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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
Page 3 Therapeutic Area Partnerships 2014 © Defined Health
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.
Page 4 Therapeutic Area Partnerships 2014 © Defined Health
Stabilizing Post Patent Cliff, CVD Is Still a Sizable Market; While Metabolics Is Steadily Growing – Nearing Parity With The Entire CV Market
0
20,000
40,000
60,000
80,000
100,000
120,000
140,000
160,000
180,000
200,000
$WW
Sal
es M
illio
ns
Worldwide Sales by Therapy Area, 2004-2020 CV
Diabetes
Obesity
CNS
Genito-Urinary
Musculo-skeletal
Oncology
Respiratory
Systematic Anti-infectives Others
EvaluatePharma
♦ 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.
Page 5 Therapeutic Area Partnerships 2014 © Defined Health
Antidiabetics Leading Growth in the “Cardiometabolics” Segment – Generic Cardioprevention Now a “Medical Bargain” for Outcomes Benefit
0
10,000
20,000
30,000
40,000
50,000
60,000
70,000
$WW
Sal
es M
illio
ns
Total Worldwide CVD & Metabolics* Therapeutic Category Sales, 2000-2020
Anti-hypertensives
Anti-hyperlipidaemics
Cardiac therapy
Platelet inhibitors
Anticoagulants
Other cardiovasculars
Anti-diabetics
Anti-obesity
EvaluatePharma
* Metabolics sales 2004-2020
♦ 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)
Page 6 Therapeutic Area Partnerships 2014 © Defined Health
Breakthrough Therapies have Improved Outcomes, Yet CVD Remains the Highest Unmet Therapeutic Need in Terms of M&M
♦ 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
♦ 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.
0
100
200
300
400
500
600
700
Num
ber o
f dea
ths (
Thou
sand
s)
Leading Causes of Death Annually in the US (2011)
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
Page 7 Therapeutic Area Partnerships 2014 © Defined Health
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
ToC
Page 8 Therapeutic Area Partnerships 2014 © Defined Health
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
International Diabetes Federation
♦ 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.
Page 9 Therapeutic Area Partnerships 2014 © Defined Health
>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.
OECD (2014), OECD Health Statistics 2014
0%
5%
10%
15%
20%
25%
30%
35%
40%
Perc
ent o
f Adu
lts, A
ged
15 a
nd O
lder
, w
ith a
BM
I >=
30
Prevalence of Obesity by Country and Gender in 2010
Male Female
Page 10 Therapeutic Area Partnerships 2014 © Defined Health
Cost of Diabetes Complications
♦ Much of the morbidity and cost of diabetes management is attributable to long-term, diabetes-related complications, particularly cardiovascular disease (CVD).
CVD Complications Generate The Greatest % Cost Of Chronic Diabetes Management
Defined Health Research
Page 11 Therapeutic Area Partnerships 2014 © Defined Health
To Address Residual Risk, Pharma Has Many Shots on Goal Towards Introducing New Drug Classes and Proving CV Outcomes Improvement
Defined Health, EvaluatePharma, *HoFH launches based on surrogate only
Timeline of Anticipated Launches Late Stage CV & Diabetes Drugs With Outcomes Data*
2010 2011 2012 2013 2014 2015 2016 2017
Victoza US Launch
Launch Date
Dapagliflozin US Launch
Invokana Canaglflozin US Launch
Nesina (alogliptin) US Launch
2009
Onglyza US Launch
CV a
gent
s T2
DM a
gent
s
Estimated Anacetrapib US Launch PPAR
Niacin
CETP
Lp-PLA2
GLP-1
DPP-IV
SGLT2 Estimated Evacetrapib US Launch
Statin/CAI
Vytorin (IMPROVE-IT,
Outcomes) PCSK9
ApoB 100
Kynamro HoFH Launch
MTP Juxtapid HoFH Launch
AMG145 Launch
REGN 727 Launch
Omega-3
Vascepa Reduce-It
Victoza LEADER Trial
Jardiance empagliflozi US Launch Tanzeum
(albiglutide) US Launch
Januvia CV Outcome Study TECOS
Page 12 Therapeutic Area Partnerships 2014 © Defined Health
Post Patent Cliff, Many Pharma Companies Announced Plans to Reallocate R&D $ To Higher Growth Areas
Bubble size correlates to 2013 WW Therapeutic Area Sales
CVD
Diabetes
Obesity
CNS
Genitourinary
Musculoskeletal
Oncology
Respiratory
Anti-infectives Other
-10%
-5%
0%
5%
10%
15%
20%
25%
30%
-10% -5% 0% 5% 10% 15% 20% 25% 30%
Expe
cted
Sal
es G
row
th C
AGR
(201
3-20
20)
Historical Sales Growth CAGR (2005-2012)
CVD yielded double-digit growth experienced since the 1980s.
Obesity has a CAGR of 28% between 2013-2020
EvaluatePharma
Page 13 Therapeutic Area Partnerships 2014 © Defined Health
Metabolics and CVD Deals Lag Other Large Therapeutic Areas
0
50
100
150
200
250
300
350
Metabolics CVD Cancer CNS Infectious & viral disease
Musculoskeletal Respiratory Opthamology
Num
ber o
f Dea
ls
Number of Deals From 2012 to 2014 by Therapeutic Area
2014
2013
2012
Informa
Page 14 Therapeutic Area Partnerships 2014 © Defined Health
Majority of CVD Deals Occur After Phase III PoC; Whereas Most Metabolics Deals Based on Preclinical Evidence (Exception of Obesity)
Informa
Marketed 19%
Phase III/Filed
7%
Phase II 12%
Preclinical 62%
Metabolics
Marketed 60% Phase
III/Filed 11%
Phase II 8%
Phase I 5%
Preclinical 16%
Cardiovascular
Latest Development Phase At Time of Deal
Page 15 Therapeutic Area Partnerships 2014 © Defined Health
Dearth of CV and Metabolic Agents in Both Preclinical and Clinical Development Compared to Other TAs
0
1000
2000
3000
4000
5000
6000
7000
8000
9000
10000
Filed Phase III Phase II Phase I Preclinical Research
Num
ber o
f Dru
gs
Number of Drugs in Development by Therapeutic Category
CVD Diabetes Obesity CNS Genito-Urinary Musculo-skeletal Oncology Respiratory Systematic Anti-infectives Others
Thomson Reuters Cortellis, ADIS R&D Insight
CVM
Page 16 Therapeutic Area Partnerships 2014 © Defined Health
Pharma Doing Early Stage Deals to Enrich Cardiometabolic Clinical Pipeline
Company website, FiercePharma
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 Licensing fee and an initial equity investment.
$200.0 Equity investments, milestone payments and R&D services through 2018
• 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.
• 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
Page 17 Therapeutic Area Partnerships 2014 © Defined Health
10%
9%
8%
7%
5% 5%
3% 3% 3% 3%
44%
Novartis Sanofi AZN Pfizer Actelion Bayer Servier Takeda Merck Daiichi Sankyo Other
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.
Cowen Therapeutic Categories, Oct 2014, EvaluatePharma
Cardiovascular Category Market Share By $ Sales 2013: $78B 2020e: $76B
11%
9%
8%
7%
7% 6% 4%
4% 3% 3%
38%
AZN Merck Novartis Sanofi Pfizer Daiichi Sankyo Boehringer Ingelheim Servier Takeda Astellas Other
Page 18 Therapeutic Area Partnerships 2014 © Defined Health
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.
Cowen Therapeutic Categories, Oct 2014, EvaluatePharma
29%
16%
11%
10%
6%
5%
4%
3% 2% 14%
Novo Nordisk Sanofi Merck & Co Eli Lilly AstraZeneca Boehringer Ingelheim Johnson & Johnson Novartis Takeda Other
Metabolics Category Market Share By $ Sales 2013: $38B 2020e: $68B
30%
22% 15%
10%
4% 3%
3% 13% Novo Nordisk
Sanofi
Merck & Co
Eli Lilly
Bristol-Myers Squibb
Novartis
Takeda
Other
Page 19 Therapeutic Area Partnerships 2014 © Defined Health
2014 Cardiometabolic Projects To Watch
Page 20 Therapeutic Area Partnerships 2014 © Defined Health
Remove Unavailable
Agents
Evaluate Based on Selection
Criteria
Compare and Prioritize
Create CVD + Met Investigational Agent Database (Phase I to registered)
Invite Selected
Companies
Adis R&D Insight 1
2
Thomson Reuters Cortellis
Defined Health Ad-hoc Additions
1,110 CVD + Met Agents in Active Clinical Development WW
The Initial List of CVD and Metabolics Agents in Development was Compiled From Licensed Databases and Industry Experience
Combine, Remove
Redundancies
Defined Health analysis
Page 21 Therapeutic Area Partnerships 2014 © Defined Health
Diabetes Projects Represent Half the Pipeline – Most CVD Agents in Development for Thrombosis, HTN and CAD
0
100
200
300
400
500
600
Registered Phase III Phase II Phase I
Num
ber o
f Dru
gs
Number of CVD and Metabolics Drugs in Development by Indication
Diabetes
Obesity
Thrombosis/ Vascular Hypertension
Heart failure
CAD/Dyslipidemia
Arrythmia
ACS
Thomson Reuters Cortellis, ADIS R&D Insight
1,110 CVD + Met Agents in Active Clinical Development WW
Page 22 Therapeutic Area Partnerships 2014 © Defined Health
Remove Unavailable
Agents
Evaluate Based on Selection
Criteria
Compare and Prioritize
Create CVD Investigational Agent Database (Phase I to registered)
Invite Selected
Companies
1,100 CVD + Met Agents in Ph I to Registration WW
809 Available Agents
495 Optimistic Projects
Top CVD & Metabolics
Projects selected
CVD & Metabolics
Top 10 Projects
Projects Developed by Big Pharma or Companies with Substantial CVD or Metabolics Development Capabilities Were Deprioritized in the Database
Originator / Developer • Big Pharma • Specialty Franchise
Defined Health analysis
Page 23 Therapeutic Area Partnerships 2014 © Defined Health
While Pharma Controls More of the Metabolics Pipeline, Nearly 3/4 of All Cardiometabolics Drugs Are Being Developed by Biotech
Pharma 22%
Biotech 78%
CVD Drugs in Development by Company Type
Pharma 32%
Biotech 68%
Metabolics Drugs in Development by Company Type
Thomson Reuters Cortellis, ADIS R&D Insight
Page 24 Therapeutic Area Partnerships 2014 © Defined Health
Remove Unavailable
Agents
Evaluate Based on Selection
Criteria
Compare and Prioritize
Create CVD Investigational Agent Database (Phase I to registered)
Invite Selected
Companies
1,100 CVD + Met Agents in Ph I to Registration WW
809 Available Agents
495 Optimistic Projects
Top CVD & Metabolics
Projects selected
CVD & Metabolics
Top 10 Projects
Projects were Rated Based on Selection Criteria Indicating Project Attractiveness to a Potential Partner
Intrinsic to Agent • Unmet Needs • Market Potential • Novelty • Precedents • Clinical • Competition
Defined Health analysis
Page 25 Therapeutic Area Partnerships 2014 © Defined Health
Remove Unavailable
Agents
Evaluate Based on Selection
Criteria
Compare and Prioritize
Create CVD Investigational Agent Database (Phase I to registered)
Invite Selected
Companies
809 Available Agents
495 Optimistic Projects
Top 20 CVD & Metabolics
Projects selected
CVD & Metabolics
Top 10 Projects
Amongst Peers: • Exclude Prior Top 10 unless
clear progress achieved. • Exclude DH current client target • Company Management • WW Availability • “For out-license”
1,100 CVD + Met Agents in Ph I to Registration WW
Projects were Compared Among Their Peers Based On Company Attributes and Advertised Availability
Defined Health analysis
Page 26 Therapeutic Area Partnerships 2014 © Defined Health
Remove Unavailable
Agents
Evaluate Based on Selection
Criteria
Compare and Prioritize
Create CVD Investigational Agent Database (Phase I to registered)
Invite Selected
Companies
Companies Making the Final List were Invited
Defined Health analysis, Company websites
Page 27 Therapeutic Area Partnerships 2014 © Defined Health
2014 Cardiometabolic Projects To Watch*
*Not presented in any particular order
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 ultra-low 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 Type 2 Diabetes Phase II
Liver selective - Reduces blood glucose levels by increased hepatic glucose uptake.
TTP 399 TransTech Pharma Glucokinase Activator Type 2 Diabetes Phase
I/II
Clinical evidence of normalizing HbA1c without inducing hypoglycemia in T2DM subjects on stable doses of metformin.
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
Page 29 Therapeutic Area Partnerships 2014 © Defined Health
2014 Cardiometabolic Projects To Watch
Company Profiles
Page 30 Therapeutic Area Partnerships 2014 © Defined Health
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 co-development and co-promotion rights in order to evolve to a commercial-stage specialty pharmaceutical company Cerenis is also exploring partnering options and ways to collaborate on non-core strategic assets outside of cardiology.
Company website, Google Finance, EvaluatePharma
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 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.
Page 31 Therapeutic Area Partnerships 2014 © Defined Health
Company Overview
Financial Market Cap: $590.35M (as of 4-Nov-2014) No sales data available
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 Esperion is interested in partnering but currently has no industry partners
Company website, Google Finance, EvaluatePharma
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
Page 32 Therapeutic Area Partnerships 2014 © Defined Health
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).
PRONOVA (BASF) Company Profile
Company Overview
Company Pronova Biopharma Norge AS (part of BASF Omega-3 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 website, Google Finance, EvaluatePharma
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.
Page 33 Therapeutic Area Partnerships 2014 © Defined Health
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.
Partners
In 2011, Juventas Therapeutics spun-off SironRX Therapeutics to focus on development of dermal and bone related applications for JVS-100. 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 website, Google Finance, EvaluatePharma
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 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.
Page 34 Therapeutic Area Partnerships 2014 © Defined Health
Company Overview
Financial Market Cap: $981M (as of 12-Nov-2014)
Sales
Arena Pharmaceuticals Company Profile
Company Overview
Company Arena Pharmaceuticals
Location San Diego, CA
Description
Arena Pharmaceuticals is a biopharmaceutical company focused on discovering, developing and commercializing novel drugs that target G protein-coupled receptors (GPCRs).
Partners Arena Pharmaceuticals has granted exclusive marketing and distribution rights for Belviq to Eisai for most worldwide territories.
Company website, Google Finance, EvaluatePharma
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.
Page 35 Therapeutic Area Partnerships 2014 © Defined Health
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 website, Google Finance, EvaluatePharma
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 Overview
Financial
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 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.
Page 36 Therapeutic Area Partnerships 2014 © Defined Health
TransTech Pharma Company Profile
Company Overview
Company TransTech Pharma
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.
Partners TransTech has strategic alliances with Novo Nordisk, Cephalon, Forest Laboratories, Siga, Merck, Boehringer Ingelheim, and Pfizer.
Company website, Google Finance, EvaluatePharma
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 Overview
Financial
TransTech Pharma is a privately held company. • 2003: TransTech announced the completion of two round of investments totaling $34.5M
• 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
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