LIVE 1:40 pm – 3:50 pm 4/26/2016 Fireside Chat: Patients Driving Innovation & Immunotherapy II: Cell Based Therapies & Financing Breakthrough Cancer Companies @2016 World Medical Innovation Forum: CANCER, April 25-27, 2016, Westin Hotel, Boston | Leaders in Pharmaceutical Business Intelligence (LPBI) Group

10 new multiple myeloma drugs approved with the activity on Kathy

Business disruptive Patients and Business Executive for the Foundation

ex-Pharma executive

How were you received

democratization of genome

Stop treatment

Dean of HBS – Kraft $20 MIllions

Kathy Giusti, Founder, Multiple Myeloma Research Foundation and Multiple Myeloma Research Consortium

Scientists and business people from Millenium, payers

how many drugs got approved?

now 40 drugs are approved

disease is fatal

Campus Trial: Whole Genome sequenced – Consortia and Industry each paid 50%, African American affected,

Science, business, technology

At Myeloma – Data sharing

WSJ article to educate patients to agree to get their genome sequenced

$300Million collected an dgiven to the Scientific Community – Crowd sourcing

when she was diagnosed there was no Internet

Not focus is Cancers

Shawn Parker – Immune

Now involved with HBS – for the Kraft Fund of $20 Million for Cancer research of Mutations

Under went Myeloma stem cell transplant – no more maintenance therapy decision

2:10 pm – 3:00 pm
Novartis Ballroom

Immunotherapy II: Cell Based Therapies – CAR-T

Oncology sits on the cusp of a new revolution thanks to the use of human cells as versatile therapeutic engines. By modifying T cells to express chimeric antigen receptors (CARs) that recognize cancer-specific antigens, our panelists describe how they have been able to prime cells to recognize and kill tumor cells that would otherwise escape immune detection. Panelists detail the various “living drugs” that kill cancer cells and could replace standard oncology treatments in the future.

Moderator: David Fisher, MD, PhD, Chief, Dermatology Service, Director, Melanoma Program, Director, Cutaneous Biology Research Center, MGH Cancer Center, Edward Wigglesworth Professor of Dermatology, Harvard Medical School

Combinations

combinatorial Partners

Challenges lymph node what strategies can be developed

Pricing for CAR-T

epitome spreading – additional autoimmune response awakening — is it possible?

role of Academic center

Versions 2,3,4 — ten years from now

Usman (Oz) Azam, MD, Global Head, Cell & Gene Therapies Unit, Novartis

hematological – amazing response

benefit is great

condition of disease — hope for durable response for patients that the only treatment can be bone marrw transplant

CAR-T prevent the need for bone marrow transplant

not a biologic but boost to own immune system

solid tumor: Brain – combinatorial therapies will be the future

Gene editing

combination therapies will be the future – most innovations will be here

Treatment will show results soon, science get refined

scalability seen

expensive treatment if- refructory vs bone marraw – this is up to $1Million in cost

small molecule and biologics – CAR-T more expensive

if curative – economic can be supported

Regulators need be convienced on not a small molecule and not a Biologics

long term outcome – reassure substancial benefits

Inside development and collaboration with UPenn

Castody and longterm follow up

centralized manufactory

hematologic concured and some succeses with solid tumor burden

Mark Frohlich, MD, Executive Vice President, Portfolio Strategy, Juno Therapeutics

Solid tumors vs Hematological Cancers — different strategies

persistance use vaccine to enhance

differentiate

tumor micro environment

expression

nano particles

gene editing

administration – cost is high

if replace Transplant of Bone marrow — cost of goods will be lower

compatibility can be shown

efficacy will be achieved in 2nd generation

novel receptor – T cell receptors may be activated

Manufacturing must be centralized, educated personnel working on asseys

Academic Centers will explore problems cropping along

explore new targets

Marcela Maus, MD, PhD, Director of Cellular Immunotherapy, Massachusetts General Hospital, Member of the Faculty, Harvard Medical School

ALL more suseptable to CAR-T??

clearing bone marrow – can be inhibited

T-Cells can go anywhere

T-Cells may not be a model for platform suppresion vs enhanced immune response

automated manufacturing will lower costs

of the shelf T-cells,

Regulatory environment has changed

200 patient – investigational drug

T-cell repertoire antigens

CAR-T will become platform for Solid Tumors

Gene editing will be dominate

Chuck Wilson, PhD, CEO, Unum Therapeutics

response rate — optimistic

T-cells Therpies — most complex TCRL, CheckPoint – if T -cell are not directed, no response

modify and super charge alter pathways to enhance response

economic implications – PD1 additional functionality

Target a gene to knock in using CRISPR signal pathways suppressed

T-cells from a donor

each patient is a batch thus, cost of goods will be high

a bottle with antibodies is less expensive than cells sent to lab for treatment back to hospital fro transplantation

safety at present is limiting applications

adaptive immune response combine Vaccines

3:00 pm – 3:50 pm
Novartis Ballroom

Financing Breakthrough Cancer Companies

The global market for oncology therapies is currently more than $100 billion annually, and could reach $147 billion in two years. Panel experts discuss the most compelling technologies, trends driving investment considerations and strategies they are using as leading oncology investors in both early stage and established companies.

Introduction by: Roger Kitterman, Managing Partner, Partners Innovation

Innovations drive Healthcare

Moderator: Meg Tirrell, Biotech and Pharma Reporter, CNBC

regulatory

Jean-François Formela, MD, Partner, Atlas Ventures

Early stage – supply is as in the past robust

funding and exit — many opportunities

CRISPR – value novel science

translation medicine faster

IO – young technology

Pricing – disingenious statement – abuse of Pricing by Pharma

ecosystem – Policy to prevail

drug vs diagnostics

epigenetics drugs

old mechanism of action can be better understood today

CRISPR will be applied to CAR-T cell drugs deletion, change genes

Jonathan Leff, Partner, Deerfield Management

Public market was enthusiasm

it came down

less capacity to do out of Academia

candidates to go publis less than years

mismatch in capital available and the need

quality of science is outstanding

better drugs

every model assumes that you will charge a price that will deliver value to Patients

high price had drawn investments to drugs

risk involved on Capital

Pharma abuse d charging on innovative drugs

ability to charge is related with the Regulatory process

2012- transforation how to get drugs faster to market by FDA

FDA said to oncology community – approval rate should not be accelerated

FDA – went to the opposite direction

targeting and identify the therapies that will ease the investment in oncology

concern that sponsors do not follwo by after market studies – Confirmatory studies for 35% response for drugs with accelerated path to approval

Amir Nashat, PhD, Managing Partner, Polaris Partners

Immuno-oncology – early stage – many great ideas

gene control

gene editing

cancer vaccine

innate immune system

invigoration and rannaisnce

IO – excitement about the results- success beget success

existing industry to screen and early diagnosed

continuum of care intrigued

investment in diagnostics companies

transparency – cost

increase in longevity – will increase productivity along a longer life

Buncrupcy caused by medical costs

underwriting drug costs

figure out how to fund health care

functional biology

Christopher Viehbacher, Managing Partner, Gurnet Point Capital – $2Billion Early stage clinical and non invasive tech

BioTech Index Funds in last 5 years outperformed S&P

Cancer – immuno-oncology is nor ALL the cancers needs

technology for colon cancer – easier a colonoscopy

drug delivery

lower toxicity

nanotechnology

Drugs is 10% of total Healthcare cost

morgage model for pricing drugs – spread the expense along many years

2015 – dug cost environment — if Value based – it will become cost effective

AZ had a drug with 10% response – lead to targeted drugs

combination therapy checkpoint inhibitors

drug here diagnostics there

if you own you can control the drug

safety of old mechanism of action are now better understood