Forward Looking Statements

Transcription

1 March 12,

2 Forward Looking Statements This presenta5on contains forward- looking statements within the meaning of the Private Securi5es Li5ga5on Reform Act of 1995 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 our Annual Report on Form 10- K and subsequent quarterly reports, all of which are filed with the SEC, par5cularly in the sec5ons 5tled Risk Factors and Management s Discussion and Analysis of Financial Condi5on and Results of Opera5ons. 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 awer 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

3 KYTHERA Acquires Worldwide Rights for Poten5al Novel Treatment of Hair Loss 3

4 Key Elements to a Successful Program are in Place Scien5fic Insight Company Capabili5es Market Poten5al Right Molecule Cri5cal IP 4

5 Scien5fic Insight PGD 2 Plays Key Role in Hair Loss George Cotsarelis MD, head of dermatology at the University of Pennsylvania Discovered that prostaglandin D 2 (PGD 2 ) played major role in hair loss 1 - Defined PGD 2 as an inhibitor of hair growth in male pabern hair loss - Suggested inhibi5on of the PGD 2 receptor as a poten5al target for treatment 2.5x higher amount of PGD 2 in bald spots than where hair growing* High PGD 2 PGD 2 Produc4on in Bald Scalp as Measured by HPLC Mass Spectrometry Low PGD 2 PGD 2 found to inhibit hair growth; blocking PGD 2 receptor may promote hair growth or retard hair loss 1 Science Transla5onal Medicine, Med 21 March 2012:Vol. 4, Issue 126, p. 126ra34 5

6 Scien5fic Insight Blocking PGD 2 Receptor Should Retard Hair Loss Preclinical models showed that PGD 2 receptor antagonists prevented nega5ve effect of PGD 2 on hair growth 1 I. Abolished hair growth inhibi5ng ac5vity of PGD 2 in hair follicles extracted from both male and female human donors (in vitro) II. In addi5on, PGD 2 suppression may extend anagen phase in hair In vivo shortened telogen and extend anagen phase in animals o Extension of anagen believed to be a way to combat hair loss Hair Growth Stages Anagen (growth) Catagen (regression)(res3ng) Telogen Goal: Extend anagen (growth) phase In balding scalps anagen phase shorter PGD 2 receptor antagonist extends anagen phase Research Suggests Blocking PGD 2 Extends Anagen (Growing) Phase 1 Data on file 6

7 Scien5fic Insight PGD 2 Inhibits Growth of Human Hair in vitro But PGD 2 R Antagonists Able to Reverse Inhibitory Effect PGD 2 inhibits growth of in vitro human hair follicles 1 Example of posi5ve response from hair follicles from human donor Growth of follicles from most donors nega5vely affected by PGD 2 Se5piprant and other PGD 2 R antagonists able to reverse this effect in vitro 2 Results demonstrated with five dis5nct PGD 2 R antagonists Effec5ve in reversing inhibitory effects in follicles from most but not all donors Effect is dose dependent but with plateau 2 PGD 2 + Se4piprant Population Analysis from 1,041 follicles from 13 donors Test Ar4cle Comparator P* Vehicle PGD 2 < PGD 2 + Se5piprant 10 nm PGD PGD 2 + Se5piprant 100 nm PGD PGD 2 + Se5piprant 1000 nm PGD * Differences of Least Square Means PGD 2 Appears to Arrest Hair Follicle Growth in Both Men and Women 1 Science Transla5onal Medicine, Med 21 March 2012:Vol. 4, Issue 126, p. 126ra34 2. Data on file 7

8 Scien5fic Insight Working Hypothesis of PGD 2 s Role in Hair Loss Scien5fic Insight Se4piprant Testosterone 5α- reductase Androgen Receptor Level and Ac4vity PGD2 synthase ac3vity PGD 2 Produc4on PGD 2 Receptor Ac4va4on Reduced Hair Growth and Eventual Hair Loss Dihydro- testosterone (DHT) Hormone Enzyme Receptor Prostaglandin 8

9 Company Capabili5es Fits Corporate Intent and Capabili5es Fits KYTHERA strategy to build leading aesthe4cs company o Aligns with our focus on developing drugs that enable customers to achieve a healthy and posi5ve self- image o Already had ac5ve research programs in hair o Novel approach Fits KYTHERA s lean research model o KYTHERA acted quickly to secure exclusive op5on o Commibed to long- term research collabora5on with Dr. Cotsarelis and the University of Pennsylvania o First 5me a hypothesis- driven approach taken to discovery of a hair growth treatment Fits well with exis4ng experience o Demonstrated clinical development, regulatory and manufacturing exper5se of similar- stage molecule (ATX- 101) Fits well into exis4ng clinical and regulatory capabili4es o In- house experience in developing drugs for the preven5on of hair loss KYTHERA combines the scien5fic and clinical rigor of biotechnology with the abrac5ve market poten5al of aesthe5cs 9

10 Right Molecule Se5piprant, Selec5ve PGD 2 Receptor Antagonist; Poten5al to Block Effects of PGD 2 on Hair Growth Se5piprant Is a selec5ve oral antagonist to the PGD 2 receptor Has been tested in mul5ple clinical trials as a poten5al allergic inflamma5on treatment Has undergone 8 clinical trials, including: - a Phase III study in seasonal allergic rhini5s pa5ents - a Phase IIB proof of concept study in asthma pa5ents Has a safety database of >1,000 subjects - Treatment in all studies was well tolerated across all treatment groups KYTHERA sees poten5al product advantages including: Oral dosing (improved compliance) No an5- androgenic effects expected (esp. sexual dysfunc5on) New and poten5ally complementary therapeu5c approach 10

11 Right Molecule Se5piprant is a Well- characterized Molecule with Large Safety Database Pre- Clinical Studies In vitro herg, phototoxicity studies Genotoxicology in vitro and in vivo Safety pharmacology in 3 species General toxicology in 3 species including 26- week rodent and 52- week dog Reproduc5ve toxicology in 2 species Carcinogenicity studies in 2 species (final analysis pending) Clinical Studies Phase I SAD/MAD in healthy volunteers Phase I ADME in healthy volunteers Phase I DDI with simvasta5n in healthy volunteers Phase I rela5ve BE study in healthy volunteers Phase II proof- of- mechanism study in asthma Phase IIB proof- of- concept in seasonal allergic rhini5s Phase IIB proof- of- concept study in asthma Phase III study in seasonal allergic rhini5s 11

12 Adverse Events in KYTH- 105 (Se5piprant) Phase III Seasonal Allergic Rhini5s Clinical Trial Treatment Emergent Adverse Events (including unrelated) Reported in More than 1 Subject in Se3piprant Group 1 Total pa5ents with at least one AE (%) Se4piprant 1000 mg BID n = 211 Placebo n = 208 Ce4rizine 10 mg QD n = (11.4%) 34 (16.3%) 23 (11.0%) Total number of AEs AEs reported in more than 1 pa5ent (> 0.5% ) in the se5piprant group Dry mouth (%) 4 (1.9%) 3 (1.4%) 2 (1.0%) Nausea (%) 4 (1.9%) 0 2 (1.0%) Somnolence (%) 2 (0.9%) 3 (1.4%) 4 (1.9%) Hepa5c enzyme eleva5on 2 (0.9%) 2 (1.0%) 0 Anxiety 2 (0.9%) 0 0 Majority of AEs were considered mild or moderate AEs considered by the inves5gator to be treatment- related were reported in 5.7% of se5piprant, 10.6% of placebo, and 4.3% of ce5rizine subjects 1 subject on se5piprant* (0.5%), 5 pa5ents on placebo (2.4%), and 2 pa5ents on ce5rizine (1.0%) discon5nued the study due to AEs 1 Data on file * Subject on se5piprant who discon5nued is pa5ent with SAE of cholelithiasis 12

13 Serious Adverse Events in KYTH- 105 (Se5piprant) Phase III Seasonal Allergic Rhini5s Clinical Trial Total pa5ents with at least one SAE (%) Se4piprant 1000 mg BID n = 211 Placebo n = 208 Ce4rizine 10 mg QD n = (0.5%) 0 0 Total number of SAEs SAEs Serious Adverse Events 1 Cholelithiasis 1 (0.5%) 0 0 No deaths in any study group 1 Data on file 13

14 Cri5cal IP We Have Secured Cri5cal and Broad IP University of Pennsylvania IP Exclusive worldwide licenses Method of Use patent applica5ons for the inhibi5on of PGD 2 for hair loss, filed in major markets Covers methods of use for modulators of the prostaglandin D 2 receptors for hair growth Actelion IP Exclusive worldwide licenses Covers se5piprant and analogues 48 patents and patent applica5ons; 45 of which are granted for molecules that inhibit of PGD 2 14

15 Market Poten5al Hair Loss Treatment is a Large and Growing Market Despite shortcomings of exis3ng treatments, hair loss treatment is a $2B and growing market $3.0 $2.8 Revenue ($B) $2.0 $2.0 Global alopecia therapeu5cs market forecast to grow at CAGR of 4.8% from 2010 to $ Hair loss affects a majority of men >50 years of age 80% of Caucasian men experience some degree of androgenic alopecia (AGA) before age GMRData: The Global Dermatology Market A. Alsantali, J. Shapiro, Androgens and hair loss. Curr. Opin. Endocrinol. Diabetes Obes. 16, (2009) 15

16 Market Poten5al Many Men and Women Suffer from Hair Loss, Which Can Have Significant Detrimental Effects on Self- Image Number Of: U.S. Men Experiencing Hair Loss 35 Million 1 U.S. Women Experiencing Hair Loss 21 Million 1 Hair loss sufferers, world- wide, seeking professional treatment >970 K 2 Percent of Hair Sufferers (n = 571) Who Said Their Hair Loss: Makes them feel less abrac5ve 26% 3 Makes them self- conscious 25% 3 Makes them look older 18% 3 Makes them less confident 15% 3 73% would trade a treasured personal possession for more hair 3 1 Interna5onal Society of Hair Restora5on Surgery, ISHRS Prac5ce Census Results ISHRS Hair Transplant Challenge Survey 16

17 Program Next Steps 2015 / 16 Open an IND for se5piprant Ini5ate a Proof- of- Concept study for se5piprant 17

18 Key Elements to a Successful Program are in Place Scien5fic Insight Company Capabili5es Market Poten5al Right Molecule Cri5cal IP 18

19 March 12,