Cell Therapeutics

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Cell Therapeutics

  1. 1. Investor Update esto Charting the path to profitability James A Bi J A. Bianco, M D M.D. Chief Executive Officer
  2. 2. Forward Looking Statement This presentation contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. The forward-looking statements contained in this presentation include statements about future financial and operating results and risks and uncertainties that could affect CTI’s product and results, CTI s products under development.These statements are based on management’s current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. These statements are not guarantees of future performance, involve certain risks, uncertainties and assumptions that are difficult to predict, and are based upon assumptions as to future events that may not prove accurate. Therefore, actual outcomes and results may differ materially from what is expressed h i I any t t Th f t l t d lt diff t i ll f h ti d herein. In forward-looking statement in which CTI expresses an expectation or belief as to future results, such expectation or belief is expressed in good faith and believed to have a reasonable basis, but there can be no assurance that the statement or expectation or belief will result or be achieved or accomplished. The following factors, among others, could cause actual results to differ materially from those described in the forward factors others forward- looking statements: risks associated with preclinical, clinical and sales and marketing developments in the biopharmaceutical industry in general and in particular including, without limitation, the potential failure of Opaxio™ to prove safe and effective for treatment of non-small cell lung and ovarian cancers, the potential failure of Pixuvri (pixantrone dimaleate) to prove safe and effective (including complete and overall response rates) for treatment of non- Hodgkin’s lymphoma, determinations by regulatory, patent and administrative governmental authorities, competitive factors, technological developments, costs of developing, producing and selling CTI’s products under development; and other economic, business, competitive, and/or regulatory factors affecting CTI’s business generally, including those set forth in CTI’s filings with the SEC, including its Annual Report on Form 10-K for its most recent fiscal year and its most recent Quarterly Report o Form 10-Q, espec a y in the “Factors Affecting Ou Ope at g Results” a d “Management’s ece t Qua te y epo t on o 0 Q, especially t e acto s ect g Our Operating esu ts and a age e t s Discussion and Analysis of Financial Condition and Results of Operations” sections, and its Current Reports on Form 8- K. Except as may be required by law, CTI does not intend to update or alter its forward-looking statements whether as a result of new information, future events, or otherwise.
  3. 3. Overview Focused on Profitability Successfully turning around the business Reduced Opex 72% y/y while advancing key assets toward market Raised $68mm YTD R i d $68 $31.5mm without selling stock (Zevalin asset sale) Removed $52.9mm of debt increased shareholder equity by$43.7mm q y y Listed on Russell 3000, Russell 2000 and Russell Global Indexes Positioned to break even in Q4 2009 attractive EPS growth 2010 Successful Pixuvri phase III trial, NDA submission and product launch will drive near term value Attractive Oncology Pipeline Management team experienced in oncology product sales
  4. 4. Building a Powerful Cancer Drug Portfolio Phase Preclinical Phase I Phase II Marketed III NDA review i NP sales EU NSCL Lung cancer MAA review 1° line Ovarain cancer Phase 3 Brostallicin Bisplatinates NP sales = named patient sales
  5. 5. Charting the Path to Profitability ands) ($ thousa $
  6. 6. Charting the Path to Profitability ands) ($ thousa $
  7. 7. Charting the Path to Profitability Cleaned up Capital Structure • Retired all preferred; only common stock & warrants to purchase p ; y p common stock outstanding Cleaning up Balance Sheet • Removed $52.9 mm ($1.89/share) of debt increased shareholder $52 9 ($1 89/share) equity by $43.7 million Increasing Shareholder Equity* Historical Proforma Proforma** Accumulated Deficit $1,325,444 $1,318,342 CTI Total Shareholder Deficit <$115,984> <$54,131> Total Liabilities & Shareholder Deficit $42,933 $54,572 *All values thousands **Proforma based on unaudited financial data for quarter ended March 31, 2009 adjusted for sale of shares in May 2009 and debt redemption.
  8. 8. Stock Price and Market Cap Appreciation Cell Therapuetics Inc. (Mercato Closing Price) *expressed in Euros € 1.40 € 1.20 Mercato Closing Price € 1.00 g € 0.80 6/2/2009 1.301 Euros (A.H.) € 0.60 € 0.40 1/23/2009 0.04 Euros € 0.20 (A.L.) € 0.00 Cell Therapeutics Inc. (Market Cap.) *expressed in Euros € 700,000,000.00 € 650,000,000.00 6/2/2009 € 600,000,000.00 650.5 m illion € 550,000,000.00 Euros ation € 500,000,000.00 Market Capitaliza € 450 000 000 00 450,000,000.00 € 400,000,000.00 € 350,000,000.00 € 300,000,000.00 € 250,000,000.00 1/23/2009 € 200,000,000.00 20.0 m illion € 150,000,000.00 Euros € 100,000,000.00 € 50,000,000.00 € 0.00 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 3/ 9/ 4/ 0/ 3/ 7/ 5/ 1/ 7/ 4/ 0/ 3/ 9/ 7/ 3/ 9/ 6/ 2/ 8/ 2/ 8/ 3/ 9/ 5/ 9/ 5/ /2 /2 /0 /1 /2 /2 /0 /1 /1 /2 /3 /0 /0 /1 /2 /2 /0 /1 /1 /2 /2 /0 /0 /1 /1 /2 01 01 02 02 02 02 03 03 03 03 03 04 04 04 04 04 05 05 05 05 05 06 06 06 06 06
  9. 9. Cell Therapuetics Inc. (NASDAQ Closing Price) $2.50 6/1/2009 $2.09 (YTD High) $2.00 rice asdaq Closing Pr $1.50 $1.00 1/23/2009 Na $0.50 $0.05 (YTD Low) $0.00 Cell Therapeutics Inc. (Market Capitalization) $1,050,000,000.00 $1,000,000,000.00 6/1/2009 $950,000,000.00 $1.049 billion $900,000,000.00 $900 000 000 00 (YTD High) $850,000,000.00 $800,000,000.00 Market Capitalization $750,000,000.00 $700,000,000.00 $650,000,000.00 $600,000,000.00 $550,000,000.00 $500,000,000.00 $450,000,000.00 $400,000,000.00 $350,000,000.00 $300,000,000.00 1/23/2009 $250,000,000.00 $200,000,000.00 $20.0 m illion $150,000,000.00 (YTD Low ) $100,000,000.00 $50,000,000.00 $0.00 08 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 09 1/ 7/ 3/ 0/ 6/ 0/ 5/ 8/ 4/ 2/ 6/ 2/ 8/ 4/ 0/ 3/ 9/ 6/ 2/ 8/ 4/ 8/ 4/ 0/ 7/ 2/ 8/ 2/ 8/ 4/ 0/ /3 /0 /1 /2 /2 /3 /0 /1 /2 /0 /0 /1 /1 /2 /3 /0 /0 /1 /2 /2 /0 /0 /1 /2 /2 /0 /0 /1 /1 /2 /3 12 01 01 01 01 01 02 02 02 03 03 03 03 03 03 04 04 04 04 04 05 05 05 05 05 06 06 06 06 06 06
  10. 10. CTI’s Successful Cancer Treatments Highly effective therapy for first and subsequent relapse in acute promyelocytic leukemia (APL) Excellent Return on Investment • Purchased in 2000 for $12mm in stock • Stock price appreciation 5000% ($1.50 to $77.00) • Peak sales $22mm • Sold to Cephalon in 2005 for $70mm cash Highly effective therapy for patients with relapsed or refractory, low-grade non-Hodgkin's lymphoma (NHL) Excellent Return on Investment • Purchased from Biogen-Idec $10mm December 2007 • Sold to Spectrum in March 2009 $31.5mm
  11. 11. CTI’s Successful Cancer Treatments Highly effective therapy for patients with relapsed aggressive NHL Pixuvri History • Discovered by Novuspharma, acquired by CTI in January 2004 y p q y y Patient benefit • Initial Label Indication - 9,760 US patients + 6,832 EU patients • Label expansion could reach additional 51,000 patients US/EU Potential to Take Company to Profitability • T t l Potential Market Size 66,592 patients/yr Total P t ti l M k t Si 66 592 ti t / • $44,000 per patient/yr
  12. 12. The superior choice for  Th i h i f an anthracycline an anthracycline in NHL Successful Phase III Trial results
  13. 13. Opportunity Assessment • Anthracyclines – 3rd most commonly used class anti- y y cancer agents • >300,000 pts receive an anthracycline each year • Potentially curative in – Lymphoma (NHL) – Leukemia ((AML) ) – Breast cancer – Sarcoma • Cornerstone class in 1st line treatment regimens
  14. 14. Anthracycline Use (US+EU) 231,417 302,000 Patients/year tients Pat 20,200 15,794 11,706 11,650 11,346 Source: Tandem Cancer Audit 2004
  15. 15. Aggressive NHL – Treatment Regimens (US data) Aggressive NHL – Anthracyclines Standard of Care in 1st line Treatment Standard of Care in 1 line Treatment 120,0% Other bortezomib 100,0% 12,3% R‐CNOP 0,8% CVP 1,4% 3,0% 0,4% 37,8% R‐CHOPM 80,0% 8,9% 45,7% lenalidomide 11,3% CAV 2,9% 1,7% 1,7% 60,0% 3,5% Treanda+ 1,1% 4,5% 3,4% 3,5% R‐ESHAP 5,2% 1,8% 5,4% RF 11,2% 1,8% 40,0% 40 0% 3,3% 60,9% 3,3% RICE 4,5% R‐CVP 25,2% 19,1% 20,0% R R + CHOP 6,3% 7,1% 0,0% 1st LOT 2nd LOT 3rd+ LOT 1st Line 2nd Line 3rd Line+ Patient # 30,029 10,150 9,767 ™ IntrinsiQ March 09
  16. 16. Heart Failure Limits Re‐treatment Heart Failure Limits Re‐ cidence of Percent (%) Inc ailure  f Heart Fa 16
  17. 17. Like cardiac toxicity, extravasation is a serious  concern with current anthracyclines concern with current anthracyclines
  18. 18. Next Generation Anthracycline  • Rationally Designed to Reduce Oxygen Radical Rationally Designed to Reduce Oxygen Radical  Generation – Significant reduction in cardiac damage in animal models Vs Significant reduction in cardiac damage in animal models Vs  current marketed anthracyclines – No extravasation concerns‐ peripheral vein infusion No extravasation concerns‐ peripheral vein infusion O OH O OH H NH 2 OH N O HN OH O HN OH COOH N O O OH O 2 HCl 2 O COOH HO H OH O HN OH O HN N NH 2 H H2N Doxorubicin Mitoxantrone pixantrone
  19. 19. PIX 301Phase III Study Design ‐ Randomized Controlled International Multicenter Phase III trial ‐ Relapsed or refractory aggressive NHL >2nd relapse ‐ 140 patients • Treatment every  month up to 6 cycles Primary Endpoint* Standard  • CT scan every 2 cycles CT scan every 2 cycles  Chemotherapy ‐ Complete Remission (CR/CRu) Complete Remission (CR/CRu) x 3 then every 3  months Secondary Endpoints* ‐ Overall Response Rate (ORR) ‐ Responses > 4months • Treatment every 21  ‐ Time to response days up to 6 cycles ‐ Progression Free Survival Pixantrone Pi t • CT CT scan every 2 cycles  2 l ‐ Safety x 3 then every 3  months *All response and progression data determined by IAP 19
  20. 20. PIX 301 Phase III Trial in Relapsed NHL p Intent to Treat :Primary endpoint achieved Pixantrone (n=70) Control (n=70) P‐value CR 11.4% 11 4% (8) 0% (0) CRu 8.6%  (6) 5.7% (4) CR/CRu 20.0% (14) 20.0% (14) 5.7% (4) 5.7% (4) 0.021 Intent to Treat : Secondary endpoint achieved Population Pixantrone Control P‐value ORR (PR+CR/CRu) 37% (26/70) 14.3% (10/70) 0.003
  21. 21. PIX 301 Phase III Trial in Relapsed NHL PIX 301 Phase III Trial in Relapsed NHL Pixantrone increased progression free survival > 80% (ITT) Median PFS 4.7 Vs 2.6 months HR 0.60 (p=0.007) Pixantrone Standard py Chemotherapy
  22. 22. Lifetime Anthracycline/Anthracenedione Exposure (Doxorubicin Equivalent Dose) (Doxorubicin Equivalent Dose) Treatment  Pixantrone Treatment  Lifetime Cycle Cumulative Dose* Cumulative Dose** N Median  (mg/m2) Median  (mg/m2) 1 68 74 365.2 2 54 148 430.2 3 43 209 498.1 498 1 4 36 275 571.8 5 25 356 631.5 631 5 6 22 427 695.0 * Pixantrone dose converted to dox equivalent dose by factor 3.4 **  Dox equivalent dose based on  “P. McLaughlin JCO Vol14, No 4, 1996”
  23. 23. Encouraging Cardiac Safety Data Frequency % CHF Expected Vs Observed by Total Doxorubicin Equivalent Exposure Doxorubicin  Doxorubicin Doxorubicin Pixantrone Equivalent  N=620 N=70 Exposure (mg/m2) <300 5.6% 2/68 500  500 15% 2/43 600 32% 0/36 >600 48% 1/25 * Swain, et al. Cancer 2003; 97(11): 2869-2879; Sonnenveld et al. JCO 1995, 13:2530-2539 23
  24. 24. Summary & Implications PIX301 Phase III Results • Superior efficacy over standard of care  • First trial  in relapsed/refractory  aNHL to demonstrate • Superior CR rates 24% Vs. 7% p=0.009* • Superior ORR  40% Vs. 14% p=0.001* Superior ORR 40% Vs 14% p=0 001* • Superior PFS 4.7 months Vs. 2.6 months p=0.007 • Superior duration of CR  7 months Vs. 3.5 months p=0.03 • Unprecedented cardiac safety profile despite median dox‐ equivalent exposure of 515mg/m2 • Non‐dose dependant occurrence of CHF (5 Vs 2) drug relationship unlikely • Cumulative occurrence  7% Vs expected  25% to 48%  standard doxorubicin • Activity preserved even among  patients with extensive prior doxorubicin and  yp g p p rituximab treatment • Available for named patient sales in Europe *Includes both treatment and follow‐up periods *Includes both treatment and follow up periods ™
  25. 25. Commercial Opportunity
  26. 26. Commercial Analysis Assumptions • Pricing $24/mg – $1 200 vial, = average use 36 vials/patient $1,200 vial • Exclusivity in the market to 2018 (US) / 2020(EU) • Regulatory approval – aggressive relapsed NHL g y pp gg p • US Q4-2009, EU Q3-2010 Modeling assumptions (US only) CTI peak Market Research • O Only NHL is included NHL in model Peak • 1st line 30,029 patients Aggressive • 2nd line 10,150 patients 1st-line 15% 26% • 3rd line 9,767 patients 9 767 2nd-line line 20% 38% 3 rd-line+ 25% 36% Peak Penetration Indolent 1st-line 4% 27% • Assumes EU ~70% US market 2nd-line line 8% 34% 3 rd-line+ 8% 32% • 15,800 pts US +10,600 pts EU • Total peak patients/yr = 26,400 • Cost per patient/yr = $44,000 $44 000
  27. 27. Novartis – CTI Agreement(s) If NVS exercises its option for Pixantrone the License would have the following features • Reimburses CTI for 50% of expenses from 9/06* >$25,000,000 p $ , , • Pays CTI $7,500,000 option fee upon license execution • Pays $10,000,000 FDA approval milestone in 3rd line aNHL • Pays additional $94 000 000 in potential future registration & sales $94,000,000 milestones • Controls future development and commercialization of product & 100% future expenses f t • CTI may field 35 FTE in US at Novartis’ expense up to $9M • Pays CTI royalties 28% to 32% on net sales beyond $50 million *from date of execution of license agreement
  28. 28. A bioengineered version of Taxol®(paclitaxel) Singer et al. In: Adv Exp Med Biol. 2003; 519:81-99
  29. 29. PG Technology: Selective Delivery of Cytotoxic Drugs Macromolecules are preferentially distributed to and retained in tumor microvasculature “EPR” effect Enhanced permeability and bilit d retention They are then taken up into the cancer cell by a process called endocytosis, where they are metabolized releasing the chemotherapy
  30. 30. OPAXIO™ Paclitaxel 15–20 minute infusion 3 to 24 hour infusion
  31. 31. Opaxio Regulatory Status EU- 1st line single agent NSCLC, PS=2 MAA review nearing completion • Submission based off non-inferior survival, better side effects US/EU- 1st line maintenance in ovarian cancer Pivotal trial (GOG212) • Targeting interim PFS results early 2010: late 2010 NDA-approval • Market size ~15,000 patients/year ($32,000/patient) Additional Registration Trial- 2010 • Esophageal/Gastric- XRT sensitization • I Impressive rates of (45%) pathologic complete remission rates i f h l i l i i • Would be 1st radiation sensitizer approved in US/EU • Phase II-III to start Q1-2010 Q
  32. 32. Novartis–CTI Agreement(s) Worldwide license to OPAXIO • $75M in potential development expense reimbursement* • $ $270M in p potential registration and sales milestones g • CTI to field 35 FTE at NVS expense up to $9M • NVS to assume all development and commercial expenses • Royalties 20-25% on WW net sales *50% expenses from date of execution of license agreement
  33. 33. Capital Structure/Financials Dual Listed NASDAQ:CTIC, MTA:CTIC.MI • Li id stock ~50mm shares/day US/EU Liquid t k 50 h /d Cleaned up Capital Structure • Retired all preferred- only common stock outstanding preferred • 502mm shares outstanding Cleaning up Balance Sheet • Redeemed $$52.9mm debt@ $ $1.89/share • Returned $43.7 mm to shareholder equity Reduced OpEx to $2 5mm/month $2.5mm/month Cash end Q1- $750,000 • Raised ~$53mm April/May, $24mm non-stock based Listed on Russell Indexes
  34. 34. Upcoming Milestones Upcoming Milestones – 2H-09 • FDA acceptance NDA - grant priority (6month review) • EMEA opinion on OPAXIO MAA for lung cancer • Potential FDA approval • Potential Novartis option exercise • Up to $45mm in payments • Phase III results on OPAXIO 1st line Ovarian cancer ti l trial
  35. 35. Charting the Path to Profitability Closing Comments Successfully turning around the business without government bail out money! • Si ifi Significant cost reductions while advancing k assets t t t d ti hil d i key t toward d market • Smart negotiation of “Zevalin sale p right” to Spectrum Pharma g put g p • Provided attractive 215% 1 yr ROI ($21.5 million net of initial $10mm investment) • Raised $44 mm at increasing valuations limiting dilution valuations, • Raised $31.5mm without selling stock (Zevalin asset sale) • International peer reviewed acknowledgement of Pixuvri benefit to patients at Amercian Society of Clinical Oncology (ASCO) Meetings • Positioned to break even Q4 2009 and profitable in 2010
  36. 36. Resources Cell Therapeutics, Inc. (CTI) 501 Elliott Ave. W., Suite 400 Ave W Seattle, WA 98119 +1 206.282.7100 www.CellTherapeutics.com Investors Contact: CTI Barabino & Partners IR Elena Murador Omar Al Bayaty T +39 02 610 35 808 T +39 02.72.02.35.35 F +39 02 610 35 601 39 F +39 02.89.00.519 39 E: elena.murador@ctimilano.com E: o.albayaty@barabino.it www.CellTherapeutics.com/investors.htm For the latest financial information, including investor updates, SEC filings, press releases, and webcasts, please visit our Web site at www.CellTherapeutics.com. For more product and clinical trial information including references, clinical trial information, and product fact sheets for pixantrone, OPAXIO™, and brostallicin, visit our Web site, www.CellTherapeutics.com. © CTI 2009

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