IMMUNOMEDICS, INC. Advanced Antibody-Based Therapeutics. Jefferies 2015 Global Healthcare Conference Peter P. Pfreundschuh, VP Finance and CFO

Transcription

1 IMMUNOMEDICS, INC. Advanced Antibody-Based Therapeutics Oncology Autoimmune Diseases Jefferies 2015 Global Healthcare Conference Peter P. Pfreundschuh, VP Finance and CFO

2 Forward-Looking Statements This presentation, in addition to historical information, contains certain forward-looking statements made pursuant to the Private Securities Litigation Reform Act of Such statements may involve significant risks and uncertainties, and actual results could differ materially from those expressed or implied herein. Factors that could cause such differences include, but are not limited to, new product development (including clinical trials outcome and regulatory requirements/actions); competitive risks to marketed products; forecasts of future operating results; availability of required financing and other sources of funds on acceptable terms, if at all; as well as those discussed in the Company's filings with the Securities and Exchange Commission. 2

3 Highlights Multiple late-stage opportunities in underserved markets Lead candidate represents potential next generation Lupus treatment Cancer programs focused on unmet need in difficult-to-treat tumors Two Phase 3 candidates and two Phase 2 candidates Next 18 months could be transformational Epratuzumab pivotal Phase 3 topline results expected in 1H15 Initial IMMU-132 & IMMU-130 Phase 2 results very encouraging; evaluating Phase 3 strategy Supports antibody-drug conjugate (ADC) technology platform Valuable assets for partnering 90 Y-clivatuzumab tetraxetan Phase 3 trial in pancreatic cancer expected to complete patient enrollment in 2016 Potentially first self-commercialized product, if approved Several additional ADCs identified to continue pipeline progress 3

4 Broad Pipeline of Antibody-Based Therapies Research/Preclinical Phase 1 Phase 2 Phase 3 Epratuzumab (humanized anti-cd22) Systemic lupus erythematosus (SLE) Top-line data 1H15 90 Y-clivatuzumab tetraxetan 3 + -line advanced pancreatic cancer Complete Phase 3 enrollment in 2016 Antibody-drug conjugates IMMU-132: anti-trop-2-sn-38 for metastatic solid tumors IMMU-130: anti-ceacam5-sn-38 for mcrc Other product candidates Veltuzumab (anti-cd20) for cancer and autoimmune diseases Milatuzumab (anti-cd74) for cancer and autoimmune diseases Multiple compounds for cancer and autoimmune diseases 4

5 Lupus Market Opportunity A chronic autoimmune disease with no cure ~650,000 patients diagnosed with primary SLE (systemic lupus erythematosus) in the U.S. and E.U.* ~$1 billion estimated size in major pharmaceutical markets** Moderate to severe SLE represent ~70% of cases*** SLE is caused when a patient s immune system attacks normal, healthy tissue Can attack heart, joints, skin, lungs, liver, kidneys, and CNS Unexpected flares Most common in women under 35 Key market needs: 1. Control of flares to allow normal organ function and improve QoL 2. Reduction in use of corticosteroids and other drugs 3. Minimal side effects to allow chronic use 5 Sources: *NIH Diagnoses Data; **Systemic lupus erythematosus , Decision Resource, December 2014; ***New Harvard Guide to Women s Health 2004

6 Common Lupus Treatments Corticosteroids Immunosuppressive to help control flares Wide range of uses Benlysta First new lupus therapy in 50 years (approved 2011) Reduces number of flares Long-term effects of systemic immunosuppression, including increased potential for: Diabetes Obesity High blood pressure Cataracts Osteoporosis No reduction in use of corticosteroids Blackbox warning for mortality Contraindicated for pregnancy 6

7 Epratuzumab Phase 2 Summary Target doses demonstrated promising efficacy and safety Statistically significant higher responder rates vs. placebo (Total cycle = 2400mg) Safety profile similar to placebo Encouraging multi-year open-label extension experience Lupus disease activity maintained or further reduced Reduced dependence on corticosteroids No new safety signals observed in extended use Improved measures of health-related quality of life 7

8 How Epratuzumab Is Designed to Work Lupus is caused by B-cells producing auto-antibodies that attack normal tissue In Blood publication, epratuzumab was shown to reduce B cells by 30-40% and connect B cells with an effector cell Designed to cause the transfer of B-cell antigens (including CD22) to the effector cell The process is called trogocytosis or shaving Without CD22, the B cells die (apoptosis) 8

9 Randomization Phase 2b EMBLEM Study Design Double-blind, dose-ranging study in moderate to severe SLE Primary endpoint: Clinical activity response index at 12 weeks Index emphasizes BILAG; also includes SLEDAI, physician global assessment and treatment failure status Screening/ Lead-in Minimum disease activity: 1 organ BILAG A, or 2 organs BILAG B N = 227 Dose Arm 1: 600mg qw, wk: 0,1,2,3 (n=37) Dose Arm 2: 1200mg q2w, wk: 0,2 (n=37) Dose Arm 3: 100mg q2w, wk: 0,2 (n=39) Dose Arm 4: 400mg q2w, wk: 0,2 (n=38) Dose Arm 5: 1800mg q2w, wk: 0,2 (n=38) Placebo (n=38) Primary Endpoint Open-Label Extension (all pts: 1200mg q2w x2 in 12 week treatment cycles) Week Week 4 Week 8 Week 12 Treatment Phase 9 Wallace DJ, et al. Ann Rheum Dis Jan;73(1):183-90

10 Results Point to Optimal Doses Statistically significant reduction in lupus disease activity versus placebo at 12 weeks with 2400mg treatment cycle (600mg qw) Combined 2400mg cycle arms (600mg qw mg q2w) produced a 12 week response rate of 43.2% (p = 0.02) Response Rate at 12 Weeks Epratuzumab 600mg qw (n=37) Epratuzumab 1200mg q2w (n=37) Epratuzumab 100mg q2w (n=39) Epratuzumab 400mg q2w (n=38) Epratuzumab 1800mg q2w (n=38) Placebo (n=38) 30.8% 26.3% 23.7% 21.1% 40.5%, 45.9 %, p = 0.03 p = 0.07 Doses selected for Phase 3 (EMBODY) 10 Wallace DJ, et al. Ann Rheum Dis Jan;73(1):

11 Treatment Responses Improved Through OLE All open-label extension (OLE) patients received epratuzumab 1200mg q2w x2 in 12-week treatment cycles Number of responders observed by week: EMBLEM group OLE Screen Week 24 Week 48 Week 72 Week 96 Week 108 Placebo (n = 35) Epratuzumab (n = 168) Total (N = 203) No new safety signals were observed during the extension period Patients reported meaningful improvements in a number of health-related quality-of-life (HRQoL) measures 11 Presented at EULAR 2013 Congress

12 Reduced Steroid Use Median corticosteroid dose during EMBLEM open-label extension Patients using <7.5mg/day rose from 36.5% to 58.0% 12 Presented at EULAR 2013 Congress

13 Randomization Phase 3 Design (EMBODY 1 & 2) Two identical, double-blind, placebo-controlled registration studies in moderate to severe SLE Trial design highly similar to Phase 2b and its Open-Label Extension Trials Primary endpoint: Clinical activity response index at 48 weeks Trials are fully enrolled Top-line data expected 1H 2015 Screening/ Lead-in Minimum disease activity: 1 organ BILAG A, or 2 organs BILAG B N = 1,560 Arm 1: 600mg qw x4 per 12 week cycle Arm 2: 1200mg q2w x2 per 12 week cycle Placebo Primary Endpoint 13 Week -2 Cycle 1 Cycle 2 Cycle 3 Cycle 4 Week 48 (Week 0) (Week 12) (Week 24) (Week 36)

14 Commercialization Plan Partnered with UCB worldwide Clinical and commercial milestones of up to $300 million Escalating double-digit tiered royalty on sales (mid-teens to midtwenties) Development and commercialization funded by UCB Immunomedics retains global rights to all oncology indications UCB is a global leader in autoimmune and CNS disorders Ideal partner with experience in commercialization of products for autoimmune diseases Intend to submit marketing application in 2H15 14

15 ADC Products - Targeting Difficult-to-Treat Tumors Potential characteristics of difficult-to-treat tumors Not responsive to prior therapies Protected by surrounding layer of connective tissue Often become metastatic Components of IMMU s antibody-drug conjugation platform (ADC) Highly specific MAb that is targeted to cancer cells Specially designed payload drug that delivers a concentrated dose to the tumor A linker designed to release the payload at the tumor site Immunomedics Next Generation ADC Technology Platform 1. Reduced toxicity 2. Greater dose of drug to tumor 3. Opportunity for long-term, repeated treatments 15

16 What Makes IMMU s ADCs Different? Unique approach to ADC therapeutics for cancer Highly cancer-specific antibodies based on 30 years of experience Utilize moderately potent payloads: increased therapeutic index Proprietary linker for rapid payload release at or inside tumor High drug-to-antibody ratio (~7.6:1) SN-38 payload Active metabolite with more potency than its parent compound, irinotecan (a commonly used chemotherapy) ADCs unique chemistry avoids low solubility and selectively delivers SN-38 to the tumor Two ADCs completed Phase 2: IMMU-132 and IMMU

17 S N -3 8 (n g /g ) IM M U tr e a te d a n im a ls S N -3 8 (n g /g ) Ir in o te c a n -tr e a te d a n im a ls IMMU-132: Increased Payload Potency Up to 135x Higher Delivery of SN IM M U : S N -3 8 Ir in o te c a n -tr e a te d : S N AUC last (µg/g h) Irinotecan-treated IMMU-132-treated IMMU-132 delivers up to 135-fold more SN-38 to Capan-1 xenografts than irinotecan H o u rs Keep in mind these facts: Mice convert irinotecan to SN-38 more efficiently than humans. Mice were given 22-fold more SN-38 equivalents with the irinotecan dose than the IMMU-132 dose, yet IMMU-132 delivers 135-fold more SN Presented at World ADC San Diego 2014

18 18 IMMU-132 Targets a Variety of Solid Tumors Mechanism of action Binds TROP-2, which is highly expressed on many epithelial cancer cells IMMU-132 is internalized into the tumor cells before SN-38 is released Phase 1/2 clinical trial design Conducted in patients with relapsed/refractory metastatic cancers: breast, lung, esophageal, ovarian, prostate, lung, colorectal, others U.S. Fast Track designation in triple-negative breast, non-small cell and small cell lung cancers U.S. Orphan Drug status in small-cell lung and pancreatic cancers Dosing on days one and eight of 21-day cycle Encouraging results from Phase 1/2 reported Mild and manageable toxicity at recommended doses Numerous objective responses or long-term disease stabilization in heavily pretreated patients; including patients previously treated with topoisomerase inhibitors Multiple treatment cycles administered

19 IMMU-132: Summary Efficacy (Patients with at least one post-baseline CT) Meaningful responses in patients having multiple prior therapies, Phase 1 / 2 clinical trials Number of Patients % ORR1 % Disease Control Clinical Benefit PR + SD > 4 mo Clinical Benefit PR + SD > 6 mo TNBC 46 26% 74% 63% 46% NSCLC 19 32% 74% 59% SCLC 20 30% 55% 55% 37% EAC 16 13% 56% 44% 1 Objective response rate (%ORR) = (Complete response + partial response)/number of patients. Note: Clinical benefit and ORR do not include any patient currently pending, starting dose at 18 mg/kg, or who received less than three doses and terminated early due to death or disease progression. Clinical benefit calculations do not include patients with stable disease that have not been dosed > 4 months and > 6 months. 19 Presented at 2015 Annual Meeting of the American Association for Cancer Research in Philadelphia, PA, April 2015

20 IMMU-132: Best Response from TNBC Patients 46 assessable, 21 continuing treatments 07 Apr 2015 B e s t r e s p o n s e ( % c h a n g e f r o m b a s e l i n e ) Objective response (CR + PR) = 26% Disease control (CR + PR + SD) = 74% CBR* [CR + PR + (SD > 6 mo)] = 17/37 (46%) CBR* [CR + PR + (SD > 4 mo)] = 27/43 (63%) Median prior chemotherapies = 4 (range, 1 11) (CR) = 2 (PR) = 10 (SD) = 22 (PD) = % 6 p a t i e n t s w i t h P D a r e n o t s h o w n b e c a u s e t h e y h a d a n e w l e s i o n o r p r o g r e s s i o n o f n o n - t a r g e t l e s i o n s L y m p h n o d e w a s s i n g l e t a r g e t le s io n t h a t d e c r e a s e d t o < 1 0 m m a t 1 s t a s s e s s m e n t, a n d w it h d is a p p e a r a n c e o f 2 n o n - t a r g e t le s io n s, t h e p t is c o n s id e r e d C R patients enrolled; 3 have not had their first assessment; 3 excluded because 2 doses given; 1 excluded because of 18 mg/kg starting dose. *CBR, clinical benefit ratio. Presented at 2015 Annual Meeting of the American Association for Cancer Research in Philadelphia, PA, April

21 IMMU-132: Best Response from NSCLC Patients 19 assessable 07 Apr 2015 Objective response (CR + PR) = 32% Disease control (CR + PR + SD) = 74% CBR* [CR + PR + (SD > 4 mo)] = 10/17 (59%) 4 0 Median prior therapies = 3 (range, 1 8) B e s t r e s p o n s e ( % c h a n g e f r o m b a s e l i n e ) p t s w it h P D n o t s h o w n b e c a u s e t h e y h a d n e w le s io n. s q u a m o u s c e l l h i s t o p a t h o l o g y, (CR) = 0 (PR) = 6 (SD) = 8 (PD) = a l l o t h e r s a d e n o c a r c i n o m a patients enrolled; 5 have not had their first assessment; 1 excluded because 2 doses given. *CBR, clinical benefit ratio. 21 Presented at 2015 Annual Meeting of the American Association for Cancer Research in Philadelphia, PA, April 2015

22 IMMU-132: Best Response from SCLC Patients 20 assessable 07 Apr 2015 Objective response (CR + PR) = 30% Disease control (CR + PR + SD) = 55% CBR* [CR + PR + (SD > 6 mo)] = 7/19 (37%) CBR* [CR + PR + (SD > 4 mo)] = 11/20 (55%) Median prior therapies = 2.5 (range, 1 7) (CR) = 0 (PR) = 6 (SD) = 5 (PD) = 9 22 patients enrolled; 1 excluded because 2 doses given; 1 excluded because of 18 mg/kg starting dose. *CBR, clinical benefit ratio. 22 Presented at 2015 Annual Meeting of the American Association for Cancer Research in Philadelphia, PA, April 2015

23 IMMU-132: Best Response from EAC Patients 16 assessable 07 Apr 2015 Objective response (CR + PR) = 13% Disease control (CR + PR + SD) = 56% CBR* [CR + PR + (SD > 4 mo)] = 7/16 (44%) (CR) = 0 (PR) = 2 (SD) = 7 (PD) = 7 18 patients enrolled; 1 excluded because 2 doses given; 1 excluded because of 18 mg/kg starting dose. *CBR, clinical benefit ratio. 23 Presented at 2015 Annual Meeting of the American Association for Cancer Research in Philadelphia, PA, April 2015

24 IMMU-132: Mild, Predictable and Manageable Toxicity Adverse events (Grades 3 and 4): Starting dose of 8 or 10 mg/kg (N=123) Grade 3 Grade 4 Neutropenia 22 (18%) 7 (6%) Anemia 7 (6%) 0 Fatigue 6 (5%) 0 Diarrhea 4 (3%) 0 Febrile neutropenia 3 (2%) 2 (2%) Note: Grade 2 Alopecia N = 25 (20%) Grade 3 Lymphopenia, WBC count decreased, and vomiting 2 patients each Grade 3 Asthenia, dizziness, and UTI 1 patient each Camptosar (irinotecan) US Prescribing Information (USPI) boxed warnings Early and late forms of diarrhea can occur (Grades 3 & 4: 38%) Severe myelosuppression may occur (Neutropenia: Grades 3 & 4: 31%) No anti-antibody responses detected to-date, even after repeated dosing Minimal dose delays; 15%-16% of patients required dose reductions 24 Presented at 2015 Annual Meeting of the American Association for Cancer Research in Philadelphia, PA, April 2015

25 IMMU-130 in Metastatic Colorectal Cancer Mechanism of action Binds to CEACAM5 on colorectal and other tumor cells SN-38 is released locally from IMMU-130 for diffusion into tumor cells Two Phase 1 clinical trials Multiple dosing schedules evaluated Phase 2 clinical trial Enrolled relapsed/refractory metastatic colorectal cancer patients previously treated with one or more irinotecan regimens, some refractory to irinotecan regimens Patient follow-up continuing in 1H15 25

26 IMMU-130: Best Response Data (N=26) IMMU IMMU Every other week Twice weekly Once weekly PR = 1 SD = 4 PD = 7 Disease Control 5/12 (42%) PR = 1 SD = 6 PD = 3 Disease Control 7/10 (70%) PR = 0 SD = 3 PD = 1 Disease Control 3/4 (75%) 26 Presented at 2014 ASCO and 2014 AACR

27 90 Y-Clivatuzumab Tetraxetan: Phase 3 for PC Labeled with 90 Y for potential first-in-class therapy Designed to selectively deliver radiation to pancreatic tumor tissue Under investigation in combination with low dose gemcitabine for enhanced activity Orphan Drug designation in the United States and EU Fast Track status with FDA Highly specific antibody targeting mucin antigen Does not bind to normal pancreas tissue or pancreatitis while reactive to ~85% of pancreatic cancers Global randomized, double-blind, Phase 3 study; completion of enrollment expected in 2016 Product patent-protected 27

28 Number of Patients Phase 1b: Initial Experience in Refractory PC Open label study in patients with metastatic pancreatic cancer who had received two or more prior therapies Study regimen: 6.5 mci/m 2 90 Y-clivatuzumab tetraxetan qw x3 +/- low dose gemcitabine (200 mg/m 2 qw x4) Encouraging overall survival trends with minimal toxicity of the combination therapy Patients Alive at 3, 6, 9 and 12 Months Y-clivatuzumab tetraxetan + gemcitabine (Arm A) 90 Y-clivatuzumab tetraxetan monotherapy (Arm B) 3 Months 6 Months 9 Months 12 Months N 3 Months 6 Months 9 Months 12 Months Arm A (48%) 10 (34%) 6 (21%) 3 (10%) Arm B (34%) 3 (10%) 1 (3%) 0 (0%) 28 Final data presented at 2014 ASCO

29 Randomization 90 Y-Clivatuzumab Tetraxetan P3 Design (PANCRIT-1) Double-blind, placebo-controlled, registration study in advanced pancreatic cancer patients who had received two or more prior therapies Prior therapy must include gemcitabine Primary endpoint: Overall survival N = 440 Arm 1: 6.5mCi/m 2 90 Y-clivatuzumab tetraxetan qw x mg/m 2 gemcitabine qw x4 per 7-week cycle (n=293) Arm 2: Placebo qw x mg/m 2 gemcitabine qw x4 per 7-week cycle (n=147) Cycle 1 Cycle 2 Cycle 3 Cycle 4 Cycle 5 Cycle 6 (max) 29

30 Financial Highlights (As of March 31, 2015) Basic shares outstanding 93 million Market capitalization $358 million Debt (convertible senior notes) $100 million Cash, cash equivalents and marketable securities $106 million Forecast FY 2015 annual cash burn (6/30/15) $42 million 30

31 Meaningful Upcoming Anticipated Milestones Program Event Expected Timing Epratuzumab Phase 3 top line data in SLE 1H15 IMMU-132 & IMMU-130 Additional Phase 2 data 2015 IMMU-132 Phase 3 clinical program decision 2H15 90 Y-clivatuzumab tetraxetan Complete Phase 3 enrollment in 3 + -line pancreatic cancer