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.

 

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.”

Bio Investor Forum Day 2 — Biotech IPOs

–San Francisco, the Palace Hotel, Bio Investor Forum Day 2, October 9, 2013. The meeting wrapped up with its Closing Plenary: Early Stage Venture Financing — Will Current Trends Continue in 2014? The Plenary was structured as a panel discussion that included moderator Luke Timmerman — Xconomy, Srini Akkaragu — Sofinova Ventures, Brian Atwood — Versant Ventures, Alexis Borisy — 3rd Rock Ventures, Maria Chavez — AbbVie Biotech Ventures, Andrew Schwab — 5AM Ventures.  LukeTimmerman opened the discussion by saying that the big story of  2013 is the boom in biotech IPOs.  He said that in the last few years many VCs have dropped out – about half of the VCs became vampires.

The discussion opened with comments from the panel about the string of biotech IPOs that emerged in 2013. They seemed pleased that there were IPOs and wondered if the situation was sustainable into 2014 or beyond. Others commented that a biotech IPO is at an early part of a long series of steps to make a drug and that they have seen this process over and over.

Some commented about the apparent high quality of the recent crop of companies, saying that a lot of the areas in biology have a lot of unknowns and is really risky.  Companies that are able to pick out the small pieces that are well known can go forward. The advances in biology amassed over the last twenty years has enabled many of the biotech companies to do IPOs.

As the discussion continued, the topics ranged the share of VC money invested in companies, to partnerships, syndication, and the short list of “go to” financial and VC companies.  They also spoke about crowd funding, YouTube, using big data for biomarkers.  They see change coming with Accountable Care Organizations, and reimbursement issues.

From what I could see, we can expect to see more biotech IPOs in 2014.

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