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Envisagenics: Splicing with a twist!

in Entrepreneurship/Face à Face/Medness by

Innovation is a necessity for us scientists. We are encouraged to discover, invent, identify a plethora of things. Whether we hunt antibacterial compounds or work on technologies that will revolutionize gene synthesis our product is eventually innovation. We thrive on it. It is what unites us, and makes each one of us unique at the same time. Some of us take our innovation to the next level and transform it into medicine, platform, or service. Here is a two-part series discussing one scientist’s maiden venture and his journey from being a trained biologist to a bioinformatician to an entrepreneur.

In conversation with Dr. Martin Akerman

Would you like to briefly introduce yourself to our readers?

I am the co-founder and CTO of Envisagenics, originally from Argentina. During my Masters, I studied a parasitic protozoan, Leishmania, after which I got my doctorate in Bioinformatics at Technion, Israel. Then I moved to Cold Spring Harbor Laboratory (CSHL) to do my postdoc on splicing under the mentorship of Dr. Adrian R. Krainer and Dr. Michael Q. Zhang.

Tell us about your journey from being a graduate student in Israel to being a postdoc at CSHL.

As a Masters student, I was studying Kala Azar, a type of Leishmaniasis. Something happened, and I moved from being a bench scientist to computational biology. This was 2002, and there wasn’t a lot of Bioinformatics around. Hence, catching up was doable. There weren’t 100 different programming languages like today. It was mostly PERL, C++. I liked this work so much that I chose to do my Ph.D. in Bioinformatics.

When I chose my Ph.D. topic, I was very fascinated with the human genome because it was brand new. During my undergraduate days, I was taught that the human genome had 150,000 genes, and this justified the complexity of humans. However, it turned out that there are ‘only’ 20,000 genes. This revelation shocked the whole field and raised new questions, tickling my fascination like many others. I soon wanted to investigate how is it possible to have such a small number of genes and still be a complex and functional organism. Splicing seemed to be a probable answer. That’s when I made a software to study splicing regulation.

If you don’t know what splicing is, here is video for you

 

[Sorry to interrupt, but did you know programming at the time?]

No, I did not know programming; I’m self-taught. It took me a little over 2 years to learn. I mean, you never stop learning! It is impossible to score with a moving post you know. Programming is a huge part of learning bioinformatics. So I did it, and I loved it.

That is very impressive. Please continue….

So, making the SF map (Splicing Factor Motif Analysis and Prediction, published in Akerman M., 2009) software only made me more interested in splicing. I came to CSHL to continue working on splicing with two eminent scientists who were leaders in this field, Dr. Adrian R. Krainer, and Dr. Michael Q. Zhang. While the Kranier lab was primarily a biochemistry lab, the Zhang lab focused on splicing using genomic data.

What inspired you to start Envisagenics from being a postdoc?

There was something special about the Kranier lab! I was the primary bioinformatician in a lab of 20 experimental biologists. What does it imply?  Well, anything I predicted was validated experimentally. While this created a lot of pressure, it was an incredible opportunity to collaborate  and stay connected to biology.

People sought my expertise to analyze their data in various ways. I analyzed RNA-seq data, protein-protein interactions, and RNA-protein interactions in the spliceosome. I soon became addicted to the feeling of having your predictions validated in the lab. At some point, I realized that solving other’s problems was more exciting than answering questions that I came up with. I also realized that the only way to address this addiction of mine was to make a software that works and solves ‘real’ problems. The answer was in starting a company.

Did you ever want a career in academia?

Of course, I did! As a postdoc, I wanted to become a professor until I realized that I don’t. In academia I would have to move from developing one algorithm at a time, which did not allow time to focus on creating a robust piece of software available for a larger audience.

What was the turning point for your transition?

When I wanted to start a company,  I had no idea what it meant. I met some investors at the “Elevator Pitch Day” organized by the Bioenterprise Club at Cold Spring Harbor. The Bioenterprise club focuses on careers outside academia. The investors I met that day were part of the panel that listened to my first ever elevator pitch. I met my co-founder, Maria Luisa Pineda  on the same day. She was in the room working with the investors, and her job was to evaluate me. Long story short, as a result of that meeting, we started the company. She is also a trained biologist from CSHL who after graduating acquired investment experience in technology and life-sciences startup companies.

What does a typical day look like for you at work?

Well, every day is different. I coordinate with the engineering team about the technical work. We work on pilot projects and milestones with scientists in pharma company. Right now we are also spending time on how to use our latest funds; this includes planning with the Business Development team. Some grant writing is pending regarding a phase II SBIR grant. I also do coding- this is my quality time.

In short, I am involved in almost everything, which is great as I enjoy working a lot with my team.

What are the skills that help you run Envisagenics?

My knack for collaboration that I developed from my postdoc days at CSHL helps me a lot. Understanding other’s problems and using our platform in solving them is beneficial to us. Both of these support our partnerships at Pharmaceuticals.

The ability to be a team player is a considerable skill. As a leader one should also know how to distribute and delegate work, be humble, and have the ability to take and give advice from/ to your team.

Being open to taking advice from advisors and investors is a must. You are toned to listen and learn. The most essential skill is, however, to resist, be positive, and never give up.

Speaking of being a leader, do you micromanage?

No, I don’t like that. If you have to micromanage, it is not a good sign. I want people to bloom and use their creativity to see what they can do. At Envisagenics, there’s a time when we meet, and everybody brings ideas. Next, we execute the plan. You cannot deviate from the plan, because we are all connected. Everyone has ONE goal, not separate projects. So it is crucial that we plan together and stick to it. That’s what makes us a great team. However, as a leader, YOU have to make informed decisions.

Coincidence brought Martin Akerman and Maria Luisa Pineda together, and as they say, the rest is history! Keep watching this space for part 2 in this series that will discuss the challenges faced by Dr. Akerman in his transition and his visions for Envisagenics.

 

Author: Dolonchapa Chakraborty

 

Dolon is a Molecular Biologist and currently wears many hats. She freelances as a Consultant for a Toronto-based start-up, helping them with brand management, marketing, and product development. She is also an Adjunct Faculty at Mercy College in the Biology department. She blogs about various topics pertaining to Biotech and PhD in Biotech.

 

 

 

Editors: Rituparna Chakrabarti and Sayantan Chakraborty

 

 

 

 

 

 

Blog design: Rituparna Chakrabarti

Cover image: Kindly provided by ENVISAGENICS, used with permision from Dr. Martin Akerman


The contents of Club SciWri are the copyright of PhD Career Support Group for STEM PhDs (A US Non-Profit 501(c)3, PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers and entrepreneurs).

This work by Club SciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License


Organs in a dish: Are you ready for it?

in Medness by

Editor’s Note: What is common between the legendary movies Star Wars and Blade Runner? The most probable answer would be Mr. Harrison Ford. However, being years ahead of their time, both these movies introduced the transforming concept of being artificial in their unique ways. The R2D2 and C3PO versions of artificial intelligence from Star Wars are a reality now with our Mars Rovers and Amazon Echoes. But what about the concept of artificial organs, courtesy Blade Runner. More than 35 years later, we know that shopping for genetically identical, replacement body parts remains the stuff of science fiction. The last decade, however, has witnessed the first steps in the creation of miniaturised version of these body parts. In this Medness blog from #ClubSciWri, Heena Khatter assembles the stories of science, entrepreneurship and the market of artificial organogenesis stemming from 3D Cultures, that will redefine the precision of future medicine.Abhinav Dey

What is 3D culture?

via GIPHY

3D cell cultures create a physiologically relevant artificial environment for growth of cells. They have gained popularity in the past few years, as they mimic the in-vivo conditions better than 2D cultures in petri dishes- which have been used for decades for growing cells. The culture methods are broadly divided into scaffold and scaffold-free.

  • In-vivo cells are surrounded by an extra-cellular matrix and a milieu of nutrients. The scaffold serves as a support and provides the microenvironment for cellular growth. Commonly used scaffolds range from polymers, hydrogels derived from natural sources (collagen, laminin, and gelatin) to micropatterned microplates.
  • Scaffold free methods allow self-assembly of cells into 3D spheroids such as hanging drop plates, rotating bioreactors and magnetic levitation.

Shyamtanu and Orpita discuss the implications of 3D cultures in their SciWri Podcast.

 

Source

 

What’s the history?

 

 

 

Who are the key players in commercializing 3D cultures?

Apart from the big names such as Thermo Fisher Scientific, Merck, VWR International, Lonza group, Corning, 3D Biotek, Kuraray, ReproCELL, BD;  a huge number of start-ups are investing in developing 3D culture diagnostics including InSphero, Kiyatec, N3d Bioscience. Europe has had a head start in this market due to greater awareness amongst people about its benefits, extensive research and growing investments.

Here, I highlight a few start-ups and their strengths in this field.

InSphero: Spin-off from ETH, Zurich; they deal in microtissues and scaffold-free methods. They are aiming for applications in pharmascreening and diagnostics, and to this end, they have collaborated with PerkinElmer on assay development for drug toxicity.

N3d Bioscience: What sets them apart is the levitation method of the 3D culture. By attaching nanoparticles to the cell membrane, cells are magnetized and the spheroids are formed by applying magnets. The company based in Texas, was awarded SBIR grant from the National Science Foundation and their products are now available in the market. They will soon be moving into application of 3D cell culture to Regenerative Medicine.

 

300 Microns: A start-up venture of scientists from Karlsruhe institute of Technology, Germany. 300uM, happens to be the natural distance between two capillaries in an animal tissue and ‘300 Microns’ produce special polymers housing microcavities in the 300uM range. These polymers can be tailor-made to the consumer’s needs varying in geometries, diameter and shape of microcavities and permeability of the polymer.

AIM Biotech: Based in Singapore, they specialize in microfluidic chips for 3D cell culture. These chips can be utilized for co-culture of different cell types. In 2016, within 4 years of establishment, the MIT spin-off company partnered with distributors from USA, Japan, Europe and China expanding their customer-base.

 

Kiyatec: Focusing on cancer therapeutics, they create customised in-vivo like models for drug response profiling, with the aim to evaluate drug toxicity before proceeding with human clinical trials. Founded by Clemson university alumni, they were recently awarded two SBIR grants: one for progressing their work on 3D breast cancer model by the National Cancer Institute and another one for developing a microbioreactor mimicking live bone marrow.

Creative Bioarray: Initially specialising in array products, now they provide a wide range of products for research comprising of various cell types, ready-to-use 3D cell cultures and protocols for 3D model development. Based in New York, they are one of the leading producers of cell lines used in research.

Organogenix: Formerly Scivax, Japan, they develop dishes for spheroid formations. Owned by the JSR corporation, they specialize in scaffold-based 3D cell culture and provide consultation as well as contract services for establishing these methods.

 

Pandorum Technologies: Based out of Bangalore (India), Pandorum Technologies creates 3D-printed human tissues for medical research and therapeutics. Pandorum was the first in India to design and 3D-print human liver tissues for medical research. The startup is currently working on bio-engineering implantable human cornea. Its 3D-printed human tissues are applicable in medical research for drug metabolism and disease modelling. Pandorum’s bigger vision is to make personalized on-demand human organs such as lungs, liver, kidney and pancreas.

 

Applications and Major Consumers

Drug Discovery: Traditionally, tests for a new drug go from 2D cell culture to animal models, before starting with clinical trials on humans, which in itself is a long, laborious process, sometimes spanning a decade. However, 2D cultures have a major draw-back: being mono-layers, the results of drug toxicity in these conditions can be misleading. 3D cultures are much more representative of the in-vivo environment. These cells grown in 3D media and organoids will soon be able to replace the use of animal models, providing clinically relevant tests for drugs that pass to human trials.

Regenerative medicine: The shortage of organs for transplantations has led to adoption of strategies for growing tissues and organoids in petri dishes. There have been success stories for artificial implants, but it’s a long way to go before this becomes a norm. Unsurprisingly, biotechnology and pharmaceutical Industry is the leading end user for these diagnostics. The need for advanced drugs, with minimal animal testing and search for better diagnostics is pushing pharmaceutical companies to adopt 3D cultures.

Basic Research: 3D cultures are becoming popular amongst basic research scientists; since 3D cultures maintain cell morphology and give a more realistic response to candidate small molecules. Especially, the field of cancer biology is moving rapidly and the assays are now being designed to ensure cells grow in a close-to-natural environment.

Challenges and Outlook

These cultures require huge financial investments which is a major concern for wide application in basic research, restricting its usage to certain institutes. Other caveats such as scale and practicality need to be overcome in the near future. With a wide range of applications, 3D cultures are here to stay! The value for global 3D culture market, as of 2016 is US$ 456.8Mn and is projected to reach US$ 2,734.3 Mn by 2025.

 

Watch out for

Microfluidics (Organs on chip) and Organoids

In a Nutshell

Applications

Drug Discovery

Organ Transplant

Cancer Research

Stem Cell

Tissue Regeneration

 

Leading Markets

In North America- USA and in Europe- Germany.

Fastest Growing market- Asia Pacific.

As of 2016, value for global 3D cell culture market, is US$ 456.8Mn and is projected to reach US$ 2,734.3 Mn by 2025.

 

 

And finally, for a career inspiration hear this TED talk by the Cellular Therapeutics CEO, Robert Hariri, talks about his research in using placenta-derived stem cells as novel therapy for Crohn’s disease, and to create new “organoids” for research and transplants.

In his career as neurosurgeon and trauma specialist at Cornell University, biotechnology executive, military surgeon and aviator, Dr. Hariri is most recognized for his discovery of pluripotent stem cells from placenta and as a member of the team which discovered TNF (tumor necrosis factor). Dr. Hariri was awarded the Thomas Alva Edison Award in 2007 for his discovery of placental stem cells and again in 2011 for engineering tissues and organs from stem cells.- From TedMed

References and further reading:

  1. https://www.gesundheitsindustrie-bw.de/en/article/news/300microns-3d-cell-culture-solutions-tailored-to-customer-needs/
  2. http://www.elveflow.com/organs-on-chip/3d-cell-culture-methods-and-applications-a-short-review/
  3. https://www.gesundheitsindustrie-bw.de/en/article/news/eric-gottwald-innovative-3d-cell-culture-systems-for-pharmaceutical-testing/
  4. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4517320/
  5. https://www.nature.com/nature/journal/v496/n7444/full/496253a.html
  6. http://www.sigmaaldrich.com/technical-documents/articles/biology/3d-biomatrix-white-paper-3d-cell-culture-101.html
  7. http://www.wtva.com/story/35673624/global-3d-cell-culture-market-expanding-at-a-cagr-of-200-from-2017-to-2025-credence-research

About the Author:

Heena is interested in scientific writing, communication and outreach. She did her PhD in Strasbourg and is currently working as a postdoctoral fellow at EMBL, Heidelberg; with experience in the field of molecular, cellular and structural biology. When she is not in the lab, she can be found promoting open access to other researchers or discussing scientific research with students, and travelling to offbeat destinations

About the Podcasters:

Orpita Dey has pursued her M.Sc in Microbiology from Bangalore University. She is currently working as a consultant copy editor for Newgen Knowledge Works. She edits STEM publications and also books on Humanities. She has worked in the past with organizations like Thomson Reuters, ANSR Source, and Education First. She is a science enthusiast and loves to learn new languages. Presently, she is learning German from Dallas Goethe Institute.

Shyamtanu Datta pursued his Ph.D. in Biomedicine (Molecular and Cellular Ophthalmology) from Universität Klinikum Regensburg (Regensburg, Germany). He is currently working as a postdoc at department of Ophthalmology in UT Southwestern Medical Center (Dallas, Texas). He is passionate in learning about business of science which translates from bench to bedside. With over 8 years of experience in research and education, he also wants to contribute to educational innovation for the next generation by making science education more personalized, interesting and fun for the next generation. He loves to travel and learn new technologies.

 

Cover Image: Vinita Bharat

Infographics and Blog design: Abhinav Dey

Creative Commons License
This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

Global oncology – Equitable global health

in Medness/SciBiz by
  • 19749360_1493159330748408_1042093450_o-1.jpg?fit=1280%2C1047
    Diagonal Solution to the Problem by Michail Serebrjakov, a photograph by Supriya Pratihar

Cancer care – not only for the rich

Keeping the key numbers in mind

On addressing cancer control, the WHO’s website says: “The key mission of WHO’s work in cancer control is to promote national cancer control policies, plans and programs that are harmonized with strategies for noncommunicable diseases and other related health concerns. Our core functions are to set norms and standards for cancer control including the development of evidence-based prevention, early diagnosis, screening, treatment and palliative care programs as well as to promote monitoring and evaluation through registries and research that are tailored to the local disease burden and available resources.”

A quick look at cancer control and therapies

Cancer – a disease that can arguably be called as one of the most complex enigmas in medical science – has been a challenge in many different ways. Not only has it been extremely difficult to elucidate the molecular causes of the disease to develop cost-effective treatments but also training and development of medical professionals in the field, provision of timely and appropriate therapeutic interventions and public education to ensure possible prevention and control of the disease.

This matter warrants an immediate reality check in the lower and middle-income countries (LMICs) where a huge disparity exists compared to high income countries. Nearly 80% of cancer cases occur in LMICs where the infrastructural support is dismal in most regions of these countries. Without public health insurance system, the enormous cost of present day cancer care is beyond the reach for majority of population, especially in the LMICs. As population in these countries is on the rise, without a proper tab on the cancer care in these countries, we risk a huge part of human population suffering without access to medical health care – that will directly affect economic and social development in these regions.

Diagonal approaches to cancer care – Educating masses on the prevention and providing therapeutic interventions go hand in hand

Cancer care is now a global issue that invokes organizations like WHO to enable local governments to deal with cancer care effectively. But in line with the WHO objectives, local and government structures in India, North east Africa, Mexico and Middle eastern countries have developed diagonal approaches to strengthen the health system by improving human resource development, drug supply, service provision, quality assurance, financing – of which a few are cited below (5).

Most of the cancer incidences in LMICs are due to cancers that are highly preventable but with limited awareness amongst masses. Various liberating structures and community events have now been in use in combination with didactic education to impart awareness on cancer prevention. While working with people directly, it is imperative to be aware of their lifestyles and cultures to be able to impact the most – a learning useful for the local health workers.

– Better communication between specialist oncologists and primary care providers – doctors and nurses, in local Indian hospitals via WhatsApp based interfaces to consult on complex chemotherapy protocols and management of side effects has enabled easier access to better treatments at local district hospitals.

– While financing cancer care remains a huge challenge worldwide, especially in LMICs, Mexico made a remarkable health reform by introduction of a publicly funded health insurance scheme that covers an increasing list of cancers and encourages preventive measures like timely mammograms.

From educating masses and medical professionals to better health insurance policies – these local experiments serve useful lessons to implement on a larger scale to address disease remediation by combining treatment with preventive measures and better care. While we still await more effective and affordable cancer treatment, there are millions out there who can benefit from the existing pool of knowledge and infrastructure. Let’s at least make sure that we use it equitably without restricting cancer treatment to those born in the first world countries.

References:
1. Cancer Care and Control as a Human Right: Recognizing Global Oncology as an Academic Field, Alexandru E. Eniu, MD, PhD, Yehoda M. Martei, MD, Edward L. Trimble, MD, MPH, and Lawrence N.Shulman, MD, ASCO Eductional Book 2017
2. World Bank definition of 2016
3. Global Health Initiatives of the International Oncology Community, Sana Al-Sukhun, MD, MSc, Gilberto de Lima Lopes Jr., MD, MBA, FAMS, Mary Gospodarowicz, MD, FRCPC, FRCR(Hon), Ophira Ginsburg, MD, MSc, FRCPC, and Peter Paul Yu, MD, FACP, FASCO, ASCO Educational Book 2017
4. Need for Radiotherapy in Low and Middle Income Countries – The Silent Crisis Continues, E.H.Zubizarreta, E.Fidarova, B.Healy, E.Rosenblatt, Clinical Oncology, 2015
5. Thinking Differently in Global Health in Oncology Using a Diagonal Approach: Harnessing Similarities, Improving
Education, and Empowering an Alternative Oncology Workforce, Natalia M. Rodriguez, PhD, Jeannine M. Brant, PhD, APRN, AOCN, FAAN, Dinesh Pendharkar, MD, PhD, MBA, Hector Arreola-Ornelas, MSc, Afsan Bhadelia, MS, Gilberto de Lima Lopes Jr., MD, MBA, FAMS, and Felicia M. Knaul, PhD, ASCO Educational Book 2017

Featured images are by Fuzzy Synapse (on Facebook, Twitter and Instagram) and Supriya Pratihar.

About the author:

Somdatta Karak works with Club SciWri as a project coordinator 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.

About the editor:

Imit Kaur, Ph.D. is a freelance scientific advisor, medical writer, editor, and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from the University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development. Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)

 

 

 

 

 

© The contents of Medness are the copyright of the PhD Career Support Group for STEM PhDs (A US Non-Profit 501(c)3, PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers and entrepreneurs)

Creative Commons License

This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

MedNess – The highs in cancer and sickle cell anemia treatments

in Medness by

MedNess is back with big news from the pharma companies. CAR-T therapy is getting closer to the market and sickle cell anemia sees a new yet ‘not-so-new’ therapeutic development. Stock prices of Indian pharma giant Biocon  soar to a record high with a recommendation for a biosimilar that can be used for treating breast cancer. Do not miss out on the details of what these companies have in mind, and how are their competitors faring.

via GIPHY (http://mistyscience.com/post/120326066555/cytotoxic-killer-t-cells-killing-a-cancer-cell)

 

MedNess North America

Novartis poised to win CAR-T race

In a historic move on July 12, the US Food and Drug Administration (FDA)’s Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of Novartis’ CTL019 (tisagenlecleucel-T), a groundbreaking CAR-T therapy for treating relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) in children and young adults. This decision is expected to lead to approval of the therapy by US FDA on or before October 3.

Due to the complexity of the therapy and expert care needed to manage side-effects, Novartis upon approval will restrict CTL019 use to 30-35 treatment centers in the U.S. who will be trained on a comprehensive risk-mitigation strategy that drew high praise from several ODAC members during the hearing. Novartis has plans for additional filings for CTL019 later this year, including applications with the FDA and European Medicines Agency (EMA) for the treatment of adults with r/r diffuse large B-cell lymphoma (DLBCL).

Close rival Kite Pharma’s CAR-T application for the treatment of adults with advanced and aggressive lymphoma will receive a decision from FDA in November. Other key players being keenly watched in this field are Juno Therapeutics, Bluebird Bio, and Cellectis.

The announcement, however, did not affect Novartis’ or Oxford BioMedica’s shares, the companies supplying raw materials for CTL019. Before this announcement, Oxford BioMedica renewed its contract with Novartis extending its partnership for the commercial and clinical supply of its LentiVector gene delivery technology. On the other hand, Kite Pharma shares have seen big swings and is currently trading 24% higher than Novartis.

 

FDA approves first sickle cell disease treatment in 20 years

via GIPHY

On July 7, the US FDA approved Endari for patients with Sickle Cell Disease (SCD) to reduce severe complications associated with the disorder. Endari, developed by Emmaus Lifesciences Inc. based in Torrance, CA, is the first FDA-approved treatment for pediatric patients with SCD and the first new treatment in almost 20 years for adult patients. The therapy is an orally administered pharmaceutical grade L-glutamine. The therapy’s efficacy and safety were evaluated in a 48-week Phase 3 trial (NCT01179217) involving sickle cell patients between the ages of 5 and 58 who had two or more painful crises in 12 months prior to enrollment. Results from the trial demonstrated that Endari reduced the frequency of sickle cell crises by 25 percent and hospitalizations by 33 percent.

Sickle cell disease is a rare, inherited disorder characterized by abnormally sickle-shaped red blood cells. These malformed red blood cells clog blood vessels and cut off oxygen to the body’s tissues, leading to episodes of severe pain and organ damage. Approximately 100,000 people in the U.S. have sickle cell disease, according to the National Institutes of Health. Endari helps increasing free glutamine in blood, which is taken up by the sickle red blood cells and used to generate anti-oxidant molecules as a product of glutamine degradation. These new antioxidants help neutralize the oxidative stress in sickle cells, allowing them to regain the flexibility needed to travel through blood vessels and capillaries, carrying oxygen to tissues.
L-glutamine, the active ingredient in Endari can be purchased over the counter, which could complicate Emmaus’s ability to obtain insurance coverage. Ontario-based Generex Biotechnology acquired controlling interest in Emmaus LifeSciences in March 2017. The recent FDA approval saw an 8% increase in share value for Generex Biotechnology.

MedNess Asia

Biocon flying high with US FDA recommendation

via GIPHY

(source)

Biocon’s biosimilar of Trastuzumab, a breast cancer drug obtained unanimous recommendation (16-0) from US FDA ODAC – taking the drug closer to procuring US FDA’s marketing approval. Trastuzumab, originally developed by Roche is commonly used to treat HER2-positive breast cancer and has a multi billion-dollar market globally with an estimated market of $6.9 bn in the US alone. Biocon chairperson Kiran Mazumdar-Shaw said that they expect to obtain approval to launch their product by September 3.

Biocon collaborated with US drug maker Mylan to develop this biosimilar. Biocon will manufacture the product for all markets globally with a profit sharing arrangement with Mylan for its markets. However, with numerous observations from US FDA and European regulators, Biocon faces concerns regarding quality lapses. To circumvent these Ms. Mazumdar-Shaw mentioned that the company has GMP certification for its drug substance facility for trastuzumab and will expedite the process to seek an early re-inspection.

It is important that the approvals work swiftly for Biocon due to the risk of losing market share to other generics manufacturing competitors . One company to watch out for will be Aurobindo Pharma – the newest entrant in oncology segment in India. It plans to file for 15-17 products in oncology this year and expects to be able to start selling by end of the year. Last month, Dr. Reddy’s Laboratories and its partner Natco Pharma obtained US FDA approval for the generic version of Doxil, a chemotherapy drug used for ovarian cancer, Kaposi’s sarcoma, etc. to launch in the US market.

MedNess

While the US FDA approval recommendation has already caused a surge of 11.8% in the stock prices of Biocon, it will need to concentrate highly on strengthening its technical capabilities and avoid quality lapses. It cannot afford to lose time in getting the approvals in order to avoid losing the first mover’s advantage in the market. Aurobindo Pharma has already seen a surge of 22.8% since it announced plans to file its oncology products.

1) https://www.novartis.com/news/media-releases/novartis-car-t-cell-therapy-ctl019-unanimously-10-0-recommended-approval-fda

2) http://blogs.nature.com/tradesecrets/2017/07/15/first-approval-in-sight-for-novartis-car-t-therapy-after-expert-panel-vote

3) http://www.fiercebiotech.com/biotech/novartis-stellar-car-t-efficacy-data-steamroll-safety-doubts-to-power-landmark-cancer

4) https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm566084.htm

5) http://www.emmausmedical.com/Article.aspx?seo=141&l=EN&g=25

6) http://economictimes.indiatimes.com/markets/stocks/news/us-fda-panel-nod-for-biocon-breast-cancer-biosimilar/articleshow/59605651.cms

7) http://www.business-standard.com/article/companies/aurobindo-pharma-looks-to-sell-cancer-drugs-in-the-us-by-year-end-117061100358_1.html

8) http://www.business-standard.com/article/companies/dr-reddy-s-gets-usfda-nod-to-launch-new-ovarian-cancer-drug-in-us-117051700942_1.html

About the authors:

Radhika Gopal is currently a postdoctoral research associate at The Scripps Research Institute, La Jolla, where she works on Hepatitis C virus glycoproteins. She also chairs the Corporate Sponsorship Committee for the San Diego chapter of AWIS (Association for Women in Science). Radhika has completed project management and business fundamentals courses from UCSD, and is actively seeking opportunities in the Biotech Industry. In her spare time, she enjoys music, hiking, reading music and the beach. She can be reached here.

Somdatta Karak, PhD is interested in pharma and healthcare sector in Asia. She also works with PhD Career Support Group / Club SciWri as its project coordinator. She aims to make a more and better informed world for all, and hence experiments with making effective platforms of education. She can be reached here.

About the editor:

Sayantan Chakraborty is an IRTA postdoctoral visiting fellow at the National Institute on Aging – National Institutes of Health, Baltimore, USA. Apart from science, he invests his time in networking, writing, and organizing events. He is consolidating his efforts to build a platform that brings together scientists and industry professionals and to help spread the perception of alternate careers for life science graduates.

Follow him on Twitter @ch_sayantan

 

© The contents of Medness are the copyright of the PhD Career Support Group for STEM PhDs (A US Non-Profit 501(c)3, PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers and entrepreneurs)

Featured image: Ipsa Jain (you can find more of her works on Facebook and Instagram)

Spread of evil – Cancer cells in blood stream by IpsaWonders

Blog design: Abhinav Dey

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This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

MedNess – Bringing the latest in cancer treatment from the ASCO meet Part I

in Medness/SciBiz by

Following the American Society of Clinical Oncology (ASCO) annual meeting at Chicago from 2nd to 5th June 2017, here is a comprehensive list of studies presented that are funded by leading pharmaceutical companies worldwide. We hope to provide an overview of what is the state of art therapeutic developments in cancer treatment. Know the leaders in oncology and what molecular targets and techniques they are most interested in for the prevalent forms of cancer.

From discussing development of newer drugs for the well-known targets in cancer therapy to CAR-T therapy, here is a collation of all the ground-breaking research from the pharmaceutical companies.

Content Idea: Imit Kaur, Ph.D

Content development and execution: Somdatta Karak, Ph.D., Vinita Bharat, Ph.D.

Edited By: Imit Kaur, PhD.

Illustration: Vinita Bharat, Ph.D

About the Authors:

  Imit Kaur, Ph.D. is a freelance scientific advisor, medical writer, editor, and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from the University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development. Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)

Somdatta Karak, PhD is interested in pharma and healthcare sector in Asia. She also works with PhD Career Support Group / Club SciWri as its project coordinator. She aims to make a more and better informed world for all, and hence experiments with making effective platforms of education. She can be reached here.

Vinita Bharat Ph.D., is currently a postdoctoral research fellow at European Neuroscience Institute, Göttingen, Germany and had been an International Max Planck Research School (IMPRS) student here. Her research area focuses on cellular and molecular neuroscience. Other than enjoying ‘being a scientist’, she has also been working on science education. Presenting science in easy and fun way is what she loves doing through her platform “Fuzzy Synapse” (one can find fuzzy synapse on Facebook, Instagram and Twitter). She is a fun, enthusiastic and curious person, passionate about traveling, loves celebrations and bringing smiles around her.

From Oklahoma to Manhattan- The Genesis of Sevengenes

in Entrepreneurship/Face à Face/Medness by

Where there is a will there is a way. This is the mantra followed by scientist-turned-entrepreneur Ayyappan Subbiah. Ayyappan started his journey in the field of Material Science, obtained PhD in 1997 from IISc, Bangalore, India under the supervision of eminent Professor C.N.R. Rao. Introvert by nature, Ayyappan had a desire to bring about some kind of impact in people’s lives. This passion was rekindled during his job at ConocoPhillips where he was working as a Senior Scientist. Ayyappan did not want to entangle himself in the rut of routine work and confine himself to the realms of bench experiments. This prompted Ayyappan to break the traditional norms and set forward his journey of entrepreneurship. With the help of two friends, Ayyappan founded LivePet LLC. The first product from LivePet was an anti-inflammatory supplement for the pets. In order to take the product from the bench to over-the-counter veterinary product, Ayyappan and his mates tested their product at Liberty Research Institute, NY and carried out a couple of small trials on 50 dogs. The dogs were administered a set dose of the supplement for 30 days and were tested successfully for the safety and the change inflammatory markers. All the tested dogs were safer and healthier at the end of the study.

After developing the novel anti-inflammatory supplement for the dogs, Ayyappan started questioning himself to go further beyond pet supplements. By now he also realized that as a responsible human being, the best job he can do is to help “towards making 7 generations of human beings living together healthily/happily!” But the bigger question was what will be the source of funding for his next project? There comes a point in life of every scientist when they look for their next career move. “I just knew that after LivePet, I couldn’t go back to the monotonous life of being a lab scientist. I wanted to make an impact in people’s lives and this was the right time” reminisces Ayyappan. Ultimately, he thought of self-funding his own project. He did not know how far his self-funding will take him, but decided to begin the journey. With this dilemma being resolved, the next question was the kind of project he should start.

My first instinct was to learn about the bottle-necks in pharmaceutical industry and look for the potential problems. I wanted to find solution to the biggest challenge of the pharmaceutical industry

says Ayyappan proudly. His research yielded him the answer for his quest. He decided to work on the solubility issues of the hydrophobic drugs. He says “About 40% of drug candidates filed with FDA and 90% in the discovery pipeline are hydrophobic and possess solubility/bio-better issues. Therefore there is an immediate need for a safe and better solvent (excipient) in the pharma industry.” He wanted to test one of his novel excipient/solvent technology idea from his materials science background with a hydrophobic molecule preferably a novel molecule for the first time in the pharma industry.  Literature search yielded an ideal hydrophobic molecule called triptolide which is notoriously known to possess solubility (and toxicity) issues but never became a drug just for those reasons.

Ayyappan took it as a challenge to solubilize triptolide in the novel excipient (7GEN) or the solubilizer. That’s the birth of Ayyappan’s successful start-up called Sevengenes (www.sevengenes.com ). Since then, there has been no looking back. 7GEN significantly enhances the solubility and bioavailability of hydrophobic molecules.  7GEN is very effective compared to existing drug delivery strategies (such as lipid and nanomaterials based) used for approved drugs.

So how does he fund his start-up projects? Ayyappan is deeply passionate about his project and he used up his 401 savings to fund his project. When asked about grant funding, Ayyappan did not want to divulge the details of his idea to the federal agency before gaining patent approval. He pitched in the idea to family and friends and they acted as amazing sources of funding. His MSc classmate wanted to help him and became a co-founder. Similarly, couple of his PhD colleagues from IISc also wanted to jump in and help and they have become co-founders too.  In addition, Ayyappan’s project was also screened by a non-profit organization, i2e (www.i2e.org) that provides funds to small scale biotechs in Oklahoma. With limited funding but tons of passion and zeal, Ayyappan and Sevengenes’ cofounders outsource their experiments to CRO’s and University of Oklahoma.

They are a pre-clinical company and would like to file their Investigational New Drug (IND) application on their first drug product 7GEN-TDTM when they get their first round investment.  They would like to use their 7GEN excipient (as a platform) for many other hydrophobic drugs and take 505(b)(2) approach for a particular combination.  Meanwhile, Sevengenes recently got selected by Alexandria LaunchLabs for an incubator space in Manhattan, New York from June-2017. Their might be restricted funds but there isn’t dearth of passion inside Sevengenes.

Infoshell…..

When asked about advice he will give to aspiring entrepreneurs, Ayyappan suggests that one should chase their dreams regardless of worrying about the outcomes. He believes that once someone sets his or her brain and heart to a project, the brain will work at its best to find resources to complete the project. Ayyappan has his background in Material Science and is yet a founder of Biological Science start up. He believes in working hard and fair. He credits his spouse and family for their support. He believes that it is a two way street. He saved up enough 401(k) so as to provide secured life to his family while his family understood and supported his dream of a start-up and his project. When asked about his mantra for relieving stress, Ayyappan says he enjoys yoga and believes in the power of prayers!

Ayyappan with Ananda Ghosh (Founder of PhD Career Support Group for STEM PhDs).

About the Author:

Imit Kaur, Ph.D. is a freelance scientific advisor, medical writer, editor, and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from the University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development. Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)

 

Featured image: Ayyappan in the auspices of Alexandria Launchlabs

Blog design: Abhinav Dey

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This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

 

MedNess: FDA Approvals, Label Expansions and International Market

in Medness by

Hello and welcome to MedNess. This edition of MedNess covers the latest from small and big biotech and pharma companies. Stay tuned for MedNess Asco Coverage. In the meantime, please subscribe to ClubSciWri for our other articles and blog posts!

Celgene supports Dragonfly in enhancing NK-cell based immunotherapy

The discovery-stage company, Dragonfly Therapeutics, aims to enhance immunotherapy using Natural Killer (NK)  cells. Dragonfly aims to harness the power of body’s innate immune system to vastly improved patient outcomes. They plan to stimulate NK cells so that these cells can attack tumors directly with support of T and B cells. This strategy has attracted the attention of a leading global pharmaceutical company, Celgene along with the Duke of Bedford, Disney family members and other organizations.

Celgene bagged the option on four NK cell based cancer therapeutics to treat myeloid leukemia, multiple myeloma and other hematological cancers by investing over $33 million in Dragonfly. This collaboration by Celgene shows that they saw immense potential in this discovery stage biotech to develop innovative therapies for cancer patients (Dragonfly Therapeutics, Fierce Biotech).

MedNess: Celgene recently reported positive data from Lupus trial. Its shares have been soaring with an overall gain of 19.8% in the last one year. As per Zack’s index, Celgene stocks are a strong hold. At the present, the shares are sold for $124.82 (Zacks).

Europe, US, and Japan joins hands to boost antibiotic development

The three drug regulators of the world; European Medicines Agency (EMA), the Japanese Pharmaceuticals and Medical Devices Agency (PMDA,) and the Food and Drug Administration (FDA) participated in the tripartite meeting in Vienna, Austria  to consider a robust response to boost antibiotic development.

They agreed to align their data requirements for certain aspects of the clinical development of new antibiotics so that the new meds can come in the global market. Also, in the meeting, they discussed in detail clinical trial recommendations for certain types of bacterial infections, including infections caused by multi-drug resistant organisms. Even the areas of differences were talked about in this meeting, and an effort to work together to minimize them was discussed.

While all three of them are now working on updating their respective guidance documents, they are also willing to provide suggestions to the individual biopharma companies. The next meeting is scheduled for October 2017 ( Fierce biotech, EMA).

Astrazeneca terminates the plan for NASH drugs with SoCal’s Regulus

California based biopharmaceutical company, Regulus aims to discover and develop innovative medicines targeting microRNAs. They recently announced their pipeline updates and advancement in which a major setback was when Astrazenenca terminated the clinical development program for AZD4076 (RG-125) which is involved in the treatment of nonalcoholic steatohepatitis (NASH) in Type 2 Diabetes/Pre-diabetes.

Regulus also planned to discontinue clinical development of RG-101 for Hepatitis C Virus (HCV) upon completion of the one remaining clinical study, which is expected to occur in July 2017. Now, the company is majorly focussing on keeping the plans of the Phase II clinical programs for RG-012 which is used for the treatment of Alport syndrome on track. Also, the IND for RGLS4326 for autosomal dominant polycystic kidney disease (ADPKD) is on track for filing by year end 2017 (Regulus, Fiercebiotech).

AstraZeneca’s Lynparza slows breast cancer progression, now a potential precision drug against prostate cancer

AstraZeneca announced statistically significant positive results from Phase 3 OlympiAD tested against breast cancer patients with BRCA gene mutations. The results demonstrated a clinically-meaningful improvement in progression-free survival (PFS) for patients treated with Lynparza (olaparib) tablets (300mg twice daily), compared to chemotherapy. In addition, a 42% reduction in disease worsening or death (HR 0.58; 95% CI 0.43-0.80; p=0.0009; median 7.0 vs 4.2 months) was observed in patients treated with Lynparza when compared to those who received chemotherapy. The results were reported in New England Journal of Medicine. Lynparza was earlier approved for ovarian cancer that is caused by BRCA. Now, in another study being carried out at the Institute of Cancer Research, London, Lynparza is showing a potential towards precision drug in prostate cancer. According to the researchers, the test designed by them, could distinguish the disease severity, treatment response and if prostate cancer is evolving genetically and can potentially become drug resistant. The results of these tests were reported in Cancer Discovery (AstraZeneca, Reuters)

MedNess: AstraZeneca has a strong pipeline of drug candidates.  The stock has a market capitalization of $87 billion. Lynparza generated $218 million in sales in 2016. The current share price is a little over $34 with overall gain of 0.26% (Forbes, CNNMoney).

Clovis Oncology announces positive results from late stage ovarian cancer trial

Clovis Pharma’s, Rubraca, met its primary endpoint and a key secondary endpoint. The company is planning to request label expansion for Rubraca and gain approval for second line treatment and maintenance treatment for women with platinum-sensitive ovarian cancer who have responded to their most recent platinum therapy. Clovis Pharma’s drug is directly in competition with Tesar Inc’s Zejula and AstraZeneca Plc’s Lynparza. All the drugs belong to the class of PARP inhibitors, that blocks enzymes poly ADP ribose polymerases. These enzymes are involved in repairing damaged DNA (Clovis Oncology).

MedNess: Clovis Pharma’s shares are soaring. It shutdown at $59.97 on Friday, June 15. The stock opened at $87.73 (~ 50% surge in price) on Monday, June 19.

MedNess Asia:

Promising addition in cancer therapy from Chi-Med with a huge investment from U.S. partner Eli Lilly

Last week, Hutchison China MediTech Limited (Chi-Med) has submitted its New Drug Application (NDA) to China Food and Drug Administration (CFDA) for fruquintinib, developed jointly with Eli Lilly to treat advanced cases of colorectal cancer (CRC). In addition, fruquintinib is being tested to treat non-small cell lung cancer and gastric cancer. The drug selectively inhibits vascular endothelial growth factor receptor and helps preventing angiogenesis, i.e. development of new blood vessels essential for tumor growth and metastasis.

Chi-Med aims to break the Active Pharmaceutical Ingredient (API) manufacturer’s stereotype of Chinese pharma companies, and establish itself in bringing innovative and modern drugs in international market. The other drug that the company is evaluating at phase II trial for cancer research is sulfatinib, that also targets tumor angiogenesis and immune evasion. (Reuters, Chi-med press releases)

MedNess: CRC is the second most common cancer with 380,000 cases in China and 1.5 mn cases globally. Reports suggest that the number of new cases of CRC will increase by 13% over the next three years by 2020. Hopes are that fruquintinib can be used in combination with chemotherapy and targeted cancer therapy. Since the application of NDA for the drug on 12th June, Chi-Med has seen a surge of 2.6% in its stock prices (within two days).

Market for internationally manufactured aesthetic medicine in China

Austria-based Croma-Pharma becomes the only second European producers of intradermal fillers approved in China. Intradermal fillers (biodegradable – like different forms of collagen and hyaluronic acid, semi-permanent – like different polymers and permanent – like silicone) help in skin aging management by restoring facial volume or treating wrinkles.

Croma-Pharma (CP) has registered Princess® VOLUME, a hyaluronic acid based filler in China by joining hands with Sihuan Pharmaceutical (SP)  – CP brings in its products, experience and related intellectual property rights, whereas SP will provide its multi-channel distribution and marketing knowledge, being one of the largest pharma corporations and drug franchises in China. This joint venture aims to compete against the local manufacturers in the country and increase the quality of beauty products in China. (Croma news, Bloomberg)

MedNess: China has the largest market for aesthetic medicine in Asia-Pacific region growing at 20% annually, with its aging population, increasing awareness about aesthetic treatment and rise in disposable incomes. By taking advantage of a Chinese company’s distribution channels, Croma-Pharma hopes to reach a considerable market share, by competing against local manufacturers – an issue that has been bothering most foreign manufacturers in China.

Generic anticoagulant launched by Dr. Reddy’s Laboratories for US market

Indian multinational pharma company Dr. Reddy’s Laboratories launched bivalirudin, a therapeutic equivalent generic version of Angiomax® by the Medicines Company. Approved by US-FDA, it is to be used for injection as a blood anticoagulant.

Angiomax, a direct thrombin inhibitor (DTI) structurally similar to hirudin, is used for acute cardiovascular care. DTIs show a higher specificity in reaction with thrombin and lesser complications arising from its use, and hence are preferred over indirect thrombin inhibitors like heparin . Angiomax and its generics registered US sales of around $198 mn over the last year till March 2017. (Dr. Reddy’s Laboratories press release, The Medicines Company)

MedNess:  The anticoagulants market is expected to grow at a CAGR of more than 6% by 2020. Within a week after the launch (on 6th June) by Dr. Reddy’s its stock prices went up by 4.5%.

 

Featured Image: Vinita Bharat

About the Authors:

 Imit Kaur, Ph.D. is a freelance scientific advisor, medical writer, editor, and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from the University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development. Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)

Somdatta Karak, PhD is interested in pharma and healthcare sector in Asia. She also works with PhD Career Support Group / Club SciWri as its project coordinator. She aims to make a more and better informed world for all, and hence experiments with making effective platforms of education. She can be reached here.

Vinita Bharat Ph.D., is currently a postdoctoral research fellow at European Neuroscience Institute, Göttingen, Germany and had been an International Max Planck Research School (IMPRS) student here. Her research area focuses on cellular and molecular neuroscience. Other than enjoying ‘being a scientist’, she has also been working on science education. Presenting science in easy and fun way is what she loves doing through her platform “Fuzzy Synapse” (one can find fuzzy synapse on Facebook, Instagram and Twitter). She is a fun, enthusiastic and curious person, passionate about traveling, loves celebrations and bringing smiles around her.

 

 

MedNess Special: Bringing North America, Europe and Asia together

in Medness by

Hello everyone and welcome to MedNess Global! Here at ClubSciWri, we are constantly trying to present the most impactful news in an ever-evolving innovative fashion. In this special edition, we bring business news from pharma and healthcare sectors in North America, Europe and Asia. Know what defines the industry space in each continent. Where is big capital invested on pharma research? And where does the manufacturing happen? What regulations underlie the drug making and selling processes across continents? This is an effort to give our readers a holistic understanding of international collaborations and deals in the industry. We thank our additional team of writers for their input; MedNess North America: Vinita Bharat; MedNess Europe: Czuee Morey; and MedNess Asia: Somdatta Karak

 

We value your input, please do not forget to provide your feedback (Fill the form at the end of this blog). Please subscribe to ClubSciWri (www.sciwri.club) for weekly updates!

MedNess North America

Merck Ventures supported Inthera Bioscience with their $11M investment

Protein-protein interactions (PPIs) are the basis of multiple control points in the body and play a pivotal role in multiple diseases, including cancer. PPI’s serve as the important target classes in oncology but are extremely intractable to work with.  Inthera bioscience, a Swiss based biopharma aims to design oral small molecules which can inhibit these PPIs, leading to the targeted approach for the treatment of solid tumors.  

For this innovative, novel and promising approach, Inthera drew the seed money of $3.8M in 2005, and now once again Merck Ventures invested $10.8M to wrap up pre-clinical developments focusing on HPV-associated cancers and hypoxia-inducible signaling  (FierceBiotech).

MedNess: Inthera Biosciences is a private biopharmaceutical company focussing on PPI’s. Inthera’s research group focuses on HPV-associated cancers and hypoxic signaling. Merck Ventures is the venture capital wing of Merck. This wing invests in novel products and technologies with significant potential to impact Merck’s core research areas (Biospace)

Novartis fully concentrated on its CAR-T cell therapy for “ALL” cancer

Novartis is specifically betting on its CAR-T cell therapy even though acknowledging that there is a gap in the other major I-O driven therapies like, IDO, BTK and PARP inhibitors. CAR-T is patient specific and looks likely to be the first of this new cancer class to reach the market by this fall.

CAR-T cell therapy involves the drawing of T-cells from patient’s blood and then reprogramming it in laboratory to hunt patient’s cancer cells and other B-cells expressing a particular antigen. Novartis touts this week data of its CAR-T candidate, CTL119 which when used with Janssen/Pharmacyclics’ Imbruvica (ibrutinib) gave complete responses with no evidence of disease in the bone marrow at three months (FierceBiotech).

MedNess: Novartis on June 1 opened with 81.78 and closed by 81.86 (Nasdaq.com). Novartis stock price has increased by 12.3% in the last 12 months. According to Zack’s classified, Novartis ranks number 3 and with its innovative technology, the stocks are advised to be hold onto (Zacks.com; TheStreet.com).

 

Tesaro’s new FDA approval med Zejula is waiting for its response

Tesaro’s PARP inhibitor based drug Zejula has one of 2017’s biggest launches, with an estimate of $1.9 billion till 2022. The company requested offers from the potential bidders but seems like they are waiting to hear from them.

Apart from the high price of this drug, buyers seem to wait for ASCO meeting where they can compare the results of Zejula with AstraZeneca’s Lynparza, Merck’s Keytruda and others. Hence, Zejula clealry opens up a PARP market and its big response is now relying on its ability to rack up new indications in prostate and breast cancer (FiercePharma).

 MedNess Europe

Therapeutic antibody against non-Hodgkin’s lymphoma developed by MorphoSys enters Phase 3 clinical trials

The German biotech MorphoSys announced that its therapeutic antibody, MOR208, has entered Phase 3 clinical trials after successful completion of the phase 2 part of the combined phase 2/3 trials. The randomized, multicenter phase 2/3 study is designed to investigate the efficacy of MOR208 plus bendamustine versus rituximab plus bendamustine in patients with relapsed or refractory diffuse large B cell lymphoma (R/R DLBCL), the most common form of non-Hodgkin’s lymphoma, who are not eligible for high-dose chemotherapy and autologous stem cell transplantation.

MOR208 is an Fc-enhanced monoclonal antibody that targets CD-19 homogeneously expressed on the surface of all B-cells. MOR208 is intended to induce direct apoptosis by binding to CD-19 along with significant potentiation of antibody-dependent cell-mediated cytotoxicity and cellular phagocytosis, thus aiming to improve a key mechanism of tumor cell killing.

MOR208 can therefore be useful to treat various B-cell malignancies such as non-Hodgkin’s lymphoma and chronic lymphocytic leukemia (CLL). Each year about 150,000 people are afflicted by B cell malignancies in the seven major markets. MOR208 was granted orphan drug designation in DLBCL and CLL across US and Europe. Additionally, the FDA has granted fast track designation for the treatment of DLBCL. MOR208 is under investigation in a separate clinical trial for CLL.

The dosing of the first patient in the phase 3 part will trigger an undisclosed milestone payment to Xencor, Inc., from whom MOR208 was in-licensed in 2010. MorphoSys has worldwide rights to MOR208. If this clinical trial is successful, MOR208 will be the first therapy that MorphoSys develops in clinical trials independently. However, MorphoSys has had a lot of success with its Human Combinatorial Antibody Library (HuCAL) that generates human antibodies in bacteria, after having been screened with the aid of specific phages. In partnership with Big pharma companies, MorphoSys has funneled over 100 monoclonal antibodies into the clinic with this platform (Labiotech.eu, Morphosys website).

MedNess Asia

Mismatch between FDA regulations and its implementation in Chinese API manufacturing companies

Cheaper manufacturing processes in China and India attract pharma companies in getting their Active Pharmaceutical Ingredients (APIs) manufactured for cheap in Asia. However, the manufacturing companies have to comply with regulations of the country where the drug is sold and intended to be used. Of the companies facing non-compliance with this are Chinese API makers, Teva Pharmaceutical Industries and Changzhou Jintan Qianyao Raw Material Factory failed US FDA inspection and has consequently been banned to ship all their products to the USA. The company had failed to meet with the Good Manufacturing Practice (GMP) expectations from  the US FDA. (GEN, FiercePharma)

MedNess: China is currently the world’s leading producer of pharma ingredients, covering 40% of global production, primarily because of the price advantage that they offer. It will be interesting to see if the API companies are willing to increase their costs by adding more regulatory expenses or let go of the multinational companies. On the other hand, China FDA (CFDA) has tightened its regulations to stop sub-standard operations.

Pricing pressures and non-compliance with US FDA regulations make difficult times for Sun Pharma

At the end of FY2016, Sun Pharmaceuticals Ltd., India’s largest pharma company, took a hit in its US sales for numerous reasons. Its key API maker for the US market, Halol faces US FDA regulatory limitations since 2014. US FDA slammed Halol with warning letters twice for reasons from inadequate data protection on computer to possibilities of contamination in their products despite Sun seeking help from consultants to make the plant US FDA compliant. In addition, Sun faces pricing pressure like the other generic drug companies. In addition, its US subsidiary specialty generics drugmaker, Taro faced a 26% drop in their sales.

More than 70% of Sun’s sales are from outside India, with US contributing to 50% of its turnover. With multiple worries continuing, Sun’s Managing Director Dilip Sanghvi has warned of single digit declines in the FY2018 revenues. (FiercePharma, Business Standard)

MedNess: Over the last 6 months Sun Pharma has seen a 28% drop in its stock prices (NSE). While resolving US FDA warning letters can take long, generic drugs pricing pressures might bring more mergers and acquisitions in the field.

 

China’s steps beyond generic drug making in healthcare sector

Founded in 2012, in Beijing, privately-held and venture-capital (VC) backed, CANbridge Life Sciences Ltd. has paved its path towards developing leading candidates in solid-tumor oncology, primarily in China, Taiwan and Korea by gathering funding worth $40mn, together after its second round of funding. Currently it focuses on two of its prime compounds – CAN008 and CAN017, and the former is already in phase I/II trial for glioblastoma multiforme, in Taiwan and will begin its phase II in China in 2018. CAN017 is designed against esophageal squamous cell cancer – the most prevalent form of cancer in Asia and has already been successful in phase I study in Greater China. CANbridge partners with AVEO Oncology (for the rights on the compounds) and Böhringer Ingelheim (for manufacturing) in development of CAN017. CANbridge talks of plans of more strategic transactions in 2017 – backed by strong finances and executive team – in direction of developing specialty healthcare products.  (Fierce Biotech, Biospace)

MedNess: China’s huge aging population and increase in lifestyle disorders as well as growth in per capita earning and expenditure is a promising ground to get into specialty healthcare products in China. CANbridge is an interesting example to study commercialization of healthcare products that are under-served in China and North Asia, by partnering with its developers and manufacturers in western countries. Since CANbridge is still VC backed, stocks are not yet for sale.

 

 

Featured Image by: Vinita Bharat

 

 

 

 

 

 

 

About the authors:

 

 

 

 

 

 

Imit Kaur, Ph.D. is a freelance scientific advisor, medical writer, editor, and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from the University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development. Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)

 

 

 

 

 

 

Somdatta Karak, PhD is interested in pharma and healthcare sector in Asia. She also works with PhD Career Support Group / Club SciWri as its project coordinator. She aims to make a more and better informed world for all, and hence experiments with making effective platforms of education. She can be reached here.

Czuee Morey has a PhD from University of Lausanne and pursued a postdoc from University of Geneva. She has broad interests in healthcare and technology as evidenced by her research experience in fields such as protein biophysics and genomics. She has previously worked as a patent analyst and also built the business plan for her startup project. She is currently working in market intelligence for digital healthcare at Debiopharm in Lausanne, Switzerland.

Vinita Bharat Ph.D., is currently a postdoctoral research fellow at European Neuroscience Institute, Göttingen, Germany and had been an International Max Planck Research School (IMPRS) student here. Her research area focuses on cellular and molecular neuroscience. Other than enjoying ‘being a scientist’, she has also been working on science education. Presenting science in easy and fun way is what she loves doing through her platform “Fuzzy Synapse” (one can find fuzzy synapse on Facebook, Instagram and Twitter). She is a fun, enthusiastic and curious person, passionate about traveling, loves celebrations and bringing smiles around her.

© The contents of Medness are the copyright of the PhD Career Support Group for STEM PhDs (A US Non-Profit 501(c)3, PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers and entrepreneurs)

Blog design: Abhinav Dey

Creative Commons License
This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

The Gentleman’s Hesitation….& The Invention of Stethoscope

in Medness/SciWorld by

René Laennec (1781 – 1826) was a thorough gentleman. In retrospect, he’d turn out to be a knight in shining white apron.

In 1816, the young French doctor was worried that he could be getting ”inappropriately close’ to a young patient who had been suffering from chest infection. He would recall later, ”…. I was consulted by a young woman laboring under general symptoms of diseased heart, and in whose case percussion and the application of the hand were of little avail on account of the great degree of fatness.The… method of direct auscultation [was] being rendered inadmissible by the age and sex of the patient…”

Laennec resolved the problem of medical diagnostics and social decency in one shot. He ”rolled a quire of paper into a kind of cylinder and applied one end of it to the region of the heart and the other to my ear ”
Within a few months, he had invented that universal symbol of medical science – the STETHOSCOPE

Reference:

  1. https://www.medisave.co.uk/blog/the-invention-of-the-stethoscope/
  2. https://thechirurgeonsapprentice.com/2015/04/21/laennecs-baton-a-short-history-of-the-stethoscope/
  3. https://commons.wikimedia.org/wiki/File%3ARene-Theophile-Hyacinthe_Laennec_(1781-1826)_with_stethoscope.jpg

 

Author Profile:

for sciwri

Anirban Mitra, Ph.D.

Anirban Mitra did his PhD from the Department of Microbiology and Cell Biology, Indian Institute of Science (IISc), Bengaluru and is now a teacher of biology, based in Kolkata. His interests range from biological evolution to history of science and facets of India’s past.

Blog Design and infographics: Abhinav Dey

Featured Image: Ipsa Jain

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MedNess: Healthcare Business News from the Month of May

in Medness by

Hello everyone and welcome to MedNess: At the frontier of healthcare news. I am back with the news from healthcare business that had the most impact this month Read below to find out more.

 

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Neurotrope’s Alzheimer’s candidate fails to yield statistically significant results

Earlier this month Neurotrope reported results from its Phase 2 Alzheimer’s study of Bryostatin. The trial involving 147 patients with moderate to severe Alzheimer’s met its primary endpoint in patients that completed the full course of treatment. Bryostatin improved outcomes in cognition and ability to handle daily activities but failed to achieve statistical significance over placebo (FierceBiotech).

Neurotrope is hopeful of its candidate and plans to take the study forward.

MedNess: This is yet another Alzheimer’s candidate that failed in Phase 2 testing. The successful treatment of dementia is an enigma, and most drug candidates fail larger phase 2 trials. No new drug has been approved for Alzheimer’s for more than a decade. Neurotrope’s shares fell by 63% (Biospace)

Moderna Therapeutics reports interim Phase 1 results from mRNA based H10N8 flu vaccine

The private biotech company; Moderna Therapeutics reported positive interim results from phase 1 study of mRNA-1440 vaccine against avian H10N8 flu. The complete data and results were published in the journal Molecular Therapy.

31 subjects were enrolled in the Phase 1 study, and 23 received 100 µg of the vaccine. All participants achieved HAI titers suggesting seroprotection against seasonal flu. No response was achieved in the placebo arm (8 subjects). (FiercePharma)

MedNess: Since the company is still private, the only investment that can be made in Moderna Therapeutics is through venture capital funds. The company’s valuation is $3B, and primary sources of funding are grants and private investments (Investopedia and The Motley Fool).

Sanofi against affordable pricing for Zika vaccine

Sanofi Pasteur, the leader in vaccine manufacture, was under fire by US army officials and Senator Bernie Sanders after refusing the US Army’s plea for affordable US price for a Zika virus vaccine. Sanofi boasts of about $43 million US research grant money. Sanders and lawmakers have been requesting the US Army to reconsider its term of negotiations with Sanofi that could provide the latter an exclusive license to make and sell Zika vaccine in the US. The vaccine is being developed with the American taxpayer funds, thus prompting both the US Army and Sanders to request an affordable price for the American population.

The spokesperson from the Army states that the decision to provide Sanofi an exclusive licensing is still under consideration and the final decision will be made in the summer (STAT).

Corbus Pharma’s anabasum denied BTD for systemic sclerosis; enrolls the last patient for mid stage-study dermatomyositis

Corbus Pharma’s anabasum (Resunab) was previously provided Orphan Drug designation for Fast Track review by the FDA for systemic sclerosis. However, this month Corbus Pharmaceuticals was trying to gain Breakthrough Therapy status for the drug. The plea was rejected by the FDA. The BTD designation entails recurrent meetings with senior personnel and a rolling review of the New Drug Application. Meanwhile, in another phase 2 clinical trial where anabasum is being tested for dermatomyositis, the last patient was enrolled

MedNess: The failure to gain BTD designation by the FDA worried the investors, slipping shares down by 12%. However, with the news of last patient enrollment, the shares moved up by 3%.

The Belgian-Dutch Biotech Argenx draws $115M post-IPO filing

Argenx filed for IPO last month to draw cash from American investors; in order to push its lead candidate ARGX-113, an antibody directed against autoimmune disorders myasthenia gravis and primary immune thrombocytopenia, to phase 3 trials. It also proposed moving its lead cancer candidate ARGX-110 through mid phase studies. The biotech was able to rope in $115M, 50% above its target goal of $75M.

The biotech started their phase 2 studies with ARGX-113 earlier this year, and the results are expected in the first quarter of next year. A 30% drop in the IgG antibody level would be considered clinically significant (Fierce Biotech, Market Watch).

Incyte reports positive results from selective IDO1 enzyme inhibitor, epacadostat in two separate combined trials

Incyte reported first set of positive results from ongoing combined trials; ECHO-202 in combination with Merck (epacadostat+ Keytruda) and ECHO-204 in combination with BMS (epacadostat + Opdivo). The full sets of results will be announced at American Society of Clinical Oncology (ASCO) meeting next month.

The ECHO-202 trial is assessing epacadostat (selective IDO1 enzyme inhibitor) in combination with Merck’s Keytruda (anti-PD1 immunotherapy). The efficacy and safety results from the phase I/II trial showed that epacadostat in combination with Keytruda was well-tolerated in the following cohorts: non-small cell lung cancer, renal cell carcinoma, ovarian cancer, triple-negative breast cancer, bladder cancer, and head and neck squamous cell carcinoma.

Alternatively, data from the ECHO-204 trial evaluating the safety and efficacy of epacadostat, in combination with Bristol-Myers Squibb’s PD-1 inhibitor Opdivo showed the combination was well tolerated in melanoma, head and neck squamous cell carcinoma, ovarian cancer, and colorectal cancer (Incyte.com).

MedNess: Following the positive results, Incyte’s shares gained 2.3%, rose by 6% by mid-day which rose by 8% towards the end of the day (Thursday, May 18, 2017) with the overall increase of 14.10% by the end of the week (Zacks, Investor Place, The Street, and CNN Money).

About the Author:  

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

Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)

 

 

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