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PHARMAUST LIMITED …Fast tracking leading edge technology in cancer therapy… PharmAust Cancer Programs PHARMAUST LIMITED CORPORATE STRUCTURE PHARMAUST  LIMITED ASX:PAA 100% Sydney-­‐Based 100% Perth-­‐Based PharmAust -­> Challenging Limitations in Cancer Therapy • Drug Toxicity • Drug Resistance • Cancer Recurrence • Poor Tumour Targeting • Poor Diagnosis ­‐> Patients Progressed at Treatment • Optimising Treatment • Survival vs Quality of Life PHARMAUST STRATEGY Oncology Focus • Targeting oncology applications of well established drugs and piggy- backing” on substantive programmes of major pharmaceutical companies • Engaging a leading clinical oncology units (St George Hospital, Sydney) Leading New Drug for Cancer Management and The Royal Adelaide Hospital • Granted patents or patent filings (either owned outright or jointly with partner) Phase II Clinical Stage • Relationships with major Strategic Partnerships with two Global Corporations • Use of CROs and external global corporations R&D suppliers Targeted Therapies for Cancer Recent  Examples  of  New  Targeted  Therapies DRUG TARGET PEAK ANNUAL SALES PATIENT COST COMPANY HERCEPTIN  + PACLITAXEL BREAST  CANCER (HER2-­‐NEU) $6.5Bln $60,000/ ANNUM GENENTECH/ ROCHE GLEEVEC MULTIPLE  CANCERS TYROSINE  KINASE $4.7Bln $92,000/ ANNUM NOVARTIS AVASTIN  + 5FU COLON,  RENAL, OVARIAN  CANCER VEGF  INHIBITOR $2.6Bln $100,000 /ANNUM (US) GENENTECH ABRAXANE  + GEMCITABINE PANCREATIC CANCER $800M $60,000/ ANNUM CELGENE Pharmaust Limited Foundations, Relationships and Commercial Cornerstones Strategic Alliances NOVARTIS ANIMAL  HEALTH NEW  SOUTH WALES INNOVATIONS IP  -­‐  Granted  and Pending  Patents UNIVERSITY  OF CAMBRIDGE Listed  Japanese Group Royal  Adelaide Hospital THE  ST.  GEORGE HOSPITAL  S CMAX &  CPR St George Hospital Department of Surgery/Oncology MONEPANTEL (MPL or PPL -1) 1. MPL is a new class of anticancer drug 2. MPL can act alone or in combination with standard of care chemotherapy for treatment of cancer 3. Pharmaust has the IP for MPL assigned from the University of NSW MONEPANTEL’S UNIQUE PROPERTIES v Unlike most cancer drugs, MPL has virtually little or no toxicity based on the toxicology undertaken by Novartis Animal Health v MPL has shown a high degree of selectivity in killing cancer cells over normal cells in culture v Studies in animal models show potent tumour regression without toxicity as measured by weight loss v MPL demonstrates a potent synergistic action with many cytotoxic drugs used in “Standard of Care” EFFECT OF MPL ON TUMOUR-BEARING MICE Dose-finding study of IP administered Drug in nude mice bearing Subcutaneous ovarian tumours (OVCAR-3), SC / IP Control Group VEHICLE Low dose Group MPL (25 mg/kg) DRUG High dose group MPL (50 mg/kg) DRUG Monepantel Kills Drug Resistant Cancer Cells • Temozolamide resistant cancer cells are effectively killed by MPL • MPL has little toxicity on “Normal” Human Embryonic Kidney Cells • MPL offers Treatment Optins either singularly or with “Standard of Care” EFFECT OF MPL ON CANCER CELLS Cell Proliferation (% of Control) TEMOZOLAMIDE RESISTANT GLIOMA U251 TEMOZOLAMIDE RESISTANT GLIOMA U251 100 75 50 25 0 0 5 10 25 [MPL] (µM) HEK [Human Embyonic Kidney cells] Viability (%) 100 75 NORMAL CELLS 50 25 0 0 10 25 Monepantel [µM] 50 50 SYNERGY BETWEEN AMINOACETONITRILES AND CYTOTOXIC DRUGS • Selective Synergy between MPL and cytotoxic drugs on “cancer” cells • Little synergy between MPL and cytotoxic drugs on “normal” cells • Opportunity for “New Class” of drug for cancer • Therapy in conjunction with standard clinical practice • Optimization of Synergy Synergy with Taxol C o n tro l M P L 2 5 m g /k g M P L 5 0 m g /k g T a x o l 2 .5 m g /k g T a x o l 2 .5 m g /k g + M P L 2 5 m g /k g T a x o l 2 .5 m g /k g + M P L 5 0 m g /k g T a x o l 5 m g /k g T a x o l 5 m g /k g + M P L 5 0 m g /k g 3 T u m o r v o lu m e (m m ) T a x o l 5 m g /k g + M P L 2 5 m g /k g 200 100 0 10 13 15 17 20 D a y s a fte r tu m o r in o c u la tio n Comparison  of  tumour  volume  V.s.  treatment  ,me  in  mice  treated  with  MPL/ Taxol  alone  or  in  combina,on  (aoer  5  doses). Lack of PPL -­1 Toxicity as Measured by Weight Loss OVCAR  3  S.C  tumour;  i.p.  injection B o d y W e ig h t 21 C o n tro l A n im a l w e ig h t ( g ) 2 m g /k g G e m 20 2 m g / k g G e m + 2 5 m g /k g M P L 5 m g / k g G e m + 2 5 m g /k g M P L 5 m g /k g G e m 19 2 m g /k g G e m + 5 0 m g /k g M P L 5 m g /k g G e m + 5 0 m g /k g M P L 2 5 m g /k g M P L 18 5 0 m g /k g M P L 17 16 15 9 11 14 16 18 21 23 25 28 30 32 35 D a y s p o s t c e ll in o c u la t io n Human Safety Trial -­ Monepantel ACCELERATED ENTRY INTO MAN DUE TO EXTENSIVE TOXICOLOGY BY GLOBAL MAJOR - Patient #1 ‐­ > Discontinued after 3 days (drug-unrelated death) - Patient #2 ­‐> Completed 28 days - Patient #3 ­‐> Discontinued after 13 days (urinary tract infection) - Patient #4 ‐­ > Discontinued after 4 days (did not wish to continue) - Patient #5 ‐­ > Completed 28 days - Patient #6 ­‐> Completed 28 days Successful Demonstration of Safety and Indication of Clinical Value by Suppression of Key Cancer Marker p70S6K D e t e r m i n a t io n o f p - P 7 0 S 6 K i n P B M C o f p a t ie n t s t r e a t e d w it h M P L D a y 1 b e fo r e tr e a tm e n t 100 D ay 3 50 0 R 01 R 101 R 02 R 102 Significance  at  Day  3  p<0.0005 R 202 R 03 MPLS,  R101 PK  –  BLOOD  LEVELS MPLS,  R02 MPLS.  Average 350 Plasma  ConcentraTon  (ng/mL) p -P 7 0 S 6 K ( % c o n t r o l) D ay 7 300 250 200 150 100 50 0 0 7 14 Study  Day 21 28 Target Markets Monepantel offers a new therapeutic regimen with or without standard Chemotherapy. The current Chemotherapy market is US$42 billion/annum. Canine Trial No Adverse Events Noted • Four Dogs Received MPL (25mg/kg) compassionately • Three Dogs treated with the lowest dose of MPL (25mg/kg) as part of trial Tasteless Formulation of MPL in Soft­‐ Gel Capsules Albendazole for the Treatment of Ascites Two Clinical Trials Completed Showing: 1. Maximum tolerated dose 2. Benefits in Localised Therapy in the abdomen 3. Significant Inhibition of VEGF Albendazole Ascites I Ascites in Man Reten7on  of  Albendazole  in  the  Abdomen Albendazole  concenra/on  (mg/ml) 1.2 1 0.8 0.6 i.p Oral 0.4 0.2 0 0 0.25 1 2 4 8 12 16 24 30 48 Time  a'er  drug  administra/on  (h) 72 120 Tumour Growing in the Abdomen and producing large quantities of Ascites fluid which distends the abdomen and reduces life expectancy Mucin Dissolution Efficacy of a Novel Mucolytic Agent on Pseudomyxoma Peritonei Mucin with Potential for Treatment through Peritoneal Catheters. EPICHEM Epichem is a profitable wholly owned subsidiary of PharmAust which provides services in synthetic and medicinal chemistry to the drug discovery and pharmaceutical industries. Epichem turnover is $2.0M/annum. With two state-of-the-art laboratories in Perth and Melbourne, Epichem serves an international clientele ranging from small operations to large multinational pharmaceutical companies. Epichem’s ability to excel in a highly competitive global marketplace was recently recognized when in 2010 it won an Australian Export Award. It has achieved this by combining a world-class team (12 Ph.Ds) of highly innovative chemists and a management committed to customer service. Contact Details DR ROGER ASTON Executive Chairman PharmAust Limited Email: rogeraston@aol.com Phone: 61 (0) 402 762 204