The vaccine market is booming – and biotech companies are just one of the beneficiaries.  A recent Associated Press Article explains that an increase in government support and newer, better technologies are just a part of it.  Patients look to benefit from new vaccinations on the horizon for diseases such as Alzheimer’s, herpes, malaria, and perhaps even a universal flu vaccine.

It was just last month at the BIO Investor Forum’s H1N1 panel that panelists discussed the rise in vaccine manufacturing and the potential that market holds for growth.  While the H1N1 pandemic has certainly pushed many manufacturers to look into new techniques and processes to manufacture vaccines more quickly and in greater quantities, an increase in support and innovation could provide some new opportunities to biotech and pharmaceutical companies looking to expand into this growing field. For more, check out BioWorld Insights’ October 26 article by Randy Osborne, “Vaccine Flu Cash Flying Around: Picking Winners in H1N1 Tricky.”

Part I

Dance of the Great Egret (Part II)
14 September 2009
Dune Road, East Quogue?, NY

Som bekendt har vi desværre måtte flytte arrangementet med Don Ø fra den 29. oktober til den 23. november.

Det foregår samme sted, nemlig det store auditorium på Julius Thomsens Plads og stadig kl. 15 til 17. Tilmeldingen sker også stadig via www.nbbc.dk, hvor mange allerede har tilmeldt sig. Vi glæder os til at se mange af jer i selskab med en af dansk erhvervslivs store direktører.

Og nu er jeg ikke den store fodboldekspert – men der er da noget galt på nedenstående billede, er der ikke?

SFIP is pleased to welcome its new alliance partner, the Business Innovation Factory.  Based in Providence, RI, BIF is a national leader in systemic and business process innovation, hosts  a top-rated annual thought leaders summit, and is focused on the elder care, energy, and education areas.  Together, BIF and SFIP plan to launch the Code Green Energy Innovation Laboratory, a user-centered residential consumer lab for new energy products and systems.

Story by Nate Shanklin

The Business Instructional Facility (BIF) was awash with orange ties and blue pinstripes for the dedication this past Friday.  The Marching Illini provided a thunderous introduction as Dean Larry DeBrock served as master of ceremonies.  Dean DeBrock, along with several of his colleges both past and present spoke highly of the effort that brought forth the new home of the University Of Illinois School Of Business.  In a particularly touching moment, the new building’s architect, Cesar Pelli, an Alumni of the University of Illinois, seemed truly taken back at the response that his creation has been generating.  Holding back emotions, he thanked this son, with whom he works, the rest of his staff, and everyone at the university without whom, he said this could not have happened.  As an impromptu reminder of students’ appreciation for the new facilities, and never missing an opportunity to let the Illini pride flow, the students surrounding the lobby on all floors began to chant “ILL” which was quickly met by a resounding “INI” by all alumni, staff, funders, and faculty in presence.  As was mentioned heavily in the opening dedication, BIF with support from both corporate and alumni donors has been built as an incredibly forward thinking, environmentally sound structure.  Due to its innovative construction, BIF will likely receive LEED gold or possibly platinum certification, making it one of the most eco-friendly and energy efficient buildings in America.  The dedication concluded with a ribbon cutting and the vocals of the Illinois Men’s Choir.  Already in service, BIF will now facilitate the needs of the School of Business at both an Undergraduate and Graduate level.

Example of a successful IPO: Targacept filed an S-1 in 2004 after late stage VC rounds, then went on roadshow in 2005 and made a deal with AZ. Only after that ramp up did they go public. Like plowing the fields before planting the seeds, a biotech must develop relationships with future investors before the IPO. To this point, it helps having an analyst on board that can position your company at the forefront of the numerous other micro caps biotechs. The analyst provides the necessary attention that will be required beyond the initial pricing.

Funding the private companies: Many VCs are adapting a “dumbell” approach by investing in either seed stage or late stage companies. The early stage is high risk but requires low capital and the late stage offers visibility and somewhat lower risk but at a cheaper level than in the past. Companies in the middle may be left out.

When the opportunity arises: The IPO is not always the best option for a company due to timing of the company’s development programs. The bankers will pitch the rewards of an early exit today, but CEOs must consider the road a few years out as a public company that is expected to deliver on a conistent basis. Private company CEOs may underestimate the demands put on them once they cross into public hands. In addition, they should make sure the balance sheet is just as ready for the years ahead as the clinical data. And on a human level, make sure you CEO can handle the psychological demands that come with volatile stock price that may be down for extended periods.

4Q09 projection: We should see many more S-1 filing in 4Q.

Positives for the industry (and why IPO interest may return):

Lots of cash at big biotechs to fund smaller companies
Lots of good science and people in these companies
Still many unmet needs and money to be made
Drugs for global diseases and for emerging markets are starting to be addressed
FDA is getting better
There will be another Gilead created

Panelists in the “Making it Personal” session discussed the clinical utility, comparative effectiveness implications and reimbursement for personalized medicine.

Tod Klinger, PhD of XDx, Inc. asserted that we are still in the early stages of personalized medicine since there is a need to determine how to link measurements with relevant clinical information for the patient to enable decision making.

Furthermore, there are many other questions that still need to be considered. Ellen Sheets, MD, of Predictive Biosciences, pointed out that it is generally assumed that patients want to know potential health problems that they may be confronted with in the future. And Pamela Munster, MD of the University of California at San Francisco added that insurers must reimburse for personalized medicine technologies, or they won’t be used anyways.

Alessandra Cesano, MD, PhD of Nodality, asserted that another challenge is that there is not a lot of data on the clinical utility of personalized medicine.

The session was moderated by Ronald Lennox, DPhil, of CHL Medical Partners.

Panelists at this morning’s Therapeutic Workshop, “Predicting a Frontrunner in the Race Against Pandemic Flu” had many valuable insights into this year’s flu pandemic, how it has compared to past pandemics, and how it will shape vaccine manufacturing in the years to come.

According to Dr. Tomás Aragón, Executive Director at the Center for Infectious Disease & Emergency Readiness, “Virulence [of H1N1] is no worse than the regular flu, but a larger risk pool, consisting of pregnant women and younger people, means that there’s higher levels of infection.”

Additionally, Dr. Joseph Miller of the CDC remarked that “the amount of flu we have seen in October is equal to the levels we typically see at the peak of the flu season in the winter.”

Why the hold-up on vaccine delivery?  One big problem has been manufacturing and distribution difficulties.  George Kemble, PhD, with MedImmune remarked,

“There is room for new vaccines, but it’s not easy.  There is a lot of room for the market to grow.”

What does the future look like?  According to Kemble, one option is to move out of the 40 year old technology of using eggs as a means of producing vaccines, which limits not only the amount of vaccine that can be produced but the population which can receive it, and into the use of cell culture production.

Another option is a universal vaccine, which as describe by panelist J. Joseph Kim of Inovio Biomedical Corporation, “would combine the various dominant strains of past flu infections into one vaccine that could be used for multiple years, allowing physicians to stock up on doses each year.”  Such vaccines are currently in pre-clinical trials.

In August, I had the good fortune to attend a workshop by Mary Buckham and Dianna Love based on their great book, Break Into Fiction (hereafter called BIF). I read the book beforehand, and went through the workshop thinking how great all of the templates are because they force you to answer the tough questions about your characters and plot. But, still, I struggled with filling them out. They get into details I wasn’t ready to produce yet.

I had an “Aha!” moment yesterday when I realized that filling out the GMC charts for my characters provided me the macro view of their lives and story that I needed to have in order to complete the micro-focused BIF templates. By completing the GMC work first, I can make sure I’m not spending my time on the BIF templates until I’m fairly sure my story will work.

So, after moving 20K words (ouch!) into my Unused Scenes folder (a topic for another day), I’m pretty much starting over.  But, this time I’m going to try it with the help of the GMC and BIF tools. The great news is that I’m pumped up about my story again. My goal is to have a completed rough draft by January 31. I’ll keep you posted on how it goes.

Chalk it all up to lessons learned and, like Dory says in Finding Nemo, “Just keep moving.”

BTW, if you ever have a chance to take a class from Mary or Dianna, you won’t be disappointed. Both of them are incredibly giving of their time and insights, and will answer endless questions with patience.

Logical Therapeutics, Inc. announced positive results of a Phase I/II clinical trial evaluating the gastrointestinal (GI) safety of its investigational drug LT-NS001, the first of a new class of bio-activated prodrugs being developed for the chronic treatment of arthritic conditions.  

Read the release.

Endocyte, Inc. has been awarded a patent from the United States Patent and Trademark Office covering vitamin receptor binding anti-cancer agents. The patent, entitled “Vitamin Receptor Binding Drug Delivery Conjugates” (U.S. Patent No. 7,601,332), covers novel conjugation linkages and anti-cancer agents, including Endocyte’s EC145, which is currently in development as a potential treatment for ovarian and non-small cell lung cancers.
Read the release.

Oncolytics Biotech Inc. (TSX: ONC, NASDAQ: ONCY) has been granted its 33rd U.S. Patent, No. 7,608,257, entitled “Sensitization of Chemotherapeutic Agent Resistant Neoplastic Cells With a Virus.” The patent claims cover methods of using reovirus in combination with currently approved chemotherapeutic agents to treat patients that are refractory to those chemotherapeutic agents alone.

Read the release.

Biotechnology researchers are working to create new vaccination options to help stop the next big pandemic before it starts.  Join Dr. Targan of BIO’s BioBytes as he walks us through the current steps in vaccine production, and what biotechnology is doing to help create new vaccines in quicker timeframes to help meet the global demand (click link below for video).

For those of you attending the 8th Annual BIO Investor Forum this week in San Francisco, be sure to check out the “Predicting a Frontrunner in the Race Against Pandemic Flu” therapeutic panel on Thursday.  Panelists will discuss the challenges presented by H1N1 and focus on the companies currently working on vaccine development.

BioBytes: Biotechnology and the Flu Pandemic

Panelists in this afternoon’s business roundtable “Window Shopping – The Buyer’s Perspective on Today’s IPO Candidates” contemplated what it’s going to take to return to a vibrant IPO market.

“What we need is public market examples of IPOs that are successful,” said Ashley Dombkowski, PhD, a managing director at MPM Capital.

Daniel Lyons, PhD, CFA, an equity research analyst at Janus Capital added that having a lack of opportunities can make a company stand out even more than usual.

Targacept went public in 2006 and provides a good example of a company that met expectations after going public. But Alan Musso, vice president, CFO and treasurer of Targacept asserted that the company had a long road to going public. Musso added that his company focused on telling their story, saying that ‘without analyst coverage, you become an orphan out there.’ Timothy Rodell, MD, president and CEO of GlobeImmune, Inc. agreed and added that it is incumbent on companies to talk to customers as well.

Bryan Roberts, PhD, a partner at Venrock, put it into simple terms by stating that ‘if the product works, it’s a good investment, but most of the time, the product doesn’t work.’ 

Christopher Earl, PhD, former CEO of BIO Ventures for Global Health moderated the panel.

As a breast cancer advocate, I am seduced by the lure of translational research and the promises of personalized medicine that will make the one size fits all approach to cancer treatment obsolete and possibly reduce over-treatment, toxicity, and treatment errors.

I will be attending the BIO Technology Transfer Symposium and BIO Investor Forum next week and will look forward to hearing insights from biotech industry leaders. The Tech Transfer Symposium will address opportunities and challenges of biotechnology licensing. Personalized medicine will be the topic of a Therapeutic Workshop on Thursday morning that will focus on the molecular and genetic basis of disease that are driving new product opportunities and categories in patient diagnosis, prognosis, monitoring and treatment. 

Genomics opens the window highlighting the heterogeneity of breast cancer subtypes. Molecular spectrum and gene assays offer promising predictive and prognostic profiles, and pharmacokinetic drug targets have shown considerable potential (i.e., Cyp2D6 variants and the choice of optimal adjuvant endocrine therapy).

Many recent advances have been made characterizing molecular mechanisms, determining molecular insights into metastasis, and developing interventions/applications in the clinic. 

Although I am thrilled that innovation is headed in this direction, I also realize that translational research scientists and clinical investigators face many struggles:

• Developing tools that will identify women who will most benefit from therapy—For example, taxane modestly improves anthracyline therapy, we are still uncertain which subgroups of patients benefit from taxanes
• Integrating cutting edge technologies as early measures of efficacy
• Identifying and validating biological and genetic markers for tumor response and patient toxicity to better discriminate minimal risk from high risk disease and personalize therapy– Even when a clearly defined population of HER2 positive patients has been identified, we remain uncertain of the optimal duration and schedule of trastuzumab administration
• Interpreting indeterminate and incidental findings, and the metrics used to measure clinical impact
• Managing the unrealistic expectations of desperate patients who are motivated by the hope of therapeutic benefit even when potential benefit seems questionable or unlikely.
• Sharing technologies across disciplines and developing new frontiers for public-private partnerships, data sharing, and biospecimen accrual.
• Bringing a drug to market – Currently, the majority of the 90 Phase I & II breast cancer clinical trials will fail FDA scrutiny.  Meeting the responsibilities of drug approval and personalized medicine in a fragmentary and flawed regulatory environment is one of the biggest challenges of experimental therapeutics. 

Despite the long and winding road ahead, I have the confidence that serious technological and cost barriers can be surmounted by a strategic focus on collaborative translational research approaches emphasizing high impact biomarker development and qualification as a new means for diagnosing, preventing, targeting, and treating disease.

In this arena of challenge and opportunity, I and groups of advocate stakeholders nationwide are asserting claims as credible participants who in partnership with academic investigators, industry, medical research foundations, and non-profits wish to influence the revamping of research direction and practices, so that progress might be accelerated in the fight against cancer.

We  suggest changes in research priorities and culture by:

• Championing adaptive pragmatic trial design and non-profit disease organization research support 
• Embracing evidence based practices: distinguishing hope from hype in a regulatory arena
• Promoting patient centered priorities:  rethinking communication dissemination and consent practices
• Reinvigorating transparency in recruitment: reframing ethical priorities for urging rapid patient enrollment into clinical trials
• Reassessing Economic Support for Clinical Trials

Advocates have become involved in various types of involvements over the past decades and we can be proud of our contributions. However, much of the progress in the acquisition of cultural competence and representation on national cooperative groups, workgroups, and panels, should not be overstated.  Many of the steps taken have been notable–albeit modest baby steps, and there is still much more work to be done.

We all know that advocacy has the highest praise when the goal is to support.  Although it is less appealing when the goal is to “act up” and critique, I hope that all biotech investor stakeholders remain open to the nuances of advocacy dialogue, openness, and candor even when inconvenient.  Advocates are credible allies who share their passion as trustworthy participants in the translational research process.

Our participation in translational science will enhance the democratization of knowledge, and our interactions and brokerage with scientists and other stakeholders will lead to further needed reforms in research practices, regulatory policies, and interpretative mechanisms for evaluating the metrics of survival outcome and clinical impact.

We face challenging times and a needed paradigm shift in translational design, conduct and support requires all of us to adopt a renewed commitment to the partnership between investigators, industry, patients, advocates and their communities; a partnership based on social responsibility, authentic collaboration, and transparency; a partnership that promotes new products, reduced development costs, and faster product cycles.

 Susan Samson, UCSF Breast  SPORE Cancer Program  Advocate

Of course, the egret here is not really dancing, but landing in the marsh after circling the same pool filled with prey.

Dance of the Great Egret
14 September 2009
Dune Road, East Quogue?, NY

The Burrill Report’s Daniel Levine spoke with Jim Greenwood, president and CEO of BIO, about the upcoming Eighth Annual BIO Investor Forum’s opening plenary session on healthcare reform. Greenwood will be moderating the session, “Healthcare Reform: Sustaining Biotech Innovation in a New World.”

Listen to the podcast

Oncolytics Biotech Inc. (TSX: ONC, NASDAQ: ONCY) invested in British Canadian Biosciences Corp., a privately held biotechnology company specializing in the development of peptides for the treatment of a variety of conditions, including cancer. Oncolytics has purchased all of the convertible preferred shares of BCBC in exchange for 200,000 common shares of Oncolytics. If converted to common shares, Oncolytics would own 10% of the outstanding BCBC common shares. In addition, Oncolytics has obtained a right, under certain circumstances, to purchase the rights to BCBC’s oncology product which is entering Phase II studies for use in solid tumours. The purpose of the transaction is to gain access, at a future date, to a potential new oncology product. Read the release.

Biotech Now’s Tracy Krughoff spoke with Dr. Tim Coetzee, president of Fast Forward, a subsidiary of the National Multiple Sclerosis Society. Fast Forward will be one of the advocacy organizations presenting at the Eighth Annual BIO Investor Forum. Listen now! http://www.bio.org/podcasts/coetzee.wma

Read a recent interview with Complete Genomics Chairman, President, and CEO Clifford Reid about his company’s top priorities, what makes his company attractive to investors, and what he hopes to accomplish at the 8^th Annual BIO Investor Forum. 

Complete Genomics sequenced its first genome in early 2009 and that data is publically available in the National Center for Biotechnology Information (NCBI) database. Already, in 2009, Complete Genomics has sequenced and delivered genomes to important collaborators in academic, pharmaceutical and government research institutions. In 2010, the company intends to sequence 10,000 genomes. Complete Genomics’ mission is to become the global leader in human genome sequencing. It is currently building the world’s largest human genome sequencing center in California. Further expansion is planned by opening sequencing centers worldwide.

A year ago, CombinatoRx probably wouldn’t have guessed a multi-million dollar merger was on the horizon.  However, after crossing paths with Neuromed at the 2008 BIO Investor Forum, the company found itself en-route to a July 2009 announcement of a $30 million dollar merger.   The deal is expected to close this quarter.

Read the full story at TheDeal.com.

Cure Duchenne's Debra Miller

Biotech-Now’s Tracy Krughoff spoke with Cure Duchenne’s President, Debra Miller. Cure Duchenne will be one of the patient advocacy organizations presenting at the Eighth Annual BIO Investor Forum next week. Listen Now! http://www.bio.org/podcasts/miller.wma

Novavax, Inc. (Nasdaq: NVAX) initiated a two-stage clinical study of its virus-like-particle (VLP) H1N1 influenza vaccine in Mexico in collaboration with Avimex Laboratories (Avimex) and GE Healthcare. Avimex distributes biological and pharmaceutical products for use in Mexico and more than 25 other countries around the world. Avimex is providing financial support for the trial and is expected to distribute the H1N1 VLP vaccine in Mexico in 2010 if it is approved for commercial sale. In addition, Novavax also announced today that GE Healthcare (a unit of General Electric Company (NYSE: GE) has agreed to support this program by providing its single-use bioprocessing technologies for vaccine production.

Read the release.

Panelists agreed that it’s been a long and complicated debate and the end of year deadline is ambitious yet within the realm of possibility. Michelle Easton, a partner at Tarplin, Downs and Young, LLC, asserted that the State of the Union address may represent a secondary deadline to hope for, if the end of year deadline is not met. Darren Willcox of Dutko Worldwide emphasized that the ‘constant surge of deadlines’ is not helpful to anyone, considering the importance of sweeping changes to the health care system.

Dr. David Bowen, who is a staff director for the HELP committee, pointed out that when a bill is signed, some changes – such as insurance reforms, coverage of preventive services and lifetime limits – will change in “short order” while other changes will take longer to implement.

Panelists discussed the importance of affordability in terms of effectively implementing changes to the health care system. Rick Weissenstein, a senior vice president and healthcare services analyst at the Washington Research Group, asserted that affordability is important to Democrats and Republicans alike.

Jim Greenwood, BIO’s President and CEO moderated the panel via satellite from Washington.