Transcript Compounds For Cures

Compounds For Cures
Compounds For Cures
Safe Harbor Forward-Looking Statements
To the extent that statements in this presentation are not strictly historical, including statements as to
revenue projections, business strategy, outlook, objectives, future milestones, plans, intentions, goals,
future financial conditions, future collaboration agreements, the success of the Company's
development, events conditioned on stockholder or other approval, or otherwise as to future events,
such statements are forward-looking, and are made pursuant to the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995.
The forward-looking statements contained in this presentation are subject to certain risks and
uncertainties that could cause actual results to differ materially from the statements made. Factors
that may impact Cellceutix's success are more fully disclosed in Cellceutix's most recent public filings
with the U.S. Securities and Exchange Commission.
Management
Leo Ehrlich, CPA
CEO and Chief Financial Officer
Board of Directors
• Founder of Cellceutix in 2007
• CEO since December 2010
Management
Krishna Menon, RCM, PhD, VMD
President, Chief Scientific Officer
Board of Directors
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Dr. Menon has more than 30 years in drug development for academia and industry
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Menon's PhD work focused on anti-folate therapy of various cancers. Menon was a
Research Associate Scientist at Dana Farber (Harvard University) from 1988 to
1991 and Research Scientist, In Vivo Research (Cancer & Metabolic Diseases), at
Miles Laboratories from 1991 to 1993
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Group Leader, Cancer In Vivo Research and Clinical Development, for Eli Lilly (19952001), where he played a key role in lead selection and pre-clinical development of
Gemzar and Alimta, well known multi-billion dollar drugs
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In 1999, Lilly honored Menon with the President's Recognition Award, the most
prestigious award at Lilly
Key Acquisition
Acquired PolyMedix September 4, 2013
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Pipeline consists of 10 compounds for infectious diseases, cancer supportive care and
antimicrobial applications
Proprietary computational drug design technology
Substantial equipment assets
Brilacidin completed Phase IIa clinical trial
Delparantag - Good Efficacy but with SAE- Possible reformulation
2012 - $227 million market cap (before bankruptcy) - “Outperform” rating - strong institutional
support
Brilacidin
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Small molecule, first-in-class defensin-mimetic antibiotic
Completed 200-patient Phase II clinical trial in patients with ABSSSI caused by Staph aureus
Hit primary endpoints, high and low doses outperformed Cubist Pharmaceuticals’ Cubicin in
control arm
Prime candidate to benefit from GAIN Act of 2012
Other possible indications include blood stream infections, lung infections and oral mucositis
Delparantag
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Anticoagulant reversing agent
Multiple clinical trials showed drug to reverse heparin and low molecular weight heparin
We are in the excellent position to be “Monday Morning Quarterbacking”
Adverse side effects; re-formulation possible
Active Projects
Kevetrin
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Breakthrough p53 targeted activator
Drug resistant cancers, e.g., lung, breast, colon
Phase 1 at Dana Farber/ Harvard Cancer Center and Beth Israel
Deaconess Medical Center (BIDMC) began Nov 2012
Phase1b trial at University of Bologna for hematological cancers
Additional Phase 2 trial at BIDMC for renal cell carcinoma
Prurisol (KM-133)
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Psoriasis
FDA 505(b)(2) application to be filed
Drug manufacturing completed by Dr. Reddy’s Labs (SYMBOL:RDY)
KM-391
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Autism
Kevetrin for Cancer
• Unique chemistry
• Strong IP position; many novel compounds in patent
application
• Unique mechanism of action
 Wild type and mutant p53 (p53 is a potent tumor suppressor)
 Transcriptional dependent and independent manner
 p53 is a potent tumor suppressor
• Large therapeutic index
 Potent anti-tumor activity even in drug resistant tumors
 Excellent toxicity profile
• No development of drug resistance
• Non-genotoxic
Kevetrin Phase 1
Dana Farber / Harvard Cancer Center and
Beth Israel Deaconess Medical Center (BIDMC)
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Dose escalation study in chemotherapy-resistant subjects with solid tumors
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Cycle: treatment once/ week for 3 weeks, followed by 1 week off treatment
p53 status of tumors will be evaluated
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18 patients enrolled to date
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Endpoints
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Completed 4 cohorts, currently in 5th cohort
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Safety
Maximum Tolerated Dose (MTD)
Tumor size
Serum tumor markers
Biomarker: increased p21 expression in lymphocytes
1 subject completed 7 cycles, 1 completed 4 cycles
No dose limiting toxicity
Have not yet reached maximum tolerated dose
5th cohort dose is 7.5 times initial dose; 75mg/m2
Kevetrin Phase 1
Observed stabilization of disease by restaging in multiple types of cancer
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Evidence of stable disease by radiological examination in 6 cases, which includes head
and neck cancer, ovarian cancer, liposarcoma and clear cell carcinoma
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Disease progression occurred following stabilization of disease in some cases
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Subject 103 had 6 months of treatment and 2 successful restaging with stable disease
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Subject 202 completed 4 cycles of treatment
*Stabilization was achieved at low doses in terminal patients with relapsed tumors that were
resistant to current chemotherapies.
Pharmacokinetic profiles and parameters show no evidence of accumulation
in plasma
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This points to possibility to increase dose and frequency
p21 biomarker expression in patients
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Kevetrin activated p21 in 4 out of 5 patients treated with 20 or 30 mg/m 2
Kevetrin Phase 1
No alteration in hematological profile was observed due to Kevetrin
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No leukopenia
No neutropenia
No anemia
No thrombocytopenia
*In contrast, standard cytotoxic chemotherapeutic drugs have adverse effects on hematology
During infusion with Kevetrin:
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No neuropharmacological changes were observed
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No adverse effects on cardiovascular system
 No change in subject’s cardiac rhythm or ECG
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No change in respiratory rate, minute volume or tidal volume
Kevetrin Phase 1
Adverse Events
No observed Serious Adverse Events (Grade 3) attributable to Kevetrin
to date (5th cohort at 75 mg/m2)
Observed Adverse Events (Grade 1, Grade 2):
• Cutaneous erythema (redness of the skin) was observed in a few
subjects, lesser number had pruritus
 Reactions subsided when infusion was completed
 Reactions were manageable with antihistamines and steroids
 Occurred without hemodynamic changes
Kevetrin Phase 1
Adverse Events
The following occurred in at least 1 subject:
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Nausea
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Vomiting
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Chest tightness
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Shortness of breath
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High blood pressure
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Fatigue
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Pre-medication with antihistamines or steroids was administered
per direction of the attending physician
*None were Grade 3 Adverse Events
Active Projects
Kevetrin
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Breakthrough p53 targeted activator
Drug resistant cancers, e.g., lung, breast, colon
Phase 1 at Dana Farber/ Harvard Cancer Center and Beth Israel Deaconess
Medical Center (BIDMC) began Nov 2012
Phase1b trial at University of Bologna for hematological cancers to start 2014
Additional Phase 2 trial at BIDMC for renal cell carcinoma to start 2014
Prurisol (KM-133)
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Psoriasis
FDA 505(b)(2) application
KM-391
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Autism
Prurisol for Psoriasis
Prurisol
Controls
CD 8
CD 4
1200
#CD8+ cells/ul blood
# CD4+ cells / ul blood
4000
3000
2000
1000
1000
800
600
400
200
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0
Control
Efalizumab
KM-133 x1
KM-133 x2
Control
Efalizumab
KM-133 x1
KM-133 x2
Prurisol for Psoriasis
Drug product:
 Manufacturing has been completed by Dr. Reddy’s Laboratories, Ltd.
(NYSE: RDY)
 Tablet is ~250 mg
Pipeline
Indication
Compound
Cancer
Kevetrin (KM-3174)
Psoriasis
Prurisol (KM-133)
Autism
KM-391
ABSSSI, Blood Stream Infections,
Lung Infections
Brilacidin
Anticoagulation Reversal
Delparantag
Oral Mucositis
Brilacidin-OM
Neurological
KM-362
Arthritis
KM-277
Arthritis/Asthma
KM-278
Pipeline (cont.)
Indication
Compound
Hypertensive Emergency
KM-732
Biomaterials Applications
PolyCide
Bacterial Infections
PMX-50003
Bacterial Infections
PMX-70004
Tuberculosis
PMX-10072
Malaria
PMX-30024
Fungal Infections
PMX-10098
Biodefense
PMX-30016
Oncology
PMX-20005
www.Cellceutix.com
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