Proprietary and Confidential. © 2014 Concert Pharmaceuticals, Inc. All Rights Reserved.
Product Innovation. Patient Benefit.
March 2017
Forward-Looking Statements
This presentation contains forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. These forward-looking statements involve
substantial risks and uncertainties. All statements, other than statements of historical
facts, contained in this presentation, including statements regarding our strategy, future
operations, future financial position, future revenue, projected costs, prospects, plans
and objectives of management and expected market growth, are forward-looking
statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,”
“intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and
similar expressions are intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words.
We may not actually achieve the plans, intentions or expectations disclosed in our
forward-looking statements, and you should not place undue reliance on our forward-
looking statements. Actual results or events could differ materially from the plans,
intentions and expectations disclosed in the forward-looking statements we make as a
result of various important factors, including the factors discussed in the "Risk Factors"
section of our most recent Annual Report on Form 10-K filed with the Securities and
Exchange Commission and in other filings that we make with the Securities and
Exchange Commission. The forward-looking statements contained in this presentation
reflect our current views with respect to future events, and we assume no obligation to
update any forward-looking statements except as required by applicable law.
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This communication is being made in respect of the proposed asset sale with Vertex.
The proposed asset sale and the asset purchase agreement will be submitted to the
shareholders of the Company for their consideration and approval. In connection with
the proposed asset sale, the Company will file a proxy statement with the SEC. This
communication does not constitute a solicitation of any vote or proxy from any
shareholder of the Company. Investors are urged to read the proxy statement carefully
and in its entirety when it becomes available and any other relevant documents or
materials filed or to be filed with the SEC or incorporated by reference in the proxy
statement, because they will contain important information about the proposed asset
sale. The definitive proxy statement will be mailed to the Company’s shareholders. In
addition, the proxy statement and other documents will be available free of charge at the
SEC’s internet website, www.sec.gov. When available, the proxy statement and other
pertinent documents may also be obtained free of charge at the Investors section the
Company’s website, www.concertpharma.com, or by directing a written request to
Concert Pharmaceuticals, Inc., Attn: Investor Relations, in writing, at 99 Hayden Ave,
#500, Lexington, MA 02421.
The Company and its directors, executive officers and other members of management
and employees may be deemed to be participants in the solicitation of proxies in
connection with the proposed asset sale. Information about the Company’s directors and
executive officers is included in the Company’s Annual Report on Form 10-K for the year
ended December 31, 2016 filed with the SEC on March 6, 2017. Additional information
regarding these persons and their interests in the transaction will be included in the
proxy statement relating to the proposed asset sale when it is filed with the SEC. These
documents can be obtained free of charge from the sources indicated above.
Additional Information about the Transaction and Where to Find It
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Corporate Highlights
CTP-656, proprietary next-generation
treatment for cystic fibrosis
– Recent asset purchase agreement
with Vertex Pharmaceuticals realizes
significant value for CTP-656
Pro forma cash expected to fund operations into 2021
Multiple efficacy studies in 2017
– CTP-543, proprietary JAK 1/2 inhibitor
for moderate-to-severe alopecia areata, entering Phase 2
– CTP-656 U.S. Phase 2 study in gating mutations underway
– AVP-786, Phase 3 trial for Alzheimer’s agitation expected to
complete Q3 2018 (partnered with Avanir/Otsuka)
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Robust Clinical Pipeline
5
Phase I Phase 2
Product
Candidate
Lead
Indication(s)
Worldwide Rights
AVP-786
Deuterated dextromethorphan
CTP-730
Deuterated apremilast
Inflammatory Diseases
JZP-386
Deuterated sodium oxybate
CTP-543
Deuterated ruxolitinib
Phase 3
CTP-656*
Deuterated ivacaftor
Multiple Neurologic/Psychiatric Indications
Alzheimer’s Agitation
Cystic Fibrosis
Alopecia Areata
Narcolepsy
Market
*Subject to closing of the CTP-656 Asset Purchase Agreement with Vertex Pharmaceuticals
CTP-656: Next Generation Treatment for Cystic Fibrosis
Cystic fibrosis is a genetic disease
affecting ~75,000 patients worldwide
Average life expectancy has doubled
in last two decades
CTP-656 (deuterated analog of
ivacaftor*) is a potentiator which
improves CFTR activity
– Potential to improve upon efficacy of
Kalydeco® (ivacaftor)
– Phase 2 U.S. trial underway
Entered asset purchase agreement with
Vertex Pharmaceuticals March 2017
Treatment for Cystic Fibrosis Undergoing Major Advances
6
Source: cff.org
* Ivacaftor is marketed by Vertex Pharmaceuticals, Inc. under the brand name Kalydeco®
CTP-656: Asset Purchase Agreement
Up to $250 million agreement with Vertex Pharmaceuticals
– $160 million upfront payment with $90 million
in milestone potential
– Vertex to acquire all rights to CTP-656
Vertex is a pioneer in CF with a broad pipeline and the capability to
efficiently advance CTP-656
– Potential for combination products benefiting the larger CF patient population
Closing of transaction subject to Concert shareholder approval and HSR
clearance
Upon closing, cash projected into 2021
– Enable advancement of CTP-543 into pivotal testing
– Strengthen proprietary pipeline
– Potential to realize AVP-786 royalties
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CTP-656: U.S. Phase 2 Trial Ongoing
Phase 2 trial in patients with gating
mutations initiated December 2016
– 30-40 patients
– 4 week treatment duration
– 3 doses of CTP-656
– Placebo and Kalydeco comparators
– Key endpoints: sweat chloride and FEV1
Trial continuing as planned
Phase 2 topline data expected Q4 2017*
8*Pending transfer of program to Vertex
CTP-543: Potential First Oral Treatment for Alopecia Areata
Target indication: moderate-to-severe
alopecia areata
– Common autoimmune disorder causing
partial or widespread loss of hair on the
scalp and/or body
CTP-543: deuterated ruxolitinib
– Ruxolitinib: potent, selective JAK1/2
inhibitor approved for myelofibrosis,
polycythemia vera
Clinical proof-of-concept demonstrated
with ruxolitinib in alopecia areata
Phase 2 expected to begin Q1 2017
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Opportunity to address important unmet medical need
source. derm101.com
Alopecia Areata: A Devastating and Poorly Treated Autoimmune Disease
Up to 650,000 patients affected with
alopecia areata in the U.S. at any given
time*
Chronic condition affecting women, men
and children of all ages
Often leads to psychological
consequences, including anxiety and
depression
No FDA-approved treatment options
FDA selected alopecia areata as area of
focus under its 2016-2017 Patient-
Focused Drug Development Initiative
*Fricke M. Clinical, Cosmetic and Investigational Dermatology, 2015. 10
Clinical Evidence for Efficacy of Ruxolitinib in Alopecia Areata
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Open label pilot study of ruxolitinib in alopecia areata patients
– 20 mg BID oral ruxolitinib (approved myelofibrosis dose)
9/12 patients (75%) achieved 50%+ regrowth by end of treatment (3-6 months)
– Responders averaged 92% regrowth by end of treatment
– <12% reported historical spontaneous remission
Treatment was generally well-tolerated with no serious adverse events
JCI Insight. 2016;1(15):e89790. doi:10.1172/jci.insight.89790.
CTP-543: Phase 1 Program Complete
Phase 1 profile supports
advancement into Phase 2
Increased exposure with
increasing doses
Well-tolerated with no serious
adverse events reported
No new metabolites were
observed
Interferon g-mediated STAT1
phosphorylation was
significantly inhibited at all
doses evaluated
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CTP-543: Potential First FDA-Approved Oral Treatment for Alopecia Areata
Phase 2a trial to commence in Q1 2017
– Double-blind, placebo-controlled, dose-ranging
– Approximately 100 adults with moderate-to-
severe alopecia areata
At least 50% hair loss as measured by Severity of
Alopecia Tool (SALT)
– 12 month dosing with primary efficacy analysis at
week 24
Primary Endpoint: 50% relative reduction in SALT
between week 24 and baseline
Topline data expected Q4 2017
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AVP-786: Potential First-in-Class Treatment for Agitation in
Alzheimer’s Disease
Estimated 5.3M Americans have Alzheimer’s
disease; approximately 50% of patients
experience agitation
– No currently approved therapies
Phase 3 trials underway for blockbuster
indication
– Expected completion: Q3 2018
Otsuka (Avanir) responsible for development and
commercialization
– $170M upfront/milestone potential; $8M achieved to date
– $5M milestone on acceptance of NDA
– Mid-single to low-double digit royalties
14
2016 Financial Results
2016
Cash and Investments $ 96.2 M
Revenue $ 174,000
Operating Expenses $ 51.3 M
Shares outstanding 22.3 M
15
Following closing of the CTP-656 agreement with Vertex,
cash is expected to fund the Company into 2021
– Based on $160 million upfront
2017 Multiple Potential Value Creating Events
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CTP-656
– Closing of asset purchase agreement with Vertex
– Report US Phase 2 monotherapy top-line efficacy data (Q4 2017)*
CTP-543
Report Phase 1 PD top-line data (Q1 2017)
Report Phase 1 crossover data (Q1 2017)
– Initiate Phase 2a trial (Q1 2017)
– Report Phase 2a topline results (Q4 2017)
Upcoming Development Milestones for Proprietary Compounds
*Pending transfer of program to Vertex
Proprietary and Confidential. © 2014 Concert Pharmaceuticals, Inc. All Rights Reserved.
NASDAQ: CNCE
www.concertpharma.com
For additional information contact:
Justine Koenigsberg
ir@concertpharma.com