ARIAD November 2016 Corporate Presentation

Positioned for Growth

Sara Non-small cell lung cancer ARIAD clinical trial patient

November 16, 2016 Jefferies London Healthcare Conference

This presentation contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on managements’ current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.

2 1 1 . 1 6 . 1 6 J e f f e r i e s 2 0 1 6 L o n d o n H e a l t h c a r e C o n f e r e n c e


Clear vision, building from core strengths in precision small molecule therapies and rare cancers

• Expertise in medicinal chemistry, structure-based drug design and predictive mutational screens

Positioned for potential increase in addressable population from 1,000 - 2,000 to ~16,000 – 18,000 U.S. patients:

• Rapid near-term revenue growth through potentially becoming a two product commercial company in 2017

• Mid-term revenue growth from potential market expansion of Iclusig and brigatinib

• Longer-term growth potential from AP32788 and new opportunities from discovery platform, including immuno-oncology program

Rapid progress in transforming company strategy, culture and financial position

• Product and royalty revenue guidance for 2016 – $170-180 million

• OPEX guidance for 2016 – $295-305 million

• Projected cash balance at December 31, 2016: $280-290 million

Completed comprehensive corporate review

• Divested EU operations, licensed EU rights for Iclusig to INCY

• Scale up of US field force to increase promotion of Iclusig and strengthen preparation for brigatinib launch

Where we are today


Our Vision is to become the leader in the discovery, development, and commercialization of precision therapies for patients with rare cancers.


Core strength: Rare cancers

• Currently account for around a quarter of all cancer diagnoses and deaths*

• Expected to increase in importance as more molecular subsets of common cancers are identified*

ARIAD Focus:

• Address rare cancers/patient populations with high-value precision therapies

• Patient-centric development and commercial model

Incident Number of Patients (US)

Orphan Threshold (prevalence)

Rare Cancer Threshold**

– 200,000

– 45,000

*Lancet, February 2016 **NCI Epidemiology and Genetics Research

ALK+NSCLC

Exon 20 EGFR/HER2 NSCLC

CML/Ph+ALL

– 8,000

– 6,000

– 3,300

ARIAD Current/Potential Markets


Core strength: Robust and fully integrated discovery platform

Precisely designed kinase inhibitors to overcome mutational resistance and drive differential efficacy

Precisely defined groups of patients who have limited, or no other, options and will receive greatest benefit

Validated targets – low target-based risk

Rapid clinical proof-of-concept

Streamlined development: ~5 years first-in-human to approval

AP32788 Rimiducid Bellicum

out-license

Ridaforolimus Merck, Medinol

Partnerships

Ponatinib Brigatinib


ARIAD’s strategy

Commercial Maximization

• US direct commercialization • Leverage Iclusig to continue growth • Planned successful 2017 launch of brigatinib • Ex-US revenues with minimal costs

Development/Pipeline

• Earlier lines of treatment for brigatinib and Iclusig • AP32788 – EGFR/HER2 lung cancer • Share development costs through ex-US partnerships

Science & Discovery

• Engine for new potential products – expand development pipeline • World-class capability in designing precision cancer therapies • Expanding platform into small molecule immuno-oncology

Lean, Cost-efficient Structure

• Rare cancer model designed for efficient commercialization/R&D • Operations limited to US • Lean G&A/headquarter functions




ARIAD’s strategy


Science & Discovery





Geographic strategy: US focus with global reach through partners

Rationale

• US represents majority of industry oncology profits based on ~50% of global sales

• Can retain significant share of ex-US value through partnerships

• Maintain strategic optionality


Key Terms

Incyte acquired ARIAD EU operations and licensed Iclusig for the region (EU countries plus others, including Russia, Turkey)

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

ARIAD acquirer may terminate license following change of control

• Incyte receives financial payments & ongoing royalty from acquirer

Major Benefits

Significantly improves cash position/financial profile

Operating costs reduced by ~ $65 mil annually

Continuity of Iclusig commercialization– ongoing ARIAD royalties

Strategic flexibility

Incyte transaction: Execution of strategy


Iclusig

Katie Olson Chronic myeloid leukemia


Key Drivers of Forward Growth

• Field team expansion

• 4-year PACE data; expected label update

• Increasing clinical experience

• Patient retention programs

Iclusig Global Sales & Royalties

Iclusig: Steady growth is expected to continue

0

2016 Guidance

2014 Actual 2015 Actual

$50M

$150M

$200M

$100M

CAGR +77%


Brigatinib

BRIGATINIB

Andy Bonnet Non-small cell lung cancer ARIAD clinical trial patient


Brigatinib: Overview

NDA accepted in crizotinib-resistant metastatic ALK+ NSCLC

• Breakthrough Therapy designation from FDA

• PDUFA date of April 29, 2017

Pivotal data suggests differentiated efficacy

• PFS 12.9 months* (vs. competitors 6-9 months)

Preparing for anticipated US launch in 2017

Development for front-line indication under way

BRIGATINIB

*Secondary endpoint, comparison not head-to-head


2016 global ALK inhibitor market: Annualized market approaching $800 mil

$283 $348

$349

$420

$0.0

$100.0

$200.0

$300.0

$400.0

$500.0

$600.0

$700.0

$800.0

$900.0

2015 Q1 2016 Annualized

Ex-US In M

illi

on

s $

Global ALK Inhibitor Market

Ex-US

US US

$632

$768 Analysts projecting market growth toward ~$2 billion by 2020

34% Q1/16 vs. Q1/15 growth

Source: Pfizer 2016 Q1 Performance For Xalkori, Novartis 2016 Q1 Performance for Zykadia, Roche 2016 Q1 Performance for Alecensa




ARIAD’s strategy




Science & Discovery



Iclusig

OPTIC & OPTIC 2L

Brigatinib

ALTA and ALTA 1L

AP32788

Phase I/II

Core development programs


1st Line 6,000

2nd Line 1,900

3rd Line 920

T315i+ 200

Total 3rd line + 1,400

Iclusig: Development programs

OPTIC & OPTIC 2L Goals

Improve Iclusig benefit-risk profile through lower doses

• Evaluate 30mg and 15mg starting doses

Expand market potential through earlier lines of treatment

2L indication more than doubles available market

Demonstrate superior efficacy to drive market share

Other Considerations

Incyte partnership provides $14 million funding, plus significant potential milestones related to OPTIC 2L

Current eligible pool 1000-2000 patients


Iclusig: Dose-ranging OPTIC trial

Trial enrolling patients

Adult CP-CML patients resistant to 2 TKIs, N=450 Primary endpoint: MCyR by 12 months

Minimum follow-up of 2 years

1:1:1 Randomization

Dose reduction to 15 mg upon achievement of MCyR

Ponatinib 15 mg Ponatinib 45 mg Ponatinib 30 mg


Iclusig: Second-line 3 OPTIC-2L trial

Trial enrolling patients

Adult CP-CML patients resistant to imatinib, N=600 Primary endpoint: MMR by 12 months

Minimum follow-up of 5 years

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

1:1:1 Randomization


ALTA 1L Goals

• Expand market potential through first line indication

• Potential for long duration of treatment enhances revenue potential

• H2H superiority data would further strengthen efficacy positioning

• Confirmatory study for FDA approval

Brigatinib: Development programs

1st Line 4,300

2nd Line 2,500

3rd Line+ 1,300

Annual Incidence/ Number of Switches*

SOURCE: *SEER Cancer Statistics Review 1975-2010; UpToDate; 2012 SEER Data (Cancer Statistics Review, 1975-2008); Cancer.gov; ARIAD internal assumptions, Applies mutation and testing rate. Stage IV only and assumes 85% testing by 2017, Kantar Health Epi Report 2015; eligible patients is reflective of all patients who would be candidates for ALK treatment


ALTA-1L: Phase 3 study in TKI-naïve ALK+ NSCLC patients

• Locally advanced or metastatic ALK+ NSCLC

• ALK TKI naïve

Brigatinib 180 mg qd* (Arm A)

Crizotinib 250 mg bid (Arm B)

• PD† • Toxicity • Other

discontinuation criteria

Stratify by: • Baseline CNS metastases (yes vs no) • Prior chemotherapy (yes vs no)

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

N = 270 patients

Planned interim analyses at 50% and 75% of total expected events

*With 7-day lead-in at 90 mg. †Arm B crossover to brigatinib allowed at progression. SOURCE: https://clinicaltrials.gov/show/NCT02737501.

R 1:1

Disease assessment q8w (including brain MRI for all patients)


AP32788 program

Potential orally active TKI precision therapy

Unique profile demonstrated against EGFR and HER2 Exon 20 mutations

No current approved targeted treatments

Estimated 6,000 US patients

Phase I/II clinical study underway

• Initial data expected next year Crystal structure of EGFR in complex with AP32788


Looking ahead: Expanding addressable patient populations

AP32788 NA ~6,000

Potential Future

Eligible US Patient Population (Incidence)

Current/Initial

~1,000-2,000 ~3,000-4,000

Brigatinib ~4,000 ~7,000-8,000


Science & Discovery


ARIAD’s strategy


Science & Discovery





Innovative chemistry

Resistance mapping

Integrated automated assays and informatics

In-house structural biology and biophysics

Structure-based drug design

• Lead optimization

• Library design

Rapid chemical analoging and parallel synthesis

Predictive mutational resistance screens

Tight downstream collaborations, fast handoffs

• DMPK, Pharmacology, Manufacturing

Core strength: Robust and fully integrated discovery platform


ARIAD: Strong internally developed pipeline

* Includes investigator-sponsored trials.

PRECLINICAL PROOF OF CONCEPT

APPROVED

Ponatinib

PIVOTAL

Brigatinib (AP26113) Non-small cell lung cancer (ALK) – Resistant

AP32788 Non-small cell lung cancer (EGFR Exon 20, HER2 Exon 20)

CML, Ph+ ALL (refractory)

AML (FLT3)

Ph+ ALL (with chemo, 1st, 2nd line)

Lung cancer (FGFR, RET)

CML (2nd line)

Non-small cell lung cancer (ALK) – 1st line

CML (dose-ranging)

Immuno-kinase program Undisclosed programs




ARIAD’s strategy


Science & Discovery





Incyte transaction: $65 mil annual operating cost savings

Reinvested in new customer-facing positions

• Doubled the US customer facing roles – 2016 vs. 2015

Focused R&D activities centered on bringing innovative therapies to patients with rare cancers

Shared development costs with partners (Incyte and Otsuka)

Reduced G&A expenses in-line with a leaner, more efficient organization

Lean, cost-efficient structure

All trademarks, service marks, trade names, trade dress, product names and logos are the property of their respective owners.


2016 financial guidance

Iclusig revenue $170 - $180

Selling, general and administrative expenses $120 - $125

Research and development expenses $175 - $180

Operating expenses $295 - $305

Cash, cash equivalents, marketable securities as of 12/31/16

$280 - $290

(dollars in millions)


ARIAD: Catalysts and upcoming milestones

Start 1st line brigatinib

trial

Announced outcomes

of strategic review

Advance AP32788 into development

NDA filed for brigatinib

Received marketing approval of

Iclusig in Japan

Present pivotal ALTA data on brigatinib at

ASCO

Q2 Q3 Q4 H1 2017

Anticipated US launch of

brigatinib

Q1 2016

Initiation of strategic

review

Anticipated product

launch of Iclusig in

Japan

4 year US label update

for Iclusig

ALTA data update at

WCLC

MAA filing for

brigatinib

Brigatinib NDA accepted;

Priority Review

Documents

ARIAD November 2016 Corporate Presentation