Companion Diagnostics: A Guide to Cure?

There is an obsession by biotechs, pharma, investors and payers to pre-select responders to a therapy before they are treated. How did we end up here and how is this going to impact medicine in the long term? In certain diseases where the biology landscape is understood better, like oncology, there has been tremendous impact. However, in most other target areas we still cannot isolate the responders and use “all comers” to try treatments and hope the safety and efficacy of a drug can be shown to be statistically significant. What happens to target areas where specific responders cannot be identified? What happens to companies that have an effective treatment but cannot get funding because they cannot find their “targeted population”? By “targeted” we mean a sub-population that expresses a specific genetic mutation or biomarker. At Novateur we are often approached by creative scientists that have discovered a biomarker diagnostic or in some cases can analyze hundreds of your markers to show that you may potentially have various disorders. Would the doctors be interested to tell you that you may have early Alzheimer’s when there is no treatment available for it? And would the payers pay for it? In our experience, diagnostics will have a challenging time getting funded and partnered, unless they can be paired with an effective therapeutic. The ultimate goal is to be able to utilize the diagnostic to change the clinical outcome in a meaningful way. In a similar fashion, biotech companies developing treatments need to think how to incorporate potential diagnostics to pre-select responders. After decades of experience at large pharma, our advisors believe...

Forgoing Retirement: Why I Became an Advisor at Novateur Ventures

I have had a successful career in pharmaceutical development and operations and I was about to retire but decided not to. So why am I an Advisor at Novateur Ventures? I just saw a quote from astrophysicist Neil Degrasse Tyson and this answers part of the reason for my motivation. “When I think of science and scientists, I think of kids who never lost their curiosity and wonder, and then woke up one day as adults with the same sense of search for what is and what is not true in the world.” As a adolescent one has to select a direction for study and a potential career. Hopefully, the choice is something that ignites a spark inside rather than just a means to make a living but everyone makes individual decisions based on many factors. As a child, I helped my Dad take apart and fix just about every thing around the house that needed to be repaired. Starting out as “curious observer”, advancing to “assistant and helper” handing over tools and finally to “repair partner”. My curiosity caused me to often forgo typical childhood pursuits and yes, it had my mother concerned. Later I became fascinated with nature and biology and how living organisims work, and how they interact with the world around them. I remember being intrigued by how some cells in plant seeds turn into stems that grow upwards and other cells become root hairs and grow downwards. Also, how can you swallow an aspirin and your headache goes away? This got me hooked on medicine and the human body. Working in the pharmaceutical science field for over thirty years, I know...

Entrepreneurial Pharma: Confronting the Adage, “Experience Is Something You Get Just After You Need It.”

During my 23 years working in the entrepreneurial pharma environment I have witnessed many lessons learned and I continue to see many companies focusing primarily on reaching key funding milestones while neglecting good drug development practices. This is a mindset that can cost them dearly and deliver experience after it’s needed! It is crucial for entrepreneurs to focus on creating a strong value proposition for the company, attracting the top investors and scientific advisors and moving quickly to key milestones and the additional funding they provide. It’s the nature of the environment. However, at the same time, pharma development requires early and consistent compliance with regulatory guidelines and quality assurance standards and exemplary collaboration skills to enable critical preclinical and clinical trials. The paradox is the need to move quickly AND carefully comply with regulatory requirements and solve technical challenges. This can create confusion when weighing the “must haves” and the “nice to haves”. Often, many important elements are considered “nice to have”. Many entrepreneurs have trouble discerning which is which. Usually, a lack of time or staff or cash is reason for projects moving ahead without the “must haves” like proper documentation or quality checks. In addition, the outsourcing environment is something that is both art and science, something not fully appreciated by all entrepreneurs. Staffing at outsourcing firms varies in expertise, especially in the areas of regulatory strategy and documentation. This requires experienced oversight by the sponsor company.   Here are some real-world examples of my observations of companies that failed to focus on the fundamentals and paid the price: When I was working as and independent...

Conquering the South Pole and Lessons for Biotechs

How the Norwegians conquered the South Pole while the well funded and well resourced British all died on the way back & how that applies to biotech   December 14th, 2016 marked the 105th anniversary of the first expedition to the South Pole by the Norwegian Roald Amundsen, trained by the Inuit in Canada. Famously, the second place finishers, a British team led by Robert Scott, showed up at the South Pole 34 days later. Despite starting the race much earlier with significantly more men and resources, the Brits not only faced defeat, but also died on the return journey. This race in many ways reflects two different business models. What we can learn from it is especially applicable to biotechs and pharma. Why would the underdog end in triumph and the other well-stocked team in tragedy?     The Nordics Were Coached by the Inuit – Experience Matters Amundsen had just completed a 3 year journey successfully crossing the Northwest Passage. During that period he spent many months stuck in ice, so he had a lot of downtime to learn from the Inuit how they kept alive in extreme conditions. Thus, the Norwegian came to the southern hemisphere with what was then the cutting-edge in polar technology: Dog sleds, igloo-building techniques and loose-fitting fur parkas. Amundsen’s subject matter experts (the Inuit) were like a team of experienced pharma drug developers who know the latest drug development techniques and the regulatory/business development/reimbursement landscape. Trekking to the South Pole is like trying to get a new drug or device to approval; it is not something you want to learn “along the...

Trends at the FDA in Drug Approvals

Dr. Thomas Stephens delivered a speech at the LifeSciences BC McCarthy Spotlight Speaking Series event in Vancouver, BC on November 17, 2016. The speech was focused on the trends at the Food and Drug Administration regarding drug approvals.   View and download the full presentation View and download the full presentation   About the Author Thomas W. Stephens, Ph.D. has 28 years of pharmaceutical development preceded by 12 years of in vitro diagnostic experience with emphasis in research, regulatory, project management and administration. In his spare time Tom pursue various interests including astrophotography, underwater photography, swimming, gardening and...

How Would the Biotech Landscape Change After the Election?

This is the 10th US Election I am following personally. I remember US elections going back to 1980 (Carter vs Reagan) at a time when I was attending a kindergarten in Charlotte, North Carolina and Iran hostage crisis was unfolding. The last time I remember a contested and controversial US election was between George W. Bush and Al Gore of Election 2000. This year’s Election has taken everything to a new level (high or low) depending on what you’d be measuring. Besides all the rhetoric and unbelievable factoids we are learning about candidates everyday, we wonder how this election would impact the FDA, reimbursement and generally the life sciences sector. As many of you know, the head the the FDA (the Commissioner) reports to the Secretary of Health and Human Services who reports to the President and is part of the President’s cabinet. Both are appointed by the President and confirmed (or not) by the Senate. So the choice Americans make on the president impacts who he or she chooses for the head of the FDA and the direction FDA would take. Also, we are entering unchartered waters in the US regarding the reimbursement debate. There has not been another time in the US history where drug pricing and the value of new medicines have been so controversial and debated. In my opinion, the few bad apples (Martin Shkreli, Mylan and others) are giving the whole industry a bad reputation. At a BIO CEO/Investor Forum last week in San Francisco, a number of protestors interrupted the plenary lunch session and prevented many of us from attending the session. Drug...