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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: derekb.lowe@gmail.com Twitter: Dereklowe

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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

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January 24, 2014

PTC's Latest Ataluren Woes

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


COMMENTS

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.

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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.

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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.

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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. :-)

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