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8:00am - 8:55am

Registration and Coffee

Showing of Streams
10:30am - 11:10am

Networking Refreshment Break with Exhibit and Poster Viewing

Showing of Streams
3:30pm - 4:00pm

Networking Refreshment Break with Exhibit and Poster Viewing

Showing of Streams
5:00pm - 6:30pm

Networking Cocktail Reception in the Exhibit and Poster Hall

more
6:30pm - 6:35pm
Close of Day Two

Close of Day Two

8:00am - 8:55am 55 mins
Registration and Coffee
8:55am - 9:00am 5 mins
Regulatory Strategies for Oligonucleotides and Peptides
Chairperson’s Remarks
  • Bruce Given, MD - Chief Operating Officer, Arrowhead Research Corporation
more
9:00am - 9:30am 30 mins
Regulatory Strategies for Oligonucleotides and Peptides
Regulatory Considerations for the Development of Oligonucleotide Therapeutics in the United States
  • David Lin, Ph.D. - Senior Consultant, Biologics Consulting (Former Chemistry Team Leader and Former Acting Division Director, Division of New Drug Chemistry, CDER, FDA)
more

The typical factors for development of oligonucleotide products include potency/efficacy, stability in vivo, favorable pharmacokinetics (PK), favorable pharmacodynamics (PD), minimization of off-target effects and safety.  However, oligonucleotide drugs fall somewhere between small molecules and large-molecule biologics, creating a new set of unique regulatory challenges.  Oligonucleotides are chemically synthesized, and despite the diversity within this class of drugs, there are similarities in approaches for synthesis.  This presentation will focus on quality expectations for oligonucleotide products and will explore the application of the quality concerns to oligonucleotide products.

9:30am - 10:00am 30 mins
Regulatory Strategies for Oligonucleotides and Peptides
Regulatory Issues and Challenges during Different Phases of Peptide Product Development in US
  • Duu-Gong Wu, PhD - Senior Director, Regulatory Consulting, PPD
more

Peptides as chemically-synthesized molecules with biological activities present some unique regulatory challenges during the product development. For peptide products, the approvals for marketing as both drugs and biologics under different laws and regulations and review organizations with US FDA add additional complexities and difficulties during different phases of development in United States. The presentation will discuss: 1) Current regulatory trend in US for the review and approval of peptide products, 2) Different regulatory issues and data requirements for the peptide products to be developed as a drug or biologic and 3) How to navigate the unique regulatory and scientific issues during different phases of peptide development, e.g. to avoid clinical hold.

10:00am - 10:30am 30 mins
Regulatory Strategies for Oligonucleotides and Peptides
Novel siRNA Therapeutics for Fibrotic Disease Treatment with Polypeptide Nanoparticle Delivery Technology: Regulatory Experiences in US and China
  • Patrick Lu, PhD - President and CEO, Sirnaomics and Nanotides Pharmaceuticals, Inc.
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Using a proprietary and optimized polypeptide-based delivery technology, we have developed the novel antifibrotic therapeutics with siRNAs targeting both TGFβ1 and Cox-2 simultaneously, for initial indication of skin hypertrophic scar followed with liver fibrosis and other fibrotic conditions. With IND approvals by both US FDA and China CFDA, we have started clinical phase 2a in US and preparing clinical phase 1 in China for our leading drug candidate STP705 (Cotsiranib®) to treat human skin hypertrophic scar. I will discuss the unique advantage of our Polypeptide Nanoparticle (PNP) technology platform for safe and efficient siRNA delivery, and our strategy for advancing multiple clinical studies in both China and USA in near future.

10:30am - 11:10am 40 mins
Networking Refreshment Break with Exhibit and Poster Viewing
11:10am - 11:15am 5 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Chairperson’s Remarks
  • Chairperson William Marshall, PhD - President and Chief Executive Officer, miRagen Therapeutics, Inc.
more
11:10am - 11:15am 5 mins
Peptide Track: Peptide Discovery and Development
Chairperson’s Remarks
  • El Djouhar Rekaï, PhD - Head of Operation Products, PolyPeptide Group
more
11:15am - 11:45am 30 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Non-clinical Development of DCR-PHXC for the Treatment of Primary Hyperoxalurias
  • Bob Brown, PhD - Chief Scientific Officer, Senior Vice President, Research, Dicerna Pharmaceuticals
more

Primary hyperoxalurias are closely related genetic disorders resulting in elevated oxalate production in the liver. Excess oxalate accumulates in the urine where it often precipitates with calcium, leading to a variety of symptoms including frequent and severe kidney stone formation, nephrocalcinosis, and kidney failure. We are investigating a potential treatment for all forms of primary hyperoxaluria with a GalNAc-conjugated Dicer-substrate siRNA targeting the enzyme LDHA.

11:15am - 11:45am 30 mins
Peptide Track: Peptide Discovery and Development
Genetically-Encoded Chemically-Modified Peptides
  • Ratmir Derda, PhD - Associate Professor, Department of Chemistry, University of Alberta
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We use genetically-encoded (GE) libraries as starting materials for multi-step organic synthesis. Examples are N-terminal conjugation(1) and cyclization of linear peptides(2) with simultaneous introduction of glycan entities. We developed the Genetically-Encoded Fragment-Based Discovery (GE-FBD) platform;(3) which combines >109 peptides with GE-modifications.(4) The talk will describe the progress and challenges in application of GE-FBD platform.

References

  1. (a) J. Am. Chem. Soc., 2014, 136, 8149. (b) ACS Chem. Biol., 2012, 7, 1482
  2. (a) Chem. Sci., 2016, 7, 3785. (b) Org. Biomol. Chem., 2016, 14, 5539-5545.
  3. Ng et al., J. Am. Chem. Soc., 2015, 137, 5248
  4.  Tjhung et al., J. Am. Chem. Soc., 2016, 138, 32.
11:45am - 12:15pm 30 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Discovery and Development of microRNA Targeting Therapeutic Candidates for the Treatment of Hematological Malignancy and Pathological Fibrosis
  • William Marshall, PhD - President and Chief Executive Officer, miRagen Therapeutics, Inc.
more

MicroRNAs regulate the expression of gene networks that culminate in the control of complex cellular processes.  Dysregulation of the expression of certain microRNAs may affect pathways that contribute to disease pathology.  We have advanced two product candidates into clinical trials; MRG-106, a LNA antimiR® targeting microRNA-155 in hematological malignancies and MRG-201, a synthetic replacement (promiR) for microRNA-29 in pathological fibrosis.  An overview of our latest clinical observations will be presented.

11:45am - 12:15pm 30 mins
Peptide Track: Peptide Discovery and Development
Discovery and Development of Synthetic Polymyxins for Targeting Gram-negative ‘Superbugs’
  • Kade Roberts, PhD - Senior Research Fellow, Biomedicine Discovery Institute, Monash University
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In this presentation, we report on our ongoing pre-clinical polymyxin drug development program, a National Institutes of Health (NIH) funded joint academic-industry collaboration between Monash University (Australia) and The Medicines Company (USA). This program aims to produce new polymyxin peptide clinical candidates with improved safety and efficacy over the currently used drugs, polymyxin B and colistin. Aspects of our novel drug design strategy and lead optimization studies will be discussed. To date a number of promising lead candidates have been identified with significantly improved safety profiles and are being progressed towards clinical evaluation.

12:15pm - 12:45pm 30 mins
Oligonucleotide Track: Preclinical and Clinical Updates
RNAi Therapeutics: From Principles to Patients
  • Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc.
more
12:15pm - 12:45pm 30 mins
Peptide Track: Peptide Discovery and Development
Polypharmacy through Phage Display: Selection of Glucagon and GLP-1 Receptor Co-agonists from a Phage-Displayed Peptide Library
  • Elisabetta Bianchi - Head of Peptide Chemistry, IRBM Science Park
more

An emerging area for the treatment of obesity and diabetes is combinatorial hormone therapies, where single molecule peptides are rationally designed to integrate the complementary actions of multiple endogenous metabolically-related hormones. In this study, we report unimolecular polypharmacy agents selected on phage-displayed peptide libraries (PDL). Co-agonists of the glucagon (GCG) and GLP-1 receptors were identified after sequentially selection on GCGR- and GLP1R-overexpressing cells, showing EC50 ≤ 30 pM on each receptor. These results validate the approach for the discovery of optimized polypharmacology paradigms across several metabolically-related hormones.

12:45pm - 1:55pm 70 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Networking Luncheon with Exhibit and Poster Viewing
12:45pm - 1:55pm 70 mins
Peptide Track: Peptide Discovery and Development
Networking Luncheon with Exhibit and Poster Viewing
1:55pm - 2:00pm 5 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Chairperson’s Remarks
  • Chairperson William Marshall, PhD - President and Chief Executive Officer, miRagen Therapeutics, Inc.
more
1:55pm - 2:00pm 5 mins
Peptide Track: Peptide Discovery and Development
Chairperson’s Remarks
  • Elisabetta Bianchi - Head of Peptide Chemistry, IRBM Science Park
more
2:00pm - 2:45pm 45 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Clinical Update on DS-5141b an Oligonucleotide Drug for DMD Treatment
  • Osamu "Sam" Sato, PhD - Executive Director, R&D Planning Department, Daiichi Sankyo Co., Ltd.
more
2:00pm - 2:30pm 30 mins
Peptide Track: Peptide Discovery and Development
Intracellular Screening for Specific Peptide Antagonists of Proteins
  • Jody Mason, PhD - Senior Lecturer In Biochemistry, University of Bath
more

We use Protein-fragment Complementation to identify peptide antagonists of 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 increased by expressing off-target proteins during selection. Antagonists are downsized and refined using structure-inducing constraints and non-natural sequences.

2:30pm - 3:00pm 30 mins
Peptide Track: Peptide Discovery and Development
Synthetic Therapeutic Peptide – Challenges with Quality Standard and Impurities
  • Ranjan Chakrabarti, PhD - Vice President, Biologics & Biotechnology, United States Pharmacopeia
more

USP develops and publishes standards which are recognized in the Federal Food, Drug and Cosmetic (FD&C) Act since it was first enacted in 1938. U.S. Pharmacopeia (USP) has official monographs and reference standards (RS) for synthetic, recombinant, naturally-derived therapeutic peptides under 40 amino acids in length and insulin products. USP Therapeutic Peptides Expert Panel was formed in 2013 to evaluate quality attributes for synthetic peptide therapeutics based on currently available regulatory guidance and expectations. Quality attributes recommended by the Panel, has been incorporated during modernization of monographs for older peptide products. Compendial requirements for new monographs are aligned to the panel recommendation. The presentation will describe USP’s current synthetic peptide program and our continued effort to set quality standards for this product class incorporating the Expert Panel’s recommendations on the quality attributes.  Compendial challenges such as impurities are illustrated using case studies.  Additionally, future standardization opportunities are outlined.

2:45pm - 3:30pm 45 mins
Oligonucleotide Track: Preclinical and Clinical Updates
An Oligonucleotide for the Treatment of Duchenne Muscular Dystrophy (DMD): An Update after Launch
  • Ryszard Kole, PhD - Consultant, Ercole Biotech Consulting
more

Speaker TBA, Sarepta Therapeutics, USA

3:00pm - 3:30pm 30 mins
Peptide Track: Peptide Discovery and Development
Oral Peptide Development/CMC: Learnings from Scaling up API and Reducing Costs for Peptide Manufacturing
  • Robert Geiger, PhD, MBA - Vice President of Quality, AmbioPharm
more

Major consumable costs for peptide manufacturing include the cost of

protected amino acids, coupling reagents, and solvents. At smaller scales,

labor and manufacturing facility expenses dominate manufacturing cost.

However, at larger scales acquisition and disposition of solvents dominate cost.

The relative costs and advantages of solid phase, solution phase and hybrid

solid/solution phase manufacturing of peptides for oral use will be discussed.

3:30pm - 4:00pm 30 mins
Networking Refreshment Break with Exhibit and Poster Viewing
4:00pm - 4:30pm 30 mins
Oligonucleotide Track: Manufacturing, CMC and Purification Strategies
5'-GalNAc-Conjugated Antisense Oligonucleotides: Synthesis and Purification Challenges
  • Andrew Rodriguez, PhD - Assistant Director, Process Chemistry, Ionis Pharmaceuticals
more

GalNAc-conjugated oligonucleotides represent an exciting approach to antisense therapy. In this presentation, we will discuss the challenges associated with production of these compounds. Solution-phase and solid-phase conjugation strategies capable of generating high-quality product will be presented and compared in terms of yield and purity.

4:00pm - 4:30pm 30 mins
Peptide Track: Peptide Impurities, Control Strategies and Manufacturing Learnings
Higher Molecular Weight (HMW) Peptide Impurities – Control Strategies and Acceptance Criteria
  • Daniel Samson, PhD - Vice President, API SPPS, Bachem AG
more

For peptide drug substances in early clinical development there is often a very strong focus on fast drug substance supply. Therefore, the development of manufacturing process capabilities and economy as well as purity and impurity profile is performed in parallel. Aggregation-prone peptides implicate additional challenges for process development. Furthermore, it is known that sometimes sub-optimal quality attributes such as poor solubility, a shortened shelf life due to high HMW-content may only become apparent in subsequent drug product formulations. Different methods and strategies are applied at BACHEM to minimize and control HMW species of aggregation-prone peptides during synthetic downstream processing. Focusing on typical process stages, BACHEM’s standard approach for optimization of process parameters regarding aggregation is presented within different case studies.

4:30pm - 5:00pm 30 mins
Oligonucleotide Track: Manufacturing, CMC and Purification Strategies
New Purification Process for Antisense Oligonucleotides
  • Robert Gronke, PhD - Senior Principal Scientist, Technical Development, Biogen, Inc
more

Recent efforts in oligonucleotide purification development have shown that an all aqueous process can achieve higher purities by employing orthogonal separation mechanisms.  Higher purity is obtained by reducing challenging impurities such as N-1, P=O, N+1, without sacrificing yield.    Positioning the detritylation step between the columns provides robust reaction control.  An example of this process and its capabilities will be presented.

4:30pm - 5:00pm 30 mins
Peptide Track: Peptide Impurities, Control Strategies and Manufacturing Learnings
Control of Non-Peptide Impurities in APIs Originated from Materials/Consumables/Equipment Contact through Process Manufacturing and Storage
  • El Djouhar Rekaï, PhD - Head of Operation Products, PolyPeptide Group
more

Pharmaceutical industries devote significant efforts to identify, quantify, and minimize level of impurities in drug products and consequently in drug substances (APIs). Chemical compounds can be leached into API from various components used in the manufacturing processes leading to potential adulteration of the API, and potentially affecting safety or efficacy of the drug product. This concern drives manufacturers to develop relevant control strategies. This presentation gives an overview and background of our risk management approach to address extractables / leachables, including elemental impurities, in peptide APIs.

5:00pm - 6:30pm 90 mins
Networking Cocktail Reception in the Exhibit and Poster Hall
more
6:30pm - 6:35pm 5 mins
Close of Day Two