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New JAK inhibitor - CTP-543 by Concert Pharmaceuticals
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?
http://www.businesswire.com/news/home/20160504006458/en/Concert-Pha...
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.
Replies to This Discussion
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Permalink Reply by sadele on
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What is encouraging is there is a time-line for his to be taken care of... The FDA has 30 days once Concert submits the required paperwork so we should know in the next 2 months.
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Permalink Reply by bibby on
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Let's hope the time line is respected in reality. Looking at this product it appears to limit JAK 1 and JAK 2 where as xeljanz limits JAK 1 and JAK 3. Fundamentally they are very similar and xeljanz was cleared by the FDA so hopefully cpt543 clearance will proceed also. Question is when will we be able to purchase the drug ourselves. !
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From my understanding of what Dr King told me last time I saw him (about 2 weeks ago) those who are incomplete or non-responders on xeljanz have a good chance of responding on CPT-543. It's basically the same as Jakafi. He told me they tweaked one little ingredient when making this medication, however, the important part is that it is a Jak 1&2 inhibitor. I responded to xeljanz, although I was considered an "incomplete" responder.
He wants me to try the CPT-543. I don't know when it will actually be available to the public as they are just starting phase 2 clinical trial. This is a year long trial. Were you on xeljanz? How did you do?
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I have not started xeljanz yet. Maybe during the summer. I'm awaiting 11mg XR xeljanz to become available in turkey to exploit the cost benefit to the higher doseage . It's worth the experiment to see if I respond to xeljanz and if fails I'll fall back on cpt-543 when it comes through the trials.
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why do we need to wait for cpt543 when we can get ruxolitinib off label? like xeljanz now it is prescribed off label.
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So presumably Cpt-543 is going to be a similar price as xeljanz when it's made available after the trials! I better hope my sales in my business increase to allow me to afford it haha!!
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I think the idea behind ctp543 is that it is being developed specifically for alopecia and will be covered by insurance for that application, where xeljanz and ruxolitinib are currently being used off-label for alopecia so they are not eligible for insurance coverage until alopecia becomes an approved usage
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good catch, I've been scanning concert's press releases lately also. It's frustrating to have to wait, but safety first. seeing some of the discussion in the xeljanz thread (weight gain, fatigue) not to mention the looming risk of cancer or secondary infection really brings to life some of the risks of drug development
body of the post:
Concert Pharmaceuticals, Inc. (CNCE) today announced that the U.S. Food and Drug Administration (FDA) has notified the Company that it has lifted the clinical hold on Concert’s CTP-543 Phase 2a clinical trial for alopecia areata. The Company will amend the protocol for the Phase 2a trial to evaluate two doses of CTP-543, each for a 24 week duration. Concert will resume enrollment in the Phase 2a trial later this month and expects to complete the trial in the second half of 2018.
“We believe the FDA is taking a measured approach for the development of Janus kinases inhibitors, or JAK inhibitors, for autoimmune dermatological disorders, including alopecia areata. Accordingly, we agreed to modify the design of our Phase 2a trial to evaluate CTP-543 in the patient population in a more step-wise fashion. It is important to note that CTP-543 has been well-tolerated in clinical evaluation to date and demonstrates a non-clinical safety profile consistent with JAK inhibition,” said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “Alopecia areata is an autoimmune disease for which there is a significant unmet medical need with no FDA-approved therapy, and we intend to be at the forefront of advancing a new oral treatment for alopecia areata patients.”
The Phase 2a trial is a double-blind, randomized, placebo-controlled trial to evaluate the safety and efficacy of CTP-543 in adults with moderate-to-severe alopecia areata. Approximately 90 patients will be enrolled in the study and will be sequentially randomized to receive one of two doses of CTP-543 (4 and 8 mg twice daily) or placebo. The primary outcome measure will utilize the severity of alopecia tool (SALT) after 24 weeks of dosing. If appropriate, the protocol may be amended to explore higher doses of CTP-543. Additional information about the trial is available on www.clinicaltrials.gov.
In May 2017, the FDA informed the Company that a review of certain non-clinical toxicology studies was required before proceeding with dosing in the Phase 2a trial. Concert submitted the requested non-clinical study reports and in further discussions with FDA agreed to amend the clinical protocol leading to today’s announcement of the FDA’s lifting of the clinical hold. In non-clinical evaluation, the profile of CTP-543 was as expected given the compound’s mechanism of action.
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