TIDES: Oligonucleotide and Peptide® Research, Technology and Product Development
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重點講演
重點講演 星期一, 2012年5月21日
Courses: 星期日 | Plenary Sessions: 星期一 | Main Conference: 星期二 | 星期三
7:45
Registration and Coffee
Keynote Presentations
8:45
Chairperson's Remarks
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals
9:00
Therapeutic Oligonucleotide Development: A Promising OutlookRecent decisions by some pharmas to reduce, re-organize or establish partnerships for their oligonucleotide research programs have raised questions as to the future potential of this field. Although some early expectations for antisense and RNAi were not met, important lessons were learned. Positive human clinical data have been obtained not only in antisense and RNAi applications, but also in the development of oligonucleotides for immune stimulatory, splice altering, aptamer, and microRNA therapeutics. This presentation will provide an overview of the first decade of oligonucleotide development and highlight recent technical advances that are making increasing contributions to drug development pipelines.
Art Krieg, M.D., Entrepreneur in Residence, Atlas Ventures
9:45
Resources to Enable and Facilitate siRNA Drug Development in ChinaLiquid phase synthesis technologies have been explored by Suzhou Ribo for potential production of siRNA in multi-kg to tons scales. Kunshan, a town near Shanghai and Suzhou with most robust economy in China, has invested multi-millions of US dollars to build the biggest RNAi facility in China to facilitate nucleic acid-based technological innovations and drug development. The initial form of an "RNAi Valley" in China has now taken its shape in Kunshan.
Zicai Liang, Ph.D., Chairman, Suzhou Ribo Life Science Co., LTD, China
10:30
Networking Refreshment Break
11:00
Harnessing the "New" Endocrinology of Peptide Hormones - Discovery of Novel Therapies Targeting Metabolic DiseaseThe biology of entero-endocrine pathways (gut-derived hormones) is an area of great interest for continued drug discovery efforts. Key peptide hormones in this axis include glucagon-like peptide 1 (GLP1), gastric inhibitory polypeptide (GIP), cholecystokinin (CCK), peptide YY, oxyntomodulin, ghrelin and others. Lessons from human physiology, pathophysiology and exploratory clinical experiments have greatly informed the selection of R&D projects in this arena. Examples of current drug discovery efforts focused on several of these pathways will be presented along with selected approaches designed to leverage current technology platforms to target best-in-class therapeutics.
David E. Moller, M.D., Vice President, Endocrine and Cardiovascular Research and Clinical Investigation, Lilly Research Laboratories, Eli Lilly and Co.
11:45
RaPID Discovery of Natural Product-Like Peptides against Therapeutic TargetsWe recently devised a new means to reprogram the genetic code, which allows us to express non-standard peptides containing multiple non-proteinogenic amino acids in vitro. This lecture will describe the most recent development in the genetic code reprogramming approach that enables us to express natural product-like non-standard peptides and screen them against various drug targets inexpensively, less laboriously, and very rapidly by a selection platform, referred to as RaPID (Random Peptide Integrated Discovery) system.
Hiroaki Suga, Ph.D., Professor, Chemical Biology and Biotechnology Lab, The University of Tokyo, Japan
12:30
Lunch on your own
Updates on Late Stage Clinical Candidates
1:40
Chairperson's Remarks
John P. Mayer, Ph.D., Senior Research Advisor, Eli Lilly and Co.
John P. Mayer, Ph.D., Senior Research Advisor, Eli Lilly and Co.
New, Unpublished Data
1:45
SOM230: A New Therapeutic Modality for Cushing's Disease
SOM230 has recently shown promise as the first effective pituitary directed medical treatment for Cushing's disease. Indeed, the multiple high affinity binding of SOM230 to somatostatin receptor subtypes enables much more effective inhibition of ACTH release in-vitro and in-vivo. Recent clinical studies involving treatment of Cushing's disease with SOM230 have demonstrated that SOM230 produced a decrease in urinary free cortisol (UFC) levels in 76% of patients during 15 days, with direct effects on ACTH release, establishing a new therapeutic modality for Cushing's disease.
Ian Lewis, Ph.D., Investigator III, Global Discovery Chemistry, Novartis Institutes of Biomedical Research, Switzerland
SOM230 has recently shown promise as the first effective pituitary directed medical treatment for Cushing's disease. Indeed, the multiple high affinity binding of SOM230 to somatostatin receptor subtypes enables much more effective inhibition of ACTH release in-vitro and in-vivo. Recent clinical studies involving treatment of Cushing's disease with SOM230 have demonstrated that SOM230 produced a decrease in urinary free cortisol (UFC) levels in 76% of patients during 15 days, with direct effects on ACTH release, establishing a new therapeutic modality for Cushing's disease.
Ian Lewis, Ph.D., Investigator III, Global Discovery Chemistry, Novartis Institutes of Biomedical Research, Switzerland
2:15
A Linaclotide Program Summary
Linaclotide, an investigational drug, is an agonist of the guanylate cyclase type‐C (GC‐C) receptor located on the luminal surface of the intestine. In preclinical models, linaclotide reduced visceral hypersensitivity, increased fluid secretion, and accelerated intestinal transit. The effects on secretion and transit are mediated through cyclic guanosine monophosphate (cGMP), which is also believed to modulate the activity of local nerves to reduce pain. Linaclotide is an orally delivered peptide that acts locally in the gut with minimal systemic exposure at therapeutic doses. Data from two Phase 3 clinical trials in patients with irritable bowel syndrome with constipation (IBS-C) will also be presented.
Angelika Fretzen, Ph.D., Vice President, Pharmaceutical Chemistry and Development, Ironwood Pharmaceuticals
Linaclotide, an investigational drug, is an agonist of the guanylate cyclase type‐C (GC‐C) receptor located on the luminal surface of the intestine. In preclinical models, linaclotide reduced visceral hypersensitivity, increased fluid secretion, and accelerated intestinal transit. The effects on secretion and transit are mediated through cyclic guanosine monophosphate (cGMP), which is also believed to modulate the activity of local nerves to reduce pain. Linaclotide is an orally delivered peptide that acts locally in the gut with minimal systemic exposure at therapeutic doses. Data from two Phase 3 clinical trials in patients with irritable bowel syndrome with constipation (IBS-C) will also be presented.
Angelika Fretzen, Ph.D., Vice President, Pharmaceutical Chemistry and Development, Ironwood Pharmaceuticals
2:45
Update on Peginesatide (formerly Hematide™), an Investigational Drug for the Treatment of Anemia in CKD Patients on Dialysis
Peginesatide is a synthetic PEGylated peptidic compound that binds to and stimulates the erythropoietin receptor and acts as an erythropoiesis stimulating agent. Conjugation to PEG prolongs the in vivo half life enabling once-monthly dosing of the drug. Highlights of the discovery and development programs including clinical results evaluating the safety and efficacy of peginesatide will be presented.
Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc.
Peginesatide is a synthetic PEGylated peptidic compound that binds to and stimulates the erythropoietin receptor and acts as an erythropoiesis stimulating agent. Conjugation to PEG prolongs the in vivo half life enabling once-monthly dosing of the drug. Highlights of the discovery and development programs including clinical results evaluating the safety and efficacy of peginesatide will be presented.
Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc.
3:15
Grand Opening of Poster and Exhibit Hall with Networking Refreshment Break
3:55
Chairperson's Remarks
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc.
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc.
New, Unpublished Data
4:00
Case
Study
The REG1 Anticoagulant System: A Novel Therapeutic Platform for Use in Coronary RevascularizationStudy
The REG1 Anticoagulant System consists of pegnivacogin, an aptamer-based direct coagulation FIXa inhibitor and anivamersen, its active control agent. REG1 recently completed the phase 2b RADAR study in patients with Acute Coronary Syndromes undergoing early coronary catheterization. We will present the development strategy for REG1 in coronary revascularization indications and the results from RADAR, which provide proof-of-concept that REG1 has the potential to reduce both ischemic events and major bleeding in percutaneous coronary intervention. Manufacturing process scale-up for phase 3 development and commercialization will also be discussed.
Christopher P. Rusconi, Ph.D., Senior Vice President, Discovery/Preclinical Development, Chief Scientific Officer, Regado Biosciences, Inc.
New, Unpublished Data
4:30
RNAi Therapeutics for Genetic Diseases: TTR Amyloidosis
Therapeutics based on RNAi represent a new class of drugs with tremendous potential. Candidate siRNA drugs such as ALN-TTR targeting transthyretin for the treatment of TTR amyloidosis and ALN-PCS targeting proprotein convertase subtilisin/kexin type 9 for the treatment of hypercholesterolemia are in early clinical development. In this talk, an update on ALN-TTR as well as other programs will be discussed.
Dinah W.Y. Sah, Ph.D., Vice President, Research, Alnylam Pharmaceuticals, Inc.
Therapeutics based on RNAi represent a new class of drugs with tremendous potential. Candidate siRNA drugs such as ALN-TTR targeting transthyretin for the treatment of TTR amyloidosis and ALN-PCS targeting proprotein convertase subtilisin/kexin type 9 for the treatment of hypercholesterolemia are in early clinical development. In this talk, an update on ALN-TTR as well as other programs will be discussed.
Dinah W.Y. Sah, Ph.D., Vice President, Research, Alnylam Pharmaceuticals, Inc.
5:00
Case
Study
Clinical and Regulatory Update on HEPLISAV: An Innovative HBV Vaccine Using an Oligonucleotide TLR9 Agonist AdjuvantStudy
Dynavax has developed a novel HBV vaccine that replaces the traditional adjuvant, alum, with an oligonucleotide TLR9 agonist. This vaccine, HEPLISAV, has demonstrated earlier and higher seroprotection with fewer immunizations in a wide variety of populations including young adults, older adults and patients with Chronic Kidney Disease. Results from the clinical program and challenges faced in the development of HEPLISAV will be discussed.
J. Tyler Martin, M.D., President and Chief Medical Officer, Dynavax Technologies Corporation
New, Unpublished Data
5:30
Case
Study
An Antisense Oligonucleotide (EXC 001) Targeting Connective Tissue Growth Factor as a Novel Approach to Reduce Skin ScarringStudy
Skin scars represent a significant unmet medical need for which no approved targeted therapy exists. An ASO (EXC 001) has been developed which inhibits expression of connective tissue growth factor (CTGF), a regulator of skin scarring. The phase 2 program for EXC 001 has produced positive clinical results in reducing scar severity in multiple clinical trials, which will be presented.
Nicholas M. Dean, Ph.D., CSO, Excaliard Pharmaceuticals
6:00
Networking Reception with Poster and Exhibit Viewing