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.

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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.”

British Tech Network and New Products at MacWorld/iWorld 2014

SAN FRANCISCO (HighTech Reports), Moscone North, March 27, 2014 – MacWorld/ iWorld 2014. We watched a panel discussion session at the Second Stage presented by the British Tech Network (BTN) with speaker Paul Wheatley, User Experience Consultant and Host at the BTN. Paul and the panelists talked about the pros and cons of a bigger screen on the current or future iPhone, among other topics. He said that consumers are buying bigger iPhones, but the panel still preferred iPhones with smaller screens because they fit better in men’s pants pockets.

The panelists also talked about two interesting new products that they saw at the expo, BearExtender Edge and the Ring. The BearExtender Edge is long range Wi-Fi repeater/ booster that works with Macs, iPad/iPhone, and other devices (PCs with Wi-FI, other phones or tablets with Wi-Fi). The one-piece Wi-Fi repeater block plugs into an AC outlet and has an antenna that repeats/ boosts the Wi-Fi signal in the area. It does not require software or USB connector.  BearExtender also makes a high power standalone USB Wi-Fi radio adapter with an external antenna that increases the range of a Mac’s Wi-Fi signal by nearly four times that of an internal AirPort card. It adds Wi-Fi to older Macs or Windows PCs that do not have a built-in Wi-Fi adapter.

Paul and the panel were very intrigued by a new product called the Ring from Logbar, Inc., a San Carlos, California-based company.  It is a wearable technology product that fits on a finger as a finger ring.  The new Kickstarter funded device transmits hand movements or gestures to an iPhone or other Bluetooth enabled devices. The Ring works as an in-the-air pointing device that drew a largish crowd around its booth. A spokesperson from the Asian firm that is making the device suggested that the Ring is still in beta-test.

ThermoFisher Aims to Get FDA OK for its Ion Torrent PGM

By Paula Myers

At this year’s CHI Molecular Medicine Tri-Conference at the Moscone Convention Center, San Francisco, February 11th, there were over 3000 attendees and more than 200 exhibitors. I visited some of the many booths on the exhibit floor. One of those was the ThermoFisher booth. I spoke with Zhen Mahoney,Sr. Clinical Sales Specialist/Pharma Business. Mahoney talked about the acquisition of Life Technology and how it affects ThermoFisher.  Thermo will have to absorb Life’s 9,000 employees. Thermo currently has 40,000 employees. Mahoney pointed out that Life has a broad product portfolio. Ion Torrent, which Life acquired in 2010, is located at Oyster Point near South San Francisco. The rest of company is located at Carlsbad, California, near San Diego, including the Invitrogen and Gibco brands. These groups are staying intact, according to Mahoney.

She also pointed out that the company submitted an application for 510K marketing clearance to the FDA for its Ion PGM system for use as a diagnostics medical device. They are hoping for a 3 to 4 month approval timeframe. By comparison, the Illumina MiSeq took about 9 months to get FDA 510K approval because it was the first of its kind. The Illumina MiSeq received a relatively fast approval because the company worked closely with FDA reviewers so that they can understand its technology. The MiSeq platform serves as a template to the FDA for follow-on platforms from Illumina and other desktop sequencer vendors. Mahoney said that the next revision for their Ion Torrent electronic P-2 Chip is coming later in 2014. It will have 660 million wells.

Some of the other firms that I visited included: Guardant Health, Diagenode, and Epitomics. I focused on companies involved in epigenetics. Guardant Health is a two-year-old service company for individuals. In February, they released GUARDANT360, the first pan-cancer blood test that provides doctors with real-time genetic information to help them prescribe the right treatments for their cancer patients.

  Diagenode is a company originally from Belgium and sells Japanese made disruptor shearing machines such as the Bioruptor Pico for DNA, chromatin, and RNA shearing. Epitomics, located in Burlingame, California, offers custom antibody services for use in epigenetics research. Li Fang, Project Manager of Custom Antibody Services at Epitomics, said that Abcam is buying the company. Abcam is a supplier of antibodies,proteins, kits and reagents.

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.”

Translating Medicine from Civilian Life to the War Zone and Back Again.

I recently viewed a PBS documentary tv show called ‘Battlefield Medicine’ from the BBC hosted by a UK doctor from the UK’s NHS to see what is going on in wartime medicine that might be helpful back home in the UK.  He travelled all thje way to a medical base in Afganistan to see first hand of how doctors there save the lives of wounded soldiers.  I noticed that there are some new methods and technologies being used to help reduce the death rate of soldiers suffering from some horrific trauma injuries from gunshot, shrapnel and IED explosions.

Injured soldiers can bleed out 5 litres of blood in 5 minutes.

Solution: comrades in the field are trained to use nylon tourniquets that they all carry to stop bleed outs from arm and leg wounds. The do so in the 1st 10
mins. after a soldier is wounded.

Solution: airlift helicopter now comes with a Dr and nurse on board are prepared for incoming injured soldiers, they can do  in-air surgury, transfusions, etc. Special blood infusion technology devices was created to infuse blood into shoulder bone and or sternum in chest.

Solution: At trauma hospital ER, bleeding patients are given a 50-50 mix of whole blood and plasma rather than just whole blood because more plasma is needed to make clots faster to stop bleeding. Localized pain drip meds target nerve via catheter that connect to injured area rather than treat whole body with morphine.

The Dr traveled to the USA to an Atlanta hospital ER to learn about a clinical trial that uses progesterone to increase blood flow in patient brains that suffered from traumatic brain injury (TBI).  Rat studies showed that brain damaged rats given progesterone had less dead tissue areas than brains of untreated rats (controls).

The Dr traveled to a Pittsburg med. center and learned that ER is using extreme cold water (saline) infusion (10 deg C) to put patient in coma-like condition for nearly 1 hr (rather than 5 mins.). This procedure suspends activity in brain and heart while surgeons work in patients trauma issues. When stabilized, patients blood is re-infused to bring back body temp to normal range.
The UK Dr. concluded that advances in civilian trauma medicine often is used in military medicine but is modified to fit the field hospital situation.
Knowledge gained from the military applications can often be used in civilian trauma medical applications.  The UK Dr working with the UK NHS hopes to see
some of the military trauma procedures and technologies that he has seen would someday help save more lives in the civilian trauma ER situations such as car crash victims.

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