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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
Confidential
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
Historical Data
and trends of
CKD Market
Evolution
Company
Developments
Applications
Download Pattern
Regulatory
Landscape
Pipeline
Evolution
CKD Costs of
Burden
Hospitalization
Pattern
Readmission
Pattern
Market ShareEpidemiology
Pattern
Geographical
Usage
Market Restraints� High cost of innovative
therapies including stem cell
and gene therapies
Market Drivers� Increasing individuals suffering
from diabetes and BP
� Increasing incidence and
Market Opportunities� Acquisitions/collaborations
of companies and research
institutes
CKD MARKET: FORECASTING MODELFig 4
Factors Influencing
Market Growth
and gene therapies
� Changing reimbursement
scenario in major markets
like U.S. and U.K.
Investments�In September 2014, Covance and Frenova Renal Research
established a collaboration designed to find more effective
treatment for patients living with CKD and ESRD.
� Increasing incidence and
prevalence of chronic kidney
diseases
� Increasing aged population
.
Historical Market Data
Impact Analysis of Market
Trends
Market Size & Forecast by
Region
Major Players Profiling and
Strategies
Global CKD Market Size
2013 and Forecast to 2020Market Trends and
Forecasting
Market Penetration
�Innovative drug discovery
platforms
institutes
�Demand for novel drugs to
increase revenue of pharma
industry
Confidential
CKD - MARKET SIZING
Global CKD Drugs Market Global CKD Drugs Market by Region
Fig 6 Fig 7
Confidential
• 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
Confidential
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
Confidential
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
Confidential
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
Confidential
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
Confidential
XX XX XX XX XX
XX XX XX XX XX
LARGE MOLECULES-SEGMENTATION BY PHASES
Pre-Clinical
Discovery
Marketed
Clinical
Phase II
Fig 14
Confidential
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
Confidential
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
Confidential
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
Confidential
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.
Confidential
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
Confidential
Angion biomedica
Opko health
Regulus
Confidential
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
Confidential
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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