Concert Pharmaceuticals to Provide Overview of Clinical Pipeline at Cantor Healthcare Conference

“While we’re disappointed by the recent results generated by our collaborators in the AVP-786 Phase 3 Alzheimer’s agitation trial, we are highly focused on the distinct value of Concert’s proprietary drug candidates, CTP-543 for alopecia areata and CTP-692 for schizophrenia,” stated Roger Tung, Ph.D., President and CEO of Concert Pharmaceuticals. “We have had a consistent pipeline strategy to develop innovative medicines based on well-established biology, which is the foundation for CTP-543, a JAK inhibitor for alopecia areata, and for CTP-692, a deuterated D-serine which is an NMDA co-agonist. Concert’s financial resources are dedicated to these proprietary candidates, and our clinical programs are focused on demonstrated efficacy and safety for unmet needs in these chronic diseases affecting millions of patients.”

Dr. Tung continued, “For our partnered programs, including AVP-786, the clinical rationale and all development costs are the responsibilities of our collaborators.” On September 27, 2019, Concert’s collaborator, Avanir Pharmaceuticals, Inc., announced that the second study of its Phase 3 clinical program for AVP-786 for the treatment of moderate-to-severe agitation in patients with Alzheimer’s disease did not meet its primary and key secondary endpoints.

At the Cantor Healthcare Conference, Dr. Tung will highlight recent developments and upcoming milestones for Concert’s two wholly-owned clinical candidates during the presentation.

CTP-543: an investigational treatment for moderate-to-severe alopecia areata

• In September 2019, Concert reported positive topline results from a recently completed Phase 2 dose-ranging trial evaluating CTP-543 for the treatment of moderate-to-severe alopecia areata. CTP-543 achieved its primary endpoint in the 8 mg and 12 mg twice-daily dosing cohorts. Treatment with 8 and 12 mg twice-daily of CTP-543 for 24 weeks was generally well tolerated and resulted in significant hair regrowth and significant ratings of improvement in individuals with alopecia areata.
• Concert plans to present the Phase 2 dose-ranging results in the late-breaking news session at the European Academy of Dermatology and Venereology (EADV) Annual Congress on October 12, 2019 in Madrid, Spain.
• Concert also initiated two open-label studies in 2019 evaluating once-daily vs. twice-daily dosing. Both studies are fully enrolled. Topline data from the first study is expected by year-end 2019 and the results from the second study are expected in the first half of 2020.
• The CTP-543 development program is based on well-established biology that Janus kinase (JAK) signaling underlies the pathophysiology in alopecia areata.
• The Company intends to advance CTP-543 into Phase 3 in 2020.

CTP-692: an investigational adjunctive treatment for schizophrenia

• Concert reported positive results from its Phase 1 program evaluating CTP-692, a novel deuterium-modified form of D-serine being developed as an adjunctive treatment for schizophrenia. The safety assessments in the single- and multiple-ascending dose trials in healthy volunteers showed that the drug was well tolerated over the dose ranges tested, which include 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.
• The Phase 1 results were presented September 10, 2019 at the European College of Neuropsychopharmacology Congress in Copenhagen, Denmark. A copy of the poster presentation is available in the Scientific Presentations section of Concert’s website.
• The CTP-692 development program is based on well-established biology that D-serine is an endogenous neurotransmitter that acts as a required co-agonist at the NMDA receptor. There is evidence that plasma and CSF levels of endogenous D-serine are reduced in patients with schizophrenia. CTP-692 has the potential to help restore NMDA receptor activity in key areas of the brain and improve clinical outcomes in patients with schizophrenia.
• The Company expects to advance CTP-692 into Phase 2 testing in the fourth quarter of 2019.

Financial 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 through 2020.
• Under an asset purchase agreement with Vertex Pharmaceuticals, whereby Vertex acquired worldwide rights to VX-561 (formerly CTP-656) from Concert, the Company could receive up to an additional $90 million in milestones based on regulatory approval in the U.S. and reimbursement in the UK, Germany or France. In the second quarter of 2019, Vertex initiated a Phase 2 dose-ranging study evaluating the once-daily potentiator VX-561 as a monotherapy as requested by the FDA. The study is designed to evaluate multiple doses of VX-561 to support potential Phase 3 development of VX-561 in a once-daily triple combination regimen.

Cantor Healthcare Conference Webcast
A live webcast of the presentation at the Cantor Healthcare Conference may be accessed in the Investors section of the Company’s website at www.concertpharma.com. Please log on to the Concert website approximately 15 minutes prior to the scheduled webcast to ensure adequate time for any software downloads that may be required. A replay of the conference webcasts will be available on Concert’s website for two weeks following the presentation.

About Concert
Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform® (deuterated chemical entity platform) to create novel medicines designed to treat serious diseases and address unmet patient needs. The Company’s approach starts with previously studied compounds, including approved drugs, in which deuterium substitution has the potential to enhance clinical safety, tolerability or efficacy. Concert’s pipeline of innovative medicines targets autoimmune diseases and central nervous systems (CNS) disorders. For more information please visit www.concertpharma.com or follow us on Twitter at @ConcertPharma or on LinkedIn.

Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including, among others, statements about our expectations on the progress of clinical development of CTP-543 and CTP-692, the timing of availability of clinical trial data and the sufficiency of our cash, cash equivalents and investments to fund our operations, and any other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the “Risk Factors” section of our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.

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