this new drug was mentioned in the Xeljanz thread but I thought that since it's so difficult to keep track of information on the format of this forum it should have it's own thread

Can we try to keep this thread on topic with any information about CTP-543 that anyone happens to come across?

LEXINGTON, Mass.--(BUSINESS WIRE)--Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced its next product candidate, CTP-543, for the treatment of alopecia areata. Alopecia areata is an autoimmune disease characterized by patchy or widespread hair loss, for which there is significant unmet medical need and currently no FDA-approved treatment. CTP-543 is a deuterium-modified analog of ruxolitinib, which selectively inhibits Janus kinases 1 and 2, known as JAK1 and JAK2. Ruxolitinib is commercially available under the name Jakafi® in the United States for the treatment of myelofibrosis and polycythemia vera. Concert expects to initiate Phase 1 clinical evaluation of CTP-543 in the second quarter of 2016 with efficacy studies commencing in 2017.

Ruxolitinib has been used to treat alopecia areata in academic settings, including an investigator-sponsored clinical trial, and has been shown to promote hair growth in individuals with moderate to severe disease. Preclinical studies conducted by the Company demonstrated that CTP-543 retains ruxolitinib’s selective JAK 1/2 inhibition profile and shows improved metabolic stability.

“We are very excited about the potential of CTP-543 to greatly enhance patient care for individuals with alopecia areata. CTP-543 represents an opportunity to address an important disease where new and effective treatment options are needed,” said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “Based on demonstrated clinical activity of ruxolitinib in alopecia areata, CTP-543 has the potential to become an important first-in-class oral medicine to treat a devastating condition affecting millions of people in the U.S.”

Recently, the U.S. Patent and Trademark Office issued U.S. Patent No. 9,249,149 that claims CTP-543 as a novel composition of matter.

“CTP-543 is a prime example of the application of our deuterium platform to create proprietary new medicines which enhance and extend the utility of known molecules into new therapeutic areas. We believe we have developed a valuable, potentially first-in-class new drug candidate for an important unmet medical need and look forward to advancing CTP-543 into clinical evaluation shortly,” said Dr. Tung.

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some patent issues with CTP-543

unfortunately I can't find a date on this article

the positive is that Concert says they're going to continue developing the drug while the legal team works on the patent issues. Concert has previously won patent disputes on similar situations (lay person's explanation is that they modify existing drugs to make them safer/more effective and then patent them as unique)

Concert Pharma’s Phase IIa alopecia areata study may lack cosmetic relevance

some key parts of article

Based on animal studies and an earlier proof-of-concept trial, there is confidence the Phase IIa for CTP-543, a JAK inhibitor, will meet its co-primary efficacy endpoint of hair loss measured by SALT at 24 weeks, interviewed experts agreed. Safety is the other co-primary measure.

The pilot study showed that nine of 12 patients (75%) demonstrated response to treatment, with average hair regrowth of 92%. Both the pilot study and the Phase IIa tested CTP-543 orally. The pilot study used a dose of 20mg of Jakafi twice-daily and the current protocol is evaluating 4mg and 8mg of CTP-543 twice-daily versus placebo. If appropriate, Concert Pharma will amend the protocol to also evaluate 12mg twice-daily, a spokesperson said.

I'll be honest, I don't really understand what this report is saying. There's someone in the trial on the AA facebook group who has grown a full head of hair

We all know there are people who by all means try to prevent

CTP 543 from hitting the market. 09.04.19 is judgement day in the

patent battle which Incyte has initiated against Concert. The whole 

thing is a shame! There is a million children waiting for medication and

incyte never had any intention to work in AA.


That’s me!! 100% AU to now 100% growth from phase 2a of trial! I just made the switch to xeljanz 3 weeks ago and still so far so good!! If anyone has any questions feel free to reach out! 

Thank you, ashes1120! Did you experience any issues side-effects? 

And what was your SALT score after completion?

Sorry, just noted your score 100 vs 0 after completion, 

Wow that’s amazing to hear !! Can I ask how long was you on CTP-543 in total ? To get full regrowth?

And how long have you been AU for? 

Thank you !

Any chance of a before and after pic ? This is exciting for the likes of people who have not responded to the traditional jak-inhibitors.

Thank you the update!

24 week trial, last 4 weeks is drug free. Only side effects I really had were increased cholesterol and a little weight gain. AU since May 2016


Wow ! Amazing ! Congratulations on your results very exciting ! Thank you for posting! 

Am guessing your eyebrows grew back ? Can I ask did anyone not respond in the trial ?

absolutely unbelievable... only 24 weeks.. 

So are you on xeljanz to maintain what you grew back? Is it necessary or just being cautious?

Ive been on Xeljanz for almost 2 years now with slow regrowth



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