In-Silico Drugmaker Highlights Rapid Rx Development at BIO Investor Forum

 BIO Investor Forum meeting October 21, 2015, Parc 55 Hilton Hotel, San Francisco. Cyclica is an emerging biotech firm that invented a therapeutic platform based on technologies. It’s called the Ligand Express (TM). The company is now in a license deal with Johnson & Johnson . They repurposed a pharmaceutical product made in the US. The DoD asked them to weed out 2000 drugs down to 53 and eventually they selected one, Zoloft. It turns out that this drug works against Ebola. The Army’s recent deployment of 3000 people to Africa to set up field hospitals, used Zoloft to treat Ebola patients.

Another example, Procter & Gamble has a drug compound that was causing skin irritation in some people that use their Olay skin cream products. It caused a rash. The company isolated three compounds that cause the issue and then eliminated the compound. 

The company’s vision is to 

  •  Cut drug development costs in half
  •  FDA to approve their drugs and 
  • Be the best tech platform for every doc to do personalized medicine solution in the future. 

The goal is to create an automated platform compared to other in-Silico players. Other in Silico firms look for chemogenic compounds, but this company does it all via Ligands. The company’s business model is mainly to work with big pharmaceutical and nutraceutical projects. 

The platform is like the eBay platform. For example on a project basis they might get $10,000 $20,000 in revenue with this model. The second thing they would do is have a license model in which they would receive $25,000 or more per project. 

Their current prospects include Procter & Gamble, the Department of Defense or other similar types of firms. The CEO’s name is Paul Angelico. He said that they will need $20 million in cash by 2019. They currently seek $9.5 million from VC investors.

Speakers at Biotech Showcase See More Growth for 2015

San Francisco — January 13, 2015. The EBD Group’s Biotech Showcase conference was held at the Park 55 Wyndom hotel in San Francisco.
During lunch time they gave a plenary talk to a group of 300 or so investors. The talk consisted of a panel of about seven people talking about the prospects of biotechnology in the coming year. The bottom line is that this group of people as a group and individually pointed out aspects of what’s going on in biotech today and it’s likely that biotechnology will continue to grow in 2015 at the same pace as it was in 2014. The take away message from this panel session is that these people believe that 2015 will be a another good year for biotechnology companies in the industry in general because the trend tends to look positive. The Q and A: they focused on the exclusivity pricing for biosimilars.

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.

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.

 

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.

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.

Bay Area Biopharma Firms Evolve Their Mobile Apps

I recently attended the Apps World North America Conference on Feb. 5, 2014 held at, San Francisco’s Moscone West convention center. The event is driven by the huge interest in mobile apps by mobile device users, developers and device makers.   Since mobile apps are a core technology that makes mobile devices so useful, the subject of developing and commercializing mobile apps has become an important business topic to over 8000 visitors to this interesting event.

Apparently, a number of healthcare companies were doing some work in the mobile apps space that might benefit many kinds of enterprises.  Paul Lanzi, Senior. Manager of the Mobile, Web and Portal Team, Gilead Sciences, gave a talk about the Evolution of mobility in the enterprise. Paul’s team provides the infrastructure to support mobile solutions at Gilead. Paul said that he started in the tech industry in 1994 and worked at Genentech in a similar job prior to his Gilead role.

Lanzi cited recent mobile milestones by saying that change has come rapidly and best practices were learned from various experiences.  In 2012 his company’s apps development grew from 1 to 100 enterprise mobile apps. By 2013, they were creating enterprise apps that employees actually want to use. Going into 2014, he expects to see more of the evolution of enterprise mobility.  Paul said that the most common mobile devices ten years ago were the Motorola Razor and the Blackberry. Lanzi said that developers need to use the idea of Mobile First and said that “In the Net, Net things change really fast.

There are four evolutionary stages of mobility in the enterprise.

Brochureware. He gave an example of his local restaurant took their paper menu and put it on the web. That’s all they did.  He showed that pharmaceutical firms like Gilead or Genentech make brochureware of Rx package inserts. People can go online and read the drug package insert online.  It adds value above a paper version, its low cost, fast to deploy and easy to update.

Paul said that enterprise app developers need to watch out for certain things by saying that it is best to avoid making an evolutionary dead end with mobile apps. Paul used a biology evolution example to make his point that crocodiles and chickens have something in common.  At some intermediate points along the evolutionary path, dinosaur-like birds with teeth emerged that became evolutionary dead-ends and went extinct.  Similarly in the PC apps evolution, the keyboard driven command line interface evolved into the familiar windows and mouse click interface.

 Snackable Apps. Its best to mobilize a key subset of business processes, “instead of providing the whole ocean.”  For example, Lanzi said that a business app that enables “Review pending transactions, approve pending transaction” is a very simple user interface. Users at Genentech said “I approve with gApprove even when I’m sitting in front of my computer.”

He said that sometimes you get some surprises.  Once they need to needed to assign support tickets on mobile devices for facility power plant workers.  They had a new app designed, but no one used it. It turned out that a 37-yr-old designed it, but the average age of end-user was 58.  What happened is the workers at that age had worse eyesight and could not read the app. The app had 12 point type.  Lanzi said that mobilize the right part of the process,  get a deep understanding of process, and keep adjusting the app.

Full Business Processes. Paul said that Concur is a complete business process at his company designed for expense reports. Concur has done a great job of adapting with Blackberry and IOS devices.  Lanzi said that developers need to choose the right process and never stop evolving.

Mobile First Enterprise.  Paul said that developers need to consider mobile first, the philosophy, and leadership, procurement, finance, hiring, and so on. Lanzi said that enterprise app builders need to design for change. For example, when Paul was working at Genentech, he said that the sales team in Spain was given iPads to replace laptops. The sales people had to get used to doing work on that device instead of on their laptops and so forth. He said that you can contact Paul on Twitter using @planzi.

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