2011 Bio International Convention


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2011 Bio International Convention

  1. 1. Apricus Biosciences, Inc. Corporate presentation 2011 Bio International Convention, June 27 ‐30, Washington, DC1
  2. 2. Safe‐Harbor StatementStatements under the Private Securities Litigation Reform Act, as amended: With the  exception  of  the  historical  information  contained  in  this  presentation,  the matters  described  herein  contain  forward‐looking  statements  that  involve  risks and  uncertainties  that  may  individually,  mutually,  or  materially impact  the matters  herein  described,  including,  but  not  limited  to,  the  Company’s  ability  to execute  its  business  plan,  obtain  regulatory  approval  for  products  under development,  enter  into  partnering  agreements,  realize  revenue  and  pursue growth  opportunities,  some  of    which  are  outside  the  control  of  the  Company. Attendees  are  cautioned  not  to  place  undue  reliance  on  these  forward‐looking statements  as  actual  results  could  differ  materially  from  the  forward‐looking statements  contained  herein.  Attendees  are  urged  to  read  the  risk  factors  set forth  in  the  Company’s  most  recent  annual  report  on  Form  10‐K,  subsequent quarterly  reports  filed  on  Form  10‐Q  and  its  most  recent  SEC  filings.    Company disclaims any intention to update this presentation.2
  3. 3. Financial Snapshot• NASDAQ:  APRI• Shares Outstanding 19.6M* • Shares Fully‐diluted 22M* • Shares in the float 16M*• Cash‐position $10.2M*• Share‐price  $5.26**• Marketcap ~$105M**• Average Daily volume ~320k**• Revenues 2010 ~$5M *As of March 31,  2011 **As of June 24,  20113
  4. 4. Company Highlights• Specialty Biopharmaceuticals company with proprietary drug delivery platform  technology (NexACT®) to rapidly advance drug candidates through clinical  development• Mid‐to‐late staged pipeline with multiple, significant near‐term value drivers• Multiple Partnerships in place with additional significant partnerships expected  near term• Experienced management team, proven to deliver on milestones and objectives• Solid financial position: Current cash position through H2 2012 with goal to be cash  flow positive exiting 20114
  5. 5. NexACT®: Multi‐Route Drug Delivery Technology• Patented:  NCE  patents  based  on  proprietary  permeation  enhancers  that  are  biodegradable, biocompatible, non‐toxic ingredients that mimic the composition of  human skin and tissues.• Effective: enables rapid absorption of high concentrations of drug directly to target  site or systemically into blood stream.• Safe: excellent pre‐clinical and clinical safety dossiers through thousands of patient  exposures• Versatile: effective with wide range of drugs classes and different routes  • Small molecules, peptides, proteins, SiRNA, anti‐sense, and antibodies • Transdermal, Oral, Sub‐Q, Buccal, Rectal, Nasal, Ophthalmic5
  6. 6. NexACT® (DDAIP) MOA‐Loosening Tight Junctions6
  7. 7. Apricus Bio Product Pipeline7
  8. 8. Vitaros®(alprostadil/DDAIP) for the Treatment of Erectile Dysfunction• PGE1, potent vasodilator, topical cream, high viscosity • Only approved ED drug for all patients• Rapid onset (generally 6‐30 minutes) • Significant efficacy, including difficult to treat populations • Diabetics • Hypertensives • Patients with cardiac issues • Patients on nitrates and alpha blockers • Prostatectomy patients  • Sildenafil (Viagra®) failures• Side effects are generally mild, transient and topically related• Studied in over 3,300 patients8
  9. 9. Vitaros® Key Conclusion: Vitaros® 300 mcgs/100 ml dose strength is comparable to Viagra® 50 mgs dose strengthPhase 3 Pivotal Clinical StudiesIntegrated Efficacy Analysis – Intent to Treat Population Global Assessment Question When using the study medication, did you feel your erections improved? Vitaros® Viagra® p<0.001 50 mgs and 100 mgs Vitaros® p<0.001 Vitaros® p<0.001 Placebo (Vitaros® study) Placebo (Viagra® study) Vitaros® Studies Vitaros Vitaros Vitaros Vitaros N=394 N=408 N=392 N=398 Viagra® Studies Source: Viagra PI; Patients were started on 50 mg and allowed to adjust the dose up to 100 mg or down to 25 mg of VIAGRA; all patients, however, were receiving 50 mg or 100 mg at the end of the study. On a global improvement question, 57% of VIAGRA patients reported improved erections versus 10% on placebo. 9
  10. 10. Vitaros®(alprostadil/DDAIP) for the Treatment of Erectile Dysfunction10
  11. 11. Vitaros® Discontinuation Due to Serious Adverse Events: Orals vs. Vitaros® % of Patients Discontinuation Rhinitis/Nasal Drug Rate Headaches Flushing Congestions Back Pain Dyspepsia Abnormal VisionVitaros® 0 0 0 0 0 0 0 Viagra® 2 16 10 7 >2 7 11 Cialis® 3.1 15 3 3 6 10Levitra® 3.4 15 11 3 2 5 Key Conclusions:•Vitaros® presents an excellent safety and tolerability profile • No serious side effects • Most adverse events were localized to the site of application but were mild and short in durationSource: Vitaros PIII Clinical Trial; PDE5 Package Inserts11
  12. 12. Vitaros®(alprostadil/DDAIP) for the Treatment of Erectile Dysfunction12
  13. 13. Femprox®(alprostadil/DDAIP) for the Treatment of Female Sexual Arousal Disorder (FSAD)Premeasured unit dose 225 mg of cream containing  0.4 % alprostadil (900 µg), DDAIP 0.5%13
  14. 14. Femprox® Phase 3 Study• A Randomized, Placebo‐Controlled, Double‐Blind, Parallel Design Study of the  Efficacy and Safety of Alprostadil Cream in Patients with Female Sexual Arousal  Disorder (FSAD) o n= 400 patients placebo, 500, 700 or 900 mcg alprostadil cream groups, Application sites:  clitoris and G‐spot o Five (5) month study 14
  15. 15. Femprox® Phase 3 Study Safety and Tolerability • The most frequently reported adverse events were mild to moderate local irritations, and were 14%, 22%, 18% and 31% observed for the placebo, 500mcg, 700 mcg and 900 mcg groups, respectively. • No serious adverse events were reported. • Overall, 5 patients (1.2%) were withdrawn from the study because of adverse events.15
  16. 16. Femprox® Clinical/ Regulatory Strategy Next Steps Tasks Schedule 1. Establishment of US and EU Clinical Advisory Board • Help design of confirmatory Phase 3 trial required by FDA Q2, 2011 2. Submission of briefing books for health authority interactions aimed at: • Approvability of successful single Phase 3 trial 2 H, 2011 in Europa, Canada and Switzerland • Type A FDA meeting to agree on regulatory path forward for NDA. 3. Efforts to engage a pharmaceutical partner to continue 2 H, 2011 clinical development16
  17. 17. MycoVa™(Terbinafine/DDAIP) for the Treatment of Onychomycosis•Synthetic allylamine derivative which inhibits enzyme squalene epoxide in fungal cell•DDAIP ‐ significant drug penetration through nail plate to bed and surrounding area•Formulations advantages • Drug availability • Not trapped in lacquer matrix • Easily treat adjacent skin and folds • Patient convenience • Ease of application, quick drying MycoVa™ • Wash off, no lacquer removal•Clinical Studies in ~900 patients • 2 Phase 3 trials completed in US, EU and Canada • 1 EU comparator trial vs. Loceryl® (amorolfine) 17 10    June‐8‐2011
  18. 18. MycoVa™ ‐ Approval Path for Europe EU Regulatory Strategy: • Switch from superiority claim to non‐inferiority claim based on N2303 data  and reanalysis. • Obtain scientific advice from European health authorities. • Partnering attempts prior to market authorization. Clinical Studies conducted in more than 2000 patients world‐wide:  • China Proof of Concept Study – High clinical and mycologucal cure rate  • US Phase 1 (Safety and PK) – Relevant concentrations in nail clippings • US, Canada and EU Phase 3 (N2301 and N2302) – Primary Endpoint not met • Phase 3 comparative trial (N2303) vs amorolfine (Loceryl) – no difference • Total patients treated > 2,00018
  19. 19. EU Trial‐Non‐Inferiority Analysis Terbinafine is non‐inferior to amorolfine Mycological Cure Rates at the End of Study (ITT Population, LOCF) Mycological Cure Rate, Chi-square 95% n (%) Confidence Interval Difference Terbinafine Amorolfine Continuity (N=507) (N=522) Uncorrected Corrected 82 (16.17) 82 (15.71) 0.46 -4.01, 4.94 -4.20, 5.1319
  20. 20. MycoVa™‐ Approval Path for US and RoWNew Clinical Data Analysis• Combined analysis of N2301 and N2302 show significant efficacy vs. placebo  in mycological cure rate• Revised analysis of N2301 and N2302 without comorbid tinea pedis shows  statistically significant superiority vs. placebo, especially at later stages of the studyUS/ROW Regulatory Strategy:• Currently requesting guidance from Health Canada and FDA to achieve approval as antifungal with “mycological cure rate” as relevant endpoint 20
  21. 21. New Analyses: Combined Phase 3 studies N2301 and  N2302   Mycological cure in combined analysis Revised analysis on mycological cure rate = negative KOH microscopy & dermatophytes negative  excluding patients with concomitant tinea pedis culture Proportion Difference P< 0.0001Treatment n p-value† P=0.0003 (%) (95% CI)*Terbinafine24w 33 12.74 P=0.0058 n=174(N=259) 6.54 0.0141Vehicle ( 1.52, 11.55)24w 16 6.20 n=175(N=258)Terbinafine48w 51 18.82(N=271) 13.35 <0.0001Vehicle ( 7.93, 18.77)48w 14 5.47(N=256) [*] Difference is terbinafine minus vehicle. Two-sided 95% CI of difference is based on the normal approximation to the binomial. Mycological Cure is significantly greater in patients [†] p-value given by the normal inverse combination test. Studies included: [Study N2301] and [Study N2302] without comorbid tinea pedis (TP) treated with MycoVa™ ITT population, LOCF, at the end of the study (week 52) over 48 weeks than those receiving placebo at later Source: Table 3.2-4 – CSFO327SCE – Combined data stages of the study extending through to week 52.21
  22. 22. MycoVa™ mycological cure rates in perspective with  Penlac® dataIn the ITT analyses of Phase 3 studiesterbinafine-DDAIP/HCl shows statistically significant *mycological cure compared with placebo, slightly higher than in pivotal ciclopirox studies.Combined analysis excluding patients with tinea pedis increases the efficacy margin vs. placebo. Mycological Cure = negative KOH microscopy & dermatophytes negative culture * * * n.s . * * * n=110 n=223 n=237 n=264 n=263 n=527 n=349 Source: FDA Medical Review of ciclopirox Source: Nexmed Clinical Study Reports for terbinafine-DDAIP/HCl 22 Apricus Bio (NexMed USAs NexACT Technology) 7/29/2011
  23. 23. Market Opportunity• Multifunctional NexACT® Small Molecule Platform Targets Over $10 Billion in approved and  Late‐Stage Product Opportunities• Vitaros® For erectile dysfunction. Approved in Canada. NDA filed in US and  Europe.  Worldwide market over $4 Billion• Femprox® For FSAD. One successful Phase III. Awaiting guidance for filing in Europe  and Canada. Preparing for US Phase III for NDA filing.  Worldwide market  estimated to be up to $4 Billion• MycoVa™ For Onychomycosis. Europe Comparator Phase III trial competed. Awaiting  guidance for filing in Europe and Canada. Worldwide  market over $1 Billion• PrevOnco™ For liver cancer (HCC).  In Phase II  and heading to Phase III. Worldwide  market over $1 Billion (Partial Pipeline)23
  24. 24. Milestones Achieved Had first drug Vitaros® for erectile dysfunction approved by Health Canada Strengthened financial foundation of the company  Cash into H2 2012 Completed first partnerships for Vitaros® Bracco‐Vitaros® (Italy) Elis‐Vitaros® (Middle East) Neopharm‐Vitaros® (Israel) Unqualified audit opinion (Going Concern removed for the 1st time in 9 years) Filed for European Approval for Vitaros® in Q2 2011 Last Financing October 2010 ~$9M at $1.83 Returned on shareholder value Up over 50% YTD24
  25. 25. Near Term Upcoming Milestones Announce additional ex‐US commercial partnerships for Vitaros®:  Canada Europe Africa Latin America Announce clinical development and regulatory milestones  Goal to be cash‐flow positive by the end of 2011 through upfront payments from partnership agreements Commence sales of Vitaros® in Canada25
  26. 26. Upcoming Milestones Announce first NexACT® technology licensing deal File for marketing approval in Canada and Europe depending on regulatory  guidance for MycoVa™ Femprox® Out‐license other late‐stage clinical products Femprox® MyCova™ PrevOnco™26
  27. 27. Commercial Partners for Vitaros® USA Middle East & Gulf Israel Italy27
  28. 28. Proven & Experienced Management Team• President & CEO‐ Dr. Bassam Damaj  • Pfizer,  Genentech  (now  Roche),  Pharmacopeia  (now  Ligand),  Tanabe  Seiyaku  (now  Mitsubishi‐Tanabe), Bio‐Quant, Celltek, R&D Healthcare• Strong Finance, Operations, and Legal Team • Steve Martin‐ Gen‐Probe, Stratagene (now Agilent) • Edward Cox‐ Bio‐Quant, NexMed • Randy Berholtz‐ Nanogen, ACON Labs• Proven Senior Business Development Team  • Mark Wilson‐ Pfizer, Halozyme (Technology) • Linda Smibert‐ BMS, AstraZeneca, Santarus (Products)• Experienced Research & Development and Medical/Regulatory Affairs Team • Daniel Frank ‐ Wyeth, Pfizer • Dr. Mohamed Hachicha ‐ Forest Labs, Purdue Pharma • Dr. Richard Martin ‐ Exelixis • Dr.  Joachim P.H. Schupp – Ciba‐Geigy, Novartis28
  29. 29. In Summary• Approved drug (Vitaros®) and clinically validated drug delivery technology• Commercial partnerships in place and expanding• Efficacy and Safety of DDAIP as topical drug vehicle established thousands  of patients• Clinical  and  Regulatory  Strategy  to  drive  projects  in  place  and developing• Unique,  patented and versatile  technology  that  can  be  partnered  multiple  times to multiple partners & used to develop multiple drugs• Revenue‐generating with current cash reserves into H2 201229