March 12, 2015
Forward Looking Statements This presenta5on contains forward- looking statements within the meaning of Sec5on 27A of the Securi5es Exchange Act of 1933 and Sec5on 21E of the Securi5es Exchange Act of 1934 that involve substan5al risks and uncertain5es. In some cases, you can iden5fy forward- looking statements by the following words: may, will, could, would, should, expect, intend, plan, an5cipate, contemplate, believe, es5mate, predict, project, seek, poten5al, con5nue, ongoing or the nega5ve of these terms or other comparable terminology, although not all forward- looking statements contain these words. These statements relate to future events or our future financial performance or condi5on and involve known and unknown risks, uncertain5es and other factors that could cause our actual results, levels of ac5vity, performance or achievement to differ materially from those expressed or implied by these forward- looking statements. These risks, uncertain5es and other factors are described more fully in the prospectus supplement and accompanying prospectus to this offering and our Annual Report on Form 10- K, all of which are or will be filed with the SEC, par5cularly in the sec5ons 5tled Risk Factors and Management s Discussion and Analysis of Financial Condi5on and Results of Opera5ons, as applicable. In light of the significant uncertain5es in our forward- looking statements, you should not place undue reliance on these statements or regard these statements as a representa5on or warranty by us or any other person that we will achieve our objec5ves and plans in any specified 5meframe, or at all. The forward- looking statements contained in this presenta5on represent our es5mates and assump5ons only as of the date of this presenta5on and, except as required by law, we undertake no obliga5on to update or revise publicly any forward- looking statements, whether as a result of new informa5on, future events or otherwise ayer the date of this presenta5on. This presenta5on also contains es5mates, projec5ons and other informa5on concerning our industry, our business, and the market for our drug candidate, as well as data regarding market research, es5mates and forecasts prepared by our management. Informa5on that is based on es5mates, forecasts, projec5ons, market research or similar methodologies is inherently subject to uncertain5es and actual events or circumstances may differ materially from events and circumstances reflected in this informa5on. 2
KYTHERA Investment Highlights Poten7al Large Aesthe7c Market Opportunity Large and growing aesthe5c market opportunity es5mated to be $1.7 billion by 2017* Poten5al of ATX- 101 Revenue to Exceed $500mm in the U.S. Alone ATX- 101 fits well into exis5ng prac5ces and fills void in injectable market Increased Visibility on ATX- 101 Regulatory Approval in the U.S. and Ex- U.S. FDA Advisory Commihee recommended to approve ATX- 101 to improve the appearance of moderate to severe submental fullness U.S. FDA PDUFA date May 13, 2015 Regulatory filings in Canada, Switzerland & Australia; addi5onal ex- U.S. 2015 filings expected Executed by Aesthe7c and Biotech Veterans with Demonstrated Success Team played leadership roles in commercializing marquee aesthe7c products, including BOTOX Cosme5c, JUVEDERM, La5sse Posi5oned to drive innova5on engine for future products *Millennium Research Group; BOTOX Cosme5c, JUVEDERM, La5sse are registered trademarks of Allergan, Inc. 3
Outcome of FDA Advisory Commihee FDA Advisory Commihee convened on March 9, 2015 Panel voted (17 0) to support the approval of ATX- 101 (deoxycholic acid) injec5on for improvement in the appearance of moderate to severe convexity or fullness associated with submental fat in adults The Commihee s recommenda5on, although not binding, is an important factor considered by the FDA as it con5nues its review of ATX- 101 FDA PDUFA date May 13, 2015 4
KYTHERA Vision to Build a Leading Aesthe5cs Company Long- term Strategy Founding Vision Apply the scien7fic rigor of biotech with focus and execu7on of specialty pharma Develop and commercialize innova5ve drugs that enable a posi7ve self- image across mul5ple stages of life Focus on high- value aesthe5c products with high pa5ent sa5sfac5on and large market opportunity Near- term Execu7on Commercialize ATX- 101 in US and Canada as an injectable treatment for the improvement of submental fullness Maximize the strategic value of ATX- 101 globally Assess and priori5ze possible future indica7ons for ATX- 101 Ini7ate proof- of concept study for se5piprant in hair loss Leverage biotechnology and aesthe5cs experience Aggressively expand product porxolio/pipeline o o Licensing/partnerships R&D Be capital efficient 5
ATX- 101 Changing the Face of Facial Aesthe5cs 35- year old subject with a 1- grade CR- SMFRS and PR- SMFRS Composite Change in Submental Fat; REFINE- 2 (ATX- 101-11- 23) 6
ATX- 101 Poten5al First and Only FDA- Approved Drug for Submental Contouring Clinically Proven 18 SMF clinical trials with >2,600 pa5ents Significant, visible results Consistent efficacy and safety profile Correc7ve Destroys fat locally Leaves surrounding 5ssue largely unaffected 87% of treatment responders maintained response at 4 years (n=33) Customized Non- surgical treatment tailored to achieve a pa5ent s desired aesthe5c outcome Most pa5ents experience improvement in 2-4 treatments 7
REFINE- 1 and REFINE- 2 Pivotal Trials - Sta5s5cally Significant Results for All Primary and Secondary Endpoints Composite of clinician and pa5ent submental fat ra5ng scales 1- grade responders (68.2% ATX- 101 vs. 20.5%, p<0.001) 2- grade responders (16.0% ATX- 101 vs. 1.5% placebo, p<0.001) Visual and Emo5onal Impact of SMF Mean improvement from baseline: - 3.5 (48%) ATX- 101 vs. - 1.3 (18%) placebo, p<0.001) MRI Submental Volume Reduc5on Met All Primary and Secondary Endpoints* Achieved pre- defined reduc5on (43.3% of subjects vs. 5.3% placebo, p<0.001) Safety Profile No treatment- related serious adverse events 80.9% of AEs in ATX- 101 treatment group were mild and 17.5% were moderate; 88.9% of AEs in placebo group were mild and 10.4% were moderate 1.6% of subjects discon5nued the study due to an adverse event Analysis of : pooled data from REFINE- 1 and REFINE- 2 8
ATX- 101 Before and AYer Pa5ent Example Age 43 Baseline BMI 26.1 kg/m 2 Weight 150.6 lb CR- SMFRS Moderate (2) PR- SMFRS Moderate (2) Sa7sfac7on a Slightly Dissa5sfied (2) Skin Laxity b Mild (2) Visit 9 BMI 26.4 kg/m 2 Weight 152.8 lb CR- SMFRS None (0) PR- SMFRS None (0) Sa7sfac7on a Extremely Sa5sfied (6) Skin Laxity b None (1) a Sa5sfac5on based on SSRS b Laxity based on SMSLG (3 Treatment Sessions) 9
ATX- 101 Before and AYer Pa5ent Example Age 39 Baseline BMI 31.9 kg/m 2 Weight 209.8 lb CR- SMFRS Severe (3) PR- SMFRS Large (3) Sa7sfac7on a Extremely Dissa5sfied (0) Skin Laxity b None (1) 32 Weeks Acer Last Treatment BMI 32.6 kg/m 2 Weight 214.6 lb CR- SMFRS Moderate (2) PR- SMFRS Slight (1) Sa7sfac7on a Sa5sfied (5) Skin Laxity b None (1) a Sa5sfac5on based on SSRS b Laxity based on SMSLG (4 Treatment Sessions) 10
ATX- 101 Before and AYer Pa5ent Example Age 41 Baseline BMI 23 kg/m 2 Weight 130.1 lb CR- SMFRS Moderate (2) PR- SMFRS Large (3) Sa7sfac7on a Slightly Dissa5sfied (2) Skin Laxity b Not Applicable 12 Weeks Acer Last Treatment BMI 23.3 kg/m 2 Weight 131.6 lb CR- SMFRS None (0) PR- SMFRS None (0) Sa7sfac7on a Extremely Sa5sfied (6) Skin Laxity b Not Applicable a Sa5sfac5on based on SSRS b Laxity based on SMSLG (4 Treatment Sessions) 11
ATX- 101 Before and AYer Pa5ent Example Age 23 Baseline BMI 22.6 kg/m 2 Weight 133.6 lb CR- SMFRS Severe (3) PR- SMFRS Large (3) Sa7sfac7on a Extremely Dissa5sfied (0) Skin Laxity b Not Applicable 12 Weeks Acer Last Treatment BMI 22 kg/m 2 Weight 126.1 lb CR- SMFRS Mild (1) PR- SMFRS Slight (1) Sa7sfac7on a Sa5sfied (5) Skin Laxity b Not Applicable a Sa5sfac5on based on SSRS b Laxity based on SMSLG (3 Treatment Sessions) 12
ATX- 101 Before and AYer Pa5ent Example Age 34 Baseline BMI 24.4 kg/m 2 Weight 170 lb CR- SMFRS Moderate (2) PR- SMFRS Moderate (2) Sa7sfac7on a Dissa5sfied (1) Skin Laxity b None (1) 12 Weeks Acer Last Treatment BMI 25.8 kg/m 2 Weight 179.5 lb CR- SMFRS Mild (1) PR- SMFRS Slight (1) Sa7sfac7on a Sa5sfied (5) Skin Laxity b None (1) a Sa5sfac5on based on SSRS b Laxity based on SMSLG (5 Treatment Sessions) 13
ATX- 101 Ahrac5ve Compe55ve Profile Lihle- to- no Down5me Precise & Specific Ease of Physician Adop5on Substan5al Safety Database Customizable to Aesthe5c Goal If approved, ATX- 101 will be the first injectable drug for SMF No Capital Equipment Short Procedure Time Non- surgical Familiar Injectable Paradigm Robust Clinical Data Predictable 14
Aesthe5cs Market is Poised for Con5nued Double- Digit Growth And There is Growing Interest in the Chin Market for aesthetic injectables is large and growing $1.3 billion 1 and injectables saw a 21% increase in 2013, according to the American Society of Aesthetic Plastic Surgery 2 Jaw Makeovers for the Surgery-Averse, Superhero-Inspired Man The New York Times, December 3, 2014 1. Internal Es5mates 2. American Society of Aesthe5c Plas5c Surgery, ASAPS, 2013 15
Submental Fullness is Caused by Gene5cs and Aging - Not Just Weight Gain AGING Unfavorable changes - appearance of submental fullness Redundancy of skin, accumula5on of fat and skin laxity GENETICS Evidenced by pa5ents at any age who can present with extensive fat deposits, but not necessarily overweight Inherited, but tends to worsen with age WEIGHT GAIN Most individuals tend to gain weight as they age Moderate weight gain may result in an increased prominence of submental fat 16
ATX- 101 Opportunity Expected to Expand into Broad Consumer Base and Other Poten5al Indica5ons Exis7ng Facial Injectable Consumers Women Affected by SMF but Naïve to Injectables Men Affected by SMF but Naïve to Aesthe5c Treatments Poten5al for Future Indica7ons Small, Localized Fat Deposits with High Aesthe5c Value $ Time 17
ATX- 101 Pa5ent Pool Growth Comes from Injectable Experienced Followed by Injectable Naive Injectable Experienced In millions 8.0 7.0 6.0 5.0 4.0 3.0 2.0 1.0 0.0 Pa5ents with treatable SMF 2 Pa5ents using toxins, fillers 1 New Users Modeled at 12% CAGR 3 Pa5ents using toxins, fillers 1 2014 Est 2022 Est Pa5ents with treatable SMF 150 Injectable Naive Popula7on Growing at 3% 4 Age and Income Eligible 125 Age and Income Eligible 4 100 in millions 75 50 25 0 Aesthe5cs Considerers 5 Bothered by SMF 2014 Est 2022 Est Aesthe5cs Considerers Bothered by SMF Sources: 1. ASAPS 2013 (assumes 3 procedures/pa5ent) 2. KYTHERA Market Research 2009 and 2010 3. Millennium Research Group 4. U.S. Census 2013 5. Industry Es5mates 18
Our Approach to Commercializa5on Build Passionate Team Experienced in Bringing Blockbuster Aesthe7cs to Market Plan, Plan, Plan, but Adapt to Market Feedback Engage and Train Physicians to Become Brand Ambassadors Emphasize Significant Physician Prac7ce Growth Opportunity with ATX- 101 A T X 1 0 1 Leverage Growing Interest in the Chin Targe7ng Those Already Using Injectables First Execute with Excellence 19
ATX- 101 Commercializa5on Success Plan KYTHERA S Commercializa7on Strategy to Capture the Poten7al $500+ Million U.S. Opportunity for ATX- 101 reate physician awareness of condition & ATX-101 rm & engage KOLs and high-value physicians ropel physicians to start patient conversation urn patients into advocates nleash consumer demand with push-pull strategy ecruit top sales team xecute on operations, manufacturing and technology 20
Create Physician Awareness of Submental Fullness Through Unbranded Campaign Kicked Off At ASDS Nov 2014 SwiY A, Remington K. Beau5PHica5on: a global approach to facial beauty. Clin Plast Surg. 2011; 38:347-77. Dayan S. Neck rejuvena5on. In: Hirsch, Aesthe5c Rejuvena5on: A Regional Approach. 1st ed. New York, NY: McGraw Hill Professional Publishing; 2008: 123-147. 21
Arm & Engage Physicians With Educa5on and Training SEE IT Make Physicians Aware of New Treatment Op7on BELIEVE IT Communicate the Value Proposi7on TREAT IT Maximize Physician Produc7vity Pa7ent Conversion WHO: WHAT: WHY: HOW: Target top 3,000 high- value facial aesthe5c accounts, beginning with KOLs, including dermatologists and plas5c surgeons Promote ATX- 101 usage in accounts with large number of exis5ng injectable pa5ents Enable rapid adop5on by physicians and exis5ng injectable pa5ents, then expand into other pa5ent targets Physician and pa5ent engagement strategies 22
Robust Post- Approval Training Program Planned if Approved Physician Training Modules Condi7on & unmet need in submental contouring Anatomy of the cervicomental region ATX- 101 MOA Phase III clinical data Injec7ng ATX- 101 Op7mizing the pa7ent experience Timing Objec7ve On-Demand Learning 2H 2015 Robust online video learning Physicians have ongoing 24-hour availability to access KYTHERA Educational Congress 2Q 2015 KOL faculty training meeting Small Group Training 2H 2015 Faculty-led small-group hands-on injection training Live Webinars 4Q 2015 Live faculty presenter Injection training Mix of pre-recorded and live 23
Recruit and Train Top Sales Team Ready at Launch Mapped out top 3,000 accounts concentrated around main metropolitan areas. 34% of the accounts are in CA, TX and FL* Plan: 8 regional managers & 73 sales reps in 2015 to reach targeted accounts *Internal research 24
Consumers Place High Value on Aesthe5c Outcomes Facial Injectable Products (Repeated) 1 Surgical (One 5me) 1 Treatment Per Treatment Dura7on # Treatments Per Year Avg Consumer Cost Per Year Treatment Avg Consumer Cost Per Treatment BOTOX 3 To 4 Months Restylane / 6 To 12 Juvederm Months Voluma 12 To 24 Months 2 to 4 $800-1,600 2 to 3 $1,200-1,800 ½ to 1 $1,600-3,200 Chin Liposuc7on $3,500-5,000 Breast Implants $5,000-7,000 Blepharoplasty $2,700-3,800 For Consumers Who Get Combina7ons of Injectables, the Range of Annual Costs Can Be $2,000 3,600 Assump5ons for ATX- 101 High- Value Correc5ve Aesthe5c Outcome with Long- las5ng Results Physician Time and Value Similar to Exis5ng Injectable Products 1. American Society for Aesthe5c Plas5c Surgery, Your Plas5c Surgery Guide; Beauty World News; American Academy of Facial Plas5c and Reconstruc5ve Surgery, Inc. 25
Roadmap to Successful Product Launch 2014 Q1 2015 Q2 2015 Q3 2015 Q4 2015 2016 May 13, 2015 PDUFA Date Commercial Product Availability Medical Affairs Field Deployment SMF Condi7on Registry (CONTOUR) KOL Engagement Physician Training Hire Field Force Finalize Pricing Infrastructure Launch Readiness (ERP, CRM, Distribu7on) CMC Prep (FDA Pre- Approval Inspec7ons, Distribu7on System) Promo7onal Campaign Direct to Pa7ent Marke7ng (DTP) DTC Marke7ng 26
KYTH- 105 (se5piprant) KYTH- 105 is a Well Characterized Molecule Is believed to directly affect the hair loss pathway by blocking a prostaglandin in the scalp that prevents hair from growing, and by extending the growth phase of the hair cycle Has undergone more than 8 clinical trials in allergic rhini5s and seasonal allergies and has a safety database of >1,000 subjects o Treatment in all studies was well tolerated across all treatment groups Fits Well With Company Strategy Aligns with our focus on developing drugs that enable customers to achieve a healthy and posi5ve self- image Fits well with exis5ng clinical and regulatory experience Could be a new treatment to the large and s5ll highly unsa5sfied hair loss market Cri7cal and Broad IP Acquired exclusive worldwide rights to KYTH- 105 Acquired certain patent rights covering the use of PGD 2 receptor antagonists for the treatment of hair loss (oyen presen5ng as male pahern baldness, or androgenic alopecia) Next Steps Plan to file an Inves5ga5onal New Drug (IND) applica5on Plan to ini5ate a proof- of- concept study to establish the efficacy of KYTH- 105 as an oral therapy in male subjects with androgenic alopecia (AGA) in late 2015/early 2016 27
We Have Secured Cri5cal and Broad IP Worldwide patent estate includes over 100 issued or allowed patents and over 100 pending patent applica5ons with claims rela5ng to ATX- 101 Patents expire 2025 through 2031 Patents Include: Methods of Use Formula5on Composi5on Manufacturing ATX- 101 KYTH- 105 (se7piprant) Worldwide Method of Use patent applica5ons for the inhibi5on of PGD 2 for hair loss, filed in major markets o Covers methods of use for modulators of the prostaglandin D 2 receptors for hair growth Covers KYTH- 105 (se5piprant) and analogues o 48 worldwide patents and patent applica5ons; 45 of which are granted for molecules that inhibit PGD 2 28
Team Proven in Commercializing Aesthe5cs and Driving Scien5fic Innova5on Keith Leonard M.S., M.B.A. Co-Founder, President and CEO Tom Grady VP, Sales Heather Rowe M.A. Director, Investor Relations Frederick Beddingfield, III M.D. Ph.D. Chief Medical Officer Ryan Irvine Ph.D. VP, Medical Affairs Diane Stroehmann M.S. VP, Regulatory Affairs, Pharmacovigilance, Clinical Quality Elisabeth Sandoval M.B.A Chief Commercial Officer Keith Klein J.D. General Counsel Frank Watanabe VP, Strategy & Corporate Development John Smither Chief Financial Officer Susan Lundeen VP, Human Resources Doug Rich VP, Operations 29
Financial Posi5on (As of December 31, 2014) Cash, Cash Equivalents, and Marketable Securi5es of Debt Comprised of Note Payable to Bayer with a Face Value of $51M o Net of debt discount at date of issuance: $22.1M; due in 2024 $6M Note Payable to Lighthouse o due in 2016 $99.7 M $28.1 M 30
Recent Achievements and Upcoming Milestones Upcoming Milestones q KYTHERA Repurchased Rights to ATX- 101 outside the U.S. and Canada from Bayer q KYTHERA ATX- 101 US NDA Filed q KYTHERA ATX- 101 Canadian NDS Submihed Recent Achievements q KYTHERA ATX- 101 MAA Submihed in Switzerland q KYTHERA ATX- 101 Drug Applica5on Submihed in Australia q Advisory Commihee (March 9) Ini5ate SMF Condi5on Registry (CONTOUR) U.S. FDA PDUFA Date (May 13, 2015) Commercial Launch Addi5onal ex- U.S. Filings Ini5ate Proof- of- Concept Study for Se5piprant for Hair Loss Build Pipeline Licensing/Partnerships R&D Development of Innova5ve Technologies 31
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