PROSPECTUS SUPPLEMENT SUMMARY
This summary highlights selected information contained elsewhere in this prospectus supplement and the accompanying prospectus and in the documents we incorporate by reference. This summary does not contain all of the information you should consider before investing in our common stock. You should read this entire prospectus supplement and the accompanying prospectus carefully, especially the risks of investing in our common stock discussed under “Risk Factors” beginning on page S-5 of this prospectus supplement and in Part II, Item 1A “Risk Factors” of our quarterly report on Form 10-Q filed with the SEC on May 5, 2022, which is incorporated by reference in this prospectus supplement, along with our consolidated financial statements and notes to those consolidated financial statements and the other information incorporated by reference in this prospectus supplement and the accompanying prospectus, before making an investment decision.
Corporate Overview
We are a late-stage clinical biopharmaceutical company that is developing CTP-543, a Janus Kinase 1 and Janus Kinase 2 (JAK 1/2) inhibitor that we discovered through the application of our DCE Platform® (deuterated chemical entity platform). We are evaluating CTP-543 in a Phase 3 clinical program for the treatment of adults with moderate to severe alopecia areata, a serious autoimmune dermatological condition. Based on the results of our Phase 3 clinical program of CTP-543, described further below, we intend to file a New Drug Application, or NDA, with the U.S. Food and Drug Administration, or FDA, in the first half of 2023. There are currently no FDA-approved treatments for alopecia areata.
We are conducting pre-commercial activities, with the intent of commercializing CTP-543 in the United States ourselves or with strategic partners. If we are successful in obtaining FDA approval for CTP-543 for the treatment of adults with moderate to severe alopecia areata, we intend to evaluate CTP-543 in younger patients with alopecia areata and explore expanding CTP-543 into additional indications. We are also assessing both internal and potential in-licensed candidates for pipeline expansion.
Recent Developments
Warrant Amendment Agreements
Concurrent with the offering to which this prospectus relates, we have entered into warrant amendment agreements with holders of tranche 1 warrants to purchase shares of our Series X1 Preferred Stock at an exercise price on a common stock equivalent basis of $5.34 per share that were issued in November 2021, which we collectively refer to as the Tranche 1 Warrants. Pursuant to such amendments, in consideration for such holders’ collective exercise of Tranche 1 Warrants to purchase 3,981 shares of Series X1 Preferred Stock for approximately $18.9 million concurrently with and contingent upon the offering, which we refer to as the Exercise Commitments, we have agreed to (i) reduce the exercise price of the Tranche 1 Warrants that are subject to the Exercise Commitments from $5.34 per share on a common stock equivalent basis to $4.75 per share on a common stock equivalent basis and (ii) extend the expiration date of the Tranche 1 Warrants that remain outstanding after exercise of the Exercise Commitments to the later of (x) August 21, 2022 or (y) the twenty-first (21st) day after the occurrence of our public disclosure of the topline results of our CTP-543 THRIVE-AA2 Phase 3 clinical trial, effective upon the consummation of the offering to which this prospectus relates and confirmation that the holders have satisfied the Exercise Commitments. Following satisfaction of the Exercise Commitments, there will be Tranche 1 Warrants to purchase 4,144 shares of Series X1 Preferred Stock and warrants to purchase an aggregate of an additional 8,125 shares of Series X1 Preferred Stock at an initial exercise price (on a common stock equivalent basis) of $7.35 per share, which we collectively refer to as the Tranche 2 Warrants, outstanding. In addition, following the offering and pursuant to the terms of the Tranche 1 Warrants and Tranche 2 Warrants, respectively, the exercise price of the Tranche 1 Warrants will be adjusted to $5.05, the midpoint of the initial exercise price of $5.34 and the public offering price per share of $4.75 (rounded up to the nearest cent), and the exercise price of the Tranche 2 Warrants will be adjusted to $6.05, the midpoint of the initial exercise price of $7.35 and the public offering price per share of $4.75.
CTP-543 Phase 3 Topline Results
On May 23, 2022, we announced positive topline results from our recently completed Phase 3 clinical trial, THRIVE-AA1, evaluating our oral investigational medicine CTP-543 in adult patients with moderate to severe alopecia areata, an autoimmune disorder that results in patchy or complete scalp hair loss. The primary efficacy endpoint for THRIVE-AA1 was the percentage of patients achieving an absolute Severity of Alopecia Tool, or SALT, score of 20 or less at Week 24 of treatment, which was met with statistical significance in both the 8 mg twice-daily and 12 mg twice-daily dose groups relative to placebo. Treatment with CTP-543 was generally well tolerated.
The key secondary endpoints were the percentage of responders on a Hair Satisfaction Patient Reported Outcome scale at Week 24 and the percentage of patients achieving absolute SALT scores of 20 or less at each of Weeks 20, 16, 12 and 8. All key secondary endpoints were met with statistical significance in both dose groups.
Patients enrolled in THRIVE-AA1 were required to have at least 50 percent scalp hair loss due to alopecia areata, as measured by SALT. A SALT score of 100 represents total scalp hair loss whereas a score of 0 represents no scalp hair loss. The average baseline SALT score across all patients was approximately 85.9 (corresponding to less than 15% average scalp hair coverage).
A statistically significant proportion of patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 experienced greater scalp regrowth compared to placebo. The proportion of patients achieving a SALT score of 20 or less (meaning that 80 percent or more scalp hair coverage was achieved) was 41.5 percent in the 12 mg twice-daily dose group and 29.6 percent in the 8 mg twice-daily dose group, compared to 0.8 percent of patients in the placebo group, at the 24-week endpoint. The treatment difference for both dose groups of CTP-543 relative to placebo was statistically significant (p<0.0001).