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Archive for the ‘Pharmaceutical R&D Investment’ Category


Tweets and Retweets by @AVIVA1950 and by @pharma_BI for @USAIC and #USAIC2019 at the 13th Annual BioPharma & Healthcare Summit, Thursday, May 9, 2019, Marriott Hotel, Cambridge, MA

 

  1.  liked Tweets you were included in

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  1.   Retweeted

    Alise Reicin discusses endpoints needed in and effect of cost of in Panel Discussion morning Networking break

  2.   Retweeted

    Alise Reicin Panel Discussion: best done as Basket&Umbrella trial; response rate 30-50% but Phase3 negative

    Translate Tweet

  3.   Retweeted

    Dr. William Chin design challenges with newer in recruiting patients; use Basket&Umbrella Trial design Live

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On risk | benefit: Long term impacts of treatment may be present for the life of the patient

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    Discussion of cyber security at panel moderated by – realization that everything is interconnected: risks to business-critical functions. Much to learn from FinTech & others’ prior experience.

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    Unexpected best thing about – at least three Zimbabweans in the crowd / on the stage.

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    segueing into a discussion of safety, and risk tolerance. Prevention has a higher safety bar than treatment later in the disease process.

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    Most humbling & touching moment: meeting an exec who nodded & teared up as I told him what we do His 9 yo child needs frequent contrast enhanced MRI scans for a rare disease. We felt like the smallest co at yet so important for hope & health

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    Great initiatives to bring CAR-T to India an automation to drug manufacturion

    Amazing moderator asking provoking question of best panel in responses by experts made known were different for competing rivals the aim is same best faster largest at lowest keeping maximized

  7.   Retweeted

    Dr. Bruce Chabner Talk: Old Phase 1,2,3 design not needed for & era based . very accommodating in Phase 1 trial design.

  8.   Retweeted

    Dr. Bruce Chabner panel discussion: selection based on new design paradigm; 40 drugs approved by accelerated approval for

  9.   Retweeted

    Dr. Mace Rothenberg talks for approvals vary greatly over multiple countries makes issues of ong-term design and post approval reimbursement

  10.   Retweeted

    Talk : design now depends on systems e.g. organelle delivery. only wants accelerated approval not conditional approval. Surrogate markers critical for new trial design

  11.   Retweeted

    Dr. Rob Scott Panel Talk crisis in not in . Lowest investment in development. Physicians using SAME design for . use in trails increasing

  12.   Retweeted

    Dr. Rob Scott on design @usaic2019: Regulators as partners not barriers but burden of efficacy on . Can use advertising to increase recruitment as 70% willing participants live too far away so use &

  13.   Retweeted

    Dr. Rob Scott discusses recruitment and burdens : prefers to do in house & not use CRO as CRO less effective in monitoring trial

  14.   Retweeted

    “Some drug platforms are mature enough to fall under the practice of medicine” – Tim Yu of

  15.   Retweeted

    Closing R&D strategies panel at is with NIBR’s Janssen’s and Takeda’s Andy Plump. Moderator Martin Mackay (now of newco Rallybio) asks: What are you most excited about?

  16.   Retweeted

    Talk of indexed pricing model in the US may be a challenge for access to other parts of the world, says a speaker on rare disease panel

  17.   Retweeted

    The biggest problem that we have in the industry is the lack of empathy, says Chris Viehbacher

  18.   Retweeted

    Tim Yu’s example left a room full of seasoned biopharma R&D execs wowed. More background here:

  19.   Retweeted

    (May 9, Boston) will feature plenary panel on Emerging R&D Strategies moderated by Dr. Martin Mackay, Co-Founder, Rallybio, with of , Andy Plump of , of Janssen, Michael Ehler of -> register today

  20.   Retweeted

    Check out my latest article: Where is Oncology Drug Development Heading Next? Hear From Top KOLs at 13th BioPharma Summit May 9, Boston via

  21.   Retweeted

    Check out my latest article: One in a Million: Emerging Trends in Rare Diseases at 13th annual BioPharma Summit- May 9, Boston via

  22.   Retweeted

    Check out my latest article: Chris Viehbacher, Gurnet Point Capital joins the USAIC Advisory Board. Please join Chris & other leaders at our annual BioPharma Summit, May 9, Boston via

  23.   Retweeted

    Check out my latest article: R&D Panel: BioPharma KOLs Debate R&D Strategies & Trends at 13th annual BioPharma Summit, May 9, Boston via

  24.   Retweeted

    Check out my latest article: What Does The Future Hold For Drug Development & Clinical Trials? Hear Predictions From Top Drug Developers at the 13th BioPharma Summit May Boston 9 via

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    Value and Access – The Ongoing Debate.The BioPharma Summit will feature this special session. Join the discussion with BioPharma KOLs via

  27.   Retweeted

    BioPharma Manufacturing in the Future: Hear KOLs Debate the Challenges and Opportunities at the 13th annual BioPharma Summit, May 9, Boston via

  28.   Retweeted

    Our 13th Biopharma & Healthcare Summit has kicked off with introductory remarks from USAIC President Karun Rishi and emcee Dr. Andrew Plump, President of R&D for Takeda.

  29.   Retweeted

    The session is starting as I attend the focused on US-India bio-pharma healthcare summit. The focus is on and to deliver compelling affordable care with key role for technologies.

  30.   Retweeted

    Carl June believes we’re only a few years away from outpatient CAR therapies, with no need for intensive infrastructure with ICU.

  31.   Retweeted

    Dr. Maus ⁩ monitoring data from clinical trial is very important development of new targets multiple drugs multiple mechanism multiple specificities more modification to one cell contamination results

  32.   Retweeted

    “Cancer is a collection of rare diseases” – , Director of Clinical Research, Cancer

  33.   Retweeted

    Sanat Chattopadhyay of US Merck says costs of manufacturing in US/Europe is significantly higher because technology deployed is ancient, both in small molecules and biologics.

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    Rare disease taking center stage as technologies mature, panel moderated by CRISPR Therapeutics CEO Samarth Kulkarni

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    Replying to  

    Enjoyed moderating the panel on manufacturing in the future as bio pharma companies explore ways to deliver drugs at affordable price and address access challenges. Digital innovation in manufacturing and supply chains will be key.

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    Price is part of doing business…if you are not able to define value, you are sunk, says Chris Viehbacher Gurnet Point Capital

  37.   Retweeted

    All diseases are unique Your perspective changes when you or someone you love is diagnosed with a life-altering disease – rare diseases panel

  38.   Retweeted

    Arun Singhal, addl secretary, health ministry speaks about Ayushman Bharat, trade margin rationalisation, clinical trial rules at meeting

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    Clinical trials in India picking up steadily, says Eswara Reddy, DCGI

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    Regulators will “provide complete support” for clinical trials in India provided drug developers meet the new requirements – Drug Controller General of India Eswara Reddy

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    discusses prevention and treatment of early disease e.g. precancer The challenge is to work out the commercial model says organize around prevention Early diagnosis / discovery of disease processes saves lives (’s raison d’etre)

  42. AMAZING EVENT @AVIVA11950 13th Annual & Healthcare Summit, Thursday, May 9, 2019, Marriott Hotel, Cambridge, MA via

  43. Dr. James Bradner, President, Novartis Institutes for BioMedical Research biophysical biochemical protein degradation – rewire disease cells with biomolecules combing propertitie of permiability of small molecules

  44. World PGD Growth of 4% in India 31%

  45.   Retweeted

    Takeda R&D Head Andy Plump asks question from audience to peers on stage: “I’ve been in the industry for 18 years and I can’t understand why a clinical trial costs so much. Why does it?”

  46.   Retweeted

    Manufacturing in the age of individualized medicine? We may need a completely different thinking for that says Paul Mckenzie, Biogen

  47.   Retweeted

    The so-called low-cost manufacturing edge of India will go away in a few years, says Hari Bhartia, Jubilant…being closer to the customer will be important

  48.   Retweeted

    Tricky question: getting patients to cancer centers to participate in clinical trials. Should patients be reimbursed for long travels and other expenses or will it be seen as an inducement?

  49.   Retweeted

    A chance to collaborate with my old colleague & friend ! -> another point from same panel: AbbVie CMO Rob Scott predicts tele-health solutions for clinical trial patients will be scalable soon

  50.   Retweeted

    Carl June: There are no CAR-T clinical trials in India. But says countries like India could eventually leapfrog to next gen (outpatient) cell therapy which will require less infrastructure + lower COG

  51.   Retweeted

    The 13th Annual BioPharma & Healthcare Summit is being kicked off by Andrew Plump. In his opening remarks, he commented that we should feel privileged as attendees because not even or is invited to this meeting.

  52.   Retweeted

    A well-represented panel of scientists, CEOs and entrepreneurs discuss a range of discovery research from CAR-Ts to small molecules…on the same panel is Arjun Surya of Curadev that licensed a preclinical oncology lead to Takeda.

  53. The promise of India is the largest democracy, the educated workforce the size of the market for therapeutics, access and price, reimbursement and regulation. DCGI the analogue of FDA is very active and innovated the challenge of 1.3 Billion a population of patients

  54. Great Leader in immunotherapy, Carl June early inventor and endless commitment to patient a Pro for BioPharma

  55. GREAT Conference of who is who in BioPharma, Boston at the top 500 startats of Biotech in Cambridge, MA ten years afo only a handful, boost by Novartis HQS in Cambridge, MA

  56. Liked the analogy of

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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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From Thalidomide to Revlimid: Celgene to Bristol Myers to possibly Pfizer; A Curation of Deals, Discovery and the State of Pharma

 

Curator: Stephen J. Williams, Ph.D.

Updated 4/12/2019

Updated 2/28/2019

Lenalidomide (brand name Revlimid) is an approved chemotherapeutic used to treat multiple myeloma, mantle cell lymphoma, and certain myedysplastic syndromes.  It is chemically related to thalidomide analog with potential antineoplastic activity. Lenalidomide inhibits TNF-alpha production, stimulates T cells, reduces serum levels of the cytokines vascular endothelial growth factor (VEGF) and basic fibroblast growth factor (bFGF), and inhibits angiogenesis. This agent also promotes G1 cell cycle arrest and apoptosis of malignant cells.  It is usually given with dexamethasone for multiple myeloma. Revlimid was developed and sold by Celgene Corp.  However, recent news of deals with Bristol Myers Squib

 

Revlimid Approval History 

FDA Approved: Yes (First approved December 27, 2005)
Brand name: Revlimid
Generic name: lenalidomide
Dosage form: Capsules
Company: Celgene Corporation
Treatment for: Myelodysplastic SyndromeMultiple MyelomaLymphoma 

Revlimid (lenalidomide) is an immunomodulatory drug indicated for the treatment of patients with multiple myeloma, transfusion-dependent anemia due myelodysplastic syndromes (MDS), and mantle cell lymphoma. 

Development History and FDA Approval Process for Revlimid 

Date  Article 
Feb 22, 2017   FDA Expands Indication for Revlimid (lenalidomide) as a Maintenance Treatment for Patients with Multiple Myeloma Following Autologous Hematopoietic Stem Cell Transplant (auto-HSCT) 
Feb 18, 2015   FDA Expands Indication for Revlimid (lenalidomide) in Combination with Dexamethasone to Include Patients Newly Diagnosed with Multiple Myeloma 
Jun  5, 2013   FDA Approves Revlimid (lenalidomide) for the Treatment of Patients with Relapsed or Refractory Mantle Cell Lymphoma 
Oct  3, 2005  Revlimid PDUFA Date Extended Three Months By FDA 
Sep 14, 2005  FDA Oncologic Drugs Advisory Committee Recommends Revlimid for Full Approval 
Sep 13, 2005  FDA and Celgene Revlimid Briefing Documents for Advisory Committee Meeting Available Online 
Jun 21, 2005  FDA Grants Priority Review for Revlimid NDA for Treatment of Low- and Intermediate- Risk MDS With Deletion 5q Chromosomal Abnormality 
Jun  7, 2005  Revlimid (lenalidomide) New Drug Application Accepted for Review by FDA 
Apr  8, 2005  Revlimid New Drug Application Submitted to FDA for Review 

  

  

 

 

M&A Deals Now and On The Horizon

  1. Right before the 2019 JP Morgan Healthcare Conference and a month before Bristol Myers quarterly earings reports, Bristol Myers Squib (BMY) announes a $74 Billion offer for Celgene Corp.  From the Bristol Myers website press realease:

Bristol-Myers Squibb to Acquire Celgene to Create a Premier Innovative Biopharma Company

  • Highly Complementary Portfolios with Leading Franchises in Oncology, Immunology and Inflammation and Cardiovascular Disease
  • Significantly Expands Phase III Assets with Six Expected Near-Term Product Launches, Representing Greater Than $15 Billion in Revenue Potential
  • Registrational Trial Opportunities and Early-Stage Pipeline Position Combined Company for Sustained Leadership Underpinned by Cutting-Edge Technologies and Discovery Platforms
  • Strong Combined Cash Flows, Enhanced Margins and EPS Accretion of Greater Than 40% in First Full Year
  • Approximately $2.5 Billion of Expected Run-Rate Cost Synergies to Be Achieved by 2022
THURSDAY, JANUARY 3, 2019 6:58 AM EST

NEW YORK & SUMMIT, N.J.,–(BUSINESS WIRE)–Bristol-Myers Squibb Company (NYSE:BMY) and Celgene Corporation (NASDAQ:CELG) today announced that they have entered into a definitive merger agreement under which Bristol-Myers Squibb will acquire Celgene in a cash and stock transaction with an equity value of approximately $74 billion. Under the terms of the agreement, Celgene shareholders will receive 1.0 Bristol-Myers Squibb share and $50.00 in cash for each share of Celgene. Celgene shareholders will also receive one tradeable Contingent Value Right (CVR) for each share of Celgene, which will entitle the holder to receive a payment for the achievement of future regulatory milestones. The Boards of Directors of both companies have approved the combination.

The transaction will create a leading focused specialty biopharma company well positioned to address the needs of patients with cancer, inflammatory and immunologic disease and cardiovascular disease through high-value innovative medicines and leading scientific capabilities. With complementary areas of focus, the combined company will operate with global reach and scale, maintaining the speed and agility that is core to each company’s strategic approach.

Based on the closing price of Bristol-Myers Squibb stock of $52.43 on January 2, 2019, the cash and stock consideration to be received by Celgene shareholders at closing is valued at $102.43 per Celgene share and one CVR (as described below). When completed, Bristol-Myers Squibb shareholders are expected to own approximately 69 percent of the company, and Celgene shareholders are expected to own approximately 31 percent.

“Together with Celgene, we are creating an innovative biopharma leader, with leading franchises and a deep and broad pipeline that will drive sustainable growth and deliver new options for patients across a range of serious diseases,” said Giovanni Caforio, M.D., Chairman and Chief Executive Officer of Bristol-Myers Squibb. “As a combined entity, we will enhance our leadership positions across our portfolio, including in cancer and immunology and inflammation. We will also benefit from an expanded early- and late-stage pipeline that includes six expected near-term product launches. Together, our pipeline holds significant promise for patients, allowing us to accelerate new options through a broader range of cutting-edge technologies and discovery platforms.”

Dr. Caforio continued, “We are impressed by what Celgene has accomplished for patients, and we look forward to welcoming Celgene employees to Bristol-Myers Squibb. Our new company will continue the strong patient focus that is core to both companies’ missions, creating a shared organization with a goal of discovering, developing and delivering innovative medicines for patients with serious diseases. We are confident we will drive value for shareholders and create opportunities for employees.”

“For more than 30 years, Celgene’s commitment to leading innovation has allowed us to deliver life-changing treatments to patients in areas of high unmet need. Combining with Bristol-Myers Squibb, we are delivering immediate and substantial value to Celgene shareholders and providing them meaningful participation in the long-term growth opportunities created by the combined company,” said Mark Alles, Chairman and Chief Executive Officer of Celgene. “Our employees should be incredibly proud of what we have accomplished together and excited for the opportunities ahead of us as we join with Bristol-Myers Squibb, where we can further advance our mission for patients. We look forward to working with the Bristol-Myers Squibb team as we bring our two companies together.”

Compelling Strategic Benefits

  • Leading franchises with complementary product portfolios provide enhanced scale and balance. The combination creates:
    • Leading oncology franchises in both solid tumors and hematologic malignancies led by Opdivo and Yervoy as well as Revlimid and Pomalyst;
    • A top five immunology and inflammation franchise led by Orencia and Otezla; and
    • The #1 cardiovascular franchise led by Eliquis.

The combined company will have nine products with more than $1 billion in annual sales and significant potential for growth in the core disease areas of oncology, immunology and inflammation and cardiovascular disease.

  • Near-term launch opportunities representing greater than $15 billion in revenue potential. The combined company will have six expected near-term product launches:
    • Two in immunology and inflammation, TYK2 and ozanimod; and
    • Four in hematology, luspatercept, liso-cel (JCAR017), bb2121 and fedratinib.

These launches leverage the combined commercial capabilities of the two companies and will broaden and enhance Bristol-Myers Squibb’s market position with innovative and differentiated products. This is in addition to a significant number of lifecycle management registrational readouts expected in Immuno-Oncology (IO).

  • Early-stage pipeline builds sustainable platform for growth. The combined company will have a deep and diverse early-stage pipeline across solid tumors and hematologic malignancies, immunology and inflammation, cardiovascular disease and fibrotic disease leveraging combined strengths in innovation. The early-stage pipeline includes 50 high potential assets, many with important data readouts in the near-term. With a significantly enhanced early-stage pipeline, Bristol-Myers Squibb will be well positioned for long-term growth and significant value creation.
  • Powerful combined discovery capabilities with world-class expertise in a broad range of modalities. Together, the Company will have expanded innovation capabilities in small molecule design, biologics/synthetic biologics, protein homeostasis, antibody engineering and cell therapy. Furthermore, strong external partnerships provide access to additional modalities.

Compelling Financial Benefits

  • Strong returns and significant immediate EPS accretion. The transaction’s internal rate of return is expected to be well in excess of Celgene’s and Bristol-Myers Squibb’s cost of capital. The combination is expected to be more than 40 percent accretive to Bristol-Myers Squibb’s EPS on a standalone basis in the first full year following close of the transaction.
  • Strong balance sheet and cash flow generation to enable significant investment in innovation. With more than $45 billion of expected free cash flow generation over the first three full years post-closing, the Company is committed to maintaining strong investment grade credit ratings while continuing its dividend policy for the benefit of Bristol-Myers Squibb and Celgene shareholders. Bristol-Myers Squibb will also have significant financial flexibility to realize the full potential of the enhanced late- and early-stage pipeline.
  • Meaningful cost synergies. Bristol-Myers Squibb expects to realize run-rate cost synergies of approximately $2.5 billion by 2022. Bristol-Myers Squibb is confident it will achieve efficiencies across the organization while maintaining a strong, core commitment to innovation and delivering the value of the portfolio.

Terms and Financing

Based on the closing price of Bristol-Myers Squibb stock on January 2, 2019, the cash and stock consideration to be received by Celgene shareholders is valued at $102.43 per share. The cash and stock consideration represents an approximately 51 percent premium to Celgene shareholders based on the 30-day volume weighted average closing stock price of Celgene prior to signing and an approximately 54 percent premium to Celgene shareholders based on the closing stock price of Celgene on January 2, 2019. Each share also will receive one tradeable CVR, which will entitle its holder to receive a one-time potential payment of $9.00 in cash upon FDA approval of all three of ozanimod (by December 31, 2020), liso-cel (JCAR017) (by December 31, 2020) and bb2121 (by March 31, 2021), in each case for a specified indication.

The transaction is not subject to a financing condition. The cash portion will be funded through a combination of cash on hand and debt financing. Bristol-Myers Squibb has obtained fully committed debt financing from Morgan Stanley Senior Funding, Inc. and MUFG Bank, Ltd. Following the close of the transaction, Bristol-Myers Squibb expects that substantially all of the debt of the combined company will be pari passu.

Accelerated Share Repurchase Program

Bristol-Myers Squibb expects to execute an accelerated share repurchase program of up to approximately $5 billion, subject to the closing of the transaction, market conditions and Board approval.

Corporate Governance

Following the close of the transaction, Dr. Caforio will continue to serve as Chairman of the Board and Chief Executive Officer of the company. Two members from Celgene’s Board will be added to the Board of Directors of Bristol-Myers Squibb. The combined company will continue to have a strong presence throughout New Jersey.

Approvals and Timing to Close

The transaction is subject to approval by Bristol-Myers Squibb and Celgene shareholders and the satisfaction of customary closing conditions and regulatory approvals. Bristol-Myers Squibb and Celgene expect to complete the transaction in the third quarter of 2019.

Advisors

Morgan Stanley & Co. LLC is serving as lead financial advisor to Bristol-Myers Squibb, and Evercore and Dyal Co. LLC are serving as financial advisors to Bristol-Myers Squibb. Kirkland & Ellis LLP is serving as Bristol-Myers Squibb’s legal counsel. J.P. Morgan Securities LLC is serving as lead financial advisor and Citi is acting as financial advisor to Celgene. Wachtell, Lipton, Rosen & Katz is serving as legal counsel to Celgene.

Bristol-Myers Squibb 2019 EPS Guidance

In a separate press release issued today, Bristol-Myers Squibb announced its 2019 EPS guidance for full-year 2019, which is available on the “Investor Relations” section of the Bristol-Myers Squibb website at https://www.bms.com/investors.html.

Conference Call

Bristol-Myers Squibb and Celgene will host a conference call today, at 8:00 a.m. ET to discuss the transaction. The conference call can be accessed by dialing (800) 347-6311 (U.S. / Canada) or (786) 460-7199 (International) and giving the passcode 4935567. A replay of the call will be available from January 3, 2019 until January 17, 2019 by dialing (888) 203-1112 (U.S. / Canada) or (719) 457-0820 (International) and giving the passcode 4935567.

A live webcast of the conference call will be available on the investor relations section of each company’s website at Bristol-Myers Squibb https://www.bms.com/investors.html and Celgene https://ir.celgene.com/investors/default.aspx.

Presentation and Infographic

Associated presentation materials and an infographic regarding the transaction will be available on the investor relations section of each company’s website at Bristol-Myers Squibb https://www.bms.com/investors.html and Celgene https://ir.celgene.com/investors/default.aspx as well as a joint transaction website at www.bestofbiopharma.com.

2.  Then through news on Bloomberg and some other financial sites on a possible interest of a merged Celgene-Bristol Myers from Pfizer as well as other pharma groups

Here’s How John Paulson Is Positioning His Celgene/Bristol Trade

Billionaire John Paulson sees a 10 percent to 20 percent chance that Bristol-Myers Squibb Co. receives a takeover bid and he’s positioning his Celgene Corp. trade based on that risk, he said in an interview on Mike Samuels’ “According to Sources” podcast.

Bristol-Myers “is vulnerable and it has an attractive pipeline to several potential acquirers,” Paulson said in the podcast released Monday. “It’s a reasonable probability,” he said. “You have to be prepared someone may show up. It’s an attractive spread, but you can’t take that big a position.”

John Paulson

Photographer: Jin Lee/Bloomberg

Paulson has the Celgene/Bristol-Myers trade as a 3 percent portfolio position, though his firm is short a pharma index rather than Bristol-Myers for about half of the position. If an activist did show up, it would likely blow out the spread from its current $13.85 to probably $20 and, if an actual bid arrived, he said the spread could move out to $40.

“I just don’t feel comfortable being short Bristol in this environment,” Paulson said. “You can sort of get the same economics by shorting an index, maybe even do better because, since Bristol came down, if the pharma sector goes up, Bristol may go up more than the pharma sector, which would increase the profitability on the Celgene. ”

Celgene fell as much as 2.2 percent on Tuesday, its biggest intraday drop since Dec. 27. Bristol-Myers also sank as much as 2.2 percent, the most since Jan. 9.

The question of whether Bristol-Myers receives a hostile takeover offerhas been the top issue for investors since the Celgene deal was announced. The drugmaker was pressured in February 2017 to add three new directors after holding talks with activist hedge fund Jana Partners LLC. The same month, the Wall Street Journal reported that Carl Icahn had taken a stake and saw Bristol-Myers as a takeover target.

Pfizer Inc., AbbVie Inc. or Amgen Inc. “make varying amounts of sense as suitors, though we see many barriers to someone making an offer,” Credit Suisse analyst Vamil Divan wrote in a note earlier this month. AbbVie and Amgen “have the balance sheet strength and could look to beef up their oncology presence.”

CNBC’s David Faber said Jan. 3 — the day the Celgene deal was announced — that there had been “absolutely” no talks between Bristol-Myers and potential acquirers.

Jefferies analyst Michael Yee wrote in note Tuesday that he doesn’t expect an unsolicited offer for Bristol-Myers to “thwart” its Celgene purchase. He sees the deal spread as “quite attractive” again at the current range of 18 percent to 20 percent after it had earlier narrowed to 11 percent to 12 percent.

Paulson managed about $8.7 billion at the the beginning of November.

From StatNews.com at https://www.statnews.com/2019/01/22/celgene-legacy-chutzpah-science-drug-pricing/

 

Nina Kjellson was just two years out of college, working as a research associate at Oracle Partners, a hedge fund in New York, when a cabbie gave her a stock tip. There was a company in New Jersey, he told her, trying to resurrect thalidomide, a drug that was infamous for causing severe birth defects, as a treatment for cancer.

Kjellson was born in Finland, where the memory of thalidomide, which was given to mothers to treat morning sickness but led to babies born without arms or legs, was particularly raw because the drug hit Northern Europe hard. But she was on the hunt for new cancer drugs, and her interest was piqued. She ended up investing a small amount of her own money in Celgene. That was 1999.

Since then, Celgene shares have risen more than 100-fold; the company became one of the largest biotechnology firms in the world. Earlier this month, rival Bristol-Myers Squibb announced plans to purchase Celgene for $74 billion in cash and stock.

Reflecting on a company she watched for two decades, Kjellson, now a venture capitalist at Canaan Partners in San Francisco, marveled at the “grit and chutzpah” that it took to push thalidomide back onto the market. “The company started taking off,” she remembered, “but not without an incredible reversal.” Celgene faced resistance from some thalidomide victims, and the Food and Drug Administration was lobbied not to revive the drug. In the end, she said, it built a golden egg and became a favorite partner of smaller biotech companies like the ones she funds. And it populated the rest of the pharmaceutical industry with its alumni. “If I had a nickel for every company that says we want to do Celgene-like deals,” she said, “I’d have better returns than from my venture career.”

But there’s another side to Celgene. When the company launched thalidomide as a treatment for leprosy in 1998, it cost $6 a pill. As it became clear that it was also an effective cancer drug, Celgene slowly raised the price, quadrupling it by the time it received approval for an improved molecule, Revlimid. Then, it slowly increased the price of Revlimid by a total of 145 percent, according to Sector & Sovereign LLC, a pharmaceutical consultancy.

Revlimid now costs $693 a pill. In 2017, Revlimid and another thalidomide-derived cancer drug represented 76 percent of Celgene’s $12.9 billion in annual sales. Kjellson gives the company credit for guts in science, for taking a terrible drug and resurrecting it. But it also had chutzpah when it came to what it charged.

A pioneer in ‘modern pricing’

How did the price of thalidomide, and then Revlimid, increase so much? Celgene explained it in a 2004 front-page story in the Wall Street Journal. “When we launched it, it was going to be an AIDS-wasting drug,” Celgene’s chief executive at the time, John Jackson, said. “We couldn’t charge more or there would have been demonstrations outside the company.” But once Celgene realized that the drug was a cancer treatment, the company decided to slowly bring thalidomide’s price more in line with other cancer medicines, such as Velcade, a rival medicine now sold by the Japanese drug giant Takeda. In 2003, it cost more than twice as much as thalidomide. “By bringing [the price] up every year, it was heading toward where it should be as a cancer drug,” Jackson told the Journal.

Thalidomide was not actually approved as a myeloma treatment until 2006. That same year, Revlimid, which causes less sleepiness and nerve pain than thalidomide, was approved, and Barer, the chemist behind Celgene’s thalidomide strategy, took over as chief executive. He made good on thalidomide’s promise, churning out one blockbuster after another. In 2017 Revlimid generated $8.2 billion. Another cancer drug derived from thalidomide, Pomalyst, generated $1.6 billion. Otezla, a very different drug also based on thalidomide’s chemistry, treats psoriasis and psoriatic arthritis. Its 2017 sales: $1.3 billion.

With persistent price increases, quarter after quarter, Celgene pioneered something else: what Wall Street calls “modern pricing.” Cancer drug prices have risen inexorably.

 

Updated 2/28/2019

From FiercePharma.com

BMS’ largest investor condemns Celgene deal—and it’s music to activists’ ears

Activist investor Starboard Value is officially rallying the troops against Bristol-Myers Squibb’s $74 billion Celgene deal, and thanks to a big investor’s thumbs-down, it’ll have more support than some expected. But the question is whether it’ll be enough to scuttle the merger.

Starboard CEO Jeffrey Smith penned a letter (PDF) to Bristol-Myers’ shareholders on Thursday labeling the transaction “poorly conceived and ill-advised.” It intends to vote its shares—which number 1.63 million, though the hedge fund is seeking more—against the deal, and it wants to see other shareholders do the same. It’ll be filing proxy materials “in the coming days” to solicit “no” votes from BMS investors, Smith said.

Starboard picked up its stake early this year after the deal was announced, BMS confirmed last week, but until now, the activist fund hasn’t been forthcoming about its intentions. But the timing of its reveal is likely no coincidence; just Wednesday, Wellington Management—which owns about 8% of Bristol-Myers’ shares and ranked as its largest institutional shareholder as of earlier this week—came out publicly against the “risky” buyout.

But while “we believe it is possible at least one other long-term top-five [shareholder] may disagree with the transaction, too,” RBC Capital Markets’ Michael Yee wrote in his own investor note, he—as many of his fellow analysts do—still expects to see the deal go through. “We think the vast majority of the acquirer holder base that would not like the deal already voted by selling their shares earlier, leaving investors who are mostly supportive of the deal,” he wrote.

Meanwhile, Starboard has been clear about one other thing: It wants board seats. It’s nominated five new directors, including CEO Smith, and investors will vote on that group at an as-yet-unscheduled meeting. Thing is, that meeting will take place after BMS investors vote on the Celgene deal in April, so Starboard will have to rally sufficient support against the deal if it wants to see them installed.

The “probability of a third-party buyer for Bristol-Myers Squibb” before the April vote is “very low,” BMO Capital Markets analysts wrote recently, adding that “we do not believe a potential activist can change that.” Barclays analysts agreed Wednesday, pointing to a “lack of realistic, potential alternatives that could collectively provide a similar level of upside.”

Updated 4/12/2019

Bristol-Myers Squibb Shareholders Approve Celgene Tie-Up

Three quarters of Bristol-Myers Squibb shareholders vote to approve the deal with Celgene, paving the way for the largest pharmaceutical takeover in history.

Bristol-Myers Squibb (BMY – Get Report) on Friday announced that it had secured enough shareholder votes to approve its roughly $74 billion takeover of Celgene (CELG – Get Report) , putting the company closer to finalizing the largest pharmaceutical merger in history.

More than 75% of Bristol-Myers shareholders voted to approve the deal, according to a preliminary tally announced by Bristol-Myers on Friday.

Bristol-Myers’ position took a positive turn in late March after an influential shareholder advisory group recommended investors vote in favor of the cancer drug specialist’s takeover,  and a key activist dropped its opposition to the deal.

Institutional Shareholder Services recommended the deal, which had been challenged by key Bristol-Myers shareholders Starboard Value and Wellington Management, ahead of Friday’s vote.

Additional posts on Pharma Mergers and Deals on this Open Access Journal include:

Live Conference Coverage Medcity Converge 2018 Philadelphia: Clinical Trials and Mega Health Mergers

First Annual FierceBiotech Drug Development Forum (DDF). Event covers the drug development process from basic research through clinical trials. InterContinental Hotel, Boston, September 19-21, 2016.

Pfizer Near Allergan Buyout Deal But Will Fed Allow It?

New Values for Capital Investment in Technology Disruption: Life Sciences Group @Google and the Future of the Rest of the Biotech Industry

Mapping the Universe of Pharmaceutical Business Intelligence: The Model developed by LPBI and the Model of Best Practices LLC

 

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In 2018, FDA approved an all-time record of 62 new therapeutic drugs (NTDs) [Not including diagnostic imaging agents, included are combination products with at least one new molecular entity as an active ingredient] with average Peak Sales per NTD $1.2Billion.

 

Reporter: Aviva Lev-Ari, PhD, RN

BIOBUSINESS BRIEFS

2018 FDA approvals hit all-time high — but average value slips again

In 2018, the FDA approved an all-time record of 62 new therapeutic drugs (NTDs; see Fig. 1 for the definition and the difference compared with new molecular entities). This is consistent with the increase we predicted last year (Nat. Rev. Drug Discov. 17, 87; 2018) and the overall resurgence of R&D in the last 5 years, with an average of 51 approvals per year in this period even with a low count in 2016. This is substantially more than the average of 31 approvals per year in the period 2000–2013 (Fig. 1).

Fig. 1 | FDA approvals of new therapeutic drugs and aggregate projected peak global annual sales: 2000–2018. We analysed 2018 FDA approvals of new therapeutic drugs (NTDs), defined as new molecular entities approved by the FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), but with two adjustments: first, we excluded diagnostic imaging agents; and second, we included combination products with at least one new molecular entity as an active ingredient. The analysis is based exclusively on approvals by the FDA and the year in which the first indication approval took place. All peak sales values were obtained from EvaluatePharma and were inflation-adjusted to 2018 using standard global GDP-based inflators sourced from the Economist Intelligence Unit. To arrive at peak sales for each NTD, we reviewed both historical actual sales as well as the full range of forecast sales that are available from EvaluatePharma and selected the highest value. Sources: EvaluatePharma, FDA and Boston Consulting Group analysis.

SOURCE

https://www.nature.com/articles/d41573-019-00004-z

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Reporter: Gail S. Thornton

 

From The Wall Street Journal (www.wsj.com)

Published January 9, 2019

Health-Care CEOs Outline Strategies at J.P. Morgan Conference

Chiefs at Johnson & Johnson, CVS discuss what’s next on a range of industry issues

One of the biggest health conferences of the year for investors, the J.P. Morgan Health-Care Conference, is taking place this week in San Francisco. Here are some of the hot topics covered at the four-day event, which wraps up Thursday.

BioMarin Mulls Payment Plans

BioMarin Pharmaceutical Inc. CEO Jean-Jacques Bienaimé said he would consider pursuing installment payment arrangements for the biotech’s experimental gene therapy for hemophilia. At the conference, Mr. Bienaimé told the Wall Street Journal that the one-time infusion, Valrox, is likely to cost in the millions because studies have shown it can eliminate bleeding episodes in patients, and current hemophilia treatments taken chronically can cost millions over several years. “We’re not trying to charge more than existing therapies,” he said. “We want to offer a better treatment at the same or lower cost.”

Johnson & Johnson Warns on Pricing

As politicians hammer drug prices, Johnson & Johnson CEO Alex Gorsky suggested companies need to police themselves. At the conference, Mr. Gorsky told investors that drug companies should price drugs reasonably and be transparent. “If we don’t do this as an industry, I think there will be other alternatives that will be more onerous for us,” Mr. Gorsky says. Some drugmakers pulled back from price increases in mid-2018 amid heightened political scrutiny, but prices went up for many drugs at the start of 2019.

Marijuana-Derived Drugs Show Promise

 

CVS Discusses New Stores

CVS Health Corp. Chief Executive Larry Merlo began showing initial concepts the company will be testing as it begins piloting new models of its drugstores that incorporate its Aetna combination. The first new test store will open next month in Houston, he told investors, and it will include expanded health-care services including a new concierge who will help patients with questions. 

Aetna Savings On the Way

Mr. Merlo also spelled out when the company will achieve the initial $750 million in synergies it has promised from the CVS-Aetna deal. In the first quarter, he said the company will see benefits from consolidating corporate functions. Savings from procurement and aligning lists of covered drugs should be seen in the first half, he says. Medical-cost savings will start affecting results toward the end of the year, he noted. 

Lilly Cuts Price

Drugmaker Eli Lilly & Co. expects average net US pricing for its drugs–after rebates and discounts–to decline in the low- to mid-single digits on a percentage basis this year, Chief Financial Officer Josh Smiley told the Journal. Lilly’s net prices had risen during the first half of 2018, but dropped in the third quarter as the company took a “restrained approach,” Mr. Smiley said. Lilly, which hasn’t yet reported fourth-quarter results, took some list price increases for cancer drugs in late December but hasn’t raised prices in the new year, he said.

Peter Loftus at peter.loftus@wsj.com and Anna Wilde Mathews at anna.mathews@wsj.com

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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.

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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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