecules
Dong-ki Lee, Ph.D., Professor, Sungkyunkwan University, Korea

10:30
Break

11:00
Use of Nanoparticles for Delivery of RNAi Therapeutic molecules
Patrick Lu, Ph.D., President and CEO, Sirnaomics, USA/China

11:40
Multiplicity of Considerations in Selecting Delivery Approaches for Therapeutic Oligonucleotides
Julja Burchard, Ph.D., Principal Scientist, Merck, USA

12:20
Conclusions and Discussion
Dmitry Samarsky, Ph.D., Executive Vice President, Technology Development, RiboBio, China

12:30
Lunch

Tutorial #3

Introduction to Peptide Manufacturing: General Specifications of a Peptide API

Tutorial Leader: Robert Hagopian, Director Business Development, PolyPeptide Group, USA

8:30
Introduction to Peptide Manufacturing: General Specifications of a Peptide API
Robert Hagopian, Director Business Development, PolyPeptide Group, USA

9:45
The Basics of a CMC Section of an IND
Ve T. Vuong, Director, Regulatory Affairs, PolyPeptide Group, USA

10:30
Networking Refreshment Break

11:00
Changes to the CMC from Pre-Clinical to Product Approval
Ve T. Vuong, Director, Regulatory Affairs, PolyPeptide Group, USA

11:30
NA-1: FDA Guidance from Preclinical through Pivotal Studies
Dave Garman, Ph.D., Technology Officer, NoNO Inc., Canada

12:00
Open Question and Answers

12:30
Networking Luncheon

Main Conference · Plenary Keynote Presentations
Tuesday, February 25, 2014

12:00
Registration and Coffee/Tea

1:25
Chairman's Remarks
James D. Thompson, Ph.D., Head of Process Sciences, Moderna Therapeutics, Inc., USA

1:30
Andrew A. Young, M.D., Ph.D. Attributes Necessary for a Commercially Successful Metabolic Peptide
Efficacy and safety do not guarantee commercial success. There are patient/user-related, prescriber-related, and payer-related attributes of the approved product that can promote or defeat commercial success. Patient-related convenience factors, such as simplicity of use, absence of pain on injection, infrequency of injection, and absence of nausea, affect uptake, retention and compliance, and thereby efficacy. This is especially the case with chronic conditions such as type 2 diabetes and obesity. Prescribers are encouraged by efficacy, wide therapeutic margins and simple dosing schedules. Payers are encouraged by reductions in the cost of care that don't come at the expense of increased treatment costs. And because burdens of care differ, reimburseability will also differ for different indications. While a limited subset of peptides has thus-far proven safe and effective for treating diabetes and obesity, it is a still smaller subset that may also score well on factors related to commercial success. These factors will be discussed.
Andrew A. Young, MD, Ph.D., Vice President and Head of Enteroendocrine Biology, GlaxoSmithKline, USA

2:00
Hiroaki Suga, Ph.D. Natural Product-Inspired Peptide Discovery
A next-generation technology that enables enrichment of bioactive pseudo-natural products and amplification is discussed. The technology is based on an in-vitro display in the combination of FIT (Flexible In-vitro Translation) system with post-translational modifying enzymes.
Hiroaki Suga, Ph.D., Professor, Chemical and Biotechnology Lab, University of Tokyo, Japan

2:30
Bruce Morimoto, Ph.D. Trials and Tribulations for an Intranasal Peptide: Davunetide
Davunetide is an eight amino acid, neuropeptide. In numerous preclinical models of efficacy, davunetide showed both neuro- and cognitive-protection. A series of Phase 2a, proof-of-principle studies were conducted and recently a large, Phase 2/3 clinical study in progressive supranuclear palsy failed to show clinical benefit. This talk will be a case study on "lessons learned" for an intranasal delivered peptides, highlighting the challenges and providing a retrospective analysis of what could have been done to mitigate the risk of failure.
Bruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

3:00
Networking Refreshment Break

3:30
Dave Garman, Ph.D. From Molecular Mechanisms to Multi-Center Clinical Trials: Translation of a Peptide Inhibitor of PSD95 for the Treatment of Acute Ischemic Stroke
Fifteen million people suffer a stroke each year, two thirds of which die or are left permanently disabled. Fewer than 5% of victims receive drugs to reduce these deficits. NA-1 is a novel peptide agent being developed to reduce disability in patients with acute stroke that has completed Phase 1 and 2 clinical trials in the United States and Canada.
Dave Garman, Ph.D., Technology Officer, NoNO Inc., Canada

4:00
Henrik Ørum, Ph.D. Antisense: Within Sight of the Finish Line
Translating the seductive simplicity of antisense therapy into clinical reality has proven much more difficult than initially thought. In fact, more than 30 years after first being proposed as a novel approach to human medicine, antisense therapy has yet to realize its broad impact on human health. Thus, major technological breakthroughs in the chemistries from which antisense drugs are made, combined with novel antisense drug formats, have made significant advances in the last decade and brought antisense therapy within sight of the finish line. The presentation provides an overview of the antisense field, its past and present, and a view of its future.
Henrik Ørum, Chief Scientific Officer, Santaris Pharma A/S, Denmark

Late-Breaking Presentation

4:30
Ian MacLachlan, Ph.D. Recent Advances in the Lipid Nanoparticle-Mediated Delivery of Messenger RNA
Seven products based on Tekmira's lipid nanoparticle (LNP) delivery platform have entered clinical trials providing increasingly robust proof of clinical activity in multiple disease areas using small interfering RNA as the active pharmaceutical ingredient. We have recently applied the LNP technology to the efficient encapsulation and delivery of chemically modified mRNA in preclinical models demonstrating robust expression in numerous healthy tissues and tumors following intravenous administration. These encouraging results, orders of magnitude greater than that achieved using previously published, commercially available delivery agents, suggest that there may be numerous opportunities for the development of mRNA therapeutics using LNP delivery.
Ian MacLachlan, Ph.D., Executive Vice President and Chief Technology Officer, Tekmira Pharmaceuticals Corp., Canada

5:00
Close of AsiaTIDES Day One Sessions

6:30
Networking Dinner
Join fellow attendees in a fantastic networking and dining opportunity. Space is limited and additional fee applies. Please indicate when you register if you plan to join the dinner.

Main Conference · Concurrent Tracks
Wednesday, February 26, 2014

8:30
Registration and Coffee/Tea

Updates on Oligonucleotide-Based Therapeutics in Preclinical and Clinical Development

8:55
Chairman's Remarks
Dmitry Samarsky, Ph.D., Executive Vice President, Technology Development, RiboBio, China

9:00
New Data
Update on Isis's Cardiovascular Franchise Progress
Isis development leadership will present a clinical trial update on their cardiovascular franchise including updates on ISIS-APOCIIIrx, ISIS-APOArx, ISIS-FXIrx and ISIS-CRPrx. The cardiovascular franchise covers lipid, inflammation and thrombosis targeting of high risk targets that are not amenable to small molecule or antibody approaches. An overall strategic approach to antisense drug development will be included.
Scott Henry, Vice President, Preclinical Development, Isis Pharmaceuticals, Inc., USA

9:30
Intervening in Complex Disease Biology with microRNA-Targeting Therapeutics
microRNAs modulate the expression of multiple mRNAs in specific biological pathways, thereby regulating complex gene expression networks. Over the last decade, multiple studies have shown that microRNAs appear to have a magnified role in regulation of cellular behavior under conditions of physiologic and pathophysiologic stress. This makes them attractive targets for potential intervention in pathological settings. Our latest observations on the pharmacology of microRNA-targeting drug candidates and their activities in animal models of disease will be presented.
William S. Marshall, Ph.D., President and Chief Executive Officer, miRagen Therapeutics Inc., USA

10:00
Case Study
New Data
Clinical Development of RXI-109 to Prevent Dermal Scarring
RXI-109 targets connective tissue growth factor (CTGF), a key regulator of fibrosis. In Phase 1 clinical trials, RXI-109 was safe and well tolerated and reduced CTGF mRNA and protein locally following intradermal administration. In Phase 2, RXI-109 is being evaluated to prevent recurrent scarring in patients undergoing scar revision surgery. In preclinical work, reduction of retinal scarring by RXI-109 is also being evaluated.
Pamela A. Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals, USA

10:30
Grand Opening of the Poster and Exhibit Hall with Refreshments Sponsored by

Business Considerations and Intellectual Property

11:15
The Business of RNA(i) Therapeutics in 2014
The year 2013 marks the time when RNA Therapeutics became widely recognized as the next major drug development platform behind small molecules and recombinant proteins/monoclonal antibodies. Drug approvals and demonstrations of target engagements contributed to the change in perception. This presentation discusses the valuation drivers in the current market and related strategies to increase valuations.
Dirk Haussecker, Ph.D., Consultant and Blogger, The RNAi Therapeutics Blog, Germany

11:45
A Current and Historical look at the Nucleic Acid Therapeutics Market
For the past 15 years, the field of nucleic acid therapeutics has seen significant investment with less than desirable returns including clinical failures and two compounds approved but yielding modest sales. In 2013, the nucleic acid therapeutic landscape began to change with the recent approval of Kynamro™ and a healthy pipeline of Phase lll candidates approaching commercialization. This presentation will include a historical look at the nucleic acid therapeutic market and explore current macro trends in investment and indication selection with particular focus on rare & orphan diseases vs. traditional large market blockbuster opportunities.
Blake Unterreiner, Business Development Manager, Agilent NASD, USA

12:15
Luncheon in Poster and Exhibit Hall

Featured Presentation

1:30
Economic Continuous Reversed Phase Mode Chromatography
As the biopharmaceutical industry evolves, the need for advanced biomanufacturing becomes critical, especially for downstream solutions where increased productivity and improved economics without sacrificing process robustness should be offered. In this presentation, we explore the advantages of continuous chromatography through reversed phase chromatography. In major tested applications, experimental data confirm significant increase in productivity as well as reduced buffer consumption and enhanced resin utilization without impacting critical product quality attributes. In combination, these features enable continuous chromatography to be easily adapted to standard purification templates, but also potentially make disposable chromatography a reality.
Romas Skudas, Ph.D., Lab Leader, Process Purification Downstream Technologies, Merck Millipore, Germany

Updates on Oligonucleotide-Based Therapeutics in Clinical Development

2:00
Chairman's Remarks
Blake Unterreiner, Business Development Manager, Agilent NASD, USA

2:15
Clinical Data of anti-Chemokine Mirror-Image Aptamers
This talk discusses the development of mirror-image aptamers (so-called Spiegelmers) against different chemokine targets, i.e. MCP-1 (monocyte chemoattractant protein-1) and SDF-1 (stromal cell-derived factor-1). Both drugs drugs, NOX-E36 and NOX-A12, respectively, were profiled in preclinical studies as well as early clinical studies. Currently, Phase IIa studies are conducted with NOX-E36 for diabetic nephropathy and NOX-A12 for hematological tumors.
Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany

2:15
Case Study
Peptide-LNA Oligonucleotide Conjugates
This talk discusses internal incorporation of peptides into LNA/DNA strands using highly efficient "click" chemistry and novel 2′-alkyne-2′-amino-LNA scaffold, high DNA/RNA target binding affinity and selectivity of the resulting peptide-oligonucleotide conjugates, and their improved stability in human serum compared to natural nucleic acids and LNA/DNA references.
I. Kira Astakhova, Ph.D., Associate Professor, Nucleic Acid Center, Department of Physics, Chemistry & Pharmacy, University of Southern Denmark, Denmark

2:45
SAMiRNA, a Novel RNAi Prodrug Technology
This presentation highlights the therapeutic potential of SAMiRNA by virtue of its low toxicity and superb in vivo serum stability as well as its target gene silencing efficiency in a mouse tumor model. Topics of discussion include SAMiRNA's flexibility to incorporate siRNA sequences against any disease target, as well as enhancement of its therapeutic potential as a delivery platform through the use of cell-type specific targeting ligands. Using this novel RNAi prodrug technology, Bioneer is currently advancing clinical development of pipeline programs in previously non-druggable diseases, including cancer and COPD, through partnerships with major global pharmaceutical companies.
Jeiwook Chae, Ph.D., Head of Therapeutic RNA Development, BIONEER Corporation, Korea

3:15
Networking Refreshment Break in Poster and Exhibit Hall Sponsored by

4:00
New Data
Synergistic Antiviral Activity Covering Broad Influenza Strains Using Combinations of siRNAs Targeting Conserved Viral Sequences
By combining the selected siRNA pair against the conserved regions of M2 and PA gene segments, it was found that not only the pair covers over 97% of influenza strains in the database, but also exhibits synergistic anti-influenza activities against H1N1, H3N2, H5N2 and H7N9 strains in MDCK cell culture. Through an H1N1-challenged mouse model, this dual-targeted siRNA pair at a 10mg/kg dosage is highly active equivalent to the effect of Tamiflu at 25mg/kg dosage. The evidence led to developing a novel prophylactic and therapeutic mean combining siRNAs targeting the conserved regions of influenza gene segments, through an international collaborative effort with Scientists from US, China and Russia.
Patrick Y. Lu, Ph.D., President and CEO, Sirnaomics, Inc., USA

4:30
Clinical Development of DIMS0150, An Immunomodulatory Oligonucleotide
InDex Pharmaceuticals´ lead compound DIMS0150 is being developed for ulcerative colitis (UC) in patients with treatment refractory disease. DIMS0150 has proven well tolerated and effective after local, single administration to patients. A current phase III trial will be completed during 2014. The preclinical/clinical development history will be presented.
Ann-Kristin Spiik, Project Manager CMC and Pharmaceutical Development, PM Clinical Supplies at InDex Pharmaceuticals AB, Sweden

5:00
New Data
2′-OMe-Phosphorodithioate Nucleic Acid for Improving RNA Therapeutics
Results from in vitro and in vivo studies will be reported to document that 2'-OMe-phosphorodithioate (MS2) monomers are remarkably useful as novel constituents of siRNA duplexes, anti-miRNAs, and aptamers. The introduction of MS2 monomer substitutions have improved the potency of unmodified siRNA duplexes 6-fold, increased the binding affinity of unmodified therapeutic aptamers 1000-fold (KD from nM to pM), and significantly increased the ability of anti-miRNAs to inhibit miRNAs.
Xianbin Yang, Ph.D., Director of R&D, AM Biotechnologies, USA

Updates on Peptide-Based Therapeutics in Preclinical and Clinical Development

8:55
Chairman's Remarks
Robert Hagopian, Director Business Development, PolyPeptide Laboratories, USA

Featured Presentation

9:00
Case Study
New Data
Clinical Development for Therapeutic Cancer Vaccine Using Cocktail Peptides Targeting Novel Tumor Specific and Tumor-Angiogenesis Targets, Learn from Phase III
A multicenter, placebo control, double blinded, Phase III, pivotal clinical trial against pancreatic cancer using a peptide cocktail is demonstrated, with 300 pancreatic cancer patients enrolled in this clinical trial. Target molecules induced cytotoxic lymphocytes are tumor specific molecule and tumor angiogenesis factors. In this session, the concept of this clinical trial and updated data will be presented.
Takuya Tsunoda, MD, Ph.D., President & CEO, OncoTherapy Science Inc., Japan

9:30
Case Study
New Data
Effect of Dose Regimen and Combination Therapy on Efficacy of the Survivin Vaccine DPX-Survivac
A study in advanced ovarian cancer patients demonstrated that combining DPX-Survivac with metronomic cyclophosphamide enhanced immune responses. A dose response was also observed, indicating that larger priming doses may be required for achieving best immunogenicity. The influence of dose and regimen on the magnitude and quality of T cells generated by the vaccine will be discussed.
Marc Mansour, Ph.D., Chief Science Officer, Immunovaccine Inc., Canada

10:00
New Data
Designed Nanocage Displaying Ligand-Specific Peptide Bunches for High Affinity and Activity
Specific peptides can be genetically fused onto the surface of cage proteins to promote the association of nanoparticles with a particular cell type or tissue. Upon symmetrical assembly of the cage peptides are clustered on the surface of the cage protein in bunches, offering the potential of synergistic increasing the avidity of the peptide ligands, thereby enhancing their blocking ability for therapeutic purposes.
In-San Kim, Department of Biochemistry and Cell Biology, Cell and Matrix Research Institute, School of Medicine, Kyungpook National University, Korea

10:30
Grand Opening of the Poster and Exhibit Hall with Refreshments Sponsored by

11:15
Case Study
New Data
Accessing the "Undrugable" Target Space for Drug Discovery - Cell-Permeable Peptides Targeting the Wnt Pathway
So far the pharmacologic access to "un-druggable" target proteins has been very limited. Nexigen identified cell-permeable peptides capable of modulating a non-enzymatic target protein in the Wnt-pathway. These peptides show strong, target specific in vitro activity and a significant anti-tumoral activity in various animal tumor models. These results indicate that the un-druggable target space can be used successfully for drug discovery using cell permeable peptides.
Joerg Vollmer, Ph.D., Chief Executive Officer, Nexigen GmbH, Germany

11:45
Case Study
New Data
Bi-Cyclic Peptides to Target Protein-Protein Interactions
The Bicycle technology is based on repertoires of peptides displayed on the surface of bacteriophages which can be modified with an organochemical scaffold 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. Results will be presented that exemplify the potential of the technology and its application to animal models of diseases.
Christophe Bonny, Chief Scientific Officer, Bicycle Therapeutics, United Kingdom

12:15
Luncheon in Exhibit and Poster Hall

Updates on Peptide-Based Therapeutics and Vaccines in Preclinical and Clinical Development

1:30
Chairman's Remarks
Robert Hagopian, Director Business Development, PolyPeptide Laboratories, USA

1:45
Case Study
New Data
From Discovery to the Clinic: Successful Applications of Protein Epitope Mimetics
This presentation describes successful case studies of applying the PEM approach from discovery to the clinic. POL7080 is a novel anti-pseudomonal antibiotic with a new mode of action (MOA). Target elucidation and MOA studies revealed that POL7080 inhibits outer membrane (OM) biosynthesis by blocking lipopoylsaccharide transport from the periplasm to the OM [2]. POL7080 has successfully completed Phase I and is now rapidly advanced to the next stage of development. POL6326, a novel potent and selective CXCR4 antagonist, is currently in Phase II clinical trials for stem cell transplantation.
Daniel Obrecht, Ph.D., Chief Scientific Officer, Drug Discovery, Polyphor Ltd., Switzerland

2:15
Case Study
New Data
Immune Function: How Much Should We Be Modifying This Complex and Sensitive System with Peptide-Based Therapeutics?
This presentation discusses what we do and don't know about IG E factors associated with allergic diseases. The talk builds on this knowledge base by incorporating current advances in T cell epitopes which do not actuate non-specific immune-cell function. All of this provides a primer for the main focus of the talk which is taking how much we know about what we've covered and using it to our advantage to promote these advanced therapies in light of our overall target - the corpus variare (the changing body). The presentation explores the hysteresis mechanisms of the body, and how far we can extend them before it becomes an out-of-control process for the body's stabilizing mechanisms.
Ben Locwin, Ph.D., Head of T & D, Sr. QA, Pharma Quality Assurance, Lonza Biopharmaceuticals, USA

2:45
New Data
Transforming Stapled Peptides into High-Impact Medicines: Full p53 Re-activator for Cancer Treatment and Long-Acting GHRH Agonist for Metabolic Disorders
Aileron's Stapled Peptide technology utilizes optimized cross-linking chemistry to stabilize alpha-helical peptides that mimic native structures. The resulting drug products exhibit extended half-life, retain molecular specificity/potency, and in select cases penetrate cells to modulate intracellular targets. These attributes have been rationally incorporated into two clinical candidates: full p53 re-activator targeting MDM2/MDMX, and long-acting GHRH agonist that re-creates physiological GH profile.
Hubert Chen, MD, Vice President, Clinical Development, Aileron Therapeutics Inc., USA

3:15
Networking Refreshment Break in Poster and Exhibit Hall Sponsored by

4:00
Case Study
New Data
Clinical Experience of Integrative Cancer Immunotherapy with GcMAF
GcMAF is derived from the group-specific component (Gc) protein and activates macrophages. Our integrative immunotherapy approach is as follows: i) 0.5 ml GcMAF once or twice per week; ii) hyper T/natural killer (NK) cell therapy once per week; iii) high-dose vitamin C twice per week; iv) alpha lipoic acid daily; v) vitamin D3 daily. Three patient cases are also discussed.
Norihiro Sakamoto, Ph.D., Division of Food and Drug Evaluation Science, Department of Community Medicine and Social Healthcare Science, Kobe University Graduate School of Medicine, Japan

4:30
New Approach in Obesity Therapy Via Vascular-Targeted Nano-Particulate System
Anti-angiogenesis has been the focus of a new strategy for the treatment of obesity. We will introduce the development of vascular-targeted nanoparticles with peptide ligands for obesity control. The advantageous effects of nanoparticle-targeted therapeutics over that achieved when a peptide mimetic composed of the same targeting peptide and the proapoptotic peptide is used for obesity therapy are also discussed.
Kazuaki Kajimoto, Ph.D., Associate Professor, Laboratory for Molecular Design of Pharmaceutics Faculty of Pharmaceutical Sciences, Hokkaido University, Japan

5:00
Case Study
A Clinical Scale Dendritic Cell-Based Vaccine for the Treatment of Cancer in Japan
A large number of autologous dendritic cell cancer vaccines have been provided by tella Inc. to the tella's affiliated hospitals and clinics for treating various solid tumors in Japan, estimating more than 7,000 cases. Although data were analyzed retrospectively, we identified strong responders to our DC vaccines among the patients, suggesting that our DC vaccines are promising to a certain population.
Sei-ichi Yusa, Ph.D., Executive Officer, tella Inc., Japan

5:30
Networking Reception in Exhibit and Poster Hall Co-Sponsored by and

Main Conference · Concurrent Tracks
Thursday, February 27, 2014

8:30
Networking Coffee and Tea

Manufacturing and Analytical Development for Peptides and Oligos

8:55
Chairman's Remarks
Detlef Rethage, President, Nitto Avecia, USA

Updates on Oligonucleotide-Based Therapeutics in Preclinical and Clinical Development

10:00
New Data
Management of Tumor and Autoimmune Diseases Via Regulation of Dendritic Cell Activities by TLR Modulators
Dendritic cells are professional antigen processing cells and a conductor of immune reactions. pDCs is a distinct population of DCs and plays central role for induction of both innate and acquired immunity. As pDCs specifically express TLR9, pDC function is positively or negatively regulated by TLR9 modulators. Management of tumor and autoimmune diseases by TLR9 modulators will be presented.
Esashi Eiji, Ph.D., Director, Ginkgo Biomedical Research Institute, SBI Biotech Co. Ltd., Japan

Manufacturing and Analytical Development for Peptides and Oligos

9:30
Sarepta's PMO Manufacturing Platform - A Manufacturing Update
Recent Phase II clinical data (96 weeks of treatment) indicates Eteplirsen assists in significant slowdown of the fatal disease, Duchenne Muscular Dystrophy (DMD) in young boys as evident by significant increase in Dystrophin levels and the 6MWT results. Currently there is no therapy available for treating DMD. A manufacturing update for Sarepta's DMD franchise will be discussed from a technology platform perspective.
Jayant Aphale, Ph.D., MBA, RAC, Senior Vice President, Technical Operations, Sarepta Therapeutics, USA

10:00
New Data
Chemical Protein Synthesis with the KAHA-Ligation
Chemical ligation reactions allow the assembly of proteins by combining smaller, unprotected fragments by chemoselective amide-bond formation. We have developed a new approach to protein synthesis using the alpha-ketoacid--hydroxylamine (KAHA) ligation for the preparation of proteins up to 200 residues by the assembly of multiple segments. Other applications include the synthesis of peptide macrocycles without protecting groups or reagents and the PEGylation of peptide drugs.
Jeffrey W. Bode, Ph.D., Professor of Organic Chemistry, ETH Zurich, Switzerland

10:30
Networking Refreshment Break in Poster and Exhibit Hall

11:15
Spray Drying: The Alternative Peptide Isolation Technology to Lyophilization
Freeze-drying is the universal technology used for the isolation of peptides. However, this technology has its limitations in terms of productivity and huge investments are needed to install big freeze-dryers for commercial scale peptide isolation. Therefore Lonza initiated about 10 years ago a research program to find alternative and cheaper isolation technologies for peptides with a higher productivity. Spray drying was selected as the best technology and was developed and installed in Lonza's facilities. The technology was successfully tested on various peptides and can now be considered as a 2nd universal technology for the isolation of peptides. Since 2006 spray drying technology is used next to freeze-drying at Lonza for the isolation of commercial peptides.
Paul Tastenhoye, Head of Peptide Product Managers, Lonza Biopharmaceuticals, Belgium

11:45
Application of Process Analytical Technology (PAT) to Oligonucleotide Synthesis
Huseyin Aygun, Ph.D., CSO, Nucleic Acid Technologies, BioSpring GmbH, Germany

12:15
The Chemical Synthesis of Interferon β-1a
GlyTech, Inc. (Kyoto, Japan) and Bachem AG (Bubendorf, Switzerland) have successfully co-developed a chemical synthesisof Interferon β-1a for industrial scale. Interferon β-1a is a 166 amino acid long glycosylated protein and an approved drug substance to treat multiple sclerosis. There are currently three recombinant products on the market which are a mixture of at least 10 glycoforms. Bachem and GlyTech achieved a pioneering breakthrough with the successful chemical synthesis of this protein featuring a specific, single glycosylation. First bioactivity studies demonstrated that the new product is as good as or better than the recombinant Interferon β-1a. Bachem and GlyTech with their know how in peptide and carbohydrate chemistry are looking forward to a future partnership with a pharma or biotech company interested in conducting all further development activities through to market approval.
José Pierre de Chastonay, Ph.D., Chief Marketing Officer, Bachem Holding AG, Switzerland

12:45
Networking Luncheon in Poster and Exhibit Hall

1:55
Chairman's Remarks
Detlef Rethage, President, Nitto Avecia, USA

2:00
Case Study
New Data
Therapeutic Aptamer Discovery
RIBOMIC Inc. has been engaged in creating therapeutic RNA aptamers for unmet medical needs using the RiboART system for Ribomic Aptamer Refined Therapeutics. One such aptamer against NGF (nerve growth factor) acquired the pain-relief activity equivalent or superior to morphine. Other pipelines that passed in vivo animal efficacy test and toxicity study in RIBOMIC will be presented.
Yoshikazu Nakamura, Ph.D., President and CEO, Ribomic Inc., Japan

2:30
Case Study
New Data
Identifying Peptides and Sequence Simultaneously: Tools for Efficient Analysis
This presentation features data from impurity analyses that show how peptides in a highly complex mixture at low abundance can be identified, sequenced, and reported to the user in minutes. The presentation also covers the ability to overcome the challenge of manual data interpretation, human judgment and error, and to increase an organization's efficiency. The mechanisms are relevant to organizations that who are characterizing or monitoring bio therapeutics, working with synthetic peptides, and looking for process impurities.
StJohn Skilton, Senior Global Marketing Manager, Biologics, ABSciex, USA

3:00
Insights into shRNA Maturation and siRNA Specificity using Next Generation Sequencing
The talk contains data that challenges the accepted precision of siRNAs in substrate cleavage, established since 2002. The consequences of these findings are far-reaching with regards to the mode of action of these oligonucleotide drugs in terms of on- and off-target effects, with impact on clinical development. The data has been orally presented already in UK and EU meetings on RNAi and Next Gen Sequencing with very positive feedback, resulting in further invitations in Europe and the USA.
Dr. Sterghios A. Moschos, M.S.B., Reader in Industrial Biotechnology & Biochemistry, Department of Molecular and Applied Biosciences, School of Life Sciences, University of Westminster, UK

3:30
Spatial Proteomics - New Workflows for a New Paradigm in Protein and Peptide Localization and Detection
New developments in MALDI instrumentation, laser technology and sample preparation techniques have turned research-style MALDI imaging of tryptic peptides into a routine technique. This presentation is focused on a novel workflow called ImageID (Bruker Daltonics) that combines LC-MALDI with MALDI imaging thus correlating protein/peptide identification results using bottom-up proteomics approach with protein spatial localization in tissue.
Jason Wood, Ph.D., Market Area Manager, Biopharmaceuticals, Bruker Daltonics

DDS (Drug Delivery Systems) for Peptides and Oligonucleotides

8:55
Chairman's Remarks
Bob Brown, Ph.D., Chief Scientific Officer and Senior Vice President of Research, Dicerna Pharmaceuticals, USA

9:00
New Data
ArisGen Technologies: Novel Platforms to Enhance Peptide Therapeutics: Intracellular Delivery and Non-invasive Administration
ArisGen is striving to tackle current pharmaceuticals delivery/administration challenges of peptides. ArisGen developed two revolutionary platform technologies, ArisGen Targeting Technology (ArisTarget) for Intracellular delivery and ArisCrown a Formulation technology for peptide non-invasive (oral & buccal) administration. ArisTarget allows peptide intracellular delivery more efficiently with less toxicity compared to classical CPP vectorization like TAT, Antennopedia et others. Besides numerous collaborations, ArisGen is currently developing on its own a pipeline of peptide therapeutics to treat of (i) fibrocontractive diseases and (ii) a specific type of cancer. Many examples will be presented. ArisCrown formulation technology owns many proofs of concepts (PD and PK data) with a variety of peptides both with sub-lingual and oral administration. Extensive studies conducted with Exendin-4 by buccal administration showed remarkable results in all animal models tested.
Paolo Botti, Ph.D., Chief Scientific Officer, ArisGen SA, Switzerland

9:30
Topical Delivery of Nucleic Acids and Macromolecules
An overview of ultrasound, chemical enhancers, liquid microjets and peptide enhancers to enhance skin permeability. A high-throughput method to screen thousands of chemicals and combinations for safely and reversibly increasing skin permeability is also demonstrated, including the administrating of leuprolide acetate for the treatment of prostate cancer. Additionally, the presentation discusses a pulsatile jet injection method that can deliver drugs across the skin with nanoliter resolution for applications such as patient-controlled analgesia. Finally, peptide enhancers have been developed to deliver siRNA into the skin for treating dermatological diseases. Collectively, the above methods make up a versatile toolbox for designing novel transdermal therapies. One or more of these methods can be combined with a therapeutic of choice to achieve painless and needle-free drug administration.
Samir Mitragotri, Ph.D., Professor, Department of Chemical Engineering, University of California, Santa Barbara, USA

10:00
Physical and Chemical Characterization of LNP Formulations of Oligonucleotides vs. Biological Activities
Lipid-based nanoparticles (LNPs) containing oligonucleotides are advancing through preclinical and clinical development in multiple indications. While some standard USP release criteria clearly apply to this class of drug leads, significant questions remain about what unique release assays and specifications should be applied to these agents due to different compositions and manufacturing methods and the resulting variable characteristics within the class. The physical and chemical properties of some LNPs and their influence on biological effects in preclinical models, including efficacy and toxicity, will be described, along with assays indicative of quality, stability, and efficacy.
Bob Brown, Ph.D., Chief Scientific Officer and Senior Vice President of Research, Dicerna Pharmaceuticals, USA

10:30
Networking Refreshment Break in Poster and Exhibit Hall

11:15
pH-Sensitive Polymers for Drug Delivery
Polymers responsive to pH-gradients in the human body can deliver oligonucleotide- and peptide-drugs into the target sites. pH-sensitive polymers can be utilized for preventing drug degradation, colon drug delivery, tumor-targeting drug delivery, intracellular cytosol drug delivery by endosomal escape. Development of oligonucleotide and peptide products using pH-sensitive drug delivery system must considerably contribute to patients' health.
Takayuki Yoshida, Ph.D., Senior Researcher, Drug Delivery, Pharmaceutical Research and Technology Labs, Astellas Pharma Inc., Japan

11:45
Cytoplasm-Responsive Delivery Systems for siRNA Using Tat-Like Peptide Nanomicelles
To develop a gene carrier for siRNA, we synthesized the stable artificial cytoplasm-responsive cell-penetrating peptide conjugated with MPEG-PCL. Nucleotides were rapidly and rigidly packed in the nanomicelles by disulfide bonding, protected against nucleases in blood and persistently released into the reductive cytosol environment. The anti-VEGF siRNA complexes exerted a strong anti-tumor effect after intravenous injection in S-180 sarcoma-bearing mice.
Hiroaki Okada, Ph.D., Director (CEO), Okada DDS Research Institute, Inc.; Professor Emeritus, Tokyo University of Pharmacy & Life Sciences, Japan

Interactive Panel Discussion:

12:15
Delivery Approaches and Challenges
Moderator: Paolo Botti, Ph.D., Chief Scientific Officer, ArisGen SA, Switzerland
Panelist:
Bob Brown, Ph.D., Chief Scientific Officer and Senior Vice President of Research, Dicerna Pharmaceuticals, USA
Hiroaki Okada, Ph.D., Director (CEO), Okada DDS Research Institute, Inc.; Professor Emeritus, Tokyo University of Pharmacy & Life Sciences
Takayuki Yoshida, Ph.D., Senior Researcher, Drug Delivery, Pharmaceutical Research and Technology Labs, Astellas Pharma Inc., Japan
Samir Mitragotri, Ph.D., Professor, Department of Chemical Engineering, University of California, Santa Barbara, USA

12:45
Networking Luncheon in Exhibit and Poster Hall

Regulatory Considerations: Preclinical and Clinical Safety Assessment of Peptide and Oligonucleotides

1:55
Chairman's Remarks
Shawn Lee, President and CEO, CPC Scientific, USA

2:00
FDA Requirements for the Investigational Use of Peptide Products
Peptides bring new challenges when establishing adequate quality attributes and profile for the intended clinical use. Although synthetic peptides are manufactured using well understood manufacturing processes the potential complexity of their structure could complicate the characterization of the active substance as well as product related impurities in a way similar to protein biologic products. Therefore, this introduces the requirement and expectation of specifications that apply to small molecule synthetic drugs and large molecule biologics. The regulatory approach taken to establish the quality in relation to the established efficacy and safety profile of peptides will be presented.
David T. Lin, Ph.D., MBA, Senior Consultant, Biologics Consulting Group, Inc., and former acting Division Director in the Office of New Drug Chemistry in the Center for Drug Evaluation and Research (CDER), U.S. FDA, USA

2:30
Case Study
Is ICH-S6(R1) Guideline Applicable for Oligonucleotides?
According to the scope of ICH S6(R1), the principle of the guideline may be applicable for oligonucleotides. However, clarification and amplification are needed. This talk explains how much ICH S6(R1) would be applicable, and lessons learned are shared from biopharmaceuticals, as similar issues, such as the use of surrogates for oligonucleotides are faced.
Takahiro Nakazawa, Senior Director Research and Development, Anges MG Inc., Japan

3:00
Presentation to Be Announced

Regulatory Considerations and OSWG and JPMA Joint Session:
Preclinical and Clinical Safety Assessment of Peptide and Oligonucleotides

3:30
Regulatory and Safety Assessment Considerations for Peptides and Oligonucleotides
In recent years, with the advanced technology that allows the large scale production of high quality product, the peptide-based therapies are moving into the main stream of drug development. However, lack of harmonized international guidance and changes of technologies still present significant challenges for pharmaceutical companies during the drug development process. In the US, the regulatory issues have been made even more complicated by the new disease areas, the approval of peptide products by different centers under different laws (e.g. biologic, new drug, generic drug, and biosimilar). The presentation will update the recent changes of laws and review organizations with FDA and discuss how to navigate the regulatory process for the development of peptide products in the US.
Duu-Gong Wu, Ph.D., Director, former US FDA Supervisory Chemist and Regulatory Consulting, PPD, USA

4:00
Interactive Panel Discussion
Moderator: Shawn Lee, President and CEO, CPC Scientific, USA
Panelists:
David T. Lin, Ph.D., MBA, Senior Consultant, Biologics Consulting Group, Inc., and former acting Division Director in the Office of New Drug Chemistry in the Center for Drug Evaluation and Research (CDER), U.S. FDA, USA Takahiro Nakazawa, Senior Director Research and Development, Anges MG Inc., Japan Duu-Gong Wu, Ph.D., Director, former US FDA Supervisory Chemist and Regulatory Consulting, PPD, USA

4:30
Close of AsiaTIDES

TIDES 2014