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MedNess: At the Frontier of Healthcare Business/ March For Science- Special Report

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Cover Design: Ipsa Jain

Hello everyone and welcome to MedNess: At the frontier of healthcare news. The month of April was pretty crucial for Gilead Sciences and Novartis. Read below to find out more.

Also, this issue of MedNess is special as we are covering a report on March for Science from Club SciWri’s “Reporting from the lab” led by Radhika Raheja, Ph.D. Why are we covering March for Science on MedNess? Science affects us; the scientists, science affects our decisions and perspectives..still need more reasons? Check out our section on March for Science.

To stay on top of major scientific advancements, subscribe to ClubSciWri (www.sciwri.club)

Gilead’s NASH candidate clears early proof concept study

Gilead Sciences presented the first clinical trial data for GS-0976, an acetyl–CoA carboxylase inhibitor in patients with non-alcoholic steatohepatitis (NASH) at the 2017 International Liver Congress. The results are very preliminary but hopeful.

Gilead acquired GS-0976 from Nimbus Therapeutics in a billion-dollar deal last year.

This chronic liver disease affects around 15 million Americans. It is manifested by fat deposition in the liver and can cause scarring and liver fibrosis leading to liver failure (FierceBiotech, SeekingAlpha).

The clinical trial consisted of only 10 patients treated with 20mg dose OD for 12-weeks. GS-0976 blocked the formation of new fat in the liver by 29% and reduced liver fat by 43%. There was also a statistically significant decline in liver stiffness, marker for liver fibrosis, from 3.4 to 3.1kPa.

MedNess: Although on Friday, April 21, 2017, we did not see Gilead stocks soaring after the news, based on 19 analysts polled by TipRanks, majority approve buying Gilead stock while 7 maintain a hold and 0 recommend selling (SmarterAnalyst).

 FDA issues new warnings against the use of opioids in kids and nursing mothers

The Food and Drug Administration (FDA) issued a consumer safety alert on April 20, 2017, ordering major label changes on prescription drugs containing codeine and tramadol. Codeine is found in some prescription pain, and cough medicines and some over-the-counter cough medicines and tramadol is found in some prescription pain medicines. The opioid drugs are metabolized rapidly by children which can lead to breathing problems.

The FDA listed 15 medications and their generics that will be affected by this warning ranging from J&J’s Tylenol with codeine and Ultracet with tramadol, Vertical’s ConZip with tramadol, and Allergan’s migraine medication Fiorinol with codeine (FDA.gov)

FDA approves Roche’s Tecentriq as first line treatment for certain patients with advanced bladder cancer treatment

Roche’s Tecentriq gained accelerated approval from the FDA as a first-line treatment in patients with advanced bladder cancer who are ineligible for cisplatin chemotherapy. Tecentriq was earlier approved for treatment in patients with advanced or metastatic bladder cancer whose disease worsened within one year of standard chemotherapy.

MedNess: This immunotherapy was approved earlier for the treatment of non-small cell lung cancer (Roche.com). The current approval enables label expansion of this immunotherapy. As per Zacks ranking list, Roche stocks are strongly recommended for buying (Zack.com)

Novartis’ CAR-T CTL019 receives FDA “Breakthrough” Tag for the treatment of most common form of lymphoma

Novartis received the US Food and Drug Administration (FDA) Breakthrough Therapy designation for CTL019, an investigational chimeric antigen receptor T-cell (CAR-T) therapy. Last month, CTL109 received the breakthrough designation for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients.

This is the second indication for which CTL019 has received this designation for relapsed/refractory diffuse large B-cell lymphoma (DLBCL) for the treatment of adults who have failed two or more prior therapies.

The Breakthrough Therapy designation is based on data from the multi-center Phase II JULIET study. The results from JULIET study are expected to be presented soon (Novartis.com).

MedNess: Novartis is competing with Kite Pharma over CAR-T therapy. Kite already has breakthrough designations for DLBCL, transformed follicular lymphoma (TFL) and primary mediastinal B-cell lymphoma. After the announcement, Kites shares dropped by 1% and Novartis’s by 0.3% (FierceBiotech)

MedNess # March for Science by Radhika Raheja Ph.D

While we look at FDA approvals and how they affect pharmaceutical companies, it is important to acknowledge the science coming out of academic institutions that steers translational discoveries. Here is a brief report on how science impacts lives and why it is necessary to support scientific research.

Reporting from outside the lab – #marchforscience #Boston

This week we are not ‘Reporting from the lab” but from outside the lab where most of the excitement was happening. Thousands of scientists all over the world took to the streets to “March for Science” on April 22, usually observed as Earth Day.

Why do we march for science? We march for science because “ Science is real, denial is deadly”, “ No science, no beer”, “ Progress in science = progress in humanity”, “Climate change is real” and several other reasons. Here in Boston, the research community in the Longwood medical area marched under the motto “ Science is good for your health“. Our rally kick-started with a lineup of extremely eloquent speakers, faculty, students, patients and the Dean of Harvard Medical School (HMS) who spoke about the impact of science on our lives and the ramifications that reckless changes in science policy can have on all of us.

Why do we march for science? Science gives us the opportunity to give back to the community. The Dean for Students at HMS, Fidencio Saldana, emphasized that science affects all of us. “Science is for people who want to invest in the future of our children” he said, as science education creates awareness, teaches our children to think critically, ask questions and creates opportunities for them in the future. Fidencio Saldana ended by saying that there is, “too much at stake for us to remain silent anymore”. On a similar note, Senan Ebrahim, an MD-PhD student at the HMS reminded us, “to raise our voices and speak the truth, because we are blessed with knowledge and it is our responsibility to act on it and share it.” It is our duty to leave behind a legacy of advanced engineering, improved medical care and to safeguard the future for young and bright scientists.

Why do we march for science? Science gives us hope when we are afflicted with disease. This was further exemplified by the stories of patients and doctors on the innovative cures for various diseases including sickle cell anemia, acute lymphoblastic leukemia, neuroblastoma, lymphangioleiomyomatosis that have saved lives, thanks to fundamental scientific research conducted in laboratories within Boston and the nation. In order to continue fostering the promise of scientific discoveries, it is important to ensure our voices are heard. “Scientific research is one of the fundamental pillars of our society, … this is not a fight for our livelihoods, this is a fight for human lives “ said Elorm Avakame, an MD/MPH student at Kennedy School of Public Health.

Why do we march for science? “We march because we are facing a threat to humanity “. The proposed budget cuts within federal agencies like the National Institute of health itself will be dramatic as it has an annual budget of $32billion and propels scientific progress not only in the United states but in the world. “Such budget cuts, if applied, will have tragic consequences that will haunt us for generations, destabilize our economy and pose an existential threat to America’s preeminence as a world leader in biomedicine”, said George Daley, Dean of HMS. He also added that NIH funding supports over 380,000 jobs nationwide and over 31,000 jobs in the state of Massachusetts alone. Research funded by the NIH drives an economic activity of over $65 billion a year. “Scientific progress, scientific discovery is an enduring symbol of what is best and what is most noble about this great nation. Cutting biomedical research funding will eviscerate our ability to relieve suffering here and around the world. It threatens the very core of our mission”.

Boston plays a historic role in creating and nurturing some of the best scientists and physicians trained to alleviate human suffering caused by disease. Nearly half of new cancer drugs in the last 5 years emerged from the hard work and curiosity of scientists in the laboratories at Harvard University funded by grants from the National Institute of Health. In this era of phenomenal advancements in science, it is terrifying to envision the therapeutic landscape for various diseases without support for scientific research.

It was a cold, rainy day in Boston, with temperatures as low as 3oC (37oF) , yet this did not deter the spirit of the students, scientists, physicians, patients and people whose lives have been positively impacted by science to get out and stand up for science. We marched to reaffirm the importance of science and how it benefits our lives, our country, and our planet. We marched because science truly matters!

About the Authors:

Imit Kaur is a freelance medical writer, editor and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development.

 

Radhika completed her PhD from Cornell University and is currently a Postdoctoral fellow at the Brigham and Women’s Hospital. Her research interests have centered around oncology and neuroimmunology. Among other things, she is striving to effectively communicate scientific discoveries to the community.

 

 

The week that it was- 27th March to 2nd April, 2017

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  • 920864_10151596531186703_1560058850_o.jpg?fit=2048%2C1389

The new week starts, with the cherry blossoms bringing in optimism. And we are steadfast in bringing you the resources that you might need to alleviate your apprehensions, regarding funding, next steps in careers and the cutting edge science happening worldwide.

If you are a researcher in the US, and the NIH budget cuts are keeping you worried, you might want to see what does the proposal say to mitigate the cuts. Will it work or not, is yet to be seen. On the other hand, stable funding and impressive infrastructure attract researchers from all over the world to Canada. Is it your destination next too?

On the medical health and research front, researchers from IIT-Kanpur, India repurpose malaria drug to fight drug resistance in metastatic cancer. While efforts to eradicate tuberculosis are still on – Researchers in Houston, USA come up with a blood-based technique to detect TB, which costs less than 10$ while Serum Institute, India start with trial II/III vaccine trial for TB.

The entrepreneurs are taking health care to the next level with machine learning. Is really Elon Musk hacking the human brain? Or are NVidia graphic processors change the face of health care? Welcome to the future!!

Doudna and Charpentier rewarded on the European front – CRISPR Therapeutics win  a broad CRISPR patent in Europe.

After the last antibiotic developed in 1984, here is funding again in market by BARDA and Wellcome Trust to foster research in the not-so-economics-friendly antibiotic development.

Open science efforts, while are undeniably necessary, are also marred by fake predatory journals. But there are ways to identify them in the pool. Open science gets a boost as now big names start backing it – Bill and Melinda Gates Foundation and Chan Zuckerberg Biohub join the league. And if you are a researcher in Europe, do your bit by filling up the EU survey form to help the community understand how should they develop the infrastructure.

Depression and academia: the story isn’t new. It is not surprising that science and engineering population shows aging. The current researchers should know the different measures to prevent/ circumvent depression. And it is high time that we start investing in projecting science as a viable career option for it to thrive. Microsoft joins hands with Nat Geo in the pursuit by getting explorers to encourage girls take up STEM careers.

Ever felt retracting a paper might kill your career? Then you might love to know that the community rather rewards such an effort to correct ‘honest errors’.

Are you planning to further your career in data science? Then you should know what your CV should look like.

If you think that staying at home means a stepback in career, think again. Here is a list to help you pick from the jobs in life science that suit working from home.

Contract jobs are becoming more and more common each day in life science industry. If you are confused how to evaluate them, here is some help.

Max Planck for Molecular Physiology, Dortmund, Germany offers diverse postdoctoral openings for biochemistry, microbiology, mass spectrometry and electron microscopy backgrounds.

For those inclined towards industry-

  • Scientist I/II position at Bioverativ, Waltham, Massachusetts, USA, to work on blood disorders
  • Multiple positions at Entasis, Waltham, Massachusetts, USA
  • Scientist position, Neuroscience at Genentech, San Francisco, USA to work on nervous system diseases
  • Scientist, Electrophysiology at Sanford Burnham Prebys, La Jolla, USA in collaboration with GSK, to work on neurodegeneration
  • Senior scientist, Neuroscience Medicinal Chemistry at Johnson and Johnson, Flanders, Belgium to work in development of large and small molecules
  • Head of Immune Biology at Roche, Munich, Germany to work in protein therapeutics in Large Molecule Research group
  • Director for Research Communications at Spiegel lab at Yale University, USA

And for those who are still in the phase of exploring, here is a list of opportunities –

  • A 3 day insider’s look at management consultancy at McKinsey for those interested in Health Care (from 1st June to 4th June, 2017 at Philadelphia), and Science and Engineering (from 8th to 11th June, 2017 at Washington, DC) – open only for residents of US and Canada, application deadline – 9th April, 2017
  • Want to know more about filing patents? Here is a free webinar by IPWatchdog.com on 5th April that might be of use

And if you still think you are yet to find your right fit, be patient and steady. Take advantage of the resources here to know what suits your aptitude and attitude, and what skills need to be developed for a career appropriate for it.

About the author:

Somdatta Karak works with Club SciWri as a project co ordinator and Corporate Liaison. She is a doctorate in neuroscience from Georg August University, Göttingen, Germany and has been a Teach for India fellow (2014-16). She loves putting her analytical skills to build newer and more sustainable solutions, enjoys traveling and communicating and takes every opportunity to expand her horizon.

You can reach her here.

 

 

 

 

 

 

 

 

 

 

Multiple Sclerosis, Single Lead- Medness Focus on Ocrevus

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What’s the news?

The multiple sclerosis community has been eagerly awaiting the approval of the drug Ocrevus (ocrelizumab), which will be used to treat patients who have relapsing MS (RMS) and primary progressive MS (PPMS). The FDA’s decision of final approval came on March 28, coinciding with Multiple Sclerosis Awareness Week.

Ocrelizumab is the first and only investigational drug

  • to show greater efficacy in both RMS and PPMS in clinical studies.
  • to consistently and significantly reduce disease activity and disability progression compared with a standard-of-care high-dose interferon (Rebif®).
  • to significantly reduce the progression of physical disability in primary progressive MS in a large Phase III study (ORATORIO).
  • that has the potential to address an important unmet need in MS.

What is Multiple Sclerosis?

MS is a chronic, typically progressive disease involving damage to the sheaths of nerve cells in the brain and spinal cord.

Patients with MS may show paresthesias (tingling sensation), blurred vision, optic neuritis (painful unilateral vision loss), clumsiness, muscle weakness, cognitive decline, and urinary dysfunction. Unfortunately, the neuron in the picture is also feeling some of these signs. The Lhermitte sign is caused when neck flexion creates electric shock-like sensations down the back and limbs.

Types of MS

1.    Clinically Isolated Syndrome (CIS) First episode of neurologic symptoms caused by inflammation and demyelination in the central nervous system.

2.    Relapsing-remitting MS (RRMS) is characterized by clearly defined attacks of new or increasing neurologic symptoms.

3.    Primary Progressive MS (PPMS) worsening neurologic function (accumulation of disability) from the onset of symptoms, without early relapses or remissions.

4.    Secondary Progressive MS (SPMS) Most people who are diagnosed with RRMS will eventually transition to a secondary progressive course in which there is a progressive worsening of neurologic function (accumulation of disability) over time.

The History

In the past 20 years, we’ve seen a lot of improvement in the battle against MS to benefit the 2.3 million people worldwide who have this ailment. Notwithstanding these developments, people with RMS continue to need medications that offer the possibility for greater efficacy than standard-of-care interferons, with a favorable safety profile.

For people with PPMS there were no approved treatments before Ocrevus. Previous Phase III trials with investigational medicines have been unsuccessful in demonstrating a significant effect on disability progression in PPMS.

What is the science behind it?

Ocrevus

  • targets myelin-attacking B-cells (unlike similar medications attacking T-cells)
  • is an anti-CD20 humanized monoclonal antibody

Image source

How was the drug developed?

Genentech’s Medical Director, Peter Chin, said “The journey of ocrelizumab in MS started about 15 years ago, when Genentech began collaborating with academic researchers at major universities to investigate the importance of B cells in MS and their potential as a therapeutic target. The first small proof-of-concept studies showed that CD20+ B cells appeared to play a more important role in MS than anybody previously thought.”

Read the full interview with Genentech’s Peter Chin here

What was the outcome of the trials?

More than 1,600 MS patients enrolled in clinical trials for Ocrevus and 94 percent of participants had fewer brain lesions during the 96 weeks of treatment.

A summary of the data from the OPERA I, OPERA II and ORATORIO studies that support this approval can be found here.

What are some of the doubts?

There was little increased risk of infection (link). 

The clinical trials for Ocrevus also found that while patients taking the drug did have a slightly increased risk of common colds and flu, they had no significant increased risk of other infections when compared to patients taking the alternative medication, Rebif.

Some concerns like “Targeting B cells in MS patients appears to be Ocrevus’ strength, but depleting B cells also raises safety concerns” were addressed to Dr Chin. He responded saying, “Ocrelizumab selectively binds to CD20, a cell surface antigen expressed by a subset of B cells. CD20 is not expressed on stem cells or antibody-producing plasma cells, and therefore pre-existing humoral immunity and the ability to reconstitute B cells may be preserved. Since the CD20 protein is not found on many other cells of the immune system, they can continue to fight infection and other illnesses.

What should the patients and their families know?

Ocrevus

  • will be administered by intravenous infusion every six months.
  • will be used for treating primary progressive MS.
  • will also be used for treating relapsing MS patients.
  • may have potential serious side effects which may include infusion reactions, infections and malignancies where only routine screening is required based on age and medical history

Medness Quotient from Imit Kaur

“Analysts forecast annual sales exceeding $3 billion by 2021 as reported by Reuters. After the approval news, Biogen stock fell by 2%, and Roche stock rose by a fraction. Novartis’s drug for MS treatment, BAF312, for secondary progressive MS is expected to receive regulatory approval in the first half of 2017. Until then, Roche can bask in glory  (Reuters, Investor’s Business Daily, FiercePharma).”

Featured image source: Pixabay

References:

  1. https://www.gene.com/media/press-releases/14657/2017-03-28/fda-approves-genentechs-ocrevus-ocrelizu?platform=hootsuite
  2. http://www.nationalmssociety.org/What-is-MS/Types-of-MS
  3. https://multiplesclerosisnewstoday.com/2017/02/28/transcript-of-interview-with-genentech-peter-chin-on-ocrevus-for-multiple-sclerosis/

Disclaimer: This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

MedNess- March Mania

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Hello and welcome to yet another exciting week of MedNess. In this March mania, we bring the news from medicine and healthcare with the greatest impact.

Tom Price justifies NIH’s “indirect” budget cuts

Secretary of Health and Human Services (HHS) defended Trump’s administration proposed National Institute of Health’s budget cuts. The Trump administration proposed $5.8 billion, about 18% cut for the fiscal year 2018. In addition to that, an addendum proposed an additional $1.2 billion cut for the current fiscal year. When questioned by both democrats and republicans about the nature of budget cuts, Price explained to reduce the “overhead” costs to streamline the system. According to the Secretary of HHS, the “indirect” cost takes up about 30% of the grant money, which could otherwise be available for research.
This came in as a second blow to the medical research community. Earlier this year, Trump administration pushed deregulation of FDA to accelerate the drug approval process. The research community did not welcome this proposal. Also, both pharmaceutical companies and insurance companies did not approve of the proposition (STAT and Science).

 

March for Science, a scientist’s view:  For our readers, “overhead” or “indirect” costs constitute of anything required for carrying out research safely, smoothly and efficiently! Some of the “indirect” costs include lab equipment, electricity, custodial services and other utilities. The list is not inclusive but clearly, emphasizes the importance of overhead charges. The budget cuts will not only affect the advancement of research but will also impact jobs and outreach of science. For non-science professionals, say, politicians, the overhead cost will include, electricity, custodial services, security, dinner, travel, etc. Again, the list is not inclusive! Of course, these expenses are required for the smooth functioning of the government. Before I wrap up the section, just a thought: yes, we need the stronger military to defend the country, but we need to ask this question, who are we protecting; the people and the Mother Nature. Therefore, we need an effective EPA and NIH. We need the healthy and clean environment and disease-free children and adults.

Amgen’s LDL-lowering drug Repatha: effective drug with good data for a bad price?
Amgen presented Phase III FOURIER results on Repatha (Evolocumab) at the 2017 American College of Cardiology conference. Repatha is an LDL-lowering PCSK9 inhibitor. Repatha targets PCSK9 proteins in the blood stream thus preventing it from binding to and breaking down LDL cholesterol receptors in the liver. The trial results were impressive. This wonder drug is believed to break down the most stubborn cholesterol. The FDA approved drug marketing in 2015 after the drug’s addition to statins reduced the LDL levels by about 63%. At the ACC, Amgen reported the outcome of long-term FOURIER trial and the results showed that Repatha reduced the risk of heart attack and stroke by 15% or more. Repatha met both its primary and secondary composite endpoint in the secondary prevention trial demonstrating superiority to statin therapy. However, analysts are not too impressed with the data and question the high price of the drug. Earlier, analysts with BioPharmInsight suggested that the high price of Repatha could be justified if the cardiovascular event risk reduction is at least 35%. Repatha has been price tagged for $14,000 annually. The trial findings and analyst’s reports will also affect insurance coverage of the drug (MedPage Today).

MedNess: PCSK9 is the hottest target in the field of cardiovascular research. While Amgen’s drug can crush the most stubborn cholesterol molecules, the investors were not impressed, or at least the stock market trend did not concede with it. After the results had been announced, Amgen’s stock price went down by 10%. However, a lot of analysts are still keeping their faith in Amgen’s stocks and considering this temporary dip as an opportunity for investment. On the contrary, the competition from other biosimilars is getting fiercer, and the dip in stock price might not be temporary after all. Other contenders in this area with Amgen, are the drugs from Sanofi and Regeneron. Both the companies are locked in a patent battle with Amgen. Another drug in the race is The Medicine’s Company’s inclisiran. Unlike Amgen’s, Sanofi’s and Regeneron’s drug, inclisiran interrupts PCSK9 synthesis. The analysts look at this drug as efficacious as Repatha but with fewer annual doses. If this assumption is correct, Amgen will have a hard time convincing insurance companies for their drug price. The best bet might be therefore to either wait or invest wisely (The Motley Fool and Seeking alpha).

FDA approves Roche MS drug Ocrevus after 3-month delay

The FDA approved Roche MS drug Ocrevus after initial delay caused by regulator’s concerns over manufacturing issues.
Ocrelizumab, becomes the first U.S.-approved medicine for the primary progressive multiple sclerosis. It has also been approved for relapsing-remitting multiple sclerosis (RRMS). Biogen’s MS drug has been used to treat RRMS. Biogen will receive up to 24% royalty on U.S. sales of Ocrevus. According to the pharmaceutical giant; Ocrevus is expected to be available for use to people within two weeks.

 

MedNess: Analysts forecast annual sales exceeding $3 billion by 2021 as reported by Reuters. After the approval news, Biogen stock fell by 2%, and Roche stock rose by a fraction. Novartis’s drug for MS treatment, BAF312, for secondary progressive MS is expected to receive regulatory approval in the first half of 2017. Until then, Roche can bask in glory  (Reuters, Investor’s Business Daily, FiercePharma).

FDA approves Tesaro Inc’s Niraparib for the treatment of Ovarian Cancer

Tesaro’s Niraprib (Zejula) gained an early approval by FDA for the treatment of recurrent ovarian cancer. Zejula is a PARP inhibitor causing DNA damage. It is a first drug in the class that can be used to treat all women with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer without requiring BRCA mutation or biomarker evaluation. This is unlike the rival drug Lynparza by AstraZeneca. In addition, Zejula acquired orphan drug designation for its use in the treatment of recurrent epithelial ovarian cancer.

MedNess: According to EvaluatePharma, Zejula is one of the top drug launches of 2017 with 2022 sales expectations of $1.9 billion. Tesaro Inc’s shares were up 7.78 percent after the drug gained FDA approval (FiercePharma and BusinessInsider).

Illustration: Ipsa Jain

About the Author

Imit Kaur is a freelance medical writer, editor and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development.

MedNess- Immuno Oncology, Precision Medicine and more….

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Picture Illustration: Ipsa Jain

CAR-T therapy- a step closer to precision medicine?

Kite Pharma’s CAR-T candidate axicabtagene ciloleucel (previously referred to as KTE-C19) might be the first gene therapy to gain approval from FDA. The candidate therapy attained primary endpoints in a major study. The study encompassed patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma. The study showed that out of 101 patients enrolled in ZUMA-1 trial, 82% had their cancer shrunk at least by half after six months. In addition, 41% had partial response while 36% of patients went on complete remission. The therapy comes with its own share of risks. 3 patients in the study died and 2 of the deaths were attributed to the treatment.

CAR-T therapy, one of the most controversial treatments involving gene therapy, utilizes reengineered patient’s T cells. These T cells (immune system’s killer cells) are filtered from patients blood and altered in the lab and injected back intravenously making it a “living drug”.

Novartis and Juno Therapeutics are also in the race for CAR-T therapy. However, Juno Therapeutics announced the discontinuation of their experimental product early this month. This is because thirteen percent of patient deaths were reported, majorly due to cerebral edema and brain swelling. Kite Pharma’s CART-T cell product is safer in this regard. Kite Pharma’s axicabtagene ciloleucel was granted Breakthrough Therapy Designation status for diffuse large B cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma by the FDA and by the Food Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

MedNess: Although Kite Pharma has not presented formal results of the trial and is expected to present their report at the annual conference of American Association of Cancer Research in April this year, the reports of axicabtegene ciloleucel meeting primary endpoints raised the stock prices by 13% of this California-based biopharmaceutical company. Kite Pharma’s CAR-T product might be first in line to gain approval from FDA by the end of this year leaving behind the products from its competitors. Therefore, stocks of Kite Pharma hold a lucrative future. However, a lot can change in further studies and it all comes down to safety and efficacy of the final product (FiercePharma, STAT).

Barclays analyst’s stern advice to Gilead Sciences

Geoff Meacham, Barclays senior analyst apparently lost patience with Gilead Sciences, urging it to “do something”. He sent an open letter to the management prompting the company to either make an acquisition or take strict measures to improve sales and profit growth. Meacham suggested measures including Gilead’s orphan drugs diversification, cost cutting in Hep-C business due to declining market, HIV franchise clarification and/ or in-licensing deals in order to gain trust of its investors (Seekingalpha)

MedNess: Gilead Sciences sales and profit growth have eroded as its hepatitis C franchise has declined, the shares have slipped by 1% and profits have declined in each of the past five quarters.

Scott Gottlieb to lead FDA under Trump administration

Scott Gottlieb who has served as a practicing physician, clinical assistant professor at New York University and health information technology adviser for the department of Health and Human Services, has been selected as Trump’s nominee to lead FDA. The president’s selection is expected to yield support from biopharma industry. Gottlieb, if confirmed, is likely to hasten the drug approval process that might have earned him Trump’s support. He was a former deputy commissioner for medical and scientific affairs at the FDA under George W. Bush (FiercePharma).

Earlier this month, Club SciWri initiated a new section on Science and Policy, emphasizing on the involvement of scientists in the healthcare policy decisions. Gottlieb’s nomination to lead FDA will serve as a perfect example, underscoring the relevance of this sensitive subject.

BMS appoints Thomas Lynch as its new Chief Scientific Officer

BMS has been in news for quite a while now and that too for all the wrong reasons. Amongst the turmoil and speculations of its buyout, BMS appointed Thomas Lynch as its new Chief Scientific Officer (CSO) while the former CSO Francis Cuff made the exit this Wednesday. Thomas Lynch, an oncologist, was a former board member of BMS. With the new appointment, hopes are high on the Opdivo front as well. Recently, Opdivo fell short in a major clinical trial when tested on previously untreated lung cancer patients. Opdivo is PD-L1 checkpoint inhibitor and therefore its efficacy is expected to be better in patients with higher levels of this biomarker. However, BMS lost its non-small cell lung cancer lead to rival Merck’s Keytruda that succeeded in patients with a PD-L1 score of 50% or more. Also, earlier this year, BMS decided to not to seek accelerated approval for their Opdivo-plus-Yervoy combination in lung cancer. These two events combined with new checkpoint inhibitors expected from Roche, AstraZeneca, Merck and Pfizer have put BMS’s shares down. However, analysts suggest, BMS share might still be a great bargain if the drug succeeds in other arms of the trial, testing Opdivo as a monotherapy in first-line lung cancer. The company also awaits readouts from Phase 2 and Phase 3 studies evaluating Opdivo and Yervoy in other types of cancer (FiercePharma, The Motley Fool).

 

MedNess-Pill for Alzheimer’s?

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Hello and welcome to yet another exciting week of MedNess. We bring the news from medicine and healthcare with greatest impact. It seems like; year 2017 will be the year of neurology! It is just the second month of the year and treatment strategies for various neurological disorders are making headlines.

Merck halts Phase 3 study on Alzheimer’s drug- another setback for amyloid theory

Clinical trials on Verubecestat- a small molecule BACE 1 and BACE2 inhibitor were called off after an interim analysis on Phase 2/3 studies did not show promising results. The analysis team concluded that there was “virtually no chance of finding a positive clinical effect”. However, another trial on patients with early symptoms of Alzheimer’s will continue. It has been speculated that the drug was too weak, or was dosed inadequately or the disease had progressed too far in patients for the drug to show concrete effect. The failure of this trial is another blow to the famous “amyloid theory”. According to this theory, the amyloid plaques are believed to be cause of the disease. Verubecestat is a beta secretase inhibitor. This disappointing cessation of clinical trial came months after Eli Lilly’s Alzheimer’s drug; Solanezumab failed in Phase 3 clinical trials in November last year. Unlike Verubecestat, Solanezumab targets plaque rather than beta secretase enzyme. This brings in disappointment not only for the patients but also for the researchers. The evidence suggests that once the disease has advanced and patients have established dementia, the removal of amyloid plaque might not yield effective outcome.

                              Do we have a pill to cure Alzheimer’s? Some quick facts:

  • Alzheimer’s is an irreversible brain disorder causing cognitive impairment
  • More than 5 million Americans are expected to suffer from Alzheimer’s
  • Sixth leading cause of death in the USA
  • No new drug has been introduced to provide symptomatic relief or to halt its progression since last decade

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There are couple of drugs at various stages of trial that are being tested under the amyloid plaque hypothesis. These drugs either act on the plaque or beta secretase enzyme (BACE inhibitor) or available as amyloid immunotherapy. These candidate drugs are from Biogen, AstraZeneca, Eli Lilly, Amgen and Novartis. Apart from BACE inhibitors, hopes are also high for Axovant’s intepirdine. Intepirdine is believed to improve cognitive symptoms by targeting receptor 5-HT6 that stimulates the release of a neurotransmitter. Interestingly, intepirdine was abandoned by GSK in 2010. The drug failed when compared to placebo. However, one study showed tangible effect on cognitive symptom when intepirdine was paired with the approved Alzheimer’s drug Aricept.

MedNess: Merck’s stock suffered severe blow after the announcement of cessation of clinical trial. On the contrary, shares of Eli Lilly, AstraZeneca, Biogen and Roche, the fellow Alzheimer’s drug makers, increased. (Fierce Biotech, Business Insider, STAT news, The Boston Globe)

Axovant’s nelotanserin passes phase 2 study for Lewy body dementia

Axovant Sciences declared successful completion of phase 2 study of nelotanserin. The company is now setting its foot forward for phase 3 study that is expected to initiate later this year. Axovant Sciences reported preliminary results from the first small group of 11 patients.

Lewy body dementia or LBD is the second most common form of dementia. The hallmark characteristic of this form of dementia is the build up of abnormal proteins i.e. Lewy bodies thus affecting cognition, movement, behavior and alertness.

The study included patients with either LBD or Parkinson’s disease dementia. These patients experienced frequent hallucinations as assessed by mini mental state examination (Pharmaceutical Business Review)

CRISPR battle of patents: The Broad institute and MIT wins!

The scientists who first demonstrated the use of most powerful gene editing technology in biotech suffered a major blow on Wednesday, February 15, 2017, in their fight to gain exclusive rights on their invention. CRISPR gene editing system has revolutionized the field of biotechnology enabling scientists to make changes in DNA. Jennifer Doudna, a UC Berkely biochemist and her European collaborator Emmanuelle Charpentier first published this gene editing technology in prokaryotic system (type of bacterial system) in 2012 in Science. UC Berkely and University of Vienna filed for U.S. patent in March 2013. There were 155 broad claims to the CRISPR-Cas9 technology. Feng Zhang, a biologist at the Broad Institute, demonstrated the use of this technology in eukaryotic cells (type of plant cells, animal cells and human cells). The Broad Institute filed their patent in 2013; months after Berkeley group filed their patent. Since the patent claims by Broad Institute were fewer than Berkeley’s, the Broad Institute’s patent was issued on April 15 2014 through accelerated approval while Berkeley group is still awaiting their approval. After the Broad Institute was granted their patent, UC Berkeley filed an interference claiming that the Broad Institute should not have been granted the patent since Doudna’s and Charpentier’s CRISPR research outlined in 2012 paved the way for Zhang’s research in eukaryotic system. The Broad Institute argued that the research was not obvious and the patent claims from both the institutes were different. The federal Patent Trial and Appeal Board ruled out UC Berkeley’s claims and sided with the Broad Institute. With this decision, UC Berkeley plans to move forward with their patent application, which if approved, will provide them right on the use of CRISPR on all cells. This would also mean that if the technology will be employed commercially, the companies would have to get licenses from both the Broad and the Berkeley group.

MedNess: The patent decision in the favor of the Broad institute increased the stocks of Editas Medicine by 30%. Editas Medicine licenses Broad’s patents for human genetic disorders. (Fierce Biotech, STAT News, The LA Times, NPR, Wired)

Are pharmaceutical industries in favor of Trump’s FDA pick? The story so far….

Donald Trump is pushing deregulation of FDA in order to accelerate the drug approval process. His ideology: drug costs are higher, drug approval process through FDA takes forever, drug companies are involved in “unfair foreign trade”, drugs should be manufactured in the USA and finally, drug companies should add the innovation factor for the better cure of the diseases. This recipe will work in favor of patients to bring the overall drug costs down and patients can have quicker access to the newer agents. Not to forget, drug manufacturing in the USA brings back jobs and the “fair trade” promotes revenue generation. This all sounds good, except, the pharmaceutical industries have opposing views. The most common complaint of every patient and every healthcare researcher is the never-ending drug approval process by the FDA. So suddenly, when we might be able to overcome this hurdle, why is everyone (read the researchers, pharmaceutical companies and informed patients) so anxious? The truth is bitter sweet. Even though we rant over the FDA, we still knew, the FDA has best interests at heart and such a tight screen is probably important for the safety of the patients. In addition, a 2011, study found that the FDA usually approves cancer drugs before Europe does. Moreover, the researchers at Yale found the FDA’s drug review is at least a month faster than Europe’s or Canada’s.

The pharmaceutical industries on the other hand are concerned about the high drug costs. In addition to the limited patient safety, deregulation in the FDA might not provide enough time for pharmaceutical companies to justify high costs of the drugs to patients and to insurance companies. The pharmaceutical companies will not be able to account for high costs of the drugs owing the limited safety and efficacy analysis that ultimately affects both the patients and the companies. President Trump said last month he has a “fantastic person” lined up for the role of the FDA commissioner. A survey conducted by Mizuho Securities of drug company executives indicated that 72 percent agreed Scot Gottlieb should be Trump’s pick to head the FDA. Until then, we all wait! (Reuters, The New York Times, Forbes)

 

              

MedNess- At the frontier of Medicine, Pharmaceutical and Healthcare Business

in Poli-Scie/SciBiz/Uncategorized by

Hello and welcome to the biweekly roundup of Healthcare business top stories. Please follow us on Twitter and LinkedIn

MedNess 

BMS’s injectable Opdivo approved by FDA for bladder cancer

FDA approved intravenous use of Opdivo (nivolumab), a PD-1 checkpoint inhibitor for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have not benefitted from platinum-containing chemotherapy or in cases where the disease progressed within 12 months of neoadjuvant or adjuvant treatment with platinum containing chemotherapy. Last year, FDA approved Roche’s Tecentriq, a checkpoint inhibitor, for the treatement of bladder cancer.

From the business standpoint, this was much awaited good news for BMS as the Opdivo did not make the cut as first line monotherapy study in non small cell lung cancer (NSCLC) in 2016. However, Merck’s Keytruda gained FDA approval soon after Opdivo failed in NSCLC study (Fierce Pharma).

 

The battle of patents: bad news for Teva Pharmaceuticals

Genric drug maker giant: Teva Pharmaceuticals lost the patent challenge in U.S. District Court, safeguarding their star drug Copaxone against generic competition. Copaxone, approved in 1996, became the most prescribed drug for the treatment of multiple sclerosis. The patents protecting Copaxone against generic competition expired two years ago for 20mg dose. Novartis and Momenta launched their 20 mg alternative (Glatopa) in 2015. To recuperate, Teva launched a 40 mg formulation of Copaxone. However, this week, U.S. District Court invalidated Teva’s last and fourth key patent protecting 40 mg Copaxone from generic drug competition. Teva lost other 3 patents last year (Madison.com).

Trump pledges to bring drug costs down

Pharmaceutical industries were told by Trump that the drugs should be manufactured in the USA and the foreign countries buying US manufactured drugs should pay “fair share”. These changes in addition to “better innovation” will help bringing prices down for the US patients (CNBC).

MedNess from MedPol: Amgen CEO Robert Bradway announced that soon nearly 1600 jobs will be added. Bank of America Merrill Lynch predicted that Trump’s policies could help Amgen recover their stocks by 23% in the next 12 months (CNBC)

MedPol

US President’s executive order on immigration: the aftermaths

This is not a political blog, but the executive order has a very significant impact on the scientific, medical and healthcare community. In the following paragraphs, I will brief you with the sectors that have been affected.

  • NRMP issues the statement for the upcoming Match

Nearly 260 people from seven nations affected by travel ban, applied through National Resident Matching Program (NRMP) for medical residency in the USA (Association of American Medical Colleges, AAMC). Both the applicants and the hospital programs are concerned and affected by the travel ban. However, NRMP has urged the programs and the applicants to be discrete in their decisions that are in the interest of healthcare. The official statement issued by NRMP on their website states, “The medical education community must support all international medical graduates and their families during these difficult times. As for the current Match cycle, NRMP encourages applicants and programs to make the best decisions they can under current circumstances. For its part, NRMP will be liberal in granting waivers to applicants and programs if they cannot meet their respective Match obligations because of the effects of the Executive Order” (NRMP.org).

  • Dark times for the US hospitals and patients from seven nations affected by travel ban

Ill patients scheduled for treatment at the USA’s premier healthcare centers, John Hopkins Medicine and Cleveland Clinic are uncertain of their treatment options. Hopkins is taking a step ahead by either urging the patients to postpone their travel or sending their staff abroad for their treatment (STAT News)

We wrap up our biweekly MedNess and MedPol news section. Have a great weekend!

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