Compounds For Cures

Compounds For Cures

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.

Safe Harbor Forward-Looking Statements

Management

Leo Ehrlich, CPA

CEO and Chief Financial OfficerBoard of Directors

• Founder of Cellceutix in 2007

• CEO since December 2010

Management

Krishna Menon, RCM, PhD, VMD

President, Chief Scientific OfficerBoard of Directors

• Dr. Menon has more than 30 years in drug development for academia and industry

• 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

• Group Leader, Cancer In Vivo Research and Clinical Development, for Eli Lilly (1995-2001), where he played a key role in lead selection and pre-clinical development of Gemzar and Alimta, well known multi-billion dollar drugs

• In 1999, Lilly honored Menon with the President's Recognition Award, the most prestigious award at Lilly

Key Acquisition

Acquired PolyMedix September 4, 2013• 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• 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• 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• 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)• Psoriasis• FDA 505(b)(2) application to be filed• Drug manufacturing completed by Dr. Reddy’s Labs (SYMBOL:RDY)

KM-391• 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)

• Dose escalation study in chemotherapy-resistant subjects with solid tumors Cycle: treatment once/ week for 3 weeks, followed by 1 week off treatment p53 status of tumors will be evaluated

• 18 patients enrolled to date

• Endpoints Safety Maximum Tolerated Dose (MTD) Tumor size Serum tumor markers Biomarker: increased p21 expression in lymphocytes

• Completed 4 cohorts, currently in 5th cohort 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• Evidence of stable disease by radiological examination in 6 cases, which includes head

and neck cancer, ovarian cancer, liposarcoma and clear cell carcinoma

• Disease progression occurred following stabilization of disease in some cases

• Subject 103 had 6 months of treatment and 2 successful restaging with stable disease

• 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

• This points to possibility to increase dose and frequency

p21 biomarker expression in patients•Kevetrin activated p21 in 4 out of 5 patients treated with 20 or 30 mg/m2

Kevetrin Phase 1

No alteration in hematological profile was observed due to Kevetrin•No leukopenia•No neutropenia•No anemia•No thrombocytopenia

*In contrast, standard cytotoxic chemotherapeutic drugs have adverse effects on hematology

During infusion with Kevetrin:

• No neuropharmacological changes were observed

• No adverse effects on cardiovascular system

No change in subject’s cardiac rhythm or ECG

• 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:

• Nausea

• Vomiting

• Chest tightness

• Shortness of breath

• High blood pressure

• Fatigue

• Pre-medication with antihistamines or steroids was administered per direction of the attending physician

*None were Grade 3 Adverse Events

Active Projects

Kevetrin• 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)• Psoriasis

• FDA 505(b)(2) application

KM-391• Autism

Prurisol for PsoriasisPrurisol

Controls

0

1000

2000

3000

4000

Control Efalizumab KM-133 x1 KM-133 x2

# CD

4+ c

ells

/ u

l blo

od

CD 4

0

200

400

600

800

1000

1200

Control Efalizumab KM-133 x1 KM-133 x2

#CD

8+ ce

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CD 8

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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