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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
GW Pharmaceuticals PLC last year secured the first U.S. FDA approval of a prescription drug derived from the marijuana plant to treat rare forms of epilepsy. Discussing the drug, Epidiolex, GW CEO Justin Gover told the Journal that the launch is going well and that there is a lot of demand. GW is pursuing additional uses of Epidiolex and is developing other experimental cannabinoid-based drugs to treat diseases including autism. “We’ve broken through all the stigma,” he said. “We’ve shown how cannabinoids can be bona fide medical treatments.”
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.
JP Morgan Healthcare Conference Update: Sage, Mersana, Shutdown Woes and Babies
Published: Jan 10, 2019By Alex Keown
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:
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.
bluebird bio’s chief executive officer, Nick Leschly, gave 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 acquiringLoxo Oncology, offered 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 CramerinterviewedAllergan 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 Bancel, spent 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:
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, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 1: Next Generation Sequencing (NGS)
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
Reporter: Stephen J. Williams, PhD
The annual J.P. Morgan Healthcare Conference is the largest and most informative healthcare investment symposium in the industry, bringing together industry leaders, emerging fast-growth companies, innovative technology creators, and members of the investment community.
Joe Biden on the Fight Against Cancer
Former Vice President of the United States joined the J.P. Morgan Healthcare Conference to discuss cancer initiatives.
Novartis Talks Move to Cell and Gene Therapies at JPM
Published: Jan 08, 2019By Alex Keown from Biospace.com
Denis Linine / Shutterstock
Following a strong post-hoc analysis of mid-stage data in the fall of 2018, Novartis announced this morning the company’s experimental humanized anti-P-selectin monoclonal antibody was crizanlizumab granted Breakthrough Therapy Status by the U.S.Food and Drug Administration (FDA).
Crizanlizumab received the designation as a treatment for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD). VOCs, which can be extremely painful for patients, happen when multiple blood cells stick to each other and to blood vessels, causing blockages.
The designation was awarded following results from the Phase II SUSTAIN trial, which showed that crizanlizumab reduced the median annual rate of VOCs leading to health care visits by 45.3 percent compared to placebo. The SUSTAIN study also showed that crizanlizumab significantly increased the percentage of patients who did not experience any VOCs vs placebo, 35.8 percent vs. 16.9 percent.
The FDA designation came one day after the Swiss pharma giant laid out its map for a future of success, sustainability and, if things work out, respect from consumers. In an interview with CNBC Monday, Novartis Chief Executive Officer Vas Narasimhan noted that the company is looking to become an entity that doesn’t draw its profits from treating disease, but will make money by providing cures. He pointed to the moves Novartis has made toward gene and cellular therapies that have the potential to cure patients of various diseases in what many researchers hope could be a “one-and-done” treatment. Narasimhan told CNBC that cures are what society wants and that is something they will value. The challenge will be determining the payment system.
As an example, the company is eying potential approval of a gene therapy for spinal muscular atrophy (SMA), a fatal genetic disease marked by progressive, debilitating muscle weakness in infants and toddlers. Novartis’ gene therapy Zolgensma is expected to be approved by the FDA this year and could have a price tag of between $4 and $5 million. While significantly high, non-profit SMA groups have already suggested that the gene therapy treatment could be more cost-effective than Spinraza, the only approved SMA treatment on the market.
During its presentation at J.P. Morgan, Novartis pointed to the moves it has made as the company pivots to this future of gene and cell therapies. The presentation noted that over the course of 2018, the company made several deals to sell off non-essential businesses, such as the $13 billion sale of its share of a consumer health business to partner GlaxoSmithKline. Not only that, but Novartis also made significant acquisitions to reshape its portfolio, including the $8.7 billion acquisition of AveXis for the SMA gene therapy. The deal for AveXis wasn’t the only gene therapy deal the company struck. Novartis began 2018 with a deal for Spark Therapeutics’ gene therapy Luxturna, a one-time gene therapy to restore functional vision in children and adult patients with biallelic mutations of the RPE65 (retinal pigment epithelial 65 kDa protein) gene.
In his interview with CNBC, Narasimhan said the company is about “platforms,” which also includes radio-ligand therapy. The company forged ahead in that area with two acquisitions, Advanced Accelerator Applications and Endocyte. Radiopharmaceuticals like Endocyte’s Lu-PSMA-617 are innovative medicinal formulations containing radioisotopes used clinically for both diagnosis and therapy. When the Endocyte deal was announced, Novartis noted the field is expected to become an increasingly important treatment option for patients, as well as a key growth driver for the company’s oncology business.
Other posts on the JP Morgan 2019 Healthcare Conference on this Open Access Journal include:
The total revenue from branded cardiovascular drugs in 2017 is projected to be just over $40 billion. Top companies in this space are putting a heavy focus on the anti-thrombotic segment, as these drugs continue to bring in the biggest profit and dominate the cardiovascular market.
Figures ($M) represent projected 2017 global sales of cardiovascular products. Credit: GlobalData.
Bayer currently holds the top spot in the market with 2017 sales of $5.4 billion; however, Sanofi and BMS come in a close second and third with sales of $5.1 and $4.7 billion, respectively.
Overall, apart from the anticoagulants segment, companies are investing less frequently due to the heavily genericized and mature nature of the cardiovascular market. This and the high cost of drug development makes the cardiovascular market hard to penetrate, especially for smaller, less established companies.
The clear leaders in sales of cardiovascular drugs are Eliquis and Xarelto (NOACs). These two drugs currently represent over 30% of the total cardiovascular market and their shares will continue to expand.
Despite high clinical unmet needs, this market is not expected to grow significantly in the near future, with the exception of the NOACs class.
Figures ($M) represent projected 2017 global sales. Credit: GlobalData.
Leadership we provide on curation of scientific findings in the eScientific publishing for Medical Education contents.
In Section 1, the Leadership we provide on curation of scientific findings in the eScientific publishing for Medical Education contents is demonstrated by a subset of several outstanding curations with high electronic Viewer volume. Each article included presents unique content contribution to Medical Clinical Education.
· These articles are extracted from the list of all Journal articles with >1,000 eReaders, 4/28/2012 to 1/29/2018.
Article Title, # of electronic Viewers, Author(s) Name
As BioMed e-Series Editor–in-Chief, I was responsible for the following functions of product design and product launch
· 16 Title creations for e-Books
· Designed 16 Cover Pages for a 16-Volume e-Books e-Series in BioMed
· Designed Series A eTOCs and approved of all 16 electronic Table of Contents (eTOCs), working in tandem with all the Editors of each volume and all the Author contributors of article contents in the Journal.
· Commissioned Articles by Authors/Curators per Author’s expertise on a daily basis
The Immune System, Stress Signaling, Infectious Diseases and Therapeutic Implications
9/4/2017
3747 pages
The VOICES of Patients, Hospitals CEOs, Health Care Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures
10/16/2017
826 pages
Medical Scientific Discoveries for the 21st Century & Interviews with Scientific Leaders
12/9/2017
2862 pages
Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics
12/27/2015
11125 KB
Medical 3D BioPrinting – The Revolution in Medicine, Technologies for Patient-centered Medicine: From R&D in Biologics to New Medical Devices
12/30/2017
1005 pages
Pharmacological Agents in Treatment of Cardiovascular Disease
Work-in-Progress, Expected Publishing date in 2018
???
Interventional Cardiology and Cardiac Surgery for Disease Diagnosis and Guidance of Treatment
Work-in-Progress, Expected Publishing date in 2018
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
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?)
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.
“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.”
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.
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 byMark Bonyhadi, Ph.D., Head, Research and Academic Affairs, Juno Therapeutics
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.
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.
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
istock/Astakhova
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.
Article ID #210: Value for Patients – Turning Advances in Science: A Case Study of a Leading Global Pharmaceutical Company – Astellas Pharma Inc. Published on 9/24/2016
WordCloud Image Produced by Adam Tubman
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.
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.
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.
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.
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).
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.
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
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)
Leaders in Pharmaceutical Business Intelligence (LPBI) Group is a Media Partner of CHI for CHI’s 14th AnnualDiscovery 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!
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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.
Leaders in Pharmaceutical Business Intelligence (LPBI) Group is a Media Partner of CHI for CHI’s 14th AnnualDiscovery 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.
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