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Feb 28
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8:00am - 8:40am

Registration and Coffee

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Showing of Streams
10:15am - 10:55am

Networking Refreshment Break with Poster and Exhibit Viewing

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Showing of Streams
12:30pm - 1:40pm

Networking Luncheon with Exhibit and Poster Viewing

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Showing of Streams
3:15pm - 3:45pm

Networking Refreshment Break with Poster and Exhibit Viewing

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Showing of Streams
5:15pm - 5:25pm

Close of Conference

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8:00am - 8:40am 40 mins
Registration and Coffee
8:40am - 8:45am 5 mins
Main Conference
Chairperson's Remarks
8:45am - 9:15am 30 mins
Info
Main Conference
Regulatory and Clinical Development Strategy for Peptides in the Era of ICH; Oligonucleotide Products - Comparing the Regulatory Environment in US and China
  • Dan Zhang, PhD - Co-Founder & CEO, Fountain Medical Development

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

9:15am - 9:45am 30 mins
Info
Main Conference
US Regulatory Update: Peptide and Device Combination Products and the Lessons Learned from the Approval of Oligonucleotide Product, Macugen®
  • Duu-Gong Wu, PhD - Senior Director, Regulatory Consulting, PPD

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

9:45am - 10:15am 30 mins
Info
Main Conference
Understanding FDA’s Draft Guidance for a Generic Product of a Highly Purified Synthetic Peptide that References an Approved Application for a Recombinant Peptide Product
  • David Lin, PhD, MBA - Senior Consultant, Biologics Consulting Group, Inc.

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

10:15am - 10:55am 40 mins
Networking Refreshment Break with Poster and Exhibit Viewing
10:55am - 11:00am 5 mins
Oligonucleotide Track: Delivery Strategies
Chairperson's Remarks
10:55am - 11:00am 5 mins
Peptide Track: Peptide CMC Strategies
Chairperson's Remarks
11:00am - 11:30am 30 mins
Info
Oligonucleotide Track: Delivery Strategies
Emerging RNA Therapeutics: Delivery Approaches for mRNA, siRNA and Beyond
  • Shiro Akinaga, PhD - Director and CSO, AccuRna

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

11:00am - 11:30am 30 mins
Info
Peptide Track: Peptide CMC Strategies
Introducing Green and Sustainable Solvents in Liquid Phase Peptide Manufacturing Processes

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

11:30am - 12:00pm 30 mins
Info
Oligonucleotide Track: Delivery Strategies
siRNA Delivery is Still the Key to Therapeutic Success
  • David Evans, PhD - Co-Founder and CSO, Sirnaomics

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

11:30am - 12:00pm 30 mins
Info
Peptide Track: Peptide CMC Strategies
Peptide Quality Attributes and USP Standards
  • Ranjan Chakrabarti, PhD - Vice President, Biologics & Biotechnology, United States Pharmacopeia

United States Pharmacopeia (USP) has official monographs for peptides.
To understand and evaluate the current regulatory considerations and
expectations, an expert panel was formed. The Panel after detailed
deliberation, recommended key Quality Attributes for incorporation in USP
standards. Compendial requirements for new monographs are aligned
to the panel recommendation. The presentation will describe the Expert
Panel’s recommendations on the quality attributes with case studies from
USP’s current synthetic peptide program.

12:00pm - 12:30pm 30 mins
Info
Oligonucleotide Track: Delivery Strategies
NJA-730, The First Therapeutic Compound from NapaJen’s Platform Delivery Technology for aGvHD (acute Graft versus Host Disease) Is in Clinical Stage
  • Kenji Arima, PhD - Chief Development Officer, NapaJen Pharma, Co. Ltd.

Our platform technology is targeted delivery of oligonucleotide therapeutic
compounds to antigen presenting cells using SPG (schizophyllan) as a
delivery vehicle. We will introduce NapaJen’s platform delivery technology
and report progress in Phase 1 trial of NJA-730, a CD40-targeting siRNA
complexed with SPG, which shows potent pharmacological activity.

12:00pm - 12:30pm 30 mins
Info
Peptide Track: Peptide CMC Strategies
Cost Efficient Peptide Purification via ZEOsphere DRP Mixed-Mode Chromatography
  • Jurgen Machielse - Business Development Director, Zeochem AG

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

12:30pm - 1:40pm 70 mins
Networking Luncheon with Exhibit and Poster Viewing
1:40pm - 1:45pm 5 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Chairperson's Remarks
1:40pm - 1:45pm 5 mins
Peptide Track: Peptide Case Studies
Chairperson's Remarks
1:45pm - 2:15pm 30 mins
Info
Oligonucleotide Track: Oligonucleotide Case Studies
Anti-FGF2 Aptamer RBM-007 in Phase I/IIa trials for Wet AMD
  • Yoshikazu Nakamura, PhD - President and CEO, Ribomic Inc

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

1:45pm - 2:15pm 30 mins
Info
Peptide Track: Peptide Case Studies
Linear and Convergent Approaches to Peptide Synthesis - A Comparison from A Manufacturer’s Point of View
  • Daniel Samson, PhD - Vice President, API SPPS, Bachem AG

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

2:15pm - 2:45pm 30 mins
Info
Oligonucleotide Track: Oligonucleotide Case Studies
Therapeutic RNA Medicines: The Silence Therapeutics Experience in Haematology and Rare Diseases
  • David Horn Solomon, M.D - CEO, Silence Therapeutics

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

2:15pm - 2:45pm 30 mins
Info
Peptide Track: Peptide Case Studies
ESCAPE-NA1 and FRONTIER: Two Complementary Phase 3 Trials to Demonstrate the Safety and Efficacy of NA-1 in Acute Ischemic Stroke
  • Dave Garman, PhD - Chief Technology Officer, NoNO, Inc.

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

2:45pm - 3:15pm 30 mins
Info
Oligonucleotide Track: Oligonucleotide Case Studies
TANGO – Targeted-Augmentation of Nuclear Gene Output – for the Treatment of Genetic Diseases
  • Huw Nash, PhD - Chief Operating Officer and Chief Business Officer, Stoke Therapeutics

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

2:45pm - 3:15pm 30 mins
Info
Peptide Track: Peptide Case Studies
The Orally Available Clinical Stage All-D-enantiomeric Peptide PRI-002 Reverses Cognition Deficits and Decelerates Neurodegeneration in Transgenic Alzheimer’s Mouse Models
  • Dieter Willbold, Ph.D. - Director, ICS-6 Structural Biochemistry, Forschungszentrum Jülich, Germany

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

3:15pm - 3:45pm 30 mins
Networking Refreshment Break with Poster and Exhibit Viewing
3:45pm - 4:15pm 30 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Latest Clinical Results from an Oligonucleotide in Development
  • Osamu Sato, PhD - Executive Director, R&D Planning Department, Daiichi Sankyo Co., Ltd.
3:45pm - 4:15pm 30 mins
Info
Peptide Track: Peptide Case Studies
NRP2945 with Anti-epileptic Activities in Patients with Drug-resistant Absence Epilepsy
  • Frank Sieg, PhD - Chief Scientist, CuroNZ Limited

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

4:15pm - 4:45pm 30 mins
Info
Oligonucleotide Track: Oligonucleotide Case Studies
Asymmetric siRNA Targeting Fibrotic and Ocular Disorders
  • Dong-Ki Lee, PhD - CEO, OliX Pharmaceuticals

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

4:15pm - 4:45pm 30 mins
Info
Peptide Track: Peptide Case Studies
Development of a Peptide Vaccine for the Induction of Epitope-specific Antibodies
  • Hideki Tomioka, PhD - Member of the Board, FunPep Co., Ltd.

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

4:45pm - 5:15pm 30 mins
Info
Oligonucleotide Track: Oligonucleotide Case Studies
MicroRNA-155 Inhibition by Cobomarsen Demonstrates Encouraging Activity in Hematological Malignancies
  • William Marshall, PhD - President and Chief Executive Officer, miRagen Therapeutics, Inc.

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

4:45pm - 5:15pm 30 mins
Info
Peptide Track: Peptide Case Studies
Osseotide, A Synthetic, Selective Osteogenic Peptide for the Treatment of Osteoporosis
  • Yoon-Jeong Park, PhD - Professor, Seoul National University

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

5:15pm - 5:25pm 10 mins
Close of Conference