ARIAD Pharmaceuticals, Inc. June 8, 2016 David Sachs Non-small cell lung cancer 1 ARIAD clinical trial patient
Some of the statements in this presentation constitute forward looking statements under the Private Securities Litigation Reform Act of 1995. Such statements are subject to factors, risks and uncertainties (such as those detailed in the Company s periodic filings with the SEC) that may cause actual results to differ materially from those expressed or implied by such forward looking statements. 2
ARIAD 2016: our focus on oncology Precision therapies Rare cancers Patients with limited options Patients with limited options 3
Targeting rare cancers with precision therapies: rapid discovery and development Integrated small-molecule discovery platform - Innovative chemistry - Computation - Predictive biology Predict and overcome resistance to current therapies Targeting genetically characterized rare cancer populations Rapid clinical proof-of-concept 4
ARIAD 2016: strategic review ongoing Areas of Focus Commercial Initiatives Geographic Review Cost Efficiencies R&D Portfolio Business Development 5
ARIAD 2016: Iclusig commercial strategy 2016 Product and Royalty Revenue Guidance: $170-$180M Strategic Imperatives 1 2 3 4 Build confidence in Iclusig Own the treatment of 2 nd gen. failures Maintain patients on therapy Increase U.S. field footprint 6
Iclusig: as a third-line treatment option Proportion of Patients Achieving CCyR 0.0 0.2 0.4 0.6 0.8 1.0 PROBABILITY BAF, BOS, DAS, OR NIL: [Cortes, 2011] BOS: [Khoury, 2012] DAS: [Garg, 2009] DAS: [Quintas-Cardama, 2007] DAS or NIL: [Garcia-Gutierrez, 2012] DAS or NIL: [Ibrahim, 2010] DAS or NIL: [Rossi, 2011] NIL: [Garg, 2009] NIL: [Giles, 2010] NIL: [Nicolini, 2009] PONATINIB: [Cortes, 2012] PONATINIB (non-t315i): [Cortes, 2012] PONATINIB: PACE PONATINIB: PACE non-t315i 22%-26% 60.5% (95% CI [52.1-68.6]) These data suggest sequencing of second-generation TKIs may be a suboptimal treatment approach SOURCE: Lipton, et al. Blood. 2013;122 (abstr 4010) BAF=bafetinib; BOS=bosutinib; DAS=dasatinib; NIL=nilotinib. NOTE: Node size in graph represents patient numbers; line signifies derived 95% confidence interval. 7
ARIAD 2016: strategic review ongoing Areas of Focus Commercial Initiatives Geographic Review Cost Efficiencies R&D Portfolio Business Development 8
Iclusig: geographic presence in 2015 9
ARIAD 2016: divestiture of European business Strategic Rationale: Re-focuses ARIAD on the valuable US market Significantly improves financial position Continuity of Iclusig commercialization in Europe New partner for Iclusig development Continued employment for ARIAD European team Preserves flexibility under change-of-control scenario 10
ARIAD 2016: Incyte transaction key terms Incyte acquired ARIAD EU operations and licensed Iclusig for the region Financials: - $140 mil upfront & 32-50% royalties to ARIAD - $14 mil in development cost sharing for OPTIC/OPTIC2L - $135 mil potential milestones in oncology indications Commercialization is coordinated through JCC Joint development for new indications is possible ARIAD acquirer may terminate License following change of control of ARIAD 11
ARIAD 2016: strategic review ongoing Areas of Focus Commercial Initiatives Geographic Review Cost Efficiencies R&D Portfolio Business Development 12
ARIAD 2016: cost efficiencies Reduction Off Our Prior Cost Base: Eliminated 25 percent of headquarter positions No customer-facing positions in Commercial or Medical Affairs impacted Created greater operation efficiencies 13
ARIAD 2016: financial guidance (dollars in millions) Iclusig product and royalty revenue $170 - $180 Selling, general and administrative expenses $120 - $125 Research and development expenses $175 - $180 Cash, cash equivalents, marketable securities as of 12/31/16 $280 - $290 14
ARIAD 2016: strategic review ongoing Areas of Focus Commercial Initiatives Geographic Review Cost Efficiencies R&D Portfolio Business Development 15
Lung Cancer: two distinct opportunities Brigatinib ALK+ NSCLC AP32788 NSCLC - Potential best-in-class orally active TKI - Compelling profile expected to garner significant market share - Global market approaches $2B by 2020 - Orally active TKI - Unique profile against EGFR and HER2 exon 20 mutations - No current targeted treatments - Estimated 6,000 U.S. patients 16
Brigatinib: ALK + NSCLC market 2016 annual ALK+ first-line and second-line treatable patients* U.S. Europe Japan 1 st line ~4,300 ~6,400 ~1,900 2 nd line ~2,000 ~3,000 ~1,000 *Stage IV Europe=EU28 Sources: Kantar Health CancerMPact Drug Therapy NSCLC. ARIAD Internal Estimates 17
Brigatinib: pivotal ALTA trial in refractory ALK+ NSCLC Data presented at ASCO 2016 Global Trial N= 222 patients Includes patients with brain metastases Brigatinib 90 mg All Patients Randomized 1:1 Increase to 180 mg 110 patients Continue on 90 mg 1 Week 112 patients Primary endpoint = ORR Randomized Phase 2 design NDA filing expected 3Q 2016; potential approval in early 2017 18
Brigatinib: ALTA trial: 54% confirmed response rate Investigator-Assessed Efficacy Parameter 90 mg qd (n=112) 180 mg qd * (n=110) Confirmed ORR, n (%) 50 (45) [97.5% CI] [34-56] 59 (54) [43-65] Confirmed CR, n (%) 1 (1) 4 (4) Confirmed PR, n (%) 49 (44) 55 (50) Single PR awaiting confirmation, n (%) 2 (2) 2 (2) Disease control rate, n (%) [95% CI] Confirmed ORR by history of prior chemotherapy, n/n (%) 92 (82) [74-89] 95 (86) [79-92] Yes 35/83 (42) 44/81 (54) No 15/29 (52) 15/29 (52) Responses include a confirmed PR at 180 mg in a patient with G1202R at baseline * 180 mg qd with 7-d lead-in at 90 mg. Primary endpoint tested at 0.025 level for each dose. Data as of February 29, 2016 CR = complete response, PR = partial response 19 SOURCE: Kim D-W, et al. Presented at: ASCO. 2016 (abstr 9007).
Brigatinib: ALTA trial median PFS more than one year Probability of PFS (%) 100 90 80 70 60 50 40 30 20 90 mg qd * Median Median PFS PFS exceeds exceeds 1 year 1 year (13 (12.9 months) months) with with 180 180 mg mg brigatinib brigatinib 10 180 mg qd* 0 0 6 12 18 24 Time (mo) 90 mg qd Events/Total (%) 50/112 (45) 180 mg qd * 31/110 (28) 1-Year PFS Probability, % (95% CI) 39 (27-52) 54 (37-68) Median PFS (95% CI) Hazard Ratio (95% CI) 9.2 months (7.4-15.6) 0.55 12.9 months (11.1-not reached) (0.35-0.86) SOURCE: Kim D-W, et al. Presented at: ASCO. 2016 (abstr 9007). * 180 mg qd with 7-d lead-in at 90 mg. Study was not designed to compare treatment arms statistically; however, post hoc comparisons were performed to support dose selection. Data as of February 29, 2016 20
Brigatinib: ALTA trial demonstrates high intracranial response rates IRC-Assessed Efficacy Parameter * Confirmed intracranial ORR, n (%) [97.5% CI] Best overall response, n (%) Patients With Measurable ( 10 mm) Brain Metastases 90 mg qd (n=25) 9 (36) [18-58] 180 mg qd (n=18) 12 (67) [41-87] Patients With Only Nonmeasurable Brain Metastases 90 mg qd (n=54) 3 (6) [1-15] 180 mg qd (n=54) 10 (19) [9-31] Confirmed intracranial CR 2 (8) 0 3 (6) 10 (19) Confirmed intracranial PR 7 (28) 12 (67) NA NA Intracranial CR awaiting confirmation Intracranial PR awaiting confirmation Intracranial disease control rate, n (%) [95% CI] 0 0 0 1 (2) 3 (12) 0 NA NA 22 (88) [69-98] 15 (83) [59-96] 39 (72) [58-84] Among patients with measurable, active ǁ brain metastases at baseline, IRC-assessed intracranial ORR: - 37% (7/19) at 90 mg - 73% (11/15) at 180 mg 47 (87) [75-95] * Of 222 randomized patients, 215 had a baseline brain MRI evaluated by the IRC, with 151 identified as having brain metastases at baseline. Intracranial response defined as a 30% decrease in measurable lesions or complete disappearance of lesions in patients with only nonmeasurable lesions. 180 mg qd with 7-day lead-in at 90 mg. ǁ Active brain metastases were defined as lesions with no prior radiotherapy or those with investigator-assessed progression after prior radiotherapy. Data as of February 29, 2016 NA=not applicable. 21 SOURCE: Kim D-W, et al. Presented at: ASCO. 2016 (abstr 9007).
Brigatinib: ALTA trial treatment-emergent adverse events 90 mg qd 180 mg qd Treatment-Emergent AEs Reported in 10% n=109 n=110 of All Patients Any Grade, % Grade 3, % Any Grade, % Grade 3, % Nausea 33 1 40 1 Diarrhea 19 0 38 0 Headache 28 0 27 1 Cough 18 0 34 0 Fatigue 20 1 27 0 Vomiting 24 2 23 0 Dyspnea 21 3 21 2 Increased blood creatine phosphokinase 11 3 30 9 Decreased appetite 22 1 15 1 Constipation 19 1 15 0 Hypertension 11 6 21 6 Muscle spasms 12 0 17 0 Arthralgia 14 1 14 0 Back pain 10 2 15 2 Abdominal pain 17 0 8 0 Rash 7 1 16 3 Increased amylase 8 1 15 1 Increased aspartate aminotransferase 8 0 15 0 Pyrexia 14 0 6 1 Some AEs appear dose-related; the increased rates are mainly in grade 1-2 events Subset of early pulmonary AEs in 6% pts (3% grade > 3). Managed primarily with dose interruption Data as of February 29, 2016 SOURCE: Kim D-W, et al. Presented at: ASCO. 2016 (abstr 9007). * 180 mg qd with 7-d lead-in at 90 mg; median time on treatment was 7.5 months in 90 mg qd arm and 7.8 months in 180 mg qd arm. 22
ALTA-1L: Frontline ALK+ NSCLC phase 3 study Locally advanced or metastatic ALK+ NSCLC ALK TKI naïve N=270 R 1:1 Brigatinib 180 mg qd * (Arm A) Stratify by: Baseline CNS metastases (yes vs no) Prior chemotherapy (yes vs no) Crizotinib 250 mg bid (Arm B) PD Toxicity Other discontinuation criteria Disease assessment q8w (including brain MRI for all patients) Additional key inclusion criteria: ECOG PS 0-2, measurable disease, 1 prior regimen of systemic therapy in the advanced setting Primary endpoint: IRC-assessed PFS per RECIST 1.1 *With 7-day lead-in at 90 mg. Arm B crossover to brigatinib allowed at progression. 23 SOURCE: https://clinicaltrials.gov/show/nct02737501.
Iclusig: dose-ranging OPTIC trial Trial enrolling patients Adult CP-CML patients resistant to 2 TKIs, N=450 Primary endpoint: MCyR by 12 months 1:1:1 Randomization Ponatinib 15 mg Ponatinib 30 mg Ponatinib 45 mg Dose reduction to 15 mg upon achievement of MCyR Minimum follow-up of 2 years 24
Iclusig: second-line OPTIC-2L trial Trial enrolling patients Adult CP-CML patients resistant to imatinib, N=600 Primary endpoint: MMR by 12 months 1:1:1 Randomization Ponatinib 30 mg Ponatinib 15 mg Nilotinib 400 mg Dose reduction to 15 mg upon achievement of MMR Dose reduction to 10 mg upon achievement of MMR Minimum follow-up of 5 years 25
ARIAD: strong internally developed pipeline Iclusig (ponatinib)* PRECLINICAL PROOF OF CONCEPT PIVOTAL APPROVED CML, Ph+ ALL (refractory) CML (2 nd line) CML (dose-ranging) Ph+ ALL (with chemo, 1st, 2nd line) AML (FLT3) Lung cancer (FGFR, RET) Brigatinib (AP26113) Non-small cell lung cancer (ALK) Resistant Non-small cell lung cancer (ALK) 1 st line AP32788 Non-small cell lung cancer (EGFR Exon 20, HER2 Exon 20) * Includes investigator-sponsored trials. 26
ARIAD 2016: potential value-driving catalysts Q2 Q3 Q4 Advance AP32788 into development Announce outcome of strategic review File for U.S. approval of brigatinib Receive marketing approval of Iclusig in Japan Start 1 st line brigatinib trial Present pivotal ALTA data on brigatinib at ASCO ARIAD: Targeting rare cancers with precision therapies A potential two-product commercial company by early 2017 27