kinnear pharmaceuticals_bio 2015_final_short
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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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Ceragenins are Innate Immune ModulatorsNovel, proprietary small molecule mimetics of defensins, key components of the innate immune system
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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Kinnear Pharmaceuticals’ Emerging PortfolioBuilding a portfolio that leverages ceragenins’ anti-inflammatory and ulceration healing and prevention activities
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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Mucositis is the Leading Radiation Oncology ToxicityOral mucositis is a serious side effect of head and neck cancer therapy that often stop oncology treatment
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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The Human Innate Immune System Plays a Key Role in the Development of Oral MucositisInnate immune response modulation during initiation phase is considered the best therapeutic strategy
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 responseProvides broad-spectrum antimicrobial protectionAids lesion healing or host repair processesActs prophylactically and therapeuticallySmall molecule providing cost and stability advantage
Normal Epithelium Initiation Amplification Ulceration Healing
Damaged epithelium increases risk of infection
Mucositis Phases
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KIN-219 Reduced Severe Oral Mucositis >90%Unsurpassed, “spectacular” performance by a non-peptide or non-polymer small molecule drug 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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Next Study Will Evaluate Prophylaxis and TreatmentStudy design includes fractionated radiation and 2 treatment arms; to be conducted by Biomodels (Watertown, MA)
KIN-219
KIN-219
KIN-219
KIN-219
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KIN-219 Oral Rinse for Oral MucositisKinnear Pharmaceutical’s lead program with expected composition of matter coverage beyond 2030
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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Oral Mucositis Mouth Rinse Development PlanPreliminary draft development plan shows exit potential in ~3 years; assumes a capital raise in October 2015
Opportunity Validation• Confirmatory efficacy• Screening Tox + IND
enabling toxicology• $1.8M + overhead
Preclinical Development• API process development• Doseform development• Clinical planning• Regulatory interactions• $1.7M + overhead
Phase 1a/b Trials• First-in-human clinical trials• Phase 2 enabling toxicology• Potential Breakthrough
designation• Potential Orphan designation• $3M + overhead
Phase 2a Trial• ~48 patients• 3 dose levels + placebo• Human efficacy POC• $3.3M + overhead
Phase 2b or 3 Trial• ~90 patients• 2 dose levels• Human efficacy• $5.5 + overhead
2015 2016 2017 2018 2019
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CSA-13 Reduced Severity of Colitis in an IBD modelData generated in an in vivo colitis model at UCLA; currently planning a follow up study at Cincinnati Children’s Hospital
Figure 3:(A) Experimental plan of chronicTNBS colitis in mice. n=10 mice pergroup. (B) H&E staining. CSA13reduced TNBS colitis-mediatedmucosal transformation and roundcell infiltration in colons. (C)Histology score. (D) CSA13 reducedTNBS colitis-induced colonicproinflammatory cytokine TNFaexpression. . (E) Mouse Raw264.7macrophages were incubated withLPS and CSA13 for 16 hours. CSA13inhibited LPS-induced mouse TNFaprotein expression.
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Ceragenins Upregulate Hyaluronan and CYR61/CCN1Key drivers in epithelial cell proliferation and migration, as well as tissue healing, positively impacted by CSA-13
Figure 4:(A) Human non-transformed colonic NCM460epithelial cells incubated with CSA13 for 8hours induced CYR61/CCN1 proteinexpression. (B) Human intestinalmicrovascular endothelial cells incubatedwith CSA13, anti-CYR61 neutralizing antibody(20mg/ml) and VEGF for 24 hours. CSA13induced tube formation was blocked by anti-CYR61 neutralizing antibody. (C) NCM460cells were incubated with CSA13 and anti-CYR61 antibody (20mg/ml, #NB100-356,Novus Biologicals) for 72 hours. CSA13induced cell proliferation was blocked by anti-CYR61 neutralizing antibody. (D) ColonicCyr61/Ccn1 mRNA expression in mice wasincreased in DSS colitis and was augmentedby intracolonic CSA13 administration.
D.
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IBD Development PlanPreliminary development plan shows exit potential in ~3 - 4 years, assuming KIN-219 is lead candidate
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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Potential Anti-inflammatory Expansion Indications Based on similar clinical pathophysiology to oral mucositis and IBD
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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Capital Requirements from May 2015 ForwardMarginal costs associated with a second indication largely attributable to direct 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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Oral Mucositis alone is a >$500 million OpportunityKIN-219 is 3x more effective in translatable animal efficacy models than Soligenix’s SGX942
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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Kinnear Pharmaceuticals Leadership TeamSuccessful track record of monetizing novel technologies and driving innovation agendas
•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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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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M I C H A E L T R I P L E T T, P H . D.
C H I E F E X E C U T I V E O F F I C E R
6 1 4 . 3 6 0 . 3 1 6 0
M T R I P L E T T@ K I N N E A R P H A R M A . C O M
B R U C E H A L P RY N, P H . D.
C H I E F O P E R AT I N G O F F I C E R
5 1 3 - 6 5 9 - 4 6 9 0
B H A L P RY N @ K I N N E A R P H A R M A . C O M
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DISCLAIMERCertain statements made in this presentation may include historical information and forward lookingactions that Kinnear Pharmaceuticals anticipates based on certain assumptions. These statements,indicated by words such as “expect,” “anticipate,” “should,” etc., denote uncertainty in facts, figuresand outcomes. While Kinnear Pharmaceuticals believes that the expectations reflected in suchforward-looking statements are reasonable, it can give no assurance that such statements will prove tobe correct. Please contact Kinnear Pharmaceuticals for a full explanation of the risks associated withan investment in Kinnear Pharmaceuticals.
The information provided in this presentation is being supplied to the best of the presenter’sknowledge. You are not entitled to rely upon any information that is provided to you by KinnearPharmaceuticals in this presentation. In deciding to enter into any transaction, you must rely uponyour own independent due diligence and investigations. Information presented in this presentationdoes not constitute legal or financial advice, and you are encouraged to consult your professionaladvisors regarding any such advice.
This presentation is copyright 2015 Kinnear Pharmaceuticals. All Rights Reserved.
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