(State or Other Jurisdiction
65 Hayden Avenue, Suite 3000N
(Address of Principal Executive Offices)
Title of each class
Name of each exchange on which registered
Common Stock, par value $0.001 per share
The NASDAQ Global Market
CONCERT PHARMACEUTICALS, INC.
/s/ Roger D. Tung
Date: August 1, 2019
Roger D. Tung
President and Chief Executive Officer
CTP-543 Third Dose, 12 mg Twice-Daily, Phase 2 Topline Data Expected Third Quarter of 2019. The Company expects to report topline results from the final cohort in its ongoing CTP-543 Phase 2 trial in the third quarter of 2019. The Phase 2 trial was designed to evaluate the safety and efficacy of three doses (4, 8, and 12 mg twice-daily) of CTP-543 compared to placebo in patients with moderate-to-severe alopecia areata. Interim results from the initial two cohorts for this dose-ranging trial, first reported in November 2018, showed that treatment with CTP-543 administered at an 8 mg twice-daily dose for 24 weeks met the primary endpoint with a statistically significant greater hair regrowth rate compared to placebo.
CTP-543 Open Label Trial Evaluating 8 mg Twice-Daily vs. 16 mg Once-Daily Fully Enrolled. In May 2019, the Company announced that an open-label dose regimen trial evaluating CTP-543 for the treatment of alopecia areata at a dose of 8 mg twice-daily compared to a dose of 16 mg once-daily was fully enrolled. Topline results from the dose regimen trial are expected by year-end 2019. Data from this study, along with results from the Phase 2 dose-ranging trial, are intended to support an end of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) in the first quarter of 2020 to review and discuss the Company’s registration pathway for CTP-543 for the treatment of alopecia areata.
CTP-543 Open Label Trial Evaluating 12 mg Twice-Daily vs. 24 mg Once-Daily Initiated. In May 2019, the Company initiated a second open label clinical trial to evaluate twice-daily compared to once-daily dosing of CTP‑543 in patients with alopecia areata. Patients in this trial are randomized to receive either 12 mg twice-daily or 24 mg once-daily of CTP-543 over a 24 week treatment period. Topline data is expected in the first half of 2020.
CTP-543 Open Label Extension Study Initiated April 2019. In April 2019, the Company initiated an open label extension study to continue to evaluate long-term safety and efficacy of CTP-543 in adult patients who complete 24 weeks of treatment in a previous qualifying CTP-543 clinical trial. Patients participating in the 12 mg cohort in the Phase 2 trial and the two open label dose regimen studies evaluating once-daily compared to twice-daily dosing, as well as future trials with CTP-543, are eligible to enroll in this study.
The Company Filed Notice of Appeal in PTAB Proceeding. On June 7, 2019, the Company filed a Notice of Appeal with the Court of Appeals for the Federal Circuit in connection with the inter partes review (IPR) decision of the Company’s U.S. Patent No. 9,249,149.
CTP-692 Phase 1 Positive Topline Results Reported. The safety assessments in the single- and multiple-ascending dose Phase 1 trials in healthy volunteers showed that CTP-692 was well tolerated over the dose ranges tested, which included the doses expected to be evaluated in Phase 2 testing. Importantly, key blood and urine markers of kidney function did not indicate any signs of renal impairment. These data are consistent with preclinical findings showing an improved renal safety profile for CTP-692 compared to non-deuterated D-serine which is known to produce renal toxicity in rats. Despite published clinical studies reporting the benefit of non-deuterated D-serine in the treatment of schizophrenia, its further development has been limited by renal safety concerns. The Company intends to advance CTP-692 into Phase 2 testing in the fourth quarter of 2019.
CTP-692 Phase 1 Results to be Presented at ECNP Annual Congress. The Company will present its CTP-692 Phase 1 single- and multiple-ascending dose clinical results during a poster session at the European College of Neuropsychopharmacology Congress taking place September 7-10, 2019 in Copenhagen, Denmark.
CTP-692 Poster Presentation at ASCP. The initial Phase 1 CTP-692 trial evaluated the safety, tolerability, and pharmacokinetics of a single oral dose of CTP-692 versus D-serine in a crossover study conducted in Australia. In the study, CTP-692 was shown to have increased plasma exposure compared to D-serine. In addition, CTP-692 was shown to be well tolerated in healthy volunteers. These results were presented at a poster session at The American Society of Clinical Psychopharmacology (ASCP) annual meeting, held May 28-31, 2019 in Scottsdale, AZ.
Enrollment in the Second AVP-786 Phase 3 Alzheimer’s Agitation Trial is Complete. In May 2019, Avanir Pharmaceuticals, a subsidiary of Otsuka Pharmaceuticals, announced that it completed enrollment in a second U.S. Phase 3 trial evaluating AVP-786 for the treatment of agitation in patients with dementia of the Alzheimer's type. The study is expected to complete in October 2019 and topline results are expected to be reported before year end. Under a licensing agreement whereby Concert granted Avanir worldwide development and commercialization rights to AVP-786, Concert is eligible to receive additional milestone payments as well as tiered royalties on worldwide sales.
Cash and Investment Position. Cash, cash equivalents and investments as of June 30, 2019, totaled $136.6 million. The Company expects its cash, cash equivalents and investments to be sufficient to fund the Company into the second half of 2020.
Revenues. For the six months ended June 30, 2019, revenue was $1.1 million, compared to $10.5 million for the six months ended June 30, 2018. Revenue recognized in the 2019 and 2018 periods was due to upfront consideration for license arrangements with Cipla Technologies and Processa Pharmaceuticals, respectively.
R&D Expenses. Research and development expenses were $14.5 million for the quarter ended June 30, 2019, compared to $8.9 million for the same period in 2018. For the six months ended June 30, 2019, research and development expenses were $30.3 million, compared to $17.5 million for the same period in 2018. The increase in research and development
G&A Expenses. General and administrative expenses were $5.0 million for the quarter ended June 30, 2019, compared to $5.5 million for the same period in 2018. For the six months ended June 30, 2019, G&A expenses were $10.6 million compared to $11.1 million for the same period in 2018. Decreases in legal and employee-related expenses were partially offset by an increase in professional fees.
Net Loss. For the quarter ended June 30, 2019, net loss applicable to common stockholders was $18.7 million, or $0.78 per share, compared with a net loss applicable to common stockholders of $13.3 million, or $0.57 per share, for the quarter ended June 30, 2018.
Concert Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
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Three Months Ended June 30,
Six Months Ended June 30,
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Unrealized (loss) gain on marketable equity securities
Loss before tax provision
Provision for income taxes
Net loss per share applicable to common stockholders — basic and diluted
Weighted-average number of common shares used in net loss per share applicable to common stockholders— basic and diluted
Concert Pharmaceuticals, Inc.
Summary Balance Sheet Data
June 30, 2019
December 31, 2018
Cash and cash equivalents
Investments, available for sale
Total stockholders’ equity