Concert Pharmaceuticals, Inc.

Good day and thank you for standing by. Welcome to the Concert Pharmaceuticals First Quarter 2021 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Justine Konigsberg. Please go ahead.

Good morning and welcome to Concert Pharmaceuticals first quarter 2021 investor update. Our prepared comments today will be brief, so we can jump right into the Q&A portion of the call. Roger Tung, our CEO, will provide the CTP 543 key highlights, and then Mark Becker, our CFO, will walk you through the first quarter financials. We will then be joined by Nancy Stewart, our Chief Operating Officer, and Jim Casella, our Chief Development Officer, for the Q&A portion of the call. As a reminder, today's discussion will include forward-looking statements about our future expectations, plans, and prospects. These statements are subject to risks and uncertainties that may cause actual results to differ materially from those projected. A description of these risks can be found in our most recent 10-Q filed with the SEC. Any forward-looking statements speak only as of today's date, and we assume no obligation to update any forward-looking statements made on today's call. With that, I would now like to turn the call over to Roger.

Thank you, Justine. We're committed to advancing CTP543 through the Phase 3 program and anticipate filing our NDA in early 2023. We're very happy with the efficacy and safety profile CTP543 has shown thus far, and based on the competitive data we've seen to date, continue to believe that it may be best-in-class treatment for alopecia areata currently in development. We're pleased with the overall progress of the CTP543 program, which is advancing as planned. We're on track to initiate ThriveAA2, our second Phase 3 trial this quarter, as projected. The study will enroll approximately 440 patients with moderate to severe alopecia areata and is similar to ThriveAA1 in design. The Phase 3 data are expected next year. As a reminder, our robust clinical results, which have been presented at a number of medical meetings, including, most recently, data from our open-label long-term extension study, show that CTP543 treatment maintains or improves hair regrowth beyond 52 weeks. We plan to present an update on the ongoing long-term extension study during the second JAK Inhibitors Drug Development Summit on July 1st. Additionally, looking at our SALT20 analysis in the Phase 2 study, which is the primary efficacy endpoint in our Phase 3 trials, we saw statistically significant differences from placebo for the CTP543 8 mg and 12 mg twice daily cohorts at 24 weeks. Specifically, 42% of patients treated with 12 mg of CTP543 twice daily achieved an absolute salt score of less than or equal to 20. In the 8-milligram cohort, 26% of patients achieved an absolute salt score of less than or equal to 20. These results are clinically meaningful. Based on the data presented to date from industry-sponsored trials, we believe our findings represent the most robust efficacy results of any compound being developed to treat alopecia areata. A recent epidemiological assessment of alopecia areata in the U.S. indicates that between 700,000 and 1.6 million patients currently have the disease with upwards of 40% of those patients suffering scalp hair loss of 50% or greater. It's becoming increasingly widely recognized that alopecia areata is an important medical condition with no approved treatment and in many patients exerts significant emotional and psychological impact including depression or anxiety, and is associated with other comorbid autoimmune conditions. There's an enormous need for an FDA-approved treatment, and we're proud that Concert was one of the first in the industry to take notice of this important disease by advancing CTP543. We continue to push forward its development with a goal to file our NDA in early 2023. For the immediate future, we intend to invest our resources in advancing CTP543. Patients with alopecia areata have waited a long time for an effective treatment. We're developing something that we believe will be clinically meaningful to patients and expect to continue to be a major force in the field. Let me pause here and turn the call over to Mark.