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New Hope for Serious Infections Corporate Presentation August 2019 © Cidara Therapeutics 2019

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Page 1: New Hope for Serious Infections - ir.cidara.com

New Hope for Serious InfectionsCorporate PresentationAugust 2019

© Cidara Therapeutics 2019

Page 2: New Hope for Serious Infections - ir.cidara.com

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Forward-Looking Statements

These slides and the accompanying oral presentation contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.

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 of rezafungin, the potential for rezafungin to treat and/or prevent infections, whether the top line results of the STRIVE Part B clinical trial will be supported in the full analysis of the STRIVE Part B clinical data; whether the success of the STRIVE Part B clinical trial indicates a successful outcome in the Phase 3 ReSTORE clinical trial, including whether or not rezafunginwill meet the primary endpoints in the ReSTORE trial; and whether Cidara will be able to successfully develop and commercialize rezafungin; as well as the potential market size for rezafungin, ability of rezafungin to capture market share from existing therapies, and the advantages of rezafungin in other settings of care. Certain statements regarding our Cloudbreak platform are also forward-looking including statements regarding whether our Cloudbreak platform can identify product candidates with intrinsic antimicrobial activity and immune engagement that will increase efficacy or represent an improvement over existing anti-infective agents; whether Cloudbreak candidates, including CB-012, will achieve the major attributes believed to be needed in flu such as broad spectrum, superior resistance profile, protection for high-risk populations, expanded efficacy window, long duration of action and rapid onset of activity, or flexible administration; whether results observed with Cloudreakinfluenza candidates, including CB-012, in-vitro or in animal studies, including, potency and broad coverage, activity against resistant strains, activity in immune compromised patients, extending the treatment window, extended half-life and long duration of action, improved viral clearance in the lungs, improved reduction in inflammatory cytokines, and a robust safety profile or other observed attributes represent an improvement over existing therapies or will also be observed in human use;

and whether our Cloudbreak platform can be expanded to identify product candidates to treat or prevent other viral diseases, such as RSV, HIV, Dengue or Zika. This presentation also contains estimates and other statistical data made by independent parties and by Cidara relating to market size and growth and other data about Cidara's industryThese data involve a number ofassumptions and limitations, and you are cautioned not to give undue weight to such estimates. Projections, assumptions and estimates of the future performance of the markets in which Cidara operates are necessarily subject to a high degree of uncertainty and risk. Risks that contribute to the uncertain nature of the forward-looking statements include: Cidara’s abilityto obtain additional financing; the success and timing of Cidara’spreclinical studies, clinical trials and other research and development activities; receipt of necessary regulatory approvals for development and commercialization, as well as changes to applicable regulatory laws in the United States and foreign countries; changes in Cidara’s plans to develop and commercialize its product candidates; 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 as most recently filed with the United States Securities and Exchange Commission (SEC), under the heading “Risk Factors.” All forward-looking statements contained in this 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.

Page 3: New Hope for Serious Infections - ir.cidara.com

Cidara’s pipeline addresses multiple unmet needs

CandidaTreatment Intravenous

Fungal Prophylaxis Intravenous

CLOUDBREAK

Indication Program Discov. in vitro in vivoIND-

enable Ph 1 Ph 2 Ph 3

Influenza Prevention &Treatment

Antiviral Fc Conjugates (AVCs)

RSV, HIV, Dengue, Zika AVCs

REZAFUNGIN

3

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Cloudbreak

Cidara addresses multiple unmet medical needs

4

Rezafungin

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28%

37%

43%

50%

59%

60%

C. parapsilosis

C. albicans

C. tropicalis

C. glabrata

C. krusei

C. auris

High mortality reflects fungal disease severityOveruse of azoles has driven resistant strains

Crude Mortality (%)n=1,890 cases between 1995 and 20021

>30x mortality risk if:

incorrect drug used treatment delayed ≥24 hrs3

1 Wisplinghoff H et al. Clin Infect Dis. 2004;39(3):309-317 for all species other than C. auris. 2 Clin Infect Dis. 2018 Jan 6; 66(2): 306-311.3 Kollef CID 2012:54 (15 June). 5

2

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Invasive fungal infections cause high mortality post transplant

1 The PATH (Prospective Antifungal Therapy) Alliance registry and invasive fungal infections: update 2012 (2012).

90-day mortality % by patient category1

63%

52%

40%

38%

26%

24%

23%

HSCT

Hematologic malignancy

Solid tumor

General medicine

Surgical (nontransplant)

HIV/AIDS

Solid organ transplant

Prophylaxis focusin Phase 3 trial

Bone and marrow transplant

6

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Antifungal drug development has dwindled

1980 1990 2000 2010 2020

6 5 1Number of New Antifungals

fluconazole

itraconazole

terbinafineamphotericin lipid forms (3)

micafungin

anidulafungin

voriconazole

caspofungin

posaconazole isavuconazole

Page 8: New Hope for Serious Infections - ir.cidara.com

POLYENES

AZOLES

1ST GEN ECHINOCANDINS

REZAFUNGIN (ECHINOCANDIN)

POLYENES

8

Rezafungin for treatment in the critically ill

AZOLES

1ST GEN ECHINOCANDINS

REZAFUNGIN (ECHINOCANDIN)

Renal toxicitiesOnce-daily IV dosing

High levels of resistanceDrug-drug interactionsHepatic toxicities

Under-dosedOnce-daily IV dosing limits outpatient use

Establish novel drug in market

Spectrum

Oral formulation

No drug-drug interactionsEstablished safety

High exposure (efficacy in critically ill)Once-weekly dosing(early discharge and outpatients)

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Rezafungin for prophylaxis in hem/onc patients

Host and Macro-environment• Chronic immunosuppression• Novel biologics• Epidemiology - azole resistance

Antifungal therapies• Drug-drug interactions• Toxicities (bone marrow, liver, kidney) • Under-dosing

Page 10: New Hope for Serious Infections - ir.cidara.com

The Rezafungin opportunity spans ID and hematology

Mostly Treatment Mostly Prophylaxis

Cancidas(caspofungin)

~$700M

Noxafil(posaconazole)

~$700M

Infectious Disease Hematology

REZAFUNGIN

Source: Cancidas – IQVIA; Noxafil – 2018 Merck Annual Report10

Page 11: New Hope for Serious Infections - ir.cidara.com

Rezafungin overall phase 3 development plan

Phase 3 Treatment Trial

Indication

Phase 3 Size

Duration of Therapy, Endpoints, Comparators

Treatment of candidemia & invasive candidiasis in patients with limited treatment options

184 patients

2 to 4 weeks of treatment,Day 30 all-cause mortality (US)Day 14 global response (EMA)Caspofungin

Phase 3 Prophylaxis Trial

Prophylaxis against Aspergillus, Candida & PCP in patients undergoing allogeneic blood and marrow transplant

~450 patients with adaptive design

90 days of prophylaxisDay 90 fungal-free survival (FFS)Fluconazole, posaconazole, Bactrim

* Final trial design and timing are subject to obtaining adequate funding and the approval of regulatory authorities.

*

11

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STRIVE B Phase 2 data in candidemia & invasive candidiasisCorroborates STRIVE A results and supports ReSTORE Phase 3

12

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Week 1 2 3 4 5 6 7 8 9

Day1 5 8 15 22 28

Dose Optional dose

Mycological & clinical response

Overall Response (Mycological & clinical response): 1° ENDPOINT

Mycological & clinical response (IC only)

4535 42 49 56 59

Mycological & clinical response

Week 1 2 3 4 5 6 7 8 9

Day1 5 8 15 22 28 4535 42 49 56 59

Dose

All cause mortality

Analysis Populations: The Intent-to-treat (ITT) population: all randomized subjects The Safety population: all subjects who received any amount of study drug The Microbiological Intent-to-treat population (mITT): all subjects in safety population who had documented

Candida infection

Randomization 2:1

Caspofungin

Rezafungin

Optional dose

P2 STRIVE Part B: Candidemia & Invasive CandidiasisNot powered for inferential statistics

13

Page 14: New Hope for Serious Infections - ir.cidara.com

Rezafungin 400 / 200mg

RESTORE (n=184)

STRIVE B: the bridge from STRIVE A to ReSTORE

Rezafungin 400 / 400mg

Caspofungin

2017 2018 2019 2020

STRIVE B (n=91)STRIVE A (n=92)

STUDY SIZE: STRIVE A + B (n=183) ≈ RESTORE (n=184)

CRITERIA: Similar inclusion/exclusion, except STRIVE B enrolled patients with invasive candidiasis from the beginning

STRATEGY: STRIVE B expands safety data; maintains enrollment momentum

14

Page 15: New Hope for Serious Infections - ir.cidara.com

Similar to the ReSTORE trial primary endpoint for FDA30-Day All Cause Mortality

15.2%

6.7%

16.3%

Caspofungin

Rezafungin 400/200

Rezafungin 400/400

13.1%

4.4%

15.8%

Caspofungin

Rezafungin 400/200

Rezafungin 400/400

STRIVE B

STRIVE A + B

7/43

1/15

5/33

12/76

2/46

8/61

n/N=

n/N=

Death at Day 30 (%)mITT Population

1

2

3

1

2

3

1. 400 mg dose once weekly for two to four weeks.2. 400 mg dose for the initial week followed by 200 mg dose once weekly for an additional one to three weeks.3. 70 mg day one, followed by daily doses of 50mg. 15

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Similar to the ReSTORE trial primary endpoint for EMAInvestigator assessment of clinical response

69.7%

86.7%

65.1%

Caspofungin

Rezafungin 400/200

Rezafungin 400/400

70.5%

80.4%

69.7%

Caspofungin

Rezafungin 400/200

Rezafungin 400/400

STRIVE B

STRIVE A + B

28/43

13/15

23/33

53/76

37/46

43/61

n/N=

n/N=

Clinical Cure (%) at Day 14mITT Population

1. 400 mg dose once weekly for two to four weeks.2. 400 mg dose for the initial week followed by 200 mg dose once weekly for an additional one to three weeks.3. 70 mg day one, followed by daily doses of 50mg.

1

2

3

1

2

3

16

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STRIVE B primary endpoint: combination of clinical and mycological responsesOverall response

69.7%

86.7%

62.8%

Caspofungin

Rezafungin 400/200

Rezafungin 400/400

67.2%

76.1%

60.5%

Caspofungin

Rezafungin 400/200

Rezafungin 400/400

STRIVE B

STRIVE A + B

27/43

13/15

23/33

46/76

35/46

41/61

n/N=

n/N=

Overall Success (%) at Day 14mITT Population

1

2

3

1

2

3

1. 400 mg dose once weekly for two to four weeks.2. 400 mg dose for the initial week followed by 200 mg dose once weekly for an additional one to three weeks.3. 70 mg day one, followed by daily doses of 50mg. 17

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Topline summary of adverse events in safety population

400/400 mg (QWk)

400/200 mg (QWk)

PooledGroups

N=46 N=18 N=64

n (%)

All Related TEAEs 3 (6.5) 0 3 (4.7)

Leading to study D/C 2 (4.3) 0 2 (3.1)

Serious AE 1 (2.2) 0 1 (1.6)

70/50 mg (QD)

N=34

n (%)

5 (14.7)

3 (8.8)

1 (2.9)

REZAFUNGIN CASPOFUNGINAs expected and observed in STRIVE A, the majority of subjects had at least one TEAE and 40-50% had at least one Serious AE, reflecting the high morbidity of the underlying population.

There were no AE trends; % of TEAEs and SAEs were approximately even across study groups.

D/C=discontinuation; TEAE (treatment-emergent adverse event)=AE that occurs after first dose of study drug is administered.

N=81 N=53 N=134

All Related TEAEs 7 (8.6) 6 (11.3) 13 (9.7)

Leading to study D/C 3 (3.7) 0 3 (2.2)

Serious AE 1 (1.2) 1 (1.9) 2 (1.5)

N=68

9 (13.2)

1 (1.5)

2 (2.9)

STRIVE A + B

STRIVE B

Study-Drug Related TEAEs

18

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Our Phase 3 trial design mirrors the Phase 2 design

Phase 2

Week 1 2 3 4 5 6 7 8 9

1

Dose Optional dose

Mycological & clinical response: 1° ENDPOINT

All cause mortality

Day 8 15 22 28 45 59

Phase 3

Week 1 2 3 4 5 6 7 8 9

Overall response: 1° ENDPOINT EMA

All cause mortality: 1° ENDPOINT FDA

Objective: demonstrate non-inferiority to caspofungin

Page 20: New Hope for Serious Infections - ir.cidara.com

Rezafungin (STRIVE A + B, 400/200) vs. Caspofungin (STRIVE A + B)30-Day All Cause Mortality – Post Hoc Analysis*

SUPERIORITY NON-INFERIORITY

0-20% 10%-10%

-24.7% +0.41%

FAVORS REZAFUNGIN FAVORS CASPOFUNGIN

-8.8%

20%

ReSTOREPhase 3 trial endpoint requires upper limit of confidence interval be below 20% threshold

Non-inferiority margin

95% confidence interval

*Using the same analysis method as planned for the Phase 3 study, a two-sided 95% confidence interval (CI) for the observed difference in the ACM rate (Rezafungin 400/200 group minus caspofungin group) was calculated using the unadjusted method of Miettinen and Nurminen.

Rezafungin mITT: 2/46= 4.4% ACM; Caspofungin mITT: 8/61= 13.1% ACM

20

Page 21: New Hope for Serious Infections - ir.cidara.com

Rezafungin (STRIVE A + B, 400/200) vs. Caspofungin (STRIVE A + B)Day 14 Clinical Response – Post Hoc Analysis*

SUPERIORITY NON-INFERIORITY

020% -10%10%

26.6% -6.9%

FAVORS REZAFUNGIN FAVORS CASPOFUNGIN

9.9%

-20%

ReSTOREPhase 3 trial endpoint requires lower limit of confidence interval be above 20% threshold

Non-inferiority margin

95% confidence interval

*Using the same analysis method as planned for the Phase 3 study, a two-sided 95% confidence interval (CI) for the observed difference in the Clinical Response rate (Rezafungin 400/200 group minus caspofungin group) was calculated using the unadjusted method of Miettinen and Nurminen.

Rezafungin mITT: 37/46= 80.4% Cure; Caspofungin mITT: 43/61= 70.5% Cure

21

Page 22: New Hope for Serious Infections - ir.cidara.com

Cidara

Opportunity

Commercial

Competition

Business Development

Treatment: in- and outpatientProphylaxis: BMT/Hematology

Outpatient IV pricing & reimbursement (Part B)

13 years since last Candidaor prophylaxis launch

ID & Hem/Onc supportive care companies

”Typical” Abx company

Inpatient treatment

Hospital inpatient DRG

~15 Ph3 programs Multiple launches in 2018-19

ID focused companies

Advantages of expanding outside of the in-hospital market

22

Page 23: New Hope for Serious Infections - ir.cidara.com

Antifungal (Cresemba) launch outpaces recent antibiotics

Cresemba (isavuconazole) is a triazole launched in 2015 by Astellas in the US. In 2017, Pfizer acquired rights to izavuconazole from Basilea for EU, China, 16 Asia Pac countries. Source for sales data: IMS

23

0

10

20

30

1 2 3 4 5 6 7 8 9 10 11 12 13Launch Quarter

U.S. Sales Post Launch: Cresemba vs. Antibiotics

CRESEMBA (ANTIFUNGAL)

AVYCAZ

DALVANCEZERBAXAAVERAGE

ORBACTIV

ANTIBIOTICS

Sale

s ($

M)

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Antifungals are historically big drugs globally

BIG PHARMA ANTIFUNGALS Company Product Peak Global Sales ($) Class

MERCK Noxafil(posaconazole) 720M Triazole

Cancidas(caspofungin) 680M 1st Gen

EchinocandinPFIZER Vfend

(voriconazole) 800M Triazole

Diflucan(fluconazole) 1,000M Triazole

Eraxis(anidulafungin) 180M 1st Gen

EchinocandinPFIZER & ASTELLAS Cresemba

(isavuconazole)Launched

in 2015 Triazole

ASTELLAS Mycamine(micafungin) 370M 1st Gen

EchinocandinASTELLAS & GILEAD

Ambisome(amphotericin B) 510M Polyene

Source: IQVIA for all products other than Noxafil (2018 Merck Annual Report) and Diflucan (www.pharmaceuticalonline.com Feb 7, 2000)

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Cidara addresses multiple unmet medical needs

25

Rezafungin

Cloudbreak

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10%-60% effective(2004-2018)1

~2-week lag time to achieve full protection2

Difficult to scale, low yields can limit production capacity3

26

Vaccines for influenza have limitations

1. https://www.cdc.gov/flu/professionals/vaccination/effectiveness-studies.htm2. https://www.cdc.gov/flu/protect/keyfacts.htm3. https://www.cdc.gov/flu/professionals/antivirals/summary-clinicians.htm

Viral coverage Patient Manufacturing

Less effective in elderly & immune compromised

Challenging in a pandemic: long, complex production

Strain-specific, variable coverage

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Treatments for influenza also have limitations

Resistance emerges rapidly

48 hour window Limited in patients with complicated & severe disease

Resistance Administration Efficacy

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Cloudbreak antiviral conjugates (AVCs) for influenza:Potential single dose universal protection and treatment

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Cloudbreak platform – multimodal mechanism of action: intrinsic antimicrobial activity & immune engagement

Binds conserved surface targetDirect antimicrobial activity

Engages innate or adaptive immune system

TARGETING MOIETY

Pathogen ImmuneComponent

Fc MOIETY

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Cloudbreak AVCs combine the power of small molecules (SMs) and monoclonal antibodies

VIRUS

• High potency SMs

• Extended half-life

• Broad spectrum (influenza A&B)

• Combining multiple MOAs

Intrinsic antiviral activity

Conserved, essential target

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What would an “ideal” product look like?

Broad spectrum, universal coverage

Superior resistance profile

Protection for High-Risk Populations

Expanded efficacy window

Long duration of action

Rapid onset of activity

Flexible administration

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Potential for “universal” activity in vivo

Coverage

Resistance

Vulnerable

Efficacy

Duration

Rapid onset

Administration

100%

0%0 14Days

Surv

ival

CB-012 0.4 mg/kg

A/Texas/36/91 H1N1

Neg control (Fc only) 14Days

100%

0%

0

Surv

ival

A/Hong Kong/68 H3N2

CB-012 0.4 mg/kg

14Days

100%

0%0

Surv

ival

B/Malaysia/04

CB-012 0.4 mg/kg

Dose

Single low doses of CB-012 protect mice in lethal infection models

Similar in vivo results found in: A/Puerto Rico/8/34 (H1N1); A/WSN/1933 (H1N1); A/California/07/09 pandemic (H1N1); A/Perth/261/2009 (H275Y).

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Coverage of drug resistant strains

Coverage

Resistance

Vulnerable

Efficacy

Duration

Rapid onset

Administration

14Days

100%

0%

0

Surv

ival

Neg control (Fc only)Tamiflu 20mg/kg10 doses

Dose

CB-012 in Tamiflu-resistant H1N1

DosesCB-012, 2mg/kg1 dose

1. A/Perth/261/2009, Strain H275Y

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Potential for protection in vulnerable patients

Coverage

Resistance

Vulnerable

Efficacy

Duration

Rapid onset

Administration

Severe Combined ImmunoDeficiencyImmune competentBALB/c SCID miceBALB/c mice

Lethal influenza model (H1N1: A/Puerto Rico/8/34)

0%

Vehicle

0.3 mg/kg

0 14DaysSu

rviv

al

Dose100%

CB-012

0%

Vehicle

0.3 mg/kg

0 14Days

Surv

ival

Dose100%

CB-012

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Expansion of the treatment window

Coverage

Resistance

Vulnerable

Efficacy

Duration

Rapid onset

Administration

0 72 9624HOURS

48

CB-012

Tamiflu

INFECTION

CB-012:Tamiflu:

10 mg/kg20 mg/kg

Dose

Doses

Treatment initiated 72 HOURS post-infection

Mouse lethal endpoint model H1N1

Fc only

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Single dose given 28 days prior to viral challenge

36

Potential for long-term single dose protection

Coverage

Resistance

Vulnerable

Efficacy

Duration

Rapid onset

Administration

-28 0 14-14

100%

0%

Surv

ival

DAYS

Dose Infect

CB-012

Control

2.5 mg/kg

Lethal influenza model (H1N1: TX/36/91 in mice)

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Rapid therapeutic exposure in key tissues

Coverage

Resistance

Vulnerable

Efficacy

Duration

Rapid onset

Administration

Cmax 1 hr

Con

cent

ratio

n

Lung

Plasma

CB-012: MINUTES

VACCINES: WEEKS

CB-012 lung distribution10 mg/kg IV dose, mouse

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Flexible routes of administration

Coverage

Resistance

Vulnerable

Efficacy

Duration

Rapid onset

Administration

CB-012 dosed by different routes5 mg/kg, mouse

SubqIMIV

Cmax 4 hr

Cmax 24 hr

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Dose-dependent viral clearance in lungs

PBS control

Fc control (3 mg/kg)

Oseltamivir (5 mg/kg, BID x 4)

CB-012 (0.1 mg/kg)

CB-012 (0.3 mg/kg)

CB-012 (1.0 mg/kg)

CB-012 (3.0 mg/kg)

PFU / g (Log10)103 104 105 106 107 108

Viral burden (H1N1) in mouse lung – Day 4 post-infection

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40

Dose-dependent reduction in inflammatory cytokines

Concentration (pg/mL)0 400 800

PBS control

Fc control (3 mg/kg)

Oseltamivir (5 mg/kg, BID x 4)

CB-012 (0.1 mg/kg)

CB-012 (1.0 mg/kg)

CB-012 (3.0 mg/kg)

Uninfected

TNFα (Mouse lung)

Concentration (pg/mL)

IL-6 (Mouse Lung)

0 1000 1500

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Broad safety margin in rats and primatesResults of 14-day toxicity testing

0

20000

40000

60000

Rat Primate

Area under the curve (AUC) for maximum

dose tested (hr*µg/mL)

Therapeutic Margins

AUC required for efficacy in mice (hr*µg/mL)

15x 10x

Clinical observationsHematologyClinical ChemistryCoagulationUrinalysisHistopathology

NO FINDINGS FOR:

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Platform expansion beyond influenza

INFLUENZA

HIV

RSV

DENGUEZIKA

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Upcoming milestones

AugustOptions X: new Cloudbreak data

2nd half 2019Investor DayPhase 1 subcutaneous

OctoberIDWeek/TIMM - STRIVE A&B data

2019 2020MidyearPhase 3 ReSTORE topline

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Financial overview

1 Includes 26,767,989 common shares and assumes conversion of 565,231 shares of Series X Convertible Preferred into 5,652,310 common shares at June 30, 2019. Each share of Series X Convertible Preferred is convertible into 10 shares of common.

Summary Information ($M) June 30, 2019

Cash $44.6

Common shares issued 26.8

Common equivalent shares issued1 32.4

We expect to finance our cash needs through:• equity and debt financings,• entering into collaborations,

strategic alliances and licensing arrangements,

• receiving government and/or charitable grants or contracts.

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Cidara is much more than a typical ID company

Rezafungin Treatment

Rezafungin Prophylaxis

Cloudbreak AVC

Strategic Focus

Our Team

Large market with low dev risk (STRIVE A + B)

Hem/onc supportive care, high unmet need

Enormous influenza market potential & expansion opportunities

Not a ‘typical’ Infectious Disease company

Experienced creators of shareholder value

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New Hope for Serious InfectionsCorporate PresentationAugust 2019

© Cidara Therapeutics 2019 | Confidential