While the OPDP’s move is a welcome step in shutting down Burzynski’s clinical trial pyramid scheme, in which, according to some patient fundraising blogs, trial participants pay more than $100,000 to become guinea pigs for antineoplaston therapy, the letter provides little clarity regarding what is or is not a promotional claim.

The letter specifies, “This provision is not intended to restrict the full exchange of scientific information concerning the drug, including dissemination of scientific findings in scientific or lay media. Rather, its intent is to restrict promotional claims of safety or effectiveness of the drug for a use for which it is under investigation and to preclude commercialization of the drug before it is approved for commercial distribution.”

However, some of the so-called violations strongly resemble claims made in countless biotech and pharma press releases posted on company websites.

For example, “ANP was well tolerated with easy manageable side effects of fatigue, skin rash, and electrolyte abnormalities and no chronic toxicities . . . These results compared favorably to radiation therapy and chemotherapy (Mandell, et al. 1999, 7 percent overall survival at 2 years and 0 percent at 5 years), but should be confirmed in phase III trials scheduled to begin in 2009.”

On the face of it, that statement seems identical to hundreds of statements made every day by other companies, so what distinguishes Burzynski’s regulation-violating promotion from permitted “exchange of scientific information?”

Clarity is on Hold

Crystal clarity on that question may be a long time coming. The biopharma community has been clamoring for the FDA’s guidance on a related question – how to use social media – for nearly two years, and the agency has answered with silence – a silence set to continue for another two-year period, under the new FDA Safety and Innovation Act.

That silence has led to a “gotcha mentality” that uses enforcement, rather than guidance, to communicate policy.

For companies that want to avoid running into trouble with their public communications, warning letters such as the Untitled Letter issued to the Burzynski Clinic are some of the only clues available as to what the regulations allow and do not allow.

Stephen King, a public affairs specialist for the FDA’s Center for Drug Evaluation and Research, offered a statement that provided a few more hints on the Burzynski case.

“The FDA takes seriously its role in assuring Americans the drugs they use are safe and effective and manufactured according to current good manufacturing practices (cGMP). The agency also seeks to ensure patients who are participating in clinical trials and expanded access programs are appropriately protected,” King said. “To date, no randomized, controlled trials showing the effectiveness of antineoplastons have been published in peer-reviewed scientific journals nor have all of the trials needed to approve antineoplastons as a treatment for cancer been conducted.”

So far, so good. But again the FDA’s communication falls short of total clarity as to why certain claims violate regulations and others don’t. In the Burzynski case, the clinic’s lack of evidence to back up its claims, and track record of plundering its patients’ retirement funds may contribute to the OPDP’s actions, but nothing in the OPDP letter actually cites false claims or misunderstanding by patients. “The totality of these claims suggest that Antineoplastons, investigational new drugs, are safe and/or effective for the treatment of various types of brain tumors indicated above, when they have not been approved for these uses,” the letter states, suggesting that the only and entire violation by the Burzynski clinic is that it said the words “safe and effective” before the drugs had been approved ‑ words which are ubiquitous in drug company statements regarding clinical trials of unapproved drugs.

“The promotion of investigational new drugs is prohibited by law and the agency is concerned BRI’s promotional claims will mislead patients about the safety and efficacy of unapproved antineoplastons,” said King, providing few hints as to how other companies should modify their language regarding safety and efficacy of their own unapproved drugs.

OPDP instructed the Burzynski Clinic to immediately cease dissemination of “violative promotional materials” for antineoplastons and to submit a written response by Nov. 1.

A company spokesman for the Burzynski Clinic, Azad Rastegar, told BioWorld, “Yes, we have received a letter from the FDA. While we don’t think we have done anything wrong, we have complied with their requests.”

Pharma may be prehistoric in terms of age compared to biotech, but it maneuvers more like a velociraptor than lumbering brontosaurus, as it commonly consumes everything in its path that can impend, contest or abet its dominance.

Likely, 100 years from now, pharma will still be standing and bio-who? will be so assimilated into the DNA of pharma that its individuality is what will be extinct.

Increasingly, pharma is obliging all of us to disregard the biotech industry as a standalone entity and acknowledge it as part of a seamless “biopharma” industry.

Sure, the biotech sector is projected to be the leading drug development market component by 2020 in terms of growth, R&D, new product development and well, just about everything except the executive management of the market.

Often, biotechnology company evolution entails the biotech progressively learning to stand on its own, just in time to be rancorously swept off its nimble feet in a late-stage takeover by a supposedly unwieldy pharma antagonist.

Resistance is Futile

The pharmeleon is trans-pharming again to assimilate with biotech, its latest target. Since 2000, pharma has recurrently been able to assume the persona or façade of a biotechnology entity, a VC, a med-tech, a generics developer or whatever was necessary to fend off internal and external potential maladies such as patent cliff obsession, pipeline arrhythmia, product envy, market share anxiety and stockholder disorder.

Pfizer Inc. is staving off the evolution of the generics companies by extending the reign of the Lipitor pharm-ulary with a discount on the brand drug that undercuts the generic price. It won’t work forever, but the tactic is, so far, out of the scope of regulation and has kept Lipitor from the extinction list past its expiry date. It’s not new product development, but it is innovative survival skill.

Selling brand drugs at generic prices to thwart generics cannibalization and instead parasitize the 180-day generics exclusivity period? That’s pharma-tastic!

The top-selling pharma drug list leaders will likely change every quarter for the remainder of 2012 and into 2013, reflecting the patent loss frenzy. Much analyst speculation exempts none of biotech’s best and brightest – ranging from the rare disease prodigy BioMarin Pharmaceutical Inc., to Amgen Inc., the market’s most profitable biotech – from the pull of pharma’s big deal crusade, as biotech’s vanguard is projected to be continually conscripted into pharma’s M&A time machine throughout the decade in order to keep repeating the history of pharma’s success.

So, Sanofi’s road to the future suddenly looks clotted with regulatory setbacks and blockbuster expiries? Implement Plan B: move Genzyme Corp. into pharm-ation to attack the clinic!

It’s a Pharma World!

Pharma permeates all it encounters. If you are waiting for the patent cliff asteroid to knock the pharma market off its axis and into extinction – give up on that thought! That threat is being flicked off more like aggravating grains of sand than a deadly space rock in hurtling free-fall.

After all, this may be pharma’s most serious patent cliff, but it isn’t its first, as the species has survived the end of gilded eras including that of Valium, Prozac, amoxicillin, Ritalin and more, and has also regularly withstood periods when more than 100 pharma patents per year expire.

Around 2030, when market archeologists excavate for clues to the fate of the self-sustaining biotechnology industry, the only artifacts they will find will be the well-preserved in-house carcasses and the fossilized stock tickers of the legendary alpha male biotechs such as Human Genome Sciences Inc., Genentech, MedImmune Inc. and also the likely additions of current market leaders such as Celgene Corp., Gilead Sciences Inc. and any other brave souls game enough to evolve to the large cap food chain menu.

In maintaining its status as the pharm-most drug development market, pharma has mastered the pharm-ula for success that relies on making the biotech market dependent on its pharm subsidies to survive.

Biotech may be first-in-brains, but pharma is first-in-business. Original innovation may be coming from biotech, but just about all else resides in the Cave of the Small Molecule Clan.

Where would the biotech market be without pharma, and more to the point: where would you, I and about a pharma-trillion other patients who have popped a pill within the last century be without the reliable presence of the ancient ones?

We all want the biotech industry to succeed and it likely will – under the general auspices of pharma.

I politely inquired whether he had considered dieting and exercise, which could produce similar results without the potential side effects of a prescribed drug – especially one in a category that’s been dogged by cardiovascular risks.

“That’s just too hard,” my uncle replied, with a mixture of helplessness and disdain.

Sadly, that’s exactly the attitude parents seem to be passing on to their children, and the results have been catastrophic.

Last week, the journal Pediatrics published Prevalence of Cardiovascular Disease Risk Factors Among U.S. Adolescents, 1999-2008, which provided a sobering look at the twin epidemics of obesity and diabetes in teenagers.

Authors Ashleigh L. May, MS, PhD, Elena V. Kuklina, MD, PhD, and Paula W. Yoon, ScD, MPH, of the U.S. Centers for Disease Control and Prevention in Atlanta concluded that obesity in the teenage population was unchanged, with one in three adolescents between the ages of 12 and 19 officially overweight or obese, with a body mass index of 25 to 30 or greater than 30, respectively. That figure is shocking in its own right.

But the horrifying change was a doubling in the prevalence of prediabetes and diabetes in teens over the same period, from 9 percent to 23 percent, along with cardiovascular disease risk factors formerly seen only in adults.

Really, parents? What part of this picture do you not understand?

Diabetes is a devastating chronic condition that can cause life-changing complications such as nerve damage, blindness, amputation, kidney damage, heart attack, stroke and, of course, death. And unlike the pounds that can be shed from an overweight teen, diabetes is irreversible and incurable. It’s a life sentence.

What’s the best prevention for Type II diabetes, which results from lifestyle factors and accounts for more than 90 percent of cases of the disease? Not a pill or a shot, but simple exercise and a diet that maintains normal body weight.

No right-minded parent would push their child in front of a moving train, but that’s exactly what many parents are doing when it comes to the health of their children. It’s hard – but not too hard – to fix that. Trust me. I raised two teens and I’ve heard all the excuses.

For starters, get them off the couch before their only muscles are located in the fingers that do their texting. Make them walk the dog, cut the grass, take out the trash and ride a bike. Even better, encourage them to play soccer, swim, or run track. I started jogging at the age of 40 with my 12-year-old daughter, who wanted to make her middle school basketball team. It’s turned into a family pastime.

Substitute milk or water for soda. Treat French fries as a luxury instead of a staple. Give them carrots and celery – without the dip. Contrary to popular belief, fresh vegetables are cheap and don’t require a culinary degree to prepare. The U.S. Department of Agriculture’s ChooseMyPlate.gov offers simple tips for healthy eating on a budget.

And while you’re at it, take the same medicine, because children are smart. They watch their parents sit on their smartphones, upsize their burgers and guzzle their mocha Frappuccinos, and believe those habits signify adulthood. Instead, many children are slowly being steered into a lifetime of blood sugar monitoring, special diets and medications – and that’s if they’re lucky enough to avoid the more dreadful complications of diabetes.

Which brings me back to my uncle. It’s one thing for a 60-something adult to pack on 50 extra pounds and become a ticking time bomb, all the while hoping the approval of Qnexa, Contrave  or lorcaserin will avert an explosion. It’s quite another for an adolescent to face the same potential fate.

Parents need to step up to the plate, and make it a healthier one, at that. The future of our children is too important to abdicate this responsibility to ignorance or indifference.

Although drug lords and cartels are not apt to file annual reports, the United Nations estimates that 5 percent of the global population now takes illegal drugs that account for an approximate $430 billion black market industry that, fortunately, still trails the $800-plus billion prescription drug market.

Of course, government keeps both markets in check for the sake of public safety. It seizes illegal drugs and takes the criminal kingpins to trial in the illegal market, thereby devaluing the illicit drug revenue stream. In the legal drug market, government comparatively acts by using the regulatory approval process as the legal system corporate kingpins must deal with, and the clinic as its institution of incarceration.

Drug dealers do have one advantage over drug makers, though. Before the first pill is even developed, the legitimate drug peddlers are sentenced to 10 years’ confinement behind clinic bars and fined $1 billion as conditions of their parole allowing them to perform their potential blockbuster community service. Fortunately, conjugal M&A visits and paid-escort partner deals that can often serve to relieve the inmates’ executives’ stress and frustration, are allowed.

Arguably, the key dynamic in the drug development market is the marriage of convenience between the pharmaceutical and biotechnology industries.

OK, they are shacking up! They’re consummating the deal like rabbits! And that’s about as subtle as I can be without descending into pornographic allusion!

Big Pharma is now Bio Pharma. They’ve been discreetly dating for some time now, but may have thrown many of you off the trail with their entertaining diversions in order to attempt to keep their affair somewhat under wraps. But the fact is that pharma and biotech are intent on producing offspring. In fact, they are planning a brood of May-December biopharma marriages that they hope will translate into a family of drugs that will be delivered into the world by the doctors at the FDA and regulatory agencies worldwide, beginning in 2012.

Canoodling @BIO

You may have seen the two of them canoodling in one-on-ones at the annual Biotechnology Industry Organization’s meetings or overheard them playing cute on boardroom phone lines late into the night. Perhaps you’ve witnessed some of the play-acting they have put on display in all-too public spats over who was going to move in with whom, as they postured for the advantage in the pre-nup merger negotiations.

Usually, when we are initially apprised of the news of a hostile takeover and the biotech’s parent board balking at the size of the pharma dowry, we assume it’s just the bluster of the mating dance and not the beginning of the breakup. So now, we just wait for the ultimate invitation to the merger ceremony.

Perfect storms can create frenzied Black Holes or dazzling landscapes, but either end result is usually borne of chaos. Think economic turmoil, patent cliffs, regulatory irregularity, R&D senior moments, failed partnerships, etc. But usually, just like in personal relationships, money issues tend to be at once, the lure that initially attracts opposites, as well as the irreconcilable difference that often renders R&D agendas or companies asunder. No matter how it ends, biopharma’s perfect storm is upon us.

The “P” Word is Becoming Offensive

How long into this decade will it be before we see pharma drop all pretenses and declare its affection for the much younger industry by going all BioPfizer, Merck & BioCo., or Johnson & BioJohnson?

The oft-married GlaxoSmithKline plc has declared its intention to shed its small molecule wardrobe and dress itself up in large molecule hoodie and skinny jeans and hang out more as a “biotech-like” company, while Merck & Co. now sees itself as “a global biopharma leader,” after dropping a few corporate pounds and getting a life sciences facelift.

The former Sanofi-Aventis, which changes its last name more times than P. Diddy or Elizabeth Taylor, has just deleted its post-hyphen once again – for what? Perhaps in preparation of patently betrothing itself to biotech as “Sanofi-Biopharma, Sanofi-Amgen …?”

So, you begin to see that it’s too late to deny the biopharma relationship is passed the “we’re just friends” stage,” as the two smitten markets continue to moving in together on Wall Street.

So, we get a lot of adorable misdirection, designed to keep the markets’ hearts beating in rhythm and to minimize investor arrhythmia, but the fact is that big pharma is shedding its small molecule skin and evolving into the hybrid entity that will characterize the drug company of the 21st century.

It promises to be a decade-long dual bash: big pharma’s retirement get-together is also biotech’s engagement party.

Distraction is so rampant in society that it hardly even seems rude any more to multitask in conference sessions. There is so much competition for our attention that we are perpetually too preoccupied to focus on a primary task.

Out of 173 audience members in attendance at a panel discussion at the BIO CEO and Investor Conference in New York in February, I counted 52 who were giving undistracted attention to the goings-on. The rest were either texting, tweeting, web surfing, playing games(!), or actually talking on their phones. Four were dozing, so I assume their batteries had drained!

A recent performance of the New York Symphony at Lincoln Center was stopped by the conductor when a cell phone repeatedly rang during the performance. The owner of the evidently-too-smart-for him device claimed it was a recent purchase and that he did not know how to silence the ring (or turn it off either, evidently).

In a fleeting newscast pan shot this week, I saw four people participating in the 2012 Selma-to-Montgomery March re-enactment who were talking on cellphones as they passed the camera. I couldn’t help but think Martin Luther King Jr. and his fellow marchers were dodging bricks, attack dogs, high-pressure water and epitaphs – but not annoying, intrusive phone conversations.

I was at a comedy show about four years ago and a ticketholder was talking loud on a cell and fingering her smart device throughout the set. When the comedian could take no more, his act abruptly turned into a five-minute personal insult tirade that started out with the offender trying to trade barbs with the professional, but ended with her storming out in tears and the audience giving the comedian a standing ovation. I am not so sure the audience would react the same today. They’d either sympathize with the serial talker or be too involved themselves cleaning out their inboxes, tweeting a recap of the jokes to their followers or capturing the war of words to upload to video.

Biotech is a big and serious business, but courtesy and good manners have been around even longer than the first biologics license application, so let’s not lose our civility. After all, how would you feel if an FDA advisory panel member felt obliged to interrupt the meeting to tweet and take phone calls?

It’s no excuse, but part of the problem that emboldens this impoliteness may be the relaxed setting of the spacious conference rooms. I do not witness such discourtesy in the more intimate milieus for board meetings, staff meetings or one-on-one engagements. I can’t imagine a biotech executive not giving his undivided attention in wooing a VC in a conference tête-à-tête by cleaning out his inbox or cutting off the dialogue with, “I have to take this call – it’s my wife – No dear, spaghetti’s fine – Now, where were we?”

Financing denied!

Upon contemplating the ever-increasing divided attention order-of-things-to-come trend, I’d like to know what does everyone think?

• Do you consider it discourteous to interface with smart devices while speakers are engaged in communication on stage?
• With live webcasts and other high-tech options emerging, is physical attendance even no longer necessary to produce a successful conference?

Well, I’ve exceeded 400 words, which is the new media attention-span article threshold. So, I need to wrap this up before the auto-censoring feature on my tablet termina – - -

But the movie’s tag line is also worth keeping in mind: “Nothing spreads like fear.”

Another round of alarming news has brought the real H5N1 back into the headlines in recent weeks. Experimental findings that it only takes a few mutations for the virus to become highly contagious via human-to-human transmission were deemed so dangerous that publication of the experimental details has been delayed, and there is currently a voluntary moratorium on H5N1 research.

But a study published today suggests that H5N1 infections are already more frequent, but milder, than the current WHO data indicates.

The WHO’s official count as of Feb. 22, 2012: There have been 586 humans infected with the H5N1 virus since 2003; 346, or 59 percent, of those infections have been fatal.

But the authors of the new paper, which appears in the Feb. 24, 2012 issue of Science, believe that the WHO’s stringent criteria end up missing most cases of H5N1.

Believe me, I am not trying to sneer at the WHO here. Public health organizations face a difficult task, needing to alert but not unduly alarm the public, encouraging prudence without panic. The WHO walks that line well.

But it is worth noting that the criteria for confirmed H5N1 infections make it unlikely that mild cases of H5N1 will be confirmed in the first place.

The new paper is a meta-analysis of prior studies that looked at serum antibodies to H5N1 as a way to estimate true rates of exposure to the virus. Such antibodies can identify people who have been exposed to H5N1 but are not counted in the WHO tally, because their case was too mild to meet WHO criteria ‑ or for them to seek medical attention at all.

Overall, those numbers tell a very different story than the official 586 cases. The best estimate for rates of exposure were in the range of 1 percent to 2 percent of the population, which, the authors said, would translate into millions of infected individuals.

Figuring out the true death rate is harder still than determining how many people have been infected with H5N1.

Even in the U.S., with its high rates of access to healthcare and mountains of medical records, figuring out how many people get infected with annual flu, and how many die from it, is an imprecise science. For one thing, not everyone with the flu goes to see a doctor. For another, flu deaths are indirect. The virus does its damage by weakening the body, especially the lungs, and the direct cause of death is most often pneumonia.

Such uncertainties are multiplied in the developing areas where new flu strains set out on their annual global trek, and where H5N1 has been circling in its animal reservoirs and popping out every now and then. Just like there appear to be many people who are infected with H5N1 without making it into WHO statistics, so there are undoubtedly people who have died of H5N1 without the medical community’s being any the wiser.

Still, even if the new paper had somehow managed to overestimate the number infections tenfold – which is hard to imagine, given that senior author Peter Palese is an extremely well-respected influenza researcher – then a death rate of nearly 60 percent would mean that there were hundreds of thousands of H5N1 deaths that have somehow gone unnoticed. This seems a lot less likely than the alternative possibility, namely, that H5N1 may not be as deadly as we think.

On Business

“Biotech is all about picking the exception. Granting access to capital to everyone doesn’t strike me as the right idea.”

‑ Bob More, general partner with Frazier Healthcare Ventures, taking an optimistic view on capital constraints. Private biotechs continued to struggle in 2011, raising about as much money as in 2010.

"I think we're going to see a regression to quality."

‑ Jim Healy, general partner at Sofinnova Ventures, on how the venture contraction will mean that only the highest quality companies get funded. Several venture groups pulled out of biotech investing during 2011.

“The take-home message is that it’s easy to build a $25 million company with $100 million cash.”

‑ Tillman Gerngross, CEO of Adimab LLC, on the trend of sliding valuations post-initial public offering. Although a handful of biotechs went public in 2011, the window did not open as wide as some had hoped.

"You can't save your way to success in this business."

‑ Marina Biotech Inc.'s CEO Michael French on the tough financial choices small biotechs must make, such as accepting harsh terms to keep a company moving forward.

“Monday, I was in Chicago, presenting at ASCO. Tuesday, I’m in California, pitching at Goldman (Sachs). And Wednesday, I’m in New York, presenting at Jefferies. How many red-eye medallion miles does it take to get one deal?”

‑ A jet-lagged biopharma CEO, taking a break at the Jefferies 2011 Global Healthcare Conference, and illustrating just how hard biotechs had to work to close financings and partnerships in 2011

On Science

“What happens when the next 10 patients you see require eight different drug combinations based on the mutations in their tumors?”

‑ Outgoing ASCO president George Sledge, on the exponential increases in complexity required to tackle “chaotic” tumors with high mutational load. Personalization of medicine continued to be a theme at ASCO and AACR in 2011.

"You do have to be somewhat of a Talmudic scholar to prescribe this drug."

‑ Lawrence Friedman, member of the FDA Antiviral Drugs Advisory Committee, referring to the regimen for Merck & Co. Inc.'s hepatitis C candidate boceprevir. His comments illustrate the difficulties of putting more personalized treatment regimens into practice.

"The fact that it's inefficient doesn't mean it hasn't made great contributions."

‑ John Mendelsohn, president of the M.D. Anderson Cancer Center, on the National Cancer Institute-sponsored collaborative clinical trials program

“It disturbs me that people are willing to accept this: treating the symptoms and allowing the disease to progress.”

‑ Jerry Colca, president and chief scientific officer at Metabolic Solutions Development Co., on new diabetes drugs

“For every target, almost every company has an inhibitor against it. Medically, as well as economically, we truly need something to get beyond that."

‑ Chiang Li, CEO of Boston Biomedical Inc.

General Wisdom

"A lot of innovation is incremental, not breakthrough."

‑ Glen Giovannetti, global biotechnology leader at Ernst & Young

“I realize it’s absurd to talk about 10Xs or even 5Xs these days.”

‑ Roger Longman, CEO of Real Endpoints LLC

“A combination of good science, good collaborations, good timing and some good luck.”

‑ Mace Rothenberg, Pfizer Inc.’s senior vice president of clinical development and medical affairs in the Oncology Business Unit, on the secrets to the success of lung cancer drug Xalkori (crizotinib)

"You can only set yourself on fire once."

‑ Oleg Nodelman, partner at Biotechnology Value Fund, referring to how pharma stocks tend to jump when they severely cut R&D budgets

"Clearly, there are easier and less insane ways to make a living."

‑ Jim Greenwood, BIO president and CEO, at the BIO 2011 keynote luncheon

The past year brought the biotech sector a mixed bag of news. Some of it was positive – for instance, the FDA picked up its drug approval pace in 2011 and, in some cases, even surprised the most hardened biotech investors by granting approval of some drugs a month or two before their PDUFA dates – while other headlines hailed discouraging trends such as the failure of Prospect Ventures to close its latest round, confirming predictions of a venture capital contraction.

As we get ready to head into 2012 (and possibly an apocalyptic count-down, if those Mayans are to be believed), let’s take a look back at some of the highs and lows of the biotech industry in 2011.

‘Me-First’ Instead of ‘Me-Too’

Biotech execs always talk about tackling unmet medical needs. So it was exciting to see in 2011 the flurry of breakthrough drugs hitting the market. An informal BioWorld poll tagged Seattle Genetics Inc.’s lymphoma drug Adcetris (brentuximab vedotin), the first approved antibody-drug conjugate, as the biggest drug approval of the year, tied with HCV game-changers Incivek (telaprevir) from Vertex Pharmaceuticals Inc. and Victrelis (boceprevir) from Merck & Co. Inc. But other notable approvals included Human Genome Sciences Inc.’s Benlysta (belimumab), the first lupus drug approved in 50-plus years, and Yervoy (ipilimumab) from Bristol-Myers Squibb Co. and Zelboraf (vemurafenib) from Roche AG and Daiichi Sankyo Co. Ltd. as the first two drugs approved for melanoma that actually improved overall survival in clinical testing. And, late in the year, Incyte Corp. scored a win with Jakafi (ruxolitinib), the first approved JAK inhibitor and the first drug for myelofibrosis.

Let’s all hope that trend of “firsts” continues in 2012.

A ‘Nobel’ Death

In a bit of irony (in an Alanis Morissette kind of way), the Nobel committee awarded one-half of the 2011 Nobel prize in physiology or medicine to Ralph Steinman, unaware that the Rockefeller University professor had died of pancreatic cancer three days earlier. Posthumous Nobels are unusual but, after brief debate, the committee wisely decided to allow Steinman to remain a Nobelist. And I’m glad. He certainly earned the title for his discovery of the dendritic cell and its role in adaptive immunity. Plus, according to BioWorld’s science editor, Anette Breindl, who heard Steinman speak in 2007, he was a pretty nice guy.

Best Bang for the Buck?

The biggest M&A deal in 2011 was easily Sanofi SA’s $20-billion-plus-contingent value rights buyout of Genzyme Corp., which followed nine months of often less-than-friendly negotiations. But it wasn’t the best deal for biotech investors. Genzyme shareholders already have lost out on the $1 CVR connected to manufacturing capacity for enzyme replacement therapies Cerezyme and Fabrazyme. Whether they will be able to earn the remaining $13 linked to multiple sclerosis drug Lemtrada (alemtuzumab) is up for debate, and, for the most part, analysts are not optimistic.

If I were to choose, I’d say that Daiichi Sankyo Co. Ltd.’s bid for Plexxikon Inc. and Shire plc’s buyout of Advanced BioHealing Inc. were far more successful M&A deals. Daiichi’s hefty $935 million payment for Plexxikon – a move validated when Zelboraf gained approval in August – came despite only getting U.S. co-promotion rights to the melanoma drug under Plexxikon’s existing partnership with Roche AG. The Shire/ABH deal, meanwhile, showcased the kind of investor returns rarely seen these days. The big pharma firm shelled out $750 million for ABH, a whopping 15x return for ABH’s largest shareholder.

‘Weighed’ Down by Safety

Despite health experts clamoring for new ways to treat the so-called obesity epidemic, drugs aimed at helping people lose weight have not had an easy time of it at the FDA. Industry observers had expected 2011 to usher in not one, but three obesity therapies; instead, all three were rejected by the FDA. Orexigen Inc.’s Contrave got stalled on cardiovascular concerns, while Vivus Inc.’s Qnexa raised worries of birth defects related to one of its generic components and Arena Pharmaceuticals Inc.’s Lorquess bumped up against carcinogenicity concerns. Contrave is set to start a large cardiovascular outcomes study, which would delay its approval until 2014. But Qnexa and Lorquess could have a shot at getting approved in 2012. That’s assuming that no additional safety issues crop up. Given the track record in the obesity space, that’s definitely no guarantee.

The Rough Side of Town

While 2011 had its much-lauded drug approvals (as noted above), the year was not without its disappointments. Two promising areas of research suffered blows – Roche pulled out of the RNAi space, dumping a high-dollar collaboration with RNAi powerhouse Alnylam Inc., while Geron Inc. abandoned its position as embryonic stem cell pioneer to focus on a less-risky cancer drug. But the biggest disappointment of the year – as chosen by 41 percent of respondents to a BioWorld poll – was the slow sales of Dendreon Corp.’s prostate cancer vaccine Provenge (sipuleucel-T). Sales fell way below estimates, and, over the course of the year, Dendreon’s shares have lost about 80 percent of their value. The Seattle-based company has since retrenched and is hoping European approval, now pending, will help accelerate revenue growth. We’ll be watching in 2012.

And the Strange Bedfellows Award Goes to . . .

Probably one of the most bizarre deals ever in biotech was 2011’s short-lived merger agreement between Allos Therapeutics Inc. and AMAG Pharmaceuticals Inc. Having in common only commercially underperforming products – Allos’ Folotyn (pralatrexate) for peripheral T-cell lymphoma and AMAG's iron deficiency drug Feraheme (ferumoxytol) – execs from both firms tried hard to convince investors and analysts of the logic in combining the companies by pointing out a handful of “cost synergies.” But the merger was met with opposition from the get-go and even prompted hedge fund MSMB Capital Management to make an unsolicited bid for AMAG. Unsurprisingly, the deal was voted down by AMAG shareholders. Seriously, what were they thinking?

Well, that’s my list of headline-grabbing biotech news in 2011. But it’s hard to remember a whole year, and I’m sure I’ve forgotten something. What do you think were the biggest events in biotech in 2011?

Some watchers of Pfizer’s R&D efforts are victims of several “brutal layoffs” within the company. Others are pharma outsiders who debated with management as to whether there actually was a “Pfizer model of drug discovery” and, if so, the advisability of trying to emulate it.

The description of Pfizer as a “dysfunctional pharmaceutical giant” and the drama and politics of Kindler’s ouster came as confirmation that Pfizer was no better than any other pharma in ensuring the sustainability of its pipeline especially with its “ossified culture.”

Makings of a Best-Selling Movie

The Fortune piece has all the makings of a best seller along the lines of Smartest Guys in The Room (Enron), Barbarians at the Gate (RJR) or Too Big To Fail (Wall Street). The elements for a great movie that would appeal to the general public also are present: money; the politically adept; a micromanaging, indecisive and abrasive CEO; the antagonistic and manipulative head of HR, primary confidant of the CEO; and the éminence grise in the form of a previous CEO. Oh ‑ and money. An actual character for the head of R&D, “a ship passing in the night” is not covered in the article.

In considering a movie, much amusement can be derived in contemplating actors for the lead character roles. The Kindler part would be well served by Donald Sutherland reprising his politically adept, ruthless CEO role in Disclosure.

Based on the article, the HR head would be a natural for Meryl Streep ‑ not a reprise of her bubbly performance in Mamma Mia, but an amalgam of parts she has deliciously (The Devil Wears Prada) and maliciously (Doubt) played.

The éminence grise would be a perfect role – minus the gun ‑ for Edward Fox (The Day of the Jackal).

Casting the head of R&D ‑ like that in real-world Pfizer ‑ is challenging given that three individuals held this role in the five years or so that Kindler was CEO. While many researchers might consider Bruce Willis or Tom Cruise playing this role, Tom Hanks reprising his role as the improbable Robert Langdon from The Da Vinci Code would be a better candidate as might Russell Crowe (The Insider) or Matt Damon (The Informant!)

The options remain endless. Notwithstanding, the Pfizer palace coup may be a timely, if not enjoyable movie as it documents the case against pharma megamergers, the cadres of “outsourced” scientists in Ann Arbor, Fresnes, Groton and Sandwich and the helicopters traversing the East Coast.

By Mike Williams, BioWorld Perspectives contributing writer