Leaders in Pharmaceutical Business Intelligence (LPBI) Group

LIVE 11/16 8AM – noon The 12th Annual Personalized Medicine Conference, HARVARD MEDICAL SCHOOL, Joseph B. Martin Conference Center, 77 Avenue Louis Pasteur, Boston

Leaders in Pharmaceutical Business intelligence (LPBI) Group

Covering in Real Time using Social Media this Event on

Personalized Medicine

Aviva Lev-Ari, PhD, RN, Founder LPBI Group & Editor-in-Chief

http://pharmaceuticalintelligence.com

Streaming LIVE @ HARVARD MEDICAL SCHOOL,

Joseph B. Martin Conference Center

@pharma_BI

@AVIVA1950

 

November 16

#PMConf

Joseph B. Martin Conference Center
77 Avenue Louis Pasteur
Boston, MA 02115

7:00 a.m. — Registration and Breakfast

8:00 a.m. — Opening Remarks – [Personalized Medicine = PM]

• Edward Abrahams, Ph.D., President, Personalized Medicine Coalition
• PM offers a more efficient HealthCare: Gov’t, Academia, research, pharma and Patients collaborations = working together
• PMC goal – solutions in the field, stacks are high
• Corporate sponsors: Genetech-Roache, astellas, Intermountain Precision Medicine

8:05 a.m. — The Personalized Medicine Report

• William S. Dalton, Ph.D., M.D., CEO, M2Gen, Chairman, Personalized Medicine Coalition
• Five medicines in 2016 are defined as PM
• Efficiences in Care by Precision Medicine
• Sequencing, molecular medicine, Genomics in all Medical Record

8:15 a.m. — Keynote Speaker

• Greg Simon, Executive Director, Cancer Moonshot Task Force
• 400 people in Washington at the Launch of Moonshot, 7,000 in audio/video attendance around the US
• Video by all stackholders
• Joe Bidan, today we are more ready than one year ago – ergency of now – a challenge we are not willing to postpone
• Example CLL therapeutics today vs 20 years ago
• Precision Medicine is the technology & Big Dat aand Science and Survivorship; PM is about the Patient
• TASK FORCE on Cancer: added EPA, Council for the Arts, DoD
• Double impact – How, Partnerships
• VA – many Veterans with Cancers – Sequence effort, Watson-IBM, to review sequences from VA – new partnerships: IBM + VA – Walter Reed Initiative
• VA tishue bank with 20 Companies – new Partnership
• DoD and VA
• Private initiatives on Moonshot topics: American Cancer SOciety – does their own, Foundations, Academia,
• Health Systems, several not one — join together Dana Farbers, Sloan Katering
• Accessible Medications is very important for Patients
• The significance of 9 month to start changing the World

8:45 a.m. — Pioneering Precision: Charting a Course for Cutting-Edge Innovations

Many scientists believe innovations in personalized medicine are poised to yield major breakthroughs in coming years, but not all members of the health care system are clear on which research topics have the most potential. The participants in this panel will identify the most encouraging scientific directions for personalized medicine and point to the most promising topics for future research.

• Moderator: Stephen Eck, M.D., Ph.D., Vice President, Oncology Medical Sciences, Astellas Pharma Global Development

1. Oncology drugs were prescribed by histology no molecular diagnostics
2. 2015 – 25% of FDA approved medicines are PM
3. In 2016 – PM and molecular diagnostics – needs to be a SYSTEM not disjoint solutions
4. Value for Price, small indications, cost containment
5. Pricing treatment if several drugs are at use wihtout knowing the realtive contribution of each

 

• David Altshuler, M.D., Ph.D., Executive Vice President, Global Research and Chief Scientific Officer, Vertex

1. Prevent and modify the course, Human genetics and somatic genetics in cancer, discovery started with Mandelian, moved to somatic, now use of catelogue for common mutations,
2. Test existing hypothesis on pathphysiology: LDL and heart disease vs HDL – genetics variance for Heart attack – predictor not modifier
3. Predicting with Genome in PM, who respond to which medication
4. New Hypothesis for new Paradigms
5. Cyctic FIbrosis: Gene found, took a histology based disease CF – to foundational genomics – mutation in gene on cell surface – POTENTIATOR of opening the channel by a small molecule — these small molecule were developed in the last 15 years, follow Patients in Registries – reduction in rate of decline of lung function — Drug – is changing the course of the disease and intervention early
6. small molecule in combination into cells that have only one copy, in a dish – two medications in Phase II
7. Personalization is a statement of not ahving yet an effective therapy but it is identification of the Pathway
8. Common genotype – small groupsidentification
9. target diseases that will show doubling efficacy, curative therapies, new models: PENETRATION OF LYTHAL DISEASES
10. Annuity model only in the future the outcome allows measurement – REGISTRIES are very important and need be used for outcomes in the future

• Michael Panzara, M.D., Head of Neurology Franchise, WAVE Life Sciences

1. Synthesis of nucleaic acid focusing on neurology – STEREOCHEMISTRY
2. modify sulfor – phorphate new modification – mixure of isomeres stereo molecules – systhesize nuclaic acids
3. design nucleic acids
4. Pipeline: CNS, MUSCLES, EYE, Liver, Skin, GI
5. HD – Huntington’s Disease [Mutant HTT] – cognitive decline
6. WAVE approach: target only the mutant HTT while leaving wild-type intact – disease modifier
7. Targeted therpy  mHTT RNA – transcript of mutant, SNP
8. Complement activation assay vs HTT transcript – wtHTT: Patients with long CAG repeat associated with T isoform will be selected for trial.
9. DMD – Exom Skipping,vs Dysfunctional Splicing: Dystrophin (increase production) vs Vinculin

• Barbara Weber, M.D., Interim Chief Medical Officer, Neon Therapeutics

1. Neoantigen biology: Native antigens (targets are expressed in both tumor and normal cells vs Neoantigens (Targets are specific to each Tumor)

Checkpoint inhibitor drug class: Neoantigen-based Therapies may expand ACTIVITY of Checkpoint Inhibitor – Nature 2013

1. Ipilimumab in melanoma (NEJM 2014
2. Pembrolizumab in NSCLC, Science 2015
3. Pembrolizumab in colonorectal cancer, NEJM 2015

NEON: Personalized Neoantigens

• personalized CAR-T
• personalized Vaccines
• Epitope Librart – Epitope selection
• NT-001 PM Clinical Trial: combination of Neoantigen Vaccine wiht Checkpoint Inhibitor – PD-1
• Monitor Immune response after insertion of neoantigents

9:45 a.m. — Networking Break

10:15 a.m. — Money Talks: The Future of Investment in Personalized Medicinemolecular diagnostics

Innovation requires investment. During this discussion, a panel of diverse investors will illuminate the most promising business opportunities for advancing personalized medicine, focusing on both macro and micro environments while also discussing the barriers to investment and potential solutions for removing them.

• Moderator: Edward Winnick, Editor in Chief, GenomeWeb

1. Beyond Ocology

• Alexis Borisy, M.S., Partner, Third Rock Ventures

1. What excite now – Breakthrough therapies in the RIGHT Patient populations
2. FOundation Medicine – 1,000 Patients treated with FOundationOne across diagnostics and drug – genomics and disease and therapy in One
3. New Diagnostics company creation – is very hard, not many, only if it is an extraordinary compelling idea, If FDA approves and the Payers do not endorse
4. Therapeutics – have a solution, nucleaic based drug, gene editing, Payers are iterested, FDA is interested vs historical standdards, translational medicine – drug will drive diagnostics
5. Reimbursement, Capital mobilization, new molecular diagnostics, challenges: Technical risk Clinical Risk
6. Cut off for AstraZeneca vs BMS are different
7. number tumor cells vs penetration of tumor cells vs infiltration of immune cells
8. Merck  has a different strategy – precise indicator of I/O agents: Single vs. multiple combinations
9. WHen population of patients is identified well: understand response rate go for 80% response rate inside this population identified
10. beyond oncology MI and CVD, cardiomyopathy – genetic mutations, familial vs sporadic,
11. Immunology: Inflammation, innume conditions, understand what is the microtrend in subpopulations
12. Infectious diseases: organisms given narrow cast antibiotics and trade-offs PM antibiotics is a fundamental challengeresistence to antibiotics
13. Diagnostics in Cancer – a small fraction vs the cost of Oncology drugs
14. Longevity drive costs,
15. Physician diagnostics is critical

• Vamil Divan, M.D., M.B.A., Senior Research Analyst, Credit Suisse

1. BMS is a leader in immunotherapy and Roache as well

• Ryan Lindquist, M.B.A., Director, Investment Banking, Leerink Partners

1. Diagnostics and Precision Medicine
2. Diagnostics – reimbursement
3. winners and loosers in Diagnostics
4. Performance, cost reduction, Payers have rules of engagement
5. Differentiation of generic drugs: COST OF TREATMENT FO RDRUG $30,000 COST RUNNING TEST $200, Pharma was willing to cut the cost creatively
6. Drug pricing a point in investing in Biotech
7. Neuroscience: many drugs are not effication – swab of mouth saliva allows to determine which pshychtropic drug will work for which patient on psych therapies
8. Data companies with Regulatories
9. Payers: Kaiser – has Outcome data and cost data
10. Healthcare system: Payers and Delivery is separate
11. Patient advocacy on reimbursement is critical and DOctor education on new Diagnostics at Academic Centers

Questions from the audience:

1. Therapeutics: IP and reimbursement is straight forward
2. Diagnostics: No IP therefore reimburcement more complex
3. Data companies
4. Foundation Medicine: Reimbursement is a challenge – what should the Payer do vs the DIagnostics companies, regulatory, American Pathology Guidelines
5. Engagement Providers – what is their role?

 

11:15 a.m. — You + 999,999: How a One Million Person Cohort Can Pave the Way for Personalized Care

• Introduction: Paolo Narvaez, Ph.D., Senior Principal Engineer, Director of Engineering, Intel Corporation

1. 1. How to use technology for PM – Intel Life Sciences

    

• Keynote: Eric Dishman, Director, All of Us Research Program, National Institutes of Health

1. PM Precision vs Personalized Health
2. All of Us Research Program, National Institutes of Health funded – is PM 
3. Framework Like the Framingham Study
4. One million Patients,  – longitudinal: lifestyle, genes, environmental
5. HUGE data not a study for one disease,  – accelerate Science
6. Infectious,Neuro, mental, Heart& Lung, Musculo-Skeletal

• DIrect Volunteer
• Health Provider Org
• Enroll & Consent
• Surveys Journals
• Baseline Measrement
• Mission to accelerate Knowledge Turns & Breakthroughs
• Participation – Transformational Approach
• 1,000 vs One million
• Diversity: People , geography health Status,
• Data Types: Genomics, Claims – Data Access
• High schools, community colleges
• User-Centered approach
• gathering Input
• Personas: ready to go, determined, too much Govenment, No time, Suspitious
• Platform Innovation Mindset & Process –>> Innovation Fannel
• Baseline Physical Evaluation not a Physical Exam
• PPI – Participant Provided Information
• Mayo Clinic: Biospecimens: Blood and Urine

Research ROADMAP Workshops

• Near Term
• Mid Term
• Long Term

Kind of Attendees: experts network (50 organization, co-funders, advoccy

Biobank – 35Million vials

IT infrafaces–>> security,  Enrollement 1-800-

New Eng PM Consortium, Bosotn, MA

 

 

 

12:00 p.m. — Luncheon

 

 

 

#PMConf

SOURCE

http://www.personalizedmedicinecoalition.org/Conference/November_16_Program