Skip to Content

PTC’s Latest Ataluren Woes

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. . .

7 comments on “PTC’s Latest Ataluren Woes”

  1. beentheredonethat says:

    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.

  2. Anonymous says:

    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.

  3. johnnyboy says:

    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.

  4. SteveM says:

    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.

  5. JigJag says:

    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.

  6. Anonymous says:

    “These days, a biotech could be pushing bellybutton lint as a cure for death …”
    I’ll have to try that one. 🙂

  7. Anon says:

    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

Comments are closed.