Day 1 | Day 2
DAY TWO: WEDNESDAY, OCTOBER 14, 2009
Sponsored by
7:45 am Continental Breakfast Presentation 505(b)(2): Not Just for Modifications of Approved Drugs
Ken Phelps, President & Chief Executive Officer, Camargo Pharmaceutical Services
Historically, most sponsors have used the 505(b)(2) pathway to make improvements to existing approved drugs. Yet, the regulations state that a 505(b)(2) can be used for any drug product where pivotal information is not conducted by the applicant. Thus, even a new chemical entity can be approved under 505(b)(2). This understanding will open up a vast source of potential drug products for development over the next decade. This Session will review the regulatory requirements for a 505 (b)(2) NDA and the kinds of information that may be used in support of an NDA submission, as well as the process needed to gain FDA buy-in to the 505 (b)(2) development plan and some of the products currently being developed without a U.S.-approved reference drug.
8:30 Chairperson’s Opening Remarks
8:40 Translational Research in the Development of Novel Nanobody-Based Therapies
Josi Holz, M.D., Chief Medical Officer, Ablynx NV
Nanobodies are a novel class of antibody derived therapeutic proteins. Because of their small size, unique structure and extreme stability, nanobodies combine the advantages of conventional antibody therapeutics with the key features of small molecule drugs. This talk will illustrate in a case study for an anti-vWF Nanobody how the translation of results in pre-clinical development (e.g. use of biomarker, PK and PD modeling) into clinical development accelerated the achievement of proof-of-concept. The presented case study illustrates the rapid advancement of a novel protein drug from target identification to completion of phase I with biological proof-of-concept in the clinic in less than 4 years.
9:10 Imaging Biomarkers in Early-Phase CNS Studies
Alan Evans, Ph.D., Professor, Neurology & Neurosurgery, Montreal Neurological Institute, McGill University
Imaging biomarkers based on the quantitative analysis of MRI and PET images are increasingly utilized in early-phase studies of CNS diseases, such as Alzheimer’s disease, Parkinson’s disease, and multiple sclerosis. The combination of data from structural, functional, molecular imaging studies provide new insight into the biodistribution, mechanism-of-action, and efficacy of novel therapeutic agents for CNS diseases. This presentation will discuss the use of advanced imaging biomarkers to allow for earlier go/no go decision making, as well as for more efficient and cost-effective early phase studies.
9:40 Reducing Attrition due to Hepatotoxicity through Early Safety Assessments of Mitochondrial Dysfunction
Sashi Nadanaciva, Ph.D., Principal Scientist, Compound Safety Prediction, Pfizer Global R&D Groton Labs
Recent retrospective analysis of drugs which have received black-box warnings and drugs which have been withdrawn due to post-market safety issues has revealed that mitochondrial dysfunction is a key factor in hepatotoxicity. The mechanisms by which drug-induced mitochondrial dysfunction occurs and the high-throughput in vitro screening methods being used to identify this type of impairment, in order to reduce compound attrition, will be discussed.
10:10 Networking Coffee Break with Exhibit and Poster Viewing
10:50 Sponsored Presentation (Opportunities Available)
11:20 Biomarkers in Early Stage Drug Development: Challenges and Opportunities – The Changing Face of Early Drug Development at AstraZeneca
Victor Sandor, M.D., Global Product Head, Executive Director, Oncology, Clinical Development, AstraZeneca Pharmaceuticals (Tentative)
The talk will focus on the practical use of biomarkers in drug development. It will focus on specific examples of how various biomarkers that cover the spectrum of biological changes that make cells malignant (growth factor signalling, angiogenesis, metastasis and genetic plasticity) were used in real development programs at AstraZeneca to make decisions to either move a drug forward or to stop development. In addition, issues related to how these markers were developed and validated will be reviewed.
11:50 Panel Discussion with Speakers
12:10 pm Lunch Presentation (Opportunity Available) or Lunch on Own
1:25 Chairperson’s Remarks
1:30 Translational imaging in Drug Discovery and Development
Daniel Bradley, Ph.D., Scientist II, Millennium The Takeda Oncology Co
Imaging is becoming well recognized as a keystone technology internally or collaboratively for many large pharmaceutical companies across multiple disease functions in early discovery molecular imaging and translational biomarker development. DCE- & DWI- MRI and FDG- & FLT- PET are well recognized examples of translational imaging biomarkers deployed in oncology drug discovery and development. The use of translational imaging in pre-clinical drug discovery is more so to qualify the value of the biomarker than discontinue a therapeutic; or at least contextualize the importance of the imaging biomarker when used to measure the effect of a novel therapeutic development. The presentation will focus on a number of examples where imaging in drug discovery can aid in clinical deployment and a number of the factors that must be considered when appropriately ‘translating’.
2:00 Mechanisms to Reduce Cost in Getting to Clinical Proof-of-Concept
Stephen Donahue, M.D., Senior Vice President, Clinical Development, Ore Pharmaceuticals
A number of options have evolved over the years that provide the drug developer effective routes to clinical proof-of-concept. Some of these can save the sponsoring pharmaceutical company resources.
We will examine some of the basic routes to cost reduction involving:
Focus on critical path; appreciated but difficult to implement
Selection of study designs; re-examination of the pilot study
Selection of indication; consideration of the demonstration indication
Relieving the proof-of-concept teams of some of the practices of phase 3 programs
Partnering before proof-of-concept
These routes will be presented with attention to items of interest to the small company or the large pharmaceutical company.
2:30 Networking Refreshment Break with Exhibit and Poster Viewing
3:10 Accelerating Proof-of-Concept Round Table Discussions
Moderator: Terri A. Roberson, MT (ASCP), M.B.A., Manager, Chorus Operations, Eli Lilly and Company
Additional Moderators to be Announced
3:40 Accelerating Proof-of-Concept – Report out of Round Table Discussions
Terri A. Roberson, MT (ASCP), M.B.A., Manager, Chorus Operations, Eli Lilly and Company
4:10 Towards a Mechanism of Action Informed Accelerated POC Model
Rumin Zhang, Ph.D., Senior Principal Scientist, New Lead Discovery, Schering-Plough Research Institute
Proof-of-Concept (POC) studies must be designed with mechanism of action (MOA) in mind. We will explore the following questions: Can a biological target be kinetically differentiated from collaterial targets in an open system such as the human body? Are test compounds with a relevant range of kinetic attributes available to test the question of druggability? What mode of binding best address the underlying pathology?
4:40 Close of Accelerating Proof-of-Concept Conference
For more information, please contact:
Christina Lingham
Cambridge Healthtech Institute
250 First Avenue, Suite 300
Needham, MA 02494
Phone: 781-972-5464
Fax: 781-972-5425
E-mail: clingham@healthtech.com
For partnering and sponsorship information, please contact:
Arnie Wolfson
Business Development Manager
781-972-5431
awolfson@healthtech.com