Summary 2011

1. Summary 2011

2. About • Strategic Focus • Repurposed drugs with less regulatory, safety and efficacy risk • Cancer treatment with a better quality of life Founded 2010 in Houston Vision: To be the leader in the development of metronomic therapies for cancer $4.2M Series A: Founded by seasoned biotech, pharma and generics executives Initial indications prioritized for fast clinical & regulatory path (later expansion to wider markets) Confidential Property of MetronomX

3. Childhood Neuroblastoma (neuronal tumors) 7%–10% of childhood cancers of pediatric cancer deaths in U.S. 15% HIGH RISK RELAPSED / REFRACTORY (60% of TOTAL) <5% long-term survival average progression- free survival 42 days Confidential Property of MetronomX

4. A Critical Unmet Need Neuroblastoma Progress Lags Behind Other Childhood Cancers Low Survival Even with Dose Intensity Neuroblastoma • Dose Intensity  survival by ~ 4x (26%) • Need new agents with non- overlapping toxicities to chemo Change in Death Rate Acute Lymphoblastic Leukemia 1999 2009 1969 1979 1989 Translations of the American Clinical and Climatological Association, vol. 121, page 183, 2010 1960 2011 NO SIGNIFICANT PROGRESS IN 50 YEARS Confidential Property of MetronomX

5. Current Treatment • 60% relapse<1 year 40% 20% 40% • Low • Risk • Surgery & Observation • Survival: >90% • High • Risk • Initial Treatment: • Chemotherapy, surgery, double autologous BMT, radiation, retinoic acid +/- antibody therapy • 1st relapse treatment: • Cytoxan + • topotecan therapy • Survival: • <5% • Intermediate Risk • Chemotherapy • Carboplatin, Etoposide, Cytoxan, Doxorubicin • Survival: • 80%–90% Confidential Property of MetronomX

6. Repurposed Drugs:A Successful Strategy MNX-100 • Strong Clinical & preclinical data • Patent applications filed • Exclusive worldwide rights Approved Drug Leverage New Clinical Activity H O C=N O2N N CH3 S • Established Drug Profile: • Approved for Chagas in South American and Central American countries starting in 1972 • >1000s of patients treated >40 years • Rapid, Lower-Risk Development: • Well understood pharmacologically • Not in US formulary (never approved) • Good toxicology, pharmacology, carcinogenicity and teratogenicity publications O O Confidential Property of MetronomX

7. Discovery (2002): MNX-100 Cytoxan & topotecan PLUS MNX-100 Initial Tumor Metastatic neuroblastoma & Chagas disease MNX-100 3 wks REMISSION J PediatrHematolOncol28: 10, 693-6, October 2006 MNX-100 12 wks MNX-100 8 wks 6 additional relapsing and refractory neuroblastoma patients 2 PR’s 4 SD’s Necrosis Necrosis Rationale for initiating Phase I clinical trial

8. Preclinical Studies: Summary Tumor Growth • Directly slows tumor growth in a variety of tumor types • Active in a variety of neuroblastoma and medulloblastoma cell lines (including TICs) and mouse models of neuroblastoma and medulloblastoma • Interacts with catecholamines to enhance toxicity • Selective for neuroblastoma and possibly small cell cancer, adrenal cancer, glioblastoma, melanoma, stellate pancreatic, pheochromocytoma, adrenal carcinoma, etc. • Results in Oxidative Stress • Nitroreduction and damage to nucleus • Induces Apoptosis • Induction of caspase 3 • Suppresses AKT phosphorylation • An important mediator of tumor resistance • Increases tumor response in combination with cytoxan, topotecan and rapamycin • Drug classes widely used across many human tumors, including NSCLC, breast, colon, ovarian, renal, glioblastoma, CHOP, etc. • Synergistic activity with rapamycin opens door to additional ‘maintenance’ schedules Neuro- endocrine Oxidative Stress Apoptosis AKT-P Synergy

9. Phase I Clinical Trial Results PR+SD=80% J Pediatr Hematol Oncol 33:1, 23-30, January 2011 *Defined as patients who had radiological stable disease with either a 50% decrease in urinary catecholamines if elevated at study entry, clearance of bone marrow disease on greater than 2 biopsies, or resolution of MIBG activity 9.1 months PFS Mean PFS: • Maximum tolerated dose reached: 30 mg/kg/day • Dose-limiting toxicities were reversible nausea and neuropathy; no hematological or cardiac side effects • Tumor responses seen both as single agent and in combination • 6/14 alive 12 months following treatment; greatly improved quality of life including reduced narcotic use, improved mobility, return to school MNX-100: ✓activityas single agent ✓ efficacy as combination therapy ✓ mild toxicity ✓ oral administration

10. MNX-100 Phase II: Response to Date PFS Increase PR +400% +5 months SD +224% +2.2 months PR+SD=70% Multiple relapsed, chemotherapy- refractory patients 1 19/20 actively progressing at time of study 2 patients treated with two 21 day cycles of NFX alone Confidential Property of MetronomX

11. Phase II Responses: Prestudy vs. Treatment NFX + CTX + TOPO NFX Alone NFX + CTX + TOPO

12. MNX-100: Summary • Follow-on multicenter Phase II studies of MNX-100 shows promising clinical activity in relapsing and refractory neuroblastoma, especially when combined with cytoxan and topotecan • RECIST response rate of 20-30% • PFS in responding patients 4X’s that of nonresponders • Average PFS for all 43 patients (PD, PR, SD) = 154 days • Accrual rate average 0.23 patients/center/month; top centers at 0.5 to 0.6 • Combined therapy has acceptable safety profile • Preclinical data suggests AKT and GSH may be important targets • Synergy as well as activity in other cancer cell lines, particularly neuroendocrine (e.g., SCLC) • Next step is to undertake randomized study with appropriate power to prove activity for accelerated approval registration (1992 –314 subpart H) • Applies in the setting of a new drug for a serious or life-threatening illness • Study may use a surrogate endpoint, reasonably likely to predict clinical benefit (PFS, RR) • Show meaningful therapeutic benefit over existing therapy or improved patient response over available therapy • Magnitude and consistency of effect important Confidential Property of MetronomX

13. THANK YOU