Corporate Overview May 8, 2014

Transcription

1 0 Corporate Overview May 8, 2014 NASDAQ: CRIS

2 Forward Looking Statements This presentation contains statements about Curis future expectations, plans and prospects that constitute forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of Actual results or events could differ materially from the expectations, plans and prospects disclosed in the forward-looking statements that the Company makes due to a number of important factors, including risks related to: the Company s expectations for the timing of conducting ongoing and planned clinical studies with CUDC-427 and CUDC-907, as well as the potential benefits and safety of these drug candidates; Genentech and Roche s ability to successfully develop and commercialize Erivedge; the Company s and its collaborators ability to successfully research, obtain regulatory approvals for, develop and commercialize products based upon the Company s technologies; the Erivedge royalty-collateralized loan transaction entered into by the Company s wholly-owned subsidiary, including the risk that it may not receive sufficient levels of royalty revenue from sales of Erivedge to satisfy the debt obligation or may otherwise lose its rights to royalties and royalty related payments as a result of a foreclosure of the loan; the Company s ability to obtain and maintain proprietary protection for its technologies and product candidates; competitive pressures; the Company s ability to raise additional funds to finance its operations; and those risk factors discussed in the Company s Annual Report on Form 10-K for the year ended December 31, 2013, and other reports that it files with SEC. The forward-looking statements included in this presentation represent our views as of the date of this presentation. We anticipate that subsequent events and developments will cause our views to change. While we may elect to update these forward-looking statements in the future, we specifically disclaim any obligation to do so. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this presentation. 1

3 CURIS: Approved Drug with Royalty Revenues, Proprietary Oncology Pipeline & Strong Team Erivedge Roche/Genentech partnership Approved for advanced BCC in nearly 50 countries Anticipated sales growth in US and now EU, royalty revenue to Curis CUDC-907 proprietary oral drug candidate First dual inhibitor of HDAC and PI3K in clinical development Phase 1 ongoing in lymphomas and multiple myeloma 29 patients to date Expansion stage in DLBCL, multiple myeloma and others in 2H this year CUDC-427 proprietary oral drug candidate Most potent oral IAP antagonist in the field, and sustained dosing ability Clinical trials back on track for mono- and combination therapy Strong clinical and corporate development teams built Professional product development team brought in-house Poised for execution and strategic growth 2

4 Pipeline Novel, Targeted Cancer Therapeutics Program Target Indication Preclinical Phase 1 Phase 2 Phase 3 Market Proprietary Programs CUDC-907 HDAC/PI3K Lymphomas and Multiple Myeloma Phase 1 CUDC-427 IAP Breast, Ovarian, Lymphomas, and Other Cancers Phase 1 Phase 1 Partnered Programs Erivedge Hedgehog Advanced Basal Cell Carcinoma AML and MDS Approved in US, AUS, EU & Others Phase Phase 1/22 Debio 0932 Hsp90 NSCLC Renal Cancer Phase 1/2 Phase 1 Phase 1b/2 Phase 1/2 3

5 Erivedge Roche/Genentech partnership Partnership established in 2003, drug approved for BCC in U.S. in 2012 Roche and Genentech executing worldwide commercialization US and European sales Year-over-year total sales growth Anticipated sales in additional territories Royalty revenues to Curis on global sales Patent protection to 2028 provides revenues for years to come Tiered, escalating sales royalty starting at mid-single digit Continued Roche/Genentech investment in additional indications AML / MDS trial initiated in 2013 Trials in earlier forms of BCC 4

6 CUDC-907: First-in-Class, Potent Inhibitor of HDAC and PI3K Phase 1: Lymphoma & Multiple Myeloma 5

7 CUDC-907 Preclinical Profile Dual inhibition affects multiple signaling pathways Dual HDAC and PI3K enzyme inhibition in one oral small molecule Affects PI3K, JAK/STAT, and MAPK signaling pathways Affects tumor cell, as well as tumor microenvironment Control Pictilisib Panobinostat CUDC-907 RTK R HDACi R PI3Ki R Ac-H3 Ac-Tub Ac-p53 p53 p-akt p-4ebp1 JAK STAT RAS RAF MEK PI3K AKT PTEN mtor Tubulin p-stat3 (S727) p-stat3 (Y705) STAT3 S6K ERK Proliferation & Survival 6

8 CUDC-907 Clinical Target Modulation HDAC & PI3K inhibition in patient PBMCs *** ** * Ac-H3 (% Control) HDAC inhibition *** *** pras40 PI3K inhibition Day post-treatment * p-value < 0.05 ** p-value < 0.01 *** p-value <

9 HDAC & PI3K Inhibition Affect Tumor Cell and Microenvironment Multiple cytokines/ chemokines produced by cancer cells or tumor microenvironment are modulated by HDAC and PI3K inhibitors Drug activity likely mediated through effects on STAT proteins Produced by tumor HDACi CCL3 CCL4 CCL22 TARC PI3Ki Produced by microenvironment HDACi TARC CD40L CXCL12 CXCL13 CCL7 IL6 IL10 TNFa BAFF PI3Ki 8

10 CUDC-907 Reduces TARC Expression - Preclinical RPMI-8226: multiple myeloma cell line CUDC-907 panobinostat idelalisib CUDC-907 panobinostat idelalisib 9

11 CUDC-907 Trial, Clinical TARC Levels Correlative trend between plasma TARC levels & clinical effect (per Revised Response Criteria for Malignant Lymphoma) Preliminary assessment of multiple cytokine and chemokines in 11 response-evaluable patients Potential for predictive value of either baseline or post-treatment change in chemokine levels 10

12 CUDC-907 Phase 1 Clinical Update Dose escalation continuing with rapid patient enrollment at all 3 centers (MSKCC, MD Anderson, Sarah Cannon) 2x/ week and 3x/week intermittent dosing schedules continuing 29 patients treated to date Currently completing 120 mg dose cohorts Safety No DLTs thus far using intermittent dosing No consistent serious AEs emerging at current doses PK / PD Increased drug exposure with dose escalation Target modulation and cytokine level changes observed in patient samples Efficacy update to be presented at scientific conference 11

13 CUDC-907 Summary Oral, highly potent & selective dual inhibitor of HDAC and PI3K-α, δ & β Preclinical highlights Inhibits PI3K, as well as JAK/STAT and MAPK pathways Affects tumor cells as well as microenvironment In vivo activity in DLBCL and multiple myeloma xenograft models Clinical highlights 29 patients treated to-date, dose escalation continuing, intermittent dosing Target modulation & effect on cytokine levels observed Preliminary evidence of anti-tumor activity - PR and SD (ASH 2013) Development plan Phase 1 expansion cohorts in 2H 2014: DLBCL and multiple myeloma likely Projecting Phase 2 plans in heme cancer: early 2015 Exploring trial in solid tumor indication subset of breast cancer patients 12

14 CUDC-427: Potent Antagonist of IAP (Inhibitor of Apoptosis) Proteins Phase 1: Solid Tumors or Lymphoma 13

15 CUDC-427 and Apoptosis Induction Oral small molecule drug Monovalent SMAC mimetic Synergy with anti-cancer agents: Chemotherapy, targeted therapies and TNF 14

16 CUDC-427 Activates Caspases 15

17 CUDC-427 is Active as Monotherapy and in Combination In Vivo Preclinical Profile Vehicle CUDC-427 Breast cancer model 5FU CUDC-427 Breast cancer model Combo Lymphoma model Docetaxel CUDC-427 Breast cancer model Combo 16

18 CUDC-427 Monotherapy Clinical Activity Complete response cases Two patients had complete response MALT lymphoma (300 mg): CR by PET let in Cycle 5 (off study patient choice) Ovarian cancer (450 mg): CR end of Cycle 2 (off study due to AE, rash) Sustained CR per imaging Feb 2013 April 18, 2012 July 31,

19 CUDC-427 Clinical Development Status Genentech-initiated dose escalation Phase 1 study completed 2 patients with unconfirmed complete responses (ucr) 4 patients with SD 3 months Monotherapy treatment plan Re-initiation of enrollment in Phase 1 trial expected shortly Once daily doses of 100mg 300mg; 14 days on / 7 days off schedule Expansion stage to enroll ovarian and lymphoma patients, including MALT lymphoma, and patients with potential IAP pathway genetic alterations Phase 2 initiation based on clinical results Combination treatment plan Phase 1b study in combination with standard-of-care chemotherapy agents (including capecitabine and taxanes in parallel cohorts), initiation expected 2H 2014 Phase 2 randomized portion, initiation expected in

20 CUDC-427 Summary Orally available and potent antagonist of IAP proteins Small molecule, monovalent SMAC mimetic drug candidate Preclinical efficacy and synergy with chemotherapy or TNF Phase 1 monotherapy studies 14 days on/ 7 days off treatment schedule well tolerated Clinical benefit observed in a patient with IAP pathway genetic alteration MALT lymphoma patient amplification of ciap2 gene Patient enrollment re-initiating expected shortly Combination therapy Combination trial planned with standard-of-care chemotherapy agents (including capecitabine and taxanes): 2H 2014 Additional combination trials projected based on preclinical data 19

21 Partnered Programs Erivedge First-in-class Hedgehog Pathway Inhibitor Debio 0932 Oral HSP90 Inhibitor Ph 1/2 : NSCLC, Ph 1: RCC 20

22 Erivedge Developed Under Collaboration Genentech and Roche Erivedge (vismodegib) Oral inhibitor of Hedgehog signaling pathway Discovered under broad pathway collaboration formed in 2003 Approved for treatment of advanced basal cell carcinoma (BCC) Genentech/Roche-sponsored trial ongoing in AML and MDS Phase 1b/2 trial initiated in Q4 2013; 60 patients as monotherapy and combination with cytarabine Milestone and royalty-based revenue stream for Curis Potential for $115 million in development & regulatory milestones $56 million received to-date Royalties on global net sales 21

23 Erivedge Launched in Multiple Countries Genentech and Roche Marketed in the U.S. for the treatment of advanced BCC Roche reported ~ $27 million net sales for first quarter 2014 Includes ~$11.1 million in ex-us sales Anticipate continued sales growth in 2014 Expand global marketing Roche Approved in U.S., Australia, European Union and multiple other countries Reimbursement discussions ongoing in Ex-US territories Filed with health authorities in many other countries 22

24 Debio 0932 Oral small molecule Hsp90 inhibitor Second generation oral, small molecule Hsp90 inhibitor Exclusive WW license with Debiopharm $90 million in potential payments ($13 million received to-date) Royalties on net sales; share of certain sublicensing payments Clinical development status Debiopharm Phase 1 portion of Phase 1/2 HALO study in NSCLC patients ongoing In combination with chemotherapy: front line and previously treated patients Phase 2 portion expected to initiate in 2014 (milestone payment) Phase 1 study in renal cell carcinoma ongoing (initiated in 2013) In combination with everolimus 23

25 Summary Financial Data March 31, 2014 (000 s) Cash, cash equivalents, investments $ 63,800 Erivedge secured debt (non-recourse to Curis) $ 30,200 Basic shares outstanding 85,900 Fully-diluted shares outstanding (1) 98,700 YTD net loss through March 31, 2014 $ (5,600) (1) Comprised of: (i) 85.9 million shares outstanding, (ii) warrants to purchase 1.4 million shares at $3.55 per share, and (iii) options to purchase 11.3 million shares at a weighted average exercise price of ~$2.80 per share. 24

26 NASDAQ: CRIS 25