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Corporate Presentation

Corporate Presentation. September 2013. Safe Harbor Statement.

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Corporate Presentation

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  1. Corporate Presentation September 2013

  2. Safe Harbor Statement • During the course of this presentation we will make statements that constitute forward-looking statements. These statements may include operating expense projections, the initiation, timing and results of pending or future clinical trials, the actions or potential action of the FDA, the status and timing of ongoing research, corporate partnering activities and other factors affecting Adherex Technologies’ financial condition or operations. Such forward looking statements are not guarantees of future performance and involve risk, uncertainties and other factors that may cause actual results, performance or achievements to vary materially from those expressed or implied in such statements. These and other risk factors are listed from time to time in reports filed with the SEDAR and the Securities and Exchange Commission, including but not limited to, reports on Forms 10-Q and 10-K. Adherex does not intend to update any forward looking information to reflect actual results or changes in the factors affecting forward-looking information.

  3. Company Overview • Biopharmaceutical company dedicated to the discovery and development of novel cancer therapeutics • Two late stage oncology clinical products: Sodium Thiosulfate (STS) and Eniluracil (EU) • STS: pending favorable data will file for NDA • EU: pending partnering discussions advance to Phase III • US based management team • Headquarters in Research Triangle Park, NC • Ticker: ADHXF – USA, AHX – Toronto • Market Cap: $10 MM; 25.1 MM shares outstanding • $1.0 MM in cash at 6/30/13, no debt • Large insider ownership with aligned shareholder incentives

  4. Management and Board of Directors • RostyRaykov – Chairman and CEO • Bear Stearns, Tiedemann Group, John Levin & Co., Alchem and DCML Co. • Chris Rallis – Director • 30 years of business development, legal and operating experience at Wellcome and Triangle Pharmaceuticals. Currently, executive in residence with Pappas Ventures. • Steve Skolsky – Director • 30 years of operating experience, including Head of GlaxoWelllcome’sDivision of HIV/Oncology, Chief Executive at Trimeris and Sequoia Pharmaceuticals. Currently, Global Head of Clinical and Data Operations at Quintiles.

  5. STS Investment Highlights • STS is a chemo-protectant agent being developed exclusively in children to prevent hearing loss caused by cisplatin • Received Orphan Drug Designation in 2004 with 7.5 years exclusivity upon approval • Phase III trial conducted by Children’s Oncology Group is fully enrolled with data on 135 patients expected in October 2013 • Phase III trial conducted by SIOPEL6 in children with liver cancer 80/102 patients enrolled • Clinical trial costs covered by government grants • Adherex has exclusive rights to data from both studies • Potential for Rare Pediatric Disease Voucher: upon approval of STS • 6 month priority review to any other new NDA or BLA application • Voucher can be transferred or sold with no restrictions • Intellectual property: Use-patent as chemo-protectant in-licensed from OHSU • Issued European and Japanese patents expire 2021, US pending prosecution

  6. Platinum Hearing Loss is Frequent, Severe and Irreversible • Platinum drugs are widelyused anti cancer agents in pediatric oncology • Produce profound, irreversible, cumulative hearing loss • Destroy the cochlear hair cells of inner ear • Effect can be seen after as little as the second or third dose • Hearing loss (ototoxicity) is a dose-limiting side effect • Up to 2,000 children receive platinum based chemotherapy every year in the US: 40-90% develop irreversible ototoxicity* • Loss of high frequency hearing sensitivity - loss of high frequency consonants (s/f/th/p/k/h/t) • Background noise compounds disability in critical settings - distance hearing and hearing in the classroom • Infants and young children at critical stage of development lack speech language development and literacy • Older children and adolescents lack social-emotional development and educational achievement *Neuwelt and Brock. J ClinOncol 2010;28:1630-1632

  7. Ototoxicityin Children Treated with Cisplatin and/or Carboplatin 61% bilateral hearing loss (ASHA criteria) at the end of treatment 41% required hearing aids that only partially restore hearing 22% of patients had dose reductions due to ototoxicity N=67 age 8 m -20 years *Gilmer-Knight et al., Journal of Clinical Oncology 88 75 67 50 11

  8. CurrentApproach to Platinum Induced Hearing Loss Detectable hearing loss generally begins after about two or three cycles of cisplatin, and may continue to occur for months (or longer) after use is discontinued Hearing Loss Detected or Communicated by Patient Hearing Loss Diagnosed Continue Course Dose Modify Switch therapies Leads to more severe hearing loss Less effective dosing/treatment, potentially shortening survival

  9. Target and Proposed STS Mechanism Requires both Cl unbound to crosslink DNA Binding to plasma proteins occurs within first hour which inactivates one binding site Free cDDP (unbound) short t1/2:1.5 hr Requires one Cl unbound to affect cochlear hair cells Binding to plasma proteins occurs within first hour which inactivates one binding site STS will bind second site preventing ototoxicity AntitumorEffect NH3 Cl Pt NH3 Cl Ototoxicity Effect NH3 STS NH3 Cl Pt NH3 Cl Pt NH3 Cl Protein Cl Protein

  10. STS Protects Against Cisplatin Ototoxicity in the Rat Change from baseline hearing threshold. Effect of STS (8 g/m2 IV) 4 hrs, 8 hrs, or 12 hrs after administration of cisplatin (6 mg/kg IA) Delayed administration of STS after platinum agents in animals reduces ototoxicity *Dickey DT et al. J Pharmacol Exp Therapeut 2005;314:1052-1058

  11. Cisplatin and STS animal PKs Cisplatin clearance is complete by 6 hrs with or without STS, when STS levels are at or higher than clinically achievable levels Cisplatin Pharmacokineticsnu/nu mice administered 4 mg/kg CDDP i.p. STS Pharmacokinetics nu/nu mice administered 3.5 g/kg STS i.p. *Gregory Reaman et al

  12. Time-Dependent Tumor Protection of STS after Administration of Cisplatin in Nude Mice STS allows for anti-tumor activity when given properly *HarnedTM et al. Clin Cancer Res 2008;14:533-540

  13. COG ACCL0431: Randomized Study of STS for Prevention of Cisplatin-induced Hearing Loss Newly diagnosed children with hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma Study Chair: David Freyer, DO, MS 135 randomized patients fully enrolled and study completed in 1Q 2012 Futility analysis conducted and reviewed by COG DSMC August 2011 with recommendation at the time to continue study Expect data in October 2013

  14. COG ACCL0431: Randomized Study of STS for Prevention of Cisplatin-induced Hearing Loss Audiology done with each cycle of therapy 80% power to detect 22.5% vs 45% change in hearing 80% power to rule out >12% difference in 3 yr EFS Secondary objectives measurement of nephrotoxicity and neurotoxicity Note: Patient must first be enrolled on the COG hearing assessment study, ACCL05C1 Newly diagnosed germ cell tumor, hepatoblastoma, medulloblastoma, neuroblastoma, or osteocarcoma Diagnosis Planned treatment program includes  200 mg/m2cisplatin(administered according to the disease-specific regimen) Study entry onto ACCL0431 Randomization Sodium thiosulfate given intravenously over 15 minutes starting 6 hours after completion of each cisplatin infusion (STS Arm) No sodium thiosulfate treatment given(Observation Arm) Protocol therapy ends when patient completes planned treatment regimen containing cisplatin

  15. SIOPEL 6: Efficacy of STS in Reducing Ototoxicity in Patients Receiving Cisplatin for Standard Risk Hepatoblastoma Newly diagnosed children with standard risk hepatoblastoma Study Chair: Peppy Brock, MD 80 randomized patients fully enrolled out of 102 Interim evaluations of efficacy of the chemotherapy carried out and reviewed by IDMC after 20, 40, 60 and 80 patients are evaluable for response Early stopping will be considered in case of concerns on efficacy of chemotherapy in either treatment arm The first two interim safety analysis after 20 and 40 patients were conducted with IDMC recommending study to continue Two interim and one final efficacy analyses planned for early stopping in case of a greater than expected difference between treatment arms in terms of hearing loss

  16. SIOPEL 6: Efficacy of STS in Reducing Ototoxicity in Patients Receiving Cisplatin for Standard Risk Hepatoblastoma DELAYED SURGERY RANDOMISATION Diagnostic biopsy Tumour storage Radiological staging RRR if required Registration to remote data entry site via web cDDP 80 mg/m2 4 Cycles cDDP 2 Cycles cDDP 80 mg/m2 STS: 10-20 gm/m2 depending on age/weight 4 Cycles cDDP + STS 2 Cycles 80% power to detect 60% vs 35% hearing loss Two interim and one final efficacy analyses planned for early stopping in case of a greater than expected difference between treatment arms in terms of hearing loss

  17. STS: Development Timeline

  18. STS Market Opportunity Pediatric Market Opportunity 12,000 children develop cancer in the US every year 2,000 children will receive platinum-based chemotherapy, 3x ROW Pricing to be determined based on available therapies in the market Competitive Position Significance of injury increases value of STS Limited competition – hearing aids and cochlear implants do not prevent hearing loss Hearing aids cost $2000 to $6000 each Cochlear implants cost up to $75,000 each Third party market research shows strong adoption characteristics* Physician approval very high Payors feedback positive *Campbell Alliance and Panel Intelligence market research, analysis and surveys

  19. Summary STS has Two Potential Near Term Value Drivers Positive data from COG Phase III trial will support filing of NDA Upon approval of NDA, receipt of Rare Pediatric Disease Voucher Attractive Commercial Market Opportiunity 2,000 patients in the US (3x ROW), limited competition Third party market research shows potential for strong adoption Eniluracilis active and well tolerated Indications where 5-FU is administered potentially in excess of $1 BLN market Seek partnership for further development of Eniluracil

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