CORPORATE PRESENTATION
1 | L E A D I N G T H E S C I E N C E O F P R O T E C T I O N | J A N U A R Y 2 0 2 1 |
F O R WA R D - LO O K I N G S TAT E M E N T S
T h e s e s l i d e s c o n t a i n f o r w a r d - l o o k i n g s t a t e m e n t s w i t h i n t h e m e a n i n g o f t h e P r i v a t e S e c u r i t i e s L i t i g a t i o n R e f o r m A c t o f 1 9 9 5 .
The words "believe," "may," "will," "estimate," "promise," "plan", "continue," "anticipate," "intend," "expect," "potential" and similar expressions (including the negative thereof), are intended to identify forward-looking statements. 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 Cidara's research and development efforts; preclinical and clinical development activities; plans, projections and expectations for and the potential effectiveness, safety and benefits of, its product candidates, including rezafungin, the Cloudbreak platform and CD377; Cidara's ability to successfully commercialize its product candidates; and potential ability to achieve milestones under its collaboration with Mundipharma, and receipt of the related milestone payments; and advancement of its strategic plans.
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 industry. These data involve a number of assumptions 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, including, Cidara's ability to obtain additional financing; the success and timing of Cidara's preclinical 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 Securities and Exchange Commission, under the heading "Risk Factors."
Additional risks and uncertainties may emerge from time to time, and it is not possible for Cidara's management to predict all risk factors and uncertainties. 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.
These slides are not intended to and do not constitute an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities in any jurisdiction, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. No offer of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended.
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C I DA R A I N V ES T M E N T T H ES I S
L e a d i n g s c i e n c e o n l o n g - a c t i n g a n t i f u n g a l a n d a n t i v i r a l p r e v e n t i o n a n d t r e a t m e n t
REZAFUNGIN | 1st antifungal under development in 14 years for treatment and prophylaxis |
Treatment - Phase 3 | ReSTORE: treatment of candidemia and invasive candidiasis |
Prophylaxis - Phase 3 | ReSPECT: prevention in high risk hematology (BMT) setting |
Validation | Significant ex-US/ex-Japan partnership with Mundipharma |
CLOUDBREAK AVCs | Modular antiviral platform for prevention & treatment |
Influenza A+B | Target: 1st effective 'universal flu' product |
RSV, HIV, CoV | Progressing multiple pre-clinical programs |
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L EA D I N G T H E S C I E N C E O N A N T I F U N G A L & A N T I V I R A L P R E V E N T I O N & T R EAT M E N T
ANTIFUNGAL PROGRAMS
Product | Indications | IND-Enabling | Phase 1 | Phase 2 | Phase 3 |
Rezafungin Treatment | Candidemia and Invasive Candidiasis | ||||
Rezafungin Prevention | IFD in Blood & Marrow Transplant Patients | ||||
ANTIVIRAL PROGRAMS | |||||
Product | Indications | Discovery | Preclinical | IND-Enabling | Phase 1 |
CD377 | Single Dose | ̴4 months | |||
Prevention & Treatment | Influenza - 3 months | ||||
CD388 | Influenza - Single Dose | 6̴ months | |||
Prevention & Treatment | |||||
RSV AVC | RSV | ||||
Prevention & Treatment | |||||
HIV AVC | HIV | ||||
Prevention & Treatment | |||||
SARS-CoV-2 AVC | SARS-CoV-2 | ||||
Prevention & Treatment |
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C I DA R A' S P I P E L I N E TA RG E T S M U LT I P L E U N M E T M E D I C A L N E E D S
REZAFUNGINCLOUDBREAK
ANTIFUNGALANTIVIRAL
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A N T I F U N G A L S A R E H I S T O R I C A L L Y B I G D R U G S G L O B A L L Y
P e a k A n n u a l G l o b a l S a l e s ( $ M )
Diflucan 1,000
Vfend 800
Noxafil 720
Cancidas 680
Ambisome 510
Mycamine 370
6 Source: IQVIA for all products in their respective peak sales year, other than Noxafil (2018 Merck Annual Report) and Diflucan (www.pharmaceuticalonline.com Feb 7, 2000)
A N T I F U N G A L D R U G D E V E LO P M E N T H A S DW I N D L E D
Number of New Antifungals | ||
5 | 5 | 1 |
1980
POLYENES
1990 | 2000 | 2010 | 2020 |
- amphotericin lipid forms (3)
AZOLES
• | itraconazole | • | posaconazole |
• | fluconazole | • | voriconazole |
ECHINOCANDINS
• | micafungin | |
• | caspofungin | rezafungin |
• | anidulafungin |
7 Includes antifungals approved for systemic fungal infections.
R E Z A F U N G I N I S D E S I G N E D T O T A R G E T A L A R G E M A R K E T S E G M E N T
CANDIDEMIA | 73% | Percentage of | ||
Invasive Fungal | ||||
AND CANDIDIASIS | Infections | |||
Rezafungin
OTHER
12%
ASPERGILLOSIS | |||
15% | AND MUCORMYCOSIS | ||
- Non-inferiorityto generic voriconazole
- Failed Phase 3 Candida trial
- No prophylaxis label
8 Source: Diagnostic Microbiology and Infectious Disease. The PATH (Prospective Antifungal Therapy) Alliance registry and invasive fungal infections: update 2012
A N T I F U N G A L L AU N C H O U T PA C ES A N T I BA C T E R I A L S
Cresemba's launch in the US has been a success, despite its limitations
U . S . S A L E S P O S T L A U N C H : C R E S E M B A V S . A N T I B A C T E R I A L S
Annualized Sales ($M)
120 | ANTIFUNGAL | ||||||||||||
80 | AVYCAZ | ||||||||||||
DALVANCE | ANTIBACTERIALS | ||||||||||||
40 | ZERBAXA | ||||||||||||
ORBACTIV | |||||||||||||
0 | |||||||||||||
1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 | 10 | 11 | 12 | 13 |
Launch Quarter
9 Cresemba (isavuconazole) is a triazole launched in Q2 2015 by Astellas in the US. In 2017, Pfizer acquired rights to izavuconazole from Basilea for EU, China, 16 Asia Pac countries. Source for sales data: IQVIA
A N T I F U N G A L S C A N H AV E G R EAT E R VA LU E T H A N A N T I BA C T E R I A L S
ANTIFUNGALS
RIGHTS | DEAL ($ MILLION) | DATA AVAILABLE AT SIGNING |
0 | 200 | 400 | 600 | P3 | NDA | Marketed | ||||||||||||
P2 | ||||||||||||||||||
Cidara / Mundipharma: Rezafungin | Ex-US;ex-Japan | |||||||||||||||||
568 | ||||||||||||||||||
Basilea / Pfizer: | Ex-US;ex-Japan | |||||||||||||||||
725 | ||||||||||||||||||
ANTIBACTERIALS | 0 | 200 400 600 | P3 | NDA | Marketed | |||||||||||||
P2 | ||||||||||||||||||
Melinta / Menarini: Delafloxacin | Ex-US;ex-Japan | |||||||||||||||||
120 | ||||||||||||||||||
Paratek / Zai Labs: Omadacycline | China | |||||||||||||||||
Melinta / Menarini: | ||||||||||||||||||
Ex-US;ex-Japan | ||||||||||||||||||
265 | ||||||||||||||||||
Vabomere, Orbactiv & Minocin | ||||||||||||||||||
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R E Z A F U N G I N : A N OV E L LO N G - A C T I N G EC H I N O C A N D I N I N P H A S E 3
N+
O
N+ | O | OH | O | - | |
O | |||||
O | H O | ||||
HO | |||||
N | N | ||||
H3C | H | ||||
O | |||||
N | |||||
H3C | O HN | OH | |||
HO | NH O | N | CH3 | O | |
O | H | ||||
N | |||||
HO | |||||
OH | |||||
O | |||||
OH | |||||
HO |
STRUCTURAL MODIFICATION IS DESIGNED TO YIELD IMPROVED CHEMICAL & BIOLOGICAL PROPERTIES
Design Features | Potential Benefit |
Designed for prolonged PK1 | once weekly dosing in clinical studies1 |
Designed for high exposures2,3 | potential for improved efficacy |
Observed absence of toxic degradation products4 | potential for improved safety |
No DDIs5 and favorable hepatic & renal safety1,4 | compatible with other medications |
11 | 1. | STRIVE Ph2 Trial. Thompson et al. CID Sept 2020 | 4. | Ong et al. AAC November 2016 |
2. | Lakota et al. AAC November 2017 | 5. | Ong et al. BBMT March 2019 |
3. Zhao et al. AAC October 2017
L A RG E M A R K E T, B U T EC H I N O C A N D I N S A R E CO N S T R A I N E D TO DAY
$4.2 Billion
$0.5 Polyenes
$1.1 Echinocandins
$2.6 Azoles
OUTPATIENT
INPATIENT
TREATMENTPROPHYLAXIS
AzolesAzoles/Bactrim
MarketedAzoles/Bactrim Echinocandins12 Source: IQVIA, as of December 31, 2017
A P P R O VA L W I T H N O N - I N F E R I O R I T Y C O U L D E N A B L E L A U N C H O F R E Z A F U N G I N
Ta r g e t s : o u t p a t i e n t s , e a r l y h o s p i t a l d i s c h a r g e a n d p r o p h y l a x i s s e g m e n t s
$4.2 Billion
$0.5 Polyenes
$1.1 Echinocandins
$2.6 Azoles
OUTPATIENT
INPATIENT
TREATMENTPROPHYLAXIS
AzolesAzoles/Bactrim
Rezafungin
Targets
Marketed
Echinocandins
Azoles/Bactrim
13 Source: IQVIA, as of December 31, 2017
R E Z A F U N G I N ' S G LO BA L O P P O RT U N I T Y S PA N S I D A N D H E M ATO LO GY
$ 4 . 2 B I L L I O N
GLOBAL ANTIFUNGAL MARKET
INFECTIOUS DISEASE | HEMATOLOGY |
MOSTLY TREATMENTMOSTLY PROPHYLAXIS
Cancidas | Noxafil |
(caspofungin) | (posaconazole) |
~$700M | ~$700M |
14 Source: Global sales for Cancidas - IQVIA; Noxafil - 2018 Merck Annual Report. Antifungal market data from IQVIA, as of December 31, 2017.
U S R E Z A F U N G I N P EA K S A L ES O P P O RT U N I T Y: ~ $ 7 5 0 M
INFECTIOUS DISEASE | HEMATOLOGY |
MOSTLY TREATMENT | MOSTLY PROPHYLAXIS |
Rezafungin in Treatment | Rezafungin in Prevention |
̴$375M | ̴$375M |
15 | Revenue projections are for peak year 2032 based on estimates and assumptions, including estimated patient populations, market share and pricing assumptions. Actual results could differ materially. Prophylaxis revenue is an estimate of the peak | |
sales potential, of which ~$223M is attributed to spontaneous product use that may occur outside of anticipated initial indication. Any such use will not be supported by Company promotion. | ||
R E Z A F U N G I N OV E R A L L P H A S E 3 D E V E LO P M E N T P L A N
P H A S E 3 T R E A T M E N T T R I A L | P H A S E 3 P R O P H Y L A X I S T R I A L |
POTENTIAL INDICATION | Treatment of candidemia & invasive |
candidiasis | |
PHASE 3 SIZE | 184 patients1 (20% NI margin) |
OVERALL OBJECTIVE | Improve outcomes, allow early hospital discharge |
and enable transition to outpatient use | |
Expect topline data by end of 2021 |
Prophylaxis against Aspergillus, Candida &
Pneumocystis in allogeneic blood and
marrow transplant patients
462 patients (12.5% NI margin)
Transform post-blood & marrow
transplant standard of care
16 | 1. | Phase 3 Primary Evaluable Population size. |
S T R I V E P H A S E 2 D A T A I N C A N D I D E M I A & I N V A S I V E C A N D I D I A S I S
P o s i t i v e d a t a s u p p o r t s R e S T O R E P h a s e 3 c l i n i c a l t r i a l
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S T R I V E P H A S E 2 P RO G R A M : C A N D I D E M I A & I N VA S I V E C A N D I D I A S I S
Tr i a l n o t p o w e r e d f o r i n f e r e n t i a l s t a t i s t i c a l a n a l y s i s
Overall Response | ||||||||||||||||||||||
(Mycological & clinical | ||||||||||||||||||||||
N= 183 | response) | Overall Response | All cause mortality | End of Follow Up Period | ||||||||||||||||||
Dose | Optional dose | |||||||||||||||||||||
Rezafungin | ||||||||||||||||||||||
Week | 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 | |||||||||||||
N= 122 | Day | |||||||||||||||||||||
5 | 14 | 30 |
Dose | Optional dose | ||||||||||
Caspofungin | Week 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 |
N= 61
A N A LY S I S | • The Intent-to-treat (ITT) population: all randomized subjects | |
• | The Safety population: all subjects who received any amount of study drug | |
P O P U L A T I O N S : | • | The Microbiological Intent-to-treat population (mITT): all subjects in safety population who had |
documented Candida infection |
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R E Z A F U N G I N S H O W E D S U P E R I O R I T Y O N T I M E T O N E G A T I V E B L O O D C U L T U R E O V E R C A S P O F U N G I N
T I M E T O N E G AT I V E B L O O D C U LT U R E ( m I T T P O P U L AT I O N )
Probability of negative blood culture
100% | Rezafungin IV 400/200mg |
80% | Caspofungin IV 70/50mg |
60% | Daily doses |
40% | Log-rank test (post hoc): |
RZF vs CSP | |
20% | p=0.02 |
Weekly | |
Dose |
0% | 0 | 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 |
Days since first dose |
19 Data on file from STRIVE A and B combined
3 0 - DAY A L L C AU S E M O RTA L I T Y - P O S T H O C A N A LY S I S
FA V O R S R E Z A F U N G I N
-8.8%
-24.7% Rezafungin 400/200; n = 46
95% confidence interval
S U P E R I O R I T Y
F A V O R S C A S P O F U N G I N
ReSTORE Phase 3 trial endpoint requires upper limit of
+0.41%confidence interval be below
20% threshold for FDA
20%
Non-inferiority margin
N O N - I N F E R I O R I T Y
-20% | -10% | 0 | 10% |
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 (relevant Rezafungin group minus caspofungin | ||
20 | group) was calculated using the unadjusted method of Miettinen and Nurminen. | |
Post-hoc analyses do not establish effectiveness and should not be assumed to establish the same outcome in Phase 3. | ||
DAY 1 4 C L I N I C A L R ES P O N S E - P O S T H O C A N A LY S I S
F A V O R S R E Z A F U N G I N FA V O R S C A S P O F U N G I N
ReSTORE Phase 3 trial endpoint | |||||||||||||||||||
9.9% | requires upper limit of | ||||||||||||||||||
confidence interval be below | |||||||||||||||||||
26.6% | Rezafungin 400/200; n = 46 | -6.9% | 20% threshold for EMA | ||||||||||||||||
95% confidence interval | |||||||||||||||||||
-20% | |||||||||||||||||||
Non-inferiority | |||||||||||||||||||
margin | |||||||||||||||||||
S U P E R I O R I T Y | N O N - I N F E R I O R | I T Y | |||||||||||||||||
20% | 10% | 0 | -10% |
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 (relevant Rezafungin group minus caspofungin group) was calculated using the unadjusted method of Miettinen and Nurminen.
21 Post-hoc analyses do not establish effectiveness and should not be assumed to establish the same outcome in Phase 3.
S T R I V E P H A S E 2 : S H O R T E R A V E R A G E I C U S T A Y F O R P A T I E N T S O N R E Z A F U N G I N 1
% Neg. Blood Culture2 | % Overall Cure | Average Days in ICU | ||||||||||||||||||||||||||||||
74.4 | 54.2 | 1 DAY | 73.9 | 55.7 | 5 DAYS | REZAFUNGIN | ||||||||||||||||||||||||||
n/N | n/N | |||||||||||||||||||||||||||||||
32/43 | 34/46 | 13 DAYS | ||||||||||||||||||||||||||||||
32/59 | 34/61 | CASPOFUNGIN | ||||||||||||||||||||||||||||||
Rz Cf2 | Rz | Cf | ||||||||||||||||||||||||||||||
16 DAYS | ||||||||||||||||||||||||||||||||
Days 0 | 5 | 10 | 15 | |||||||||||||||||||||||||||||
85.7 | 2 DAYS | 3 | ||||||||||||||||||||||||||||||
65.5 | ||||||||||||||||||||||||||||||||
2 | ||||||||||||||||||||||||||||||||
n/N | ||||||||||||||||||||||||||||||||
36/42 | ||||||||||||||||||||||||||||||||
Rz | Cf | 38/58 | ||||||||||||||||||||||||||||||
% Neg. Blood Culture | ICU DAYS | |||||||||||||||||||||||||||||||
SAVED |
22 1. Data on File.
2. Proportion of patients with negative blood culture, mITT population (%; n/N).
R e S T O R E P H A S E 3 T R I A L D ES I G N M I R RO RS S T R I V E P H A S E 2 T R I A L
Global | Global Response | All Cause Mortality | End of Follow Up Period | |||||||||||||||
Response | (1° ENDPOINT - EMA) | (1° ENDPOINT - FDA) | ||||||||||||||||
Rezafungin | Dose | Optional dose | ||||||||||||||||
Week | 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 | |||||||||
N= 92 | ||||||||||||||||||
400/200mg | Day | |||||||||||||||||
5 | 14 | 30 | ||||||||||||||||
Caspofungin | Dose | Optional dose | |||||||
Week 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 | |
N= 92 | |||||||||
70/50mg |
23 | Global Response is defined as Clinical Response (as assessed by the Primary Investigator), Mycological Response and Radiological Response (for qualifying invasive candidiasis patients only). |
T H E D I S A R M A C T: A F U N DA M E N TA L I M P ROV E M E N T I N R E I M B U RS E M E N T
The DISARM Act
Proposed, but not yet enacted, legislation to encourage the development of antimicrobial drugs
Currently proposed terms of the DISARM Act
- Removes QIDP-designated products - like rezafungin - from DRG1 reimbursement and, instead, allows for separate reimbursement
- Generic echinocandins are not QIDP-designated and would not be eligible for separate reimbursement under DISARM Act
DISARM Act status
- Bipartisan introduction in 2019 by Senators Isakson (R) and Casey (D) and Representatives Davis (D) and Marchant (R)
- Expected to be reintroduced in 117th Congress in early 2021
- Co-sponsorsin the Senate and House affirm that this is a high priority
DISARM Act terms from H.R.4100 - 116th Congress (2019-2020).https://www.congress.gov/bill/116th-congress/house-bill/4100/text
24 1. Under the diagnosis-related group (DRG) payment system, health care providers receive a fixed payment based on the DRG assigned to the patient at discharge regardless of the cost of actually treating the patient.
R E Z A F U N G I N O V E R A L L P H A S E 3 D E V E L O P M E N T P L A N
P H A S E 3 T R E A T M E N T T R I A L | P H A S E 3 P R O P H Y L A X I S T R I A L |
POTENTIAL INDICATION | Treatment of candidemia & invasive |
candidiasis | |
PHASE 3 SIZE | 184 patients1 (20% NI margin) |
Improve outcomes, allow early hospital discharge | |
OVERALL OBJECTIVE | and enable transition to outpatient use |
Expect topline data by end of 2021 |
Prophylaxis against Aspergillus, Candida &
Pneumocystis in allogeneic blood and
marrow transplant patients
462 patients (12.5% NI margin)
Transform post-blood & marrow
transplant standard of care
25 | 1. | Phase 3 Primary Evaluable Population size. |
C U R R E N T A N T I F U N G A L P R O P H Y L A X I S S T A N D A R D O F C A R E I S I N A D E Q U A T E
H I G H D I S C O N T I N U AT I O N R AT E
BMT patients discontinue prophylaxis due to adverse
events and low tolerability
Posaconazole1 | 34% |
Fluconazole 1 | 38% |
Bactrim2 | 35% |
H I G H M O R T A L I T Y
90-day mortality in patients with invasive fungal infection3
Blood and marrow transplant | 63% |
Hem Malignancy | 52% |
- Ullmann AJ et al. Posaconazole or fluconazole for prophylaxis in severe graft-versus-host disease. N Engl J Med. 2007 Jan 25;356(4):335-47.
- Vasconcelles MJ et al. Aerosolzed Pentamidine and Pneumocystis Prophylaxis after Bone Marrow Transplantation is Inferior to Other Regimens and is Associated with Decreased Survival and Increased
26 Risk of Other Infections. A. Society for BMT. 2000.
3. The PATH (Prospective Antifungal Therapy) Alliance registry and invasive fungal infections: update 2012.
R E Z A F U N G I N : P O T E N T I A L S I M P L I F I E D S I N G L E D R U G P A R A D I G M
A n t i f u n g a l p r o p h y l a x i s i n a l l o g e n e i c b l o o d a n d m a r r o w t r a n s p l a n t s e t t i n g
Current
Antifungal
Prophylaxis
Regimens
Fluconazole
Fluconazole | Posaconazole or voriconazole |
Posaconazole or voriconazole
Bactrim, dapsone or atovaquone
Rezafungin 400/200 once weekly
-10 | 0 | 10 | 20 | 30 | 40 | 50 | 60 | 70 | 80 | 90 | ||||||||||
Transplant | Day |
SOC for Candida and Aspergillus
SOC for
Pneumocystis
Candida, Aspergillus and Pneumocystis
27
R e S P E C T P H A S E 3 P R O P H Y L A X I S T R I A L D E S I G N
REZAFUNGIN ARM (N=~300) | Dose | 1° Endpoint: Fungal Free Survival at Day 90 | Follow up | |||||||||||||||||||||||||
Week 1 | 2 | 3 | 4 | 5 | 12 | 13 | 17 | |||||||||||||||||||||
Rezafungin | ||||||||||||||||||||||||||||
Azole placebo | ||||||||||||||||||||||||||||
Bactrim placebo | ||||||||||||||||||||||||||||
Day | 1 | 90 | 120 |
COMPARATOR ARM (N=~150) | ||||||||||||||||||||||
Week 1 | 2 | 3 | 4 | 5 | 12 | 13 | 17 | |||||||||||||||
Rezafungin Placebo | ||||||||||||||||||||||
Azole1 | ||||||||||||||||||||||
Bactrim | ||||||||||||||||||||||
Day | 1 | 90 | 120 |
28 | 1. Fluconazole to start in all patients. Posaconazole optional in patients who develop GVHD per label. |
P A T I E N T E X P E R I E N C E - E X P A N D E D A C C E S S
P a t i e n t A - r e s i s t a n t C . g l a b r a t a a n d t r e a t m e n t - i n d u c e d r e n a l f a i l u r e
B A C K G R O U N D
Outpatient adult male with multiple aortic surgeries and chronic Candida glabrata infection requiring life-long suppression
Initially treated with once daily
intravenous infusions of caspofungin
C L I N I C A L
O B S E R V A T I O N S
S I T U A T I O N | E X P A N D E D A C C E S S |
The C. glabrata strain acquired an | On May 7, 2020 Patient A received |
fks mutation that confers resistance to | the first 1x/weekly IV infusion of 400 |
the caspofungin treatment | mg of rezafungin |
Patient A was switched to | Patient A continues to receive |
amphotericin B and flucytosine, | 1x/weekly IV infusions of 200 mg |
but then developed renal failure | rezafungin as an outpatient |
and was running out of | |
treatment options |
- Chronic resistant C. glabrata infection remains suppressed to date
- Renal function recovered with discontinuation of amphotericin B; avoided dialysis
- To date, no adverse events related to this treatment
29 | Patient A's response may not be typical of all patients. Individual results may vary. Information above is as of December 2020. |
PAT I E N T E X P E R I E N C E - E X PA N D E D A C C ES S
P a t i e n t B - u n r e s o l v e d r e s i s t a n t i n f e c t i o n r e q u i r i n g e x t e n d e d i n p a t i e n t t r e a t m e n t
B A C K G R O U N D | S I T U AT I O N | E X PA N D E D A C C E S S |
Inpatient adult female with recent | Patient B was not considered a | Patient B administered 400 mg of |
liver transplant complicated by | good candidate for additional | rezafungin the first week followed by |
intra-abdominal abscesses with | surgery | 1x/weekly doses of 200 mg rezafungin |
chronic Candida krusei | Physician believed infection was | Patient B improved and was discharged, |
Infection did not clear with | ||
unresponsive to treatment due to | while continuing to receive 1x/weekly | |
repeated surgical washouts and | inadequate tissue penetration of | 200 mg rezafungin on an |
prior antifungal therapy with | prior echinocandin therapy | outpatient basis |
micafungin | 12 total treatments | |
• Patient B responded to 1x/weekly rezafungin IV treatments, was discharged and able to continue 1x/weekly treatment as | |
C L I N I C A L | an outpatient |
O B S E R V A T I O N S | • Imaging at week 12 confirmed that the critical deep tissue infection was resolved and Patient B was able to cease treatment |
• To date, no adverse events related to this treatment |
30 Patient B's response may not be typical of all patients. Individual results may vary.
O U R P A R T N E R F O R R E Z A F U N G I N E X - U S / J A P A N
1 2 0
M A R K E T S
Active in more than 30
in Europe
O v e r € 2
B I L L I O N
European sales
exceeding €1 billion
Over €2B ex-US
Y E6A0R S
Over 60 years of
asset-led innovation
5 0 %
Over 50% revenue derived from new products launched in the last five years in Europe
Product: Rezafungin | Rights: ex-US/Japan
$ 5 6 8 M
P h a s e 2 d a t a
- $30M upfront
- $9M equity investment
- Up to $42M in development support
- Up to $487M clin/reg/comm milestones
- Double-digitroyalties in the teens
31
C I D A R A ' S P I P E L I N E T A R G E T S M U L T I P L E U N M E T M E D I C A L N E E D S
REZAFUNGINCLOUDBREAK
ANTIFUNGALANTIVIRAL
32
C L O U D B R E A K AV C P L AT FO R M : A P OT E N T I A L N E W C L A S S O F A N T I V I R A L
ANTIVIRAL CONJUGATE (AVC)
A POTENTIAL NEW CLASS OF LONG-ACTING DRUGS
- Not a vaccine, monoclonal antibody or traditional therapeutic
- AVCs designed to be stable conjugates of a potent antiviral with an Fc antibody fragment
- Designed for rapid onset, potent activity coupled with ̴4 - 6 months of protection
33
C L O U D B R E A K A V C s : P O T E N T I A L T O T A R G E T M U L T I P L E V I R U S E S
T W O P R O D U C T O P P O R T U N I T I E S F O R I N F L U E N Z A
C D 3 7 7 | C D 3 8 8 |
(Development Candidate) | (Development Candidate) |
• | Single dose / ̴4 months | • | Single dose / 6̴ months | |
• | Full immune engagement | • | Attenuated immune engagement | |
PLATFORM EXPANSION | RSV | HIV | SARS-CoV-2 | |
PROGRAMS UNDERWAY | ||||
34
C L O U D B R E A K I N F L U E N Z A A V C s : A F U N D A M E N T A L L Y N E W A P P R O A C H
Potential Design Benefits
Potent, universal activity against
influenza A and B
̴4 - 6 months of protection Engages immune system
Neuraminidase | Fc domain of |
inhibitor | human IgG1 |
35
A V C s M E C H A N I S M O F A C T I O N A G A I N S T I N F L U E N Z A
FLU VIRUS REPLICATION | CD377 / CD388 ACTIVITY AGAINST FLU |
NEURAMINIDASE | |||||||||
RELEASE OF | VIRION | NO VIRION | |||||||
VIRION | RELEASED | HALTED | |||||||
NEURAMINIDASE | NEW VIRIONS | CONTINUED | |||||||
AVC INHIBITS | VIRAL | ||||||||
CLEAVES RECEPTOR | VIRAL | ||||||||
REPLICATION | |||||||||
REPLICATION | NEURAMINIDASE | ||||||||
ACTIVITY | |||||||||
BUDDING | RECEPTOR | RECEPTOR | |||||||
CONTAINING SIALIC | |||||||||
VIRUS | CONTAINING SIALIC | ||||||||
ACID | |||||||||
ACID | |||||||||
H O S T | C E L L | H O S T | C E L L | ||||||
36
F L U V A C C I N E S H A V E W E L L - K N O W N L I M I T A T I O N S
Historically flu vaccine effectiveness has been limited
Flu vaccines don't cover all strains | Flu vaccines don't cover most people |
- Vaccines cover 3 to 4 of the dozens of flu strains
- Strains selected 6 months before the flu season
- Flu viruses are constantly changing
- Certain strains (H3N2) grow poorly in eggs
2018-2019 flu season | |||
Age | Vaccine | ||
Effectiveness | |||
Vaccine | 6 mos-8 y | 48% | |
Effectiveness (VE) | |||
9-17 y | 7% | ||
in 2018-2019 | |||
29% | 18-49 y | 25% | |
50-64 y | 14% | ||
≥65 y | 12% | ||
37 Centers for Disease Control and Prevention: Selecting Viruses for the Seasonal Influenza Vaccine and Seasonal influenza vaccine effectiveness.
~ 1 0 0 M P E O P L E I N U S A T H I G H R I S K F O R F L U C O M P L I C A T I O N S 1 , 2
Many higher risk patients have lower Vaccine Effectiveness
Immuno- | Pregnant | Elderly | Children | Respiratory co- |
compromised3 | women | over 65 | under 5 | morbidities4 |
HIGH RISK |
- High risk groups:https://www.cdc.gov/flu/highrisk/index.htm
- Census Bureau 2017, CDC.gov and cancer.gov
38 3. The Prevalence of Immunocompromised Adults: United States, 2013. Harpaz. JAMA December 20, 2016 Volume 316, Number 23
4. Includes asthma and COPD
V I S I O N F O R A V C s : S I N G L E - D O S E , U N I V E R S A L , S E A S O N A L P R O T E C T I O N
AVC VISION
AVCs designed to cover | AVCs aim to protect |
ALL STRAINS ALL PEOPLE
39
P R E - C L I N I C A L V A L I D A T I O N O F A V C F L U P R O G R A M
BROAD SPECTRUM COVERAGE | PROTECTION FOR HIGH-RISK |
POPULATIONS | |
POTENCY VS. | PROLONGED SINGLE-DOSE |
TAMIFLU-RESISTANT VIRUSES | PROTECTION |
SUPERIOR RESISTANCE PROFILE | BROAD SAFETY MARGIN |
40
C D 3 7 7 : E F F E C T I V E I N V I V O A G A I N S T A L L F L U A & B S T R A I N S T E S T E D
L E T H A L I N F L U E N Z A I N F E C T I O N M O D E L S
CD377 - 0.3 mg/kg | ||
A/California/07/2009 | Vehicle | A/Hong Kong/1/68 H3N2 |
H1N1 Pandemic | ||
CD377 - 0.03 mg/kg
B/Florida/4/2006 | B/Malaysia (Victoria) |
(Yamagata) | |
Vehicle | |
CD377 - 0.3 mg/kg Vehicle
CD377 - 0.1 mg/kg
Vehicle
41 5 BALB/c mice per cohort. CD377 dosed once 2 hours post infection (SC). Döhrmann and Levin et al, IDWeek 2020
C D 3 7 7 : P O T E N T A G A I N S T T A M I F L U - R E S I S T A N T V I R U S E S
L E T H A L I N F L U E N Z A I N F E C T I O N M O D E L
Dose
Doses
Survival | Tamiflu 20 mg/kg |
% | BID x 5 |
CD377 - 0.3 mg/kg dosed once
Vehicle
Days Post Infection
42 5 mice per cohort. CD377 dosed 2 hours post infection (SC), Tamiflu dose 2 hours post infection BID x 5 days (PO). Levin et al, IDWeek 2020
C D 3 7 7 : L O W E R R E S I S T A N C E P O T E N T I A L
Viral titer, (PFU/mL)
S E R I A L P A S S A G E S T U D Y
107
Vehicle | Tamiflu (100 nM) | |||||
Baloxavir (4 nM) | ||||||
108 | ||||||
105 | ||||||
CD377 (4 nM) | ||||||
104 | ||||||
103 | 2 | 4 | 6 | 8 | 10 | |
Passage Number
43 Almaguer et al, ECCMID 2020
C D 3 7 7 : P O T E N T I A L F O R L O N G - T E R M , S I N G L E D O S E P R O T E C T I O N
Single 1 mg/kg SC dose was fully protective vs:
- H1N1 (right)
- A/Hong Kong/1/68 H3N2
- Influenza B (Malaysia)
- Influenza B (Florida)
L O N G T E R M P R E V E N T I O N M O D E L
CD377: 1 mg/kg
% Survival
Vehicle
DoseInfect
Day Post Infection
44 5 mice per cohort. CD377 dosed 28 days prior to infection (SC). Levin et al, IDWeek 2020
C D 3 7 7 : E F F E C T I V E I N I M M U N E D E F I C I E N T H O S T S
LETHAL INFLUENZA INFECTION MODELS
BALB/c immune competent | SCID immune deficient |
CD377 - 0.1mg/kg | CD377 - 0.1mg/kg |
Vehicle | Vehicle |
45 5 BALB/c or SCID mice per cohort. CD377 dosed once 2 hours post A/PR/8/1934 H1N1 infection (SC). Levin et al, IDWeek 2020
C D 3 7 7 : B R O A D S A F E T Y M A R G I N I N P R I M A T E S
R e s u l t s o f 1 4 - d a y t o x i c i t y t e s t i n g
THERAPEUTIC MARGIN
500000
Area under the curve (AUC) for maximum dose tested (hr*mg/mL)
25000095x
Predicted AUC required for efficacy in humans (hr*mg/mL)
0
Primate
NO ADVERSE FINDINGS FOR:
Clinical observations
Hematology
Clinical Chemistry
Coagulation
Urinalysis
Immunophenotyping
Cytokines
Histopathology
46 Cidara data on file
F I N A N C I A L O V E R V I E W
Important Information | September 30, 20201 |
Cash and restricted cash | $53.7M |
PacWest Term Loan - Remaining Principal | $8.1M |
Expected Mundipharma Milestone2 | $11.1M |
Common stock issued | 43,936,026 |
Common equivalent shares issued3 | 54,378,806 |
Summary Consolidated
Cash Flow Information
Operating Cash Burn5
Mundipharma Cost Share Payments
Net Operating Cash Burn
ATM Equity Proceeds
Term Loan Principal Payments
Net Cash Use
Rolling two-quarter period ended September 30, 20204
$(36.8)M
$8.6M
$(28.2)M
$9.5M
$(1.9)M
$(20.6)M
- Information listed here is as of September 30, 2020 (as disclosed in our 10-Q filing) with the exception of the Expected Mundipharma Milestone (see footnote 2).
- As of November 27, 2020, Cidara met the necessary conditions under the Collaboration and License Agreement with Mundipharma dated September 3, 2019 to require Mundipharma to pay the previously disclosed $11.1 million milestone payment on or before January 10, 2021.
- Includes (i) 43,936,026 shares of common stock and (ii) 10,442,780 shares of common stock issuable upon the conversion of Series X Convertible Preferred stock, both as of September 30, 2020. Each share of
Series X Convertible Preferred is convertible into 10 shares of common stock.
47 4. Amounts reflect a rolling two quarter period ending on the date noted. Amounts shown are historical and may not be indicative of future results.
5. Represents net cash used in operations of $28.2M adjusted to remove $8.6M received pursuant to the Mundipharma cost share.
C I DA R A I S M U C H M O R E T H A N A T Y P I C A L I D CO M PA N Y
S T R AT E G I C F O C U S
R E Z A F U N G I N
T R E AT M E N T
R E Z A F U N G I N P R O P H Y L A X I S
Transformative approaches to infectious disease
Potential to enable fast clearance of infection, early discharge vs SOC
Potential to transform the care of BMT patients
C L O U D B R E A K A V C
Radically different approach to prevent and treat viral disease
O U R T E A M
Experienced creators of shareholder value
48
CORPORATE PRESENTATION
49 | L E A D I N G T H E S C I E N C E O F P R O T E C T I O N |
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Cidara Therapeutics Inc. published this content on 05 January 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 05 January 2021 19:49:02 UTC