About this Author
DBL%20Hendrix%20small.png College chemistry, 1983

Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: Twitter: Dereklowe

Chemistry and Drug Data: Drugbank
Chempedia Lab
Synthetic Pages
Organic Chemistry Portal
Not Voodoo

Chemistry and Pharma Blogs:
Org Prep Daily
The Haystack
A New Merck, Reviewed
Liberal Arts Chemistry
Electron Pusher
All Things Metathesis
C&E News Blogs
Chemiotics II
Chemical Space
Noel O'Blog
In Vivo Blog
Terra Sigilatta
BBSRC/Douglas Kell
Realizations in Biostatistics
ChemSpider Blog
Organic Chem - Education & Industry
Pharma Strategy Blog
No Name No Slogan
Practical Fragments
The Curious Wavefunction
Natural Product Man
Fragment Literature
Chemistry World Blog
Synthetic Nature
Chemistry Blog
Synthesizing Ideas
Eye on FDA
Chemical Forums
Symyx Blog
Sceptical Chymist
Lamentations on Chemistry
Computational Organic Chemistry
Mining Drugs
Henry Rzepa

Science Blogs and News:
Bad Science
The Loom
Uncertain Principles
Fierce Biotech
Blogs for Industry
Omics! Omics!
Young Female Scientist
Notional Slurry
Nobel Intent
SciTech Daily
Science Blog
Gene Expression (I)
Gene Expression (II)
Adventures in Ethics and Science
Transterrestrial Musings
Slashdot Science
Cosmic Variance
Biology News Net

Medical Blogs
DB's Medical Rants
Science-Based Medicine
Respectful Insolence
Diabetes Mine

Economics and Business
Marginal Revolution
The Volokh Conspiracy
Knowledge Problem

Politics / Current Events
Virginia Postrel
Belmont Club
Mickey Kaus

Belles Lettres
Uncouth Reflections
Arts and Letters Daily
In the Pipeline: Don't miss Derek Lowe's excellent commentary on drug discovery and the pharma industry in general at In the Pipeline

In the Pipeline

« Go Home, Gaijin | Main | Taking Risks - You Have To, So Do It Right »

June 24, 2014


Email This Entry

Posted by Derek

No time for a morning blog post - I'm too busy exhaling sighs of relief around here. I'll see everyone later on in the day (on some other topic entirely). What a business this is!

Comments (29) + TrackBacks (0) | Category: Clinical Trials


2. breathedeep on June 24, 2014 7:33 AM writes...

If it's related to the CF trials announcement, that's good news indeed. Congrats to patients and scientists alike.

Permalink to Comment

3. A Nonny Mouse on June 24, 2014 7:42 AM writes...

Well I hope that it goes better than the Pharmaxis treatment......

Permalink to Comment

5. Anonymous on June 24, 2014 8:51 AM writes...

50% jump in stock price. Nice if you have any. :-(

Permalink to Comment

6. PPedroso on June 24, 2014 9:10 AM writes...

I wonder what will be cost of the combination?

Nevertheless, great news for science and especially for the patients of this terrible disease!

Permalink to Comment

7. Anonymous on June 24, 2014 9:13 AM writes...

Congrats. Moving the needle at all in dF508 patients is a huge accomplishment and truly amazing for the patients and families.

Permalink to Comment

8. Hap on June 24, 2014 9:19 AM writes...

@6: the article suggested $160K/year. Vertex didn't say anything, though.

Permalink to Comment

9. TEd on June 24, 2014 10:15 AM writes...

Congratulations to all involved!

I tell people that good scientists are able to derive deep satisfaction from infrequent success. With that in mind, I hope the Vertex crowd enjoys a rich and boisterous day of celebration before waking to the failed hypotheses of tomorrow...

Permalink to Comment

10. Anonymous on June 24, 2014 10:19 AM writes...

Kalydeco offers a stark validation of the idea that--even if you start with a phenotypic screen--you need to characterize your target to:
1-show that your dose-limiting tox. (in animals) is on-target
2-rationally combine therapeutics. Drug space is too big to run all the clinicals without the insight

Permalink to Comment

11. David Formerly Known as a Chemist on June 24, 2014 10:22 AM writes...

Vertex is now bleeding in Pfizer-infested waters....

Permalink to Comment

12. PPedroso on June 24, 2014 10:27 AM writes...


Vertex is American. Pfizer wants fish from foreign waters.

Permalink to Comment

13. Ed on June 24, 2014 10:39 AM writes...

#12 expect an AZN-VRTX merger first then!

Permalink to Comment

14. Anonymous on June 24, 2014 11:14 AM writes...

Promising news indeed, for the patients, the company, its employees and shareholders. Congratulations!

Permalink to Comment

15. SteveM on June 24, 2014 11:59 AM writes...

Yes, congrats to the scientists.

Now comes the messy part - the business end. The therapy increased lung function from 10 to ??? percent for (only) 30% of the patients who received it.

So a 10% increase in function for 160 Grand a year? And for the 70% of the patients who had some benefit but less than 10%, will they still be prescribed it because it's better than nothing? Again, at 160 Grand a year?

There is no way that this R&D business model is sustainable once a broad spectrum of hyper-expensive niche drugs are placed in formularies.

This generally is going to devolve into a political and economic quagmire.

Permalink to Comment

16. anon on June 24, 2014 1:18 PM writes...

As a researcher that works for another rare disease company. I want to extend my congratulations! This is a great win for patients and Vertex. It's great to see a company stick to their guns from the beginning to the end. The slog through compound screening, pre-clinical efficacy studies, and now to the benefits observed in Ph3 is a great testament to what makes this industry great.

Permalink to Comment

17. Anon on June 24, 2014 1:22 PM writes...

Congratulations to Vertex scientists and patients!

Now we just have to get ready for the physician and politicians to complain about its price and efficacy...And journalists to botch the story.

Permalink to Comment

18. MoBio on June 24, 2014 1:45 PM writes...

The thing that caught my eye was not the small increase in FEV1 but rather the decrease in 'episodes' of the sort that frequently necessitate hospitalization. I'm interested to see if the FDA panel agrees the the primary end point of FEV1 (4% increase) is 'meaningful'....

Permalink to Comment

19. bank on June 24, 2014 2:20 PM writes...

@ SteveM,

A 10% increase in function may not appear to be a lot, but it can have big functional effects if your capacity is already very low.

Imagine trying to lift a weight that is 5% heavier then you can manage, and along comes something that boosts your strength by 10%.

Permalink to Comment

20. ronathan richardson on June 24, 2014 2:31 PM writes...

The decrease in rate of hospitalization during the study period (~45% in placebo vs 18% in 600mg combo) justifies a pretty high (though not astronomical) price tag--I think 150k is reasonable; 300k is not.

Permalink to Comment

21. Vaudaux on June 24, 2014 3:31 PM writes...

#15 et al: Note that FEV1 actually decreased a little in the placebo group, consistent with the gradual decline in lung function that these patients typically experience despite the standard-of-care treatment they are getting.

If long-term treatment with the combination yields further improvement of a couple of percentage points every year (which is completely unknown), the value over time will look pretty good - both for the patient's quality of life and for parameters with $$ values like hospitalization and time lost from work.

Permalink to Comment

22. newnickname on June 24, 2014 5:28 PM writes...

Kalydeco targets CFTR dysfunctional mutation G551D and VX-809 targets CFTR misfolding mutation F508del (taken from wikipedia). So VX-809 rescues some or all the misfolding and that makes it good enough for Kalydeco to come in and potentiate the ion channel?

Kalydeco has no effect on F508del but some effect on a better folded, even if not perfectly folded, F508del?

Permalink to Comment

23. jbosch on June 24, 2014 9:55 PM writes...

Regarding the CF story what saddens me a bit is the talk of how the stock prize will develop for Vertex - in particular at the Fierce Biotech blog. How about putting the patients and not the stock prize into focus? If those guys who make a good deal on the stock would donate to such programs would that be much better ?

The 10% improvement can mean a lot for lung function. I react with asthma on particular pollen, that luckily do not exist as much in US as they do in Germany. The FEV in the presence of those allergens led to a 50% decrease in lung volume. This is a not so funny experiment but it was part of a trial that I played the Guinea pig in. After receiving the drug the improvement was dramatic within 2 hours.

What I'm trying to say is that although the number seems low and perhaps minimal because it's not 100% for the individual it might really mean a lot.

Permalink to Comment

24. MDACC GS on June 25, 2014 12:07 AM writes...

To those who are overlooking it, the 30+% improvement in pulmonary exacerbation is a pretty big deal (as is the weight gain for patients).

Permalink to Comment

25. Bagnar on June 25, 2014 1:47 AM writes...

Yesterday. Conference with a french scientist...

And on one of his slides, a long citation from... Guess who ?

M. Derek Lowe !
Incredible !

Permalink to Comment

26. John on June 25, 2014 6:41 AM writes...

This is a very good news. Congratulations!

Permalink to Comment

27. Da Vinci on June 25, 2014 7:03 AM writes...

@23, Forget it, this is industry, nobody cares about the patients.

Permalink to Comment

28. emjeff on June 25, 2014 10:13 AM writes...

Good news for Vertex, but if they followed Andrew Witty's philosophy, they would immediately sell the combination for some quick cash and lay off everyone who was involved in the development.

Permalink to Comment

29. cliffintokyo on June 26, 2014 9:18 AM writes...

Now I undestand what dis-allusioning (sic) cynicism is...

Permalink to Comment


Remember Me?


Email this entry to:

Your email address:

Message (optional):

The Last Post
The GSK Layoffs Continue, By Proxy
The Move is Nigh
Another Alzheimer's IPO
Cutbacks at C&E News
Sanofi Pays to Get Back Into Oncology
An Irresponsible Statement About Curing Cancer
Oliver Sacks on Turning Back to Chemistry