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# Drug Baron

Drug Baron - news and opinions on life sciences from leading uk consultancy

## Sitemaps

- [XML Sitemap](https://drugbaron.com/sitemap.xml): Contains all public & indexable URLs for this website.

## Posts

- [Lessons from Monte Carlo Models: Why Drug Development is Hard](https://drugbaron.com/lessons-from-monte-carlo-models-why-drug-development-is-hard/) - “There is more than one way to skin a cat” is a rather gruesome British idiom, but its sentiment surely applies to running a successful pharmaceutical portfolio. It is now more than a decade since Francesco De Rubertis, together with Kevin Johnson and Michele Olier, coined the term “asset-centric” investing to describe the approach to portfolio
- [Claims that AI will “revolutionise pharma R&D” are (almost entirely) hype](https://drugbaron.com/claims-that-ai-will-revolutionise-pharma-rd-are-almost-entirely-hype/) - This month’s announcement that Alphabet’s Isomorphic Labs, led by the talented and charismatic Deepmind co-founder Demis Hassabis, had inked discovery collaboration deals with both Lilly and Novartis was just the latest indication that AI-enabled pharma R&D was ready for the big time. Billions of dollars have been invested in companies promising to “revolutionise pharma R&D” through
- [A quite revolution in the world of proteomics](https://drugbaron.com/a-quiet-revolution-in-the-world-of-proteomics/) - Proteomics – examining the panoply of proteins within a complex sample – has been around much longer than the word we use today to describe it. Proteomics the word was coined in around 1997 as a portmanteau coupling the now-ubiquitous “omics” suffix to the word protein. But two decades earlier two-dimensional gel electrophoresis was the cutting-edge technology
- [#Unshackled: The evolving definition of asset-centricity](https://drugbaron.com/unshackled-the-evolving-definition-of-asset-centricity/) - Asset-centric is a term coined by Medicxi founding partner Francesco De Rubertis to describe an investment strategy he pioneered, together with Kevin Johnson and Michele Ollier, in the early years of the 21st Century. The key was focusing investment on a single asset to avoid the problem with pipelines in early-stage biotech companies. While it seemed superficially attractive
- [How to find a drug: the past, present and future of small molecule drug discovery](https://drugbaron.com/how-to-find-a-drug-the-past-present-and-future-of-small-molecule-drug-discovery/) - Despite the current hype around so called “advanced therapies”, which range from gene editing to cell therapies, and the inexorable advance of biologic therapeutics such as monoclonal antibodies, even in 2022 the majority of drugs in development and reaching patients are still small organic molecules. From enzyme inhibitors to receptor antagonists, allosteric modulators to suicide
- [Re-Imagining Med Chem Strategies: the Tyranny of the n+1 Compound](https://drugbaron.com/re-imagining-med-chem-strategies-the-tyranny-of-the-n1-compound/) - Finding small molecule drugs is much harder than finding a needle in a haystack – discovering the right arrangement of atoms to bind precisely to a protein target to elicit a particular response is a problem of vast dimensionality. We are most familiar with the numbers involved when dealing with antibodies: a typical antibody library
- [An Open Letter To FDA Regarding Eteplirse](https://drugbaron.com/an-open-letter-to-fda-regarding-eteplirse/) - Dear FDA, I am writing to you about the forthcoming meeting to consider the marketing application for eteplirsen, a morpholino-oligo that induces exon-skipping to generate functional dystrophin protein and therefore, it is hoped, improved outcomes for patients with Duchenne Muscular Dystrophy (DMD). I should, before continuing, declare my conflict of interest. DMD is a terrible
- [Why Small Beats Big: the Hidden Cost of Too Many Voices](https://drugbaron.com/why-small-beats-big-the-hidden-cost-of-too-many-voices/) - The coronavirus pandemic has taught us a lot of lessons. But some, such as exactly why small biotech companies are increasingly making all the running in early-stage drug discovery and development, are slightly less obvious than others. Just this week, we have seen the UK Scientific Advisory Group for Emergencies (SAGE) urge the Government to slow the
- [The Hidden Superpower of Strategic Focus](https://drugbaron.com/the-hidden-superpower-of-strategic-focus/) - The first death unequivocally caused by COVID was reported to the world on January 11th 2020 – that's just 303 days ago (even if it feels like a lifetime away). And yet, with interim data announced today showing a vaccine candidate from Pfizer and BioNTech to have at least 90% efficacy, it seems certain a vaccine to protect against a virus
- [Cellular social networks give cancer cells a cloak of invincibility](https://drugbaron.com/cellular-social-networks/) - Ever since DrugBaron studied biochemistry at the end of the 1980s, one question about cancer has always perplexed him: why can we induce almost complete remission and yet the cancer can come back stronger and invariably kill the patient? The answer now, as then, has always been selection of the rare, resistant mutant. But DrugBaron
- [The history – and the future - of antibody discovery technologies](https://drugbaron.com/the-history-and-the-future-of-antibody-discovery-technologies/) - Monoclonal antibodies are now well-established as a mainstay of the therapeutic armoury. More than a hundred antibodies have now been approved as drugs, making up around a third of all approvals over the last three years. This reflects a number of inherent advantages of antibodies over “conventional” small-molecule drugs: their inherent specificity allows off-target toxicities
- [Why Sarepta's most recent failure in DMD was entirely predictable](https://drugbaron.com/why-sareptas-most-recent-failure-in-dmd-was-entirely-predictable/) - Yesterday, Sarpeta (NASDAQ: $SRPT) announced that its gene therapy for Duchenne Muscular Dystrophy failed to improve muscle function in a study of 40 boys, despite achieving impressive expression of micro-dystrophin (at least in the short term). However, you spin it – and this company is world-class at spinning a story – that represents a serious blow.
- [One dose or two? What the debate about COVID vaccination teaches us about science – and its limitations](https://drugbaron.com/one-dose-or-two-what-the-debate-about-covid-vaccination-teaches-us-about-science-and-its-limitations/) - Over the past week a furious debate has broken out over the merits of delaying the second dose of the approved COVID vaccines, to increase the number of people who can be given a single dose. The case against doing so is clear: the labels of the approved vaccines are unambiguous that two doses should
- [COVID19: Serology is harder than it looks](https://drugbaron.com/covid19-serology-is-harder-than-it-looks/) - More than a month after the World Health Organisation declared COVID19 a global pandemic on 11th March we are still missing one crucial piece of data from our modelling: the fraction of people who have established productive immunity against the SARS-CoV2 virus. While lockdowns may slow the rate of viral transmission in the short term,
- [Even scale-up chemistry is not "just cooking"](https://drugbaron.com/even-scale-up-chemistry-is-not-just-cooking/) - Ask star antibody developer and Medicxi partner Kevin Johnson to name the biggest challenge in bringing a new antibody into the clinic, and his immediate answer has only three letters: CMC. Everyone knows how big a challenge it is to get the manufacturing piece right for an antibody. And many promising projects stumble due to
- [Forget about greed, President Trump: the villain responsible for high drug prices is inefficiency](https://drugbaron.com/forget-about-greed-president-trump-the-villain-responsible-for-high-drug-prices-is-inefficiency/) - “I have directed my administration to make fixing the injustice of high drug prices one of my top priorities.” – President Donald Trump 2018 State of the Union address Directing people to fix problems is one thing, but actually fixing them is quite another. And while President Trump may see high drug prices as an “injustice”,
- [The Cult Of DNA-centricity](https://drugbaron.com/the-cult-of-dna-centricity/) - Understanding the role of DNA in biology is arguably the single most important scientific advance of the 20th century. It provides the molecular basis of inheritance, and understanding how the genetic code is translated into proteins providing a whole new insight into the workings of the human body. The fundamental unit of inheritance is
- [Why Too Many Clinical Trials Fail -- And A Simple Solution That Could Increase Returns On Pharma R&D](https://drugbaron.com/why-too-many-clinical-trials-fail-and-a-simple-solution-that-could-increase-returns-on-pharma-rd/) - The rate of late stage clinical trial failures is the single biggest determinant of returns on pharmaceutical R&D. The lion’s share of discovery and development costs come at the end of the process, and if those trials fail (whether for safety or lack of efficacy), all the capital invested up to that point is lost.
- [The Lone Biotech Bear?](https://drugbaron.com/the-lone-biotech-bear/) - Judging from the atmosphere at last week’s JP Morgan Healthcare conference in San Francisco – which industry veteran and long-time participant Henri Termeer described as the “most optimistic” he had every experienced – the industry’s well-documented problems are clearly behind us. After a decade in the doldrums, the biotech market is on a bull run
- [Beta-Amyloid: The Emperor Really Does Have No Clothes](https://drugbaron.com/beta-amyloid-the-emperor-really-does-have-no-clothes/) - Yesterday, Roche (OTCQX:RHHBY) announced its decision to discontinue its Phase 3 study of gantenerumab, its experimental anti-amyloid antibody, in early Alzheimer’s Disease. The study failed a futility analysis – the clearest possible evidence of a lack of signal. This marks the latest nadir for the amyloid hypothesis – the dominant collective understanding of the molecular
- [Is This The Future Of Late-Stage Drug Development?](https://drugbaron.com/is-this-the-future-of-late-stage-drug-development/) - On Friday, Novartis announced that its anti-IL17A antibody secukinumab (Cosentyx™) demonstrated clear superiority over its rival, Stelara™ ustekinumab from Johnson & Johnson, an antibody against IL-12 and IL-23, in the treatment of psoriasis. What marks this out as significant is not the potential commercial impact for $NVS, nor the step-change in efficacy this delivers for
- [Spotting Baby Unicorns](https://drugbaron.com/spotting-baby-unicorns/) - Valuing biotech assets, whether an early-stage private investment in a preclinical molecule or a substantial trade in a public stock, is notoriously challenging. Both the length and the complexity of drug development, with multiple parallel risks, are unusual and extreme compared with most other sectors. This multiplicity of risks, leading to high failure rates,
- [Why clinical CROs hate eCRF systems - and why you should love them](https://drugbaron.com/why-clinical-cros-hate-ecrf-systems-and-why-you-should-love-them/) - Everything from banking to government services, from shopping to gambling has moved on-line in the past decade, yielding huge efficiency gains for suppliers and (for the most part) an improved experience for the customer. Suppliers that have failed to adjust their business model are being slowly (or not slowly) ejected from the marketplace. Against
- [Constructing better multivariate biomarker composites](https://drugbaron.com/constructing-better-multivariate-biomarker-composites/) - The earliest biomarkers, such a body temperature or blood pressure, were single measurements that reflected multiple physiological processes. Today, though, our reductionist approach to biology has turned up the resolution of our lens: we can measure levels of individual proteins, metabolites and nucleic acid species, opening the biomarker floodgates. But this increased resolution has
- [The HDL myth: how misuse of biomarker data cost Roche and its investors $5billion](https://drugbaron.com/the-hdl-myth-how-misuse-of-biomarker-data-cost-roche-and-its-investors-5billion/) - On May 7th 2012, Roche terminated the entire dal-HEART phase III programme looking at the effects of their CETP inhibitor dalcetrapib in patients with acute coronary syndrome. The immediate cause was the report from the data management committee of the dal-OUTCOMES trial in 15,000 patients that there was now no chance of reporting a 15%
- [Environmental Pollutants: Opening a Soup-Can of Worms](https://drugbaron.com/environmental-pollutants-opening-a-soup-can-of-worms/) - They are everywhere: so called ‘present organic pollutants’, or POPs for short. Since almost all the everyday items that make modern life so much easier emerged from a chemical factory, its not surprising that environmental contamination with organic chemicals is increasing all the time – even ‘environmentally aware’ Western countries. But maybe it will surprise
- [The interleukin lottery: playing the odds on numbers 9 and 16](https://drugbaron.com/the-interleukin-lottery-playing-the-odds-on-numbers-9-and-16/) - The interleukins are an odd family. One name encompasses dozens of secreted proteins that are linked by function rather than by structure. And even that common function is very broadly defined: cytokines that communicate between cells of the immune system. Defined in such a way, its perhaps not surprising that the interleukins have yielded
- [Smoke Screen: The intensifying debate about population screening generates more heat than light](https://drugbaron.com/smoke-screen-the-intensifying-debate-about-population-screening-generates-more-heat-than-light/) - If a test with prognostic value exists, should it be used for population screening? On the face of it, it's a simple question, but it doesn't have a simple answer. Like most things in life, it depends on the context: how prevalent and how dangerous is the disease? How invasive and how expensive is the
- [Personalized Medicine Demands Investment in Innovative Diagnostics: Will the Returns be High Enough?](https://drugbaron.com/personalized-medicine-demands-investment-in-innovative-diagnostics-will-the-returns-be-high-enough/) - Several very senior pharma executives were recently overheard by a journalist discussing what each of them viewed as the most important changes in the way healthcare will be delivered over the coming decade. Each of them listed several such factors, including increased payor pressure on prices, the mounting regulatory burden and the shift toward orphan
- [Ultra-sensitive NMR-based diagnosis for infectious diseases: the tortoise races the hare again](https://drugbaron.com/ultra-sensitive-nmr-based-diagnosis-for-infectious-diseases-the-tortoise-races-the-hare-again/) - Obtaining rapid and reliable diagnosis of infectious diseases is usually limited by the sensitivity of the detection technology. Even in severe sepsis, accompanied by organ failure and admission to an intensive care unit, the causative organism is often present at a level of less than one bacterium per milliliter of blood. Similarly, in candidiasis the
- [Chemokines as biomarkers for cancer: Time to revisit an old friend?](https://drugbaron.com/chemokines-as-biomarkers-for-cancer-time-to-revisit-an-old-friend/) - A wide-ranging study pre-published on-line in Nature last month points the finger at the chemokine CCL2 (also known as MCP-1, or JE in mice) as a key regulator of tumour metastasis. Intriguingly, CCL2 seems to participate in the generation of clinically-relevant metastatic disease on multiple levels: it promotes seeding of the shed metastatic cells, but it
- [Finding exogenous biomarkers of heart disease: humans are ecosystems too!](https://drugbaron.com/finding-exogenous-biomarkers-of-heart-disease-humans-are-ecosystems-too/) - It is ten years this week since the Total Scientific team, together with our collaborators at Imperial College in London submitted the first large-scale clinical metabolomics study for publication in Nature Medicine. We applied proton NMR spectroscopy to serum samples collected from patients with coronary heart disease (defined by angiography), as well as control subjects
- [Biomarkers:  lessons from history](https://drugbaron.com/biomarkers-lessons-from-history/) - The increase in the use of the term biomarker is a recent one. When one looks back at the use of this term in the literature over the last fifty years, there was an explosive increase in its use in the 1980s and 1990s, and it continues to grow today. However, biomarker research as we
- [The final frontier - post-genomic biomarkers](https://drugbaron.com/the-final-frontier-post-genomic-biomarkers/) - Some biomarkers are easier to find than others. Once a class of molecules has been noticed, and the assay methodology to measure their levels has been optimized, data rapidly accumulates. Related molecules frequently pop up (often as a result of artifacts appearing in the assays under certain conditions or when particular samples are analysed). Its
- [Biomarkers: A Band-Aid for Bioscience](https://drugbaron.com/hello-world-2/) - In this kick-off article to the new Total Scientific biomarker blog, we discuss the potential for biomarkers to improve the R&D productivity of the pharmaceutical industry. Please read the full article bandaid_blog1. Read the original article here
- [FDA guidance on the use of biomarkers as drug development tools](https://drugbaron.com/fda-guidance-on-the-use-of-biomarkers-as-drug-development-tools/) - Back in September the US Food and Drug Administration announced that it was going to delay its publication of draft guidance on the qualification of drug development tools, originally promised for the summer. However, this draft guidance was finally published at the end of October. While still in draft form, the Guidance substantially expands on
- [Biomarkers:  standing the test of time means good initial study design](https://drugbaron.com/biomarkers-standing-the-test-of-time-means-good-initial-study-design/) - It feels like every other day that another putative biomarker is identified that will predict presence or extent of some disease or another, usually with an absurdly low p value. So, if these biomarkers are so common, why is the subsequent commercialisation and clinical use of these potential diagnostics so difficult to achieve? I
- [Anti-PCSK9 Drugs: An Ingenious Solution For A Problem That's Mostly Already Solved](https://drugbaron.com/anti-pcsk9-drugs-an-ingenious-solution-for-a-problem-thats-mostly-already-solved/) - On Friday, for the first time, we saw the full data from the FOURIER clinical trial of Amgen’s cholesterol-lowering antibody, evolocumab (Repatha). Depending on who you listen to, it was either “the most important study in at least 20 years” or a damp squib. So what does FOURIER really tell us? And how will
- [Portola's APEX Trial Design Was Too Clever By Half](https://drugbaron.com/portolas-apex-trial-design-was-too-clever-by-half/) - On Thursday, March 24, Portola announced the top-line results from APEX, a large phase 3 trial of the novel oral anticoagulant betrixaban. With two other Factor Xa inhibitors already on the market (and no head-to-head comparison data) the findings of APEX are of more immediate interest to clinical trial designers than patients. The drug
- [Viagra, Melanoma And The Problem With Big Data](https://drugbaron.com/viagra-melanoma-and-the-problem-with-big-data/) - This week saw the climax (or, for those who like to demonize the global pharmaceutical industry, perhaps the anti-climax) of a year-long saga linking an increased risk of melanoma with drugs such as sildenafil (better known as Viagra) used to treat erectile dysfunction. The story began in 2014 when a team of Boston scientists
- [New Data On Alzheimer's Drug Candidates Changes Nothing](https://drugbaron.com/new-data-on-alzheimers-drug-candidates-changes-nothing/) - Sometimes data just speaks for itself. But more often, its left to scientists and journalists to try and draw useful conclusions from complex observations. And so is the case with the latest data reported at the Alzheimer's Association International Conference in Washington, D.C on two clinical-stage drug candidates. Predictably, there has been no shortage of
- [Is The Superiority Of Entresto Just An Illusion?](https://drugbaron.com/is-the-superiority-of-entresto-just-an-illusion/) - Entresto, a combination of an old drug valsartan with a novel neprilysin inhibitor sacubitril from Novartis , which was approved in July for treating heart failure, has been ear-marked by analysts as a mega-blockbuster. Some suggest $10 billion per annum peak sales may be achievable, making it one of the biggest selling drugs of all time. A 20% reduction
- [DMD Drugs: An Existential Threat To The FDA?](https://drugbaron.com/dmd-drugs-an-existential-threat-to-the-fda/) - Last week saw the advisory committee meeting for drisapersen, the last of three new candidate treatments for Duchenne Muscular Dystrophy (DMD) to begin the process for approval in 2015. The outcome is clearly important for patients with DMD and their families. Equally, it is important to the companies who have developed these drugs, and their
- [How Does Meldonium, The Drug Maria Sharapova Took, Work?](https://drugbaron.com/how-does-meldonium-the-drug-maria-sharapova-took-work/) - The news of Maria Sharapova’s failed drug test at the Australian Open tennis championships in January thrust a little-known drug–meldonium–into the spotlight. Almost unheard of outside of Russia, where it is approved for use in cardiovascular disease, the properties of meldonium offer an insight into the mind of a tennis superstar and the team that surrounds her.
- [Cult of Crystallography](https://drugbaron.com/cult-of-crystallography/) - Biological macromolecules, such as proteins and DNA, are very small and very complex. It is something of a technological miracle, therefore, that we can visualise their three-dimensional structures at atomic resolution. The first, and still by far the dominant, method of solving these structures is crystallography. The principal is simple: by packing many millions of
- [A DrugBaron Glossary](https://drugbaron.com/a-drugbaron-glossary/) - To mark the fifth anniversary of this blog, as well as the launch of the new website, DrugBaron has collated some of his favorite phrases, each encapsulating an important concept in pharma R&D. Most of these terms were initially coined by DrugBaron in these pages, but have entered the wider biotech lexicon, and frequently feature
- [The (Statistical) Power and the Glory](https://drugbaron.com/the-statistical-power-and-the-glory/) - The worst possible outcome from a clinical trial is a negative result with a drug that actually works in the chosen indication. These ‘false negatives’ almost invariably destroy real value, and for all but the largest pharmaceutical companies spell the death not only of the programme but also the company itself. In theory, drawing the
- [To be or not to be: The Pros and Cons of the Virtual Biotech Company](https://drugbaron.com/to-be-or-not-to-be-the-pros-and-cons-of-the-virtual-biotech-company/) - It is more than a decade since the classical model of a biotech company ruled unchallenged: the glass-fronted building on the gleaming modern science park, with the best part of a hundred doctoral level scientists working seemingly double shifts to deliver an entire pipeline of high value product candidates. The central principle underpinning the investment
- [Monte Carlo models of drug R&D focus attention on cutting costs - Part 1](https://drugbaron.com/monte-carlo-models-of-drug-rd-focus-attention-on-cutting-costs-part-1/) - There is a theme behind many of DrugBaron’s musings over the last three years: pharma R&D is just too expensive to make economic sense. Given high failure rates throughout the process, including in particular a significant rate of late stage failures when the capital at risk is very high, either attrition must fall or costs
- [What price an orphan this Christmas?](https://drugbaron.com/what-price-an-orphan-this-christmas/) - Tiny Tim wasn't the only orphan to suffer a miserable Christmas at the hands of a merciless Scrooge. The same fate awaits so-called orphan disease patients in the UK. This year, it's the turn of around 400 cystic fibrosis patients, who cannot access newly-approved Kalydeco on the supposedly comprehensive National Health Service (NHS). Its easy
- [The perils of ignoring negative data](https://drugbaron.com/the-perils-of-ignoring-negative-data/) - This week, Eleven Biotherapeutics ($EBIO) announced the failure of its Phase 3 trial of an IL1 antagonist in dry eye. The company indicated they saw no way forward for the product candidate in this indication, which with a little translation, indicates a comprehensive failure of the study (rather than a near-miss on the primary end-point,
- [What Thomas Dimsdale, arguably the world’s first biotech entrepreneur, tells us about drug pricing](https://drugbaron.com/what-thomas-dimsdale-arguably-the-worlds-first-biotech-entrepreneur-tells-us-about-drug-pricing/) - Thomas Dimsdale (born in 1712) was arguably the world’s first biotech entrepreneur. Even before Edward Jenner famously ‘invented’ vaccination in the 1770s, Dimsdale was making money from selling smallpox inoculations. And his customers included Catherine the Great of Russia, whom Dimsdale successfully inoculated in 1769 on a visit to St Petersberg. In return he
- [What the travails of the UK’s largest grocer can teach the pharmaceutical industry](https://drugbaron.com/what-the-travails-of-the-uks-largest-grocer-can-teach-the-pharmaceutical-industry/) - In the five years since DrugBaron posted his first article, entitled “Hard Times, Harder Times”, it seems that in the business of drug discovery and development everything has changed – and at the same time nothing. On the one hand, the ubiquitous gloom that surrounded the industry has lifted, to be replaced with a
- [What Angelina Jolie's Very Personal Medicine Tells Us About Personalized Medicine](https://drugbaron.com/what-angelina-jolies-very-personal-medicine-tells-us-about-personalized-medicine/) - Angelina Jolie told the story yesterday of her decision to have her ovaries and fallopian tubes surgically removed to reduce the risk of ovarian cancer due to the faulty BRCA1 gene she was born with. This follows a similar decision to undergo a double mastectomy in 2013 to reduce the even higher risk of breast
- [“Idea Bubbles”:  The dangers of big theories in low-validity environments](https://drugbaron.com/idea-bubbles-the-dangers-of-big-theories-in-low-validity-environments/) - This week, Pfizer and J&J finally discontinued development of iv bapineuzumab for Alzheimer’s Disease (AD) after a second spectacular failure in Phase 3. That they discontinued the programme surprised almost no-one. Everyone, it seems, is smart after the event, but the potential for anti-amyloid therapies was not always viewed with such negativity. Once upon a
- [PheWAS – the tool that's revolutionizing drug development that you’ve likely never heard of](https://drugbaron.com/phewas-the-tool-thats-revolutionizing-drug-development-that-youve-likely-never-heard-of/) - Late-stage attrition kills returns on pharma R&D investment. One way to reduce attrition is to take less risk – incremental innovation reduces the risk of failing in development, but increases the risk of failing in the marketplace with even bigger (though delayed and less sudden) consequences. The other solution is to address the principle causes
- [“On the way to changing the world, there are no signposts” : the illusory power of precedents](https://drugbaron.com/on-the-way-to-changing-the-world-there-are-no-signposts-the-illusory-power-of-precedents/) - Precedents play a central role in the drug development industry. Clinical trials are based on earlier studies with ostensibly similar drug candidates. Business models are judged against the current landscape. Assets are valued by looking for comparables. Indeed, big pharma consider the institutional accumulation of such experience to be a significant competitive advantage. But
- [D&D On R&D - Lessons For Pharma From The Dungeon Master](https://drugbaron.com/dd-on-rd-lessons-for-pharma-from-the-dungeon-master/) - The discovery and early development phases of pharmaceutical R&D are notoriously challenging. Success (even marked by regulatory approval, let alone meaningful sales) is a distant vision. The cost of prosecuting each project is high, and rises very rapidly as development progresses, yet the vast majority of projects – no matter how compelling initially – eventually
- [The Problem with Patient Power](https://drugbaron.com/the-problem-with-patient-power/) - The democratization of opinion is the dominant social meme of the 21st Century. Driven by the power of the internet and social media, the opinion of everyman, thus aggregated, dominates decision-making like never before – in healthcare, as in almost every other sphere. Jargon like “patient-centric development” and “putting the patient at the centre
- [Twenty six of the best candidate protein biomarkers have no ability to predict Alzheimer's Disease](https://drugbaron.com/twenty-six-of-the-best-candidate-protein-biomarkers-have-no-ability-to-predict-conversion-to-dementia-from-prodromal-disease-2/) - DrugBaron woke up this morning to the latest chapter in the “blood test for Alzheimer’s Disease” soap opera, with this article from the BBC trumpeting the latest study to be published as a “major step forward”. The study behind the headlines was published (unusually for a “major breakthrough”) in the journal Alzheimer’s & Dementia, and
- [Did I say asset-centric? I meant team-centric!](https://drugbaron.com/did-i-say-asset-centric-i-meant-team-centric/) - In many Buddhist traditions there is a dislike of naming things. A name can be very limiting: it often focuses on a particular property of the thing, and as a result ignores all the other infinite complexity of the item or concept in question. Worse, it can lead the listener to assume properties that are
- [Never mind how many, feel the sales potential – the Class of 2013 FDA Approvals](https://drugbaron.com/never-mind-how-many-feel-the-sales-potential-the-class-of-2013-fda-approvals/) - With thirty-nine approvals from the FDA, 2012 triggered a wave of optimism in the pharmaceutical industry. But if 2012 was characterized by an increased number of approvals, the sales potential of many of those new products was underwhelming. For 2013, the number of approvals may have gone down, but the quality (in terms of clinical
- [Even odds-on favourites can lose: lessons from the Prosensa story](https://drugbaron.com/even-odds-on-favourites-can-lose-lessons-from-the-prosensa-story/) - With the details from the DEMAND-III study, the full extent of the failure of Prosensa’s exon-skipping drug drisapersen to arrest progression of Duchenne Muscular Dystrophy (DMD) has become clear. There are few grounds for optimism among the plethora of secondary end-points, no trends, not even a statistically significant post hoc sub-group analysis. Only pages
- [The all new, good old-fashioned, solution to the "replication crisis" in science](https://drugbaron.com/the-all-new-good-old-fashioned-solution-to-the-replication-crisis-in-science/) - As the number of reports highlighting the difficulty replicating academic studies proliferate, the clamour for better frameworks to ensure the repeatability of published science is becoming deafening. But is replication the incomparable paragon it is held up to be? Should the scientific community be wasting resources doing the same things over and over again,
- [Medicalizing biomarkers – the sure-fire road to commercial success](https://drugbaron.com/medicalizing-biomarkers-the-sure-fire-road-to-commercial-success/) - Creating new drugs is a process fraught with risk. The risk involved with discovery and early development is obvious, but increasingly the greatest risk lies at the very end of the process: winning a decent market for your approved product. But history teaches us there is a sure-fire trick to eliminate a large portion
- [An open letter to Congressman Waxman on the subject of drug pricing](https://drugbaron.com/an-open-letter-to-congressman-waxman-on-the-subject-of-drug-pricing/) - Dear Congressman Waxman (and anyone else concerned about the price of Sovaldi™), I write to you in response to your letter to John Martin, Chief Excutive at Gilead, on March 20th this year. With spiraling healthcare costs and government debt at an all-time high, taking aim at high-priced drugs seems certain to win
- [Drug prices are (unnecessarily) high because R&D is (unnecessarily) inefficient](https://drugbaron.com/drug-prices-are-unnecessarily-high-because-rd-is-unnecessarily-inefficient/) - Let me put it in bold text and a large font, lest there be any misunderstanding of DrugBaron’s position: Healthcare costs are manifestly too high and completely unsustainable in the medium term. That includes drug prices. This is not inconsistent with the two recent articles (here, here) on drug pricing, which set out to
- [Cometh the saviour? Casting Valeant’s J. Michael Pearson in a new role](https://drugbaron.com/cometh-the-saviour-casting-valeants-j-michael-pearson-in-a-new-role-2/) - There has been much written about the proposed acquisition of Allergan by Canada’s Valeant – most of it doom-laden. Much of the commentary paints a picture of a post-apocalyptic landscape, with marauding capitalists slashing and burning cherished R&D facilities. And the villains of the piece are Valeant’s CEO J. Michael Pearson, playing Darth Vader
- [After NICE is it time for NASTY? National Academy for the Study of Things that harm You](https://drugbaron.com/after-nice-is-it-time-for-nasty-national-academy-for-the-study-of-things-that-harm-you/) - The United Kingdom's National Institute for Clinical Excellence (NICE) has proven its worth as an independent arbiter of value-for-money when it comes to medical interventions. People may question the details of the methodology they employ to compare effectiveness of drugs and procedures, but few doubt the principle of its existence. Unfortunately, there is no equivalent
- [Another Grim Day for Big Science...](https://drugbaron.com/another-grim-day-for-big-science/) - Science pervades our lives at every level, supposedly guiding public policy in almost every area, and thanks to the internet increasingly affecting our behaviour directly. Its easy to assume that this rational approach does nothing but good. But fashion affects scientific thinking as much as every other human endeavour, and even the what seems to
- [Finding Safer Medicines](https://drugbaron.com/finding-safer-medicines/) - Medicines need to be safe and effective. Once upon a time there was a trade-off between the two that was assessed at the population level – over the entire group of people receiving the medicine was there more benefit than harm. Across a broad swathe of an indication, a positive answer to this risk:benefit equation
- [Going Against The Grain](https://drugbaron.com/going-against-the-grain/) - In his 2004 Chairman’s Letter at Berkshire Hathaway, Warren Buffet offered sound advice to those trying to find the best time to invest in equities: “be fearful when others are greedy and greedy when others are fearful”. For equity investing, this strategy seems to work very well. Selling at the end of a long bull
- [Why killing “above average” projects is the only way to rescue biotech’s return on investment](https://drugbaron.com/why-killing-above-average-projects-is-the-only-way-to-rescue-biotechs-return-on-investment/) - Across the whole sector, the return on investment in biotech (broadly defined to encompass diagnostic and medical device companies, as well as therapeutic developers) is poor, whether you look at publically traded stocks or private investments through venture capital funds. So poor, in fact, that no ‘generalist’ investor would touch biotech as an asset class.
- [Steps to rebuilding pharma R&D efficiency: “The Exupéry Principle” for medicinal chemistry](https://drugbaron.com/steps-to-rebuilding-pharma-rd-efficiency-the-exupery-principle-for-medicinal-chemistry/) - The efficiency of pharmaceutical R&D is declining. That much is not in doubt: record R&D expenditure in the last decade has led to fewer drug approvals overall, and almost a complete drought in genuine, first-in-class innovation. Set aside biologicals, and the performance in small molecules has been even more dire. But that's where the unanimity
- [The 39 (mostly small) Steps: The Class of 2012 FDA Approvals](https://drugbaron.com/the-39-mostly-small-steps-the-class-of-2012-fda-approvals/) - The pharmaceutical industry achieved 39 approvals in 2012 – the highest number for several years – triggering a much-needed wave of optimism. But sadly approval is not a guarantee of either patient benefit or commercial success. So DrugBaron looked a little deeper at the drugs behind the headline numbers. For a start, only 35 of
- [The Man Who Cured Ageing?](https://drugbaron.com/the-man-who-cured-ageing/) - When Disraeli supposedly said “The only two certainties in life are death and taxes” he could not have imagined that little over a century later, such rock-solid logic could be under threat. If a team of Croatian scientists, led by Professor Miroslav Radman, are correct, death need be no certainty. DrugBaron was fortunate to spend
- [Monte Carlo models of drug R&D focus attention on cutting costs – Part 2 (the caveat!)](https://drugbaron.com/monte-carlo-models-of-drug-rd-focus-attention-on-cutting-costs-part-2-the-caveat/) - “So DrugBaron tells me that drug discovery and early stage development is essentially random, and so all I have do is cut costs to the bone. Then if I have enough shots on goal, even if I don't really know what Im doing, then I will hit big after a while. In other words, drug
- [Evidence that "pick the winners" is precisely the wrong strategy](https://drugbaron.com/evidence-that-pick-the-winners-is-precisely-the-wrong-strategy/) - The asset-centric platform at Index Ventures is built around stringency. The entire concept arose from the realization that drug discovery and development is a complex process, in a low-validity environment (meaning there is often too little data available to make decisions), where the amount of capital required is steeply graded from little to vast sums.
- [Clinical trial transparency: time for some carrot as well as more stick?](https://drugbaron.com/clinical-trial-transparency/) - Today, the majority of clinical trials that are run never report their results in a form that is accessible to the public. To many people that is unacceptable for several reasons: it makes it possible for drug companies to selectively marshal the evidence base surrounding their drugs (if not to subvert the regulatory process, then
- [On the origins of hypochondria](https://drugbaron.com/on-the-origins-of-hypochondria/) - DrugBaron suffers from a condition for which there are no approved drugs. At times it can be a debilitating condition, with obscure causes. Like many psychological illnesses, sufferers are often dismissed as slightly crazy, suffering from something that (to a non-sufferer) appears to have no basis in reality. DrugBaron suffers from a (relatively mild) case
- [Asset-centric project financing is AN answer - not THE answer](https://drugbaron.com/asset-centric-project-financing-is-an-answer-not-the-answer/) - As In Vivo blog noted last week, asset-centric project financing in biotech is everywhere. As they said: "looking at the biopharma newsflow, you would think the entire industry was restructuring itself to find, develop and sell single products" But they also noted that a small, but growing, minority of VC investors are not interested in
- [Platform Technologies – the foundations of big pharma or their nemesis?](https://drugbaron.com/platform-technologies-the-foundations-of-big-pharma-or-their-nemesis/) - It has often been said that one of the key features that distinguishes “big pharma” from biotech is access to the latest technological platforms to aid efficient drug discovery and development. These platforms range from vast chemical libraries, ultra high throughput screening and huge genetic databases in discovery, to predictive toxicology platforms, cutting-edge ‘omics’ and
- [Dendreon proves the risk of investing in "Silver Medal" companies](https://drugbaron.com/dendreon-proves-the-risk-of-investing-in-silver-medal-companies/) - Once upon a time, getting a drug approved by the FDA was an automatic green light for commercial success. If a drug was good enough to pass muster with the regulators, it was a virtual certainty it would find favour with physicians, payers and patients. There was no such thing as market risk for an
- [The zero person biotech company](https://drugbaron.com/the-zero-person-biotech-company/) - Comparing the founding of two virtual drug development companies, Funxional Therapeutics in 2006 and XO1 earlier in 2013 proves at least one thing: in just seven years it has become dramatically easier and quicker to create a virtual drug development company. Today it is even possible to take virtual to its logical extreme: a zero
- [An Open and Shut Case: The Madness of the IPO window](https://drugbaron.com/an-open-and-shut-case-the-madness-of-the-ipo-window/) - As biotech companies queue up like lemmings on a cliff top to hurl themselves through the ‘IPO window’ while its open, DrugBaron tried (and failed) to understand the mentality of public market investors who shun biotech assets for years, and then suddenly buy anything (or so it seems). The trigger for many, if not all,
- [Incremental innovation lives on](https://drugbaron.com/incremental-innovation-lives-on/) - In 2010, DrugBaron declared the death of incremental innovation and wrote the obituary. It didnt seem a very bold statement at the time - increasing payor pressure was beginning to bite, and it seemed obvious that the entrenched practice of paying a premium for small (often imperceptible) improvements was passing. If payors, whether governments, private
- [Positive publication bias IS harmful](https://drugbaron.com/positive-publication-bias-is-harmful/) - DrugBaron was intrigued to see a paper published in PLOS One on June 20th entitled “Why selective publication of statistically significant results can be effective”. After all, selective publication of positive results is one of the key phenomena that underpins the replicability crisis that afflicts science. Could it really be that positive publication bias is a
- [Ichorcumab: the blood of the gods?](https://drugbaron.com/ichorcumab-the-blood-of-the-gods/) - Index Ventures have announced the completion of an $11M Series A investment in XO1, an asset-centric company established with the sole objective of developing the monoclonal antibody Ichorcumab under license from Cambridge University. The story of its discovery has striking similarity with Alexander Fleming’s discovery of penicillin -- combining luck and skill in equal measure,
- [Soriot and AstraZeneca face increasing headwinds: can the acquisitions of Omthera and Pearl boost the engine?](https://drugbaron.com/soriot-and-astrazeneca-face-increasing-headwinds-can-the-acquisitions-of-omthera-and-pearl-boost-the-engine/) - The acquisition of Pearl Therapeutics for up to $1.15 billion, hot on the heals of last months $430 million take-out of Omthera, is an important deal for Pascal Soriot and AstraZeneca. Following the decision on June 4th to return the rights to fostamatinib, further depleting the late stage pipeline, all eyes will be focused on
- [Better drugs cannot cure healthcare system woes](https://drugbaron.com/better-drugs-cannot-cure-healthcare-system-woes/) - For those of us investing money or our careers developing new and better drugs, its easy to assume that we are tackling the biggest unmet need in healthcare. New drug approvals (and even positive early stage studies) are, after all, the biggest news in healthcare. But to be effective, drugs need to be efficiently delivered
- [How NOT to cut costs](https://drugbaron.com/how-not-to-cut-costs/) - DrugBaron’s mantra is simple: drug development is too expensive to make economic sense. Because its impossible (not just difficult) to know whether a given asset is going to make it to the next value inflection point, the numbers only add up if you can ask the value-adding questions as cheaply as possible. Paying for more
- [The power of crowds – and their limits](https://drugbaron.com/the-power-of-crowds-and-their-limits/) - The first internet revolution in the 1990s saw the emergence of on-line service providers who offered the same service as off-line competitors, but cut costs. Big winners included Amazon and LastMinute.com. But the second, and arguably bigger, internet revolution in the 2000’s was the emergence of social networks, with Facebook, LinkedIn and Twitter in the
- [Biosimilars are not similar enough: A proposal for true 'biogenerics'](https://drugbaron.com/biosimilars-are-not-similar-enough-a-proposal-for-true-biogenerics/) - As the debate rumbles on about biosimilars, DrugBaron cannot help wondering if they are not in any case the wrong solution to a very real problem. After all, when it comes to biologic drugs is biosimilar similar enough? The principle of generic drugs mostly works very well, ensuring that at the end of the state-sponsored
- [Please DO cut the science budget!](https://drugbaron.com/please-do-cut-the-science-budget/) - As President Obama signs the sequester cuts into the budget, amid dire predictions of global economic melt-down and howls of protest at the impending reduction of the science budget, there is at least one UK scientist who wishes the same were happening here. DrugBaron would cut the science budget in the UK overnight by 10%
- [Venture Capital 2.0](https://drugbaron.com/venture-capital-2-0/) - Innovation is the life-blood of venture capital. Whether the innovation is in marketing, business model or the product itself, the principle of venture funding is to identify that novel spark that disrupts markets and delivers supra-normal returns – albeit accompanied by abnormally high risk. But for an industry focused on innovation, the business model of
- [The Primacy of Statistics: In defense of the pivotal Phase 3 Clinical Trial](https://drugbaron.com/the-primacy-of-statistics-in-defense-of-the-pivotal-phase-3-clinical-trial/) - Statistics have always had a bad press. Ever since Disraeli’s supposed utterance “lies, damn lies and statistics” the public have treated any claim based on statistical analysis with appropriate caution. But if it is indeed true that statistics can be used (or more likely abused) to ‘prove’ almost anything, it is equally true that without
- [The REAL scandal of drug pricing](https://drugbaron.com/the-real-scandal-of-drug-pricing/) - Healthcare provision, at least in the Western world, is in a permanent state of turmoil, as costs rise inexorably and politicians search for ways to cut spending without angering voters. But why do costs keep on rising so steeply? High priced drugs often get the blame. After all, every time new medicines come on line
- [Why hubris kills returns on pharma R&D](https://drugbaron.com/why-hubris-kills-returns-on-pharma-rd/) - On January 11th, David Shaywitz (@DShaywitz) posted an excellent article on Forbes entitled “Are we the problem?”, in which he attributes many of difficulties in pharmaceutical R&D to the gap between how much we know about biology and how much we THINK we know. In other words, the hubris of researchers and managers across the
- [Healthcare-omics: why we should close down every angiography suite and hand out free statins to everyone](https://drugbaron.com/healthcare-omics-why-we-should-close-down-every-angiography-suite-and-hand-out-free-statins-to-everyone/) - Billions are spent discovering new medicines every year – even with the knowledge that most projects (and even many vastly expensive Phase 3 trials) will fail. But the aim is noble, the prize for success high, and despite debatable return on capital, pharmaceutical R&D has grown exponentially over the last three decades. The presumption behind
- [Bardoxolone and BG-12: A Tale of Two CITIEs (Covalently-Interacting Therapeutic IntErventions!)](https://drugbaron.com/bardoxolone-and-bg-12-a-tale-of-two-cities-covalently-interacting-therapeutic-interventions/) - Until 18th October, bardoxolone methyl (Abbott/Reata) and BG-12 (Biogen) had two things in common: the nrf2 transcription factor complex as their molecular target; and their candidacy for blockbuster status. Then Reata halted clinical development of bardoxolone, following an excess of unspecified serious adverse events and mortality among the treated patients in the Phase 3 BEACON
- [Lessons Learned in the Playground: Why the UK lags behind the US when it comes to biotech entrepreneurs](https://drugbaron.com/lessons-learned-in-the-playground-why-the-uk-lags-behind-the-us-when-it-comes-to-biotech-entrepreneurs/) - Some of the most important lessons in life are learned in the playground. And so it proved this week, when more than 100 wannabe biotech entrepreneurs gathered in London for the Playground event organized by Index Ventures and Nature Biotechnology. The goal was to inspire the next generation of entrepreneurs to take the plunge in
- [From Luddite to Guru: a Cynic’s Guide to Twitter for Biotech Professionals](https://drugbaron.com/from-luddite-to-guru-a-cynics-guide-to-twitter-for-biotech-professionals/) - There was something quite ironic when Luke Timmerman of Xconomy described DrugBaron as “Twitter-savvy”. Even allowing for poetic license that represents a turnaround to match the heroics of the European Ryder Cup golf team last weekend. When it comes to social media, DrugBaron is a renowned luddite – seemingly one of a dying breed who
- [Managing failure is the key to success](https://drugbaron.com/managing-failure-is-the-key-to-success/) - The pharma industry, with decadal time lines and multi-billion dollar R&D budgets, is particularly sensitive to progressing projects that should have been killed. Occasionally failure is perfectly obvious, and the decision to kill a project is trivial. But much more often, there is no certainty whether you are killing a future blockbuster or saving millions
- [Phase 2a proof-of-clinical-concept trials: the single most important determinant of R&D efficiency in drug development](https://drugbaron.com/phase-2a-proof-of-clinical-concept-trials-the-single-most-important-determinant-of-rd-efficiency-in-drug-development/) - The future of the pharmaceutical industry depends on improving our approach to the early Phase 2 trials. After all, three of the most important decisions relating to a drug product candidate are founded on the data emerging from Phase 2a trials: do we believe it will work in pivotal trials with regulatory end-points that will
- [Funxional Screening: The Functional Therapeutics Story](https://drugbaron.com/funxional-screening-the-functional-therapeutics-story/) - FX125L is a fascinating molecule. A first-in-class anti-inflammatory agent, targeting a newly discovered pathway promoting the resolution of chronic inflammation, it holds exceptional promise as a first line treatment option in a number of major disease areas, including asthma, COPD and rheumatoid arthritis. As Boehringer Ingelheim announce the acquisition of the FX125L from Funxional Therapeutics,
- [Boehringer Ingelheim and Funxional Therapeutics announce acquisition of Funxional’s FX125L and the somatotaxin portfolio to treat inflammation](https://drugbaron.com/boehringer-ingelheim-and-funxional-therapeutics-announce-acquisition-of-funxionals-fx125l-and-the-somatotaxin-portfolio-to-treat-inflammation/) - Ingelheim, Germany and Cambridge, UK – 23 July 2012 - Today, Boehringer Ingelheim, one of the world’s leading pharmaceutical companies, and Funxional Therapeutics, a clinical-stage, asset-centric, private company, announced an agreement under which Boehringer Ingelheim will acquire the global rights to Funxional Therapeutics’ FX125L compound and somatotaxin programme. FX125L is a small molecule to treat
- [Autophagy: The Higgs Particle of Biology?](https://drugbaron.com/autophagy-the-higgs-particle-of-biology/) - With all the excitement surrounding the discovery of a particle that might be the elusive Higgs Boson this week, it set DrugBaron wondering what might be the equivalent in biology. The Higgs particle suffuses the entire universe, giving mass to all the other particles. Is there anything so ubiquitous, so central to everything else, in
- [Safety First: An open letter to biomedical inventors](https://drugbaron.com/safety-first-an-open-letter-to-biomedical-inventors/) - DrugBaron is fortunate enough to review hundreds of exciting early-stage drug development projects every year, for organizations as diverse as the British Heart Foundation, Wellcome Trust and US National Institutes of Health as well as Index Ventures and Total Medical Ventures. Such is the competition for resources, that only a small minority win support. Thus
- [The mirage of “big data” in clinical trials](https://drugbaron.com/the-mirage-of-big-data-in-clinical-trials/) - It is inevitable that clinical trials will fail. No matter how good the hypothesis, how promising the compound, sometimes our understanding of the biology was just too patchy. Humans are, after all, complex creatures. But for every “acceptable failure” when the hypothesis being tested was just plain wrong, there is another failure that was entirely
- [Aiming for Average: Three traits where being best means being bang in the middle](https://drugbaron.com/aiming-for-average-three-traits-where-being-best-means-being-bang-in-the-middle/) - Winning entrepreneurs tend not be average. On traits as diverse as energy levels, intelligence and gut feeling the best entrepreneurs are usually off the graph. Whether in sport, culture or business being average just doesn't cut it. Extremes often bring with them big benefits – but sometimes also big handicaps. And working with some of
- [Is academic research finally coming of age?](https://drugbaron.com/is-academic-research-finally-coming-of-age/) - DrugBaron has been concerned about the damage that failures in the system of publishing academic research is doing to the global pharmaceutical industry (and to much else besides) long before it became fashionable. In the six months since 'Rotten Foundations' was published here, we have seen an explosion in studies demonstrating the lack of reproducibility
- [The UK Patent Box: a welcome gift for small biotech as well as large pharma](https://drugbaron.com/the-uk-patent-box-a-welcome-gift-for-small-biotech-as-well-as-large-pharma/) - Confirming the launch of the Patent Box tax break for innovative technologies from April 2013, the UK government has made one of the most far reaching changes to corporate tax structure in recent times. The full implications of the new UK corporate tax regime are still being unraveled, but within days of the announcement global
- [Five Vital Statistics of a Successful Venture Fund – How does the new Index Ventures Life Science Fund Measure up?](https://drugbaron.com/five-vital-statistics-of-a-successful-venture-fund-how-does-the-new-index-ventures-life-science-fund-measure-up/) - The launch of Index Ventures’ new life sciences fund on March 21st is another big step in the reincarnation of the biotech investment model. As a smaller, focused fund it will carry even further the “asset-centric” strategy Index have championed. But the most obvious new feature is the inclusion of two global pharmaceutical giants as
- [Drugging the apolioprotein E pathway – the starters gun has sounded](https://drugbaron.com/drugging-the-apolioprotein-e-pathway-the-starters-gun-has-sounded/) - It can take years or even decades to properly understand a pathway. But when a certain threshold is passed, new therapeutic opportunities can be revealed at an exciting rate. The apolipoprotein E (apoE) pathway has passed that threshold, and VC-backed start ups to exploit this new understanding are springing up like mushrooms. As early as
- [Combinatorial animal study designs](https://drugbaron.com/combinatorial-animal-study-designs/) - It is sometimes assumed that government regulations governing the use of animal models in drug development hamper good science, either by accident or design. But reality is rather different: focus on the 3Rs of replacement, reduction and refinement can lead to more reliable results, quicker, at lower cost and with improved animal welfare and reduced
- [Avoiding the pain of the not-so-mystical placebo effect with the almost-magical run-in period](https://drugbaron.com/avoiding-the-pain-of-the-not-so-mystical-placebo-effect-with-the-almost-magical-run-in-period/) - Nothing kills biotech companies like an active placebo. More often than not the press release after a failed trial points the finger at a "surprisingly large placebo response" against which the active drug had little scope to excel. It's easy to assume that this placebo effect has its origins in some almost mystical neuropharmacology, where
- [Targeting tumour metabolism: the next land grab?](https://drugbaron.com/targeting-tumour-metabolism-the-next-land-grab/) - The sale of Avila to Celgene may mark the pinnacle of the market for kinase inhibitors in cancer. Where might early stage drug discoverers in cancer start looking now if they want to create successes like Avila in five to eight years time? Excitement for different kinds of therapeutic target in cancer goes in waves
- [Merck’s $800M bet on Sir Austin Bradford Hill](https://drugbaron.com/mercks-880m-bet-on-sir-austin-bradford-hill/) - Why Merck’s CETP Inhibitor programme is such a huge gamble – and why DrugBaron fears the odds are stacked against them. Few programmes in the pharmaceutical industry were born with such optimism as the search for CETP inhibitors. Decades of epidemiology had already pointed to the importance of HDL-cholesterol (the so-called “good cholesterol”) as a
- [New Year, Same Message: Ignore Commercial Risk at your Peril](https://drugbaron.com/new-year-same-message-ignore-commercial-risk-at-your-peril/) - DrugBaron wishes his readers a Happy New Year. But how happy 2012 will really be, at least from a biotech perspective, must be open to doubt. If the health of the drug industry depends on the uptake of its products, then 2011 casts a long, gloomy shadow over the New Year celebrations. That shadow comes
- [Chocolate-flavoured Poison: Like red wine and debt, the company-building model of biotech investing tastes good but kills you eventually](https://drugbaron.com/chocolate-flavoured-poison-like-red-wine-and-government-debt-the-company-building-model-of-biotech-investing-tastes-good-but-it-will-kill-you-eventually/) - The most dangerous imperatives are those that you actually like. Hearing, and half believing, that red wine keeps the French safe from heart disease has no doubt led to many a hangover. Hearing, and half understanding, John Maynard Keynes’ advice to spend your way out of recession is the root cause of the raging global
- [Too Much Skin in the Game?](https://drugbaron.com/too-much-skin-in-the-game/) - There are very few rules for the biotech investor that win universal approval. But the first commandment attracts few dissenters: the inventors and developers of the technologies in which you invest your cash must, as a condition of attracting the cash, invest their life and soul in parallel with your money. Or as its usually
- [Choosing the Right Path](https://drugbaron.com/choosing-the-right-path/) - Choosing the right path in clinical development – as in life – is a huge challenge. For small companies it can be the difference between success and failure, survival and implosion. But divergent clinical data published in the last month on two apparently similar members of the Factor Xa inhibitor class demonstrate that the largest
- [The End of a Dream for Population Screening as Diagnostic's "Blockbuster" Product](https://drugbaron.com/the-end-of-a-dream-for-population-screening-as-diagnostics-blockbuster-product/) - For a decade investors in the development of therapeutic products have been coming to terms with the shrinking market sizes of approved products. The focus today is on avoiding “busters” (drugs that are registered, but never achieve any meaningful sales) rather than relying on “blockbusters”. But for investors in diagnostics, the only bright light on
- [Rotten Foundations: How defects in academic publishing are the biggest threat to the commercial life sciences sector](https://drugbaron.com/rotten-foundations-how-defects-in-the-academic-publishing-system-are-the-biggest-threat-to-innovation-in-the-commercial-life-sciences-sector/) - The cherished principles governing the publication of academic science haven’t changed in a hundred years, with concept of peer review at its core - but this ancient system is failing, with catastrophic consequences for life science innovators and investors alike. It might serve academia well enough, but they are not the only constituency who rely
- [Prospect of Failure for the “Company Building” Model of Venture Investing](https://drugbaron.com/prospect-of-failure-for-the-company-building-model-of-venture-investing/) - Prospect Venture Partners surprised the sector with its announcement on 5th October that current commitments, at $150M, were insufficient to support a new fund. That life science VCs might find capital raising challenging in the current economic climate is not in itself all that surprising – as DrugBaron noted last month, the returns of the
- [Crestor™ versus Lipitor™: King Canute Fails to Stem the Tide](https://drugbaron.com/crestor-versus-lipitor-king-canute-fails-to-stem-the-tide/) - As high stakes battles go, the 20-year competition between the various marketed statins in the cholesterol-lowering indication has been one of the most fascinating, and commercially important, in the history of the pharmaceutical industry. The prize is the largest annual sales of any drug ever, thanks in no small part to the controversial decision of
- [Apixaban and Prasugrel: Blockbusters or White Elephants?](https://drugbaron.com/apixaban-and-ticagrelor-blockbusters-or-white-elephants/) - A new study suggests that factor Xa inhibitors, such Eliquis™ apixaban from BMS and partner Pfizer, can be superior to warfarin. On a composite efficacy end-point in atrial fibrillation patients apixaban was significantly better than warfarin (1.3% versus 1.6%; p
- [Making Drugs Safer Might Cost You Your Job - Or Even Your Life!](https://drugbaron.com/making-drugs-safer-might-cost-you-your-job-or-even-your-life/) - On the 1st February 2011, one of the largest and most successful global pharmaceutical companies, Pfizer, announced the closure of its Sandwich (Kent, UK) site, with the loss of the 2,400 jobs. Some, such as John Gapper at the Financial Times, see this as another step on the road to the complete out-sourcing of drug
- [The Death of Incremental Innovation](https://drugbaron.com/the-death-of-incremental-innovation/) - Does it work? Once upon a time it was sufficient to determine if a drug, diagnostic or device delivered some benefit. A significant effect in a randomized, placebo-controlled trial was necessary and sufficient to identify efficacy. Combined with demonstration of safety, this was enough to garner regulatory approval and with it a sizeable profit. But
- [A penny for your thoughts –  how much value does an idea have today?](https://drugbaron.com/a-penny-for-your-thoughts-how-much-value-does-an-idea-have-today/) - I remember very well a meeting in the mid 1990s when a venture capital partner told me that “a good idea is worth a million’. He didn’t specify whether his bon mot was denominated in pounds, dollars or euros, but as an inventor of early stage technologies it was music to my ears. Perhaps more
- [Hard Times, Harder Times](https://drugbaron.com/hard-times-harder-times/) - No one is in any doubt that these are hard times for life sciences companies. Some commentators have even suggested that these are the hardest times. The cause is obvious enough: the paucity of capital that has blighted the ‘real’ economy has been felt even harder in the cash-hungry world of technology companies that lack

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## Resources

- [The Cambridge Partnership](https://drugbaron.com/resource/the-cambridge-partnership/) - The Cambridge Partnership is the only professional services company in the UK exclusively dedicated to supporting companies in the biotechnology industry. We specialize in providing a “one-stop shop” for accountancy, company secretarial, IP management and admin services. The Cambridge Partnership was founded in 2012 to fill a gap. Running a biotechnology company has little in
- [RxCelerate Limited](https://drugbaron.com/resource/rxcelerate-limited/) - RxCelerate Ltd is an outsourced drug development platform based near Cambridge, UK. We specialize in delivering an entire road map of drug development services from discovery and medicinal chemistry through to formal preclinical development and clinical up to Phase IIa. In the last five years, we have witnessed dramatic changes in the drug development landscape,

## Categories

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## Tags

- [popular](https://drugbaron.com/tag/popular/)
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## Category

- [Out Sourced Drug Developer](https://drugbaron.com/resources/out-sourced-drug-developer/) - Specialising in delivering an entire road map of drug development services from discovery through to preclinical/clinical Phase IIa.
- [Accounting & Back-Office Services](https://drugbaron.com/resources/accounting-and-back-end-services/) - A professional services company exclusively dedicated to supporting companies within the biotechnology industry.