Building Shareholder Value - Jefferies · Building Shareholder Value Hans Loland with wife Cynthia...

Jefferies Healthcare Conference 06.04.2014 1

Building Shareholder Value

Hans Loland with wife Cynthia

Chronic myeloid leukemia ARIAD clinical trial patient

June 4, 2014

Jefferies Healthcare Conference

Tim Clackson, Ph.D.

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

A R I A D P h a r m a c e u t i c a l s , I n c .


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.


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 AP26113

- New first-in-class TKI


Iclusig

Justin Ozuna Chronic myeloid leukemia ARIAD clinical trial patient


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


> 400 commercial U.S. patients through April

- 210 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

Iclusig: early U.S. progress

Beth Galliart Chronic myeloid leukemia ARIAD clinical trial patient


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

Iclusig: maximizing the European commercial opportunity

~2,500 Current eligible

patients

15 Key Countries


1H 2014 2H 2014

Advancing pricing and reimbursement in Europe

Iclusig: maximizing the European commercial opportunity

Germany

Switzerland

Ireland

Italy

France

U.K.

Spain

Nordics

Benelux

Portugal


Advancing clinical development

Jim Logan Chronic myeloid leukemia ARIAD clinical trial patient

Iclusig


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 2014

- Determine optimal starting dose, dose-reduction

- Balance of safety and efficacy in earlier and later lines of treatment

Iclusig: improving the benefit/risk


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


Phase 2 Trial of Ponatinib in GIST: Time On Treatment

Time on Treatment (Weeks)

P a t i

e n t s

0 4 8 12 16 20 24 28 32 36 40 44 48

Discontinued

Ongoing

• 14 out of 35 patients remain on therapy and have received treatment for at least 6 months

Heinrich et al, ASCO 2014: abstract #10506 Data as of 07 Apr 2014


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

Data as of 07 Apr 2014 Heinrich et al, ASCO 2014: abstract #10506

GIST: phase 2 data at ASCO


Disease Patient population/ design Country

Ph+ ALL Combination with hyper-CVAD in 1st/2nd-line Ph+ ALL U.S.

CP-CML Frontline treatment in chronic phase CML

2nd-line treatment in chronic phase CML U.S.

AML Single agent in FLT3+ AML Combination with cytarabine for consolidation in FLT3+ AML

Hong Kong France

NSCLC NSCLC with FGFR1 amplification or RET translocation

NSCLC with RET translocations

U.S.

MTC Medullary thyroid carcinoma with or without RET mutations U.S.

Endometrial Endometrial cancer with FGFR2 mutations U.S., Australia

Iclusig: ISTs in progress


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.


AP26113

ALK Inhibitor in NSCLC


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 1.6 – 14.7 mo (ongoing) • Median PFS is 10.9 mo (N=49)

aTKI-naïve- 5/6 pts had best target lesion response data entered at time of analysis; bReceived prior crizotinib and ceritinib

Data as of 17 March 2014 Gettinger et al, ASCO 2014: abstract #8047


35/51 (69%) crizotinib refractory ALK+ NSCLC patients responded to AP26113

- 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

AP26113: updated phase 1/2 data at ASCO


AP26113: pivotal phase 2 trial in refractory ALK+ NSCLC

Primary endpoint = ORR Non-comparative trial

Trial began 1Q 2014

90 mg QD

All Patients

Global Trial (N= 220 patients)

Includes patients with brain metastases

Randomized 1:1

1 Week

Continue on 90 mg QD

110 patients

Increase to 180 mg QD

110 patients

20 06.04.2014 Jefferies Healthcare Conference

Strong internal drug discovery: five novel molecules

ridaforolimus

AP1903

First Generation

ponatinib

AP26113

Current Generation

New small- molecule TKI

Next Generation


ARIAD: building shareholder value

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