1. CORPORATE OVERVIEW FOR BIO 2015
J U N E 2 0 1 5
P H A R M A C E U T I C A L S I N S P I R E D B Y T H E H U M A N I N N AT E I M M U N E S Y S T E M
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2. Ceragenins are Innate Immune Modulators
Novel,proprietarysmallmoleculemimeticsofdefensins,keycomponentsoftheinnateimmunesystem
Proprietary, small molecule functional mimetics of defensins or cationic antimicrobial peptides (AMPs)
Ceragenins therapeutically modulate the innate immune system
Portfolio of >100 compounds that differentially exhibit immunomodulatory, anti-inflammatory,
antimicrobial, antiviral, anticancer, and osteoinduction activities
Subject of >40 peer reviewed scientific publications, $ millions of public and private investment, and
multiple >$10 million transactions in pharmaceuticals and animal health products
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3. Kinnear Pharmaceuticals’ Emerging Portfolio
Building a portfoliothatleveragesceragenins’anti-inflammatoryandulcerationhealing andpreventionactivities
Program in vitro Efficacy in vivo Efficacy Preclinical Safety Phase 1
Oral Mucositis 2015-16* 2017*
Inflammatory Bowel Disease 2015-16* 2017*
Bone (Osteoinduction)
Cystic Fibrosis Inhaled Anti-infective
Multiple Myeloma
*The optimized portfolio strategy, after oral mucositis, will be determined by end of Q4 2015; actual timing depends upon capital availability.
Current base case assumes 2 – 3 programs move forward:
Oral mucositis is lead program with KIN-219 as lead candidate
IBD is second program with KIN-219 as lead candidate, leveraging oral toxicology and CMC investments made for oral mucositis
Undisclosed radiation countermeasure program under evaluation by NIH and DOD
Bone and cystic fibrosis programs move forward with non-dilutive, foundation or partner funding only
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4. Mucositis is the Leading Radiation Oncology Toxicity
Oralmucositisisa serioussideeffectof headandneckcancertherapythatoftenstoponcologytreatment
Complication of several types of cancer therapy
◦ Radiation for head and neck cancers
◦ Conventional, cycled chemotherapy for solid tumors
◦ Hematopoietic stem cell transplantation (HSCT)
Painful and debilitating inflammation and ulceration
◦ Moderate to severe cases inhibit eating and drinking; can lead to death
◦ Can be dose limiting, requiring reduction or stoppage of cancer therapy
◦ Secondary bacterial or fungal infections, including a 4x septicemia risk
Significant pharmacoeconomic burden
◦ ~$21,800 (2012) incremental cost per head and neck patient1
◦ ~$33,000 (2012) incremental cost per 1-pt increase in HSCT patients2
Significant unmet clinical need
◦ Few and mostly ineffective prophylactics and therapies
◦ Options range from homeopathic, devices, to a single Rx for HSCT
Grade 1 (Mild)
Oral soreness, erythema
Grade 2 (Moderate)
Oral erythema, ulcers,
solid diet tolerated
Grade 3 (Severe)
Oral ulcers, liquid diet
only
Grade 4 (Life-threatening)
Parenteral or enteral
feeding only, intubation
Grade 0 (None)3
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5. The Human Innate Immune System Plays a Key Role in
the Development of Oral Mucositis
Innateimmuneresponsemodulationduring initiationphaseisconsideredthebesttherapeuticstrategy
Radiation and chemotherapy induce an excessive
innate immune response in the targeted tissues,
producing a damaging inflammatory response
Ideal oral mucositis drug profile:
Reduces the innate inflammatory response
Provides broad-spectrum antimicrobial protection
Aids lesion healing or host repair processes
Acts prophylactically and therapeutically
Small molecule providing cost and stability advantage
Normal
Epithelium
Initiation Amplification Ulceration Healing
Damaged epithelium
increases risk of infection
Mucositis Phases
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6. KIN-219 Reduced Severe Oral Mucositis >90%
Unsurpassed,“spectacular”performancebya non-peptideornon-polymersmallmoleculedrug candidate
>20% REDUCTION REQUIRED FOR DRUG VIABILITY
KIN-009 & KIN-219 BOTH EXCEEDED 90%
KIN-219 (LIGHT BLUE) PROVIDED THE STRONGEST
AND LONGEST LASTING REDUCTION
Data generated by Biomodels (Watertown, MA) in a translatable oral mucositis model
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7. Next Study Will Evaluate Prophylaxis and Treatment
Studydesignincludesfractionatedradiationand2 treatmentarms;tobeconductedbyBiomodels(Watertown,MA)
KIN-219
KIN-219
KIN-219
KIN-219
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8. KIN-219 Oral Rinse for Oral Mucositis
KinnearPharmaceutical’sleadprogramwithexpectedcompositionofmattercoveragebeyond2030
KIN-219 PROPERTIES SUPPORT DEVELOPMENT
~8 and 3 step, scalable synthetic pathways
Adequate solubility in water
Stable in solution and in biological environments
Demonstrates secondary antimicrobial activity
Low oral bioavailability expected
CERAGENINS DISPLAY ANTI-INFLAMMATORY,
ANTIMICROBIAL AND LESION HEALING ACTIVITIES
Ceragenins down regulate critical inflammatory markers:
◦ TNFα
◦ NF-kB
◦ IL-6
Ceragenins upregulate hyaluronan and CYR61/CCN1, key
factors in epithelial cell proliferation and migration as well
as tissue healing
CSA-13 showed in vivo efficacy for oral mucositis and IBD;
KIN-219 is a close analog of CSA-13
Ceragenins demonstrate broad-spectrum antimicrobial
activity, including multidrug resistant strains and biofilms
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10. CSA-13 Reduced Severity of Colitis in an IBD model
Data generatedinaninvivocolitismodelatUCLA;currentlyplanning a followupstudyatCincinnatiChildren’sHospital
Figure 3:
(A) Experimental plan of chronic
TNBS colitis in mice. n=10 mice per
group. (B) H&E staining. CSA13
reduced TNBS colitis-mediated
mucosal transformation and round
cell infiltration in colons. (C)
Histology score. (D) CSA13 reduced
TNBS colitis-induced colonic
proinflammatory cytokine TNFa
expression. . (E) Mouse Raw264.7
macrophages were incubated with
LPS and CSA13 for 16 hours. CSA13
inhibited LPS-induced mouse TNFa
protein expression.
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11. Ceragenins Upregulate Hyaluronan and CYR61/CCN1
Keydriversinepithelialcellproliferationandmigration,aswellastissuehealing,positivelyimpactedbyCSA-13
Figure 4:
(A) Human non-transformed colonic NCM460
epithelial cells incubated with CSA13 for 8
hours induced CYR61/CCN1 protein
expression. (B) Human intestinal
microvascular endothelial cells incubated
with CSA13, anti-CYR61 neutralizing antibody
(20mg/ml) and VEGF for 24 hours. CSA13
induced tube formation was blocked by anti-
CYR61 neutralizing antibody. (C) NCM460
cells were incubated with CSA13 and anti-
CYR61 antibody (20mg/ml, #NB100-356,
Novus Biologicals) for 72 hours. CSA13
induced cell proliferation was blocked by anti-
CYR61 neutralizing antibody. (D) Colonic
Cyr61/Ccn1 mRNA expression in mice was
increased in DSS colitis and was augmented
by intracolonic CSA13 administration.
D.
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12. IBD Development Plan
Preliminarydevelopmentplanshowsexitpotentialin~3- 4 years,assuming KIN-219isleadcandidate
Opportunity Validation
•Confirmatory efficacy
•$85K + overhead
Preclinical Development
•Candidate screening for process R&D
•FIH enabling toxicology, if needed
•Modified release dose form development +
animal testing
•Clinical planning
•Regulatory interactions
• $300K + overhead
Phase 1a/1b Trial
•FIH clinical trials
•Human indication of activity
•Potential Breakthrough designation
•$2.8M + overhead
Phase 2a Trial
•~90 patients
•2 dose levels
•Human efficacy
•$7.5M + overhead
Phase 2b Trial
2015 2016 2017 2018 2019
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13. Potential Anti-inflammatory Expansion Indications
BasedonsimilarclinicalpathophysiologytooralmucositisandIBD
Mucositis as a result of radiation oncology therapy and/or radiation + chemotherapy
◦ Oropharyngeal
◦ Proctitis
◦ Gastric mucositis
Post-radiation exposure gastric mucositis
◦ NIH and DOD priority countermeasure
◦ ‘Dirty bomb’ treatment for Strategic National Stockpile
◦ 4 May 2015 review of data with NIH and DOD for potential inclusion in free drug screening program
◦ Gateway to significant and expedited federal funding
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14. Capital Requirements from May 2015 Forward
Marginal costs associatedwitha secondindicationlargelyattributabletodirect R&D expenses
Seeking a $6 million raise in Q4 2015 to fund preclinical development of oral mucositis and IBD
Will require a $7.5 million raise to fund Phase 1 studies in Q4 2016 (plan to conduct studies in
Australia; potential 43.5% tax credit not reflected in capital requirements)
Expense Category Opportunity Validation
(May 2015 – June 2016)
Preclinical
(July 2016 – Nov 2016)
Phase 1
(Dec 2016 – Dec 2017)
Phase 2a
(Jan 2018 – Dec 2018)
Oral mucositis R&D $1.8 M $1.7 M $3.0 M $3.3 M
IBD R&D $0.09 M $0.30 M $2.8 M $7.5 M
Pharma G&A $1.6 M $0.55 M $1.5 M $1.3 M
IP & License $0.85 M $0.06 M $0.24 M $0.22 M
Total by Phase $4.3 M $2.6 M $7.5 M $12 M
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15. Oral Mucositis alone is a >$500 million Opportunity
KIN-219is3xmoreeffectiveintranslatableanimalefficacymodelsthanSoligenix’sSGX942
Oral mucositis market estimates:
◦ MuGard believes it can achieve sales of
$350 million by 2020
◦ RxKinetix believes the mucositis market
potential is in excess of $1 billion
IBD is a significantly larger market
opportunity than oral mucositis
Potential medical countermeasure
programs could provide non-dilutive R&D
capital with possible Strategic National
Stockpile sales
In total, Kinnear Pharmaceuticals
represents >>$100 million opportunity
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16. Kinnear Pharmaceuticals Leadership Team
Successfultrackrecordof monetizing noveltechnologiesanddriving innovationagendas
•Progressive leadership roles at Procter & Gamble, Ventaira Pharmaceuticals and Battelle in R&D, technology
commercialization, and corporate strategy
Michael Triplett, PhD - Chief Executive Officer
•Former director of early stage drug R&D, liaison to venture capital funds, and leader of technical due diligence for
Procter & Gamble Pharmaceuticals
Bruce Halpryn, PhD - Chief Operating Officer
•KPMG, Borden Chemical, venture backed companies, and private equity experience
Ken Leachman - Chief Financial Officer
•VP of Empire Advisors where he is instrumental in fund operations, negotiations, and portfolio company
operations; formerly with Latham & Watkins LLP
David V. Richards, JD - VP, Corporate Development
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17. Kinnear Pharmaceuticals Summary
•Ceragenins are small molecule mimetics of human antimicrobial peptides, innate immune modulators often called defensins,
that express anti-inflammatory, antimicrobial, anticancer and osteoinduction pharmacological activities
Development-stage pharmaceutical company leveraging the ceragenin small molecule platform
•Additional programs inflammatory bowel disease (IBD), cystic fibrosis, bone growth (osteoinduction), and multiple myeloma
Building a gastrointestinal anti-inflammatory pipeline with oral mucositis as the lead program and
inflammatory bowel a successor program utilizing the same active ingredient as oral mucositis
•The company was founded in 2015 as a successor to N8 Medical, which split into two entities, a medical device company
which retained the N8 Medical name and Kinnear Pharmaceuticals, each pursuing ceragenin applications.
Seeking a $6 million equity investment to advance the oral mucositis and inflammatory bowel
disease programs, supported by strong preclinical efficacy data, through preclinical completion
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18. MICHAEL TRIPLETT, PH.D.
CHIEF EXECUTIVE OFFICER
614.360.3160
MTRIPLETT@KINNEARPHARMA.COM
BRUCE HALPRYN, PH.D.
CHIEF OPERATING OFFICER
513-659-4690
BHALPRYN@KINNEARPHARMA.COM
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19. DISCLAIMER
Certain statements made in this presentation may include historical information and forward looking
actions that Kinnear Pharmaceuticals anticipates based on certain assumptions. These statements,
indicated by words such as “expect,” “anticipate,” “should,” etc., denote uncertainty in facts, figures
and outcomes. While Kinnear Pharmaceuticals believes that the expectations reflected in such
forward-looking statements are reasonable, it can give no assurance that such statements will prove to
be correct. Please contact Kinnear Pharmaceuticals for a full explanation of the risks associated with
an investment in Kinnear Pharmaceuticals.
The information provided in this presentation is being supplied to the best of the presenter’s
knowledge. You are not entitled to rely upon any information that is provided to you by Kinnear
Pharmaceuticals in this presentation. In deciding to enter into any transaction, you must rely upon
your own independent due diligence and investigations. Information presented in this presentation
does not constitute legal or financial advice, and you are encouraged to consult your professional
advisors regarding any such advice.
This presentation is copyright 2015 Kinnear Pharmaceuticals.
All Rights Reserved.
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