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Archive for the ‘BioTechnology – Venture Creation, Venture Capital’ Category


2019 Biotechnology Sector and Artificial Intelligence in Healthcare

Reporter: Aviva Lev-Ari, PhD, RN

 

AI Ushers in a New Era

The implications of AI, cloud-based technologies and increased R&D focus have lent a competitive edge to companies within the biotech space. The use of AI has gradually begun to revolutionize research activities in the industry as it can drastically reduce time and costs involved in developing life-saving drugs.

Let’s take a look at some instances on how AI is being used to advance in biotech. AiCure has developed an application that uses AI to govern if and at what time the patient takes a pill. Moreover, it is now being used regularly in many clinical trials. SOPHiA Genetics ‘ AI system is used for genomics analysis of next-generation sequencing data from hospitals and research institutions globally.

Moreover, Illumina ILMN released an open source artificial intelligence software for discovering previously overlooked noncoding mutations in patients with rare genetic diseases in the beginning of 2019.

In fact, J&J JNJ , Pfizer PFE and Novartis NVS have tie-ups with IBM’s Watson Health. Per the deals, the companies can use Watson Health’s AI solutions and applications for drug discovery and to accelerate cancer research efforts.

SOURCE

https://m.nasdaq.com/article/biotechnology-market-on-a-tear-5-etfs-in-spotlight-cm1128200

 

The biotech industry has kept its promise for solid returns so far. The rally in some major biotechnology indexes reflects the same. In this context,

SOURCE

https://m.nasdaq.com/article/biotechnology-market-on-a-tear-5-etfs-in-spotlight-cm1128200

BioPharma

Novartis AG (NVS): Free Stock Analysis Report

Eli Lilly and Company (LLY): Free Stock Analysis Report

Roche Holding AG (RHHBY): Free Stock Analysis Report

Pfizer Inc. (PFE): Free Stock Analysis Report

Johnson & Johnson (JNJ): Free Stock Analysis Report

ALPS Medical Breakthroughs ETF (SBIO): ETF Research Reports

Principal Healthcare Innovators Index ETF (BTEC): ETF Research Reports

Virtus LifeSci Biotech Products ETF (BBP): ETF Research Reports

SPDR S&P Biotech ETF (XBI): ETF Research Reports

Spark Therapeutics, Inc. (ONCE): Free Stock Analysis Report

Illumina, Inc. (ILMN): Free Stock Analysis Report

ARK Genomic Revolution Multi-Sector ETF (ARKG): ETF Research Reports

SOURCE

To read this article on Zacks.com click here.

Zacks Investment Research

 

2019 M&A in Biotech

Mergers and acquisitions (M&As) are dominating the sector as sluggishness in mature products has forced companies to explore acquisitions to bolster their pipeline. The biggest deal of the year was Bristol-Myers’ acquisition offer of $74 billion to buy Celgene. Also, Eli Lilly and Company LLY has announced that it will take over Loxo Oncology for $8 billion to broaden its oncology suite to precision medicines or targeted therapies. (read: What’s Behind the Biotech ETF Rally to Start 2019? )

Several other large-cap pharma as well as bigger biotech companies are entering collaboration deals with smaller ones to boost their pipeline. Notably, Swiss pharma giant Roche Holdings RHHBY has bet big on U.S.-based gene therapy company Spark Therapeutics ONCE in an effort to strengthen its presence in gene therapy. Similarly, in order to develop gene therapies targeting rare indications, Biogen has offered to buy Nightstar Therapeutics.

Furthermore, in-licensing deals are consistently rising with bigwigs partnering with smaller and mid-sized players that own promising mid-to-late stage pipeline candidates or interesting technology.

SOURCE

https://m.nasdaq.com/article/biotechnology-market-on-a-tear-5-etfs-in-spotlight-cm1128200

 

Takeda-Novartis, Daiichi-AZ and more—FiercePharmaAsia
Takeda sells meds to Novartis and J&J; Daiichi’s AZ-shared HER2 antibody-drug conjugate hits key trial goal; Sun scouts for Chinese partner.
Takeda HQ
Novartis buys Takeda’s Xiidra, gets 400 staffers in $3.4B deal
Novartis hopes the deal, potentially worth $5.3 billion, could better position itself in front-of-the-eye therapies.
Asia Map
AZ, BeiGene, Kangmei and more—FiercePharmaAsia
AZ warns of slower China growth; BeiGene chief ranks among highest-paid biopharma CEOs; Kangmei faces delisting over huge accounting “error.”
Sanofi Pasteur HQ
After safety scare, Sanofi’s Dengvaxia nabs limited FDA nod
The FDA limited Dengvaxia to older children and teenagers living in endemic regions—and only if a diagnostic test confirms a prior dengue infection.
Takeda US facility
Takeda’s new Trintellix ad celebrates everyday wins
Takeda highlights everyday joys in new TV ads for major depressive disorder treatment Trintellix.
ReputationSign
HIV drugmakers ViiV, Gilead top pharma reputation survey
Pharma’s reputation is holding steady with patient groups with an annual study finding 41% giving pharma good marks, similar to 43% the year before.
Asia Map
PD-1 royalty dispute, Takeda and more—FiercePharmaAsia
Nobel laureate wants bigger PD-1 revenue cut; Takeda scouts buyers for Latin America business; Chinese genomics investor is forced out of U.S. firm.
Takeda scouts buyers for Latin American business: report
Takeda sold its Brazil-based unit Multilab right after it confirmed its plan to buy Shire, and now it’s reportedly mulling another sale in the region.
Woman typing on computer
Repackager recalls 40 lots of tainted losartan—News of Note
CDMOs Cambrex and Ajinomoto Bio-Pharma Services upgraded manufacturing plants, Takeda scored an albumin approval via its Shire deal, and more.
Darzalex
NICE limits coverage of J&J, Takeda myeloma combo
J&J’s Darzalex is on track to nab a second first-line myeloma nod in the U.S., but its reimbursement journey in England hasn’t been so smooth.

Other related 260 articles published in this Open Access Online Scientific Journal include the following:

https://pharmaceuticalintelligence.com/?s=Artificial+Intelligence

To access 260 articles:

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Artificial Intelligence per Ontology on this topic [multiple nested categories]

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LIVE 13th Annual BioPharma and Healthcare Summit, Thursday, May 9, 2019, Marriott Hotel, Cambridge, MA

 

http://www.usaindiachamber.org

8:40 AM – 9:10 AM Registration and Networking
9:10 AM – 9:20 AM Welcome addressKarun Rishi, President, USAIC

Opening comments: Dr Andrew Plump, President R&D and Director, Takeda Pharmaceuticals

9:20 AM – 9:40 AM Fireside Chat

  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration
  • Dr Eswara Reddy, Drug Controller General of India, Central Drug Control Organization

Moderator: Sanat Chattopadhyay, President, Merck Manufacturing Division; Merck & Co.

9:40 AM – 10:00 AM Presentation on CAR (chimeric antigen receptor) T-cell Therapies
Dr. Carl June, Director of Translational Research, Abramson Cancer Center University of Pennsylvania Moderator: Dr. Raju Kucherlapati, Professor of Genetics, Harvard Medical School
10:00 AM – 10:50 AM Panel Discussion: Oncology – The Emperor of BioPharma Development

Panelists:

Moderator: Dr. Christiana Bardon, Managing Director, MPM Capital

10:50 AM – 11:20 AM Networking Break
11:20 AM – 12:10 PM Panel Discussion: Future of Clinical Trials and Drug Development

Panelists:

Moderator: Dr. William Chin, Professor of Medicine, Emeritus, Harvard Medical School

12:10 PM – 1:00 PM Panel Discussion: Manufacturing in the Future

Panelists:

  • Hari Bhartia, Founder and Co-Chairman, Jubilant Bhartia Group
  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration
  • Dr. Paul McKenzie, Executive Vice President, Pharma Operations & Technology, Biogen
  • Sanat Chattopadhyay, President, Merck Manufacturing Division; Merck & Co.
  • Vinay Ranade, Chief Executive Officer, Reliance Life Sciences

Moderator: Professor N. Venkat Venkatraman, Boston University Questrom School of Business

1:00 PM – 1:50 PM Lunch
1:50 PM – 1:55 PM Video message from Suresh Prabhu, Hon’ble Minister of Commerce & Industry, Gov. of India
1:55 PM – 2:45 PM Panel Discussion: One in a million – Emerging trends in Rare Diseases

Panelists:

Moderator: Dr. Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics

2:45 PM – 3:20 PM Networking & Tea Break
3:20 PM – 3:50 PM Fireside Chat: Value and Access – The ongoing debate

Moderator: Dr Andrew Plump, President R&D, Takeda Pharmaceuticals

3:50 PM – 4:10 PM India update on Clinical Trial Regulations

  • Arun Singhal, Additional Secretary, Ministry of Health & Family Welfare, India
  • Dr Eswara Reddy, Drug Controller General of India, Central Drug Control Organization
4:10 PM – 5:00 PM Panel Discussion: Research and Development Strategies and Trends

Panelists:

Moderator: Dr. Martin Mackay, Co-Founde, Rallybio

5:00 PM – 5:05 PM Closing Remarks
5:05 PM – 6:15 PM Cocktails & Networking Reception

Aviva Lev-Ari, PhD, RN & Leaders in Pharmaceutical Business Intelligence (LPBI) Group

will cover the event in Real Time

REAL TIME COVERAGE USING SOCIAL MEDIA

 

LIVE Images taken by @AVIVA1950

 

 

 

9:10 AM – 9:20 AM

Welcome addressKarun Rishi, President, USAIC

Opening comments: Dr Andrew Plump, President R&D and Director, Takeda Pharmaceuticals

  • tomorrow announcement @Shire
  • India 1.3Billion in India, each person is a potential patient in the largest democracy in the World
  • China – transformation takes place every day
  • The Patient and the Pricing of Drugs the biggest issue missing the ball dialoguing on Panel today

9:20 AM – 9:40 AM

Fireside Chat

  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration (FDA)
  • Dr Eswara Reddy, Drug Controller General of India (DCGI), Central Drug Control Organization

Moderator: Sanat Chattopadhyay, President, Merck Manufacturing Division; Merck & Co.

9:40 AM – 10:00 AM Presentation on CAR (chimeric antigen receptor) T-cell Therapies
Dr. Carl June, Director of Translational Research, Abramson Cancer Center University of Pennsylvania Moderator: Dr. Raju Kucherlapati, Professor of Genetics, Harvard Medical School

  • Video on child with recurrent twice of leukhimia
  • T-cell HIV Virus infect

 

10:00 AM – 10:50 AM

Panel Discussion: Oncology – The Emperor of BioPharma Development

Panelists:

  1. solid vs blood tumors
  2. T-Cells amplification microenvironment and biology
  3. PD-1 in combination therapies thousand Trials
  4. Biomarker allows to check response in conjunction with genomics data brings insights
  5. Tumors World, Biomarkers in Immuno oncology respond to PD-1 no response to other drug
  6. stratify patients
  1. Protein experimental data compound design from simulations of VIRTUAL compounds,
  2. how to incentivise to take on new innovations
  1. more that one single administration by injection
  2. response rates different even in one patient let alone among patients
  3. detection gene
  4. CAR-T glioblastoma
  5. pancreatic cancer good responses in combination therapies
  6. immunr repertoire biology so complex that biomarkers are limited

Moderator: Dr. Christiana Bardon, Managing Director, MPM Capital

  • 30% patinets with complete cure

10:50 AM – 11:20 AM Networking Break11:20 AM – 12:10 PM

Panel Discussion: Future of Clinical Trials and Drug Development

Panelists:

  1. endpoints need to be redefined it effect price of drug development
  2. in Oncology – Basket and Umbrellas Trial – two stufies approval for melanoma, biomarker
  3. Is response rate is 30% va 50% and Phase 3 is negative Kertuda when worked at Merck dose ranging last phase when response dropped from 60% to 30% in the case of Study C3
  4. 30% of the cost of the study – 30% was translational
  5. CRO model appropriate oversite vs douplication of tasks
  • Dr. Bruce Chabner, Director of Clinical Research, Mass General Hospital Cancer Center
  1. Old paradigm Phase 1,2,3 – off the board now, New drugs do not need the old paradigm
  2. Phase i1 changed if genomics is involved multiple cohorts at same time
  3. FDA play amazing role
  4. patient selection is key
  5. mutations in rare disease vs mutations in cancer
  6. immunotherapy and endogenic drugs with chemo in RENAL cancer
  7. check-points – lung cancer understood money spent to find responders
  8. HOW to select which cheno therapy — no improvement today vs past
  9. 40 drugs approved by accelerated approval one came back on the market
  10. Financial burden of being in a clinical trial
  11. Foundation gives money to Institutions to reimburse patients for flights, meals, acommodation, Pharma are reluctant to participants due to potential accusation of bias id Pharma pays Patients that participate in Clinical Trials
  1. FDA recognizes approval process – systems involved AFTER approval for reimbursement and monitoring after market
  2. regulatory by countires are different
  3. which factors are sacrifiable in the long tern in clinical trial design
  1. Safety – benefit risk is what physicians work with every day
  2. Drugs paradign of small molecules does not hold is you have a drug that deliver entire organelle – how you dose for half life how you prive the rate of replication in the body
  3. Surrogate markers
  4. Taking a drug off the market ->>  conditional approvals [approval can be taken back or require additional studies] not a favorable view of Pharma in the present to support Conditional approval vs accelerated approval

 

  1. speed
  2. differentiation from competition
  3. drug development in crisis is CVD not cancer, US and the rest of the world – lowest investment in drugs is CVD
  4. Studies designed by Physicians using SAME design
  5. need to create experts to use ML in the course of clinical trial design
  6. regulators as Partners not as Barriers
  7. Proof of efficacy is a burden on the developers of the drug not on the Regulatory
  8. Increase use of advertising to recruit
  9. 70% OF PATIENTS WILLING TO PARTICIPATE  lives to far from site of trials
  10. Telecommunication between administrators of study ans clinical Trials participants
  11. Back when I was at Pfizer, designing study – patients burden relieved more willingness to participate
  12. Preferrs to run studies in house vs use CRO they are not effective in monitoring like study run in house

Moderator: Dr. William Chin, Professor of Medicine, Emeritus, Harvard Medical School

  • Probability of success to clinic has not changed
  • challenge is design and execution in clinical trials
  • changes in drug modalities: RNA, DNA,
  • which combination to use
  • how to find the many patients needed
  • Basket and Umbrellas Trial

12:10 PM – 1:00 PM

Panel Discussion: Manufacturing in the Future

Panelists:

  • Hari Bhartia, Founder and Co-Chairman, Jubilant Bhartia Group
  1. supply change
  2. blockchain
  3. quality by design
  4. CPK
  5. productivity will go up variability will decrease
  6. manufacturng must happen in India
  7. Genetics price selection
  8. Secure system, data quality the data logic and the analytics
  9. infrastructure in manufacturing is not completed yet
  10. Training by augmented reality Turnover high in India
  11. cyber security – digitization and central control
  12. demonstration data offense
  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration
  1. next 10 years India and China will improve regulatory activities and match better the US requirements
  2. review foreign hosts
  3. skills and location of hosts:
  4. India: Standards and unannounced inspections and
  5. China: same
  6. Blockchain is experienced as experimentation at FDA across each all parts of the Agency
  • Dr. Paul McKenzie, Executive Vice President, Pharma Operations & Technology, Biogen
  1. raw material to patients: Pharma very slow than other industries Reliable needs be very high, relationships
  2. Hurrican in PortoRIco affected supply chain
  3. Reality, every one HAVE to be in China
  4. Platforming for each modality for Scaling out vs Scaling up
  5. diversify vs modality x
  6. build capacity and capabilities customization of ultra filtration different in two plants lowers standardizations
  7. Training on Demand, Virtually, documnetation needs to change to electronic
  8. Continueous manufacturing Academic contribution
  • Vinay Ranade, Chief Executive Officer, Reliance Life Sciences
  1. Pharma was slow in India the manufacturing
  2. infantile diarreha vaccine 70,000 in 4 years needs that drug,
  3. massive intellectual capital in India
  4. How to implement and make best use of data to improve processes
  5. cyber security was not experiences
  1. Phase 1 scaling out vs up – it is different in vaccine field
  2. ML, Block chain, supply chain and manufacturing will be adapted in supply chain
  3. Apply analytics and relationships in manufacturing
  4. obsolescence and upgrades
  5. capture data electronically
  6. cyber security can be a hazard hard to mitigate when all systems are down
  7. significant challenges in manufacturing and data security

Moderator: Professor N. Venkat Venkatraman, Boston University Questrom School of Business

  • How can Pharma become leaner
  • heterogenuious environment for production
  • cyber security

1:00 PM – 1:50 PMLunch1:50 PM – 1:55 PM Video message from Suresh Prabhu, Hon’ble Minister of Commerce & Industry, Gov. of India1:55 PM – 2:45 PM

Panel Discussion: One in a million – Emerging trends in Rare Diseases

Panelists:

  1. worked with Academic community on how to treat rare disease in the future
  1. Show clinical benefit and impact multiplemyeloma
  2. patients becoming activists
  3. access
  4. foundation by patients
  5. Patient to get cloud
  • Dr. Dhaval Patel, Executive Vice President  and Chief Scientific Officer, UCB
  1. if a modality will cure a disease justify innovation Model for payment: Mortgage Model
  2. Access INDEX pricing – US will benchmark the price in other parts of the world
  3. Gene therapy is not only got monognenic diseases but for
  4. decrease work involved in development of drugs
  • Dr. James Wilson, Director – Gene Therapy Program, University of Pennsylvania
  1. tension between physicians and development of the perfect drug.
  2. AV
  3. Protein replacement therapy repeated infusion gene therapy infrastructure develop in China for China, Develop in India for India vs develop in US for India or China
  4. Cost of manufacturing to decrease
  • Dr. Timothy Yu, Assistant Professor in Pediatrics, Harvard Medical School
  1. Scalability beyond the one case: the mechanism for the drug has generability for other aptients iwth same mutation the method has no limit
  2. Molecular type of mutation Spice Switching strategy, just-in-time manufacturing

Moderator: Dr. Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics

  1. Rare diseases, potential for cure
  2. Academia, Hospitals, biotech
  3. commercial model of the disease

2:45 PM – 3:20 PMNetworking & Tea Break3:20 PM – 3:50 PM

Fireside Chat: Value and Access – The ongoing debate

  1. since 2003 testify in the House, against Canadian  David Brenner was asked about importation from Canada of breast cancer tamoxiphen at a lower price than in the US.
  2. From importation crisis to Obama Care – stable system Medicare Part D – drug coverage for Olderly
  3. After Obama – Price is part of doing business REBATES $100Billion the valur of REBATES
  4. Co-Insurance
  1. right for innovation will be preserved
  2. price increase
  3. give and take
  4. Co-pay – We need lower co-pay
  5. with current administration, sink finding the Well instead of Well funding the sick
  6. CHange is coming, co-pay will change
  1. Genzyme days vs 2019
  2. changes how drugs are priced?
  3. Flaws of the system:Gevernment induce prices that will change
  4. $800,000 drug is now $80 [ala Regeneron] – R&D was $2Billion
  5. CO-pay for hospital stay is lower than co-pay on drugs – 10% twice a year

Moderator: Dr Andrew Plump, President R&D, Takeda Pharmaceuticals

3:50 PM – 4:10 PM

India update on Clinical Trial Regulations

  • Arun Singhal, Additional Secretary, Ministry of Health & Family Welfare, India
  1. Each patient deserve access to healthcare in India
  2. experimenting
  • Dr Eswara Reddy, Drug Controller General of India, Central Drug Control Organization
  1. Time line for Application approval for drugs, if approved in another country 60 days
  2. Gov’t hospitals can import New drugs which have not been permitted in India

4:10 PM – 5:00 PM

Panel Discussion: Research and Development Strategies and Trends

Panelists:

  1. Neuroscience – Pharma understand biomarkers and now genetics
  2. Vaccines – across species in the animal WORLD
  1. Attempt not to tweak the PIPELINE: CVD, NEUROSCIENCE AND CANCER
  2. 485 Teams doing R&D convluence of interests to develop cure
  3. Modularity – BioMolecule — multimodality biophysical biochemical protein degradation – rewire disease cells with biomolecules combing propertitie of permiability of small molecules
  4. PHARMACOLOGICAL PREVENTION – biotech is inspiring only Pharma can solve
  1. immunooncology – mutation signature – marker protein signature — that group of diseases respond to
  2. colon cancer and multiple myeloma — understanding of the biology was deep

Moderator: Dr. Martin Mackay, Co-Founder, Rallybio

5:00 PM – 5:05 PM Closing Remarks

5:05 PM – 6:15 PM Cocktails & Networking Reception

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LIVE Day Two: 2019 MassBio Annual Meeting, State of Possible Conference, Sonesta Hotel, Cambridge, March 28, 2019

http://files.massbio.org/file/MassBio-State-Of-Possible-Conference-Agenda-Feb-22-2019.pdf

Leaders in Pharmaceutical Business Intelligence (LPBI) Group

represented by Founder & Director, Aviva Lev-Ari, PhD, RN will cover this event in REAL TIME using Social Media

@pharma_BI

@AVIVA1950

#StateofPossible19

 

Day 2 Agenda – March 28, 2019

Possible Talk – The New Role of Police: Creating Non-Arrest Pathways to Treatment and Recovery Conference Wide 3/28/2019 8:45:00 AM 3/28/2019 9:00:00 AM
Future of Human Biology: How we are Already Evolving Ourselves Conference Wide 3/28/2019 9:00:00 AM 3/28/2019 9:45:00 AM
Possible Talk – The Evolution of the Investment Landscape to Support Future Innovations in Biotech Conference Wide 3/28/2019 9:45:00 AM 3/28/2019 10:00:00 AM
CFIUS: Proposed Rules Impacting Global Investments for Biotech Breakout – Business Track 3/28/2019 10:15:00 AM 3/28/2019 11:00:00 AM
Exploring the Possibilities of Microbiome Beyond GI Diseases Breakout – Science Track 3/28/2019 10:15:00 AM 3/28/2019 11:00:00 AM
Advancing from ‘R’ to ‘D’: Various Routes to Building Successful Clinical-stage Companies Breakout – Science Track 3/28/2019 11:30:00 AM 3/28/2019 12:15:00 PM
Financing Outlook for 2019-2020: Will the Bubble Burst? Breakout – Business Track 3/28/2019 11:30:00 AM 3/28/2019 12:15:00 PM
Lunch & Henri Termeer Innovative Leadership Award Conference Wide 3/28/2019 12:15:00 PM 3/28/2019 1:15:00 PM
Enabling Digital Health in the Healthcare Value Chain Conference Wide 3/28/2019 1:15:00 PM 3/28/2019 2:00:00 PM

Day 2 Agenda – March 28, 2019

8:15am – 8:45am: Breakfast

8:45am – 9:00am: Possible Talk

Possible Talk – The New Role of Police: Creating Non-Arrest Pathways to Treatment and Recovery

  • Treatment of excellence in opiods draws patients fro all the MA state to Boston Programs and from Outside of MA
  • 94% walking on the door receive referrals to treatment centers in MA
  • Prediction of the future of persistence of the phonomenon
  • Funding partnerships for reducing time in treatment due to efficacy and patients engagement
  • 500 police personnel are involved in Treatment available on Demand referral
  • Substance abuse programs that work using institutions as problem solvers
  • Urgent and important because people die every day

 

9:00am – 9:45am: Future of Human Biology: How we are Already Evolving Ourselves

Megan Thielking, STAT News

  • Ethics of Human germ line
  • George M. Church PhD, Professor of Genetics, Harvard Medical School
  1. Multiplexing and gene editing for reverse aging
  2. Doing to much by adding new forms somatic therapies feedback loop is long from young adults to aging
  3. Small pox: Gene therapy can becaome non expensive
  • Juan Enriquez, Managing Director, Excel Venture Management, Prof. @Harvard in Genomics and Manage Start up in Genomics
  1. conception – identical twins bred 20 years apart
  2. fetus external to body and male can have children
  • Greg Verdine, President, CEO & CSO, Board Member, LifeMine
  1. Embryos IVF,, fully sequence the embroy and
  2. Democratice technologies at high school age –
  3. Educators will play a role for informed decisions public education
  4. AI harder to democratize
  5. Ethics consideration in patient selection for certification

9:45am – 10:00am: Possible Talk – 

Possible Talk – The Evolution of the Investment Landscape to Support Future Innovations in Biotech

  • communication what Biotech do, how position biotech start up and how raise fund
  • Thematic Investing: ALPHA CURRENTS: AI, Medical Technologies, communication technologies
  • Mother test – Communication of thoughts

 

10:15am – 11:00am: CFIUS: Proposed Rules Impacting Global Investment for Biotech [Business Track]

  • Review committee foreign investment, purchasing entire company –
  • new regulation 8% foreign investment is the largest allowed share as equity position and
  • not to negatively affect cyber security and national security
  • In BioTech (i.e. gene editing technologies developed already overseas and all other ecosystems
  • Green Card Holder investor are not excempt of CFIUS is different that US Citizen Investors which are exempts
  • Vikas Goyal, Principal, S. R. One, GlasxoSK Limited
  • Linda Ji, Partner, McDermott Will & Emory
  1. Disclosure about the investment and investors
  2. neurotech, genetics, genomics
  3. Critical tech in Biotech: Foundational vs
  4. US EXPERT CONTROLLED Technology
  5. License needed for licensing US EXPERT CONTROLLED Technology
  6. CFIUS does not handle licensing
  • Lisa Schaefer, Director of Financial Services and Tax Policy, Biotechnology Innovation Organization (BIO)
  1. Expert controlled technology
  2. AI in Drug discovery -is emerging foundational technology
  • Johhy Hu – VC in Boston – FUND owners are Swiss & Italian citizenship

10:15am – 11:00am: Exploring the Possibilities of Microbiome Beyond GI Diseases [Science Track]

  • David Donabedian, Co-founder, CEO & Director, Axial Biotherapeutics
  • Justin Chakma, Head of BD and Strategy, Vedanta
  • Chris Howerton, Biotechnology Equity Analyst, Jeffries

11:00am – 11:30am: Networking Break 

11:30am – 12:15pm: Advancing from ‘R’ to ‘D’: Various Routes to Building Successful Clinical-stage Companies [Science Track]

  • Laura Indolfi, Founder & CEO, PanTher Therapeutics
  • Sven Karlsson, Co-Founder, CEO,& CBO, Platelet Biogenesis
  • Leslie Williams, Director, Founder, President and CEO, ImmusanT

11:30am – 12:15pm: Financing Outlook for 2019-2020: Will the Bubble Burst? [Business Track]

  • Bruce Booth, Partner, Atlas Ventures
  1. Great time of capital rich, 2018 2Q – largest M&A in Biotech
  2. staggering increase in funding
  3. capital is flowing not from VCs, bur large hedge funds and foreign investment – China related 40% and Abu Dabi invest in Biotech
  4. First IPO in 2012 was Biotech
  5. Investor more selective and descerned : Preclinical Phase 1
  6. 2009-2010 – cool off market
  7. 2019 at constructive market for growing companies

 

  • Kathy Bersteinsson, Morgan Stanley 
  1. $30 Billion in 2018
  2. 8 IPO, back
  3. 2019 will be constructive, capital for biotech ecosysytem 50Billion needed, @25 Billion is secured without convertibles
  4. back log of IPO for 2Q in 2019
  5. investors wish 30% returns – adjust IPO valuation some are 1.5 times and fees on top
  6. Investors willing to participate VC not participate in good markets
  7. Good IPO no VCs
  8. Oncology, Selling genes, CNS
  9. Maturing the crop takes time

 

  • Christine Brennan, Partner, MRL Ventures Fund, Merck US
  1. RNA landscape
  2. oncology

 

  • Barbara Dalton, Pfizer Ventures
  1. Neurosciences, Biogen failure, need for new drugs
  2. Newrodegeneration
  3. Venture group does invest in neuro because Pfizer left Neuro
  4. investment in IMMUNITY
  5. M&A pipeline to move the needle does not happens for small biotech
  6. Pfizer and all BIG Pharma do not but even though Tax Reform favor Big Pharma

 

  • Steve Wilcox, Lawyer – largest Biotech
  1. what is hot and not
  2. Option to Purchase is lousy for sellers you cap the upper bound

 

12:15pm – 1:15pm: Lunch and MassBio Innovation Award Presentation

  • Lita Nelson, fear of genetics lead to development of 1980s legislation ownership and Patent exploitation by institutions, Universities started to collect royalties from Tech Transfer, Gov’t supported 120 Billion in basic research since the 70s.
  • 200 Research Universities and Hospitals Tech Transfer Office research shows:
  • 6300 licenses granted
  • early investment in innovations – in MA, 30 years ago no Pharma in MA today 18 of 20 big Pharma are in Cambridge and other locations in mA
  • People: accelerate biotech faced scarce talent not scarce capital
  • networking – dense clusters in Cambridge
  • Cambridge is the envy of the World in BioTech

 

1:15pm – 2:00pm: Enabling Digital Health in the Healthcare Value Chain

Moderator: Hussain Mooraj, Delloitte

  • Jeff Elton, CEO, Concerto Health AI
  • Cluster of centers of excellence
  • Moderna, VP IT
  1. cross pollination =: CS + Biology form MIT
  2. Top management understand technologies in Biotech
  3. data collection
  4. blueprint to design a digital platform – from the start to do it right vs retrofits at later stage

 

  • Luba Greenwood, Google – Verily – IP was the asset in 2019 it is the DATA  not the IP – use of data to improve drug discovery
  1. Starting up a Biotech company
  2. Advice is available

 

  • ThermoFischer – R&D DIgital Science,
  • Pear Therapeutics, Brooke Paige, VP, Alliance ManagementBrooke Page

2:00pm – 2:30pm: Keynote Address

 

2:30pm – 3:00pm: Closing Networking Dessert Reception

@@@@@

8:15am – 8:45am: Breakfast

8:45am – 9:00am: Possible Talk

9:00am – 9:45am: Future of Human Biology: How we are Already Evolving Ourselves Humans began changing our environment early on through fire, agriculture and early medicines, and later with biopharmaceuticals and even CRISPR babies. Come hear futurists in human biology offer provocative thoughts on how we can wisely and ethically embrace next generation science to change our own DNA. – George M. Church Phd, Professor of Genetics, Harvard Medical School – Juan Enriquez, Managing Director, Excel Venture Management – Greg Verdine, President, CEO & CSO, Board Member, LifeMine

9:45am – 10:00am: Possible Talk

10:15am – 11:00am: CFIUS: Will New Regulatory Threats Reduce Global Investment in Biotech? [Business Track] CFIUS’ newly expanded scope and authority have the potential to severely disrupt investment in the U.S. biotech sector. A new federal law now includes biotechnology on a list of 27 “critical” industries where all foreign investment must be reviewed by the CFIUS office. With this new rule in place, questions abound about its impact on the biotech industry. Will raising money take longer? Will it deter foreign investors? Will it limit companies’ leverage during investment negotiations? Come hear legal, investor, and company experts discuss what companies and investors need to know about CFIUS’ impact on future investments in biotech companies. – Vikas Goyal, Principal, S. R. One, Limited – Linda Ji, Partner, McDermott Will & Emory – Lisa Schaefer, Director of Financial Services and Tax Policy, Biotechnology Innovation Organization (BIO)

10:15am – 11:00am: Exploring the Possibilities of Microbiome Beyond GI Diseases [Science Track] The specific balance of bacteria in your gut affects your digestive system, but new research also shows these bacteria affect your behavior, energy, heart, brain and other unexpected organ systems. Pioneers in microbiome expound on the possibilities. – David Donabedian, Co-founder, CEO & Director, Axial Biotherapeutics – Justin Chakma, Head of BD and Strategy, Vedanta – Chris Howerton, Biotechnology Equity Analyst, Jeffries

11:00am – 11:30am: Networking Break

11:30am – 12:15pm: Advancing from ‘R’ to ‘D’: Various Routes to Building Successful Clinical-stage Companies [Science Track] Massachusetts is the epicenter of startup creation for biotech, but it’s not always easy to go from research to commercialization. Come hear founders and industry leaders offer lessons learned on intelligently scaling your company amid rapid growth including when to partner, sell or IPO, or when to build or buy preclinical, manufacturing, and clinical development capabilities. – Laura Indolfi, Founder & CEO, PanTher Therapeutics – Sven Karlsson, Co-Founder, CEO,& CBO, Platelet Biogenesis – Leslie Williams, Director, Founder, President and CEO, ImmusanT

11:30am – 12:15pm: Financing Outlook for 2019-2020: Will the Bubble Burst? [Business Track] 2018 was a record year for biotech IPOs, the XBI hit an all-time high in August, and the FDA approved a record number of novel drugs. What will 2019 bring after late 2018’s many signals of a slowing market? Will M&A pick up? Can new IPOs continue at last year’s pace? How will public markets respond? Investment bankers, private equity, and industry leaders peer into their crystal ball to predict the financial outlook for the next few years. – Bruce Booth, Partner, Atlas Ventures

12:15pm – 1:15pm: Lunch and MassBio Innovation Award Presentation

1:15pm – 2:00pm: Enabling Digital Health in the Healthcare Value Chain At its core, digital technologies are increasingly critical in enabling the growth of biotechnology companies– from the development of better research and development insight engines, to the commercialization of next generation therapies via digital channels. This panel will explore best practices that enable the embedding of digital health technologies in the value chain for both life sciences companies, payers and providers.

2:00pm – 2:30pm: Keynote Address

2:30pm – 3:00pm: Closing Networking Dessert Reception

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LIVE Day One: 2019 MassBio Annual Meeting, State of Possible Conference, Sonesta Hotel, Cambridge, March 27, 2019

http://files.massbio.org/file/MassBio-State-Of-Possible-Conference-Agenda-Feb-22-2019.pdf

Leaders in Pharmaceutical Business Intelligence (LPBI) Group

represented by Founder & Director, Aviva Lev-Ari, PhD, RN will cover this event in REAL TIME using Social Media

@pharma_BI

@AVIVA1950

#StateofPossible19

State of Possible Conference Day 1 Agenda – March 27, 2019

Session Title Session Type Start Time End Time
Breakfast & Registration 3/27/2019 8:15:00 AM 3/27/2019 8:45:00 AM
Breakfast 3/27/2019 8:15:00 AM 3/27/2019 8:45:00 AM
Keynote: State of the State of Possible: Where are we in the history of Biotech and What Lies Ahead? Conference Wide 3/27/2019 9:00:00 AM 3/27/2019 9:30:00 AM
‘Fireside Chat’ on RNAi and CRISPR: What Can the Past Teach Us and What Opportunities Await? Conference Wide 3/27/2019 9:30:00 AM 3/27/2019 10:15:00 AM
Possible Talk – The Possibilities of Gene Editing for Transforming the Practice of Medicine Conference Wide 3/27/2019 10:15:00 AM 3/27/2019 10:30:00 AM
Possible Talk – Leveraging Artificial Intelligence to Revolutionize Drug Discovery Conference Wide 3/27/2019 11:00:00 AM 3/27/2019 11:15:00 AM
Solving the Value Equation to Fix our Broken Healthcare System & Ensure Patient Access Conference Wide 3/27/2019 11:15:00 AM 3/27/2019 12:00:00 PM
Possible Talk: Opening Doors for the Future, a Patient’s Perspective Conference Wide 3/27/2019 12:00:00 PM 3/27/2019 12:15:00 PM
Early Stage Funding in the Era of Venture Creation Breakout – Business Track 3/27/2019 2:00:00 PM 3/27/2019 3:00:00 PM
From Academia to Commercialization: What’s the Threshold of Data Needed to Start a New Company? Breakout – Science Track 3/27/2019 2:00:00 PM 3/27/2019 3:00:00 PM
The Evolving Massachusetts Ecosystem: How Can we Continue to Sustain our Growth? Breakout – Business Track 3/27/2019 3:00:00 PM 3/27/2019 4:00:00 PM
Addressing the challenges of Large Population Diseases. Breakout – Science Track 3/27/2019 3:00:00 PM 3/27/2019 4:00:00 PM
The State of Neurodegenerative Disease: Drug Targets and Alternative, Patient-Centric Clinical Endpo Breakout – Science Track 3/27/2019 4:30:00 PM 3/27/2019 5:30:00 PM
Addressing the Opioid Crisis Breakout – Business Track 3/27/2019 4:30:00 PM 3/27/2019 5:30:00 PM
 

The State of Possible Celebration 3/27/2019 6:00:00 PM 3/27/2019 9:00:00 PM

 

Day 1 Agenda – March 27, 2019

8:45am – 9:00am: Welcome

9:00am – 9:30am: Keynote – State of the State of Possible: Where are we in the History of Biotech and What Lies Ahead?

  • Jeremy Levin, Chairman & CEO, Ovid Therapeutics, Inc.

No way to predict the future: rich and diverse, existential moment

  • impact of life expectancy
  • FDA in 2018 – record of Dr. Gottlieb in approval of new drugs
  • capital is flowing in LARGE amounts into innovations: new medical modalities: Gene therapy, immune oncology, RNA, CAR-T – 2500 clinical trials in immune oncology – product emerging as BIOSIMILARS
  • cell therapy, gene therapy
  • Health Insurers matters, consolidation, CIGNA partners with pharma
  • Insulin is $400 patients that can’t pay — covenant: Health Care must be universal for Economic growth considerations
  • Innovators part of the pie shrinks
  • Drug Pricing shifts the political balance
  • Bipartisan Pressure on the Pharma industry
  • WHAT IS COMING
  1. consolidations
  2. massive innovations
  3. innovations is essential – Financial burden
  4. CNS innovations – Alzermer’s Disease: Epilepsy,
  5. BIOSIMILARS: Crushing wave will create monopolies on OLD molecules PD!
  6. Organizational changes: Patient centricity using AI and precision
  7. Investors in Biotech in 2007 investors did not believe in immuno-oncology
  8. Pricing controversy – access and affordalitiry of cost out of pocket
  9. Bad democracy: if access is not to all
  10. CEO’s compensation need be tied to product innovations not to drug pricing
  11. IP Protection
  12. BIOTECH – matters: Sustainability if patient is the purpose
  13. Democracy is by all and for all

9:30am – 10:15am: ‘Fireside Chat’ on RNAi and CRISPR: What Can the Past Teach Us and What Opportunities Await?

Mike, Nikitas, Moderator

  • John Maraganore, Chief Executive Officer, Alnylam
  1. Tissue to benefit from RNA therapies only the beginning, not yet for cancer
  2. FDA has amazing scientists to continue Dr. Scott Gottlieb’s legacy
  3. Patients at the center and the Team move Science forward

Katrine Bosley, former Chief Executive Officer, Editas MedicineGenetic repair by gene deletions – as advances are achieved knowledge is shared cross projects on multiple indications

    1. regulators need to move faster as they see the innovations for tissue by gene medicine
    2. Dialog with FDA will continue Human germ line is NOT appropriate

10:15am – 10:30am: Possible Talk – Morgan Stanley

Possible Talk – The Possibilities of Gene Editing for Transforming the Practice of Medicine

Base Editing – Repair DNA

Creation of a new base editor – platform creation – platform to enable development – mammal cell lines

  • Cytidine Base Editor: (CxG -> TxA)
  • ApoE4 genetic factor fo rAlzheimer’s DIsease
  • Adenine Base Editor: (AxT ->GxC??)
  • Substrate specificity, delivery, product selectivity
  • @ BEAM Base editing as a Human Therapy 0 genetic disease faulty gene expression
  1. Gene correction,
  2. Gene regulation,
  3. Gene silencing,
  4. Gene reprogramming

Treating genetic disease ONE LETTER AT A TIME

10:30am – 11:00am: Networking Break 

11:00am – 11:15am: Possible Talk

Possible Talk – Leveraging Artificial Intelligence to Revolutionize Drug Discovery

  • Historical evolution of AI started on the 50s
  • Is Pharma ready for AI?
  • Publications on AI in Drug Discovery is th elargest growth in Publishing
  • Pharma Corporations, AI conpanies, Tech corporations
  • Potential contributions of AI to Human Biology

 

11:15am – 12:00pm: Solving the Value Equation to Fix our Broken Healthcare System & Ensure Patient Access

Margaux Hall, Moderator

  • Erin Mistry, Managing Director Pricing and Access, Syneos Health
  • Mike Bonney, Executive Chair, Kaleido Lifesciences
  1. Payers need to change their system for each new product they will reimburse for – IT system change
  2. Value based scheme are challenging due to data collections need by Payer to define contracts for VALUE definition and contract terms
  3. Will the value change on the next year is more enrolled as users?
  4. value-based constructs of therapeutics: population impact when patients start a given therapy – research shows that change in x yield favorable outcomes thus, approval of drug coverage by Payer  – ALWAYS have population effects
  5. Payors can loose cohorts and gain other cohorts by incentives
  6. Healthcare policies must be long term and not by Party election peril ti the Healthcare system
  7. Show progress that business strategy bring scoring on outcomes
  8. Saving life of patients, it is expensive, then it becomes free almost for developing countries, like vaccines and generics
  • Tamar Thompson, Executive Director, State Government Affairs & Federal Payment, Bristol-Myers Squibb
  1. pay less if pay cash without insurance vs pay insurance and pay co-pay — ?? affordability and gaining it fairly

12:00pm – 12:15pm: Possible Talk

Possible Talk: Opening Doors for the Future, a Patient’s Perspective

Make a change in life of Patients every day

12:15pm – 12:45pm: MassBio Presidential Address – Robert K. Coughlin, President and CEO, MassBio

12:45pm – 2:00pm: Lunch

2:00pm – 3:00pm: From Academia to Commercialization: What’s the Threshold of Data Needed to Start a New Company? [Science Track]

  • Irene Abrams, Vice President, Technology Development and New Ventures, Boston Children’s Hospital
  • Irena Ivanovska, Boston Site Head, Celdara Medical
  • Issi Rozen, Chief Business Officer, Broad Institute

2:00pm – 3:00pm: Early Stage Funding in the Era of Venture Creation [Business Track]

  • Todd Zion ex-Smart Cell, CEO
  • Paula Ragan, CEO, X4Pharmaceuticals
  • Rosemary Reilly, Partner, WilmerHale
  • Ramani Varanasi, CEO, X-Biotix

3:00pm – 4:00pm: Addressing the Challenges of Large Population Diseases [Science Track]

  • Calum MacRae, Vice Chair for Scientific Innovation, Brigham and Women’s Hospital, Harvard Medical School

3:00pm – 4:00pm: The Evolving Massachusetts Ecosystem: How Can we Continue to Sustain our Growth? [Business Track]

  • Tanisha Sullivan, Associate General Counsel, Industrial Affairs, Sanofi Genzyme
  • Doug Fambrough
  • Chandra Ramanthan
  • Johannes Fruehauf, Founder & CEO, LabCentral

4:00pm – 4:30pm: Networking Break 

4:30pm – 5:30pm: The State of Neurodegenerative Disease: Drug Targets & Alternative, Patient-Centric Clinical Endpoints [Science Track]

  • Robert Alexander, Vice President and Head, Global Clinical Science Neuroscience, Takeda
  • Joseph J. Higgins, MD FAAN, Vice President, Clinical Development, Huntington’s Disease Program Lead, Uniqure
  • John Torous, Director, Digital Psychiatry Division, Beth Israel Deaconess Medical Center
  • Sally Johnson, Biogen
  • Gilmore O”Neill, Serepta
  • Steve Wood – biomarkers (are LAB specific not regulated by FDA)
  • Ecosystem will continue to make bets, concept of POOLING FUNDING for bedding in paralllel and develop in parallel several therapeutic targets
  • Upside – investment will flow, Novertis and AztraZeneca joined funding on one development
  • Placing bets on Neurological diseases
  • neurogeneration of inherited diseases
  • Earlier intervention is better
  • target identification AUGMENTATION VS REMOVAL not clear what
  • Biogen measuring SNIPS Partnerships Academia and Industry

4:30pm – 5:30pm: Addressing the Opioid Crisis [Business Track]

  • Kerry Wentworth, Chief Regulatory Officer, Flexion Therapeutics

6:00pm – 9:00pm: State of Possible Celebration at the Museum of Science

Join us for drinks, food and networking at the Museum of Science! 

@@@@

8:45am – 9:00am: Welcome

9:00am – 9:30am: Keynote – State of the State of Possible: Where are we in the History of Biotech and What Lies Ahead? – Jeremy Levin, Chairman & CEO, Ovid Therapeutics, Inc.

9:30am – 10:15am: ‘Fireside Chat’ on RNAi and CRISPR: What Can the Past Teach Us and What Opportunities Await? Although researchers have been experimenting with gene therapy since the 1980s, 2018 was a major turning point in making these technologies a reality for patients. Not only was the first therapeutic using RNA interference (RNAi) approved last year, the first human clinical trials started for a CRISPR product. Come hear the two superstars of RNAi and CRISPR compare stories, lessons learned, thoughts on the future of gene editing, and more. – John Maraganore, Chief Executive Officer, Alnylam – Katrine Bosley, former Chief Executive Officer, Editas Medicine

10:15am – 10:30am: Possible Talk – Morgan Stanley

10:30am – 11:00am: Networking Break

11:00am – 11:15am: Possible Talk

11:15am – 12:00pm: Solving the Value Equation to Fix our Broken Healthcare System & Ensure Patient Access FDA approval used to mean access – but that’s no longer the case. More insurers are restricting access to new drugs, all at the expense of patients, using the argument that they do not bring enough value to the healthcare system. But what does value really mean? Experts in pricing and market access offer thoughts on how we might solve the value equation to improve patient access while continuing to reward innovation. – Erin Mistry, Managing Director Pricing and Access, Syneos Health – Mike Bonney, Executive Chair, Kaleido Lifesciences – Tamar Thompson, Executive Director, State Government Affairs & Federal Payment, Bristol-Myers Squibb

12:00pm – 12:15pm: Possible Talk

12:15pm – 12:45pm: MassBio Presidential Address – Robert K. Coughlin, President and CEO, MassBio

12:45pm – 2:00pm: Lunch

2:00pm – 3:00pm: From Academia to Commercialization: What’s the Threshold of Data Needed to Start a New Company? [Science Track] In 2012, Nature famously published a review by a former Amgen head of oncology research that 10% of landmark studies could not be reproduced. Company founders and initial investors must be certain that the academic work underpinning the foundational science and licensed patents are grounded in a sufficiently robust body of multiple lines of evidence can be reproduced. Serial founders and prolific professors discuss how to be sure initial scientific results are both fundable and can be advanced to “pharma grade”. – Irene Abrams, Vice President, Technology Development and New Ventures, Boston Children’s Hospital – Irena Ivanovska, Boston Site Head, Celdara Medical – Issi Rozen, Chief Business Officer, Broad Institute

2:00pm – 3:00pm: Early Stage Funding in the Era of Venture Creation [Business Track] Over the past decade, new biotech company creation has shifted away from investing in entrepreneurs to the venture creation model, where venture investors themselves assess an unmet medical need and create a company to address it. Yet, companies continue to spawn outside the venture creation model. Come hear experts discuss how they’ve raised money outside of the venture creation model through new sources of capital. – Paula Ragan, CEO, X4Pharmaceuticals – Rosemary Reilly, Partner, WilmerHale – Ramani Varanasi, CEO, X-Biotix

3:00pm – 4:00pm: Addressing the challenges of Large Population Diseases. [Science Track] As we better understand the genetics of disease, we can increasingly treat the underlying disease mechanisms rather than the superficial disease etiology. Biomarker testing toward improved drug effectiveness in defined sub-groups have made this a reality. Unlike oncology, where this concept is now mainstream, how might precision medicine apply to large patient populations like cardiovascular and neurodegenerative diseases? This panel will discuss the challenges and opportunities for precision medicine in these widespread maladies. – Calum MacRae, Vice Chair for Scientific Innovation, Brigham and Women’s Hospital, Harvard Medical School

3:00pm – 4:00pm: The Evolving Massachusetts Ecosystem: How Can we Continue to Sustain our Growth? [Business Track] Massachusetts is the #1 life sciences cluster in the world, in large part due to the collaboration and partnership among academia, industry, and government. The vibrant startup scene, along with a wealth of talent, is keeping and attracting big pharma and investors to the area, creating a collaborative environment unlike anywhere else. Yet, major challenges face the industry’s continued success in Massachusetts, from workforce development to sky-high rents to traffic. Come hear Massachusetts leaders discuss how the ecosystem is evolving and what we must do to remain on top. – Tanisha Sullivan, Associate General Counsel, Industrial Affairs, Sanofi Genzyme – Johannes Fruehauf, Founder & CEO, LabCentral

4:00pm – 4:30pm: Networking Break

4:30pm – 5:30pm: The State of Neurodegenerative Disease: Drug Targets & Alternative, Patient-Centric Clinical Endpoints [Science Track] Despite massive investments in research for diseases like Alzheimer’s and Parkinson’s, treatment options for patients are largely non-existent. ‘Soft’ clinical endpoints like Cognitive Impression scores are difficult to demonstrate statistical significance even when underlying biology is being modified. Industry and practicing clinicians share how they are introducing modern imaging and biomarker measurements as ‘harder’ endpoints to the FDA, along with including diverse patients in earlier trials to better predict large pivotal trials. – Robert Alexander, Vice President and Head, Global Clinical Science Neuroscience, Takeda – Joseph J. Higgins, MD FAAN, Vice President, Clinical Development, Huntington’s Disease Program Lead, Uniqure – John Torous, Director, Digital Psychiatry Division, Beth Israel Deaconess Medical Center 4:30pm – 5:30pm: Addressing the Opioid Crisis [Business Track] Pain treatment remains a pressing need, but opioid abuse grew by >10%, killing nearly 50,000 Americans in 2018. Despite decades in the spotlight, how can policy and law enforcement, medical providers and the biopharma industry come together to reverse the trend?

6:00pm – 9:00pm: State of Possible Celebration at the Museum of Science Join us for drinks, food and networking at the Museum of Science!

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Record Innovations in Drug Discovery by Koch Institute @MIT Members and Affiliates

Reporter: Aviva Lev-Ari, PhD, RN

 

 

In Good Company

Trovagene announced a new patent for the use of the drug onvansertib in combination with other anti-androgen drugs for the treatment of prostate cancer. Last fall, Trovagene secured exclusive rights to develop combination therapies and clinical biomarkers for prostate cancer based in part on Bridge Project-funded research. Read more.

Lyndra Therapeutics, co-founded by KI member Bob Langer, raised $55 million in its Series B round, with new investors including the Bill and Melinda Gates Foundation and Gilead Sciences. Phase 2 trials for its ultra long-acting drug delivery capsule are expected to begin next year. Read more.

Dragonfly Therapeutics, co-founded by KI director Tyler Jacks, has committed $10 million to launch the first clinical studies of its TriNKETs (Tri-specific, NK cell Engager Therapies) platform for both solid tumor and hematological cancers. Read more.

Following its record-breaking IPO, Moderna Therapeutics (co-founded by KI member Bob Langer) published preclinical data in Science Translational Medicine demonstrating the promise of its mRNA-2752 program in several cancers. Read more.

Dewpoint Therapeutics launched with a $60 million Series A, aims to translate recent insights into biomolecular condensates from the laboratory of co-founder and KI member Rick Young to drug discovery. Read more.

KI member Bob Langer and collaborator Omid Farokhzad co-founded Seer— combining nanotechnology, protein chemistry, and machine learning—to develop liquid biopsy tests for the early detection of cancer and other diseases. Read more.

Epizyme, co-founded by KI member Bob Horvitz, is submitting a New Drug Application to gain accelerated approval of tazemetostat for patients with relapsed or refractory follicular lymphoma. Read more.

Ribon Therapeutics, founded by former KI member Paul Chang, launched with $65 million in a Series B funding round with Victoria Richon, a veteran of Sanofi and Epizyme, at the helm. Ribon focuses on developing PARP7 inhibitors for cancer treatment. Read more.

SOURCE

From: MIT Koch Institute for Integrative Cancer Research <cancersolutions=mit.edu@cmail19.com> on behalf of MIT Koch Institute for Integrative Cancer Research <cancersolutions@mit.edu>

Reply-To: <ki-communications@mit.edu>

Date: Wednesday, February 6, 2019 at 3:15 PM

To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: Lung Microbiome Corrupted in Cancer; Angelika Amon wins 2019 Vilcek Award; Lunch Lines of Inquiry

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37th Annual J.P. Morgan HEALTHCARE CONFERENCE: News at #JPM2019 for Jan. 10, 2019: Deals and Announcements

Reporter: Stephen J. Williams, Ph.D.

From Biospace.com

 

JP Morgan Healthcare Conference Update: Sage, Mersana, Shutdown Woes and Babies

Speaker presenting to audience at a conference

With the J.P. Morgan Healthcare Conference winding down, companies remain busy striking deals and informing investors about pipeline advances. BioSpace snagged some of the interesting news bits to come out of the conference from Wednesday.

SAGE Therapeutics – Following a positive Phase III report that its postpartum depression treatment candidate SAGE-217 hit the mark in its late-stage clinical trial, Sage Therapeutics is eying the potential to have multiple treatment options available for patients. At the start of J.P. Morgan, Sage said that patients treated with SAGE-217 had a statistically significant improvement of 17.8 points in the Hamilton Rating Scale for Depression, compared to 13.6 for placebo. The company plans to seek approval for SAGE-2017, but before that, the FDA is expected to make a decision on Zulresso in March. Zulresso already passed muster from advisory committees in November, and if approved, would be the first drug specifically for postpartum depression. In an interview with the Business Journal, Chief Business Officer Mike Cloonan said the company believes there is room in the market for both medications, particularly since the medications address different patient populations.

 

Mersana Therapeutics – After a breakup with Takeda Pharmaceutical and the shelving of its lead product, Cambridge, Mass.-based Mersana is making a new path. Even though a partial clinical hold was lifted following the death of a patient the company opted to shelve development of XMT-1522. During a presentation at JPM, CEO Anna Protopapas noted that many other companies are developing therapies that target the HER2 protein, which led to the decision, according to the Boston Business Journal. Protopapas said the HER2 space is highly competitive and now the company will focus on its other asset, XMT-1536, an ADC targeting NaPi2b, an antigen highly expressed in the majority of non-squamous NSCLC and epithelial ovarian cancer. XMT-1536 is currently in Phase 1 clinical trials for NaPi2b-expressing cancers, including ovarian cancer, non-small cell lung cancer and other cancers. Data on XMT-1536 is expected in the first half of 2019.

Novavax – During a JPM presentation, Stan Erck, CEO of Novavax, pointed to the company’s RSV vaccine, which is in late-stage development. The vaccine is being developed for the mother, in order to protect an infant. The mother transfers the antibodies to the infant, which will provide the baby with protection from RSV in its first six months. Erck called the program historic. He said the Phase III program is in its fourth year and the company has vaccinated 4,636 women. He said they are tracking the women and the babies. Researchers call the mothers every week through the first six months of the baby’s life to acquire data. Erck said the company anticipates announcing trial data this quarter. If approved, Erck said the market for the vaccine could be a significant revenue driver.

“You have 3.9 million birth cohorts and we expect 80 percent to 90 percent of those mothers to be vaccinated as a pediatric vaccine and in the U.S. the market rate is somewhere between $750 million and a $1 billion and then double that for worldwide market. So it’s a large market and we will be first to market in this,” Erck said, according to a transcript of the presentation.

Denali Therapeutics – Denali forged a collaboration with Germany-based SIRION Biotech to develop gene therapies for central nervous disorders. The two companies plan to develop adeno-associated virus (AAV) vectors to enable therapeutics to cross the blood-brain barrier for clinical applications in neurodegenerative diseases including Parkinson’s, Alzheimer’s disease, ALS and certain other diseases of the CNS.

AstraZeneca – Pharma giant AstraZeneca reported that in 2019 net prices on average across the portfolio will decrease versus 2018. With a backdrop of intense public and government scrutiny over pricing, Market Access head Rick Suarez said the company is increasing its pricing transparency. Additionally, he said the company is looking at new ways to price drugs, such as value-based reimbursement agreements with payers, Pink Sheet reported.

Amarin Corporation – As the company eyes a potential label expansion approval for its cardiovascular disease treatment Vascepa, Amarin Corporation has been proactively hiring hundreds of sales reps. In the fourth quarter, the company hired 265 new sales reps, giving the company a sales team of more than 400, CEO John Thero said. Thero noted that is a label expansion is granted by the FDA, “revenues will increase at least 50 percent over what we did in the prior year, which would give us revenues of approximate $350 million in 2019.”

Government Woes – As the partial government shutdown in the United States continues into its third week, biotech leaders at JPM raised concern as the FDA’s carryover funds are dwindling. With no new funding coming in, reviews of New Drug Applications won’t be able to continue past February, Pink Sheet said. While reviews are currently ongoing, no New Drug Applications are being accepted by the FDA at this time. With the halt of NDA applications, that has also caused some companies to delay plans for an initial public offering. It’s hard to raise potential investor excitement without the regulatory support of a potential drug approval. During a panel discussion, Jonathan Leff, a partner at Deerfield Management, noted that the ongoing government shutdown is a reminder of how “overwhelmingly dependent the whole industry of biotech and drug development is on government,” Pink Sheet said.

Other posts on the JP Morgan 2019 Healthcare Conference on this Open Access Journal include:

#JPM19 Conference: Lilly Announces Agreement To Acquire Loxo Oncology

36th Annual J.P. Morgan HEALTHCARE CONFERENCE January 8 – 11, 2018

37th Annual J.P. Morgan HEALTHCARE CONFERENCE: #JPM2019 for Jan. 8, 2019; Opening Videos, Novartis expands Cell Therapies, January 7 – 10, 2019, Westin St. Francis Hotel | San Francisco, California

37th Annual J.P. Morgan HEALTHCARE CONFERENCE: News at #JPM2019 for Jan. 8, 2019: Deals and Announcements

 

Read Full Post »


37th Annual J.P. Morgan HEALTHCARE CONFERENCE: News at #JPM2019 for Jan. 8, 2019: Deals and Announcements

Reporter: Stephen J. Williams, Ph.D.

From Biospace.com

JP Morgan Healthcare Conference Update: FDA, bluebird, Moderna and the Price of Coffee

Researcher holding test tube up behind circle of animated research icons

Tuesday, January 8, was another busy day in San Francisco for the JP Morgan Healthcare Conference. One interesting sideline was the idea that the current government shutdown could complicate some deals. Kent Thiry, chief executive officer of dialysis provider DaVita, who is working on the sale of its medical group to UnitedHealth Group this quarter, said, “We couldn’t guarantee that even if the government wasn’t shut down, but we and the buyer are both working toward that goal with the same intensity if not more.”

And in a slightly amusing bit of synchrony, U.S.Food and Drug Administration (FDA)Commissioner Scott Gottlieb’s keynote address that was delivered by way of video conference from Washington, D.C., had his audio cut out in the middle of the presentation. Gottlieb was talking about teen nicotine use and continued talking, unaware that his audio had shut off for 30 seconds. When it reconnected, the sound quality was reportedly poor.

Click to search for life sciences jobs

bluebird bio’s chief executive officer, Nick Leschlygave an update of his company’s pipeline, with a particular emphasis on a proposed payment model for its upcoming LentiGlobin, a gene therapy being evaluated for transfusion-dependent ß-thalassemia (TDT). The gene therapy is expected to be approved in Europe this year and in the U.S. in 2020. Although the price hasn’t been set, figures up to $2.1 million per treatment have been floated. Bluebird is proposing a five-year payment program, a pledge to not raise prices above CPI, and no costs after the payment period.

Eli Lilly’s chief executive officer David Ricks, just days after acquiring Loxo Oncologyoffered up projections for this year, noting that 45 percent of its revenue will be created by drugs launched in 2015. Those include Trulicity, Taltz and Verzenio. The company also expects to launch two new molecular entities this year—nasal glucagons, a rescue medicine for high blood sugar (hyperglycemia), and Lasmiditan, a rescue drug for migraine headaches.

CNBC’s Jim Cramer interviewed Allergan chief executive officer Brent Saunders, in particular discussing the fact the company’s shares traded in 2015 for $331.15 but were now trading for $145.60. Cramer noted that the company’s internal fundamentals were strong, with multiple pipeline assets and a strong leadership team. Some of the stock problems are related to what Saunders said were “unforced errors,” including intellectual property rights to Restasis, its dry-eye drug, and Allergan’s dubious scheme to protect those patents by transferring the rights to the Saint Regis Mohawk Tribe in New York. On the positive side, the company’s medical aesthetics portfolio, dominated by Botox, is very strong and the overall market is expected to double.

One of the big areas of conversation is so-called “flyover tech.” Biopharma startups are dominant in Boston and in San Francisco, but suddenly venture capital investors have realized there’s a lot going on in between. New York City-based Radian Capital, for example, invests exclusively in markets outside major U.S. cities.

“At Radian, we partner with entrepreneurs who have built their businesses with a focus on strong economics rather than growth at all costs,” Aly Lovett, partner at Radian, told The Observer. “Historically, given the amount of money required to stand up a product, the software knowledge base, and coastal access to capital, health start-ups were concentrated in a handful of cities. As those dynamics have inverted and as the quality of living becomes a more important factor in attracting talent, we’re not seeing a significant increase in the number of amazing companies being built outside of the Bay Area.”

“Flyover companies” mentioned include Bind in Minneapolis, Minnesota; Solera Health in Phoenix, Arizona; ClearDATA in Austin, Texas; Healthe, in Eden Prairie, Minnesota; HistoSonics in Ann Arbor, Michigan; and many others.

Only a month after its record-breaking IPO, Moderna Therapeutics’ chief executive officer Stephane Bancelspent time both updating the company’s clinical pipeline and justifying the company’s value despite the stock dropping off 26 percent since the IPO. Although one clinical program, a Zika vaccine, mRNA-1325, has been abandoned, the company has three new drugs coming into the clinic: mRNA-2752 for solid tumors or lymphoma; mRNA-4157, a Personalized Cancer Vaccine with Merck; and mRNA-5671, a KRAS cancer vaccine. The company also submitted an IND amendment to the FDA to add an ovarian cancer cohort to its mRNA-2416 program.

One interesting bit of trivia, supplied on Twitter by Rasu Shrestha, chief innovation officer for the University of Pittsburgh Medical Center, this year at the conference, 33 female chief executive officers were presenting corporate updates … compared to 19 men named Michael. Well, it’s a start.

And for another bit of trivia, Elisabeth Bik, of Microbiome Digest, tweeted, “San Francisco prices are so out of control that one hotel is charging the equivalent of $21.25 for a cup of coffee during a JPMorgan conference.”

Other posts on the JP Morgan 2019 Healthcare Conference on this Open Access Journal include:

#JPM19 Conference: Lilly Announces Agreement To Acquire Loxo Oncology

36th Annual J.P. Morgan HEALTHCARE CONFERENCE January 8 – 11, 2018

37th Annual J.P. Morgan HEALTHCARE CONFERENCE: #JPM2019 for Jan. 8, 2019; Opening Videos, Novartis expands Cell Therapies, January 7 – 10, 2019, Westin St. Francis Hotel | San Francisco, California

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