Big Data and Precision Medicine Trending at CHI Tri-Con in SF

Moscone North Hall, Feb. 17, 2015. After walking the halls in the exhibit area at the recent annual CHI Tri-Con event in San Francisco, I discovered that a theme came together after I passed by various booths.

For one thing, the words “precision medicine” seemed to be resonating among those firms that were exhibiting and I asked some of them, “Is that the same thing as “personalized medicine” or “individualized medicine?”” I noted to that person that President Obama had recently made some kind of a speech that was promoting the idea of precision medicine so maybe the time has come for precision medicine to take the spotlight.

In any event I also found that there are other themes there such as big data. It is being used in a number of different biomedical research areas. I stopped by the Illumina booth spoke with the lady there whose name was Kathleen. She said that she had just joined the company about two months ago from Roche where she was involved in the clinical area. She said that her firm is moving into the data management side of their business with a focus on clinical diagnostics and take advantage of the fact that a lot of NexGen sequencing is now being used for clinical types of applications and will be generating lots and lots of data.  So big date is the theme here as well. They’re hoping to sell their systems into the clinic and hospital type settings so that they develop some very useful software systems to make sense of all that data. Data analytics is going to be a big deal.

I walked around and came across another booth that was also telling the story of powerful computer power and big data and that was the guy at Cray Computer that is famous for supercomputers in the past, but today they are using many many computers together as a cluster, a Hadoop and have another one they called SPARK. I’ll have to check out what “Spark” means. It seems to me that quite a lot is happening in the software.

CHI had other usual events that they have at the Exhibit Hall such as a raffle in which an attendee might win some kind of electronic gadget. This part of the event also featured a discussion tables. There were 40 tables that could handle as many as 8 to 10 people.  I noticed that just about every table was filled up in the hall and some of the tables had probably 10 to 15 people there, so they must have had some very popular topics to discuss. Traditionally, this part of the exhibit area has been very popular in past meetings that I’ve attended.

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Simple Cancer Biomarkers are Inadequate to Enable Personalized Medicine

It seems that researchers are finding that using single cancer biomarkers to develop companion diagnostics (CDx’s) to be used with future targeted therapeutics is very challenging. An article in the November 15 issue of Genetic Engineering and Biotechnology News, called Traversing the Cancer Biomarker Labyrinth, by Kathy Liszewski, is a very interesting read.

Apparently progress in this field has slowed in finding clinically useful biomarkers for diagnostics and making other tests that guide doctors for disease prognosis and prediction. Researchers are using a variety of reductionist technical approaches that range from analysis of certain glycans, key microRNAs, and epigenetic changes, to big data analysis of massive data stores of genomic data to tease out more clues to what is going on in cancers.

Scientists seek to develop early detection blood tests that can detect cancers of interest.  Such a blood test could be considered a ‘liquid biopsy’ and might include a panel of a dozen or more miRNAs that represent a biomarker signature.  An oncologist might one day be enabled to quickly screen certain patients with a blood test that would help them diagnose, stage or predict the potential outcome of a cancer.

Achillion vs. Actelion More Biotech M&A’s to Come

Last week’s announcement by Swiss drug maker, Roche Holding AG, that it was buying biotech firm InterMune, Inc. for $8.3 billlion sparked speculation by industry watchers about who would be the biotechs that might be involved in the next round of M&As. InterMune makes a drug, Esbriet (pirefnidone) that treats a lung condition, idiopathic pulmonary fibrosis. Esbriet is approved in Canada and Europe. The drug has the potential to become a blockbuster seller. InterMune’s product would join Roche’s Pulmozyme and Xolair to build up its lung drug portfolio.

A number of names popped up such as Achillion Pharamaceuticals, Actelion, Puma Biotechnology, Intercept Pharmaceuticals, and others. Maybe people heard Achillian but were attracted to similar sounding Actelion.

  • Achillion Pharamaceuticals makes Phase II hepatitis C virus (:HCV) candidate, ACH-3102.
  • Puma Biotechnology is developing its Phase III breast cancer candidate neratinib, PB272.
  • Intercept Pharmaceuticals makes its phase II nonalcoholic steatohepatitis drug, obeticholic acid, OCA.
  • Actelion Pharmaceuticals makes a cancer drug to treat a rare form of non-Hodgekin’s lymphoma.

Some in the big pharma side are still holding out hopes of buying a biotech that can make their next blockbuster. So which will it be? Achillion? Actelion? Others? To be sure, we will need wait and see.

Acute Need for Early Warning Dx for “Silent Killer” Cancers

There is a need for early warning diagnostics to detect “silent killer” cancers such as pancreatic, liver, GI and lung cancers. Most of these cancers are discovered at the late stage when acute symptoms appear and no cure is possible.  Maybe an annual simple screening test could be developed that could accurately detect a molecular signature of these cancers.

It is a very sad situation indeed to see patients die just after two, four or six weeks post-diagnosis.  Just this past weekend, our co-writer and industry analyst for this blog and our market research firm succumbed to an aggressive cancer of unknown primary origin that spread to the liver.  She died in less than five weeks from diagnosis.  The FDG- PET CT scan showed active cancers in the liver, pancreas, uterus and breast. Ultimately the oncologist concluded that the cancer likely came from the pancreas. May she rest in peace.

Close relatives interested in getting a PET scan for themselves were advised that the Radiology Dept. would only offer a PET scan after a patient was already diagnosed to have cancer.  Whereas a PET scan is good at detecting cancer, it is a very expensive resource that hospitals choose to limit access to.

What is needed is an annual pre-symptomatic, accurate, low-cost multiple-molecular-marker, blood test that can screen for cancer at the earliest point – before a tumor is formed. The screening test would likely use a panel of biomarkers associated with the “silent cancers.”

OvaScience, Seattle Genetics, and Qiagen Present at 31st J.P. Morgan Heathcare Event

This year’s 2013 J.P. Morgan Healthcare Conference at the Westin St. Francis in San Francisco (January 7  to 10) had around 8400 attendees and around 400 company presentations.  Navigating through the hallways to the numerous presentations was a challenge.  These are three of the many company presentations I attended.

OvaScience’s CEO Michelle Dipp gave a very interesting presentation about new infertility treatment options. The U.S. fertility market is over $4 billion and it is seeing rapid worldwide growth, said Dipp.  Some other interesting statistics included that every year there are 7.3 million childbearing women that are infertile and 1.2 million women seeking treatment.  There are over 400 IVF clinics in the U.S.  Most of them are in the East and West Coast.  Unfortunately, most IVF treatments fail because many women are delaying childbirth. Apparently, energy in eggs decreases with age.

OvaScience has discovered that adding mitochondria to human eggs increases IVF success. The company’s new approach to infertility is called the Egg Precursor Cell (EggPC).  The discovery of these EggPCs (germline stem cells) that mature into eggs offers new fertility treatment options, said Dipp.

The company has two product candidates that include Augment and OvaTure.  There is a study underway for the firm’s first product, Augment.  Augment uses EggPC mitochondria to rejuvenate eggs.  The company’s second product OvaTure involves fresh, young, healthy eggs matured in the lab from EggPCs.  The OvaTure program is currently being designed.  According to Dipp, the goal is to improve the IVF success rate for older women while reducing the number of embryos that need to be transferred to the uterus thus decreasing the number of multiple births.  That would be great news for the many infertile women who could benefit from this treatment and not have to worry about the potential of multiple births.

Seattle Genetics’ President and CEO Clay Siegall began his presentation with the company’s key value drivers.  They include building the Adcetris franchise, advancing the antibody-drug conjugate (ADC) pipeline and technology along with its strong financial position and collaborations to fund a very robust research.  Adcetris targets the CD30 cell membrane protein, which is expressed on the surface of certain types of lymphoma cells.  Adcetris is FDA approved for relapsed Hodgkin lymphoma and systemic anaplastic large cell lymphoma.

The firm also received EU approval for Adcetris in October 2012.  Seattle Genetics has 20 internal and collaborator ongoing clinical programs for Hodgkin lymphoma and other cancers.  The company has collaborations with Millennium/Takeda, Genentech, Celldex, Bayer, and Abbott just to name a few.  According to Siegall, “ADC collaborations have generated over $200 million to date with the potential for around $3.8 billion in future milestones plus royalties.”  Net product sales of Adcetris since launch in August 2011 are over $145 million.  Siegall said they are “making strong progress towards a fully global brand.”

Peer Schatz, President and CEO at QIAGEN N.V., began by saying that “2012 was a very important and successful year.”  He said that QIAsymphony is the company’s fastest growing product in molecular diagnostic placements with Europe being the biggest at 40 percent and the U.S. coming in at 35 percent.  Another of its products is the Therascreen KRAS test, a companion diagnostic for metastatic colon cancer to help guide doctors in the use of Erbitux.  “The U.S. KRAS market conversion is progressing well.  Doctors are demanding the Therascreen test,” said Schatz.

Over the next 2 years, the firm will be developing several new molecular diagnostic assays.  Qiagen is also developing new biomarkers with the potential as companion diagnostics.  One of the company’s goals is to “expand NGS from research to routine clinical use.”  Qiagen is preparing the launch of its first NGS workflows in 2013, which includes a broad range of its products such as the QIAsymphony NGS version.  In closing, Schatz said the company will be “executing on its 2013 initiatives to drive growth and innovation at a faster pace.”

Diagnostic Companies Speak About Challenges With Pharma Partners at IBC Event

On August 6, 2012, I attended the IBC Life Sciences’ Drug & Diagnostic Development Conference at the Westin San Francisco Market Street Hotel.  The three-day conference consisted of four ‘summits’ that included the Future of Rx/Dx Summit, the Clinical Biomarkers and New Frontiers in Cancer Summit, the Next-Gen Sequencing Summit, and the Antibody-Drug Conjugates, Bispecfics and Empowered Antibodies Summit.  I decided to focus on the Future of Rx/Dx Summit business track.  A number of the presentations discussed the many challenges and success factors that go into developing companion diagnostics (CDx), which companies need to consider when developing a CDx.

Tips to Navigate Rx/Dx Co-Development

Patrick Goody, Divisional Vice President, R&D at Abbott Molecular brought up a number of important guidelines during his case study presentation, “Co-development of Diagnostics and Therapeutics: Abbott/Pfizer Crizotinib.”  Crizotinib (Xalkori) is Pfizer’s personalized medicine that targets a type of late stage non-small cell lung cancer (NSCLC).  Abbott Molecular developed the FDA required EML4-ALK fusion companion molecular test that enables physicians to select the sub-group of patients that would benefit from using crizotinib.

He said in order to be successful, the partners need to “capitalize on their collective strengths and establish good chemistry.”  He believes that good “communication is key.”  Other important factors include “coordination of drug/IVD submissions as well as commercial execution and worldwide distribution.”  Goody emphasized that pharmas should get involved with a diagnostic partner early. This point was mentioned in other presentations as well.  He also said that there must be business incentives for both partners.

Ron Mazumber, Global Head, Research and Product Development, at Janssen Diagnostics spoke of the “Challenges and Opportunities/Solutions of CDx in Pharma” during his presentation.  Some of the main challenges include the “complex regulatory landscape, PMA process/expectations, and variability of testing.”  He offered some solutions such as “repurposing an existing 510 (k) cleared product, bridging studies, use of CTCs (circulating tumor cells), and multiple partners/platforms.”  Mazumber said the “ideal scenario is to start at Phase 3 with an IUO (investigational use only).”

Panel Discusses Rx/Dx Business Ideas

I also attended the panel discussion about “Emerging Commercialization, Collaboration and Business Models for Rx and Dx.”  One of the major themes of the discussion was that diagnostic companies need to continue to drive the value of their product.  Panelist Michael Pellini, President and CEO, at Foundation Medicine, said, “it’s all about education.”  Another panelist, Ron Andrews, President, Medical Sciences, at Life Technologies said, “We are venturing into a more complex world.”  He said “a lot more goes into a diagnostic nowadays than even five years ago.”  To the question posed by moderator Alexis Borisy, Partner, at Third Rock Ventures, “Do Pharmas ever pay diagnostic companies big fat royalties?”  “No, pharmas are not paying diagnostic companies big fat royalties,” said panelist Ron Mazumder at Janssen.”  However he did say, “pharmas are starting to recognize value but it is still a sticky issue.”  Andrews said “there are tons of therapeutics that have been shelved that might have value if the right diagnostic is developed.”  Panelist Pellini said that he “dreams of a fat royalty if they enter into a relationship with a pharma company and salvage a drug with a diagnostic.”  “They should share revenue downstream if they add enough value,” he added.

Biotechs Seek Opportunities with Social Media, FDA Still Murky…

On 6/16/11, I attended the BioConference Live interactive online-only life science conference and attended the Panel Discussion titled, “Social Media in Regulated Industries: What are the Opportunities and where is the line?”  I thought I would share some the interesting points brought up during the discussion.  Panelists included Moderator Tina Baumgartner from Accella Group, Pamela Lund from PL Interactive, and Lianne McLean and Mya Thomae from Myraqa.

Baumgartner said that some of the opportunities in social media are the ability to “listen to customers, recruiting employees, attracting funding, and increasing visibility.”  She discussed three of the common misconceptions of social media, which include that it is primarily a tool for marketing and PR, it is too complicated and time consuming, and it has the same regulatory compliance risk as traditional PR and advertising.  None of which are true, according to Baumgartner.

McLean discussed a number of challenges to marketing in a regulated environment such as companies are only allowed to promote their product for the legally approved label and they must report adverse reactions.  She also pointed out that “the FDA’s regulatory stance has been unclear.”  Apparently, the FDA has been promising a guidance document since 1996.  In the meanwhile, the FDA has been issuing warning letters to companies that they think have crossed the line.   In July 2010, the FDA sent Novartis a letter issuing four violations for its “Facebook Share” widget saying among other things that it did not present a balanced viewpoint of its leukemia drug, Tasigna.

One of the attendees asked the panel, “will there be regulatory documents coming soon from the FDA?”  The answer was “not really.”  Another attendee asked, “what is the time horizon of widespread adoption of social media?”  Panelist McLean said “in the next couple of years or so.”  She also said that “pharma companies would adopt more quickly than diagnostic companies.”

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