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

« Are There Good Writers In This Business? | Main | The Nasty Side of HDL »

January 24, 2014

PTC's Latest Ataluren Woes

Email This Entry

Posted by Derek

I wrote here about PTC Therapeutics and their drug candidate for Duchenne muscular dystrophy (ataluren, PTC124). Opinion has been divided, to put it mildly, about how it works and whether it works at all.

Well, the saga continues. The company is having a rough time with that program these days, though. PTC applied to the European Medicines Agency for conditional approval of ataluren, but that request has just been firmly rejected.

Ataluren failed both a Phase IIb study for DMD as well as a Phase III study for cystic fibrosis, yet the biotech went on to wrap one of 2013's hottest IPOs in the resurgent biotech field, grabbing $125 million from investors. And over the last month its stock price jumped 37%.

Peltz has argued for years now that even though ataluren hasn't produced statistically significant results in later stage studies, the improvements in walking distance warranted an approval. But the EMA has now formally said no, leaving the drug's fate to be decided by a late-stage study the biotech describes as "confirmatory."

Hey, they might be right in that description - the Phase III might confirm the Phase II results and show that the drug truly does not work. And it looks like the regulatory agencies are thinking the same way. . .

Comments (7) + TrackBacks (0) | Category: Clinical Trials | Regulatory Affairs


1. beentheredonethat on January 24, 2014 1:14 PM writes...

What else can they do? Get rid of the chemists as they have done it in the past. Biology is not in doubt, but the efficacy is(if there was any!). Poorly managed from the top down.

Permalink to Comment

2. Anonymous on January 24, 2014 2:03 PM writes...

This is what happens when you push a drug too far too fast and end up with an unbalanced portfolio that depends on a single drug: you keep pushing it forwards despite the negative results, denying failure, and wasting more money. It's like trying to gamble your way out of debt. Completely stupid.

Permalink to Comment

3. johnnyboy on January 24, 2014 3:19 PM writes...

These days, a biotech could be pushing bellybutton lint as a cure for death and still have an ever-climbing stock price. PTC is following the money, like so many others.

Permalink to Comment

4. SteveM on January 25, 2014 8:05 AM writes...

Re: "Peltz has argued for years now that even though ataluren hasn't produced statistically significant results in later stage studies, the improvements in walking distance warranted an approval."

Peltz's walking distance argument is aligned with the now normative Pharma tactic of getting approval for a drug that provides only limited relief and charging $7,000 a month for it. Under that rubric, staying with a long shot makes sense.

Permalink to Comment

5. JigJag on January 25, 2014 1:19 PM writes...

Realizing the fact that the CEO and his cartel were trying to push this non-working compound into market the former chief medical officer Langdon Miller left the company.

Permalink to Comment

6. Anonymous on January 27, 2014 8:49 AM writes...

"These days, a biotech could be pushing bellybutton lint as a cure for death ..."

I'll have to try that one. :-)

Permalink to Comment

7. Anon on May 28, 2014 1:06 PM writes...

As a former PTC employee, the recent recommendation for conditional approval is a joke. None of the data are strong. The improvement for the 40 mg/kg/day dose over placebo was significant (after eliminating 2 patients whose baseline data was faulty) in the DMD trial, however the results from the higher 80 mg/kg/day dose almost perfectly matched the placebo. Moreover, the CF Phase 3 trial was not only negative on the primary endpoint, but also on pretty much all the secondaries. Most importantly, the endpoint that was the primary in the Phase 2 studies in CF, used to show proof of concept, was highly nonsignificant in the Phase 3 study. Don't be surprised to see this one land flat on its face

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