Agenda
DAY ONE - May 1st, 2008
7:30-8:30 - Workshop Registration and Breakfast
8:30 - Pre-Conference Workshop: Best Practices in Licensing and Deal-Making. View more info →
10:15-10:30 - Networking and Refreshment Break
12:00 - Workshop concludes / Conference registration begins
12:00 - 1:00 - Lunch for Workshop participants
1:00 - Chair’s opening remarks
Howard Wachtler, CEO, Actinium Pharmaceuticals
1:10 - After the Dive: Surviving and Thriving in New Pharmaceutical Industry Paradigm
Andrew Forman, Financial Analyst (#1 Pharmaceutical Analyst Wall Street Journal Survey, Forbes May 2006”)
1:40 - Electroporation as a Clinical Stage Technology for Intracellular Delivery of Nucleic Acids
In general, clinical trials using conventional injection of DNA-based products have produced disappointing results, likely due to inefficient intracellular delivery of the DNA. In comparison to conventional injection, electroporation dramatically enhances both the delivery of nucleic acids into cells and the resultant biological responses. Electroporation mediated DNA delivery is currently being evaluated in a number of clinical trials for DNA vaccines. This session will cover the following topics:
- Ability of electroporation to enhance intracellular DNA and RNA delivery, and resulting biological responses
- Overview of clinical electroporation technologies, including Ichor’s TriGridTM Delivery System
- Current clinical trials utilizing electroporation to deliver DNA vaccines
Claire Evans, Ph.D., Director Protein Therapeutics, Ichor Medical Systems
2:10 - Novel Injectable Delivery Systems for Biologicals
Biological drugs represent one of the most important and fastest growing segments of the pharmaceutical industry. Promising areas for future growth are monoclonal antibodies and vaccines. These agents present considerable delivery challenges. A number of delivery technologies are being investigated that may facilitate administration and improve outcomes.
- New Self-Injecting systems
- Subcutaneous Delivery of Monoclonal antibodies and other drugs requiring high volumes
- Enhancing vaccination using microneedles.
- Lymphatic delivery using microneedles for potential treatment of diseases such as metastatic cancers.
Philip G. Green, Ph.D., Director Alternative Delivery Systems, BD-Medical Pharmaceutical Systems
2:40 - The Staccato System: Inhaled Drug Delivery for Rapid Relief of Acute Systemic Medical Conditions
The Staccato system technology is the basis of a novel drug delivery via inhalation for treatment of acute and intermittent conditions, such as pain, migraine, panic and agitation. The technology enables precise, drug delivery via the lungs to the systemic circulation, thus generating a rapid onset of effect, comparable to IV. Its core principal is thermal transformation of pure, excipient-free drug into an aerosol that is inhaled by a patient. The Staccato system technology has been proven in 6 product candidates to date, with the most advanced program in Phase III clinical trials.
This session will cover the following:
- Key features of Staccato technology
- Differentiation of Staccato system technology vis a vis other inhalation and drug delivery systems
- Technical, strategic and commercial assessment of compounds for Staccato technology
Tatyana Naranda, Director Business Development, Alexza Pharmaceuticals
3:10 - 3:30 - Networking and Refreshment Break
3:30 - Protecting Intellectual Property in the Changing Patent Landscape
In the past few years, there has been much public debate about the quality of patents, and pressure to make patents harder to obtain and enforce based on the perception that competition is being stifled by worthless patents. Recently, the Supreme Court, the Federal Circuit (the patent appeals court), the legislature, and the Patent Office have modified or sought to modify long standing patent law and procedure in apparent response to this pressure. Both class action lawyers and the government are aggressively suing pharmaceutical companies for antitrust violations arising from alleged “sham” patent litigations and settlement agreements.
This presentation will summarize recent court decisions and pending legislation and regulations that may significantly alter what is patentable and how patents are obtained, as well as the relationships of licensors and licensees. As a result of the changing landscape, life science companies need to reevaluate how they protect their intellectual property.
Topics to be covered in this presentation include:
- Recent decisions on patent validity and the effect on drug delivery patents
- Recent decisions affecting license agreements
- The patent application process under the pending Patent Office rules
- Proposed patent reform legislation in Congress
- Listing drug delivery patents with the FDA: to list or not to list?
Janet Linn, JD, Intellectual Property Expert
4:00 - Molecular Imaging and Drug Delivery
This presentation will outline the basics of molecular imaging and how this process can aid with the drug delivery and discovery process.
This presentation will cover the following key areas:
- Lesion detection (diagnosis)
- Patient stratification
- Dose scheduling (pharmacokinetics)
- Treatment monitoring (pharmacodynamics)
- Drug screening/optimization
Shawn Chen, Ph.D., Assistant Professor, Stanford University School of Medicine
4:30 - Understanding Regional Bioavailability as a Driver for Realising the Success and Value from Modified Release Oral Formulations
The physicochemical properties of many new chemical entities present significant biopharmaceutical challenges for oral drug delivery. Furthermore, patient-friendly dosage regimens are now seen as a requirement for these new products as well as a driver for traditional life cycle management activities. The mapping of regional bioavailability in humans has been proven as a pivotal tool in providing data-driven decisions for the selection of optimal formulation strategies to achieve desired in vivo performance, thereby offering substantial advantages over historical techniques such as preclinical and in vitro models.
Evidence will therefore be presented to show how data from human drug absorption (HDA) studies can be utilised to:
- Confirm the potential oral developability of the candidate molecule vrs the proposed target product profile
- Recommend specific Modified Release (MR) formulation technologies to achieve a desired pharmacokinetic (PK) delivery profile
- Generate a proactive “MR formulation road-map” to expedite initiation of PoX studies
- Explore enhanced IP protection, particularly in light of MR formulation “obviousness” challenges
Peter Scholes, Ph.D., VP Pharmaceutical Sciences, Pharmaceutical Profiles Ltd.
5:00 - Wine & Cheese Reception
DAY TWO - May 2nd, 2008
7:30-8:15 - Breakfast
8:15 - Chair’s Opening Remarks
Howard Wachtler, CEO, Actinium Pharmaceuticals
8:25 - The Competitive Landscape of Nanotechnology in Drug Delivery
Many nanobiotech innovations are directed at improving the delivery of active molecules, with academics, startups, and corporations pursuing these new avenues. These new technologies often blur the line between drug and device, meaning that both pharma companies and devicemakers will be impacted, whether by the actions of an existing or new competitor, a new market threat or opportunity, or new regulation. In this presentation, Mark Buenger will present an overview of the companies active in this emerging space today, including their technical, operational, and IP positions.
Mark Bunger, Research Director, Lux Research
8:55 - Delayed Pulse Delivery as a Method to Overcome First Pass Effects
Oral drug delivery is the preferred route for administering drugs and extended release formulations are ideal because they give constant blood levels. However some drugs have a substantial first pass metabolism and extended release formulations exacerbate this problem. Additionally, many drugs are only absorbed in the upper gastrointestinal tract so the dosage form must be retained in this area to be effective. This presentation will focus on a recently developed gastric retentive pulsatile delivery system to over come both the first pass metabolism and the absorption window problems.
Verne Cowles, Ph.D., Sr. Director of Preclinical Studies, Depomed
9:25 - Delivering the Payload: Turbo Charging the mAb
Radiopharmaceuticals have long been used as diagnostics as well as therapeutics in different forms of cancer. Only in recent years has the real possibility emerged to use mAbs and potentially peptides as vehicles to deliver radioisotopes to cancer cells. This emerging field of Radioimmunotherapy (RIT) has already resulted in approvals of 2 drugs based on beta isotopes. Alpha emitting isotopes have distinct advantages over beta emitters. Studies with two alpha isotopes Bi 213 and Ac225 have shown very favorable results. These isotopes form the basis of the API technology platform.
This session will cover the following:
- Key features of API technology
- Differentiation of API with other radiopharmaceuticals
- Future directions of API technology
Howard Wachtler, CEO, Actinium Pharmaceuticals and Dragan Cicic, MD, Medical Director, Actinium Pharmaceuticals
9:55-10:10 - Networking and Refreshment Break
10:10 - Collaboration and Licensing as an Approach to Solving Drug Delivery Problems
Merck has been very active over past 8 years in the licensing arena. A brief overview of Merck’s current pipeline and our research and licensing strategy. The presentation will next focus on Merck’s areas of interest in the drug delivery area. Highlighted will be Merck’s drug delivery partnerships in the past as well as two recent case studies: our collaboration with SurModics in the ophthalmic area for their I-vation platform technology and our agreement with MicroDose Technologies regarding their dry powder inhaler with Merck’s proprietary products. A discussion around Merck’s commitment to RNA Therapeutics will complete the presentation. Merck’s 2006 acquisition of Sirna Therapeutics will be the focus, along with the needs for drug delivery technology in this area.
Yael Weiss, MD, Ph.D., Director Licensing and External Research
10:40 - Transdermal Delivery: Reaching Beyond the Stratum Corneum Through RF MicroChannels
Transdermal delivery, although a preferable route of administration, has always been limited in its applications and growth due to an effective barrier- the Stratum Corneum, the most upper layer of the skin. The ViaDermTM system, a novel, active transdermal drug delivery system is designed to overcome the Stratum Corneum barrier by opening microscopic pores in a precise, effective, and reproducible manner. The pores created enable the systemic delivery of a wide range of molecules, including peptides and proteins.
In today’s pharmaceutical’s pipeline, major growth is expected to come from peptides and proteins based therapeutics and a delivery method which would offer safe and painless administration of such therapeutics is bound to be implemented into many therapeutic compounds, improving patients disease management and quality of life. The ViaDerm was developed to answer this apparent need. It has a significant potential to offer an alternative delivery route which entails many advantages: It avoids the need for injections, it offers a control over the blood drug profile, and increases drug safety. This system is low cost, small, user friendly and aimed to be self used at home especially by chronically ill patients. Examples of products being developed based on the ViaDerm system and the technologies underlying the system will be presented.
Judith Kornfeld, VP Business Development, Transpharma Medical
11:10 - Waiting to Inhale: The Prospects for Inhaled Delivery Technologies
Despite the recent terminations by Pfizer/Nektar and Novo Nordisk of their respective inhaled insulin programs, many inhaled drug delivery programs continue to advance. This presentation will provide an overview of these programs and a critique of inhaled delivery technologies and commercialization strategies. At the end of the presentation and discussion, the audience will have an understanding of the opportunity for inhaled drug delivery and which technologies and strategies are best poised to capitalize on that opportunity.
Matthew Kim, JD, Founder, Vice President Licensing and Business Development, Aerovectrx
11:40 - Prefilled Products: Platforms for Today and the Future
Prefilled products have gained significant acceptance in the market and are projected to continue to grow. Drivers for this growth include product differentiation, the impact of the age wave on medical facilities and staff, and the increased trend in home administration. The session will examine the current platforms, their flexibility, the need for improved cycle time for innovation and cost reduction, and a glimpse of what future platforms may have to evolve to in order to meet industries needs and customer’s requirements.
Dr. Ron Forster, Director of Advanced Product and Materials Engineering, Amgen
12:10-12:55 - Lunch
12:55 - Keys to a Successful and Profitable Contract Manufacturing Partnership
As drug manufacturers plan for the commercialization of their products, they will need to determine how their drugs will be delivered to the patient. Many drugs will require a separate delivery device. These devices may range from a simple syringe, to a complex device designed to deliver one or multiple doses to very specific sites in the body. In many cases, drug manufacturers will partner with a contract manufacturer to build these devices. Choosing the right contract manufacturing partner may make the difference between successful commercial launch or failure. The numerous considerations to take into account include: quality and regulatory status, core competencies, design for manufacturability, scalability and effective use of capital.
Topics to be discussed in this presentation include:
- Design and Development
- Manufacturing and Assembly
- Supply Chain Management
- Packaging and Sterilization
- Order Fulfillment
Walter Gacek, Director of Business Development, Accellent
1:25 - Intracellular Drug Delivery Mediated by High Intensity Focused Ultrasound (HIFU)
Cell and tissue specific intracellular drug delivery in vivo is now possible, mediated by ultrasonic energy applied externally to specific regions of the patient. This is accomplished first by using novel, self-assembling polymeric nanocarriers to safely deliver therapeutics to the target site of the patient. Because of their multifunctionality, these therapeutic carrying vesicles may be passively or actively targeted using, for example, ligands such as antibody fragments or nucleic acid aptamers. Once the therapeutic containing nanocarriers are in treatment area, drug delivery commences following cell membrane disruption and nanocarrier disassociation by high intensity focused ultrasound (HIFU). Thus, non-invasive sonic energy being applied to the patient, directly and indirectly results in both therapeutic release and drug delivery to specific organs and tissues inside the body of the patient. Preferred therapeutics for use with this new drug delivery platform include the modern therapeutics of biotechnology, such as: nucleic acids, proteins, peptides, and other compounds.
This presentation will include the following:
- An overview of ultrasound’s use in medicine including diagnostics and drug delivery applications.
- A discussion of the complex and competitive nature of ultrasound intellectual property
- An overview of acoustic cavitation, which is integral to the successful development of this new technology
- A summary of the future therapeutic applications for ultrasound drug delivery
Charles Hardy, President, Biovaluation and Analysis
Dr. Hardy is the sole inventor on a large number of U.S. and international patent applications associated with ultrasonic intracellular drug delivery in vivo, including acoustically-responsive therapeutic carrying vesicles, instrumentation and other methods and systems.
1:55 - Needle-Free Injection Technology for Small and Large Molecules; DosePro™ - a Novel Pre-filled, Single Use, Disposable, Needle-free Drug Delivery System
Development of protein based injectable therapeutics is on the increase as is the need to commercialize these products in innovative patient preferred delivery systems that differentiate drugs in competitive markets. This presentation by Zogenix, Inc., a CNS and pain specialty pharmaceutical company will cover the following topics in relation to its’ single use, pre-filled, disposable, needle-free drug delivery technology - DosePro™:
- Development update on lead program; sumatriptan DosePro for the treatment of migraine and cluster headache.
- Market research and market needs
- Regulatory status and clinical data
- Benefits to patients and partners
- Manufacturing readiness
- Potential advantages over prefilled needle based systems
- Platform application - delivery of protein therapeutics and viscous formulations.
Jonathan M. Rigby, Co Founder and Vice President Business Development, Zogenix Inc.
2:25 - End of Conference