Corporate Presentation

Transcription

1 Corporate Presentation Leerink Global Healthcare Conference February 14 th -15 th, 2018 C O N F I D E N T I A L a n d P R O P R I E T A R Y

2 Forward Looking Statements / Safe Harbor This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding future results of the operations of ARMO BioSciences, Inc. ( We, ARMO or the Company ), including financial position, strategy, and plans and ARMO s expectations for future operations, are forward-looking statements. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: the timing and success of preclinical studies and clinical trials conducted by ARMO; the ability to obtain and maintain regulatory approval of ARMO s product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing ARMO s product candidates; ARMO s ability to obtain and maintain intellectual property protection for ARMO s product candidates; ARMO s anticipated growth strategies; ARMO s expectations regarding competition; the anticipated trends and challenges in ARMO s business and the market in which we operate; ARMO s ability to attract or retain key personnel; the size and growth of the potential markets for ARMO s product candidates and the ability to serve those markets; the rate and degree of market acceptance of any of ARMO s product candidates; ARMO s ability to establish and maintain development partnerships; ARMO s expectations regarding federal, state and foreign regulatory requirements; regulatory developments in the United States and foreign countries; and ARMO s use and sufficiency of its cash resources. Moreover, ARMO operates in a very competitive and rapidly changing environment. New risks emerge from time to time. It is not possible for ARMO s management to predict all risks, nor can ARMO assess the impact of all factors on its business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statement. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this presentation may not occur and actual results could differ materially and adversely from those anticipated or implied. Except as required by law, neither ARMO nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements. ARMO undertakes no obligation to update publicly any forward-looking statements for any reason after the date of this presentation to conform these statements to actual results or to changes in ARMO s expectations. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy securities. C O N F I D E N T I A L a n d P R O P R I E T A R Y 2

3 Investment Highlights IO Focused Business Model Clinical stage immuno-oncology (IO) company Portfolio of IO product candidates Strong and experienced management team IPO closed January 30, $147 million in total gross proceeds AM0010 (Pegilodecakin) Phase 1/1b clinical trial treated 350+ patients in multiple solid tumors - Objective responses as a single agent in cutaneous T-cell lymphoma, melanoma and RCC - Clinical efficacy seen in PDAC, NSCLC, RCC, CRC & melanoma - Improves tolerability of checkpoint and chemo regimens Phase 3 clinical trial (SEQUOIA) +/- FOLFOX in PDAC actively enrolling - Orphan drug (US & EU) and Fast Track designations received in Second interim analysis could provide basis for BLA filing as early as 2020 Two Phase 2 clinical trials (CYPRESS 1 & 2) in NSCLC enrolling patients in Q Initial data read outs on both studies expected in late 2018 Initiate RCC clinical trial with AM0010 in 2H 2018 Additional Portfolio Depth AM0001 anti-pd-1 antibody moving into Phase 1 clinical trial by Y/E Option to optimize commercial potential by combining with internal assets Additional pre-clinical IO assets such as IL-12, IL-15 and anti-lag-3 in development C O N F I D E N T I A L a n d P R O P R I E T A R Y 3

4 Management Team Peter Van Vlasselaer, PhD President and CEO Co-Founder Joseph Leveque, MD Chief Medical Officer Herb Cross Chief Financial Officer Martin Oft, MD VP Pre-Clinical and Clinical Development Co-Founder Russ Kawahata, PhD VP Technical Operations C O N F I D E N T I A L a n d P R O P R I E T A R Y 4

5 Pipeline and Development Timeline Product Candidate Indication / Combination (Clinical Trial) Current Development Stage Preclinical Pre-IND IND Phase 1 Phase 1b Phase 2 Phase 3 Development Status PDAC/FOLFOX (SEQUOIA) Enrollment of 1st patient in early 2017 First interim analysis expected in early 2018 AM0010 (PEG-IL-10) NSCLC/Anti-PD-1 (CYPRESS 1 and 2) RCC/Anti-PD-1 Enrollment of 1st patient in Phase 2 clinical trial(s) planned for 1H 2018 Evaluating Phase 2 clinical trial(s) Melanoma and Other Solid Tumor Indications Evaluating Phase 2 clinical trials in additional indications (e.g. CRC, melanoma, TNBC) AM0001 (Anti-PD-1 mab) All Tumor Indications Initiation of Phase 1 clinical trial in 2018 AM0015 (IL-15) All Tumor Indications Pre-Clinical POC AM0012 (IL-12) All Tumor Indications Pre-Clinical POC AM0003 (Anti-LAG-3 mab) All Tumor Indications Pre-Clinical POC C O N F I D E N T I A L a n d P R O P R I E T A R Y 5

6 AM0010 Mechanism of Action C O N F I D E N T I A L a n d P R O P R I E T A R Y 6

7 Rationale for AM0010 in Immuno-Oncology Intra-tumoral CD8 T cell deficiency and chronic inflammation are linked to a poor prognosis in cancer patients Efficient anti-tumor immune therapy may benefit from drugs that combine anti-inflammatory and CD8 T cell activation properties Anti-Inflammatory CD8 T cell activation IL-10 Biology IL-10 Biology AM0010 Systemic and sustained reduction of inflammatory molecules such as IL-12p40, IL-17, IL-23 and TGF-ß Stimulation of T cell activating cytokines IFNγ, IL-18, IL-7, IL-4, GM-CSF C O N F I D E N T I A L a n d P R O P R I E T A R Y 7

8 AM0010 Increases Intra-Tumoral CD8 + T Cell Numbers AM0010 Monotherapy Increases the Number of Intra-Tumoral PD-1 + CD8 + Granzyme + T Cells in Phase 1/1b Colorectal Cancer Patient Pre Treatment Day 60 of Treatment CRC Liver Metastasis Pyknotic Tumor Cells Tumor Infiltrating Cytotoxic PD-1 + CD8 + T Cells (TILs) Tumor Cells Granzyme B CD8 Note: Granzyme B CD8 staining C O N F I D E N T I A L a n d P R O P R I E T A R Y 8

9 AM0010 Clinical Trials Phase 1/1b C O N F I D E N T I A L a n d P R O P R I E T A R Y 9

10 Phase 1/1b Data Guides Registration Strategy Establish novel combinations and sequentials with IO therapies (e.g. PD-1/PD-L1 inhibitors) with emerging IO therapies with non-io therapies (e.g. SOC targeted therapies, chemotherapies) Focus on patient populations with unmet medical need none and low tumor PD-L1 expression low tumor mutational burden disseminated disease (e.g. liver) Exploit anti-inflammatory properties limit treatment-related adverse events, both immune and non-immune in origin potentiate efficacy by advancing dose intensity reduce morbidity associated with off-target effects C O N F I D E N T I A L a n d P R O P R I E T A R Y 10

11 AM0010 Phase 1/1b Clinical Trial Over 350 Patients Enrolled Across 14 Different Cancers Monotherapy Dose Expansion CRC 4 th Line AM0010 Combo-Therapy Dose Escalation PDAC 2 nd Line FOLFOX + AM0010 SEQUOIA - Phase 3 PDAC 2 nd Line FOLFOX + AM0010 Monotherapy Dose Escalation Melanoma >2 nd Line Anti-PD-1 Refractory Pembro + AM0010 Combo-Therapy Dose Escalation with Anti-PD-1 Nivo/Pembro NSCLC >2 nd Line Nivo + AM0010 RCC >2 nd Line Nivo or Pembro + AM0010 CYPRESS (1 and 2) 1. NSCLC 1 st Line w/ Pembro +/- AM0010 in PD-L1 H 2. NSCLC 2 nd Line w/ Nivo +/- AM0010 in PD-L1 L C O N F I D E N T I A L a n d P R O P R I E T A R Y 11

12 AM0010 Clinical and Regulatory Summary Assessed AM0010 s tolerability, mechanism of action and preliminary efficacy in an ongoing Phase 1/1b clinical trial - Over 350 cancer patients treated across 14 different types of cancer and several treatment settings Monotherapy Combined with checkpoint inhibitors (nivolumab, pembrolizumab) Combined with SOC chemotherapies (FOLFOX) - Well tolerated as a single agent and in combination with chemo and anti-pd-1 SOC therapy - Most common TrAEs being anemia, thrombocytopenia, fatigue and fever - AM0010 potentially reduces immune-related complications when used in combination with checkpoint inhibitors - Durable responses in PDAC, NSCLC, CRC and Melanoma* with data maturing in other indications * As measured by overall survival benefit for AM0010 alone or in combination with standard of care therapies in these populations with advanced stage diseases that are longer than those observed historically based on the medical literature in comparable or earlier stage populations. C O N F I D E N T I A L a n d P R O P R I E T A R Y 12

13 AM0010 Clinical Trials PDAC AM0010 received Orphan Drug Designation (US and EU) and Fast Track Designation in 2016 C O N F I D E N T I A L a n d P R O P R I E T A R Y 13

14 AM0010 in PDAC Phase 1/1b Clinical Trial Data AM0010 AM PDAC FOLFOX Monotherapy FOLFOX** N (Evaluable) N (ITT) Prior Therapies (Median) Prior Therapies (Range) NA (2-6) (1-5) ORR % *** CR + PR CR % DCR 36% 53% * 74% CR + PR + SD PFS (Months - Median) OS (Months - Median) OS (Landmark - One Year) 18.5% 22.7% 42.9% AM0010 data as of Zaanan et al., BMC 2014 (FOLFOX in 2L PDAC) * Based on 8 of 15 evaluable subjects with stable disease at 2 months ** Median follow-up for subjects still alive is ~20 months *** Includes two (2) CRs and one (1) PR with 100% tumor reduction DCR = Disease Control Rate; ORR = Objective Response Rate; CR = Complete Response; PFS = Progression Free Survival; OS = Overall Survival C O N F I D E N T I A L a n d P R O P R I E T A R Y 14

15 AM0010/FOLFOX Combo in PDAC Phase 1/1b Data AM0010/FOLFOX Combo Therapy Has Improved OS in Late Stage Patients (Median 3 rd LOT; Range 2 nd -6 th ) Who Progressed on a Prior Gemcitabine Containing Regimen 4.3 months mos and 18.5% 1-y OS with FOLFOX monotherapy in Zaanan et al Refs: Phase 2 (Zaanan et al, BMC, 2014) Note: AM0010/FOLOFX data as of C O N F I D E N T I A L a n d P R O P R I E T A R Y 15

16 AM0010/FOLFOX Combo is Well Tolerated in PDAC FOLFOX cycles (Median 3 rd LOT) compared to: - Oettle et al.; JCO 2014 (CONKO3) - Zaanan et al.; BMC 2014 (FIRGEM) Rate of Neuropathy (treatment related and unrelated) Study Grade 1/2 Grade 3/4 Reference OFF 2 nd LOT 38.2% 4% Oettle JCO 2014 FOLFOX 2 nd LOT 44% 7% Zaanan BMC 2014 FOLFOX 2 nd LOT NA 4.1% Gill JCO 2016 AM FOLFOX 16% 0% Current data* *AM0010 data as of NA: not available C O N F I D E N T I A L a n d P R O P R I E T A R Y 16

17 AM0010 in PDAC Phase 3 Clinical Trial Design (SEQUOIA) Arm 1 AM FOLFOX Advanced metastatic pancreatic cancer 2 nd line Tx after one prior gemcitabine regimen N=566 1:1 Randomization Interim Analysis 1 n = 60 DMC Recommendation Continue Trial Discontinue Trial Interim Analysis 2 (70% of 393 deaths) DMC Recommendation Stop for superiority Complete Trial Discontinue Trial Final Analysis (393 deaths) FOLFOX Arm 2 Proposed Timing of Events st Interim - Go/No-Go decision 2 nd Interim analysis Potential basis for BLA filing C O N F I D E N T I A L a n d P R O P R I E T A R Y 17

18 AM0010 Clinical Trials - NSCLC C O N F I D E N T I A L a n d P R O P R I E T A R Y 18

19 AM0010 in NSCLC Phase 1/1b Clinical Trial Data Anti-PD-1 mab AM0010 AM AM NSCLC Monotherapy Monotherapy Nivolumab Pembrolizumab N (Evaluable) NA 7 * 22 5 ** N (ITT) NA 9 * 29 5 ** Prior Therapies (Median) Prior Therapies (Range) NA (1-7) (1-3) (0-5) ORR 19% - 41% 40% CR + PR CR DCR 41% 57% 82% 100% CR + PR + SD PFS (Months - Median) NR 11.0 OS (Months - Median) NR (1) TBD (2) (1) Clinical trial in progress. Numbers as of October 29, Median follow-up 17.5 months (range ) (2) OS reached, To Be Disclosed at an upcoming scientific conference. As of October 29, 2017, median follow-up was 31.2 months (range ) Garon et al., NEJM 2015 (pembrolizumab in 2L NSCLC) * Only five (5) subjects available for testing, all of which were PD-L1 negative ** Only four (4) subjects available for testing, all of which were PD-L1 negative AM0010 data as of DCR = Disease Control Rate; ORR = Objective Response Rate; CR = Complete Response; PFS = Progression Free Survival; OS = Overall Survival; NR = Not reached C O N F I D E N T I A L a n d P R O P R I E T A R Y 19

20 AM0010 in NSCLC Indications of Efficacy Regardless of PD-L1 Status Anti-PD-1 mab AM ORR by PD-L1 Expression Monotherapy Anti-PD-1 mab* All 19% 41% (Responses/N) (96/495) (11/27) > 50% 44% 80% (Responses/N) (25/57) (4/5) 1-49% 16% 67% (Responses/N) (12/77) (2/3) < 1% 9% 33% (Responses/N) (2/22) (4/12) Twenty (20) of 27 subjects had PD-L1 testing Garon et al., NEJM 2015 * Includes both nivolumab and pembrolizumab treated subjects C O N F I D E N T I A L a n d P R O P R I E T A R Y 20

21 AM0010 in NSCLC Indications of Efficacy Regardless of TMB and PD-L1 Status NSCLC Tumor Mutational Burden and PD-L1 Expression in Pegilodecakin plus Nivolumab Treated Patients (n=10) Notes: 1. Includes all patients available for TMB and PD-L1 status testing 2. High/Low TMB stratification (243) used in Carbone et al, NEJM 2017 C O N F I D E N T I A L a n d P R O P R I E T A R Y 21

22 AM Pembrolizumab or Nivolumab Reduces Measurable Secondary Liver Lesions Change in Measurable Secondary Liver Lesions in NSCLC on AM0010 in Combination with Pembrolizumab or Nivolumab (n=18) Pre-Treatment 7 Months of Treatment (-81%) A total of 8/27 patients had liver metastasis 5/8 patients had a partial response (63% ORR) 8 patients had a combined total number of 18 target lesions in the liver 16/18 metastasis showed reduction in size during combo therapy 14/16 lesions showed >50% reduction in size C O N F I D E N T I A L a n d P R O P R I E T A R Y 22

23 AM Nivolumab Potentially Reduces Clinically Meaningful Immune-Related Complications Immune-Related Adverse Events (iraes) AM Nivolumab (N) 2 AM N 3 N N = = 144 N N = = 134 N N = = Event % % % Rash % % % Pruritus % % 2 6.9% Fatigue % 7 5.2% % Appetite Loss % 4 3.0% % Vitiligo 0 0.0% 2 1.5% 0 0.0% Pneumonitis 0 0.0% 6 4.5% 1 3.4% Adrenal Insufficiency 0 0.0% 0 0.0% 1 3.4% Thyroiditis/Hypothyroiditis 1 0.7% % 1 3.4% Hypophysitis 0 0.0% 1 0.7% 0 0.0% Mucocytis 1 0.7% 3 2.2% 0 0.0% Colitis 0 0.0% % 0 0.0% Hepatitis 0 0.0% 5 3.7% 0 0.0% Cholangitis 2 1.4% 2 1.5% 0 0.0% Polyarthritis 0 0.0% 1 0.7% 0 0.0% Myasthenia Gravis 0 0.0% 1 0.7% 0 0.0% Optic Neuritis 0 0.0% 0 0.0% 1 3.4% irae rate excluding rash, pruritus, fatigue and appetite loss 2.8% 30.6% * 13.8% (1) Includes phase 1/1b clinical trial monotherapy subjects from all indications (2) Haratani et al. JAMA Onc: pp E1-5 ( ); NSCLC patients (3) Includes only NSCLC patients *Assumes one irae per patient C O N F I D E N T I A L a n d P R O P R I E T A R Y 23

24 AM0010 in NSCLC CYPRESS 1 Clinical Design N ~50 AM Pembrolizumab ORR Stage IV / Metastatic WT NSCLC 1L PD-L1 H N ~100 R NSC 70 / SC 30 Limit in Each Arm Treatment to Progression Crossover Stratify by Histology Mutational Burden PS 0-1 Histology Agnostic N ~50 Pembrolizumab ORR C O N F I D E N T I A L a n d P R O P R I E T A R Y 24

25 AM0010 in NSCLC CYPRESS 2 Clinical Design N ~50 AM Nivolumab ORR Stage IV / Metastatic WT NSCLC 2L PD-L1 L PS 0-1 Histology Agnostic No Exposure to Immune-Based Therapies N ~100 R NSC 70 / SC 30 Limit in Each Arm N ~50 Nivolumab Treatment to Progression Crossover Stratify by Histology Mutational Burden Smoking Status ORR C O N F I D E N T I A L a n d P R O P R I E T A R Y 25

26 AM0010 Clinical Trials - RCC C O N F I D E N T I A L a n d P R O P R I E T A R Y 26

27 AM0010 in RCC Phase 1/1b Clinical Data Summary Nivolumab AM0010 AM AM RCC Monotherapy Monotherapy Nivolumab Pembrolizumab N (Evaluable) N (ITT) Prior Therapies (Median) Prior Therapies (Range) NA (0-7) (1-3) (0-5) ORR 20%-22% 25% 39% 50% CR + PR CR less than 1% % * DCR 57%-65% 56% 81% 100% CR + PR + SD PFS (Months - Median) NR 16.7 OS (Months - Median) NR (1) NR (2) (1) Clinical trial in progress. Numbers as of October 29, Median follow-up 13.8 months (range ) (2) Clinical trial in progress. Numbers as of October 29, Median follow-up 29.4 months (range ) Motzer et al., NEJM 2015 (nivolumab in 2L RCC) * Two (2) partial responses with 100% reduction of measurable disease AM0010 data as of DCR = Disease Control Rate; ORR = Objective Response Rate; CR = Complete Response; PFS = Progression Free Survival; OS = Overall Survival; NR = Not reached C O N F I D E N T I A L a n d P R O P R I E T A R Y 27

28 Other AM0010 Clinical Trials C O N F I D E N T I A L a n d P R O P R I E T A R Y 28

29 Melanoma & CRC Phase 1/1b Clinical Data Summary We observed encouraging survival in a number of additional tumors in our Ph1/1b clinical trial - AM0010 +pembrolizumab cohort, (n=25) Melanoma - mos of 16.7 months observed in patients resistant or refractory to anti-pd-1/ipilimumab - 1 year survival rate of 57% - Recent trial of ipilimumab+pembrolizumab in patients with a similar profile demonstrated a mos of 8 months 1 - AM0010 monotherapy cohort, (n=27) CRC - mos of 11 months observed in patients who had failed a median number of four prior therapies - Recent trial of trifluridine/tiperacil in a similar patient population demonstrated a mos of 7.1 months 2 (1) Kirchberger et al., EJC 2016 (2) Mayer et al., NEJM 2015 C O N F I D E N T I A L a n d P R O P R I E T A R Y 29

30 Other Immunotherapy Assets C O N F I D E N T I A L a n d P R O P R I E T A R Y 30

31 Robust Pipeline of Immunotherapy Assets AM0001 anti-pd-1 checkpoint inhibitor, currently undergoing Investigational New Drug Application (IND) enabling studies AM0015 IL-15, a pre-ind stage product candidate has demonstrated preclinical antitumor responses that are additive with AM0010 AM0012 IL-12, currently in preclinical studies AM0003 anti-lag-3 checkpoint inhibitor, undergoing pre-ind enabling studies C O N F I D E N T I A L a n d P R O P R I E T A R Y 31

32 ARMO Summary C O N F I D E N T I A L a n d P R O P R I E T A R Y 32

33 Finance & IP Strong Balance Sheet Strong Financial Position - $177M in private capital raised pre-ipo - IPO closed January $147M in gross proceeds Cash and cash equivalents balance of ~$67M at September 30, 2017 No debt outstanding AM0010 IP Strong Composition of Matter (COM) portfolio - Base COM claims through 2029 with possibility for extension up to 2034 Orphan designation also provides 7-year market exclusivity C O N F I D E N T I A L a n d P R O P R I E T A R Y 33

34 Key Corporate Milestones Continue data read out from Phase 1/1b (survival in PDAC, RCC, NSCLC, Melanoma and CRC as well as PFS in select cohorts) 1H 2018 Initiate CYPRESS 1 NSCLC study 1L pembrolizumab +/- AM0010 Initiate CYPRESS 2 NSCLC study 2L nivolumab +/- AM st interim analysis (safety) in AM0010 SEQUOIA PDAC study Preliminary data readouts from CYPRESS 1 NSCLC study in late H 2018 Preliminary data readouts from CYPRESS 2 NSCLC study in late 2018 Initiate RCC clinical trial with AM0010 Initiate AM0001 (anti-pd-1) clinical trial Potential pivotal NSCLC studies potentially commencing (expected 2019) Preliminary data readout from RCC study (2H 2019) 2 nd interim analysis (safety + efficacy) in SEQUOIA PDAC study (expected 2020) C O N F I D E N T I A L a n d P R O P R I E T A R Y 34

35 Investment Highlights IO Focused Business Model Clinical stage immuno-oncology (IO) company Portfolio of IO product candidates Strong and experienced management team IPO closed January 30, $147 million in total gross proceeds AM0010 (Pegilodecakin) Phase 1/1b clinical trial treated 350+ patients in multiple solid tumors - Objective responses as a single agent in cutaneous T-cell lymphoma, melanoma and RCC - Clinical efficacy seen in PDAC, NSCLC, RCC, CRC & melanoma - Improves tolerability of checkpoint and chemo regimens Phase 3 clinical trial (SEQUOIA) +/- FOLFOX in PDAC actively enrolling - Orphan drug (US & EU) and Fast Track designations received in Second interim analysis could provide basis for BLA filing as early as 2020 Two Phase 2 clinical trials (CYPRESS 1 & 2) in NSCLC enrolling patients in Q Initial data read outs on both studies expected in late 2018 Initiate RCC clinical trial with AM0010 in 2H 2018 Additional Portfolio Depth AM0001 anti-pd-1 antibody moving into Phase 1 clinical trial by Y/E Option to optimize commercial potential by combining with internal assets Additional pre-clinical IO assets such as IL-12, IL-15 and anti-lag-3 in development C O N F I D E N T I A L a n d P R O P R I E T A R Y 35