22
Sep
2016

Sarepta’s Controversial Victory, Allergan’s Buying Spree, & GSK’s History-Making CEO  

More than a little history was made the past week or so in biotech. Big burning questions were met with action, like it or not.

For the rundown on the FDA tension between science-based review and individual patient desire; growth-through-acquisition (as an alternative to price jacking); and the first female named to run a major pharma company, read Frontpoints.

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13
Sep
2016

What Phil Sharp Thinks of ‘Hood’ + 3 Book Events in Boston

When I spill a few of the shockers in “Hood: Trailblazer of the Genomics Age,” people seem surprised. “I didn’t think the book would be so tough,” one woman said.

It is tough. It’s also fair. That’s the result of poring over thousands of pages of confidential files, public records, and conducting more than 150 interviews with Hood and his family, friends, collaborators, and detractors.

Once people read Hood, the biography of a charismatic and controversial personality in science, they like it:

Phil Sharp, the Nobel laureate at MIT, compared Hood to one of the best-selling and most controversial biology books of all-time. “Timmerman captures the glory and clay feet of a great scientist, like Jim Watson did for himself in The Double Helix,” Sharp wrote.

David Shaywitz, the chief medical officer of DNAnexus, said the Hood life story effectively picks up where Horace Freeland Judson’s 1979 masterpiece, The Eighth Day of Creation leaves off. “An unobstructed view into the egos and drama of modern high-stakes research,” Shaywitz wrote.

Keith Robison, a computational biologist and well-known blogger, called Hood “a valuable exploration of one of the leading figures in the early development of genomics and proteomics.”

If you pre-ordered a signed copy, your book went out in the mail yesterday!

Need to buy your copy? Here’s how to get one:

  1. Hardcover: $29.95 on Amazon (or at University Bookstore in Seattle).

    Hood, on prominent display at the University Bookstore in Seattle, Sept. 9.

    Hood, on prominent display at the University Bookstore in Seattle, Sept. 9.

  2. Kindle: $9.95 on Amazon. (My share of the profits for the eBook is the same as for the hardcover, so for those of you asking which version is better for me, don’t worry—go with your preference.)
  3. PDF: $19.95 from TimmermanReport.com. (I get the biggest share from this method, but I realize it’s not most people’s preferred way to read.)
  4. Bulk orders. If you want to give books as corporate gifts, you can buy them from me in multiples of 14 copies. Email me at luke@timmermanreport.com. These will be shipped directly from the printer and arrive within two weeks.

Here’s where to catch me this week in Boston/Cambridge:

BioPharm America

I’m doing three formal sessions (and one informal meetup) at this big partnering event hosted by EBD Group.

11:45 am-12:15 pm, Sept. 14. Boston Marriott Copley, 110 Huntington Ave. Boston. “Maximizing Your Social Media Presence.” I’ll be moderating a panel with Adam Feuerstein of TheStreet, Laura Strong of Quintessence Bioscience, and Ryan McBride of Novartis Institutes for Biomedical Research.

12:30 pm-1:30 pm, Sept. 14 “Food for Thought: What Makes Serial Entrepreneurs Tick?” I’m moderating this panel with Michael Gilman, Tillman Gerngross, and Troy Wilson.

5 pm-6 pm, Sept. 14. Boston Marriott Copley. Book signing in the exhibit area.

6:30 pm. Champions Sports Bar at the Boston Marriott Copley. Adam Feuerstein of TheStreet and I are organizing an informal biotech chat/networking event. Join us for a beer and some biotech talk.

MIT Koch Institute

Noon-1 pm, Sept. 15. Koch Institute for Integrative Cancer Research at MIT. I’ll be doing a brownbag session on Hood with students, postdocs, faculty and staff.

Mass Innovation Labs

5:30-5:50 pm ET, Sept. 15. Mass Innovation Labs, 675 West Kendall St., Cambridge, MA. Journalist Wade Roush will facilitate a Q&A on Hood during happy hour at this startup hotspot. Free admission, and all Boston-area TR subscribers are welcome, but I ask that you RSVP at luke@timmermanreport.com so I can give an accurate headcount.

See you out there. And don’t forget to leave a review of Hood on Amazon!

9
Sep
2016

Reflections from Yellowstone, EpiPen’s Foul Aftermath, and Saunders Steps Up

I’m back in the saddle after a family camping trip in Yellowstone National Park. It was a wonderful way to see some beautiful scenery and tons of bison, elk, deer, bear, and even a wolf. My four-year-old daughter was fascinated by the geysers, and didn’t whine too much about the long drive. Importantly for those of us in the information economy, it’s a great way to go off the grid, get some digital detox, and get ready for a busy fall of biotech news and analysis.

To catch up on the major happenings from before and after Labor Day, get your Frontpoints.

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15
Aug
2016

Watching the Watchdog: How Does ICER Determine ‘Fair’ Prices for Drugs?

With recent scandals over price increases for old drugs (Turing and Valeant Pharmaceuticals), public uproar regarding the pricing of high-profile new drugs (Sovaldi), as well as the general recognition of significant financial strain for patients being treated with specialty pharmaceuticals, there has been a growing demand for methods to determine whether or not the healthcare system is getting good value for the money it spends on pharmaceuticals. In response, several organizations have created “value frameworks” used to measure medical product value and determine whether or not the manufacturers’ list prices are merited.

Leora Schiff, principal, Altius Strategy Consulting

Leora Schiff, principal, Altius Strategy Consulting

Among these organizations is the non-profit Institute for Clinical and Economic Review (ICER). ICER positions itself as a “trustworthy independent source to help assess how valuable a new drug really is”[1]. ICER’s value framework combines cost-effectiveness budget impact analyses to determine “value-based price benchmarks”, a range of drug prices that ICER considers to be reasonable given the drug’s demonstrated benefits, and impact on payer budgets.

ICER has published reports on the cost-effectiveness and budget impact of a number of important new drugs including the hepatitis C drug Sovaldi, the cholesterol-lowering PCSK9 inhibitor class of drugs and drugs for the treatment of multiple myeloma. Through press releases, conference presentations and statements in the media, ICER has been disseminating the results of its research not only to professional healthcare stakeholders, but also to a broader public audience.

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5
Aug
2016

BMS Loss is Merck’s Gain, Biogen Takeover Speculation, & GSK’s Bioelectronics Foray

This summer, I’ve been dialling down my social media consumption and dialing up my old-fashioned book reading. Neil Postman’s “Amusing Ourselves to Death” knocked me on my ass 20 years ago as a journalism student, and did it again upon re-reading a couple weeks ago. It’s about the corrosive, and dangerous, effect of television on public discourse. Given the rise of an authoritarian strongman-entertainer wannabe in the U.S. who gets excellent TV ratings, this book is even more relevant than it was when first published 30 years ago.

That’s a dark read, but fortunately I got some lighter recommendations from thoughtful biotech pros when I asked this week. I passed along some excellent suggestions on Forbes.

It’s always a good time to re-dedicate oneself to reading old-fashioned books, there was a lot of fast-moving action in biotech online. Catch up on a dizzying array of headlines, along with some healthy perspective, in this week’s Frontpoints.

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3
Aug
2016

Donating Your Personal Data: Giving Pay-to-Play a Whole New Meaning

I read an article that the San Francisco 49ers are kicking off a quarterback competition to decide whether Colin Kaepernick or Blaine Gabbert gets the starting job. They will be put through their athletic paces in training camp, where all of their teammates will participate in determining the outcome.  But based on a recent deal the 49ers struck, with Orig3n, Inc., I was wondering why are they making this so complicated?

Lisa Suennen, managing partner, Venture Valkyrie Consulting.

Lisa Suennen, managing partner, Venture Valkyrie Consulting.

Allow me to explain. The 49ers have entered into a deal with Orig3n to reward fans for making genetic contributions to populate their ever-growing database that informs  pharmaceutical research.  In fact, Orig3n has two uses for the gene samples they collect: the first is to build up LifeCapsule, which the company claims is the world’s largest blood cell repository dedicated to supporting regenerative medicine. That’s the primary focus of the 49ers deal.

The second use is for LifeProfiles, where consumers can learn more about their own genetic profiles in the quest to know their own propensity for greatness (by, for instance, comparing their sample to those from the 49ers bench).  These tests offer the following claim: “the SUPERHERO assessment decodes secret information in your DNA, giving you insights into where your super-powers lie.”.  They tell you, with some sheen of scientific specificity, whether the footnote on your cape should reflect your extreme intelligence, strength or speed.

So why not save everyone some time and just run Colin and Blaine through these tests?

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18
Jul
2016

Want to Help Biopharma’s Reputation Problem? Tackle Poverty

I make a lot of lists. It’s not the right approach for everyone, but it works for me, especially when there’s a lot going on in my life. Sometimes the lists are things to do, things to buy, or just ways to organize my thoughts. And yet my life isn’t particularly complicated or hard. When I dig into our healthcare system and how convoluted it can be, I’m thankful that I don’t have a lot of decisions to make or choices to research.

That’s not the case for everyone.

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11
Jul
2016

The Best Boring Companies in Biotech

Good companies don’t always make for good stories. Our current fragmented —and increasingly fragmenting — media environment demands minute-to-minute 24/7 excitement, drama. We need twists and turns, ups and downs, heroes and villains to grab public attention. Celebrity, controversy and plain old shouting tend to drown out serious conversation about ideas.

Ever wonder why 23andMe is a household name, yet only insiders have ever heard of Illumina? Or why Sarepta Therapeutics throws off so much heat from a mere 12-patient study?

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7
Jul
2016

How Brexit Changed the Way I Look at Biopharma’s Reputation Problem

You may have heard something recently about Britain, the European Union (EU), some vote or other, chaos, turmoil, blah, blah, blah…You might also have heard how the presumptive Republican nominee for President of the United States has gotten to that position by identifying a strong thread of anti-establishment, populist sentiment in the US. And you may have heard that biotech and pharma is suffering from a reputation problem.

Kyle Serikawa

Kyle Serikawa

One of these things is not like the other, right?

I’m not so sure.

That biopharma has a reputation problem isn’t in doubt. The question, though, is how the industry got here. I want to know this because, thinking like many drug developers, I believe that by knowing the cause of a condition a fix can more easily be found.

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5
Jul
2016

The Schultz Lab Alumni: See Where They Are Now

On the East Coast, there’s Stu Schreiber. On the West Coast, there’s Peter Schultz.

These twin towers of chemistry/chemical biology have had parallel careers, like Larry Bird and Magic Johnson back in the day. These chemistry gladiators both turn 60 this year. Each blazed trails in their field, started companies, had their rivalries, and trained a constellation of bright minds in academia and biotech.

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30
Jun
2016

Pfizer, GSK, AbbVie Join $51.5M Bet on Computational Drug Discovery Shop, Morphic Therapeutic

Integrins were a big deal in the early days of biotech in the 1980s and 1990s. They looked like a promising class of receptors found on cells implicated in many diseases. Billion-dollar dreams were in the air. Then, thunderstruck by dangerous and surprising side effects, integrin-directed drugs were tossed in the trash.

But integrin biology is making a comeback with a big-name scientific entrepreneur from Harvard Medical School, and a healthy shot of cash from Big Pharma. Waltham, Mass.-based Morphic Therapeutic is announcing today it has raised $51.5 million in a Series A venture financing to build on new understanding from Tim Springer’s lab at Harvard Medical School about the shifting, not static, state of integrin receptors. GlaxoSmithKline’s SR One venture group and Pfizer Venture Investment co-led the financing, and were joined by Omega Funds, AbbVie Ventures, Tim Springer, Polaris Partners, Schrodinger, and Shangpharma Investment Group.

Integrins have long fascinated biologists as drug targets, because they are receptors commonly found on the surface of about 90 percent of human cells, and are thought to play a role in crucial cell processes—cell survival, differentiation, and migration. Acute coronary disease, autoimmune diseases, clotting disorders, and many more common ailments seemed to be in reach. Early targeted antibodies were made to specifically inhibit integrin targets, and a few are still around—Biogen’s natalizumab (Tysabri) is one famously potent integrin-directed antibody for multiple sclerosis that also comes with some serious warnings about side effects.

Praveen Tipirneni, CEO, Morphic Therapeutic

Praveen Tipirneni, CEO, Morphic Therapeutic

Early antibodies were good at specifically binding with these targets. The flaw, as is usually the case in biotech, was a lack of understanding of the underlying biology, said Morphic CEO Praveen Tipirneni. This time around, Springer’s lab has used advances in X-ray crystallography to obtain precise structural information on integrin targets. That led to the realization that the receptors don’t behave in a static way. They “morph” into specific shapes, including a closed one and an open one.

That’s a crucial insight. It means that if you make a specific antibody that inhibits an integrin in the closed state, and it does that job well, it can also have the opposite effect, by stimulating another activity. That can cause dreaded side effects, or even make a disease worse.

Armed with the vivid (and proprietary) crystal structure information of integrins from Springer’s lab, Morphic started working with the computational group at Schrodinger. The computational group runs calculations and simulations to find small-molecule chemical compounds, the kind which can be made into oral pills, that have the optimal binding characteristics to inhibit an integrin in its closed state, and keep it locked even in an open state, Tipirneni said.

Here’s what Springer said about the opportunity, in an email:

Almost every major pharma rode a wave of excitement on integrins as targets in the 1990s but the wave crashed when orally available small molecule antagonists failed to prevent thrombosis and even made patients worse. All this work was done without benefit of crystal structures or knowledge that integrins can morph from one conformational state (shape) to another. We now have extensive structural knowledge of integrins, much of it from my lab, and my lab also knows how to avoid the harmful effects of inhibitors that relate to integrin morphing and has licensed this knowledge to Morphic Therapeutic. Recent advances with biologicals as therapeutics have further proven the value of targeting integrins. With our structural knowledge of integrins, ability to solve structures of new drug-integrin complexes, ability to morph integrins into the desired conformational state, and Schrodinger’s expertise in computational design, we are in position to ride a new wave of integrin drug discovery all the way to FDA approval.

Springer is well-known for his scientific work that dates to the early days of integrin discovery, as well as his entrepreneurial ventures, especially LeukoSite, a company acquired by Millennium Pharmaceuticals. More recently, one of Springer’s angel investments, Selecta Biosciences, went public.

Morphic got started with Springer teaming up with Polaris Partners, an early-stage venture firm, and one of its partners, Kevin Bitterman (for more on Bitterman, see “12 VCs Who Matter, But You Never Read About” in TR). Bitterman helped bring in Tipirneni, a former sales and marketing executive at Cubist Pharmaceuticals, as the permanent CEO. The operation is still small, with just 15 employees, and at an early stage. It doesn’t yet have any drug candidates in clinical trials, or a publicly declared lead candidate.

By raising a lot of money early, Morphic will be able to move forward simultaneously on several fronts. Inflammatory bowel disease is a “high priority” of focus, given the recent success of Takeda Pharmaceuticals, which analysts expect will generate $2.5 billion a year in peak sales for vedolizumab (Entyvio), a drug aimed member of the integrin family. Fibrosis is another logical disease category for an integrin discovery shop, and there are potential novel strategies for cancer as well, Tipirneni said. About two dozen members of the integrin family are known, and about 15 appear to have good potential as drug targets, Tipirneni said.

Other companies are paying attention. Pliant Therapeutics, a Third Rock Ventures-backed fibrosis drug developer that I wrote about last year for Forbes, is one company working in an overlapping area of R&D, Tipirneni said. Cambridge, Mass.-based Nimbus Therapeutics is another company that leans heavily on a computational drug discovery approach in collaboration with Schrodinger, although against a different set of biological targets, Tipirneni said.

For a detailed review of the history of trials and tribulations with integrin-targeted drug development, see this 2011 review article in the journal Theranostics.

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