Citius Pharmaceuticals, Inc. · NASDAQ:CTXR
Citius Pharmaceuticals — Investor Presentation (February 2021)
53 slides from Citius Pharmaceuticals's investor presentation, Feb 16, 2021
Slide 1: Citius Pharmaceuticals Corporate Presentation Winter 2020-21
Citius Pharmaceuticals, Inc. Corporate Presentation Winter 2020 - 21 NASDAQ: CTXR
Slide 2: Disclaimer
2 NASDAQ: CTXR This presentation has been prepared by Citius Pharmaceuticals, Inc. (the “Company”) for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the Company or any di
Slide 3: Company Overview
3 NASDAQ: CTXR Olympic Motto: “ Citius , Altius, Fortius ” ( Faster , Higher, Stronger) Company Overview Four Active Programs with a Late Stage Lead Asset Mino - Lok in late Phase 3; favorable review received for Futility Analysis by Data Monitoring Committee (12/2019); favorable review for safety a
Slide 4: Management
4 NASDAQ: CTXR Management Leonard Mazur, Director and Executive Chairman • Founder/co - founder of: Genesis, Triax , Akrimax , and others Myron Holubiak, Director, CEO and President • Former President of Roche Laboratories, Inc. Jaime Bartushak, EVP, Chief Financial Officer • Over 20 years experienc
Slide 5: Key Scientific/Medical Advisors
5 NASDAQ: CTXR Key Scientific/Medical Advisors (ID and CA) Isaam Raad , M.D • Chair of MD Anderson Cancer Center’s Dept. of Infectious Diseases • Author of the underlying patents for Mino - Lok® • Dr. Raad’s innovations have been endorsed at the highest level (Category 1A) by the Center for Disease
Slide 6: Key Cellular Therapy Advisors
6 NASDAQ: CTXR Key Cellular Therapy Advisors Michael A. Matthay, MD • Senior Associate at the Cardiovascular Research Institute, and Associate Director of the Critical Care Medicine & Professor o f M edicine and Anesthesia at the University of California at San Francisco (UCSF) • Research focused on
Slide 7: Unique Pipeline in Progressive Stages
7 NASDAQ: CTXR Program Estimated Market (Worldwide) Preclinical Phase I Phase II Phase III Mino - Lok® Treat CVC Infections > $1.5B CITI - 002 (Halo - Lido) Rx Therapy for Hemorrhoids > $2B CITI - 101 (Mino - Wrap) Prevent Infections Associated with Breast Implants > $400M CITI - 401 ( i - MSC) Trea
Slide 8: Lead Product Mino-Lok
LEAD PRODUCT Minocycline/EDTA/Ethanol Antibiotic Lock Therapy for Salvaging Catheters That Cause Bloodstream Infections Mino - Lok®
Slide 9: Central Venous Catheters
9 Central Venous Catheters Central Venous Catheters (CVCs), Peripherally Inserted Central Catheter (PICCs), and Hemodialysis Central Venous Catheter PICC Hemodialysis CTXR
Slide 10: Pathogenesis of CRBSI
1 0 Pathogenesis of CRBSI Safdar N, Maki DG. The pathogenesis of catheter - related bloodstream infection with noncuffed short - term central venous catheters. Int Care Med . 2004;30:62 - 67. CTXR Endogenous: Skin flora Extrinsic: HCW hands; Contaminated field Endogenous: Skin flora Extrinsic: HCW h
Slide 11: Biofilm Formation Protects Colonies
11 Biofilm Formation Protects Colonies • Pathogens attach to the surface of the lumen in a central venous catheter and form colonies. • Colonies grow and exude a fibrous glycocalyx that protects the organisms from antibiotics, even when shown to be sensitive in vitro CTXR Micrograph of Biofilm in Cen
Slide 12: Infection Rates and Poor SOC Lead to Death & Morbidity
12 NASDAQ: CTXR BUT Infection Rates + Poor SOC Leads to Death & Morbidity Infections are Common & Dangerous Of the 7,000,000 CVCs used annually in US, up to 472,000 become infected leading to serious, life threatening infections called CRBSI/CLABSI. 1 These infections are associated with 12 - 25% mo
Slide 13: R&R procedures are invasive and discomforting to patient
13 NASDAQ: CTXR R&R procedures are invasive and discomforting to patient R&R Procedures are costly and usually require additional hospital stay. Complications include infection, thrombosis, occlusion, and mechanical complications. • Infectious complications are reported to occur in 5% to 26% of pati
Slide 14: Locking a Central Venous Line with Mino-Lok
14 NASDAQ: CTXR Locking a Catheter is a Standard Operating Procedure 1. Using Mino - Lok does not require any novel methodologies. 2. Any RN or LPN or Technician can perform the procedure. 3. There is no change in normal workflow and does not require exceptional training. 4. The patient does not exp
Slide 15: Phase 2b Trial Results
15 NASDAQ: CTXR Phase 2b Trial Results Parameter Mino - Lok Arm N ( % ) Control Arm N ( % ) Patients 30 (100%) 60 (100%) Cancer Type - Hematologic 20 (67) 48 (80) - Solid tumor 10 (33) 12 (20) ICU Admission 4 (13) 4 (7) Mech. Ventilator 3 (10) 0 (0) Bacteremia - Gram+ 17 (57)* 32 (53) - Gram - 14 (4
Slide 16: Mino-Lok Phase 3 Pivotal Trial Design
16 NASDAQ: CTXR Active Arm (n=72) Mino - Lok® Solution Control Arm (n=72) Antibiotic Lock Patients with CRBSI/CLABSI (n~ 144) R Multi - center, randomized, open label, blinded assessor, active control superiority study (80% powered) Futility Performed at 35 - 40 Events and 60 - 70 Events for Superio
Slide 17: Time-To-Catheter Failure Trial Design
Mino - Lok® Time - To - Catheter Failure Trial Design 0 10 20 30 40 50 60 70 80 90 100 0 10 20 30 40 50 60 Catheter Survival (%) Time (days) Kaplan - Meier Model ALT MLT 21 vs. 38 Day Difference in Median Number of Catheter Failures 17
Slide 18: Mino-Lok Development Plan
18 NASDAQ: CTXR 2015 2016 2017 2018 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Pivotal Phase 3 ALT Study 2019 Q1 Q2 Q3 Q4 2020 Q1 Q2 Q3 Q4 2021 Q1 Q2 Q3 Q4 First Patient In Interim Data Safety/Efficacy FDA Interim Mtg. FDA Review Mtg. Commercial Preparation Phase 2 FDA EOP2 Meeting Registration
Slide 19: Market Opportunity, Reimbursement and Pricing
19 NASDAQ: CTXR Worldwide Ex - US United States Regulatory Updates Reimbursement Pricing With modest penetration at conservative pricing, we believe that >$500M peak year U.S. sales is achievable. DISARM Act is pending in the Senate, which would create a DRG carveout for QIDP products. This would al
Slide 20: Robust Intellectual Property Portfolio
20 NASDAQ: CTXR Mino - Lok TM is supported by a robust intellectual property portfolio Composition of Matter patent that provides protection until June 7, 2024 . Formulation Patent has been issued and will add protection through 2036. Creators Description of Patent All U.S. and Foreign Patent Applic
Slide 21: Regulatory Protection
21 NASDAQ: CTXR Regulatory Protection Qualified Infectious Disease Product (US) • Eligibility for Fast Track Status, enables frequent communication and collaboration with FDA; • Priority Review, reduces the NDA review time from 12 to 6 months; and, • Market Exclusivity, grants an additional 5 years
Slide 22: Competitive Landscape
22 NASDAQ: CTXR Company/Source Product/Components Status Features/Weaknesses CorMedix DefenCath TM ( Neutrolin ®) taurolidine, citrate, heparin NDA accepted by the FDA in August 2020 PDUFA date 2/28/2021 Available in Europe (CE Mark) Prevention only Anti - infective only being used in prophylaxis No
Slide 23: Mino-Lok Summary
23 NASDAQ: CTXR Mino - Lok® (minocycline/disodium EDTA/ethyl alcohol) Treats catheter - related blood stream infections (CRBSIs). Penetrates biofilm, eradicates bacteria and salvages infected, indwelling vascular catheters while providing anti - clotting properties. Salvages central venous access in
Slide 24: NoveCite induced Mesenchymal Stem Cells (i-MSCs)
NoveCite induced Mesenchymal Stem Cells (“ i - MSCs”)
Slide 25: Citius, Novellus and NoveCite
25 Citius Pharmaceuticals, Inc. • late - stage specialty pharmaceutical company • critical care products (anti - infectives and cancer care) • Mino - Lok, Mino - Wrap, and Halo - Lido. NASDAQ (CTXR) Novellus Therapeutics • pre - clinical stage biotechnology company • patented non - immunogenic mRNA
Slide 26: NoveCite i-MSC Unique Advantages
NoveCite i - MSC Compelling Unique Advantages 26 • Novellus mRNA cell reprogramming efficiently creates i - PSC cells ( i - PSCs are an ideal building - block for cell - based therapies) • Clonal i - PSC master cell bank is created – no batch - to - batch variation and footprint - free (no viral vec
Slide 27: Novellus mRNA Cell Reprogramming
Novellus mRNA Cell Reprogramming Proprietary induced Pluripotent Stem Cell Platform – Single donor, NO repeat harvesting adult - derived cells; NO repeat donor testing – Clonal i - PSCs differentiated into clonal i - MSCs for genetically identical manufacturing batches – i - MSCs exhibit enormous ex
Slide 28: Proprietary Non-Immunogenic mRNA Technology
Proprietary mRNA Technology – Non - Immunogenic Ribolog Œ evades the innate immune system and is recognized by the ribosome as mRNA, resulting in no immunogenicity, high protein expression and ability for repeat dose 28 Novellus’s RiboLog Ρ Native mRNA is recognized by the innate immune system so is
Slide 29: i-MSC Production Process
i - PSCs Day 0 Induction medium Day 4 MSC medium Day 6 Passage Day 9 Passage Day 13 Passage Day 19 Passage Day 26 Passage Day 29 Passage i - MSCs i - MSC Advantages: Production in 30 Days • 6 days of 5 non - immunogenic mRNAs encoding reprogramming factors • Mutation - free, extensively characterize
Slide 30: i-MSCs Advantages: Greater Expansion Potential
i - MSCs Advantages: Greater Expansion Potential 30 Growth Curve Telomere Length i - MSCs exhibit greater expansion potential – mRNA process restores telomeres to yield >70 population doublings “BM - MSCs” = bone marrow - derived MSCs *Data on file (Novellus) BM - MSCs
Slide 31: NoveCite i-MSC Summary
NoveCite i - MSC Summary 31 • Novellus mRNA cell reprogramming efficiently creates i - PSC cells ( i - PSCs are an ideal building - block for cell - based therapies) • Clonal i - PSC master cell bank is created – no batch - to - batch variation and footprint - free (no viral vectors) • Rapid product
Slide 32: NoveCite i-MSCs: ARDS in COVID
NoveCite i - MSCs: ARDS in COVID - 19 SARS - CoV - 2 Severe acute respiratory syndrome coronavirus 2
Slide 33: Acute Respiratory Distress Syndrome (ARDS)
ARDS Lung Normal ARDS a type of respiratory failure characterized by rapid onset of widespread inflammation and fluid accumulation in the lungs. ARDS impairs the lungs’ ability to exchange oxygen for carbon dioxide Symptoms includes shortness of breath, rapid breathing, and bluish skin coloration; f
Slide 34: ARDS and sepsis immune dysfunction; role of MSCs
ARDS (and sepsis) is a dysfunction of the immune system with the concomitant presence of pro - inflammatory and anti - inflammatory states • Immunosuppressive state is evidenced by : • increase in lymphocyte apoptosis , • alteration of immune cell functions , • less antigen presentation by dendritic
Slide 35: i-MSC Advantages: Levels of Immunomodulator Secretion
i - MSC Advantages: Levels of Immunomodulator Secretion 35 i - MSCs secrete higher levels of many immunomodulatory proteins NoveCite Example: MMP - 1 = 6,500 pg/ml MMP - 1 = 330 pg/ml Example: VEGF - A = 10,800 pg/ml VEGF - A = 3,000 pg/ml Example: MMP - 1 = 6,500 pg/ml MMP - 1 = 330 pg/ml *MMP - 1:
Slide 36: NoveCite i-MSC Mouse Study
NoveCite i - MSC Mouse Study
Slide 37: i-MSCs vs. MSCs: Therapeutic Effect in MS Mouse Model
i - MSCs vs. MSCs: Therapeutic Effect in MS Mouse Model 37 Novellus i - MSCs improve clinical score in an EAE mouse model – Tail - vein injection of 20 million i - MSCs/kg improved score (p<0.001) – Donor - derived MSCs had little/no effect at 20 million and 40 million cells/kg 25 3=Partial hind lim
Slide 38: ARDS Sheep Study
ARDS Sheep Study
Slide 39: Interim Results in Sheep Model of ARDS
P/F ratio Pulmonary Lymph Flow Pulmonary Lymph Protein Loss Norepinephrine Requirement Interim Results: Very Strong Beneficial Effect of i - MSCs in Sheep Model of ARDS Improved Oxygenation Reduced Lung Vascular Injury Less systemic shock Overview of findings: In this ovine model of severe ARDS the
Slide 40: Our Solution – NoveCite i-MSCs
Our Solution – NoveCite i - MSCs Description: • Allogeneic (“off - the - shelf”) i - PSC - derived induced Mesenchymal Stem Cells • High potency i - MSCs – in vitro data showing exponentially higher secretion of immunomodulatory proteins vs. BM - derived MSCs • Footprint - free i - PSCs created usin
Slide 41: Clinical Development Plan
Clinical Development Plan (as of 2/2021) Phase Type n Description 1/2 Multi - center Phase 1 dose - finding study followed by a randomized placebo - controlled Phase 2 expansion phase to assess the safety, tolerability, and efficacy of iMSCs in patients with moderate to severe ARDS due to COVID - 19
Slide 42: Clinical Development Plan timeline
Clinical Development Plan (estimated as of 2/2021*) Pre - Clinical And Clinical Regulatory Product and CMC 2020 2021 2022 2023 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Pre - Clinical (tox) Studies Chemistry and Manufacturing Control (CMC) Development Phase 3 Clinical Study POC (Sheep) studies
Slide 43: Mino-Wrap
Mino - Wrap CITI - 101 Minocycline/Rifampin (M/R) Gelatin Film Bioabsorbable Extended Release Antimicrobial Wrap for the Prevention of Breast Tissue Expander Infections
Slide 44: Rate of Infection Post-Mastectomy
44 NASDAQ: CTXR Background: Rate of Infection Post - Mastectomy • The rate of infection following mastectomy with tissue expander (TE) is 2.4 to 24%. Estimated mean is 12 - 14%*. • Once the implant becomes infected, the patient is usually hospitalized requiring approximate 2 weeks of IV and/or oral
Slide 45: Mino-Wrap: Thesis
45 NASDAQ: CTXR Mino - Wrap: Thesis • The highest risk for TE - related infections occurs at the time of surgery and as long as drains remain in place (about two weeks post - operatively) and there are portals for microbial colonization. • Mino - Wrap is a malleable, bioabsorbable, antimicrobial wra
Slide 46: Mino-Wrap Development Plan
46 NASDAQ: CTXR 2019 2020 2021 2022 2023 2024 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Orphan drug request Pre - Clinical And Clinical Regulatory Product and CMC NDA Approval Mino - Wrap Development Plan (estimated as of 2/2021*) Pre - Clinical Studies Chemistry and Ma
Slide 47: Halo-Lido CITI-002
Halobetasol /Lidocaine Prescription Strength Topical for Symptomatic Hemorrhoid Treatment Halo - Lido CITI - 002
Slide 48: First FDA-approved prescription product for hemorrhoids
48 NASDAQ: CTXR CITI - 002 ( halobetasol + lidocaine) Citius’ product candidate would be the first FDA - approved prescription product to treat hemorrhoids in the US OTC Products are the Mainstay for Treatment of Grade I and II • Up to 5% of the U.S. population suffers from hemorrhoids, but there ar
Slide 49: Current Status
49 NASDAQ: CTXR Current Status • Based on the results of phase 2 trial in 240 patients, CTXR elected to use super potent steroid Halobetasol propionate (HBP), maintained Lidocaine HCl (LH) and developed 10 prototype formulations • Two formulations selected for Vasoconstriction Assay (VCA) studies •
Slide 50: Halo/Lido Development Plan
50 NASDAQ: CTXR Halo/Lido Development Plan (estimated as of 2/2021*) 2015 2016 2017 2018 2019 2020 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Phase 2a Clinical Study Product and Formulation Development Phase 2b Clinical Study Pre - Clinical Studies FDA Type C meeting IND
Slide 51: Corporate Summary
51 NASDAQ: CTXR CITIUS Corporate Summary • Addressing attractive diversified multi - billion dollar opportunities – Adjunctive Cancer Care/Infectious Disease and Gastrointestinal Disease • Partnership with Novellus in developing highly promising i - MSC therapies • Partnership with MD Anderson Cance
Slide 52: Financial Summary
52 NASDAQ: CTXR Financial Summary (as of 02/11/21) Current Cap Table Sha r es % of Fully Diluted Basic Shares Outstanding 74,954,525 67.4 % Warrants 31,639,976 28.5 % Options 4,490,166 4.0 % Unit Purchase Options 100 , 667 0. 1 % Fully Diluted Shares Outstanding 111,185,334 100% ( 1 ) Beneficial sto
Slide 53: Citius Pharmaceuticals contact details
53 NASDAQ: CTXR Citius Pharmaceuticals, Inc. 11 Commerce Drive First Floor Cranford, NJ 07016 www.citiuspharma.com Investor Relations Contact : Andrew Scott – V.P., Corporate Development (908) 967 - 6677 x105 ascott@citiuspharma.com
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