Chronic Kidney Disease

IQ4I Research & Consultancy Pvt. Ltd.

Chronic Kidney Disease

Pipeline Analysis

CONTENTS� REPORT DESCRIPTION

� INTRODUCTION� Causes & Symptoms

� Diagnosis

� Classification

� Epidemiology

� HOT TARGETS, MECHANISMS & THERAPIES� Associated targets

� DNA vaccination

� Gene therapy

� SMALL MOLECULES

� Pipeline

� Competitive Analysis

� LARGE MOLECULE

� Pipeline

� Competitive Analysis

� MONOCLONAL ABs

� Pipeline

� Competitive Analysis

� STEMCELL THERAPY� Gene therapy

� Tissue engineering

� MARKET DATA� Forecasting model

� Market dynamics

� Market sizing

� Cost burden

� PIPELINE ANALYSIS� Development stage

� Modalities

� Leading players

� Innovative approaches

� STEMCELL THERAPY

� Pipeline

� Competitive Analysis

� RNA-BASED THERAPY

� Pipeline

� Competitive Analysis

� MAJOR PLAYERS

� Company Profiles

� Overview, Pipeline & Deals

� REFERENCES

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REPORT DESCRIPTION

� “Chronic Kidney Disease (CKD) Pipeline Analysis” gives comprehensive insight onthe various drugs being developed for the treatment of CKD. The report covers allthe drugs being developed in various phases (Discovery, Preclinical, Clinical &Marketing). The pipeline focuses on novel pharmacologic drugs & regenerativemedicines covering small molecules, monoclonal antibodies, stem cell therapiesand RNA-based therapeutics, but excludes symptom relief drugs. The report alsocovers some of the hot targets in research for CKD treatments.

� This report enables Pharmaceutical /Biotech companies, Academic institutes,Individual researchers, Investors, Medical technology companies, Service providersIndividual researchers, Investors, Medical technology companies, Service providersand other associated stake holders to identify and analyze the availablelicensing/collaborative commercial opportunities in the CKD Drug market. Thereport also provide strategic insights on medicines that are likely to have an impacton CKD treatment space and potentially alter standards of care in CKD in theforeseeable future.

� Current therapies are not effective and more than half of patients gradually facesevere renal and cardiovascular complications, hence it provides a tremendousopportunity for upcoming therapies specific to CKD such as MAbs, stem cell-basedand RNA-based therapies, which reverses disease course or slow down kidneydeterioration.

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASEINTRODUCTIONINTRODUCTION

Diabetes XX%

High blood

pressureXX%

OthersXX%

CKD is a progressive & persistent kidney disease accompanied by reduction in

Glomerular Filtration Rate (GFR) i.e; less than 60 ml/min/1.73m^2 for a period of more than 3 months and is manifested by abnormal albumin secretion.

Major Causes (2014)

STAGE 1

No symptoms

Signs of symptoms

STAGE 2

STAGE 3

Mild symptoms

Low kidney function

STAGE 4

STAGE 5

Kidney damage

Stages of CKD

CHRONIC KIDNEY DISEASE (CKD)

STAGE 2 STAGE 4

People at major risk

Comparitive to Whites risk of:

African Americans – 4 times high Americans – 1.6 times high Asians – 1.4 times high Hispanics – high

CKD Reality

• 31 million people in U.S. adults have kidney disease

• 1 in 3 American adults are prone to CKD

• CKD in women is more common than men. But 50% men with CKD will progress to kidney failure.

• Currently out of 120,000 Americans are waiting for organ transplant and more than 100,000 need a kidney.

• ~ 25.8 million people in U.S. have diabetes and 40% of people with diabetes will develop CKD.

Prevalence

Global CKD prevalence is approximately XX – XX%

India - XX%

Indonesia –XX%

Europe–XX%

China– XX%U.S.–XX%

Argentina– XX%

Mexico–XX%

Taiwan– XX%

DIAGNOSIS OF CHRONIC KIDNEY DISEASE

BLOOD TEST

BLOOD TEST

URINE TEST

URINE TEST

ULTRASULTRAS

DIAGNOSISDIAGNOSIS

URINE TEST

URINE TEST

IMAGING TEST

IMAGING TEST

KIDNEY BIOPSYKIDNEY BIOPSY

ULTRASOUND SCAN

ULTRASOUND SCAN

STAGES OF CHRONIC KIDNEY DISEASE

STAGE 1

GFR 90

STAGE 2

GFR 60 – 89

STAGE 3

GFR 30 – 59

STAGE 4

GFR 15 – 29

STAGE 5

GFR less than 15 GFR

STAGE

According to KDOQI Clinical Practice Guidelines CKD is classified into 5 stages.

GFR 90 ml/min/1.73m^2

No symptoms observed

Normal urea & creatinine level

Normal kidney function

GFR 60 – 89 ml/min/1.73m^2

No symptoms observed

Mildly elevated urea & creatinine

level

Evidence of kidney damage

GFR 30 – 59 ml/min/1.73m^2

Early symptoms observed

Excess urea & creatinine level

Signs of kidney damage

GFR 15 – 29 ml/min/1.73m^2

Tiredness, itching, poor

appetite observed

Excess urea & creatinine level

Reduced kidney function

GFR less than 15 ml/min/1.73m^2

Poor sleeping, vomiting, breathing difficulty

Excess urea & creatinine level

Damage of kidney system

GFR

SYMPTOM

DIAGNOSIS

FUNCTION

KDOQI – Kidney Disease Outcomes Quality Initiative

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASEMARKET DATAMARKET DATA

CKD - MARKET SIZING

Global CKD Drugs Market Global CKD Drugs Market by Region

Fig 6 Fig 7

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• The global CKD market is estimated to be $XX billion in 2013 and is expected to reach $XX billion

by 2020 with a CAGR of XX% (2013-2020).

• The market growth is attributed to technological advancements, which led to emergence of novel

therapeutics, expansion in number of kidney transplantation, increasing incidence of kidney diseases,

and high unmet needs.

GLOBAL CKD DRUG MARKET BY THERAPEUTIC SEGMENTATION

Fig 8

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Anemia associated CKD Diabetic Nephropathy

SHPT + Hyperphosphatemia Others

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASEPIPELINE ANALYSISPIPELINE ANALYSIS

CKD PIPELINE BY PHASESFig 10

•Pipeline analysis reveals that there are XX molecules in various stages of pipeline (focusedon Discovery, Pre-clinical, Phase I, II, III and Marketed candidates)

•The phase II stage commanded the pipeline analysis with XX molecules, followed by preclinicalwith around XX molecules.

CKD PIPELINE BY LEADING PLAYERS

Fig 11N

o. o

f m

ole

cule

sN

o. o

f m

ole

cule

s

GLOBAL CKD DRUG MARKET BY THERAPEUTIC SEGMENTATION

Small molecules

Large molecules

Monoclonal antibodies

Stem cell therapy

RNA based therapy

Fig 12

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The CKD pipeline is commanded by pharmacological drugs, accounting for XX molecules. Large molecules

accounting for XX molecules followed by Monoclonal antibodies, Stem cell therapy, RNA base therapy and

others.

RNA based therapy

Others

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASESMALL MOLECULESSMALL MOLECULES

SMALL MOLECULES MOLECULE PHASE MOA COMPANY SUMMARY

STNM310 DiscoveryGlycan

inhibitorStelic Institute

& Co.

For small molecule glycan inhibitor, the company is seekinga strategic business partner with expertise in developing andoptimizing the candidates to launching the drug into themarket.

XX XX XX XX XX

XX XX XX XX XX

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XX XX XX XX XX

XX XX XX XX XX

XX XX XX XX XX

SMALL MOLECULES-SEGMENTATION BY PHASES

Pre-Clinical

Marketed

Discovery

Clinical

Phase I

Fig 13

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Phase II

Phase III

In the small molecule segment are clinical phase dominated with XX molecules amongthem phase II is the largest with XX molecules, followed by preclinical with XXmolecules

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASELARGE MOLECULESLARGE MOLECULES

LARGE MOLECULES MOLECULE PHASE MOA COMPANY SUMMARY

BLR-103 Discovery Not Available BLR-Bio

Blr-103 is a peptide mimetic, BLR Bio is advancing its productcandidates with a focus on chronic kidney disease and thecomplications of diabetes. However, our drug target is causal inmany forms of fibrosis as well as certain cancers.

XX XX XX XX XX

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XX XX XX XX XX

XX XX XX XX XX

LARGE MOLECULES-SEGMENTATION BY PHASES

Pre-Clinical

Discovery

Marketed

Clinical

Phase II

Fig 14

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Phase II

Phase I

Phase III

In the large molecule segment are clinical phase dominated with XX molecules amongthem phase II is the largest with XX molecules, followed by preclinical with XXmolecules

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASEMONOCLONAL ANTIBODIESMONOCLONAL ANTIBODIES

MONOCLONAL ANTIBODIES MOLECULE PHASE MOA COMPANY SUMMARY

XX XX XX XX XX

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XX XX XX XX XX

XX XX XX XX XX

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASESTEM CELLSTEM CELL--BASED THERAPYBASED THERAPY

STEM CELL THERAPYMOLECULE PHASE MOA COMPANY SUMMARY

XX XX XX XX XX

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RNA-BASED THERAPYMOLECULE PHASE MOA COMPANY SUMMARY

RG-012Pre-

Clinical anti-miR-

21 Regulus

miR-21 is a 22-mer non-coding RNA that negatively regulatesgene/networks and has been reported to be up-regulated infibrotic kidney diseases in both animal models and humanpatients.Preclinical studies have demonstrated that treatmentwith an anti-miR-21 significantly attenuates chronic kidneydisease progression. In July 2014, RG-012 received orphan drugdesignation from the U.S. Food & Drug Administration as atherapeutic for the treatment of Alport syndrome. InSeptember 2014, Regulus initiated ATHENA, a natural historyof disease study to gather greater information about theprogression of Alport syndrome, particularly the rate ofCHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE

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progression of Alport syndrome, particularly the rate ofdecline in renal function in Alport syndrome patients overtime. In the first half of 2015, Regulus expects to initiate aPhase I clinical study of RG-012 and a Phase II proof-of-concept study thereafter.

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASEOTHER MOLECULESOTHER MOLECULES

OTHER MOLECULESMOLECULE PHASE MOA COMPANY SUMMARY

CureNephron DiscoveryNot

ApplicableEvotec Ag

In January 2012, Evotec AG, Harvard University, Brighamand Women's Hospital, University of South Californiaand AstraZeneca collaboratively started the programme"CureNephron", designed to deliver and exploit noveltherapeutic targets as well as biomarkers that allowmore accurate diagnosis, monitoring and treatment ofchronic and acute kidney disease. As per the companypresentation the next milestone is pharmacollaboration.

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XX XX XX XX XX

XX XX XX XX XX

CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASEMAJOR PLAYERSMAJOR PLAYERS

CKD KEY PLAYERS - HEADQUARTERS

Astellas

Angion biomedica

BLR bio

Eli lilly

Pfizer

Vidasym

AstraZeneca

Johnson & Johnson

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Angion biomedica

Opko health

Regulus

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Fig 15

COMPANY-ASTELLAS PHARMA

OVERVIEWHEADQUARTERS

Astellas pharma is a U.S. affiliate of Tokyo-based pharmaceutical companyfounded in the year 2005 and is headquartered in United States. Thecompany aims to maintain and strengthen their position as global leader intransplantation and urology areas. It also focus in other applications likeoncology, urology, cardiology, infectious disease, immunology anddermatology. Astellas’ sustained commitment to research and developmentwithin the urology scientific community which continues in 2015/2016 as acollaborative effort with the Canadian Urological Association (CUA). TheCUA Astellas Research Grant Program has been established to supportinvestigator sponsored research in urology.

ILLIONIS

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investigator sponsored research in urology.

PIPELINEPIPELINE

Discovery Preclinical Phase I Phase II Phase III Marketed

ASP-6858

XXX

XXX

XXX

XXX

XXX

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