Building Shareholder Value

Transcription

1 Building Shareholder Value June 4, 2014 Jefferies Healthcare Conference Tim Clackson, Ph.D. Hans Loland P r e s i d e n t o f R & D, C h i e f S c i e n t i f i c O f f i c e r with wife Cynthia A R I A D P h a r m a c e u t i c a l s, I n c. Chronic myeloid leukemia 1 ARIAD clinical trial patient

2 Some of the statements in this presentation constitute forward looking statements under the Private Securities Litigation Reform Act of 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

3 ARIAD: creating shareholder value Rebuild confidence in Iclusig - Resistant/intolerant CML and Ph+ ALL - Strong re-launch in the U.S. - P&R approvals and launch in Europe - Approval in Japan and other markets - Lifting clinical hold in the U.S. - Earlier lines of CML treatment - New indications Rebuild value of ARIAD - Improve benefit/risk of Iclusig - Competitive advantages of AP New first-in-class TKI 3

4 Iclusig Justin Ozuna Chronic myeloid leukemia 4 ARIAD clinical trial patient

5 Iclusig: U.S. commercial opportunity Eligible population: ~1,300 new patients annually Streamlined distribution Modest price premium to second-generation therapies ~$125,000 Comprehensive patient assistance program; $10 co-pay 5

6 Iclusig: early U.S. progress > 400 commercial U.S. patients through April IND patients successfully converted - 30% third line, 55% fourth line - ~60% chronic-phase patients - ~25% with T315I mutation Growing prescriber base with > 300 unique prescribers through April - ~60% community based physicians Beth Galliart Chronic myeloid leukemia ARIAD clinical trial patient 6

7 Iclusig: maximizing the European commercial opportunity Selling in Germany, U.K., France, Austria, Switzerland, the Netherlands, Norway and Sweden Modest commercial operations customized to the local market dynamics Article 20 Referral procedure ongoing 15 Key Countries ~2,500 Current eligible patients 7

8 Iclusig: maximizing the European commercial opportunity Advancing pricing and reimbursement in Europe 1H H 2014 Germany Switzerland Ireland Italy France U.K. Spain Nordics Benelux Portugal 8

9 Iclusig Advancing clinical development Jim Logan Chronic myeloid leukemia ARIAD clinical trial patient 9

10 Iclusig: improving the benefit/risk Better understanding of vaso-occlusive events - Longer follow up of clinical trials - Pharmacovigilance program for commercial product - Biological and molecular basis of events - Comparison to other BCR-ABL inhibitors Interventions - Role of anti-coagulants and anti-platelet drugs - CV risk factor mitigation - Treatment of concurrent CV disease Benefit-risk at lower doses - Randomized trial of multiple dose levels to begin 2H Determine optimal starting dose, dose-reduction - Balance of safety and efficacy in earlier and later lines of treatment 10

11 Iclusig: advancing the opportunity in GIST GIST - Annual U.S. incidence of 4,500 patients - KIT activation in 85% of patients - Iclusig has high potency against KIT and resistance mutants - 50% of patients fail imatinib (PFS 24 months) due to resistance mutants - Phase 2 trial ongoing in resistant GIST 11

12 P a t i e n t s Phase 2 Trial of Ponatinib in GIST: Time On Treatment Ongoing Discontinued Time on Treatment (Weeks) 14 out of 35 patients remain on therapy and have received treatment for at least 6 months 12 Heinrich et al, ASCO 2014: abstract #10506 Data as of 07 Apr 2014

13 GIST: phase 2 data at ASCO Initial analysis suggests ponatinib has activity in patients with advanced GIST, particularly KIT exon 11 patients, after failure of prior TKI therapy - Clinical Benefit Rate at 16 weeks for KIT exon 11 patients was 50% Median follow-up of 6 months, 14 patients remain on treatment - 1 partial response, 11 stable disease and 2 progressive disease Median OS not reached; median PFS 7 months Safety profile in GIST appears to be consistent with that in CML, except myelosuppression is notably less in GIST than in leukemia trials Phase 2 data provide ponatinib proof of concept in resistant GIST 13 Heinrich et al, ASCO 2014: abstract #10506 Data as of 07 Apr 2014

14 Iclusig: ISTs in progress Disease Patient population/ design Country Ph+ ALL Combination with hyper-cvad in 1st/2nd-line Ph+ ALL U.S. CP-CML AML NSCLC Frontline treatment in chronic phase CML 2nd-line treatment in chronic phase CML Single agent in FLT3+ AML Combination with cytarabine for consolidation in FLT3+ AML NSCLC with FGFR1 amplification or RET translocation NSCLC with RET translocations U.S. Hong Kong France U.S. MTC Medullary thyroid carcinoma with or without RET mutations U.S. Endometrial Endometrial cancer with FGFR2 mutations U.S., Australia 14

15 Iclusig: ISTs to open in 2014 Disease Patient population/ design Country Ph+ ALL Elderly Ph+ ALL patients unfit for chemo or transplant Italy BP-CML Combination with FLAG-IDA in CML blast crisis U.K. AML Combination with induction chemotherapy Combination with decitabine in relapsed/refractory FLT3+ AML Combination with azacytidine in relapsed FLT3+ AML U.K. France Australia Various Cancers with abnormalities in FGFRs or other ponatinib targets U.S. Bile Duct Cholangiocarcinoma/bile duct carcinoma with FGFR fusions U.S. 15

16 AP26113 ALK Inhibitor in NSCLC 16

17 AP26113: anti-tumor activity in ALK+ NSCLC patients 72% (41/57) objective response rate (95% CI: 59-83%) 100% (6/6) in TKI-naïve (incl. 1 CR) 69% (35/51) post-crizotinib (95% CI: 54-81%) 23 confirmed, 7 await confirmation Response duration mo (ongoing) Median PFS is 10.9 mo (N=49) a TKI-naïve- 5/6 pts had best target lesion response data entered at time of analysis; b Received prior crizotinib and ceritinib 17 Gettinger et al, ASCO 2014: abstract #8047 Data as of 17 March 2014

18 AP26113: updated phase 1/2 data at ASCO 35/51 (69%) crizotinib refractory ALK+ NSCLC patients responded to AP Of 49 patients with follow-up scans, median PFS is 10.9 months 9/13 (69%) patients had regression of untreated or progressing brain lesions Most common AEs were nausea, fatigue and diarrhea, which were generally grade 1 or 2 in severity; the most common SAE was dyspnea Early onset pulmonary symptoms were observed in 3% (1/38) of patients started at 90 mg daily and 14% (6/44) of patients started at 180 mg daily No early onset pulmonary symptoms were observed in 28 patients started at 90 mg and escalated to 180 mg after 7 days 18

19 AP26113: pivotal phase 2 trial in refractory ALK+ NSCLC Trial began 1Q 2014 Global Trial (N= 220 patients) Includes patients with brain metastases 90 mg QD All Patients Randomized 1:1 Increase to 180 mg QD 110 patients Continue on 90 mg QD 1 Week 110 patients Primary endpoint = ORR Non-comparative trial 19

20 Strong internal drug discovery: five novel molecules First Generation Current Generation Next Generation ridaforolimus AP1903 ponatinib AP26113 New smallmolecule TKI 20

21 ARIAD: building shareholder value Discover Develop Deliver 21

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