New Hope For Serious Infections

Transcription

1 New Hope For Serious Infections

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 and other attributes of CD101 IV and its potential to treat infections, the incidence of fungal infections, and the effectiveness and treatment protocols for competitive therapies. 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-Q 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 update: November 2015 CD101 IV Phase 1 SAD completed CD101 Topical Begin Phase 2 mid 2016 Cloudbreak Infectious disease immunotherapy

4 Cidara accomplishments since inception Series B: $42M Feb, 2015 Cidara formed Jan, 2014 Series A: $32M Jun, 2014 IPO: $77M Apr, Acquires CD101 portfolio May, 2014 Initiates CD101 Topical program Oct, 2014 Phase 1 SAD complete for CD101 IV Nov, 2015 Submits IND to FDA for CD101 IV Jun, 2015 FDA fast-track & QIDP for CD101 IV May, 2015

5 Cidara: an experienced team Jeff Stein, PhD CEO Dirk Thye, MD CMO Ken Bartizal, PhD CDO Paul Daruwala, RPh CCO Kevin Forrest, PhD COO Jim Balkovec, PhD SVP Research

6 Successful track record Antibacterial Antifungal Antiviral

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

8 CD101 Progress 4Q 2015 Program Indication Pre-Clin IND Enabling Phase 1 Phase 2 Phase 3 CD101 IV Candidemia/ Invasive Candidiasis CD101 Topical Acute and Recurrent VVC Projected 4Q 2016 Program Indication Pre-Clin IND Enabling Phase 1 Phase 2 Phase 3 CD101 IV Candidemia/ Invasive Candidiasis CD101 Topical Acute and Recurrent VVC

9 Cidara pipeline Program Indication Discovery Research/ In Vitro In Vivo IND Enabling Phase 1 Phase 2 CD101 IV Candidemia/ Invasive Candidiasis CD101 Topical Acute and Recurrent VVC Cloudbreak TM Platform Antifungal Cloudbreak Small and Large Molecule Programs Antibacterial Antiviral

10 CD101 A novel echinocandin antifungal in Phase I Structural modification yields superior chemical & biological properties Permanent charge and highly stable ring structure Prolongs PK: once weekly dosing Allows high exposures: treats less susceptible pathogens Eliminates toxic degradation products: improved safety & dose range Enables multiple formulations: systemic and topical ICAAC 2015

11 CD101 - potent against Candida and Aspergillus CD101, anidulafungin and caspofungin tested against a panel including azole & echinocandin-resistant strains Candida MIC 90 (µg/ml) Aspergillus MEC 90 (µg/ml) * albicans (n=100) glabrata (n=100) tropicalis (n=100) krusei (n=100) parapsilosis (n=100) fumigatus (n=20) terreus (n=19) niger (n=16) flavus (n=12) CD Anidulafungin < Caspofungin * CLSI methodology was employed for MIC/MEC determination, MECs vs Aspergillus were determined for CD101, anidulafungin and caspofungin. ICAAC 2014 & 2015

12 CD101 IV: superior efficacy at equivalent doses CFU in Kidneys of Mice 24 Hours after Infection with C. albicans and treatment with Anidulafungin or CD101 IV DOSE 0.0 mg/kg 0.5 mg/kg* 1.5 mg/kg** Log 10 CFU/Kidneys 5 Anidulafungin 4 3 CD101 IV Untreated Control Cohort 2 Cohort 3 *Statistically Significant at p < **Statistically Significant at p < 0.05

13 Phase 1 single ascending dose study design Number of Subjects by Dose DOSE 50mg 100mg 200mg 400mg TOTALS CD101 IV Placebo o Objective: Safety, tolerability and pharmacokinetics 32 Safety assessments included adverse events, ECGs, hematology, chemistry labs, urinalysis, vital signs, physical exam Subjects followed for 21 days after dosing

14 Phase 1 single ascending dose results Adverse Events by Dose DOSE 50mg 100mg 200mg 400mg Placebo Number of AEs Subjects w/ AEs 3 of 6 0 of 6 3 of 6 1 of 6 5 of 8 More Placebo AE than in all CD101 IV combined No serious AE No severe AE No dose-response in AE No withdrawals due to AE No vital sign, physical exam or ECG abnormalities No significant laboratory abnormalities (hematology, chemistry, LFTs)

15 Mean Plasma Conc (µg/ml) 1x weekly dosing achievable; high exposures CD101 IV Phase I PK Days 400mg 200mg

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

17 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 + High unmet need Broader initial label claim: invasive candidiasis

18 Systemic fungal infection market U.S. Days of Therapy (Millions) Inpatient M Inpatients Discharge 4.5 ~40% of inpatients on antifungals are discharged on antifungals outpatient Prophylaxis* 1.5 High risk for invasive fungal infection days of therapy per patient Global sales of the systemic antifungal market are ~$4B *High risk populations: Acute leukemia, Allo HSCT, SOT

19 Factors that favor CD101 IV 70% US % of non-albicans Candida International Epidemiology Guidelines Epidemiology International Guidelines 60% 2011 Pharmacoeconomics 50% 40% 2006 Converging factors increasing prevalence of non-albicans Candida 30% M. Pfaller. Mycoses, 2014, 57,

20 Factors converge to favor CD101 IV Epidemiology International Guidelines Pharmacoeconomics Superior efficacy of echinocandins vs azoles Rise in azole resistant pathogens Echinocandins recommended as first line therapy in candidemia Guidelines shifting to favor echinocandins Source: ESCMID* guideline for the diagnosis and management of Candida diseases 2012: non-neutropenic adult patients; European Expert Opinion on the Management of Invasive Candidiasis in Adults

21 Cost saving opportunities in multiple settings US Cost Hospital Stay* US Cost Outpatient $10,616 Epidemiology International Guidelines Pharmacoeconomics $4,550 $7,583 $3,863 $6,702 3 Days General Ward 5 Days General Ward 7 Days General Ward 7 Days Micafungin OPAT 14 Days Micafungin OPAT KOLs estimate that 5-30% of patients on an echinocandin can be discharged 3-7 days early Assumptions: General ward cost per day - $1,516 OPAT: Micafungin cost per day (WAC) - $187, Cost of PICC placement - $1,024, Per diem OPAT costs - $219

22 The differentiating value of a weekly echinocandin INPATIENT POST DISCHARGE PROPHYLAXIS TARGET Stable, eligible for early discharge; removal of central line; longer term Tx Echinocandin continuation; azole challenged; nonalbicans Azole challenged: high risk acute leukemia, allo HSCT, SOT VALUE Reduce cost and improve care Improve outcomes vs azoles, decrease OPAT costs Reduce deadly infections Echinocandins ~20% of TOTAL DOT* <5% of DOT <5% of DOT CD101 Potential Million DOT* *Days of Therapy

23 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

24 Cloudbreak concept and compound design Microbe TM EM Cell MICROBE TARGETING MOIETY EFFECTOR MOIETY HUMAN CELL Fungi Echiniocandin fmlf Neutrophil Fungal Hyphae Cidara Compound Neutrophils

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

26 Ex-vivo proof of concept for Cloudbreak No drug 1 nm C-001 (1/70 th MEC) ICAAC 2015 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.

27 Cloudbreak platform expansion Small Molecule Large Molecule Microbe TM EM Cell Microbe TM TM EM EM Cell TARGETING MOIETY EFFECTOR MOIETY TARGETING MOIETY EFFECTOR MOIETY Approved anti-microbial Small molecule & peptide-based immune engagers Antibody that binds cell-surface antigen Antibody that engages immune system INNATE IMMUNE SYSTEM ADAPTIVE IMMUNE SYSTEM

28 A450 nm Large Molecule Cloudbreak Microbe TM TM EM EM Cell Selected Immune Cells 1.5 Immune Cell Activation 1.0 Cells only TM TM-EM TM (-Antigen) TM(+Antigen) TM-EM(-Antigen) TM-EM(+Antigen) Microbe TM EM TM EM Cell Microbe TM EM TM EM Cell Microbe TM EM TM EM Cell

29 Cloudbreak Platform Expansion Anti-Bacterial Anti-Viral Indwelling Hardware Influenza

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

31 New Hope For Serious Infections