Leaders in Pharmaceutical Business Intelligence (LPBI) Group

11/19/2015 8 a.m. Building a Personalized Medicine Company & Keynote: President, Worldwide R&D, Pfizer Inc. 11th Annual Personalized Medicine Conference, November 18-19, 2015, Harvard Medical School

Reporter: Aviva Lev-Ari, PhD, RN

 

Live Press Coverage in REAL TIME

Aviva Lev-Ari, PhD, RN

Director & Founder, Leaders in Pharmaceutical Business Intelligence, Boston

Co-Founder, GDE

 

 

8:00 a.m. – Opening

• Chromosome of 12q
• Genetics association study – Vitamin D Receptor Genetic Variants related to Asthma
• Respiratory infections
• Airway smooth muscle efffects
• Effects on developing lungs
• Vitamin D Intake by Pregnant Mothers — reduction of Asthma and wheezing in newborn
• Surfactant production and Vitamin D deficiency
• Studies of Prenatal Vitamin D on early life asthma and allergy
• Supplemental Mothers during Pregnancy with Vitamin D (75nmol/L vs control at ): 4400IU vs 400IU
• Pragnancy and Infant Characteristics of Mothers whose Children are at Risk for Asthma
• REDUCTION in Respiratory infections
• Effect the strongest at newborns at age 1
• Genomic Origins of Asthma
• Vitamin D – supplementation good effects on Cancer
• Ten years of studies — Genomics used for Public Health Policy formulation
• If Pregnant Mothers are smoking, the effect is reduced with supplementation of Vitamin C and D
• Folate pathway – Vitamin B6

8:30 a.m. Harvard Business School Case Study

Building a Personalized Medicine Company: What does it take?

• Genetics Diagnostic Lab (GDL) for Profit
• In 2013 Boston Children’s Hospital (BCH) spun off GDL
• BCH invested in- bud a Genetics Testing company in Pediatrics
• Sell Lab services to other Children’s Hospitals
• Speed of innovations increased
• Involve other companies and hospitals as Strategic Partners
• It was High Price and Low Volume – Now it is High Volume and Price is getting lower
• Spin off examples: Eye surgery, Radiology, Basic Research vs Clinical Research

DISCUSSION on Pros and Cons for Hospital to  Spin off

• Diagnostics Companies: can assist Academic Hospital Labs to get to the Clinics fasters with Tests that are perfected to scale
• High Volume Test, understanding the disease better
• Diagnostics companies focus on Solutions to providers

ISSUES

1. InvestorsWho should be:

• Only non-Dilutive, i.e., Grants,
• Other Hospitals,
• VC: Phase 1 & 2, scale fast via acquisition
• Research collaborators

1. Who will be on the Board: Other Genomics Companies
2. Value of Spin off is significant to BCH that owns a piece
3. Objective, is the Primary Service to the Community of a Source of Value Creation for BCH

What actually Happened?

• Cerner Cincinnati Children’s Hospital and NextCODE became investors
• Illumina dominated the research sequencing marker
• Company developed own tests
• Recently released Claritas Clinical Exome platform
• Claritas executed a contract with the VA to sequence exomes and genomes of >20,00 veterans (1 Million Veteran Program)
• Platform: Used tests, data analysis, results interpratation and delivery to EMR
• sequence the exomes TWICE for validity, integrated with EMR, results are passed on for Research,
• Quality driven accuracy data collection and Analysis
• Volume drive Profits
• TESTING and CARE — Diagnostics became more accurate affecting Outcomes
• Research Exome, different than Clinicla Exome – ACM Criteria for interpretations
• Clinical Exome, subsequent access for research – assays
• CULTURAL changes:

Strategic relations:

1. WuXi – NextCODE: Genomic SW platform for all exome data
2. Cerner: Claritas interfaces with EMR, EPIC and Lab Information Management System (LIMS)
3. Illiumina and Life Technology: Product obtains speed and accuracy through dual platform approach
4. constituencies: Ordering the right TEST? How that is accomplished
5. Expression of the phynotype for the diagnosis and the treatment

 

 

9:30 a.m. Keynote Speaker

Looking at Personalized Medicine from a Pharmaceutical Perspective

• Right Target

Targeting the Tumor Cancer Genome, ALK 1st & 2nd Gen

• Right Drug(s)

Targeting the Immune Cancer Genome – PD-L1, 4-1BB

• Right Patient

Targeting Inherited Genome Variability – F9 Gene Therapy

LUNG CANCER Subtypes

Diversity of Oncogene Drivers:

• EGFR: Tarceva, ALK, ROS1 translocation

Resistance

• ALK mut/ampl
• RTK (EGFR, KIT)
• RTK+ALK
• ? Cycadia

Right Target
Lorlatinib: potential Next Gen ALK Inhibitor to address Resistance

Phase 1 Study with Xalkori-Resistant Brain Mets

Patient Treatment Timeline

1. Xalkori, 2011
2. mainteanime
3. Xalkori rechallenge
4. Carboplatin
5. Radiation + Cisplatin 1st dose of Lorlatinib (Phase 1 trial)

Right Drug(s)

Immuno-profiling

• Tumor: Genome Transcriptome Neoantigens Proteome (PD-L1 – Tumor Tissue
• Immune Infiltrate:

Ovarian Cancer: Immuno-oncology Targeting the Immune Cancer Genome

• Monotherapy: avelumab (PD-L1) Durable response in Ovarian Cancer
• Combo-therapy: 4-1BB + rituxumab Derable CR in Rituxan Refractory FL Screening ans wmonth CT
• immune-profiling

Exiting time for Combination Therapy and Precision Medicine based on Genomics

Gene Therapy: Targeting Inherited Genome Variability in Himpophilia

 

Glybera Gen Therapy approval in lipoprotein lipase deficiency marks

• Which Patients to Treat
• Community impact
• Collaborations
• Constituencies

Tomorrow Integrated Rx/Dx: Biomarkers for disease 

AUDIENCE QUESTIONS:

• Pfizer Inc. – Drugs to eleviate disease burden
• Reimbursement in UK: Advanced drugs denied to patients
• approach in Pharma to advance Medicine, get Patients back to work
• effective medicine and funding science
• molecular databases: development of scientific findings for clinical
• Drugs have approved diagnosis
• Experimental drug for ROS dramatic results vs ALK – regulatory
• ONE one drug One Diagnostics
• Combination of Drugs for multiple gene expressed conditions
• Drugs with benefit using molecular diagnostics are now at Academic Centers — HOW that could diffuse to larger populations
• Test to be cost effective for multiple indication

 

 

– See more at: http://personalizedmedicine.partners.org/Education/Personalized-Medicine-Conference/Program.aspx#sthash.O4Znb9kq.dpuf

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TALK SPECIFIC # and @

Day	Time	Talk Title	#	@
Thurs. Nov 19, 2015	8:00 AM	Welcome & Opening Remarks	#personalizedmedicine #genomics #Boston #Harvard #MGH #biotech #harvardhealth	@PartnersNews @MassGeneral   @HarvardHealth @harvardmed
		Greetings	#personalizedmedicine	@newsfrompmc (Personalized Medicine Coalition)
	8:30 AM	Building a Personalized Medicine Company: What does it take?	#NIH #AER15 #personalizedmedicine #pharma #startup	@HarvardHBS @BV
	9:00 AM	Looking at Personalized Medicine from a Pharmaceutical Perspective	#personalizedmedicine #pharma #biotech #biopharma	@Pfizer @FiercePharma @FierceBiotech