Ebola, Tekmira Pharma, Highlight SF BIO Investor Forum Meeting

San Francisco, The Palace Hotel, BIO Investor Forum meeting, Tues. Oct. 7, 2014. The annual BIO Investor Forum opened its meeting at midday with welcome remarks and a program change that included new information about the urgent subject about healthcare developments in Ebola among other topics. The welcome remarks featured a short discussion with Tekmira Pharmaceuticals’ CEO, Dr. Mark Murray PhD about his firm’s involvement in this fast moving healthcare area. Dr. Murray touched on the fact that Tekmira is working on a promising early stage antiviral drug to treat patients infected by the ebola virus. More details would follow at his afternoon investor talk

Dr. Murray said in his afternoon investor talk that Tekmira Pharmaceuticals uses an RNA interference based therapeutic in their ebola drug development program.  RNA interference (RNAi) is a naturally occurring internal cellular process that shuts down the production of targeted proteins. Dr. Mark Murray also talked about Tekmira’s drug therapy developments in nine clinical programs underway.  He said that Tekmira can trigger RNAi, but needs a delivery technology that uses the LNP-RNAi trigger mechanism.  They are working on the TKM-PLK1, TKM-H8v, TKM-ebola drug programs.  The firm is using the FDA orphan drug rare disease designation for HTG, GSD4 and so on. Their Lipid NanoParticle (LNP) partner, is Anylum, using their ALN-TTR02 platform.

Tekmira has nine products in its clinical pipeline. Dr. Murray said that its lead products include two antiviral programs.  The TKM-HBV is being developed to treat Hepatitis B and is being readied for sometime in 2015. The TKM-Ebola program is in phase 1 clinical testing.  The FDA gave Tekmira a‘Fast track’ designation for the program.  Dr. Murray added that the product formulation gave a very high survival result in animal testing using monkeys.  Tekmira will work with the World Health Organization (WHO) in the West African region for its ebola drug human clinical testing program.

Oncology product programs. The TEK-PLK1 product acts on pololukekine-1, which is found in many tumor types, usually ones linked to poor outcomes.  The tumor types include GI neuroendochrine tumor and adenocortico cancer.

Rare disease program. Hyper trigliceridemia. The condition leads to pancreatitis.  GSD glycogen is a rare condition.

Financials. Tekmira Pharmaceuticals (TKMR) is a public stock company located in Toronto, Canada, is on the NASDAQ stock Exchange and has a $528Million market cap.

Drug Program Status.

  • 2014    PLK1 Phase I/II
  • 2015    Follow on of clinic activity.

Dr. Murray concluded his talk by saying that for further information go to the company website at ir@tekmira.com.

Several hundred investors and presenting company officials gathered at this year’s BIO Investor Forum meeting for one-on-one partnering meetings, 128 company presentations, plenary informational talks, workshops and networking.

 

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Growing Ebola Outbreak in West Africa Fosters Urgent Need for More Drugs and Tests to Help

A man who recently returned from west Africa to Dallas, Texas yesterday was admitted to a hospital there sick from Ebola. He is the first US case of a tourist to be infected by the deadly disease. The CDC confirmed his diagnosis. The newswires are abuzz about how this man traveled from Africa on a commercial airliner to the US while infected with the Ebola virus.

This story follows the two Ebola infected US doctors who were airlifted in a specially equipped airplane to an Atlanta hospital where they were treated and recovered. This Ebola outbreak is the largest one since it was discovered in 1976 in the Congo.

Since the Ebola cases double every 21 days, public health experts estimate that there might be 300,000 to 500,000 cases in west Africa by year-end and perhaps a million cases by mid-January 2015. The US has committed 3,000 troops to the area to provide supportive care to the decimated health care workforce.

Several drug companies have developed experimental drugs and vaccines that are in clinical trials that may be fast tracked to African patients a some point in the future.

  • Mapp Biopharmaceutical of Sorrento Mesa –ZMapp serum
  • Newlink Genetics Corp.  Earlier in the month, FDA gave it the OK Phase clinical trial for its Ebola vaccine.
  • Sarepta Therapeutics Inc. Sarepta is developing a treatment designated AVI-7537 to treat the Ebola virus.
  • GlaxoSmithKline PLC .Glaxo is working with the National Institutes of Health’s Vaccine Research Center to help develop of an early stage vaccine for Ebola.
  • Tekmira Pharmaceuticals Inc. TKM-Ebola is in Phase 1, clinical trials.

MacWorld/iWorld 2014 Returns to Moscone North

SAN FRANCISCO (HighTech Reports), Moscone North, March 27, 2014 – MacWorld/ iWorld 2014. It was refreshing to see that MacWorld/ iWorld returned to Moscone North, MacWorld’s former location of years ago. There were around 30,000 attendees at this year’s MacWorld. The exhibit floor seemed bigger than last year and highlighted more booths that we visited. We spent some time in the MacIT room where companies that had booths gave presentations. While there, we learned about products from Printer Logic, Lantronix, Absolute Software, Parallels, and Crash Plan.

Printer Logic sells an enterprise software app that attaches to Windows 2008R file server. The app replaces print server boxes in the enterprise. A license for up to 50 printers costs $5,000. The spokesperson said that most users have 25-30 printers. Each client driver for Mac/Windows PCs links to the printer app via Wi-Fi that scales very big in the enterprise. For example, the Department of Homeland Security used its service to reduce support calls for print servers that went down. The app solution was more efficient and saved the client money.

Absolute Software makes software that helps organizations manage all types of computer assets: PCs, laptops, tablets, phones, etc. It helps track the life cycle of all IT equipment assets to end-of-life. For example, the software helps Apple track iTunes content licensee IP assets. The firm’s solution helped the Columbine school track tablets, laptops, and thefts from intruders entering its campus. The company puts a special sticker on devices with a phone number to call if a child gets cyber bullied. The victimized child connects to a caring person to talk to and so forth. Absolute Software is also exploring other vertical markets.

 

Emerging Changes Coming to the Drug Development Ecosystem

At last, the face of the Affordable Care Act (ACA) has entered the public arena.  Last October at the launch of the web marketplace of the ACA, the public became keenly aware that major changes in the US healthcare system were underway.  Despite its rocky beginning, by January several million customers had successfully signed up for their new healthcare insurance plans for 2014.  The ACA will have an impact on many stakeholder groups including patients, health care providers, hospitals, insurance payers, government agencies, drug developers, and others.  In particular, one can expect to see changes of the drug development ecosystem emerge over the next few years.

On January 14th at the Parc 55 Hotel in San Francisco, the Biotech Showcase presented a plenary luncheon presentation, The Changing Dynamic of the Drug Development Ecosystem.  A panel of experts shared their insights and opinions of what might play out in the industry over the next few years.  Moderator, Ellen Corenswet, Partner at Covington & Burling LLP, posed key questions to the panelists.

The panelists included:  Karen Bernstein – Co-Founder, Chairman and Editor-In-Chief, BioCentury; Anton Gopka – Managing Partner, RMI Partners; Dan Mendelson – CEO, Avalere Health; Dennis Purcell – Sr. Managing Director, Aisling Capital; Evonne Sepsis – Managing Director, ESC Advisors.

Karen Bernstein opened the initial discussion about patients, what drugs they want, how they pay for healthcare insurance and so on.  She spoke about the ACA (aka: ObamaCare) in relation to patients and how they interact with drug companies.  Dan Mendelson seemed to know many detail data points about what to expect from the ACA since his firm has done some studies that model the potential impacts of the law.  Dan said “more and more, healthcare costs are being pushed to consumers.”  He said that many of the plans offered on the Exchanges cause consumers that need Tier-4 drugs “to pay 50% of the market price of the drugs.”  Tier-4 drugs are expensive targeted cancer drugs.

Berstein said that drug companies have offered discount coupons to consumers for some expensive drugs.  Mendelson said that there is a contradiction between ObamaCare and Medicare.  For example, Medicare does not accept discount coupons.  But, ObamaCare does accept discount coupons.

Dennis Percell said “In Boston, there are three hospitals within three miles of each other that do heart transplants. In New York City, there are hospitals that repair hips. There is one that charges $15,000.  At another place it costs $60,000.”  He asked, “What’s going to happen in pricing five years from now to the hospitals that do the heart transplants and the ones that do the hip repairs?”

Dan Mendelson  said he has “seen that consolidation is already happening either through acquisition or through contracts among organizations.“  “In many areas there is significant over capacity such as from teaching hospitals and academic centers.”  He said that “a lot of the teaching hospitals are being excluded from the networks that are fielded under the exchanges.”  Dan said that the exchanges will get up to about 6 million people and will represent just two percent of the health care system.

He said that “small businesses are calling the insurance companies and asking them to design new insurance plans for them.” He expects that “over the next five years, the benefit designs for the ACA will eventually spill over to whole healthcare insurance market.”  This scenario will have an impact on biotech’s future drug development plans.

Evonne Sepsis said “Reimbursement needs to be considered at the beginning of drug development process. She said “historically, most drug development companies considered reimbursement later.”  But not now.

Bernstein noted that companies say that their partner’s drug R&D costs need to be recovered through higher prices. However, high new drug prices are not sustainable. Dennis Purcell pointed out that “the last ten of twelve new cancer drugs cost $100,000 or more” for a course of treatment.

Evonne added “A few years ago, ten years ago, we saw the orphan drug market emerge. It needed just a patient population of 1000-5000.”  But now “with smaller patient populations in personalized medicine it is similar to the orphan drug market.”

Ellen asked  “What about the role of international going forward?”

Anton Gopka said that international pharmas are doing clinical trials in Russia for proof of concept studies.  This business model might work.  After the trials they can commercialize the new drug in U.S. Dan Mendelson said “Well maybe not so, because most U.S. payers or regulators prefer drug trials to be done in U.S.”  Dennis Purcell said  “If it were a country, the U.S. healthcare system would be the 5th biggest country in world.”

The discussion turned toward disease foundations and patient groups.

Dennis Purcell said “I believe that we will see lot of disease foundations that will open a VC arm.  So VC groups should work together with them to bring their projects forward.  Dan Mendelson offered that disease groups would work as clients. That is. A diabetes group would test the glucose value of products.

Ellen asked how to engage with the patients.

Dan Mendelson said that we need to understand and look at the quality measures. “We can’t expect the payer to give you a guarantee. Its not possible.” “We need to show an advantage vs. other drug competitors.  We need payer buy-in — each payer wants something different.  Small companies need to be competitive.  This situation has ham-stringed the FDA — They are not up to speed with the leading technologies.”

Karen Bernstein wrapped up their discussion by saying that the FDA is good in certain areas.  They are the only government agency that is under funded.  She noted that Janet Woodcock is expected to return soon, others, key people are expected to leave or retire by 2016.  She said “I see the next five years as tough for the FDA.”

iPhone 5 More Powerful than the Curiosity Mars Rover

SAN FRANCISCO (Takeda Pacific HighTech Reports), Moscone West, January 30, 2013 — MacWorld/iWorld 2013.  NASA experts spoke about NASA’s Curiosity Mars Rover and its connection to Apple products at a meeting here.  The session was titled “Software, Hardware, and Flying to Mars. How We Built, Programmed and Operate NASA’s Curiosity Mars Rover.”  David Oh, Lead Flight Director and Software Engineer at JPL/Caltech Mars Science Lab, asked the question, “Why are we at MacWorld?”  The answer was shown in a slide of the NASA control room at the JPL in Pasadena, California.  On the desk was a mixture of Apple products such as MacBook Pros, iPhones and iPads during the Rover’s Mars landing.  The Macs were running Mac OS X.

Ben Cichy, Chief Flight Software Engineer, compared the processing power of the iPhone 5 to Curiosity’s processing power.  The iPhone 5 has 1.3 GHz and the Curiosity has only 132 MHz.  In addition, the iPhone 5 has 1GB of memory and 64GB of storage and the Rover has 128MB of memory and 4GB of storage.  The cost of an iPhone 5 is $399 while the cost of the Curiosity was $1.8 billion.

During the session, the audience was shown a video of the landing of the Curiosity on Mars.  It only took 7 minutes, but a very tense 7 minutes.  It was very exciting watching the landing of the Mars Rover all over again.  “The Rover’s goal is to explore and see if Mars ever sustained life or is now,” Ben said.

David showed a slide that described the different parts that make up the Rover.  He added that the heart of the mission is the SAM (Sample Analysis at Mars).  The SAM analyzes the chemical and isotopic composition of the planet’s atmosphere and surface.  He said that the “Curiosity has two brains (computers) in its belly.  One is the primary computer and the other is the backup.”  This time social media plays a big part in providing people access to seeing what is happening with the Curiosity as it explores the surface of Mars.  The Curiosity has its own Facebook page and iPhone app.

Maria Bartiromo and J.P. Morgan’s Jamie Dimon On Business in 2012 at SF Healthcare Meeting

On Monday January 9, 2012, I attended the J.P.Morgan Healthcare Conference luncheon at the Westin St. Francis Hotel in San Francisco.  During the luncheon, Maria Bartiromo, journalist and news anchor at CNBC, interviewed Jamie Dimon, Chairman, President and CEO of J.P. Morgan Chase about a number of issues facing his company, the U.S. economy and Europe in 2012.

I compiled a list of some of the answers to questions from Bartiromo and Dimon’s answers to those questions.  Bartiromo asked Dimon about the financial health of J.P. Morgan Chase.  The stock is lower, but the company is better with record earnings, Dimon said.  When asked about the U.S. economy, Dimon’s answer was that the U.S. economy is in a mild recovery.  He sees small business in better shape.  He does not see a huge formation of small businesses, however.  It is not access to capital that is the problem, but demand for their products.  There are also IPO backlogs.

He said that early indicators for the recovery include: housing improving and near bottom and shadow inventory is getting better.  He also believes that more people working, the better it is for the economy.  He is not nervous about the capital markets.  However, he did say that geo-politics is always the wild card.   When asked about the European debt crisis, he said that it has to be fixed for the health of the world.  He said that “countries have to be responsible.  They need to change their fiscal policies.”  J.P. Morgan has cut back exposure, but still is investing in Europe.

Bartiromo also asked Dimon about the “Basel Stress Test”.  He said he is in favor of a good  stress test.  Dimon said that J.P. Morgan will be fine with worst case scenario.  He added that J.P. Morgan never lost money in a quarter after the greatest stress test, which was the “Financial Crisis” in 2008.  He wants fairness around the world for derivatives.   He also commented on the Fed’s “Volcker Rule.”  “The Feds do not want us to take any risks,” Dimon said.  He wishes that people “writing these government rules were business people.” However, he is not opposed completely to regulation.

When asked about ObamaCare, he said he wants healthcare for everyone, but he believes that “ObamaCare just added to the mess.”  Bartiromo asked him about Fannie and Freddie.  He said they should have a hybrid or eliminate them.

When a member of the audience said he should run for President, there were huge laughs.  However, he said he has no plans to do so.

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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