tuesday october 16, 2018 - asdevents/ tuesday october 16, 2018 12:15 networking lunch priority...

DAY ONE / Tuesday October 16, 2018

12:15 Networking Lunch

Priority Review Vouchers Track

Strategic Partnerships for Drug Repurposing Track

Neglected Tropical Diseases Track

Investment and Business Models for Drug

Development Track

1:15 Opening RemarksDavid Ridley, Ph.D., Faculty Director, Health Sector Management, Fuqua School of Business, DUKE UNIVERSITY

Opening RemarksBruce Bloom, CEO, CURES WITHIN REACH

Opening RemarksMarco Biamonte, President, DRUGS & DIAGNOSTICS FOR TROPICAL DISEASES

Opening RemarksWilliam D. Paiva, Ph.D., Executive Director, Centre for Health Systems Innovation (CHSI), OKLAHOMA STATE UNIVERSITY; Managing Partner, OKLAHOMA LIFE SCIENCE FUND

1:30 Manage the Complexities of Clinical Trials for PRV-Eligible Products• Discover the lessons learned

from conducting clinical trials for Ebola, Chikungunya and Zika during outbreaks

• Understand how thechallenges of these trials differ from conducting research in well-equipped, experienced sites

• Review financial and logisticalconsiderations associated with conducting research in developing countries

Ted FitzGerald, M.S., Director, Global Research Services, FHI360

Strategic Partnerships for Open Clinical Innovation Successes • Address the value proposition

for repositioning through Open Innovation means

• Discuss what happenswhen you open the doors to collaboration

• Touch on how we can get anew view of old compounds through informatics approaches

• Understand the key to successin industrial-academic partnerships

Leslie Cousens, Director, Translational Medicine Emerging Innovations, ASTRAZENECA

Top Considerations to Reassess Global Health Strategies to Build Sustainable Infrastructures That Combat NTDs• Identify models of treatment

access expansion through capacity building

• Assess the long-term impactof donation programs on the allocation of resources to explore new licensing or pricing models for existing drugs

• Strengthen global strategyprograms that address the substantial NTD burden implement models that aim to utilize sustainable plans

Bill Lin, Ph.D., Global Program Leader, Neglected Tropical Diseases (retired), JOHNSON & JOHNSON

Capital Raising for a Pre-NDA PRV Candidate • Assess the development

pathway of a product that addresses an unmet medical need

• Gain an overview of thecapital raising activities and timeframe

• Review the global healthimpact obligations within the agreements

• Understand the post-PRVplans to build a sustainable global health biotechnology company

Curt H. LaBelle, M.D., MBA, President, GLOBAL HEALTH INVESTMENT FUND

8:15 Registration Opens and Continental Breakfast 9:00 Opening Remarks

9:15 A Public Policy Perspective on Market-Shaping Considerations for Emerging Infectious Diseases• Compare the systems in place for expedited access to vaccines and available therapies during public health emergencies• Evaluate existing domestic and international policies that maximize market access in emerging markets for infectious disease

products• Consider innovative business model proposals for emerging infectious diseases that leverage internal regulatory expertise and

strategic funding optionsJohn Billington, Director U.S. Public Policy Vaccines, GLAXOSMITHKLINE

10:00 Review of Advancements and Challenges in the Clinical Development of Therapies for Those With Unmet Medical Needs • Understand the impact that the current global regulatory environment has on the development of drugs for neglected diseases• Outline the market access forces driving regulatory innovations that impact clinical development• Understand how regulatory agencies have provided support to advance the control of some NTDs like malaria and TBLeonard Sacks, M.D., Associate Director for Clinical Methodology, Office of Medical Policy, CDER, FDA

10:45 Networking Break11:15 Generic Drug Incentives That Can Drive More Treatments to Patients Faster

• Grasp how recycling of CONSUMER-USED materials has been a part of life for centuries and how technology has changed it• Understand how new technology, economic incentives, and government requirements have repurposed therapy development• Describe the future of drug repurposing research and discover how to find new drug targets quickly and more efficientlyBruce Bloom, CEO, CURES WITHIN REACH

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Development Track

2:15 Overview of the Recent Pandemic and All-Hazards Preparedness and Advancing Innovation Act of 2018 (PAHPA Reauthorization) and Its Implications for MCM PRVs• Examine and discuss the

current landscape for medicalcountermeasure developmentand procurement with the U.S.government

• Understand the implicationsof the PAHPA Reauthorizationon medical countermeasurePRVs

• Discuss future opportunitiesto incentivize the developmentof medical countermeasures

Patrick Lucy, Chief Business Officer, PFENEX

Using AI and Drug Repositioning to Accelerate Solutions for Vulnerable Populations, Vulnerable Patient Populations have Zero Luxury of Time• Address every new tool,

approach, and novel operatingframeworks to advancesolutions time and cost-effectively, given the hugequantity of healthcare dataworldwide, AI is central todeveloping new solutions

• Understand AI in combinationwith drug repositioning, whichaccelerates the developmentof new therapeutic entitiesfaster than any otherdevelopment process, westand at the dawn of anew operating frameworkto develop solutions forvulnerable populations

• Hear specific case studies of the use of the Project Prodigy AI

Aris Persidis, President and CoFounder, BIOVISTA

Regulatory Considerations That Are Essential to Overcome Global Health Concerns in the Developing World • Address the challenges of

providing LMIC access toinnovative solutions

• Review current and ongoingpublic sector engagementprograms

• Industry methods that supportthe building of an independentcapacity to execute publichealth initiatives

• Discuss the potential ofthe market value of NTDvaccines as the foundationfor sustainable businessmodel for future innovations inemerging markets

Jean-Antoine Zinsou, Head of Global Public Affairs, New Vaccines, SANOFI PASTEUR

Insights From a PRV-Centered Investment Deal Between Private and Non-Profit Entities• Gain an overview of the terms

and structure of a PRV-baseddeal to fund development

• Describe the nature, timeline,and challenges associatedwith arranging such a dealand the concerns aboutongoing developments anduncertainties in this dynamicspace

• Discuss how this investmentis enabling the developmentof a new, urgently neededdrug and how it’s partners aimto have this new drug reachhundreds of millions of peopleliving in low-income countries

Scott Requadt, J.D., MBA, Managing Director, CLARUS

3:00 Networking Break

3:30 Valuation Considerations for PRVs• Assess the current supply

and demand for PRVs in themarketplace

• Evaluate whether publicperception of PRVs hasshifted and if that will impactawarding habits and futurelegislation

• Determine how geographicpricing (U.S./EU/ROW)corresponds with regionaldemand

• Review tax consequencesaround PRV sale structuring

Prashant Raykar, Senior Manager, Corporate Development, EMERGENT BIOSOLUTIONS

Explore New Therapies to Put Autoimmune Diseases Into Long-Term Remission • Hear how we have developed

a treatment to prevent and/or reverse the cognitiveimpairment due to MultipleSclerosis

• Define a novel therapeuticstrategy for the treatmentof cognitive impairment inMS, inhibition of glutamatecarboxypeptidase II (GCPII)

• Study recent clinicaldata collected at theJohns Hopkins Hospitalin MS patients and theirperformance on a battery ofcognitive tasks

• Examine a mood-trackingtechnology to help track yourdaily mood

Adam Kaplin, Assistant Professor of Psychiatry and Behavioral Sciences, JOHNS HOPKINS HOSPITAL

Global Health Partnerships Models That Encourage Sustainable Development • Implement alliance

development strategiesacross the private sector,multilateral and NGOs

• Consider policy frameworksfor public and private sectorengagements and understandcurrent policy gaps thatencourage sustainableprogram development

• Develop programs thatleverage resources to balancesocial equity with businessgoals

Philip E. Sanderson, Ph.D., Project Leader and Manager, Therapeutics for Rare and Neglected Diseases, NCATS

Impact Investing for Research Through the Leukemia & Lymphoma Society• Gain an overview of the history

of the Leukemia & LymphomaSociety

• Understand the mechanismsof how we fund

• Gain insights into the diligenceprocess, how to manage riskand what we look for in aninvestment opportunity

• Discuss successful examplesof collaboration

Kenneth LaMontagne, Ph.D., MBA, Vice President, Research, Therapy Acceleration Program, THE LEUKEMIA & LYMPHOMA SOCIETY

DAY ONE / Tuesday October 16, 2018







Development Track

4:15 Panel: The Value of Priority Review Vouchers in the Marketplace• Discuss historical transactions

and trends in pricing• Explore changes in supply and

demand effects• Share thoughts on voucher

value from the buy and sellside

• Forecast future prices forPRVs

Jennifer R. Cook, Manager— Valuation and BusinessAnalytics, BDOPrashant Raykar, SeniorManager, CorporateDevelopment, EMERGENTBIOSOLUTIONS

Case Study: Repurposing FDA-Approved Drugs for Neurodegenerative Diseases: From Mechanism to Medicine• Understand the common

mechanisms ofneurodegeneration and cancerin repurposing drugs

• Elucidate drug action at thecellular and molecular levels

• Discuss the impact of arepurposed drug on patientsincluding rare and orphandiseases

Charbel Moussa, Director,Laboratory for Dementia andParkinsonism, GEORGETOWNUNIVERSITY MEDICALCENTER

Novartis Institute of Bi-omedical Research Case Study: Strides in Drug Development to Combat Cryptosporidiosis and Leishmaniasis • Review the Leishmaniasis

program milestones andaccomplishments inidentifying ways modulatethe activity of a conservedparasite target

• Learn about the expandingdrug discovery effortsbeing made to identifynovel therapies to fightcryptosporidiosis

• Hear about the challenges ofnavigating the IND pathwayswhen working with non-ICH regulatory bodies tomake these novel therapiesaccessible

Richard Colvin, M.D., Ph.D.,Executive Director, SeniorTranslational Medicine Expert,NOVARTIS INSTITUTE FORTROPICAL DISEASES (NITD)

Funding Flexibly and Col-laboratively in a Healthcare System Focused on Cover-age and Reimbursement • Review various models

and structures thatbiopharmaceutical companiescan utilize to secure programfunding

• Discuss how alternativefunding can be aninnovative approach tosharing the inherent risksof drug development whileprogressing programs toultimately benefit patients

• Explore some advantagesto risk sharing approaches,potential funding models,how risk is viewed andassessed, and criteria usedfor determining asset fundingdecisions

Brian Axe, Principal,NOVAQUEST CAPITALMANAGEMENT

5:00 Day One Sessions Conclude

5:30

7:30

CureAccelerator Live! This is a philanthropic pitch competition that gives attendees the chance to find and fund the next

breakthrough treatment at a unique networking event for researchers, clinicians, patient groups, biomedical industry professionals, and funders. This opportunity to help harness the power of

repurposing research and help bring groundbreaking treatments to patients in need. The finalists will be announced pending review of submitted proposals.

Complimentary with conference registration, but sign-up is required to reserve your spot.

Join us as we announce the winner of the

2018 Cure Accelerator Live! program



DAY TWO / Wednesday October 17, 2018Priority Review Vouchers

TrackStrategic Partnerships for Drug Repurposing Track



Development Track

8:00 Continental Breakfast

9:00 Opening RemarksDavid Ridley, Ph.D., Faculty Director, Health Sector Management, Fuqua School of Business, DUKE UNIVERSITY

Opening RemarksBruce Bloom, CEO, CURES WITHIN REACH

Opening RemarksMarco Biamonte, President, DRUGS & DIAGNOSTICS FOR TROPICAL DISEASES

Opening RemarksWilliam D. Paiva, Ph.D., Executive Director, Centre for Health Systems Innovation (CHSI), OKLAHOMA STATE UNIVERSITY; Managing Partner, OKLAHOMA LIFE SCIENCE FUND

9:15 TPOXX®: A Case Study in Medical Countermeasure Development and Stockpiling• Review the development and

supply of a novel antiviral drug for treatment of smallpox infections

• Evaluate the opportunitiesand challenges of partnered development with the U.S. Government for a critical public health need

• Provide an overview of thebusiness opportunities and challenges for Biodefense products

Phillip L. Gomez, Ph.D., CEO, SIGA TECHNOLOGIES, INC.

Combining Chemogenomics and Phenotypic Screening With Machine Learning to Rapidly Identify Drug Targets and Anti-Targets • Explore studies have

demonstrated that drug sensitivity information at the level of the proteome is not available at the level of the corresponding DNA or mRNA

• Hear a discovery platformfor Identification of Drug Targets and Anti-targets by Cellular and Molecular Cross-referencing (idTRAX)

• Discuss a custom developedmachine learning algorithm is then used to relate the resultant cellular phenotypes to the compounds’ kinase inhibition profiles

Hassan Al-Ali, Research Assistant Professor, Department Of Neurological Surgery, UNIVERSITY OF MIAMI

The CURE Platform: An FDA and NCATS Collaboration to Capture and Organize Drug Repurposing Option For NTDs• Hear and overview of

repurposing’s promise for neglected infectious diseases

• Review repurposing examplesin the preclinical versus clinical space

• Discuss CURE platformlimitations and its potential to inspire formal drug development innovations for tropical infectious diseases

Heather Stone, Public Health Analyst, FDA

An Overview of Fifteen Years of Experience of a PDP, DNDi, to Address Disease Burdens on Developing Nations• Understand the role of DNDi

and how it contributes to the research and development of treatments for neglected diseases

• Review case examples ofsuccessful collaborations with industry partners

Serge Sagodira, Ph.D., Director of Business Development and Legal, DRUGS FOR NEGLECTED DISEASES INITIATIVE (DNDI)



DAY TWO / Wednesday October 17, 2018Priority Review Vouchers

TrackStrategic Partnerships for Drug Repurposing Track



Development Track

10:00 Incentivizing Industry Investment in Medical Countermeasure Development• Provide background on

the role of private sector in medical countermeasure development

• Review why bioterrorism-typethreats are increasing and different incentive options that have been generated to attract investment

• Identify gaps in the literatureto better understand the relative strengths of industry partners and highlight new recommendations for leveraging industry involvement

• Explore how a morenuanced understanding of industry “players,” along with a portfolio of creative incentives, targeted at different kinds of companies, would be more effective than a “one-size-fits-all” approach

Rebecca Fish, Vice President, Marketing and Product Strategy, Biodefense Division, EMERGENT BIOSOLUTIONS

Panel: Drug Development Partnerships With Stakeholders • Clarity the partnerships

between life science companies government, non-profits and patient advocacy groups

• Identify some of the issuesand challenges surrounding drug repositioning

• Incentives from the FDA fordrug repurposing for orphan and pediatric diseases

• Understand a systematicapproach to evaluate drug candidates

• Piece together fundingfor smaller companies, partnerships

PanelistsBobbie Austin, Program Officer, Drug Development Partnership Programs, NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES, NIH Heather Stone, Public Health Analyst, FDA Zhichao Liu, Staff Fellow, NCTR, FDA

Strategies to Mitigate the Financial Risk of Novel Therapeutic Development of for Neglected Patients by Bridging Funding Gaps• Outline the market access

forces that drive pricing considerations and how drug developers can react appropriately

• Cultivate strategic partnerships that lessen the risk of investing in development for both parties

What to Do When Raising Capital Locally for Life Science Companies Is a Challenge• Learn how national and local

foundations can make seed investments in life science organizations using grant dollars through Program-Related Investments (PRIs)

• Gain an overview of theVenture Philanthropy category and examine organizational and management best practices

• Review local and nationalexamples of success

William D. Paiva, Ph.D., Executive Director, Centre for Health Systems Innovation (CHSI), OKLAHOMA STATE UNIVERSITY; Managing Partner, OKLAHOMA LIFE SCIENCE FUND

10:45 Networking Break

11:15 New Models for Innovation of Access• Explore novel routes

to guaranteeing access to PRV-eligible drugs

• Discuss how imperative itis to consider the needs of patients in endemic regions when constructing a market access plan

• Discover the different waysto accommodate fair pricing for populations that actually need these products

Forrest Cox, Co-Founder and SVP, Corporate and Business Development, AFFIGEN

Develop a Repositioning Strategy to Repurpose Drugs for New Indications • Learn about drug treatments

for post-acute/chronic neurorehabilitation needs

• Delve into some of theNeuroHealing clinical programs that are in the pipeline

• Discuss innovative drugtreatments for specialty indications based on neurologically active compounds

• Review some trends andcomparisons of development paths for CNC focus

Neal M. Farber, CEO, NEUROHEALING PHARMACEUTICALS, INC.

Commercialization of Cost-Effective Diagnostics for Targeted Mass Drug Intervention for River Blindness Patients• Establish a testing strategy

to map the distribution of Loa loa infection in patients also infected with river blindness

• Discuss the low-costimplications of designing a platform that can be adapted to multiple diseases to avoid adverse events

• Construct a feasible trial planthat incorporates regional global health initiatives skill in market access strategies for underserved populations globally

Marco Biamonte, President, DRUGS & DIAGNOSTICS FOR TROPICAL DISEASES

Case Study: Adapting Investment Strategy to Business Model• Examine the different types

of investors and what they expect from traditional investments

• Learn about the benefits ofworking with patient advocacy groups and how they can raise political awareness to help define guidance for drug development

• Understand the challengesof raising awareness for a disease that lacks a definitive means of diagnosis

Amrit Shahzad, MBBS, MBA, Industry Alliance Officer, UC SAN DIEGO






Development Track

12:00 Extensions of the Priority Review Voucher Program Concept• Understand how the PRV

program creates value through faster regulatory review

• Consider how the value ofa PRV can be leveraged to encourage innovation for underserved populations

• Explore how the sameprinciple has been and can be applied in other settings, for example:

• United States Patent andTrademark Office: Patents for Humanity

• United States EnvironmentalProtection Agency: Vector control

• Europe: Regulation andreimbursement

David Ridley, Ph.D., Faculty Director, Health Sector Management, Fuqua School of Business, DUKE UNIVERSITY

Advance Precision Medicine Through an Understanding of Current and Future Strategies • Understand biomarker-based

repositioning in precision medicine (personalized medicine)

• Touch on systematicrepurposing approach and how to integrate with precision medicine

• Establish the importance ofdrug repurposing in the era of medicine

• Finding economic incentivesfor repurposing and drug pricing

• Explore bioinformatics forrepurposing in rare diseases

Mark Hasleton, Senior Director, Global NTE Pipeline Manager, TEVA PHARMACEUTICALS

DNDi Drug Discovery Booster Program: An Innovative Product Development Partnerships Spanning the Biomedical Landscape • Understand the impact of

strategic product development partnerships to accelerate innovation of novel NTD therapies

• Discuss the role PDPs play indeveloping relationships with endemic regulatory authorities that facilitate clinical work with neglected populations

• Develop partnerships withendemic country institutions to implement post registration commitments and facilitate policy adoption

Anita Staud, Senior Manager Business Development, DRUGS FOR NEGLECTED DISEASES INITIATIVE (DNDI)

Accelerate Life-Changing Breakthroughs to Cure, Prevent and Treat Type 1 Diabetes and Its Complications • Gain an overview of JDRF’s

funding mechanism and discovery and development partnerships program

• Discuss key considerations forselecting the right partners

• Examine the evolving businessmodel and the establishment of a scalable, mission-driven venture philanthropy platform exclusively focused on investment in early stage commercial opportunities

Michael Batten, M.D., MBA, Director, T1D Fund, JDRF

1:00 Networking Lunch

2:00 Closing Plenary Panel Collaborative Partnerships That Enable Multi-Phase Development Therapies for the Greatest Public Health Concerns • Gain an overview of how companies of various structures like PDPs, nonprofits, and industry collaborate to accomplish a

shared goal • Promote balanced and productive relationships with social enterprises• Build drug development programs that leverage private and public sector resources to balance social equity with business goalsModeratorBruce Bloom, CEO, CURES WITHIN REACHPanelistsWilliam D. Paiva, Ph.D., Executive Director, Center for Health Systems Innovation (CHSI), OKLAHOMA STATE UNIVERSITY; Managing Partner, OKLAHOMA LIFE SCIENCE FUNDAdam Kaplin, Assistant Professor of Psychiatry and Behavioral Sciences, JOHNS HOPKINS HOSPITALLeslie Cousens, Director, Translational Medicine Emerging Innovations, ASTRAZENECAZhichao Liu, Staff Fellow, NCTR, FDARichard Colvin, M.D., Ph.D., Executive Director, Senior Translational Medicine Expert, NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH (NIBR)

3:00 Conference Concludes

DAY TWO / Wednesday October 17, 2018



tuesday october 16, 2018 - asdevents/ tuesday october 16, 2018 12:15 networking lunch priority...

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