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Maybe it’s the summertime lulls that make it a good time for people to change jobs. For whatever reason, I seemed to notice more than the usual volume of notable personnel moves over the past week.
Catch up on those impactful switches, and other matters of import, in this week’s Frontpoints.
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.
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”. 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.
This week, I saw more than the usual volume of press releases that said “Company ABC will be presenting at Investor Conference XYZ.” Dog days of summer are here, to be sure.
If you don’t have sand between your toes, here are the deals, data, financings and worthwhile reads to catch up on in Frontpoints.
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.
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?
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?
As long as I can remember on the biotech beat, I’ve heard people talk about the potential of gene therapy for hemophilia. Now it appears to be actually happening, thanks to some encouraging data from BioMarin Pharmaceutical.
For that, and much more from the week in biotech, get your Frontpoints.
Beach reading season is here. I just got back from camping on Canada’s Vancouver Island, and marveling at the amazing diversity of marine life in its rocky beach tidepools. Hopefully, you are already on your own beach, or planning your own respite, so I’ll attempt to keep this week’s Frontpoints brief.
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.
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?
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.
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.
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.
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.
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.
Some very powerful people drove attention — positive attention — to biotech this week. It’s hard to have a shared national conversation about anything in an increasingly fragmented media world, but wealth, power, and celebrity does tend to cut through the clutter. This week, some of those folks focused on opportunities in precision medicine, cancer, antibiotics, and gene editing. Not a bad week.
For more on the ups and downs, the deals, data, and chatter that garnered attention this week, read your weekly Frontpoints.
One year ago, biotech was abuzz about the “crossover” phenomenon. Now comes the hangover.
Memories are short these days, but as a refresher, many deep-pocketed investors were making outsized returns a year ago in biotech stocks. As stocks got pricey, some chose to “crossover” to the adventurous terrain of private, venture-backed biotech. There were good technologies and management teams to be found. Prices were relatively cheap. I gathered data and wrote here about the big names who started showing up regularly in venture syndicates. Many of the private companies looked like solid bets to go public within 12 months and achieve fast liquidity for everyone involved.
This year, of course, it’s a different story.
Summer hasn’t quite arrived for me at home in Seattle, but blood temperatures seem to be rising. There was a horrific mass shooting in Orlando, a bizarre-beyond-belief Presidential election just started, and Zika posing a growing global public health threat.
Believe it or not, there was some good news from around the world of biotech. You’ll just have to take the good and the bad in the weekly Frontpoints.
BIO really, really likes Newt Gingrich. The former Speaker of the House was one of the big name guests at the BIO International Convention last week in San Francisco. Gingrich joined Stephanie Cutter and Candy Crowley on a keynote chat with BIO president Jim Greenwood discussing election-year politics. Gingrich also did a book signing.
BIO had nice things to say about its other commentators, but it heaped on the praise with a spoon for Gingrich. The man, in the trade group’s words, is a “distinguished world leader, powerful visionary, and political vanguard.”
I am available to speak about biotech trends at public and private biotech events. See me for details.