2014 Cardiometabolic Projects To Watch Michael C. Rice Senior Consultant, Defined Health

Therapeutic Area Partnerships 2014 November 19-21, 2014 The Hyatt Regency Boston, MA

The information in this report has been obtained from what are believed to be reliable sources and has been verified whenever possible. Nevertheless, we cannot guarantee the information contained herein as to accuracy or completeness. All expressions of opinion are the responsibility of Defined Health, and though current as of the date of this report, are subject to change. The opinions and information set forth herein are expressed solely for the benefit of the addressee and only for the purpose(s) for which the report was produced. Without the prior written consent of Defined Health, this report may not be relied on in whole or in part for any other purpose or by any other person or entity, provided that this report may be disclosed where disclosure is required by law. This report may contain information provided by third parties such as Thomson Reuters, Wolters Kluwer, EvaluatePharma, Datamonitor, Informa Healthcare, IMS Health and others with a proprietary interest in the data provided herein. Please note that you are not permitted to redistribute any such third party information without consent from the originator company. © Defined Health, 2014

Environment Driving This Year’s Selection Criteria ♦ CVD, the once leading pharma market, appears to be stabilizing post generic erosion of

nearly all cardiopreventative drug classes – Showing signs of renewed growth in later years.

♦ During this transitional period, many CVD franchises reallocated R&D portfolios towards

adjacent growth areas such as Type 2 Diabetes and more recently, high need niche areas.

♦ Diabetes is continuing strong growth fueled by demographics and innovation; however,

higher regulatory hurdles, need for CV outcomes and threats from genericization and biosimilars question how long this trajectory can continue.

♦ The obesity segment is expected to be among the highest growth segments in the pharma

industry; however, despite the enormous prevalence it’s growing off a tiny revenue base and the market is just being shaped with recently approved agents.

♦ Even with such progress, unmet need remains: Downstream result of Diabetes and Obesity

is worsening heart disease, macrovascular complication and significant M&M.

♦ However, it is increasingly difficult to prove outcomes benefits of new cardiometabolics

drugs in late-stage trials on top of the SOC Generic – Many failed CV outcomes trials

♦ Investment and deal making in cardiometabolics has been anemic and accordingly, there is

a dearth of novel clinical stage assets.

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Stabilizing Post Patent Cliff, CVD Is Still a Sizable Market; While Metabolics Is Steadily Growing – Nearing Parity With The Entire CV Market ♦ Total WW Pharmaceutical Sales is expected to grow by $238B to an estimated $835B by 2020 ♦ Overall, the “cardiometabolics” segment will grow to $80B by 2020, 18% of the overall drug sales. 200,000 180,000

$WW Sales Millions

160,000 140,000 120,000 100,000 80,000 60,000 40,000 20,000 0

Worldwide Sales by Therapy Area, 2004-2020 CV Diabetes Obesity CNS Genito-Urinary Musculo-skeletal Oncology Respiratory Systematic Antiinfectives Others

EvaluatePharma

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Antidiabetics Leading Growth in the “Cardiometabolics” Segment – Generic Cardioprevention Now a “Medical Bargain” for Outcomes Benefit ♦ Cardiovascular revenue is predicted to remain stagnant through 2020 while Diabetes and

Obesity revenue are predicted to grow significantly.

♦ However, some new drug classes are starting to showing signs of renewed growth in later

years: PCSK9s, Factor Xa, LCZ696 is an ARNI (Angiotensin Receptor Neprilysin Inhibitor)

70,000

Total Worldwide CVD & Metabolics* Therapeutic Category Sales, 2000-2020

$WW Sales Millions

60,000 50,000

Anti-hypertensives Anti-hyperlipidaemics Cardiac therapy

40,000

Platelet inhibitors

30,000

Anticoagulants

20,000

Other cardiovasculars

10,000 0

Anti-diabetics Anti-obesity * Metabolics sales 2004-2020

EvaluatePharma

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Breakthrough Therapies have Improved Outcomes, Yet CVD Remains the Highest Unmet Therapeutic Need in Terms of M&M Number of deaths (Thousands)

700

Leading Causes of Death Annually in the US (2011)

600 500 400

Despite increased chance of survival of acute events, heart disease is still the leading cause of death for both women and men in the United States

300 200 100 0

♦ Major breakthroughs in the treatment and

prevention of heart disease led to steady improvements in CV outcomes; however, CVD remains most prevalent of all diseases:

• ~60M+ Americans and 100M+ people in the

developed world suffer from forms of CVD

• Number one cause of death globally. ~17.3

million CVD deaths (30% of global deaths).

World Health Statistics 2014, 2013, and 2011, CDC.gov

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♦ Ischemic heart and cerebrovascular diseases

are most common causes of morbidity and mortality:

• ~20% of individuals aged > 40 years are

predicted to die within 1 year of initial MI • ~20% of ACS patients are re-hospitalized within 1 year; nearly 60% of costs result from re hospitalization ♦ Cost of CVD in the US is more than half a

trillion dollars, escalating cost of health care. Page 6

Cardiometabolic Risk Factors Act In Concert Promoting CHD and Resulting Morbidity and Mortality ♦ Hypertension, Dyslipidemia, Insulin Resistance, Inflammation, Environment/Lifestyle and

Genetics all contribute to endothelial dysfunction and development of CHD

♦ As diabetes is a significant risk factor for CVD events, the epidemic of obesity and T2DM will

increase future M&M

AHA Scientific Sessions 2011, S4; J Am Osteopath Assoc. 2007;107(suppl 2):Inside front cover–S3

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ToC

Page 7

T2DM is a Challenging Health Problem in the 21st Century It is One of the Most Common Non-Communicable Diseases Globally ♦ Global prevalence of diabetes 371M &

expected to reach 552M by 2030

• diabetes increasing in all parts of the world yet

nearly 50% of people globally undiagnosed

♦ 43% (201 Billion USD) of Global Healthcare

Expenditure Due To Diabetes in 2011 Was Generated In USA

• Large disparity in healthcare spending on

diabetes between regions and countries.

• Only 20% of global healthcare expenditures

due to diabetes were made in low- and middle-income countries, where 80% of people with diabetes live.

International Diabetes Federation

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>80% of Diabetics in 7 Major Markets are Overweight or Obese, Often Comorbid with T2DM, Hypertension and Dyslipidemia ♦ Developed countries, including U.S. and Western Europe, and women, exhibit highest rates of Obesity,

defined as body mass index (BMI) above 30

♦ A remarkable one-third of the US adult population (~75MM individuals) meet clinical definition of

Obesity (BMI>30). Another one-third is overweight (BMI 25-30).

♦ Obesity rates continue to rise steadily in other developed and developing countries of the world.

Percent of Adults, Aged 15 and Older, with a BMI >= 30

Prevalence of Obesity by Country and Gender in 2010 40% 35%

Male

Female

30% 25% 20% 15% 10% 5% 0%

OECD (2014), OECD Health Statistics 2014

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Cost of Diabetes Complications CVD Complications Generate The Greatest % Cost Of Chronic Diabetes Management ♦ Much of the morbidity and cost of diabetes management is attributable to long-term,

diabetes-related complications, particularly cardiovascular disease (CVD).

Defined Health Research

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To Address Residual Risk, Pharma Has Many Shots on Goal Towards Introducing New Drug Classes and Proving CV Outcomes Improvement

CV agents

Timeline of Anticipated Launches Late Stage CV & Diabetes Drugs With Outcomes Data*

Launch Date

Statin/CAI

DPP-IV

Niacin

GLP-1

SGLT2

CETP

Lp-PLA2

PPAR

ApoB 100

MTP

PCSK9

Omega-3

2009

2010

AMG145 Launch

T2DM agents

Vytorin (IMPROVE-IT, Outcomes)

Juxtapid HoFH Launch

2011

2012

Onglyza US Launch

Victoza US Launch

Estimated Anacetrapib US Launch

Kynamro HoFH Launch

Tanzeum (albiglutide) US Launch

2013

Invokana Canaglflozin US Launch

Nesina (alogliptin) US Launch

REGN 727 Launch

2014

2015

Vascepa Reduce-It

2016

2017

Victoza LEADER Trial

Jardiance empagliflozi US Launch

Dapagliflozin US Launch

Estimated Evacetrapib US Launch

Januvia CV Outcome Study TECOS

Defined Health, EvaluatePharma, *HoFH launches based on surrogate only

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Post Patent Cliff, Many Pharma Companies Announced Plans to Reallocate R&D $ To Higher Growth Areas

Expected Sales Growth CAGR (2013-2020)

Obesity Obesity has a CAGR of 28% between 20132020

30% 25% 20% 15% 10%

Oncology

Other CVD yielded doubledigit growth 5% experienced since CNS CVD the 1980s. 0% -10% -5% 0% -5% -10%

EvaluatePharma

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Anti-infectives

Respiratory 5% 10% Genitourinary

Diabetes Musculoskeletal 15%

20%

25%

30%

Historical Sales Growth CAGR (2005-2012) Bubble size correlates to 2013 WW Therapeutic Area Sales

Page 12

Metabolics and CVD Deals Lag Other Large Therapeutic Areas

350

Number of Deals From 2012 to 2014 by Therapeutic Area

300 2014

Number of Deals

250

2013 2012

200 150 100 50 0

Metabolics

CVD

Cancer

CNS

Infectious & Musculoskeletal viral disease

Respiratory

Opthamology

Informa

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Majority of CVD Deals Occur After Phase III PoC; Whereas Most Metabolics Deals Based on Preclinical Evidence (Exception of Obesity) Latest Development Phase At Time of Deal Cardiovascular

Metabolics

Preclinical 16%

Marketed 19%

Phase I 5%

Phase III/Filed 7%

Phase II 8% Phase III/Filed 11%

Marketed 60%

Preclinical 62%

Phase II 12%

Informa

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Dearth of CV and Metabolic Agents in Both Preclinical and Clinical Development Compared to Other TAs Number of Drugs in Development by Therapeutic Category 10000 9000 8000

Number of Drugs

7000 6000

CVD Obesity Genito-Urinary Oncology Systematic Anti-infectives

Diabetes CNS Musculo-skeletal Respiratory Others

5000 4000 3000 2000 1000

CVM

0

Filed

Phase III

Phase II

Phase I

Preclinical

Research

Thomson Reuters Cortellis, ADIS R&D Insight

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Pharma Doing Early Stage Deals to Enrich Cardiometabolic Clinical Pipeline Alliance

Type

Area (Phase)

Upfront (Mlns)

Total (Mlns)

Notes

Sanofi/ MyoKardia (Sept 2014)

Collaboration, Translation

Targeted therapies for patients with genetic forms of cardiomyopathy

$45.0

$200.0

• Collaboration represents one of the largest R&D commitments to genetic forms of cardiomyopathy and covers three MyoKardia programs: two for hypertrophic cardiopathy (HCM) and one for dilated (DCM) cardiomyopathy.

Licensing fee and an initial equity investment.

Equity investments, milestone payments and R&D services through 2018

• Sanofi and MyoKardia will equally share development costs on the HCM programs following initial demonstration of efficacy. • Sanofi will fully cover the development costs of the DCM program.

AstraZeneca/ Moderna (March 2013)

Collaboration

Messenger RNA therapeutics for the treatment of serious cardiovascular, metabolic, and renal diseases and cancer

$240.0

$420.0

Three technical milestone payments

• AstraZeneca will have exclusive access to select a target of its choice in cardiometabolic diseases over a period of five years for subsequent development of messenger RNA • AZ will lead the preclinical, clinical, development and commercialization of therapeutics resulting from the agreement. • Moderna will be responsible for designing and manufacturing the messenger RNA against selected targets

Company website, FiercePharma

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Dominant CV Franchises Changing Leadership – New Franchise Entering Specific Segments Fragmenting the Market ♦ Leading CV franchises such as Pfizer’s will become smaller in the next few years as the result of expired

brands and lack of replacements.

♦ At the same time, multiple new entrants to the CV market (AZ, Janssen, Amgen, D-S) will influence

change towards reshaping the marketplace as broad indications become sub-segmented.

♦ The epidemic of T2DM and obesity will contribute to CVD burden and the silo approach by Cardiologists

and Endocrinologists to treat high risk patients may change as well.

Cardiovascular Category Market Share By $ Sales 2013: $78B AZN Merck Novartis Sanofi Pfizer Daiichi Sankyo Boehringer Ingelheim Servier Takeda Astellas Other

2020e: $76B 10%

11% 9%

38%

9% 44%

8%

8%

7%

7% 3% 3% 4%

4% 6%

7%

5% 3% 3% 3% 3%

5%

Novartis Sanofi AZN Pfizer Actelion Bayer Servier Takeda Merck Daiichi Sankyo Other

Cowen Therapeutic Categories, Oct 2014, EvaluatePharma

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Metabolics Category Market Share ♦ In 2013, Novo Nordisk, Sanofi, Merck, and Eli Lilly led the $36B diabetes category. ♦ In 2019, we forecast that these same four companies plus AstraZeneca (post its January 2014

acquisition of Bristol’s diabetes assets) will dominate a projected $56B market, driven by growth of their insulins , GLP-1 products and DPPIVs.

♦ SGLT-2s. are the latest OAD entrant. JNJ’s Invokana, AZN’s Forxiga and Lilly/BI’s Jardiance were all

approve in the last year.

Metabolics Category Market Share By $ Sales 2013: $38B Novo Nordisk Sanofi Merck & Co Eli Lilly Bristol-Myers Squibb

3% 3% 4%

2020e: $68B

13% 30%

Takeda

Novo Nordisk

14% 29%

4%

Other

22%

Merck & Co AstraZeneca Boehringer Ingelheim

6% 15%

Sanofi Eli Lilly

5%

10%

Novartis

2% 3%

16%

10% 11%

Johnson & Johnson Novartis Takeda Other

Cowen Therapeutic Categories, Oct 2014, EvaluatePharma

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2014 Cardiometabolic Projects To Watch

Therapeutic Area Partnerships 2014 © Defined Health

Page 19

The Initial List of CVD and Metabolics Agents in Development was Compiled From Licensed Databases and Industry Experience

Create CVD + Met Investigational Agent Database

Remove Unavailable Agents

(Phase I to registered)

1

Adis R&D Insight

Evaluate Based on Selection Criteria Thomson Reuters Cortellis

Compare and Prioritize

Invite Selected Companies

Defined Health Ad-hoc Additions

Combine, Remove Redundancies

2

1,110 CVD + Met Agents in Active Clinical Development WW

Defined Health analysis

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Diabetes Projects Represent Half the Pipeline – Most CVD Agents in Development for Thrombosis, HTN and CAD Number of CVD and Metabolics Drugs in Development by Indication 600

1,110 CVD + Met Agents in Active Clinical Development WW 500 Diabetes Number of Drugs

400

Obesity Thrombosis/ Vascular Hypertension

300

Heart failure 200

CAD/Dyslipidemia Arrythmia

100

ACS

0 Registered

Phase III

Phase II

Phase I

Thomson Reuters Cortellis, ADIS R&D Insight

Therapeutic Area Partnerships 2014 © Defined Health

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Projects Developed by Big Pharma or Companies with Substantial CVD or Metabolics Development Capabilities Were Deprioritized in the Database

1,100 CVD + Met Agents in Ph I to Registration WW

Create CVD Investigational Agent Database

(Phase I to registered)

809 Available Agents

Remove Unavailable Agents

495 Optimistic Projects

Evaluate Based on Selection Criteria

Top CVD & Metabolics Projects selected

Compare and Prioritize

CVD & Metabolics Top 10 Projects

Invite Selected Companies

Originator / Developer • Big Pharma • Specialty Franchise

Defined Health analysis

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While Pharma Controls More of the Metabolics Pipeline, Nearly 3/4 of All Cardiometabolics Drugs Are Being Developed by Biotech CVD Drugs in Development by Company Type

Metabolics Drugs in Development by Company Type

Pharma 22%

Biotech 78%

Pharma 32%

Biotech 68%

Thomson Reuters Cortellis, ADIS R&D Insight

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Projects were Rated Based on Selection Criteria Indicating Project Attractiveness to a Potential Partner

1,100 CVD + Met Agents in Ph I to Registration WW

Create CVD Investigational Agent Database

(Phase I to registered)

809 Available Agents

Remove Unavailable Agents

495 Optimistic Projects

Evaluate Based on Selection Criteria

Top CVD & Metabolics Projects selected

Compare and Prioritize

CVD & Metabolics Top 10 Projects

Invite Selected Companies

Intrinsic to Agent • Unmet Needs • Market Potential • Novelty • Precedents • Clinical • Competition

Defined Health analysis

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Projects were Compared Among Their Peers Based On Company Attributes and Advertised Availability

1,100 CVD + Met Agents in Ph I to Registration WW

Create CVD Investigational Agent Database

(Phase I to registered)

809 Available Agents

Remove Unavailable Agents

Top 20 CVD & Metabolics Projects selected

495 Optimistic Projects

Evaluate Based on Selection Criteria

CVD & Metabolics Top 10 Projects

Compare and Prioritize

• • • • •

Invite Selected Companies

Amongst Peers: Exclude Prior Top 10 unless clear progress achieved. Exclude DH current client target Company Management WW Availability “For out-license”

Defined Health analysis

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Page 25

Companies Making the Final List were Invited

Create CVD Investigational Agent Database

(Phase I to registered)

Remove Unavailable Agents

Evaluate Based on Selection Criteria

Compare and Prioritize

Invite Selected Companies

Defined Health analysis, Company websites

Therapeutic Area Partnerships 2014 © Defined Health

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2014 Cardiometabolic Projects To Watch* Agent

Company

MOA

Indication

Phase Comments

CER 001

Cerenis Therapeutics

Apolipoprotein A I stimulants

FPHA ACS

Phase II

ApoAI repletion therapy.orphan designated for FPHA where ultralow ApoAI defines the disease.

ETC 1002

Esperion Therapeutics

AMP activated protein kinase stimulants

CAD, dyslipidemia

Phase II

first-in-class drug designed to LDL-C and improve other cardiometabolic risk in statin intolerant patients

PRC 4016

Pronova Biopharma (BASF)

Structurally Enhanced Fatty Acid

Mixed dyslipidemia Hypertriglyceridemia

Phase II

Best-in-class Omega-3 drug that reduces LDL-C, non-HDL-C, total cholesterol, triglycerides and ApoB

JVS 100

Juventas Therapeutics

SDF-1, CXCR4 receptor agonists

Heart failure

Phase II

improves cardiac function in advanced heart failure more than a decade after having a heart attack by promoting tissue repair.

Ralinepag (APD-811)

Arena Pharmaceuticals

Prostacyclin (IP) receptor agonists

Pulmonary Arterial Hypertension

Phase I

An orally available agonist of the prostacyclin (IP) receptor, orphan designated for PAH

GKM-001/2

Advinus Therapeutics

Glucokinase Activator

Phase II

Liver selective - Reduces blood glucose levels by increased hepatic glucose uptake.

Phase I/II

Clinical evidence of normalizing HbA1c without inducing hypoglycemia in T2DM subjects on stable doses of metformin.

TTP 399

TransTech Pharma

*Not presented in any particular order

Therapeutic Area Partnerships 2014 © Defined Health

Glucokinase Activator

Type 2 Diabetes

Type 2 Diabetes

Page 27

Roundtable Discussion: Creating New Opportunities and Partnerships in CV/Metabolic Drug Programs Moderator: • Mike Rice MS, MBA, Senior Consultant, Defined Health Panelists: • John Delyani, Executive Director, Business Development & Licensing, Merck & Co. • Barry Ticho MD, PhD, Head, Cardiovascular and Metabolic Disorder & ERDI, Pfizer • Steve Cohen MD, Executive Director ,External Scientific Affairs, Daiichi Sankyo • Tomas Landh, Director, Strategy & Innovation Sourcing, Novo Nordisk Therapeutic Area Partnerships 2014 November 19-21, 2014 The Hyatt Regency Boston, MA

2014 Cardiometabolic Projects To Watch

Company Profiles

Therapeutic Area Partnerships 2014 © Defined Health

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CERENIS

Company Profile Company Overview Company

Cerenis Therapeutics

Location

Labege, Toulouse, France

Description

Cerenis is a multinational biotechnology company dedicated to the discovery, development, and commercialization of novel HDL therapies for the treatment of cardiovascular and metabolic diseases.

Partners

In cardiology, Cerenis seeks to retain codevelopment and co-promotion rights in order to evolve to a commercial-stage specialty pharmaceutical company

Company Overview

Financial

In 2010 the company raised €50 million in Series C financing. In October 2010 the total equity investment raised to date was 117 million Euros.

Cerenis is also exploring partnering options and ways to collaborate on non-core strategic assets outside of cardiology. Technology Platform • Project to Watch: CER 001 for CAD/dyslipidemia, Phase II • Cerenis is developing a broad pipeline of HDL therapies to address the needs of a variety of patients in both the sub-acute and chronic care CVD markets. • The lead product is CER-001, a recombinant high-density lipoprotein (HDL) mimetic: a complex of a human apolipoprotein A-I (ApoA-I) and phospholipids. • The product is designed to rapidly regress atherosclerotic plaque among patients at high risk of cardiovascular events. Company website, Google Finance, EvaluatePharma

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ESPERION

Company Profile Company Overview Company

Esperion Therapeutics

Location

Ann Arbor, MI

Description

Esperion is a pharmaceutical company focused on the development and commercialization of novel therapies for the treatment of patients with elevated levels of low-density lipoprotein cholesterol (LCL-C) and other cardiometabolic risk factors

Partners

Company Overview

Financial

Market Cap: $590.35M (as of 4-Nov-2014) No sales data available

Esperion is interested in partnering but currently has no industry partners

Technology Platform • Project to Watch: ETC-1002 (Bempedoic acid) for CAD/dyslipidemia, Phase II • Esperion’s lead product candidate is an orally available, once-daily small molecule designed to lower LDL-cholesterol levels and avoid the side effects associated with currently available therapies. • ETC-1002 has a dual mechanism of action with the potential to regulate both lipid and carbohydrate metabolism and is in development primarily for patients with hypercholesterolemia and a history of statin intolerance

Company website, Google Finance, EvaluatePharma

Therapeutic Area Partnerships 2014 © Defined Health

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PRONOVA (BASF) Company Profile Company Overview Company

Pronova Biopharma Norge AS (part of BASF Omega3 Verbund)

Location

Oslo, Norway

Description

The BASF acquisition of Pronova Biopharma (2013), Equateq (2012), and Cognis (2010) has made BASF a global market leader for omega-3 fatty acids. The newly created portfolio now covers the full range of omega-3 fatty acids for pharmaceuticals, dietary supplements and clinical nutrition

Partners

BASF’s pharmaceutical business is operated through Pronova BioPharma.

Company Overview Financial

Market Cap (BASF): $63,6B (as of 12-Nov-2014)

Sales

BASF acquired Pronova BioPharma for approximately $657M (NOK 3760m) in cash at price of $2.186/share (NOK 12.50/share).

Technology Platform • Project to Watch: PRC 4016 for CAD/dyslipidemia, Phase II • Pronova BioPharma was a Norway based pharmaceutical company focused on development & manufacturing of marine-originated omega-3 derived pharmaceutical products. • PRC 4016 is a structurally enhanced omega-3 fatty acid with potent triglyceride and cholesterol-lowering effects and has been shown to prevent the development of atherosclerosis and improve liver steatosis in a CETP mouse model of mixed dyslipidaemias. • PRC 4016 may have better efficacy than currently approved drugs, with a broader therapeutic window and appears to have a better safety profile than other lipid-lowering non-fatty acid based products. Company website, Google Finance, EvaluatePharma

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Juventas Therapeutics Company Profile Company Overview Company

Juventas Therapeutics

Location

Cleveland, Ohio

Description

Juventas Therapeutics is a private clinical-stage biotechnology company focused on the development of therapies for ischemic cardiovascular disease. In 2011, Juventas Therapeutics spun-off SironRX Therapeutics to focus on development of dermal and bone related applications for JVS-100.

Partners

In addition to its clinical programs, Juventas has on-going collaborative research programs with Cleveland Clinic, Center for Stem Cell & Regenerative Medicine, Global Cardiovascular Innovation Center, Summa Health System, and SironRX Therapeutics.

Company Overview

Financial

In 2012 the company raised $22.2 Million in Series B financing co-led by Triathalon Medical Venture Partners and New Science Ventures. All previous venture firms participated in the round. New investors included Takeda Ventures, Venture Investors, Global Cardiovascular Innovation Center, Tri-State Growth Fund, Glengary and select angel investors. The company has received non-dilutive grant support through Cleveland Clinic, Ohio BioValidation Fund, Global Cardiovascular Innovation Center and Center for Stem Cell & Regenerative Medicine.

Technology Platform • Project to Watch: JVS-100 for Heart failure, Phase II • JVS-100, is a non-viral DNA plasmid that encodes from stromal cell-derived factor-1 (SDF-1). SDF-1 has been shown to significantly increase end-organ function following tissue injury by promoting cell survival, recruiting endogenous stem cells to the damaged region and promoting new blood vessel growth. Company website, Google Finance, EvaluatePharma

Therapeutic Area Partnerships 2014 © Defined Health

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Arena Pharmaceuticals Company Profile Company Overview

Company Overview

Company

Arena Pharmaceuticals

Financial

Location

San Diego, CA

Description

Arena Pharmaceuticals is a biopharmaceutical company focused on discovering, developing and commercializing novel drugs that target G proteincoupled receptors (GPCRs).

Partners

Market Cap: $981M (as of 12-Nov-2014)

Sales

Arena Pharmaceuticals has granted exclusive marketing and distribution rights for Belviq to Eisai for most worldwide territories.

Technology Platform • Project to Watch: APD-811 for Hypertension, Phase II • APD-811 is the lead from orally-active non-prostanoid prostacyclin (IP) receptor agonists, identified using the company's Constitutively Activated Receptor Technology (CART) technology, as an once-daily formulation • Approach allows identification of drug leads that act as receptor agonists or inhibitors, inverse agonists, and ligand-dependent, receptor activity. • The platform could eliminate the need to identify the native ligand for an orphan receptor and provide the ability to discover novel and improved therapeutics directed at known receptors. Company website, Google Finance, EvaluatePharma

Therapeutic Area Partnerships 2014 © Defined Health

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Advinus Therapeutics Company Profile Company Overview Company

Advinus Therapeutics

Location

Bengaluru, India

Description

A research-based pharmaceutical company and first of its kind in India to offer end-to-end development services to the global Pharma, Agro and Biotech industries. Advinus is focused on developing therapeutics in the areas of metabolism, inflammation, pain & neglected diseases.

Partners

Advinus has established successful partnership with global pharma companies such as Merck, Novartis, J&J and DNDi.

Company Overview The TATA Group is a rapidly growing business group based in India. The total revenue of Tata companies was $70.8 billion in 2008-09, with 64.8% coming from business outside India Financial

In 2012 Takeda and Advinus entered into an agreement to initiate a three-year discovery collaboration. Advinus will receive guaranteed research funding of $36 million over the term of the collaboration, $9 million in milestones leading up to candidate selection, and is eligible to receive future clinical and regulatory milestones payments of up to $45 million per product.

Technology Platform • Project to Watch: GKM-002 for Diabetes, Phase II • GKM-002, a back-up compound of GKM-001, is in development for the potential treatment of type 2 diabetes. • Diabetes approach addresses glucose overproduction, peripheral insulin resistance, and insulin deficiency. • Obesity platform focuses on mechanisms involved in abnormal energy intake and expenditure or excessive energy storage to provide safe therapies for weight loss.

Company website, Google Finance, EvaluatePharma

Therapeutic Area Partnerships 2014 © Defined Health

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TransTech Pharma Company Profile Company Overview

Company Overview

Company

TransTech Pharma

TransTech Pharma is a privately held company.

Location

High Point, NC

Description

TransTech Pharma is a clinical-stage pharmaceutical company focused on the discovery and development of human therapeutics. The pipeline includes clinical and preclinical small molecule drug candidates for the treatment of CNS disorders, diabetes and metabolic disorders, inflammation and oncology.

• 2003: TransTech announced the completion of two round of investments totaling $34.5M

Partners

TransTech has strategic alliances with Novo Nordisk, Cephalon, Forest Laboratories, Siga, Merck, Boehringer Ingelheim, and Pfizer.

Financial

• 2005: TransTech and SIGA Technologies announced the receipt of a $2.7M challenge grant from the NIH • 2010: TransTech and High Point Pharma were awarded cash grants totaling $1.96M from the US Government’s Qualifying Therapeutic Discovery Project (QTDP) program

Technology Platform • Project to Watch: TTP 399 for Diabetes, Phase I/II • TTP 399 is an orally available, small molecule glucokinase activator (GKA) for the treatment of type 2 diabetes mellitus • The proprietary drug discovery engine TTP Translational Technology is a highly automated and fully integrated drug discovery process, which translates genomic & proteomic data into safe and effective small molecule therapeutics in high throughput fashion. • Three modules of TPP Translational Technology: TTPredict, TTPSpace, and TTPScreen that are integrated with an informatics system that captures data from each optimization cycle of the drug discovery process. According to the company, TTP Translational Technology realizes higher success rates in the development phase of compounds selected versus compounds discovered traditionally. Company website, Google Finance, EvaluatePharma

Therapeutic Area Partnerships 2014 © Defined Health

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Roundtable Discussion: Creating New Opportunities and Partnerships in CV/Metabolic Drug Programs Moderator: • Mike Rice MS, MBA, Senior Consultant, Defined Health Panelists: • John Delyani, Executive Director, Business Development & Licensing, Merck & Co. • Barry Ticho MD, PhD, Head, Cardiovascular and Metabolic Disorder & ERDI, Pfizer • Steve Cohen MD, Executive Director ,External Scientific Affairs, Daiichi Sankyo • Tomas Landh, Director, Strategy & Innovation Sourcing, Novo Nordisk Therapeutic Area Partnerships 2014 November 19-21, 2014 The Hyatt Regency Boston, MA