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
2016 World Medical Innovation Forum: CANCER, April 25-27, 2016,
Partners HealthCare, Boston, at the Westin Hotel, Boston
ANNOUNCEMENT
Leaders in Pharmaceutical Business intelligence (LPBI) Group
Streaming in Real Time using Social Media this Event on
CANCER Innovations
Aviva Lev-Ari, PhD, RN, Founder LPBI Group & Editor-in-Chief
http://pharmaceuticalintelligence.com
Streaming LIVE @Westin in Boston
Novartis Ballroom
Fireside Chat: Patients Driving Innovation
- 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
- 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
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.
- 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
- 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
- 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
- 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
- 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
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.
- Innovations drive Healthcare
- regulatory
- 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
- 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
- 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
- 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
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