web_panel gene therapies for rare diseases may 6 2015

7/23/2019 Web_Panel Gene Therapies for Rare Diseases May 6 2015

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What's Hot & What's Not

in Gene Thera ies for Rare DiseasesMay 6, 2015

o era or:

Mike Rice, MS, MBASenior Consultant, Defined Health

Panelists:

Matthew Porteus, MD, PhDAssociate Professor of Pediatrics(Cancer Biology), Stanford University

Stewart Abbot, PhDExecutive Director, Integrative Research at

Paul Gallagher, MBAPresident, Compass Strategic Consulting

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allows organizationsto simply andsecurely share

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consultancy to pharmaand biotech.

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BIO also organizes theBIO International

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be held in Philadelphiafrom June 15-18.

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Logistics

The web panel discussion will last 75 minutes

.

Contribute questions via the Q&A GoToWebinar

interface.

We will address the questions intermittently or at the

end. If we dont get to your question, we will respond

The web panel discussion is being recorded, and we

will contact you when it becomes available.

We welcome your feedback after the web panel so we

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Mike RiceMS, MBA

Senior Consultant

Mike RiceMike Rice

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Pharma Has Benefitted Significantly

b Investin in Biolo icsTop-Selling Products (WW Sales $M) 2004 vs. 2014

(coded by conventional vs. biologics)

Lipitor (atorvastatin) - Pfizer Humira (adalimumab) -AbbVie

2004 2014

o og conven ona

Z rexa olanza ine - Lill

Norvasc (amlodipine) - Pfizer

Seretide/Advair (fluticasone;salmeterol) - GSK

ocor s mvas a n -

Seretide/Advair (fluticasone,Rituxan (rituximab) - Roche

Lantus (insulin glarginerecombinant) - Sanofi

Sovaldi (sofosbuvir) - Gilead

Zoloft (sertraline) - Pfizer

Procrit (epoetin alfa) - J&J

Nexium (esomeprazole) - AZN

Remicade (infliximab) - J&J

Herceptin (trastuzumab) -Roche

Avastin (bevacizumab) -Roche

sametero -

0 5,000 10,000 15,000

Plavix (clopidogrel) - BMS

Effexor (venlafaxine) - Wyeth

0 5,000 10,000 15,000

Lyrica (pregabalin) - Pfizer

Crestor (rosuvastatin) - AZN

EvaluatePharma

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Gene and Cell Therapies Next

Advancement Be ond Biolo ics

TherapeuticInterventions

SmallMoleculeModulators

ProteinAugmentation

Antibodies Peptidesand

NucleicAcidsGeneCorrection& Augmentation

CellTherapy/Regen Med

Immune

Modulators

SMIs

Plasma/tissue

derived

proteins

Plasmaderived

Polyclonal Igs

Monoclonal

Immune

Modulators

Exon skipping

Viralvectors

Retro/

Lentiviral

AdV

Autologous

andallogeneic

BMT/Cell

therapy

Chaperones

Substrate

Reduction

ecom nan

Proteins

Clotting

factors

Cytokines

an o es

mAB fragments

Scaffolds

Antisense

RNAi /miRNA

Nonviral

Plasmids/

Fragments

Othercell

sources: e.g.

ES,iPS

Transcription /

Translation

enhancers

ormones

Growth

factors

Enzyme

Intrabodies p amers

Ribozyme Geneediting

with

Meganucleases

ZincFingers

ev ces

Encapsulation

Scaffolds

Implants

Microorgans

Epigenetics Rep acement TALENS

CRISPR/Cas9

Ap aeresis


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Gene Therapy Defined

According to the FDA, gene therapy products areall products that mediate their effects by

transcription and/or translation of transferredgenetic material and/or by integrating into the

acids, viruses, or genetically engineeredmicroorganisms.

The products may be used to modify cells in vivoor transferred to cells ex vivo prior toadministration to the recipient.

FDA.gov

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Components of Gene Transfer Platforms:

Clinical Need, Intervention, Delivery VectorAAV

Adenovirus

Retro/LentiviralGene Transfer

HSV, VACV, SV40

Plasmid/Fragment

Liposome, Other?

Polygenic Diseases

Monogenic DiseaseClinicalNeed Infectious DiseaseGene

Therapy

Oncology

Suicide Gene

Gene Augmentation

Antisense, RNAi

TherapeuticIntervention

ZFs, CRISPR, TALENs

Ribozymes

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Clinical Need: Today There Are Over 500Active Gene Therapy Programs

Broadly Applicable: Investigated in >1,600 clinical trials

Cancer is By Far the Most Active Area for Clinical Trials Monogenetic Diseases May Be Most Tractable

5

Gene Therapies by All Therapeutic Areasn = 574, individual products counted multiple times

OncologySensory Disorders

20328

28

14Meta o ic Disor ersCardiovascular DisordersNeuromuscular DisordersHematological Disorders

Neurological DisordersNeurodegenerative Disorders

29

28n ec ous sease

Immunological DisordersMusculoskeletal DisordersOtherGastrointestinal DisordersLiver Disorders

69

56

39

33

Genitourinary DisordersCongenital DisordersLung DisordersAccidents & InjuryRespiratory DisordersAdis R&D Insight,

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Gene Transfer Vectors:Viral-Based Vectors Used in Majority of Pipeline

Gene Therapies in Development by Approachn=394

14

139 7

5 Adeno-associated virus (AAV)

Other gene therapy

Adenovirus

122

16

15

15 Lentiviral vector

Plasmid

Cancer vaccine

Fragment/DNA

38

23Retrovira

T cell therapy

Cell therapy

Other viral vector

Oncol tic virus

65

49

ZF

Transposon

TALEN/CRISPRAdis R&D Insight,

Thomson Reuters Cortellis

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Therapeutic Intervention:GT Originated as Functional Genomics Tools MayTrans orm In er te D sor ers an Ot er D seases

Inherited Disorders

StableTransient

Enzyme (ERT)

mRNA

Allo SCTAutologous Cell

Correction

mRNA

Exon Skipping Plasmid Transfection

Viral Integration

Recombination

Gene Activation

Triplex

Other epigeneticTransposons

Meganucleases, ZF,

Viral Episomal? Gene Editing

, ,

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Cross-Comparison of

Gene Thera PlatformsTechnology Capacity Delivery* Integration Pros/Cons

AdenovirusP: high packaging capacity

(AdV)

< n v vo p soma : e c s a po en mmune response;transiently expressed transgene

Adeno-Associated < 5kB In vivo Episomal

P: non-pathogenic; infects dividing ornon-dividing cells

C: Prior ex osure immune re ectionrus

DNA lost through cell division

Retrovirus < 8kB Ex vivo IntegratingP: stable integration into host genomeC: random insertion tumorigenesis risk;

only infects dividing cell types

Lentivirus 8-10kB Ex vivo IntegratingP: infects dividing or non-dividing cells;

reduced tendency to cause cancerC: theoretical tumorigenesis risk

*Indicates how a technology is being used in its current format

GeneEditing

n/a Ex vivo IntegratingP: Ability to add, delete or correct genesat the single nucleotide levelC: complexity vs. gene addition;


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Emerging Genome-Editing Platforms

Gene editing refers to methods that target adouble-stranded break in the genome, then directsa s ecific DNA se uence alteration.

ZFN, TALEN, andCRISPR/Cas9 genome-editing

Four families of engineered nucleases used: Meganucleases (MEGAs): highly specific due to large

recognition site (dsDNA sequences of 12 to 40 base

tools

pa rs .

Zinc finger nucleases (ZFNs): fusion of a zinc finger

DNA-binding domain to a DNA-cleavage domain.

Transcription activator-like effector nucleases(TALENs): fusion of TALE DNA binding domain to aDNA cleavage domain.

palindromic repeats (CRISPRs): Delivery of Cas9(an RNA-guided DNA endonuclease enzyme) andappropriate guide RNAs into a cell used to cut thegenome at a desired location.

roc at ca c : .; ature otec no ogy : .; ature ev ews enet cs : .


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GT May Be Inflecting in Value,

But Has Yet To Produce aCommercially Viable Product

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Old and New GT Companies

Advancin Diverse PlatformsGene EditingGene Augmentation

AAV

AdV

Ado tive Cellular I O-

Retro

Plasmid

Other

Partners

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Indicator of Maturity of GT Platforms:

-

Counts of Papers Published on PubMed Per YearMentioning Each Gene Therapy Approach

1 600

1,800

2,000

achYear

Adeno-Associated Virus Adenoviral

Retroviral Lentiviral

CRISPR TALEN

1 000

1,200

1,400

Pub

lished

s eganuc eases

600

800

ofPapers

-

200

1985 1987 1989 1991 1993 1995 1997 1999 2001 2003 2005 2007 2009 2011 2013

Numbe

PubMed

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Indicator of Maturity of GT Platforms:>200 Programs Advancing in Clinical Trials

Severa w t Conv nc ng PoC

Broadly Applicable, Cancer is By Far the Most Active Area for Clinical Trials Monogenetic Diseases May Be Most Tractable

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Gene Therapies by All Phases of Development

n = 574, individual products counted multiple times

68Preclinical

Phase 1/2

Phase 2

329158

Phase 3

Registered

Adis R&D Insight; Cortellis

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Thecombined

regenerative

medicine

field,

including

cell,

gene,

andgenemodifiedcelltherapies,generated$4.74Bthrough

, ,

investmentsfromMarch2013toMarch2014.

Thereare

close

to

700

clinical

trials

currently

underway

with

the

, ,

cardiovasculardiseases.And,theclinicalpipelineismaturing

withoveronethirdofthosetrialsinlaterstages(P2orP3). The

rateofgrowthisreflectedinthepublicmarkets:25%ofthe

biotechIPOs

in

the

second

half

of

2013

were

regenerative

medicinecompanies.

http://www.biospace.com/News/biotech-ipos-up-22-since-bubble-year-of-2000-shows/361523; http://www.poliwogg.com/news/125-sectors-to-watch-in-biotech-and-healthcare-investing

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Gene Thera IPOs & VC Financin

2015

IPO VC Financing

$29 M Series B

Dec 2014: $185 M

Jun 2014: $117 M

Apr 2014: $55MSeries B

May 2014: $20 M

Jan 2014: $50 M

Oct 2013: $44 M Nov2013: $47M Series A

2013

July 2013: $18M Seed CapitalMay 2013: $18.4M Conv. Issue

May 2013: $150M Series F

Jul 2013: $184 M

2012

.

May 2012: $10M Series DFeb 2012: $43M Series D

May 2011: $100M Series E

EvaluatePharma

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Pharma InvestmentGene Therapy Deal Timeline 2012-2015

Jan 2015$6M

Hemophilia A&B

Dec 2014$280M

Hemophilia B

June 2014$252M

Hemophilia A

Aug 2014$4M

Acquisition

June 2014$44M

Acquisition

Oct 2013$1M

Acquisition

Apr 2015$64M

VC Funding

2013 2014 2015

Feb 2015$845M

Parkinsons

July 2014$1MCV

July 2013$40M

Familial lipoproteinlipase deficiency

Jan201511M+

April 2015$1B

S100A1CV

DiseaseCRISPRplatform

EvaluatePharma, Company Website

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Gene Therapy Innovator Market Caps

Company Market CapSangamo

5,000

6,000

Bluebird

ZioPharm

GenVec

$5.7B

3,000

4,000

unts($M) AmpliPhi Biosciences

Intrexon$3.07B

$4.3 B

1 000

2,000IPOAm

AGTC

UniQure

$897M

Bloomber .com

0

,

AAV AdV Lenti ZF

va anc e

Spark

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T-Cell Therapy Deal Timeline

-

Feb2014

$150MCART

June2014

$350M

TCR

Sept2014

$350MTargetedTcells

Opus Bio

Dec2014

$20M

CART

Jan2015

$525M

CART

Jan2015

$150M

CART Mar2015

CART

Jan2015

$60M

CART

2014 2015

Jan2014

$13.5M

Acquisition

June2014

$265M

CART

Sept2014

CART

Oct2014

TCRms

Dec2014

$11m

CART

Mar2015

CART

Apr2015

CART

Feb2015

CART,TCR

EvaluatePharma, Company Website

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T-Cell Therapy Market Caps

20,000

Market Cap of Leading Biotechsin T-Cell Space by Technology

ZioPharm

Takara Bio

14,000

16,000

18,000 Sangamo Biosciences

MolMed

MacroGenics

$17B

10,000

12,000

ket

Cap

($M)

Kite Pharma

Juno Therapeutics

Emergent Biosolutions$8.6B

4,000

6,000

8,000Ma Cellular Biomedicine Grp

Cellectis

Bluebird Bio

Bellicum

0

2,000

Bi-specific CAR T TCR CTL Other T celltherapy

TIL

Atara Biotherapeutics

Affimed Therapeutics

.

$1.2B $797M$536M

Bloomberg.com

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Pharmas Have Renewed Interest in

Gene Thera iesHowever, Issues Remain

Translation of reclinical results into man

Delivery, Stability, Immunogenicity

Safety e.g. anaphylaxis, insertional mutagenesis, cytokine storm

Higher regulatory scrutiny

Innovative clinical endpoints necessary

RAC review

Does not fit nicely into Pharma scalability model Complicated IP and stacking royalties

Individualized vs. one-size fits all

Involves devices and process

anu ac ur ng: epro uc y, ca a y, g cos o goo s

Market Access:Value-based pricing in different payer systems

Bioethics: Boundaries of use, Testing in vulnerable populations

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Recent Gene Therapy News Highlights

GlaxoSmithKline files for gene therapy OK asa case of the jitters sets in

May 5, 2015

By John Carroll

GSK CEO Andrew Witty

Close to 5 years after GlaxoSmithKline ($GSK) signed on to collaborate with the San Raffaele Telethon Institute for Gene Therapy inItaly, the partners have stepped up with a European application to start marketing a gene therapy for extraordinarily rare cases of

- .recoiling from some notable setbacks that have begun to cloud what has been a bullish sector in biotech.

The drug is the rarely mentioned GSK2696273, a gene therapy which uses a viral vector to insert working copies of the ADA geneinto stem cells extracted from the bone marrow of patients. The cells are then reintroduced to the patient, who can expect to startmaking the gene on their own, repairing their immune system.

The Europeans will be reviewing an application built on data from 18 patients, with the first treatment dating back 13 years. All arealive thou h three had to have follow-u enz me re lacement thera or a bone marrow trans lant which is what most of thesepatients rely on today.

An approval for GlaxoSmithKline in Europe would have wide implications. With so few patients, the center in Italy may well becomea Mecca for patients around the world, including the U.S.

"This is an important landmark for clinicians, researchers and all the staff at TIGET who have been working side by side with GSKtowards approval of this gene therapy," says Alessandro Aiuti, the clinical research coordinator at TIGET. "In the past years wehave witnessed how a sin le infusion of ene modified stem cells has chan ed the lives of these children and their families. Ifauthorized, we will be ready to offer gene therapy at our center to ADA-SCID patients in need from Europe and other countries.

https://www.genomeweb.com/research-funding/nih-places-human-germline-out-bounds-genome-editing-funding

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Recent Gene Therapy News Highlights

NIH Places Human Germline out of Bounds forGenome Editin Fundin

April 29, 2015

NEW YORK (GenomeWeb) Reacting to research published last week in which Chinese scientists conducted germline editingon a non-viable human zygote, the National Institutes of Health today firmly came down in opposition to the use of geneediting technologies in the human germline.

"NIH will not fund any use of gene-editing technologies in human embryos," NIH Director Francis Collins wrote in a statementpu is e on t e NIH we site

While technologies like CRISPR/Cas9 provide an "elegant" way of editing the genome, Collins said safety and ethical issuesoutweighed any potential benefits and added that germline editing has been viewed almost universally as a line that shouldnot be crossed.

NIH's stand comes a week after scientists from Sun-yat Sen University in China published a study reporting CRISPR/Cas9gene editing of the beta-thalassemia gene in nonviable tripronuclear human zygotes. The study confirmed rumors thatsc en s s were us ng e ec noogy o a er e uman germ ne an prec p a e w esprea ac as n e sc en cand popular press.

In his statement, Collins mentioned several legal and regulatory barriers to conducting such research in the US, including theDickey-Wicker amendment, which forbids federal funding for the creation of human embryos for research, as well as researchin which human embryos are destroyed; NIH Guidelines pertaining to the Recombinant DNA Advisory Committee, whichstates that research proposals for germline changes will not even be considered; and the authority given to the US Food andDrug Administration to regulate gene therapy products under the Public Health Service Act and the Federal Food, Drug, andCosmetic Act

"NIH will continue to support a wide range of innovations in biomedical research, but will do so in a fashion that reflects well-established scientific and ethical principles," Collins said.

htt s: www. enomeweb.com research-fundin nih- laces-human- ermline-out-bounds- enome-editin -fundin

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Matthew Porteus, MD, PhD

Introduction:

Associate Professor of Pediatrics(Cancer Biology), Stanford University

Areas of interest in research

What is state of the art?

Gene Augmentation vs. Gene Editing

Involvement with CRISPR

Therapeutics Matthew PorteusMatthew Porteus

How is the ability to raise significant capital ina biotech company changing the advancementof the field compared to the academic setting?

to impact which disease first?

Whats down the road?

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Stewart Abbot, PhD

Introduction

Executive Director, Integrative Research atCelgene Cellular Therapeutics (CCT)

What is Going on at CCT

How is Celgene employing gene

Stewart AbbotStewart Abbot

trans er an ce u ar t erap esacross therapeutic areas?

How is a large pharma company

of individualized, potentially one-time treatments?

Infrastructure, logistics, manufacturing,

scalability, patient access

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e u ar erapeu cs

Gene Therapies for Rare Diseases

Stewart Abbot

May 2015

NON-CONFIDENTIAL


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Celgene Cellular Therapeutics (CCT)

Whats going on at CCT

CCT is a wholly owned subsidiary of Celgene Corporation

Established in 2003 as semi-autonomous group within Celgene

Longstanding interest in human-placental cells (HSC and MSC)

Internal R&D, regulatory, clinical and cell and tissue productmanufacturing capabilities

eve op ng ce , gene-mo e ce an ssue erapeu cs

Primary focus on indications with high unmet clinical needs

(rare and not-so-rare)

Clinical-stage candidates including PDA-002 Diabetic foot ulcers and PAD

Support chimeric antigen receptor-modified T cell collaborations

Revenue generating activities including biomaterial products and private

Partnered BiovanceTM and other biomaterial products with AlliquaBiomedical

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Celgene Cellular Therapeutics (CCT)

How are we employing gene transfer and cellular therapies

Mechanistic studies

Medium throughput RNA-silencing studies to define PDAC (MSC-like) cell MoA

Transfection protocols for whole genome siRNA and miRNA

Viral transduction stable cell lines for in vitro and in vivo functional studies Potential for product life cycle management

Utilize detailed MoA findin s to further au ment cell function b en ineerin in or

out CTQ functionality

Gene-modified cellular candidates

Embracing technology convergences between cell and gene-therapy platforms , -

oncology)

Gene modified HSC-derived RBC (DARPA and other partners)

Chimeric antigen modified T-cell candidates

-

R&D capabilities

Focus on scientifically-driven product development and practicality of

production, distribution etc.

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Pharma Approaches to

Individualized TreatmentsRare Disorders

The individual treatment MarmiteTM analogy:

You either love it or you hate it.

Precision medicine versus rare diseases

7 000 different rare diseases and disorders 30 million eo le in the United

States are living with rare diseases

Potential for precision medicine initiatives to define individualized treatment

subsets within highly common single disorders

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Pharma Approaches toIndividualized Treatments

CCTs Experience

Initial focus on expanded allogeneic cell therapies for common disorders

Immuno-evasive cells, scalable production, lower COGS

Business models evolving to embrace individualized treatments for orphan

indications

Reimbursement and margins dependent on magnitude of treatment effects

Ex vivo gene-modification of cellular therapies can overcome PK issues with

gene-therapies Potentia or rationa y- esigne t erapeutics to e curative or at east

transformative

Assumption that high treatment effects can facilitate smaller clinical trials

Organizational structures evolving to embrace modality diversity

CCT organized as semi-autonomous unit within Celgene to develop modality-

, .

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CCTs Experience (continued)

Rapidly adapt to evolving scientific, regulatory and commercial

bureaucracy

Understand, and potentially embrace, initiatives such as Japans Pharmaceutical

and Medical Device (PMD) Act, Safety of Regenerative Medicine Act

Focus on making the possible practical

Develop solid understanding of CTQ attributes for each product (non-modified or

gene-modified)

Consider duration of cell and gene-modified manufacturing cell processing andinfrastructure requirements (clean room suites, incubators etc.)

Time in culture = risk & cost in production

Develop robust, comparable and scalable manufacturing and distribution solutions

Leverage expertise in allogeneic cell process development and manufacturing

scale-upapproaches to develop autologous cell manufacturing scale-out

approaches

Distribution logistics for frozen and non-frozen products and feedstock

Requirement for JIT processing and rapid release tests

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Internal or external partnering to rapidly fill capability gaps

Novartis / U. Penn

GSK / Adaptimmune

CCT / Alliqua biomedical

Embrace new technology developments

CCT / miRNA-based screening

Novartis / Intellia / Caribou

GSK / Celgene / CRISPR Therapeutics Check the business model is practical

Orphan versus ultra-orphan

Patient access for trials

Patient available and reimbursement

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Ph A h t


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Summary

- -

therapeutics

Current clinical success is supporting high levels of investment,

. .

Current investment should support appropriate basic and clinical

R&D to ensure some the approaches change the near-future

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Paul Gallagher, MBA

Introduction:

President,Compass Strategic Consulting, Inc.

Will payers carve out genethera ies in health technoloassessments, pricing and coveragedecisions?

In planning will innovative

Paul GallagherPaul Gallagher

paymen sc emes, suc asannuities, apply?

What should developers of genethera ies do to o timize the value

of innovations?

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P i i d k t i f


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Pricing and market access issues of genetherapies are just beginning to be addressed

Glybera registered and pricedin Germany at 877,400

No country has carved outspecific HTA process to cover

There is not just one business model for all gene therapies.

Different diseases will find different models

Jorn Aldag, CEO, UniQure

regenerative medicine

Generally off the radar ofa ers

I dont know that this is on anybodys radar screen, thats the

other issue, I dont think a lot of senior leaders of our businessunits really understand.

Member of formulary of major US payer, Dec 2014

Advocacy groups beginningto look at reimbursementissues

Alliance for Re enerative

The original annuity payment could be set with certaintypes of 're-opener' clauses, such as with patent expiration[death], or if a less expensive new therapy came on line --

thus subjecting the gene therapy annuity to the same vagaries

Medicine

Calls for innovative payment

of market competition that standard pharmaceuticals faceThe special case of gene therapy pricing, Troyen ABrennan, James M Wilson, Nature Biotech, Sep 2014

I think its very difficult to amortize payments...the business

Pricing and budget impact

not value are issues in thepublic domain

-

regulated industryfor fully insured products there are specificrules reserve requirements

Member of formulary of major US payer, Dec 2014

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In your planning, assume current payment

until health systems show willingness tocarve out some gene therapies

Remember affordability is a major issue in ex-USmarkets and coming to the US

therapies is asking for special treatment in HTAs and offthe radar in the US, less so in the EU

specific for product and country / payer within country

Start early to understand the needs in markets meet with payers

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If new payment mechanisms are necessary


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If new payment mechanisms are necessaryfor some curative therapies, change will

require a collaborative effort amongnumerous stakeholders

Prioritize developing strong clinical, economic and long-

term follow-up data based on early analysis of genetherapy HTAs

Focus on value, start with crystalizing the burden of illness

Monitor health system structure / policy since they

are likely to change based on recent history Educate payers, payer influencers and policy makers

Collaborate with decision makersuse strength innumbersform a working group and explore options with

the budget holders Build bridges to patient advocacy groups

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Discussion and

Listeners, please type your questions into the.

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What's Hot & What's Not


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What s Hot & What s Notin Gene Thera ies for Rare Diseases

May 6, 2015

o era or:

Mike Rice, MS, MBASenior Consultant, Defined Health

Panelists:

Matthew Porteus, MD, PhD

Associate Professor of Pediatrics(Cancer Biology), Stanford University

Stewart Abbot, PhDExecutive Director, Integrative Research at

Paul Gallagher, MBAPresident, Compass Strategic Consulting

`

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joining us!

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web_panel gene therapies for rare diseases may 6 2015

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