[ NASDAQ: MSHL ] Southern California Investor Conference August 29, 2011

Transcription

1 [ NASDAQ: MSHL ] Southern California Investor Conference August 29, 2011

2 Forward-Looking Statements These slides and the accompanying oral presentation contain forward-looking statements. Actual events or results may differ materially from those projected in any of such statements. Additional information concerning factors that may cause actual events or results to differ from those projected is contained in Marshall Edwards most recent annual report on Form 10-K and quarterly reports on Form 10-Q, as well as other subsequent filings with the SEC. 2

3 Marshall Edwards (Nasdaq: MSHL) ncology company focused on the clinical development of novel therapeutics targeting cancer metabolism Extensive clinical experience with multiple compounds developed from proprietary technology platform Two lead compounds show robust activity against broad range of tumor cell lines ME-143 expected to enter clinic by September; ME-344 by Q Worldwide rights to lead compounds as well as other potential drug candidates and indications Headquartered in San Diego 3

4 Management Team Executive Management Daniel Gold, Ph.D. President & Chief Executive fficer Former Chief Scientific fficer & Founder of Favrille Thomas Zech Chief Financial fficer Former Chief Financial fficer at Pacira Pharmaceuticals Robert Mass, M.D. Chief Medical fficer Former Head of Medical Affairs, Bioncology at Genentech Board of Directors Bryan Williams, Ph.D. (Chairman) Director of the Monash Institute of Medical Research Leah Cann, MBA Two-time Wall Street Journal All-Star Analyst Daniel Gold, Ph.D. President & CE of Marshall Edwards William Rueckert Chairman of Novogen Limited Christine White, M.D. Former Senior V.P. of Global Medical Affairs at Biogen Idec 4

5 Lead Drug Candidates Derived from Proprietary Isoflavone-Based Platform H H H H H Scaffold: Genistein 1 st Generation: Phenoxodiol Next Generation H H H H NADH xidase Inhibitor: ME-143 H Mitochondrial Inhibitor: ME-344 H 5

6 NADH xidase (tnx) Inhibitors Mechanism of Action: Caspase-Mediated Apoptosis 6

7 Mitochondrial Inhibitors Mechanism of Action: Caspase-Independent Cell Death Drug Mitochondria CX IV Endo G RS ATP ADP AMPKα1 ERK Bax mtr Nucleus LC3-II DNA Fragmentation Caspase- Independent Cell Death Destructive Autophagy 7

8 Lead Candidates ME-143 & ME-344 Superior Single-Agent Anti-Tumor Activity* Compound Breast (SK-BR-3) Melanoma (MM200) Lung (NCI-H460) varian (CP70) Prostate (PC3) Phenoxodiol ME ME * IC 50 = M 8

9 tnx Inhibitor Clinical Trial Experience Platinum-Resistant or Refractory varian Cancer Route of Phenoxodiol Administration Treatment ME (N=70) ral Weekly platinum + Phenoxodiol ME (N=20) Intravenous Weekly platinum + Phenoxodiol Response Rate 0/70 = 0% 6/20 = 30% 9

10 tnx Inhibitor Clinical Trial Experience Comparable Patient Demographics (ITT Population) ME ral Phenoxodiol ME i.v. Phenoxodiol Chemotherapy Carboplatin Cisplatin Age in years Median: Range: Karnofsky performance status Median: Range: Histology Serous: Endometrioid: ther: Number of prior regimens Median: Range: Months since platinum refractoriness/resistance Median: Range: % 10% 9% % 9% 26%

11 tnx Inhibitor Program Summary Clinical experience shows insufficient levels of active drug achieved when 1 st generation Phenoxodiol is administered orally Strong Phase 2 data that suggest Phenoxodiol is active when administered intravenously Next-generation ME-143 significantly more active in pre-clinical studies Expect to initiate Phase I trial of ME-143 by September

12 Mitochondrial Inhibitor Program H H H H CH 3 Prodrug: ME-128 Demethylated in vivo Lead Candidate: ME-344 H 12

13 Mitochondrial Inhibitor: Potent Inhibitor of Chemo- Resistant Primary varian Cancer Cell Lines* ME-128 * Alvero, Montagna, Mor, et al ME-128, a novel isoflavone derivative, induces caspase independent cell death through Akt/mTR pathway. Cancer. 115:

14 Mitochondrial Inhibitor: Active on KRAS Mutant, Tarceva-Resistant NSCLC Cell Lines* A549 NCI-H1734 NCI-H1299 * Data provided by Dr. Kwok-Kin Wong, Dana Farber Cancer Center, Boston 14

15 Tumor Volume (mm 3 ) Mitochondrial Inhibitor: As Effective as Rapamycin in Pre-Clinical Studies 1000 R182 varian Xenograft Model ME-128 vehicle control ME-128 (50 mg/kg) RAPAMYCIN vehicle control RAPAMYCIN 40 mg/kg * * *p Time (Days) 15

16 Percentage Change - Body Mass but with Less Toxicity ME-128 Vehicle Control ME-128 (50mg/kg) RAPAMYCIN Vehicle Control RAPAMYCIN - 40mg/kg *p * Time (Days)

17 Mitochondrial Inhibitor Program Summary Dual mechanism of action caspase-independent cell death via DNA fragmentation and destructive autophagy Active against a broad range of major cancer cell lines, including: Chemotherapy-resistant ovarian cancer stem cells KRAS-mutant, Tarceva-resistant non-small cell lung cancer Next-generation ME-344 significantly more active in pre-clinical studies Expect to submit IND application for ME-344 by Q

18 Clinical Development Strategy Goal: Establish clear evidence for clinical activity in combination with standard chemotherapy regimens Randomized/controlled Phase II trials Assumption: Mechanism of action and preclinical results do not predict specific tumor type or chemotherapy combination Focus: Common tumors with unmet medical need, unambiguous assessment of clinical benefit and weekly/bi-weekly chemotherapy regimens e.g., metastatic breast, metastatic colorectal, ovarian 18

19 Clinical Development Timeline Q Q Q Q Q Q Q Q NADH xidase Inhibitor Program Drug manufacturing of ME-143 IND-enabling preclinical studies Single agent Phase I trial Phase II trials + chemotherapy Mitochondrial Inhibitor Program Lead candidate selection Drug manufacturing of ME-344 IND-enabling preclinical studies Single agent Phase I trial 19

20 Milestones Establish headquarters in the U.S. Add oncology expertise to Board of Directors Acquire rights to lead product candidates Appoint experienced Chief Medical fficer Bolster intellectual property portfolio NADH xidase Inhibitor Program Complete drug manufacturing of ME-143 Submit IND application to FDA for ME-143 Initiate Phase I safety trial of ME-143 (Q3 2011) Mitochondrial Inhibitor Program Complete drug manufacturing of ME-344 (Q3 2011) Submit IND application to FDA for ME-344 (Q1 2012) Initiate Phase I safety trial of ME-344 (1H 2012) 20

21 [ NASDAQ: MSHL ] Southern California Investor Conference August 29, 2011