The Big Red Biotech Blog

India and China Start Ups?

Here's a post from In the Pipeline discssing latest information on status of VC investment in biopharm R&D with start ups in India and China.  Conclusion?  VC activity is still fairly low by comparison to US standards.  The study author's estimate was that approximately $1.7 B had been invested in 116 firms over the past 12-13 years.  By comparison, the US industry would expect about that much investment activity per quarter. 

No where to go but up -- and that does appear to be the direction in which things are moving -- albeit slower perhaps than one might imagine.

Posted by Bruce Lehr Mar 8th 2013. 

EMA proposes virus testing for food sector-derived trypsin used in drugmaking

The EMA says that food industry controls on porcine-trypsin do NOT guarantee that it is virus-free. The agency is therefore proposing new procedures for drug and vaccine makers who use it as part of their process.

EMA proposes virus testing for food sector-derived trypsin used in drugmaking.

This arises most recently from the rotavirus vaccine contamination event by porcine circoviruses that was ultimately traced to porcine trypsin used in its manufacture. Based on events like this, the EMA made new recommendations:

• Use only trypsin from glands from pigs that have appropriate official health certificates
• Test for specific viruses using both porcine and primate cell lines
• It advises use of two complementary virus reduction steps in processing -- e.g. low pH and irradiation

Under the guidelines proposed the drug or vaccine producer would have the responsibility to ensure proper testing (via its vendor if desired) and safety processing steps had been undertaken.

The EMA also noted that potential alternatives to trypsin could be used from recombinant sources or could be plant-derived (like TrypZean) saying ---- "the use of bacterial or plant derived recombinant trypsin minimizes in principle the risk for animal virus contamination and the application of such alternatives is therefore encouraged."

Posted by Bruce Lehr Mar 6th 2013.

SAFC is Adding Powdered Medium Plant in Scotland

SAFC will expand manufacturing capacity for dry powdered media in its Irvine, Scotland facility to serve local European customers.  The new plant will give SAFC redundant powder manufacuring capability in both Europe and the US.

SAFC is adding large-scale production of bulk dry power media and reagents at its manufacturing plant in Irvine, Scotland.

The new facility will have the same processes, same procedures, same validation packages and use the same raw materials as the US plant in Kansas, which itself expanded in the past year. European customers have expressed a strong interest in qualifying the new plant. The plant is slated to open in the first quarter of 2014.

Posted by Bruce Lehr Mar 6th 2013.

I Liked This Post on Leadership

I read this post in Xconomy today and liked it.  I think that it rings true and they we all can relate to its message that sometimes less is more and that you know when you need to back off or ask for help.

Enjoy.

Posted by Bruce Lehr Mar 6th 2013.

 

 

SMILE! BE HAPPY!

Posted by Bruce Lehr Mar 6th 2013

EC Funds up to 200 New Rare Disease Drugs

Fierce Biotech reports this afternoon that the European commission has pledged $187M in new research funds aimed at helping develop up to 200 new drugs for rare diseases by the year 2020.  Rare diseases have become a popular target these days for drug makers so the uptake of the R&D in these projects should be good. Part of the popularity with developers is that these drugs can take a more streamlined path in clinical trials and with regulatory approval.  Another popular feature is that these drugs often command among the highest prices per patient in the biotech world.   

It's ironic that the previous post talks about Express Scripts latest analysis of drug prices. The so-called Specialty category experienced an 18%+ growth rate last year and that is expected to continue. Drugs to combat rare diseases typcially fall squarely in this category as well.  So these drugs while provided needed therapies for the afflicted will tend to further exacerbate the financial and issues that 3rd party payers and governments are grappling with. 

It's further ironic to me that many of the groups that advocate for the development of more rare disease drugs -- which have been historically neglected -- tend to also be the first groups that raise objections to ultra-high prices these drugs often command. Therein lies the rub.

Posted by Bruce Lehr Mar 5th 2013.

2012 Rx Spending Report

Every year Express Scripts publishes and analysis spending on prescripotion drugs in the US. 

Rx Spending: What Goes Down, May Go Back Up // Pharmalot.

Here are some of the factoids for 2012:

• Spending by private health insurers dropped 1.5%
• Spending on brand name medications rose 12.5%
• Generic drug prices fell a whopping 24%
• Spending on specialty meidcations rose a whopping 18.4%
• Overall this resulted in a 2.7% increase in spending on drugs

The 2.7% figure doesn't sound so bad and in fact is consistent with the previous year - but what is more worrying to 3rd party payers is that specialty drugs that account for only 2% of the populaion accounted for 24.5% of the total spending.  This is the highest percentage on record -- and more specialty medicines are on their way. 

Here's some more facts just for the specialty meds catagory:

• Spending on RA drugs rose 23%
• Spending on cancer meds increased 25.9%
• Spending on Hep C drugs went up 33.7% -- highest for an therapeutic area
• Spending on Hep C drugs (according to Express Scripts) is expected to increase another 32.3% this year and an incredible 56.3% in 2014)
• Diabetes is the category where te most money is spent and it went up 11% in 2012

What's really troublesome to payers is that more and more specialty medicines are expected to be introduced in the next several years.  Last year, 39 new drugs were approved by FDA including 22 specialty drugs, many of which cost more than $10,000 per month to utilize. Since, Big Pharma in concentrating its efforts on producing more of these drugs, the payers are worried how they will pay and control costs. 

Personally, I think this will be another economic and political driver to figure out how to make biosimilars a bigger part of the mix in te US to offer price competition in some of this specialty categories -- like RA for example. But in the real short term, we can expect more new drugs in specialty categories and more spending.  An alternative would be to adopt models to restrict access to newer drugs pending trial of older cheaper ones first -- but i don't think that will (or should) happen across the board. I do think a biosimilar model that works in the US needs to be developed. See Fierce Biotech too.  

 

Posted by Bruce Lehr Mar 5th 2012.

 

FDA Begins Crackdown On ‘Risky’ Compounders

The FDA says it has begun a new crackdown on compounding pharmacies that is targeting about 30 so-called ‘high risk’ operations in nearly a dozen states.  Late last week, it disclosed that inspections reports were issued for four compounders for a variety of serious manufacturing violations.

FDA Begins Crackdown On ‘Risky’ Compounders // Pharmalot.

The agency used a “risk-based model that relied on adverse event reports, historical inspection data, reports of quality problems and public information, to identify a number of pharmacies that may pose higher risks and that are known to have engaged in sterile compounding. These include many with which the FDA has had issues in the past.”

The agency has been criticized for moving too slow in the past and has at least one house committee looking over its shoulder.  In response to this mounting pressure, the FDA has begun coordinating its efforts with state boards of pharmacy in identifying compounders and making its findings available to all states on its website, since many of the riskier compounders ship across state lines.

As The Thing might say, "It's clobbering time."  Of course, FDA might have to ask permission to do so and be relegated to refering cases to state boards for actual action.  Not ideal.

 

 

 

Posted by Bruce Lehr Mar 4th 2013.

Build Blockbusters One Block at a Time

Fierce Biotech reports that Novartis CEO, Joe Jimenez, says that the company will switch away from expecting single indication drugs to result in blockbuster status (i.e. > $1 B in annual sales).  Instead, Novartis will introduce drugs, gain approval as quickly as possible, and then extend the drug's indications -- perhaps in niche markets -- to cobble together its $1 B in annaul sales across all the indications.

In other words, they will continue to do what they have been doing in cancer fields for a long time. Just build em one block at a time.

Posted by Bruce Lehr Mar 4th 2013.

Luke Takes Umbrage to LaSalle Biotech Hub Rankings

Here's a good article from Xconomy's BioBeat today.  In it, Luke Timmerman calls to task a recent report from Jones Lang LaSalle purportedly ranking the top biotech hubs in America and the World.  Luke argues persuasively they got it wrong and calls for someone to provide a "real analysis" and produce a better set of rankings.

I think most of his points have merit.  Read the blog post yourself and decide. I do think a careful consideration of most, if not all, of his 15 points would provide a more holistic ranking and one that would be more robust in its conclusions.

Posted by Bruce Lehr Mar 4th 2013.

India Rejects Bayer Appeal On Compulsory License

India's Intellectual Property Appellate board rejected Bayer's appeal fighting a compulsory license to its drug Nexavar.  The license was granted last year to the generic manufacturer Natco when authorities determined that Nexavar cost to much for Indian consumers -- patents or no stinkin' patents.

India Rejects Bayer Appeal On Compulsory License // Pharmalot.

Needless to say, Bayer was not impressed.   “The order of the Intellectual Property Appellate Board weakens the international patent system and endangers pharmaceutical research,” the Bayer (BAY) spokesman writes.

Consumer advocates on the other hand were dancing in the streets.  “The decision confirms that the Indian patent office is able to use all the means legally at its disposal to check the abuse of patents and open up access to affordable versions of patented medicines,” says Leena Menghaney, the access to medicines campaign manager in India for Medicins Sans Frontierers, also known as Doctors Without Borders. “Most importantly, the decision means that the way has been paved for compulsory licenses to be issued on other drugs now patented in India and priced out of affordable reach.”

This can't be good news for multi-national pharma companies in India if the decision stands -- not unless they want to drop prices by 90%.  Ouch!  Certainly, there appear to be disincentives developing for Western companies to introduce new drugs to India if their patents are not to be respected. 

Posted by Bruce Lehr Mar 4th 2013.

Life Sci VC Reviews 2013 Deals

Here's some interesting commentary and facts about some of the early biotech structured deals in 2013 between Pharma and the small pre-clin stage biotechs.  The post is from Bruce Booth of Life Sci VC.

The upshot is that the deals look good so far.   All appear to be high science plays (two orphans), share risk and capability between partners, are capital efficient, were driven by interest in the program and have low asset bases, and could all return outsized downstream returns (albeit low probability for full pay-outs). 

The upfront return (IRR) on only one deal is particularly attractive. Questions now arise as to whether there will be more movement toward pre-clin type deals.  If so, will this shorten or lengthen the time to liquidity for drug discovery companies once the big pharma bureacracy takes over?

Posted by Bruce Lehr Mar 1st 2013.

India May Drop Compulsory Licenses Based On Price

Does it really matter?  It appears that India will no longer issue compulsory licenses to generic or biosimilar manufacturers to make a patented drug based solely on whether that patented drug's price is perceived to be too high.  Good news for the multi-nationals right?  They seem to think so.

India May Drop Compulsory Licenses Based On Price // Pharmalot.

Instead India has set up a new Committee to set pricing for patented drugs by comparing to prices negotiated between manufacturers and governments in US, Canada, France, Australia and New Zealand.  The thought seems to be this comparison will lead to higher prices than what consumer advocates in India would like to see but less than what pharma companies would charge on their own, but woud still be acceptable to these same companies.

But....the Committee would take per capita gross national income (GNI) and purchasing power parity in these countries into account.  The ratio of GNI in comparison country/GNI in Indiawould be calculated and used to adjust the price of the medicine for India from that of the comparison country.  This adjusted price would also serve potentially as the starting point for future reductions.

Ok. No compulsory licensing. Yes to a Pricing Committee.  What if they price products lower that what pharma is expecting?  Are we back where we were?

Posted by Bruce Lehr Mar 1st 2013.

Baseball and New Drugs

I love baseball.  The actual game really and discussions around it.  I'm less into the statistics but they are a usual part of baseball discussions.  I'm even less into sabermetrics though I do acknowledge they provide another interesting way to look at the stats of the game and do have merit in potential decision making in the game.

So I was really happy to see this morning's Xconomy post that compared evaluating baseball with the evaluating a drug development pipeline.  I thought many of the analogis held up pretty well -- you'll have to read it and decide for yourself. But one thing is correct in my view, we should continue to strive to find the best metrics to look at the success of drug development and open our minds toward doing this and operating in a fashion different than our historic past.  It is well pas that time.

Posted by Bruce Lehr Mar 1st 2013.  

Smoky Mac N' Cheese. Yum! Yum!

Here's what you gonna need.

• 8 ounces dry macaroni
• 1/2 pound thick sliced apple wood smoked bacon
• 2 cups half and half
• 2 tablespoons all-purpose flour
• 1 tablespoon white truffle butter
• 8 ounces smoked gouda, grated
• 1  teaspoon smoked paprika
• sea salt, as needed for cooking macaroni

To Cook:   

1. Preheat oven to 400 degrees F.  Lightly butter baking dish or dishes.  This recipe will make enough for 10 1/2 cup ramekins.
2. Line a large, shallow baking sheet with aluminum foil.  Place the bacon side-by-side on
   the baking pan.  Bake in oven until crispy, about 20 minutes for thick-cut bacon.  Remove from oven and place on several layers of paper towels to drain.  (Do not turn off oven!)  Chop bacon into smallish pieces and set aside.
3. Pour 8 cups of water into a 3-quart saucepot.  Salt water according to macaroni package.  Bring the salted water to a boil and carefully add the macaroni to the water.  Cook only until macaroni is at a slightly firm al dente (approximately seven minutes, but depending on the quality of the macaroni, cooking times may vary).  DO NOT over-cook the pasta.  It is necessary for the macaroni to be a bit undercooked because it is going to absorb additional liquid from the sauce.
4. Drain cooked macaroni in a strainer/colander and rinse under cool water to stop the cooking process.  Drain well, then set aside while the sauce is prepared.
5. In a 3-quart saucepot, add the half and half (it's ok to use the same saucepot the macaroni was cooked in).  Whisk in the 2 tablespoons flour until smooth.  Add the white truffle butter and the smoked paprika to the half and half mixture.  Heat over medium or medium-low heat until hot and beginning to thicken, whisking constantly.
6. When the sauce is hot, stir in the cheese.  Continue to stir until cheese is melted and sauce is smooth.  Turn off heat.  Reserve about 1/4 cup of the chopped bacon, set aside.  Stir the remaining bacon into the sauce.  Taste and add salt, if needed.
7. Fold the macaroni and the sauce together.  Pour macaroni into the ramekins or casserole dish.  To help catch potential spills, place baking dish or dishes on a baking pan.  Top with reserved bacon.
8. Bake for approximately 10-20 minutes, depending on the size of the container, until hot and bubbly.  Serve immediately.

To Drink:  

Trenel Sanit-Veran 2010

Pale gold in color, this Saint-Veran offers up an intense and complex combination of white flowers, green apple, and ripe peach aromas.  The palate is soft on the entry with lemony apple flavors and a clean, crisp finish.

Saint-Veran is an appellation of dry white wines produced in the southern half of the Maconnais sub-region of Burgundy.  This clean and crisp 100% Chardonnay is a perfect compliment to the rich and creamy mac and cheese.

Recipe courtesy of Terri at That's Some Good Cookin' Blog.

Posted by Bruce Lehr Feb 28th 2013.

 

Ooooh! This isn't Good for US Manufacturing Economy

Here's a sobering report from Xconomy on a new study out of MIT.  In it, MIT researchers claim that US manufacturers (150 in study from diverse industries) hit problems when they go to scale operations at full commercial level.  This leads them to seek foreign investors and set up foreign facilities. Wave bye-bye to some good jobs.

That's not good and is why there is beginning to be more clamoring for manufacturing supports in the US (i.e. tax credits and the like).  It would be nice to have the manufacturing base remain strong (US is still No. 1) bu there is a trend toward moving to Asia and other sites abroad. We need to nip it.

Posted by Bruce Lehr Feb 28th 2013.

 

 

Innovation Lost

Here's the latest epistle from Innovate on Purpose on why innovation desires from the C suite are often lost in translation to the rest of the organization.  Some of the issues discussed include: conflict between near term sales/profitability goals and longer term innovation, prioritization conflicts around activities, and lack of innovation skills in the organization. 

The conclusion is that until the concept of innovation is firmly established and constantly communicated and resourced, it will be hard for firms to innovate consistently. Innovation is not a project but a corporate capability.

Posted by Bruce Lehr Feb 28th 2013. 

Roche's T-DM1 Crosses Finish Line as Kadcyla

The second ADC in the past year has crossed the finish line to be approved by the FDA.  This time it is Roche's (Genentech) Kadcyla to be used in the treatment of HER2 positive breast cancers.  The drug has performed in clinicals -- despite missing out on accelerated approval -- as it was widely expeced to be approved this time around.

"Kadcyla is an antibody-drug conjugate representing a completely new way to treat HER2-positive metastatic breast cancer, and it helped people in the EMILIA study live nearly six months longer," said Hal Barron, Roche's chief medical officer and head of global product development

Unfortunately, it is priced at $9800 per month per patient and is expected to cost about $94,000 for a single course of treatment -- about double its predeccessor (non-ADC) Herceptin alone.  While I am excited to see ADCs make it to market --- and to be successful clinically.  I do worry about pricing models that have most new biological drugs -- particularly in oncology areas --- costing a minimum of 6 figures per patient course. 

It is the type of thing that does not seem sustainable in the current world fiscal and economic crisis.  See reports from Fierce Biotech and Xconomy for more on the medicine and FDA approval.

On a happier economic note, Genetech (no doubt boosted by successes like Kadcyla) announced that it will be adding 600 new staff in the US.  Just to put that into perspective, 600 staffers would easily handle the work of more than 30 average biotechs in pursuit of their first drug approval.  Genentech says it prefers to promote from within, making a number of these new hires replacements for upward bound employees. And they pay well, with many of these positions tied to starting salaries that range from $70,000 to $80,000 a year.  At least this company is hiring for more success as it experiences new successes. Huzzah!  See Fierce Biotech.

Posted by Bruce Lehr Feb 23rd 2013

 

PS - There is no truth to the rumor that Kadcyla battled Xalkori to a draw over Tokyo for control of that fine metropolis.    

High Drug Pricing Next Industry Challenge?

The good news from 2012 might be that more drugs were actually approved (39) than in the very recent past -- but the bad news is that many were VERY expensive.  Xconomy's Luke Timmerman did a nice piece summarizing this a couple of days ago.  It seems that many of the very high priced drugs are coming into the oncology and "rare disease" or orphan arenas.  Many of these drugs carry price tags into the serious 6 digits and are $300K or more annually.  Is this sustainable?  Are any of these products worth it?

Those are questions that the industry must face as healthcare systems around the world struggle with costs.  Individual patients oft times may have to struggle as well depending on what type of insurance coverage they carry.  And even in the industry, some players must worry that the action of their peers might be in danger of killing a golden goose if these prices are perceived publicly -- even by physicians -- as being out of line with reality. 

What's a drug worth?  One answer might be what the market is willing to pay.  But will that number change as government and other third party payers become budget constricted. Yes, is the obvious answer.  From a payers perspective, they want a price that they feel is commeasurate with the benefits that are received.  Same from a patient's perspective, I hope.  Do I feel I got my money's worth in terms of improved health or quality of life? 

There is no doubt that this issue will not go away. 

Posted by Bruce Lehr Feb 13th 2013.

New Drug Approvals in 2012

Here's a quick piece from CEN-Online that overviews and discusses the 39 new drug approvals by FDA in 2012.  The good news is that we have seen a 2 year trend now where more drugs have been approved than in the recent past.  This may say that changes being made to R&D departments throughout the industry are starting to have an effect but more than likely it is too early to make such a statement -- however at least the trend is "good". 

It does appear that the relationships between FDA and the drug companies have improved and there is better information sharing and collaboration.  A troubling trend has been developing though with pricing and this may be the next hurdle that needs to be addressed in the industry and among goverments and payers -- not to mention patients and physicians.

Posted by Bruce Lehr February 13th 2013

 

FDA's Sprinter Pathway to Possible Approval

The FDA's new "breakthrough" designation may allow drug makers to get their products through to commercialization after only performing enhanced Phase I trials -- of course they have to meet the criterion and actually show their product works.  No small task.  However, this could be a game changer in terms of shortening the path to market and gaining return on investment --- not to mention giving patients a shot at a new treatment much earlier.

Vertex has already expoited this pathway twice in gaining approval for its cystic fibrosis medication Kalydeco and in testing its cobination therapy VX-809.  A third drug also has breaktrhough status and 18 more applications are pending with FDA.  It seems the race is on -- and its a sprint.

This will no doubt have many backers with drug makers, patients, payers and investors.  It offers many potential benefits for novel treatments of diseases with unmet needs in particular.  We'll just have to see if the program lives up to its advertisements.  See Fierce Biotech.

Posted by Bruce Lehr Feb 13th 2013.

A Real Cluster

Here's the new 2012 report on Life Science Clusters issued by Jones Lang LaSalle.  It highlights biotech clusters around the globe.  This year's top 10 ranking in the US follows with the change from last year's ranking in parentheses:

1. Greater Boston (no change)
2. San Diego (+5 spots)
3. San Francisco Bay area (no change)
4. Raleigh-Durham (+5 spots)
5. Philadelphia (+1 spot)
6. Washington DC metro area (-1 spot)
7. New Jersey/New York city (-5 spots)
8. Los Angeles/Orange county (-4 spots)
9. Minneapolis-St. Paul (-1 spot)
10. Seattle (no change)

Three major trends were cited in the report to account for some of the movement:

• M&As and growth of middle-market life science companies are benefiting smaller metropolitan areas
• Proximity to innovation -- including intellectual capital -- is more important than ever
• Build it (labs) and they will come -- states are agressively competing for life science as economic drivers for their region

You can access the whole report here - Download Life Sciences Cluster Report_Global_2012 

Posted by Bruce Lehr Feb 8th 2013

Where Has All The Pharma Talent Gone?

If this weren't so sad, it would be comical.  Big Pharma cans about 150,000 scientists since 2009 and now complains it can't find enough good talent to hire.  Need we say anymore? 

Where, Oh Where, Has All The Pharma Talent Gone? // Pharmalot.

  

 INTO THE MUD SCUM CHEMISTS!

Posted by Bruce Lehr Feb 5th 2013

One Little Indian

Late last year, GSK said it would release (anonymous) patient level information from clinical trials to allow researchers more access to provide independent analytics.  It is sticking to its guns by endorsing the AllTrials movement to do just that.  GSK also says that it is working on its web porthole that will provide researchers the promised open -- yet secure -- access to trial data.

Glaxo Clashes With PhRMA Over Data Disclosure // Pharmalot.

PhRMA -- which GSK belongs to -- and the Association for British Pharmaceutical Industry however both demur, saying it is a bad idea.  PhRMA claims that this level of disclosure will inhibit trial participants from signing up and also will promote second guessing of regulatory decisions -- both ideas being BAD in their view.

I guess they wouldn't be in favor of instant replay in baseball either?  Oh wait, I got them confused with Bud Selig.

Posted by Bruce Lehr Feb 5th 2013.

 

Actual Companion Diagnostics Sighting!

Xconomy published today on Qiagen's recent application to the FDA for the approval of a diagnostic test that looks at a mutation in the EGF receptor.  The test is useful in the diagnosis of the speed of disease spread in non-small cell lung cancers (NSCLC) -- 10 to 15 percent of Caucasions and 40 percent of Asians carry the mutation.  The test was developed in conjunction with Boehringer Ingelheim's cancer therapy, afatinib that targets the mutation -- and they've asked the FDA to approve both products simultaneously.

This type of "companion diagnostic" has long been talked about, particularly with cancers, as being advantageous to define the target patient population that will respond to the drug. This helps recruitment for clinical trials and to better ensure the drug will actually be effective in the trial. The proto-type for this concept is Roche's combination Herceptin and the HER2 positive breast cancer from the late 90's. But not much else has really reached the market since then.

With Qiagen's/BI's application, we expect to see a new surge in companion diagnostics-therapeutic combination being developed and brought forth. In fact, co-development of the therapy and the diagnostic is the major difference in these programs now compared to what may have been done in the past where a test was typically developed sometime well after the therapy was available.  The result is that the tests didn't work particularly well and only 1% of drugs in the US actually have a companion diagnostic -- most in cancer area.

That's changing as the FDA has encouraged this approach.  Qiagen says it now has 15 programs in progress -- including with not only BI -- but with big companies like Pfizer and AZ. Last year, Roche had a companion diagnostic it developed with its Zelboraf treatment for melanoma. Pfizer's Xalkori has a companion diagnostic developed by Abbott. Other drug-companion diagnostic programs in progress include the following companies:

• Synadex Pharmaceuticals and Ventana Medical with entinostat & NSCLC's 
• Seattle Genetics/Takeda and Ventana Medical with Adcetris & hopefully various cancers
• Qiagen and Bayer with tests therapies and tests for solid tumors
• Qiagen also has a similar deal with Pfizer
• Astellas Pharma and Roche with Tarceva and lung cancers

The industry and FDA are looking for more of these to be successful -- and would expect clinical trials to flow more smoothly and result in more approved drug applications. It remains to be seen if this will translate into reality -- though the promise is clearly there.

Posted by Bruce Lehr Jan 31st 2013.