Concert Pharmaceuticals Initiates Phase 1 Multiple Ascending Dose Trial with CTP-656 for Cystic Fibrosis

“In the pipeline” are words that make the heartstrings of anyone living with a chronic, hard-to-treat disease go twanggggg. It’s Big Pharma lingo for “drugs under development,” and generally signals happy things.

So often with rare diseases—and autoimmune diseases, particularly—a drug treatment will work at first. But gradually it stops being effective. It’s like living with the sword of Damocles hanging by a hair over your head all the time.

When will my relief end? When will I have to get back on that not-fun rollercoaster called “finding a treatment that works for me”?

Knowing that there are treatments being developed, tested, and almost ready for FDA approval is comforting. So when I read that Concert Pharmaceuticals has a new drug for cystic fibrosis (CF), CTP-656, in the Phase 1 testing portion of its pipeline, I smiled.

happy disney smile cartoon puppy
Yes, this is me. Dug is my spirit animal. Source:

The drug finished a “multiple ascending dose trial.”

That’s the drug testing/approval phase in which researchers are discovering:

  • how the drug moves through (pharmacokinetics),
  • is processed by,
  • and affects the body (pharmacodynamics).

Trial participants are given multiple low doses of the treatment being tested. Then their blood and fluid samples are analyzed. The process is repeated until the dosage meets a pre-determined level. If the results are as (or better than) expected, the drug goes on to the next stage. This is called Phase II testing.

CTP-656’s results were impressive enough to warrant Concert Pharma’s continued investment. It’s now headed on to Phase 2.

Currently, the standard for treating CF is with a drug, commercially known as Kalydeco® (ivacaftor). It helps improve the function of the CFTR “gates” that let chloride ions move into and out of cells.

Concert Pharmaceuticals is trying to come up with a treatment that is better than ivacaftor. According to information the company presented at a European Cystic Fibrosis Conference in June, 2016, things are looking good!

It’s nice to see that the future of CF care is still following an upward trajectory!



EmpatheticBadass is a young-at-heart writer from Ohio (Go, Bobcats & The Marching 110!)) who is passionate about being a voice for the patient perspective.

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