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Archive for the ‘Pharmaceutical R&D Investment’ Category


Novartis’ Kymriah (tisagenlecleucel), FDA approved genetically engineered immune cells, would charge $475,000 per patient, will use Programs that Payers will pay only for Responding Patients

Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED on 9/1/2017:

This Pioneering $475,000 Cancer Drug Comes With A Money-Back Guarantee

Novartis defends the eye-popping price of its pioneering gene therapy with arguments about its $1 billion expenditure—and novel “value-based” pricing.

https://www.fastcompany.com/40461214/how-novartis-is-defending-the-record-475000-price-of-its-pioneering-gene-therapy-cancer-drug-car-t-kymriah

 

On 8/30/2017 we wrote:

FDA has approved the world’s first CAR-T therapy, Novartis for Kymriah (tisagenlecleucel) and Gilead’s $12 billion buy of KitePharma, no approved drug and Canakinumab for Lung Cancer (may be?)

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2017/08/30/fda-has-approved-the-worlds-first-car-t-therapy-novartis-for-kymriah-tisagenlecleucel-and-gileads-12-billion-buy-of-kite-pharma-no-approved-drug-and-canakinumab-for-lung-cancer-may-be/

 

The Price for the Treatment was published on 8/31/2017, a Value-based Pricing Payment Model of a $475,000 per patient charge for the responding patients after ONE month of treatment. Novartis says it takes an average of 22 days to create the therapy, from the time a patient’s cells are removed to when they are infused back into the patient. Kymriah will initially be available at 20 U.S. hospitals within a month, Novartis says. Eventually, 32 total sites will offer the therapy. 

CAR-T gained national attention three years ago when Carl June, a researcher at the University of Pennsylvania, used to put a young girl’s acute lymphoblastic leukemia. Genetically altering the girl’s immune cells had made her deathly ill, but June had used a Roche drug, Actemra, to treat the side effects. She lived, and the results were published in The New England Journal of Medicine. Novartis bought the rights to the Penn treatment for just $20 million up front.

Pharma Buying the right to use from an Academic Institution is a known route to leap frog the R&D lengthy process of Drug discovery.

“I’ve told the team that resources are not an issue. Speed is the issue,” says Novartis’ Chief Executive Joseph Jimenez, told Forbes in a cover story about the work then.

The FDA calls this CAR-T therapy treatment, made by Novartis, the “first gene therapy” in the U.S. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults. The FDA defines gene therapy as a medicine that “introduces genetic material into a person’s DNA to replace faulty or missing genetic material” to treat a disease or medical condition. This is the first such therapy to be available in the U.S., according to the FDA.

Two gene therapies for rare, inherited diseases have already been approved in Europe.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA’s Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

FDA commissioner Scott Gottlieb in a statement.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” 

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm

The Protocol

A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.

In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived.

https://www.technologyreview.com/s/608771/the-fda-has-approved-the-first-gene-therapy-for-cancer/?utm_campaign=add_this&utm_source=email&utm_medium=post

CAR-T Therapies: Product/Molecules/MOA under Development:

  • Similar CAR-T treatments were being developed at other institutions including
  • Memorial Sloan-Kettering Cancer Center,
  • Seattle Children’s Hospital, and
  • The National Cancer Institute.
  • The Memorial and Seattle work was spun off into a startup called Juno Therapeutics, which has fallen behind. Juno Therapeutics ended a CAR-T study earlier this year after patients died from cerebral edema, or swelling in the brain.
  • The NCI work became the basis for the product being developed by Kite Pharma. Kite Pharma, which is awaiting FDA approval for its CAR-T therapy to treat a form of blood cancer in adults, was this week bought out by Gilead in a deal worth $11.9 billion.

On Cambridge Healthtech Institute’s 4th Annual Adoptive T Cell Therapy, Delivering CAR, TCR, and TIL from Research to Reality, August 29 – 30, 2017 | Sheraton Boston | Boston, MA

TUESDAY, AUGUST 29 – I covered in Real Time the talk on Juno Therapeutics: Building Better T Cell Therapies: The Power of Molecular Profiling by Mark Bonyhadi, Ph.D., Head, Research and Academic Affairs, Juno Therapeutics

https://pharmaceuticalintelligence.com/2017/08/29/live-829-chis-oncolytic-virus-immunotherapy-and-adoptive-cell-therapy-august-28-29-2017-sheraton-boston-hotel-boston-ma/

 

Precision Medicine is Costly and not a Rapid manufacturing process

All of the CAR-T products are expensive to make, and must be manufactured on an individual basis for each new patient from the patient’s own T-cells, a type of white blood cells, a process that takes weeks.

  • How quickly companies can speed up manufacturing.
  • Kymriah will be manufactured at a facility in Morris Plains, N.J.
  • CAR-T technology, which has so far been used only in patients with blood cancers that have not been cured by other treatments, can be used earlier in the disease or for solid tumors: Breast, Prostate, Melanomas.

https://www.forbes.com/sites/matthewherper/2017/08/30/fda-approves-novartis-treatment-that-alters-patients-cells-to-fight-cancer/#2aecb25b4400

Prediction How Patients will Far Well – Researchers use a big-data approach to find links between different genes and patient survival.

https://www.technologyreview.com/s/608666/a-cancer-atlas-to-predict-how-patients-will-fare/?set=

A pathology atlas of the human cancer transcriptome

+ See all authors and affiliations

Science  18 Aug 2017:
Vol. 357, Issue 6352, eaan2507
DOI: 10.1126/science.aan2507

Modeling the cancer transcriptome

Recent initiatives such as The Cancer Genome Atlas have mapped the genome-wide effect of individual genes on tumor growth. By unraveling genomic alterations in tumors, molecular subtypes of cancers have been identified, which is improving patient diagnostics and treatment. Uhlen et al. developed a computer-based modeling approach to examine different cancer types in nearly 8000 patients. They provide an open-access resource for exploring how the expression of specific genes influences patient survival in 17 different types of cancer. More than 900,000 patient survival profiles are available, including for tumors of colon, prostate, lung, and breast origin. This interactive data set can also be used to generate personalized patient models to predict how metabolic changes can influence tumor growth.

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Expedite Use of Agents in Clinical Trials: New Drug Formulary Created – The NCI Formulary is a public-private partnership between NCI, part of the National Institutes of Health, and pharmaceutical and biotechnology companies

Reporter: Aviva Lev-Ari, PhD, RN

 

Wednesday, January 11, 2017

New Drug Formulary Will Help Expedite Use of Agents in Clinical Trials

The National Cancer Institute (NCI) today launched a new drug formulary (the “NCI Formulary”) that will enable investigators at NCI-designated Cancer Centers to have quicker access to approved and investigational agents for use in preclinical studies and cancer clinical trials. The NCI Formulary could ultimately translate into speeding the availability of more-effective treatment options to patients with cancer.

The NCI Formulary is a public-private partnership between NCI, part of the National Institutes of Health, and pharmaceutical and biotechnology companies. It is also one of NCI’s efforts in support of the Cancer Moonshot, answering Vice President Biden’s call for greater collaboration and faster development of new therapies for patients. The availability of agents through the NCI Formulary will expedite the start of clinical trials by alleviating the lengthy negotiation process — sometimes up to 18 months — that has been required for investigators to access such agents on their own.

“The NCI Formulary will help researchers begin testing promising drug combinations more quickly, potentially helping patients much sooner,” said NCI Acting Director Douglas Lowy, M.D. “Rather than spending time negotiating agreements, investigators will be able to focus on the important research that can ultimately lead to improved cancer care.”

The NCI Formulary launched today with fifteen targeted agents from six pharmaceutical companies:

  • Bristol-Myers Squibb
  • Eli Lilly and Company
  • Genentech
  • Kyowa Hakko Kirin
  • Loxo Oncology
  • Xcovery Holding Company LLC

“The agreements with these companies demonstrate our shared commitment to expedite cancer clinical trials and improve outcomes for patients,” said James Doroshow, M.D., NCI Deputy Director for Clinical and Translational Research. “It represents a new drug development paradigm that will enhance the efficiency with which new treatments are discovered.”

The establishment of the NCI Formulary will enable NCI to act as an intermediary between investigators at NCI-designated Cancer Centers and participating pharmaceutical companies, facilitating and streamlining the arrangements for access to and use of pharmaceutical agents. Following company approval, investigators will be able to obtain agents from the available formulary list and test them in new preclinical or clinical studies, including combination studies of formulary agents from different companies.

The NCI Formulary leverages lessons learned through NCI’s Cancer Therapy Evaluation Program (CTEP) and the NCI-MATCH trial, a study in which targeted agents from different companies are being tested alone or in combination in patients with genetic mutations that are targeted by these drugs. As the use of genomic sequencing data becomes more common in selecting cancer therapies, requests for access to multiple targeted agents for the conduct of clinical trials are becoming more common.

“We are very pleased that several additional pharmaceutical companies have already pledged a willingness to participate and are in various stages of negotiation with NCI,” said Dr. Doroshow, who is also director of NCI’s Division of Cancer Treatment and Diagnosis. “By the end of 2017, we expect to have doubled the number of partnerships and drugs available in the NCI Formulary.”

CTEP staff continue to discuss the NCI Formulary with pharmaceutical companies to make additional proprietary agents available for studies initiated by investigators at NCI-designated Cancer Centers.

The Formulary will complement NIH’s plans for another new public-private partnership in oncology, the Partnership to Accelerate Cancer Therapies (PACT). Through PACT, the NIH, U.S. Food and Drug Administration, biopharmaceutical groups in the private sector, foundations, and cancer advocacy organizations will come together to support new research projects to accelerate progress in cancer research as part of the Cancer Moonshot. PACT research will center on the identification and validation of biomarkers of response and resistance to cancer therapies, with special emphasis on immunotherapies. PACT will also establish a platform for selecting and testing combination therapies. PACT is expected to launch in 2017.

About the National Cancer Institute (NCI): NCI leads the National Cancer Program and the NIH’s efforts to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI website at cancer.gov or call NCI’s Cancer Information Service at 1-800-4-CANCER.

About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

SOURCE

https://www.nih.gov/news-events/news-releases/new-drug-formulary-will-help-expedite-use-agents-clinical-trials

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Value for Patients – Turning Advances in Science: A Case Study of a Leading Global Pharmaceutical Company – Astellas Pharma Inc.

Astellas Pharma Inc. (https://www.astellas.com/en/) and Astellas Pharma U.S., Inc. (https://www.astellas.us/)

UPDATED on 4/3/2017

Astellas Pharma Inc. and Ogeda SA announced today that Astellas and Ogeda shareholders have entered into a definitive agreement under which Astellas has agreed to acquire Ogeda a privately owned drug discovery company. Ogeda is a clinical-stage drug discovery company that discovers and develops small molecule drugs targeting G-protein coupled receptors (GPCRs). The lead investigational candidate, fezolinetant, is a selective NK3 receptor antagonist, and the positive data from a Phase 2a study result for the non-hormonal treatment of menopause-related vasomotor symptoms (“MR-VMS”) was announced in January 2017. This transaction expands Astellas’ late stage pipeline and is expected to contribute to its mid-to-long term growth.

SOURCE

http://www.prnewswire.com/news-releases/astellas-to-acquire-ogeda-sa-300433141.html

https://endpts.com/astellas-swoops-in-on-a-mid-stage-drug-for-hot-flashes-in-860m-biotech-buyout-deal/?utm_medium=email&utm_campaign=Monday%20%20April%203%202017&utm_content=Monday%20%20April%203%202017+CID_4adac18d4a997566831a3ca0829b655e&utm_source=ENDPOINTS%20emails&utm_term=Astellas%20swoops%20in%20on%20a%20mid-stage%20drug%20for%20hot%20flashes%20in%20860M%20biotech%20buyout%20deal

UPDATED on 8/24/2016

Some analysts suggested Pfizer paid too much, particularly since it will split profits from Xtandi with Japan-based Astellas Pharma, which helps market the drug. Pfizer defended the deal, saying it would add 5 cents to its earnings per share in the first full year.

“The proposed acquisition of Medivation is expected to immediately accelerate revenue growth and drive overall earnings growth potential for Pfizer,” Ian Read, chairman and chief executive of Pfizer, said in the statement on Monday.

SOURCE

http://www.nytimes.com/2016/08/23/business/dealbook/medivation-pfizer-14-billion-deal.html?_r=0

Author: Gail S. Thornton, M.A.

Co-Editor: The VOICES of Patients, HealthCare Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures  https://pharmaceuticalintelligence.com/biomed-e-books/series-e-titles-in-the-strategic-plan-for-2014-1015/2014-the-patients-voice-personal-experience-with-invasive-medical-procedures/

 

Tokyo-based Astellas Pharma Inc., a top 20 global pharmaceutical research company, has a strong, global company legacy, precision focus and patient-centric vision in creating innovative pharmaceuticals in areas of unmet medical need.

2012-05-10 003_Astellas building

Image SOURCE: Photograph of the Astellas Pharma U.S. building. Courtesy of Astellas Pharma U.S., 5/10/2012.   

The company’s commitment to science is based on development of medicines that address high unmet medical needs in therapeutic areas that include:

  • oncology,
  • urology,
  • immunology,
  • nephrology, and
  • neuroscience.

The company is also exploring advancements in new therapeutic areas and related diseases such as,

  • ophthalmology—retinitis pigmentosa (RP), age-related macular degeneration (AMD), diabetic macular edema (DME) and Stargardt’s macular degeneration (SMD) and
  • muscle diseases.

And they are investing in new technologies and modalities, such as,

  • regenerative medicine and cell therapy, and
  • next-generation vaccines.

The company is committed to improving the lives of patients through innovative science and with the highest sense of ethics and integrity. This commitment is reflected in the Astellas Group Code of Conduct, which applies to all employees across the globe and can be accessed through the link below.

Astellas Group Code of Conduct

Boosting research and development productivity remains an important issue for Astellas Pharma Inc., because innovation is vital for the company’s success in developing new therapeutic areas, technologies and modalities of treatment.

Dr. Bernhardt Zeiher, President, Development, is responsible for the more than 800-person development organization that is involved in developing these innovative therapies through cutting-edge clinical research. Dr. Zeiher’s team conducts clinical investigations of novel biological targets and new chemical entities with unique mechanisms of action and looks to determine whether the findings in preclinical testing will translate to benefit for patients.  Clinical studies are conducted globally with operational hubs in the United States, Netherlands and Japan. Astellas relocated their Development headquarters from Japan to the United States in 2008.

Building on its 120-year heritage, Astellas uses creativity and innovation to bring patients new medicines through the more than 17,000 global employees who work to improve the lives of patients and their families. Astellas was formed through the merger of Japan’s third and fifth largest pharmaceutical companies, Yamanouchi, founded in 1923, and Fujisawa, founded in 1894. Yamanouchi brought a record of developing blockbuster drugs, a pipeline full of promising new compounds and a sales and marketing culture of deeply grounded, data-driven expertise. Fujisawa brought dominance in transplantation, a soaring reputation for in-depth understanding of the disease states and treatments within its market niches, and a track record for developing high-profile, market-leading products that become new standards of care.

The company has made steady progress; they reported annual global sales of 1,372,706 million yen (approx. $13.2 billion) through the end of fiscal year 2015, with an annual research and development investment of 225,665 million yen (approx. $2.2 billion) through the end of fiscal year 2015.

Below is my interview with Astellas Dr. Bernhardt Zeiher, President, Development, which occurred in June, 2016.

What is your overall Research & Development (R&D) strategy?

Dr. Zeiher: We are focused on turning innovative science into value for patients in areas of high unmet need where we have, or can quickly acquire, expertise and where Astellas believes new scientific understanding is poised to drive significant innovation. Our commitment to R&D is based on the development of medicines that address high unmet medical needs in our main therapeutic areas of focus: oncology, urology and immunology.  We also have increased efforts to explore advancements in new therapeutic areas such as ophthalmology, nephrology, neuroscience and muscle diseases where there is a high level of unmet medical need. Building on our patient-centric vision, Astellas has been actively investing in new technologies and modalities, such as regenerative medicine and next-generation vaccines.

What are your R&D strengths?

Dr. Zeiher: Astellas is building on its legacy of bringing transformative medications to patients by investing in some of today’s most dynamic areas of scientific exploration. Innovations delivered by Astellas have helped to address and largely solve some of the most significant scientific challenges in urology and transplant. We also have built a strong presence in oncology with treatments for difficult-to-treat cancers, such as prostate and non-small cell lung cancer.

Moving forward in oncology, Astellas has made a deliberate effort to build leadership through organic efforts with a pipeline exemplifying the “follow the biology” approach that includes treatments for prostate, non-small cell lung and pancreatic cancer, and continued research in therapies for breast cancer and acute myeloid leukemia, among others. We also have forged strategic acquisitions and collaborated with industry and academic leaders to further build our portfolio.

In addition, we are leveraging what we know across conditions with similar biologies or mechanisms, building on our expertise to expand into adjacent diseases and proactively seek new opportunities. For example, leveraging our expertise in transplantation and infectious diseases, Astellas is developing the world’s first DNA vaccine for cytomegalovirus (CMV) infections. Currently in clinical trials, ASP0113 is a potential first-in-class agent for immunocompromised individuals undergoing solid organ or hematopoietic stem cell transplant who are at high risk of viral reactivation.

Describe your near-term R&D projects and pipeline activities?

Dr. Zeiher: Currently, the company is working on 35 investigational programs in Phase II and Phase III/registration development, of which half involve new molecular entities. We have a diverse pipeline with a balance of early- and later-stage assets. Later-stage programs include novel therapies/vaccines for cancer, anemia and infectious diseases.

  • Our two most advanced novel oncology agents, ASP2215 and ASP8273, continue to progress through the pipeline. ASP2215 shows promise in the treatment of relapsed or refractory acute myeloid leukemia, and ASP8273 is being evaluated as a treatment for a type of non-small cell lung cancer.
  • Leveraging our expertise in kidney disease, we are developing a first-in-class oral treatment for anemia associated with chronic kidney disease through our licensing agreement with FibroGen.
  • Astellas is developing the world’s first DNA vaccine for cytomegalovirus (CMV) infections. Currently in clinical trials, ASP0113 is a potential first-in-class agent for immunocompromised individuals undergoing solid organ or hematopoietic stem cell transplant who are at risk of viral reactivation. We are also working on a therapeutic vaccine, ASP4070, for Japanese red cedar pollen allergy.

We are building expertise in two new therapeutic areas—ophthalmology and muscle diseases—where there is significant unmet need. Through the Astellas Institute for Regenerative Medicine (AIRM) and external collaborations, we are addressing ophthalmologic diseases with a higher risk of blindness, including age-related and Stargardt’s macular degeneration, retinitis pigmentosa (RP), and diabetic macular edema (DME). In the muscle disease area, we are collaborating with our partner, Cytokinetics, on a skeletal muscle troponin activator which is being investigated in Spinal Muscular Atrophy (SMA). In addition, Astellas and Cytokinetics have agreed to amend their collaboration agreement to enable the development of CK-2127107 for the potential treatment of ALS and to extend their joint research focused on the discovery of additional next-generation skeletal muscle activators through 2017.

The pharmaceutical industry is intensely competitive and it requires an extensive search for technological innovations. How are you positioned to be a leader in developing new medicines that address unmet medical needs in critical therapeutic areas?

Dr. Zeiher: Astellas is focused on accelerating scientific discovery with an open innovation model. The Astellas open innovation model combines in-house R&D with strategic merger and acquisition approaches to advance research in untouched and complex disease states, allowing the company to maintain steady productivity and maximize its return on R&D investment.

With open innovation, Astellas undertakes research activities in the best possible environment. In some cases, the best environment is within the Astellas research laboratories. In many other cases, we look to collaborate with top biotech and academic leaders.  By building partnerships with top researchers and companies that complement our existing expertise, Astellas is able to quickly advance into new technologies and therapeutic areas of research where there is significant unmet medical need.

This approach has helped Astellas credibly enter into, compete and lead in some segments of the most competitive therapeutic areas in the pharmaceutical industry – oncology – and is accelerating the company’s efforts to develop treatments for important emerging therapeutic categories, such as ophthalmology and musculoskeletal disease, as well as leading technologies, such as regenerative medicine and vaccines.

For example, LAMP-vax is a next-generation DNA vaccine that utilizes the body’s natural cellular processing of Lysosomal Associated Membrane Protein (LAMP) to develop a more complete immune response to a target antigen.  The ability to activate a more complete immune response gives the LAMP-vax technology potential across a number of diseases, including allergic disease and cancer immunotherapy.  In 2015, Astellas established a licensing agreement with Immunomic Therapeutics, Inc. for the LAMP-vax products for the treatment or prevention of any and all allergic diseases in humans, including ARA-LAMP-vax for peanut allergy and other research-stage programs for food or environmental allergies.

Earlier this year, Astellas acquired Ocata Therapeutics, Inc., and established the Astellas Institute for Regenerative Medicine (AIRM) to serve as the global hub for Astellas regenerative medicine and cell therapy research. Our most advanced cellular therapy programs are in ophthalmology, but we are exploring other therapeutic areas. We are working on treatments for ophthalmologic diseases that leave patients at risk for blindness, which include retinitis pigmentosa (RP), age-related macular degeneration (AMD), and Stargardt’s macular degeneration (SMD).

Zeiher_Bernie

Image SOURCE: Photograph of Dr. Bernhardt Zeiher, President of Development, at Astellas. Courtesy of Todd Rosenberg, 11/17/2014. 

Dr. Bernhardt Zeiher serves as President, Development, at Astellas. In this role, he is responsible for all phases of drug development.

Prior to his current role, Dr. Zeiher was executive vice president and Therapeutic Area head, Immunology, Infectious Diseases and Transplantation at Astellas. Of note, he led the development of CRESEMBA® (isavuconazonium sulfate), which received Qualified Infectious Disease Product (QIDP) designation from the U.S. Food and Drug Administration and was approved in 2015 for the treatment of two rare invasive fungal infections. Prior to joining Astellas, he served as vice president of the Inflammation/Immunology therapeutic area at Pfizer.

Dr. Zeiher earned his Doctor of Medicine at the Case Western Reserve University School of Medicine, and completed an internal medicine residency at University Hospitals of Cleveland as well as a fellowship in Pulmonary and Critical Care Medicine at University of Iowa Hospitals and Clinics. Dr. Zeiher has received several awards, including being named a Fellow by American College of Physicians in 2004, awarded to those who demonstrate excellence and contributions to both medicine and the broader community of internists.

Editor’s note:

We would like to thank Jeff Winton, Andrew Lewis and Julie Monzo from the Astellas communications team for the tremendous help and support they provided during this interview.

 

REFERENCE/SOURCE

Astellas Pharma Inc. (https://www.astellas.com/en/) and Astellas Pharma U.S., Inc. (https://www.astellas.us/)

Other related articles:

Retrieved from http://3blmedia.com/News/Astellas-USA-Foundation-Giving-Back-Means-Living-Smarttm-Together-Our-Communities

Retrieved from http://3blmedia.com/News/Astellas-and-World-Transplant-Games-Federation-Announce-Launch-Fit-Life-Promote-Physical

Retrieved from http://www.fiercepharma.com/pharma-asia/japan-s-astellas-shows-nearly-50-gain-q1-even-as-sales-drag-price-revisions

Retrieved from http://3blmedia.com/News/Astellas-Farma-Brasil-Ranked-Among-20-Greatest-Workplaces-Brazil

 

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

2016

LIVE 4:50 pm – 5:55 pm 4/25/2016 Early Detection and Prevention of Cancer & Innovation Break: Announcing the C³ Prize from Astellas Oncology and the World Medical Innovation Forum @2016 World Medical Innovation Forum: CANCER, April 25-27, 2016, Westin Hotel, Boston

https://pharmaceuticalintelligence.com/2016/04/25/live-450-pm-555-pm-4252016-early-detection-and-prevention-of-cancer-innovation-break-announcing-the-c%C2%B3-prize-from-astellas-oncology-and-the-world-medical-innovation-forum-2016-world/

Top Seven Big Pharma in Thomson Reuters 2015 Top 100 Global Innovators

https://pharmaceuticalintelligence.com/2016/01/04/top-seven-big-pharma-in-thomson-reuters-2015-top-100-global-innovators/

Eye Lens Regenerated

https://pharmaceuticalintelligence.com/2016/03/19/eye-lens-regenerated/

 

2012

Picturing US-Trained PhDs’ Paths and Pharmaceutical Industry’s Crisis of Productivity: Partnerships between Industry and Academia

https://pharmaceuticalintelligence.com/2012/06/27/picturing-us-trained-phds-paths-pharmaceutical-industrys-crisis-of-productivity-partnerships-between-industry-and-academia/

Medicines in Development for Cancer in 2012: An Excellent Response from America’s Biopharmaceutical Research Companies

https://pharmaceuticalintelligence.com/2012/07/31/medicines-in-development-for-cancer-in-2012-an-excellent-response-from-americas-biopharmaceutical-research-companies/

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Announcement from LPBI Group: key code LPBI16 for Exclusive Discount to attend Boston’s Discovery on Target (September 19-22, 2016, CRISPR: Mechanisms to Applications on 9/19/2016)

DOT-646x200

Leaders in Pharmaceutical Business Intelligence (LPBI) Group is a Media Partner of CHI for CHI’s 14th Annual Discovery on Target taking place September 19 – 22, 2016 in Boston.

As a proud partner of this event, Leaders in Pharmaceutical Business Intelligence Group has secured a special discounted price for you to attend, resulting in a $200 discount on a commercial registration and $100 discount on an academic registration!

*This offer is valid for new registrants only, does not apply to previously registered attendees or short courses, and cannot be combined with any other offer. You must mention key code LPBI16 to receive this discount.

Don’t miss your opportunity to network with 1,100+ of your peers at this year’s event. Special early registration savings are currently available through Friday, August 12.

Preliminary AGENDA and Registration Link

http://www.DiscoveryOnTarget.com

For sponsorship & exhibit information, please contact: Jon Stroup, Sr Business Development Manager,
(+1) 781-972-5483, jstroup@healthtech.com

 

See us in CHI’s Media Partners section online:

http://www.discoveryontarget.com/Discoveryontarget_content.aspx?id=125312

Contact: 617-244-4024, avivalev-ari@alum.berkeley.edu

@pharma_BI

@AVIVA1950

ANNOUNCEMENT

Leaders in Pharmaceutical Business Intelligence (LPBI) Group, Boston

pharma_bi-background0238

will cover in REAL TIME

Cambridge Healthtech Institute’s

Discovery on Target

September 19-22, 2016,

CRISPR: Mechanisms to Applications 

September 19, 2016

Westin Boston Waterfront, Boston, MA

In Attendance, streaming LIVE using Social Media

Aviva Lev-Ari, PhD, RN

Editor-in-Chief

http://pharmaceuticalintelligence.com

and

Stephen J Williams, PhD

Senior Editor

http://pharmaceuticalintelligence.com

flyer2forApril2016BioWorld

 

Leaders in Pharmaceutical Business Intelligence (LPBI) Group is a Media Partner of CHI for CHI’s 14th Annual Discovery on Target taking place September 19 – 22, 2016 in Boston.

 

As a proud partner of this event, Leaders in Pharmaceutical Business Intelligence Group has secured a special discounted price for you to attend, resulting in a $200 discount on a commercial registration and $100 discount on an academic registration!

*This offer is valid for new registrants only, does not apply to previously registered attendees or short courses, and cannot be combined with any other offer. You must mention key code LPBI16 to receive this discount.

Don’t miss your opportunity to network with 1,100+ of your peers at this year’s event. Special early registration savings are currently available through Friday, June 3.

 

Preliminary AGENDA and Registration Link

http://www.DiscoveryOnTarget.com

For sponsorship & exhibit information, please contact: Jon Stroup, Sr Business Development Manager,
(+1) 781-972-5483, jstroup@healthtech.com

 

See us in CHI’s Media Partners section online:

http://www.discoveryontarget.com/Discoveryontarget_content.aspx?id=125312

Contact: 617-244-4024, avivalev-ari@alum.berkeley.edu

@pharma_BI

@AVIVA1950

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Recents Thoughts of Biotech Innovation: 2015 2016

From WorldofDTCMarketing

Can’t innovate ? Buy small biotech companies that can

cloud-innovationOn a week where a lot of people are taking their final summer vacations the news is that Amgen is buying Onyx and AstraZeneca Plc took a further step to bolster its pipeline of new cancer drugs on Monday by agreeing to acquire privately held U.S. biotech company Amplimmune for up to $500 million.  On paper it’s a good business move but as big pharma companies gobble up small biotech companies they bring with then antiquated processes and business people who are thinking about the bottom line rather than patients.  The results ?  Innovation that led these smaller biotech companies to develop new drugs will be stymied by a bureaucratic business model.

There is a reason why, after being acquired, that so many employees of smaller biotech companies leave.  Either they don’t want to work for big a big pharma bureaucracy or the acquiring company determines that these people are not needed and shows them the door.  Behind all this are people who provided the start-up funding and want to cash in without awaiting the lengthy process of developing new drugs.  In the end however it’s patients who loose.

bureaucracy

Last week Steve Ballmer, the CEO of Microsoft, announced his resignation.  There is a correlation between what happened at Microsoft and the challenges for big pharma.  Steve was forced out because Microsoft became a huge bureaucracy and could not innovate fast enough.  Those of us who have worked in pharma know of the endless 9-5 meetings to move even small projects forward.  Amgen’s culture revolves around back-to-back meetings with executives from other big pharma companies who are trying to put their power on display.  It’s only a matter of time before people from Onyx leave because of Amgen’s prohibitive culture.

Unknown

Until the costs of developing and launching new drugs is lower more and more innovative biotech firms are going to have a for sale sign hanging in the window hoping big pharma can help investors cash in.

And in a Commentary on CNBC

This is biotech’s real problem

Robert J. Mulroy, president and CEO of Merrimack

Thursday, 1 Oct 2015 | 9:38 AM ET

1

COMMENTJoin the Discussion

Here’s a challenge — name a biotech that’s not a small company with one potential blockbuster in the works or an industry giant that’s acquiring the hottest new technologies. Got one? Great! Now try to name four more.

Biotech

Jian Wan | Vetta | Getty Images

The fact is, midsize biotechs (Ironwood Pharmaceuticals andMedivation are couple of examples) are a rarity these days, and that’s a problem for patients, doctors and investors. Start-ups that are in the process of developing and drawing from a foundation of knowledge are often acquired once they have a promising candidate in the pipeline. If the associated research teams aren’t immediately jettisoned (just when their potential for broader breakthroughs is surging), the top innovators go off to launch another venture that doesn’t build on their current research.

There’s also enormous pressure to focus on that “next big thing” that can crowd out other innovations for patients, while blocking valuable, in-depth examination of existing treatments. In oncology, drug combinations (like Genentech’s combination of Herceptin, pertuzumab and docetaxel to treat HER-2 breast cancer) are making huge strides in prolonging patients’ lives. Such combinations require understanding how specific tumors grow, and designing diagnostics that tell doctors whether a patient’s tumor fits that profile. The problem? Not enough small biotechs have the luxury of developing that understanding before they’re acquired so that big biotechs can gain another drug candidate.

As the CEO of a cancer-focused biotech that’s spent the last 15 years building a diverse product pipeline — the lead candidate is under FDA review with a decision expected next month — my view is that pursuing individual drug targets will bring limited success. Cancer is the ultimate engineering challenge, and effective treatments need to address more than a single facet of the problem.

The real winners in the industry will be the companies that understand how their therapies work in combinations with their own and competing therapies, and help physicians make sense of the explosion of new treatments via companion diagnostics. In fact, regulators could potentially require a more integrated approach to manage the ever-increasing influx of new drugs and data. In August, the American Society of Clinical Oncology issued guidelines for doctors on interpreting multi-gene tests for cancer susceptibility, acknowledging the need for more education and regulation.

Most oncology biotech start-ups dream of developing such an integrated approach. But it takes time and money, and an environment that prioritizes in-depth scientific research.

Doing well by patients, doctors and investors means pursuing sustainable innovation, not just one-offs or single-use purchases. Innovation drives value and can build on itself to address complex challenges. And while innovation takes time and entails risk, it mitigates that risk in the long term.

For example, if you have a deep understanding of how your drug works — say, the tumor-growth mechanisms it disrupts — you can determine whether there are signs that the mechanism it targets is present in a particular patient and then enroll only those patients in clinical trials. That allows for smaller, less expensive trials — and a higher chance of success.

An integrated approach across the industry would allow drug developers to identify responders, and then eliminate the non-responders from clinical trials and from the target population post-approval, ensuring patients only receive treatments likely to benefit them and don’t waste their time enrolling in irrelevant trials.

The current cycle of big pharma acquiring start-ups and dismantling the research teams while divesting in their own R&D appears self-perpetuating, but cracks are showing in the high cost — now in the billions — of bringing a single drug to market.

These companies are dealing with outside pressures that stymie progress. Less than 10 percent of experimental oncology drugs ever get approved. A tactical approach to the pipeline makes sense from a risk-aversion perspective. But sustainable growth requires strategy and investments in the fundamental science work that drives innovation.

Commentary by Robert J. Mulroy, president and CEO of Merrimack, a biotech company focused on cancer treatments. Prior to joining Merrimack, Mr. Mulroy worked as a management consultant in the pharmaceutical and health-care industries. He has served as an advisor to multiple start-up companies in the biotechnology industry.

The New Biotechnology Innovation Organization

Jim's CornerAt BIO, new discoveries in research and development are constantly being made by our members. We take pride in the contributions they have made across a diverse range of biotechnology industries, including: healthcare, agricultural, industrial and environmental.

As one of the world’s strongest catalysts for innovation, our role within the biotechnology community requires us to reflect on who we are, what we do and how we can better serve our members in future.

Biotechnology scientists and entrepreneurs are not just industrious – they are revolutionary, imaginative, inspired, creative, ingenious and inventive. It is these traits that produce innovation.

BIO Logo Vertical RGBAs you may already know, starting today, the Biotechnology Industry Organization will become the Biotechnology Innovation Organization. It’s a one-word name change – from industry to innovation – but the implications are substantial.

Today is a time of tremendous innovation. So much so that our current name no longer best describes our members and our role as one of the world’s leading innovators.

BIO’s members are on the cutting-edge of science and we believe our new name will allow us to build upon our relationships, create new ones and provide our members with better educational and research opportunities.

Our members are discovering scientific breakthroughs and bringing new and innovative therapies to the marketplace. With the help of biotechnology, people are living longer and healthier lives. Our industry embodies innovation and made the world a better place for people everywhere.

Our meaningful innovations also provide the tools to help feed more people, develop new sustainable fuels and products to help protect the planet and devise unique clean technologies to make our environment safer.

In the more than 22 years since its founding, BIO has united scientists, policymakers and the public in a partnership to drive our remarkable progress even further.

It’s important to note that we are not becoming a different organization. We are not altering our mission or the value we deliver to our members.

We will, however, continue to blaze the trail to accelerate cures – connecting thought leaders, building a stronger, more advanced economy and creating jobs to raise the world’s standard of living.

In the coming years, BIO’s diverse membership – from promising startups to global companies in a wide array of biotechnology and related fields – will drive health, life expectancy and improve quality of life for millions of people.

The Biotechnology Innovation Organization will be there to support our members in their tireless effort to make the world a better place to live.

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Problem of Science Doctorate Programs

Larry H. Bernstein, MD, FCAP, Curator

LPBI

 

 

The Problem in Biomedical Education

Henry Bourne (UCSF)

Dr. Henry Bourne has trained graduate students and postdocs at UCSF for over 40 years. In his iBiology talk, he discusses the imminent need for change in graduate education. With time to degrees getting longer, the biomedical community needs to create experimental graduate programs to find more effective and low cost ways to train future scientists and run successful laboratories. If we don’t start looking for solutions, the future of the biomedical enterprise will grow increasingly unstable.

Watch Henry Bourne’s iBioMagazine: The Problem in Biomedical Education

https://youtu.be/V9peRqNr-L0

Henry Bourne is Professor Emeritus and former chair of the Department of Pharmacology at the University of California – San Francisco. His research focused on trimeric G-proteins, G-protein coupled receptors, and the cellular signals responsible for polarity and direction-finding of human leukocytes. He is the author of several books including a memoir, Ambition and Delight, and has written extensively about graduate training and biomedical workforce issues. Now Dr. Bourne’s research focuses on the organization and founding of US biomedical research in the early 20th century.

Related Talks

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The Valeant Sky Dive

Reporter: Larry H. Bernstein, MD, FCAP

 

UPDATED on 8/9/2016 at noon EST

Is the new Valeant just a shiny facade?

By Lisa LaMotta | August 9, 2016

http://www.biopharmadive.com/news/valeant-papa-earnings-directors-debt/424107/

 

The Roll-Up Racket    BY

Few falls in business history have been as sudden and as steep as that of Michael Pearson, the C.E.O. of the drugmaker Valeant. Not long ago, he was heading a company whose stock price had risen more than four thousand per cent during his tenure. A former McKinsey consultant, he had developed a strategy based on acquisitions, cost-cutting, and price hikes. The influential hedge-fund manager Bill Ackman, one of Valeant’s largest shareholders, compared Pearson to Warren Buffett, citing his genius at capital allocation. No one’s calling Pearson a genius anymore. In the past six months, Valeant’s stock price has fallen almost ninety per cent, thanks to a toxic combination of sketchy accounting, political blowback, and slowing growth. Two weeks ago, the company announced terrible fourth-quarter earnings, and said that it wouldn’t be able to file its annual report on time, which drove the stock down fifty per cent in a day. Investors who once saw Pearson as a savior now consider him an albatross: when, last week, Valeant announced that he would step down, the stock price rose.

Valeant used to be a small drugmaker, struggling to stay afloat by doing what pharmaceutical companies typically do: invest heavily in R. & D. in order to discover new drugs. But Pearson, who took over in 2008, scrapped that approach. He argued that returns on R. & D. were too low and too uncertain; it made more sense to buy companies that already had products on the market, then slash costs and raise prices. So Valeant became a serial acquirer, doing more than a hundred transactions between 2008 and 2015. It invested almost nothing in its core business; R. & D. spending fell to just three per cent of sales. It was ruthless about bringing down costs, sometimes laying off more than half the workforce of a company it acquired. And though Martin Shkreli may be the public face of drug-price gouging, Valeant was the real pioneer. A 2015 analysis looked at drugs whose price had risen between three hundred per cent and twelve hundred per cent in the previous two years; of the nineteen whose prices had risen fastest, half belonged to Valeant.
The company also pulled every trick in the financial-engineering handbook. In 2010, it merged with a Canadian company, in order to bring down its tax rate, and it sheltered its intellectual property in tax havens like Luxembourg. It used opaque accounting methods that made it hard for investors to judge how well acquired companies were doing. To ward off competition from generic drugs, Valeant entered into a complicated relationship with a mail-order pharmacy called Philidor. Meanwhile, it paid its executives exceedingly well, and tied their compensation to shareholder returns, thus encouraging a single-minded focus on stock price. Valeant embodied practically everything that people hate about business today. So it’s no surprise that much of Wall Street saw it as a profit-making machine.

If Wall Street was happy, what went wrong? There were a couple of contingent problems: the dubious relationship with Philidor made people wary of Valeant’s accounting (the company just announced that it would have to re-state earnings for 2014 and part of 2015), while the political backlash provoked by Shkreli limited Valeant’s ability to raise prices. But the bigger problem was that Pearson’s buy-and-slash approach hit its inevitable limits. Valeant had become what’s known as a roll-up: a company that buys lots of other companies, trusting that they’ll be much more profitable together than they were apart. The challenge for roll-ups is that they have to keep feeding the beast: if you grow by buying, you have to keep buying to thrive. But, the bigger you get, the fewer deals there are that can truly boost your bottom line. And, because your grim reputation precedes you, you end up paying big premiums, which may mean that you have to start borrowing heavily. (Valeant’s debt is almost three times its annual sales.) Not surprisingly, roll-ups have a terrible track record. A Booz Allen study of the performance of eighty-one roll-ups between 1993 and 2000 found that only eleven did better than the market as a whole. Another study found that more than two-thirds of roll-ups created no value for investors at all. The only roll-ups that succeed are those which find, as one study put it, “a fundamentally superior way to make money.” Valeant’s collapse has shown that it had no such ability.

Valeant now says that its roll-up days are over, and that it’s going to focus on expanding its business “organically.” Yet it’s far from clear that this will be possible……

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