New Hope For Serious Infections. Jefferies 2016 Healthcare Conference June 7-10, 2016 Grand Hyatt, New York City

Transcription

1 New Hope For Serious Infections Jefferies 2016 Healthcare Conference June 7-10, 2016 Grand Hyatt, New York City

2 Forward-Looking Statements These slides and the accompanying oral presentation (the Presentation ) contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effectiveness, safety, long-acting nature, anticipated human dosing, potential to treat infections and other attributes of CD101 IV and CD101 topical, as well as the incidence of fungal infections and the effectiveness and treatment protocols for competitive therapies. Statements regarding the intended design of current and future Cloudbreak compounds are forward-looking. Risks that contribute to the uncertain nature of the forward-looking statements include: the success and timing of Cidara s preclinical studies and clinical trials; regulatory developments in the United States and foreign countries; changes in Cidara s plans to develop and commercialize its product candidates; Cidara s ability to obtain additional financing; Cidara s ability to obtain and maintain intellectual property protection for its product candidates; and the loss of key scientific or management personnel. These and other risks and uncertainties are described more fully in Cidara s Form 10-K most recently filed with the United States Securities and Exchange Commission (SEC), under the heading Risk Factors. All forward-looking statements contained in the Presentation speak only as of the date on which they were made. Cidara undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

3 Cidara April 2015: two preclinical platforms Volker Brinkmann, Wikimedia.org CD101 IV: Pre-clinical Cloudbreak : Immunotherapy

4 1 year post-ipo: substantial progress CD101 prophylaxis & other CD101 topical for VVC, Phase 2 CD101 IV for candidemia, Phase 2 CD101 topical IV prophylaxis for candidemia, for VVC, & other Phase Phase 2 2 Expanding to antiviral In-vivo proof of concept, antibacterial In-vivo proof of concept, antifungal In-vivo Expanding proof to of antiviral concept, antibacterial antifungal Volker Brinkmann, Wikimedia.org CD101 Cloudbreak

5 Solid execution beyond plan IPO: $77M Apr, Positive Phase 1 SAD/MAD CD101 IV Nov 2015/ Jan 2016 Submits IND to FDA for CD101 IV Jun, 2015 FDA fast-track & QIDP for CD101 IV May, 2015 Phase 2 start CD101 IV CD101 topical Orphan drug status CD101 IV Feb, 2016 FDA fast-track & QIDP for CD101 topical May, 2016 Cloudbreak Development candidate

6 Fungal infections: high mortality U.S. 12-Week Mortality per Annum by Species Candida 67,500 Aspergillus 22,500 Cryptococcus Rare Fungi 4,500 2,500 97,000 deaths per year

7 Echinocandins: first line therapy for candidemia DRUG PROFILE First line treatment for Candida Safe and well tolerated Superior to azoles BUT CONSTRAINED Once daily IV Low exposure suboptimal dosing Growing resistance USD Billions Polyenes Echinocandins Azoles Today

8 CD101 opportunity: expand echinocandin market ACCESS ENTIRE >$4 BILLION MARKET USD Billions Inpatient & outpatient 4.2 Treatment & prophylaxis Polyenes 0.5 Penetrate azole class Echinocandins 1.1 ~2x Expand indications Azoles 2.6 Today Future Illustrative

9 Cubicin established precedent as IV outpatient CUBICIN Outpatient CD101 Outpatient ~$720M: Outpatient sales (2015) ~$625M: Potential value* 20%: Share of outpatient market ~15%: CD101 potential share $240M: Initial projection inpatient 4%: Echinocandin current share From hospital to home From hospital to home Assumptions: 14 days OPAT: Micafungin cost per day (WAC) - $187, Cost of PICC placement - $1,024, Per diem OPAT costs - $219 *Assumes CD101 captures outpatient share = 80% current echinocandin inpatient share

10 CD101 a differentiated echinocandin 10 N + O HO H 3 C H 3 C HO O HO N + O O O - O N N H NH H N OH O HN O O OH O H N N O OH CH 3 OH Biafungin (CD101 Acetate) O HO Structural modification yields improved chemical & biological properties Prolongs PK Allows high exposures Eliminates toxic degradation products Enables multiple formulations ICAAC 2015 once weekly dosing treats less susceptible pathogens improved safety & dose range systemic and topical

11 CD101 Superior efficacy vs anidulafungin 11 CFU in kidneys of mice 24 hours after infection with C. albicans and treatment with anidulafungin or CD101 DOSE 0.0 mg/kg 0.5 mg/kg 1.5 mg/kg 5 24 hours Anidulafungin Log 10 CFU/Kidneys CD101 IV 1 0 Untreated Control Cohort 2 Cohort 3 CONFIDENTIAL

12 CD101 IV: streamlined development plan IND I II III Phase I Phase 2 Phase 3 PK and safety in healthy volunteers Double-blind vs. echinocandin in candidemia Non-inferiority vs. echinocandin in candidemia + invasive candidiasis

13 Phase 1 Single Ascending Dose (SAD) Study Design DAYS 50mg mg Each Dosing Group: 6 CD101 subjects 2 placebo subjects Dosed on day 1 200mg mg

14 Phase 1 SAD study: safe and well tolerated Adverse Events by Dose DOSE 50 mg 100mg 200 mg 400 mg Placebo Subjects w/ AEs 3 of 6 0 of 6 3 of 6 1 of 6 5 of 8 No serious or severe AEs No dose response in AEs No withdrawals due to AEs No clinically significant: Lab abnormalities ECG Vital signs

15 Once weekly high exposure dosing CD101 IV Phase I PK Mean Plasma Conc (µg/ml) AUC ~2.5x greater C max 3x higher AUC ~1.6x higher Anidulafungin 100mg x 7 400mg x 1 200mg x Days

16 Phase 1 Multiple Ascending Dose (MAD) Design DAYS 100mg Dosing day mg Each Dosing Group: 6 CD101 subjects 2 placebo subjects Dosed on days 1 and 8 400mg

17 Phase 1 MAD study: safe and well tolerated Adverse Events by Dose DOSE 100 mg 200mg 400 mg* Placebo Subjects w/ AEs 3 of 6 2 of 6 4 of 6 2 of 6 *Dosed weekly for 3 weeks No serious or severe AEs No withdrawals due to AEs No elevation of LFTs (AST, ALT, Alk Phos, Bili) in any CD101 subject Mild transient infusion reactions associated with 400mg dose (primarily with the third dose)

18 CD101 IV Phase 1 Summary CD101 IV is safe and well-tolerated up to 400mg Adverse events were similar between placebo and CD101 PK was dose-proportional and supports once weekly dosing

19 Phase 2: first of two studies for approval CD101 IV 400/400/(400)mg n=30 Dosing day Mycological response Mycological & Clinical response 1 o END POINT (optional) Mycological & Clinical response CD101 IV 400/200/(200)mg n=30 Dosing day (optional) Caspofungin IV 70/50/(50)mg n= (optional) mg 50mg daily 28 35

20 VVC/RVVC has widespread impact 75% have VVC in their lifetime US: 4-5M suffer RVVC OTC Topical US: 4.4M visits per year Rx Topical or Oral

21 Existing therapies have significant limitations % women fail acute treatment 50% relapse in RVVC No coverage of non-albicans Candida Increased risk of miscarriage (flucon) CDC guidelines recommend only using topical antifungals to treat pregnant women with VVC, including for longer periods than usual if these infections persist or recur CONFIDENTIAL

22 22 6 CD101 Topical eradicates infection in VVC Vulvovaginal Candidiasis (VVC) in Rats 5 Log 10 CFU/mL lavage Infect Treat CFU Topical Day 0 Days 2,3,4 Days 5,7,9,12 LOD D1 D5 D7 D9 D12 D5 D7 D9 D12 D5 D7 D9 D12 No Treatment Monistat tm CD 101 3% Gel ICAAC Poster: New Anti-Fungal Agents Saturday, Sept 19 Novel Echinocandin CD101 Gel Formulation is Highly Effective in Eradicating Candida albicans in a Rat Model of VVC, L. Miesel, et al. CONFIDENTIAL

23 Phase 2 VVC Study Design Objectives Safety/tolerability of CD101 Gel/Ointment Efficacy of CD101 Gel vs. CD101 Ointment vs. Fluconazole on Days 7, 14, 28 Mycological & Clinical response at days 7, 14 & 28 DAYS CD101 3% Gel n=50 Dosing day CD101 6% Ointment n=50 Fluconazole 150mg n= Efficacy

24 CD101 Topical development plan IND I II III Phase 2 Pivotal Study 1 Dose-ranging safety & efficacy in VVC patients Superiority in acute VVC Pivotal Study 2 Superiority in RVVC

25 Cloudbreak: a transformational technology Cloudbreak Physically connects the immune system with the pathogen Primes the immune system Vaccine Targets the pathogen Therapeutic The strengths of both systems

26 Cloudbreak concept and compound design Microbe TM EM Cell MICROBE TARGETING MOIETY EFFECTOR MOIETY IMMUNE CELL

27 Ex-vivo experimental design Microfluidic Schematic Actual microfluidics chamber Conidia Loading Neutrophil Input Well

28 Ex-vivo proof of concept No drug 1 nm Cloudbreak (1/70 th MEC) ICAAC Poster: New Anti-Fungal Agents Saturday, Sept 19 Bifunctional Small Molecule Immunotherapy. C-001 and C-016 Attract Neutrophils (PMNs) to Inhibit Aspergillus fumigatus (Af) Growth in Microfluidic Chambers, D. Irimia, et al.

29 Cloudbreak antibacterial in-vivo POC N=20 per cohort % Survival No treatment (vehicle) + antibody Cloudbreak treatment - antibody Cloudbreak treatment + antibody MDR E. coli model: carbapenem r, quinolone r, aminoglycoside r

30 Upcoming Milestones Program Next Milestone Expected Timing CD101 IV Phase 2 Start 1H 2016 CD101 Topical Phase 2 start Mid 2016 Cloudbreak Platform Development candidate 2016

31 New Hope For Serious Infections Jefferies 2016 Healthcare Conference June 7-10, 2016 Grand Hyatt, New York City