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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

 

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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

Read Full Post »


NIH SBIR Funding Early Ventures: September 26, 2018 sponsored by Pennovation

Stephen J. Williams PhD, Reporter

Penn Center for Innovation (Pennovation) sponsored a “Meet with NCI SBIR” program directors at University of Pennsylvania Medicine Smilow Center for Translational Research with a presentation on advice on preparing a successful SBIR/STTR application to the NCI as well as discussion of NCI SBIR current funding opportunities.   Time was allotted in the afternoon for one-on-one discussions with NCI SBIR program directors.

To find similar presentations and one-on-one discussions with NCI/SBIR program directors in an area nearest to you please go to their page at:

https://sbir.cancer.gov/newsevents/events

For more complete information on the NCI SBIR and STTR programs please go to their web page at: https://sbir.cancer.gov/about

A few notes from the meeting are given below:

  • In 2016 the SBIR/STTR 2016 funded $2.5 billion (US) of early stage companies; this is compared to the $6.6 billion invested in early  stage ventures by venture capital firms so the NCI program is very competitive with alternate sources of funding
  • It was stressed that the SBIR programs are flexible as far as ownership of a company; SBIR allows now that >50% of the sponsoring company can be owned by other ventures;  In addition they are looking more favorably on using outside contractors and giving leeway on budgetary constraints so AS THEY SUGGEST ALWAYS talk to the program director about any questions you may have well before (at least 1 month) you submit. More on eligibility criteria is found at: https://sbir.cancer.gov/about/eligibilitycriteria
  • STTR should have strong preliminary data since more competitive; if don’t have enough go for  an R21 emerging technologies grant which usually does not require preliminary data
  • For entities outside the US need a STRONG reason for needing to do work outside the US

Budget levels were discussed as well as  the waiver program, which allows for additional funds to be requested based on criteria set by NCI (usually for work that is deemed high priority or of a specialized nature which could not be covered sufficiently under the standard funding limits) as below:

Phase I: 150K standard but you can get waivers for certain work up to 300K

Phase II: 1M with waiver up to 2M

Phase IIB waiver up to 4M

You don’t need to apply for the waiver but grant offices may suggest citing a statement requesting a waiver as review panels will ask for this information

Fast Track was not discussed in the presentation but for more information of the Fast Track program please visit the website  

NCI is working hard to cut review times to 7 months between initial review to funding however at beginning of the year they set pay lines and hope to fund 50% of the well scored grants

NCI SBIR is a Centralized system with center director and then program director with specific areas of expertise: Reach out to them

IMAT Program and Low-Resource Setting new programs more suitable for initial studies and also can have non US entities

Phase IIB Bridge funding to cross “valley of death” providing up to 4M for 2-3 years: most were for drug/biological but good amount for device and diagnostics

 

Also they have announced administrative supplements for promoting diversity within a project: can add to the budget

FY18 Contracts Areas

3 on biotherapies

2 imaging related

2 on health IT

4 on radiation therapy related: NOTE They spent alot of time discussing the contracts centered on radiation therapy and seems to be an area of emphasis of the NCI SBIR program this year

4 other varied topics

 

Breakdown of funding

>70% of NCI SBIR budget went to grants (for instance Omnibus grants); about 20-30% for contracts; 16% for phase I and 34 % for phase II ;

ALSO the success rate considerably higher for companies that talk to the program director BEFORE applying than not talking to them; also contracts more successful than Omnibus applications

Take Advantage of these useful Assistance Programs through the NIH SBIR Program (Available to all SBIR grantees)

NICHE ASSESSMENT Program

From the NCI SBIR website:

The Niche Assessment Program is designed to help small businesses “jump start” their commercialization efforts. All active HHS (NIH, CDC, FDA) SBIR/STTR Phase I awardees and Phase I Fast-Track awardees (by grant or contract) are eligible to apply. Registration is on a first-come, first-serve basis!

The Niche Assessment Program provides market insight and data that can be used to help small businesses strategically position their technology in the marketplace. The results of this program can help small businesses develop their commercialization plans for their Phase II application, and be exposed to potential partners. Services are provided by Foresight Science & Technology of Providence, RI.

Technology Niche Analyses® (TNA®) are provided by Foresight, for one hundred and seventy-five (175), HHS SBIR/STTR Phase I awardees. These analyses assess potential applications for a technology and then for one viable application, it provides an assessment of the:

  1. Needs and concerns of end-users;
  2. Competing technologies and competing products;
  3. Competitive advantage of the SBIR/STTR-developed technology;
  4. Market size and potential market share (may include national and/or global markets);
  5. Barriers to market entry (may include but is not limited to pricing, competition, government regulations, manufacturing challenges, capital requirements, etc.);
  6. Market drivers;
  7. Status of market and industry trends;
  8. Potential customers, licensees, investors, or other commercialization partners; and,
  9. The price customers are likely to pay.

Commercialization Acceleration Program  (CAP)

From the NIH SBIR website:

NIH CAP is a 9-month program that is well-regarded for its combination of deep domain expertise and access to industry connections, which have resulted in measurable gains and accomplishments by participating companies. Offered since 2004 to address the commercialization objectives of companies across the spectrum of experience and stage, 1000+ companies have participated in the CAP. It is open only to HHS/NIH SBIR/STTR Phase II awardees, and 80 slots are available each year. The program enables participants to establish market and customer relevance, build commercial relationships, and focus on revenue opportunities available to them.

I-Corps Program

The I-Corps program provides funding, mentoring, and networking opportunities to help commercialize your promising biomedical technology. During this 8-week, hands-on program, you’ll learn how to focus your business plan and get the tools to bring your treatment to the patients who need it most.

Program benefits include:

  • Funding up to $50,000 to cover direct program costs
  • Training from biotech sector experts
  • Expanding your professional network
  • Building the confidence and skills to create a comprehensive business model
  • Gaining years of entrepreneurial skills in only weeks.

 

ICORPS is an Entrepreneurial Program (8 week course) to go out talk to customers, get assistance with business models, useful resource which can guide the new company where they should focus on for the commercialization aspect

THE NCI Applicant Assistance Program (AAP)

The SBIR/STTR Applicant Assistance Program (AAP) is aimed at helping eligible small R&D businesses and individuals successfully apply for Phase I SBIR/STTR funding from the National Cancer Institute (NCI), National Institute for Neurological Disorders and Stroke (NINDS), National Heart, Lung and Blood Institute (NHLBI). Participation in the AAP will be funded by the NCI, NINDS, and NHLBI with NO COST TO PARTICIPANTS. The program will include the following services:

  • Needs Assessment/Small Business Mentoring
  • Phase I Application Preparation Support
  • Application Review
  • Team/Facilities Development
  • Market Research
  • Intellectual Property Consultation

For more details about the program, please refer to NIH Notice NOT-CA-18-072.

 

These programs are free for first time grant applicants and must not have been awarded previous SBIR

Peer Learning Webinar Series goal to improve peer learning .Also they are starting to provide Regulatory Assistance (see below)

NIH also provides Mentoring programs for CEOS and C level

Application tips

  1. Start early: and obtain letters of collaboration
  2. Build a great team: PI multi PI, consider other partners to fill gaps (academic, consultants, seasoned entrepreneurs (don’t need to be paid)
  3. They will pre review 1 month before due date, use NIH Project Reporter to view previous funded grants
  4. Specify study section in SF to specify areas of expertise for review
  5. Specific aims are very important; some of the 20 reviewers focus on this page (describes goals and milestones as well; spend as much time on this page as the rest of the application
  6. Letters of support from KOLs are important to have; necessary from consultants and collaborators; helpful from clinicians
  7. Have a phase II commercialization plan
  8. Note for non US clinical trials:  They will not fund nonUS clinical trials; the company must have a FWA
  9. SBIR budgets defined by direct costs; can request a 7% fee as an indirect cost; and they have a 5,000 $ technical assistance program like regulatory consultants but if requested can’t participate in NIH technical assistance programs so most people don’t apply for TAP

 

  • They are trying to change the definition of innovation as also using innovative methods (previously reviewers liked tried and true methodology)

10.  before you submit solicit independent readers

NCI SBIR can be found on Twitter @NCIsbir ‏

Discussion with Monique Pond, Ph.D. on Establishment of a Regulatory Assistance Program for NCI SBIR

I was able to sit down with Dr. Monique Pond,  AAAS Science & Technology Policy Fellow, Health Scientist within the NCI SBIR Development Center to discuss the new assistance program in regulatory affairs she is developing for the NCI SBIR program.  Dr Pond had received her PhD in chemistry from the Pennsylvania State University, completed a postdoctoral fellow at NIST and then spent many years as a regulatory writer and consultant in the private sector.  She applied through the AAAS for this fellowship and will bring her experience and expertise in regulatory affairs from the private sector to the SBIR program. Dr. Pond discussed the difficulties that new ventures have in formulating regulatory procedures for their companies, the difficulties in getting face time with FDA regulators and helping young companies start thinking about regulatory issues such as pharmacovigilence, oversight, compliance, and navigating the complex regulatory landscape.

In addition Dr. Pond discussed the AAAS fellowship program and alternative career paths for PhD scientists.

 

A formal interview will follow on this same post.

 

Other articles on this OPEN ACCESS JOURNAL on Funding for Startups and Early Ventures are given below:

 

Mapping Medical Device Startups Across The Globe per Funding Criteria

Funding Oncorus’s Immunotherapy Platform: Next-generation Oncolytic Herpes Simplex Virus (oHSV) for Brain Cancer, Glioblastoma Multiforme (GBM)

 

Funding Opportunities for Cancer Research

 

Team Profile: DrugDiscovery @LPBI Group – A BioTech Start Up submitted for Funding Competition to MassChallenge Boston 2016 Accelerator

 

A Message from Faculty Director Lee Fleming on Latest Issue of Crowdfunding; From the Fung Institute at Berkeley

 

PROTOCOL for Drug Screening of 3rd Party Intellectual Property Presented for Funding Representation

 

Foundations as a Funding Source

 

The Bioscience Crowdfunding Environment: The Bigger Better VC?

 

Read Full Post »


Cancer Cell Therapy: Global Start up Acquisitions in Oncolytic Viruses (OV) Therapeutics – a Pipeline of 70 OVs in Clinical Development and another 95 in Preclinical Programs

 

Reporter: Aviva Lev-Ari, PhD, RN

 

September 2018 

  • Boehringer Ingelheim is buying ViraTherapeutics in $244M deal

Boehringer Ingelheim joins the crowd and goes all-in on oncolytic viruses, buying ViraTherapeutics in $244M deal

https://endpts.com/boehringer-ingelheim-joins-the-crowd-and-goes-all-in-on-oncolytic-viruses-buying-viratherapeutics-in-244m-deal/?utm_medium=email&utm_campaign=Thursday%20September%2013%202018&utm_content=Thursday%20September%2013%202018+CID_61414a80a0003abe24ea7e26fafb3eab&utm_source=ENDPOINTS%20emails&utm_term=Boehringer%20Ingelheim%20joins%20the%20major%20league%20players%20with%20a%20buyout

 

May 2018

  • J&J executed one of its classic billion-dollar deals to buy BeneVir.

J&J’s Janssen to Acquire BeneVir for $1 Billion

May 09, 2018
By Pharmaceutical Technology Editors

Janssen Biotech, part of Janssen Pharmaceutical Companies, a subsidiary of Johnson & Johnson (J&J), announced on May 2, 2018 that it has entered into a definitive agreement to acquire BeneVir Biopharm (BeneVir), a biotechnology company specializing in the development of oncolytic immunotherapies.

BeneVir is a portfolio company and subsidiary of HC2 Holdings’ Pansend Life Sciences, which is focused on developing healthcare technologies and products. Benevir uses a proprietary T-Stealth Oncolytic Virus Platform to engineer oncolytic viruses tailored to infect and eliminate cancer cells.

Under the terms of the agreement, Janssen will make an upfront cash payment of $140 million at closing of the transaction, plus additional contingent payments of up to $900 million based on achievement of certain predetermined milestones. The total amount of all payments could exceed $1 billion if all milestones are met. The closing of the transaction is subject to customary closing conditions, including clearance under the Hart-Scott-Rodino Antitrust Improvements Act. The transaction, which was facilitated by J&J Innovation, is expected to close in the second quarter of 2018.

SOURCE

http://www.pharmtech.com/jj-s-janssen-acquire-benevir-1-billion-0

 

May 2018

  • A recent study from the Cancer Research Institute found 69 OVs in clinical development and another 95 in a preclinical program.

New Report on the Global Landscape of Cancer Cell Therapy Highlights Robust International Pipeline Marked by Rapid Growth

  • A comprehensive, neutral analysis of the global cancer cell therapy landscape
  • 753 cancer cell therapies in the global development pipeline, with 375 in clinical studies
  • 350 new cancer cell therapies have entered the global development pipeline since Sep. 2017, an 87% increase in less than 7 months
  • 113 targets are being pursued in 7 different classes of cell therapies

NEW YORK, May 25, 2018—The Cancer Research Institute (CRI) announced today the publication of a report that provides a comprehensive, independent analysis of the global landscape of cancer cell therapies, including all agents from preclinical to post-market stages. This report provides a quantitative and current overview of the cancer cell therapy field, reveals the unparalleled speed of the field’s expansion, highlights exciting innovations in the development pipeline, and advises strategies to advance the field as a whole.

The report, titled “The Global Landscape of Cancer Cell Therapy,” appeared online today in Nature Reviews Drug Discovery, a premium journal from Nature Publishing Group and an authoritative source of information in drug discovery and development. This report, which expands on CRI’s previously published landscape analysis of the entire field of immuno-oncology, highlights the geographic distribution of cancer cell therapies worldwide and identifies the dominant presence of CAR T therapies in the cancer cell therapy space.

“The quantitative analyses from this report reveal unprecedented enthusiasm and innovation in the global cell therapy pipeline,” said Jill O’Donnell-Tormey, Ph.D., chief executive officer and director of scientific affairs at the Cancer Research Institute.

“In addition to traditional powerhouses of drug development such as the United States or European countries, many other countries, especially China, have significant presence in this space,” noted Jun Tang, Ph.D., a senior research analyst for the CRI Anna-Maria Kellen Clinical Accelerator program and first author on the report.

To access the interactive dashboard of the report, visit the CRI website at cancerresearch.org/io-cell-therapy.

Reference
Tang J. et al. Global landscape of cancer cell therapy. Nature Reviews Drug Discovery. 25 May 2018. doi:10.1038/nrd.2018.74

SOURCE

https://www.cancerresearch.org/news/2018/global-landscape-of-cancer-cell-therapy-report

 

February 2018

  • Merck’s R&D chief Roger Perlmutter — who steered the T-Vec deal at Amgen — bagged Viralytics for $394 million.
FEBRUARY 21, 2018 / 4:29 AM / 7 MONTHS AGO

Merck to buy virus-based cancer drug firm Viralytics for $394 million

(Reuters) – U.S. drugmaker Merck & Co (MRK.N), already one of the leaders in the hot area of cancer immunotherapy, said on Wednesday it had agreed to buy Viralytics VLA.AX for 502 million Australian dollars ($394 million) to expand its pipeline in the sector.

Merck will pay 1.75 Australian dollars per share for the Sydney-based biotech company, which uses viruses to infect and kill cancer cells.

The idea is to cause cancer cells to rupture and die, while also stimulating a wider immune system response in the body.

SOURCE

https://www.reuters.com/article/us-viralytics-m-a-merck-co/merck-to-buy-virus-based-cancer-drug-firm-viralytics-for-394-million-idUSKCN1G50ZN

 

Hum Vaccin Immunother. 2018; 14(4): 839–846.
Published online 2018 Feb 22. doi:  10.1080/21645515.2017.1412896
PMCID: PMC5893211
PMID: 29420123

Talimogene laherparepvec: First in class oncolytic virotherapy

ABSTRACT

Oncolytic viruses represent a novel drug class in which native or modified viruses mediate tumor regression through selective replication within and lysis of tumor cells as well as induction of systemic antitumor immunity capable of eradicating tumor at distant, uninjected sites. Talimogene laherparepvec (TVEC) is a type I herpes simplex virus genetically modified to preferentially replicate in tumor cells, enhance antigen loading of MHC class I molecules and express granulocyte-macrophage colony-stimulating factor to increase tumor-antigen presentation by dendritic cells. It is presently the only oncolytic virus approved by the FDA with an indication for advanced melanoma based upon improved durable response rate in a randomized, phase III trial. Clinical trials are underway in melanoma investigating TVEC as neoadjuvant monotherapy and in combination with checkpoint inhibitors for unresectable disease as well as in an array of other malignancies. It is appropriate to review TVEC’s biology mechanism of action, clinical indication and future directions as a prototype of the burgeoning class of oncolytic viruses.

SOURCE

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5893211/

October 2015 

  • Amgen’s landmark approval of T-Vec, the world’s first marketed oncolytic virus.

FDA approves cancer-killing cold sore virus as therapy for late-stage melanoma

October 28, 2015, University of Utah Health Sciences
melanoma
Melanoma in skin biopsy with H&E stain — this case may represent superficial spreading melanoma. Credit: Wikipedia/CC BY-SA 3.0

The U.S. Food and Drug Administration announced on Oct. 27 that it has approved, for the first time, an oncolytic (cancer-killing) viral therapy in the United States. The drug was approved for use against late-stage melanoma, a deadly skin cancer that can be difficult to treat.

The approval came as the result of a recent Phase III study, which showed that more patients with late-stage melanoma, treated with a herpes cold sore virus designed to kill , had a better response when compared to a different treatment. Robert Andtbacka, M.D., from Huntsman Cancer Institute at the University of Utah and Howard L. Kaufman, M.D., from Rutgers Cancer Institute of New Jersey, led the multisite study, published May 26 online in the Journal of Clinical Oncology.

SOURCE

https://medicalxpress.com/news/2015-10-fda-cancer-killing-cold-sore-virus.html

 

Additional Sources

T Cell Engineering Breakthrough Sidesteps Need for Viruses in Gene-Editing
UCSF News Center | Pete Farley | July 11, 2018

With Faster, Cheaper, More Precise Technique, Authors Say It’s ‘Off to the Races’ Toward New Cell

The basic unit of life. The number of cells in a living organism ranges from one (e.g. yeast) to quadrillions (e.g. blue whale). A cell is composed of four key macromolecules that allow it to function (protein, lipids, carbohydrates, and nucleic acids). Among other things, cells can build and break down molecules, move, grow, divide, and die.

” aria-describedby=”tt”>CellTherapies

In an achievement that has significant implications for research, medicine, and industry, UC San Francisco scientists have genetically reprogrammed the human immune cells known as T cells without using Virus

An infectious entity that can only persist by hijacking a host organism to replicate itself. Has its own genome, but is technically not considered a living organism. Viruses infect all organisms, from humans to plants to microbes. Multicellular organisms have sophisticated immune systems that combat viruses, while CRISPR systems evolved to stop viral infection in bacteria and archaea.

” aria-describedby=”tt”>viruses to insert DNA

Abbreviation of deoxyribonucleic acid, a long molecule that encodes the information needed for a cell to function or a virus to replicate. Forms a double-helix shape that resembles a twisted ladder. Different chemicals called bases, abbreviated as A, C, T, and G, are found on each side of the ladder, or strand. The bases have an attraction for each other, making A stick to T while C sticks to G. These rungs of the ladder are called base pairs. The sequence of these letters is called the genetic code.

” aria-describedby=”tt”>DNA. The researchers said they expect their technique — a rapid, versatile, and economical approach employing CRISPR

Pronounced “crisper.” An adaptive immune system found in bacteria and archaea, co-opted as a genome engineering tool. Acronym of “clustered regularly interspaced short palindromic repeats,” which refers to a section of the host genome containing alternating repetitive sequences and unique snippets of foreign DNA. CRISPR-associated surveillance proteins use these unique sequences as molecular mugshots as they seek out and destroy viral DNA to protect the cell.

” aria-describedby=”tt”>CRISPR Gene

A segment of DNA that encodes the information used to make a protein. Each gene is a set of instructions for making a particular molecular machine that helps a cell, organism, or virus function.

” aria-describedby=”tt”>gene-editing technology — to be widely adopted in the burgeoning field of cell therapy, accelerating the development of new and safer treatments for Cancer

A type of disease caused by uncontrolled growth of cells. Cancerous cells may form clumps or masses known as tumors, and can spread to other parts of the body through a process known as metastasis.

” aria-describedby=”tt”>cancer, autoimmunity, and other diseases, including rare inherited disorders.

The new method, described in the July 11, 2018 issue of Nature, offers a robust molecular “cut and paste” system to rewrite Genome

The entire DNA sequence of an organism or virus. The genome is essentially a huge set of instructions for making individual parts of a cell and directing how everything should run.

” aria-describedby=”tt”>genome sequences in human T cells. It relies on electroporation, a process in which an electrical field is applied to cells to make their membranes temporarily more permeable. After experimenting with thousands of variables over the course of a year, the UCSF researchers found that when certain quantities of T cells, DNA, and the CRISPR “scissors” are mixed together and then exposed to an appropriate electrical field, the T cells will take in these elements and integrate specified genetic sequences precisely at the site of a CRISPR-programmed cut in the genome.

“This is a rapid, flexible method that can be used to alter, enhance, and reprogram T cells so we can give them the specificity we want to destroy cancer, recognize infections, or tamp down the excessive immune response seen in autoimmune disease,” said UCSF’s Alex Marson, MD, PhD, associate professor of microbiology and immunology, member of the UCSF Helen Diller Family Comprehensive Cancer Center, and senior author of the new study. “Now we’re off to the races on all these fronts.”

But just as important as the new technique’s speed and ease of use, said Marson, also scientific director of biomedicine at the Innovative Genomics

The study of the genome, all the DNA from a given organism. Involves a genome’s DNA sequence, organization and control of genes, molecules that interact with DNA, and how these different components affect the growth and function of cells.

” aria-describedby=”tt”>Genomics Institute, is that the approach makes it possible to insert substantial stretches of DNA into T cells, which can endow the cells with powerful new properties. Members of Marson’s lab have had some success using electroporation and CRISPR to insert bits of genetic material into T cells, but until now, numerous attempts by many researchers to place long sequences of DNA into T cells had caused the cells to die, leading most to believe that large DNA sequences are excessively toxic to T cells.

SOURCE

https://innovativegenomics.org/news/t-cell-engineering-breakthrough-sidesteps-viruses/

 

Cancer Res. 2016 Aug 15; 76(16): 4627–4636.

Published online 2016 Jun 3. doi:  10.1158/0008-5472.CAN-15-3455

PMCID: PMC5295843

CAMSID: CAMS5780

PMID: 27261504

Design and Reporting of Targeted Anticancer Preclinical Studies: A Meta-Analysis of Animal Studies Investigating Sorafenib Antitumor Efficacy

James Mattina,1 Nathalie MacKinnon,1 Valerie C. Henderson,1 Dean Fergusson,2 andJonathan Kimmelman

 

Other 17 related articles published in this Online Scientific Journal include the following:

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