CKD provides a tremendous opportunity for upcoming therapies specific to CKD such as MAbs, stem cell-based and RNA-based therapies, which reverses disease course or slow down kidney deterioration.

1. Chronic Kidney Disease
IQ4I Research & Consultancy Pvt. Ltd.
Chronic Kidney Disease
Pipeline Analysis

2. 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 THERAPYGene 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

3. REPORT DESCRIPTION
“Chronic Kidney Disease (CKD) Pipeline Analysis” gives comprehensive insight on
the various drugs being developed for the treatment of CKD. The report covers all
the drugs being developed in various phases (Discovery, Preclinical, Clinical &
Marketing). The pipeline focuses on novel pharmacologic drugs & regenerative
medicines covering small molecules, monoclonal antibodies, stem cell therapies
and RNA-based therapeutics, but excludes symptom relief drugs. The report also
covers 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 providers
and other associated stake holders to identify and analyze the available
licensing/collaborative commercial opportunities in the CKD Drug market. The
report also provide strategic insights on medicines that are likely to have an impact
on CKD treatment space and potentially alter standards of care in CKD in the
foreseeable future.
Current therapies are not effective and more than half of patients gradually face
severe renal and cardiovascular complications, hence it provides a tremendous
opportunity for upcoming therapies specific to CKD such as MAbs, stem cell-based
and RNA-based therapies, which reverses disease course or slow down kidney
deterioration.

4. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
INTRODUCTIONINTRODUCTION

5. Diabetes
XX%
High
blood
pressure
XX%
Others
XX%
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%

6. DIAGNOSIS OF CHRONIC KIDNEY DISEASE
BLOOD
TEST
BLOOD
TEST
URINE
TEST
URINE
TEST
ULTRASULTRAS
DIAGNOSISDIAGNOSIS
URINE
TEST
URINE
TEST
IMAGING
TEST
IMAGING
TEST
KIDNEY
BIOPSY
KIDNEY
BIOPSY
ULTRAS
OUND
SCAN
ULTRAS
OUND
SCAN

7. 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

8. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
MARKET DATAMARKET DATA

9. 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.

10. GLOBAL CKD DRUG MARKET BY THERAPEUTIC
SEGMENTATION
Fig 8
Confidential
Anemia associated CKD Diabetic Nephropathy
SHPT + Hyperphosphatemia Others

11. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
PIPELINE ANALYSISPIPELINE ANALYSIS

12. CKD PIPELINE BY PHASES
Fig 10
•Pipeline analysis reveals that there are XX molecules in various stages of pipeline (focused
on Discovery, Pre-clinical, Phase I, II, III and Marketed candidates)
•The phase II stage commanded the pipeline analysis with XX molecules, followed by preclinical
with around XX molecules.

13. CKD PIPELINE BY LEADING PLAYERS
Fig 11No.ofmoleculesNo.ofmolecules

14. 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

15. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
SMALL MOLECULESSMALL MOLECULES

16. SMALL MOLECULES
MOLECULE PHASE MOA COMPANY SUMMARY
STNM310 Discovery
Glycan
inhibitor
Stelic Institute
& Co.
For small molecule glycan inhibitor, the company is seeking
a strategic business partner with expertise in developing and
optimizing the candidates to launching the drug into the
market.
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

17. 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 among
them phase II is the largest with XX molecules, followed by preclinical with XX
molecules

18. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
LARGE MOLECULESLARGE MOLECULES

19. 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 product
candidates with a focus on chronic kidney disease and the
complications of diabetes. However, our drug target is causal in
many forms of fibrosis as well as certain cancers.
XX XX XX XX XX
Confidential
XX XX XX XX XX
XX XX XX XX XX

20. 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 among
them phase II is the largest with XX molecules, followed by preclinical with XX
molecules

21. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
MONOCLONAL ANTIBODIESMONOCLONAL ANTIBODIES

22. MONOCLONAL ANTIBODIES
MOLECULE PHASE MOA COMPANY SUMMARY
XX XX XX XX XX
Confidential
XX XX XX XX XX
XX XX XX XX XX

23. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
STEM CELLSTEM CELL--BASED THERAPYBASED THERAPY

24. STEM CELL THERAPY
MOLECULE PHASE MOA COMPANY SUMMARY
XX XX XX XX XX
Confidential

25. RNA-BASED THERAPY
MOLECULE PHASE MOA COMPANY SUMMARY
RG-012
Pre-
Clinical
anti-miR-
21
Regulus
miR-21 is a 22-mer non-coding RNA that negatively regulates
gene/networks and has been reported to be up-regulated in
fibrotic kidney diseases in both animal models and human
patients.Preclinical studies have demonstrated that treatment
with an anti-miR-21 significantly attenuates chronic kidney
disease progression. In July 2014, RG-012 received orphan drug
designation from the U.S. Food & Drug Administration as a
therapeutic for the treatment of Alport syndrome. In
September 2014, Regulus initiated ATHENA, a natural history
of disease study to gather greater information about the
progression of Alport syndrome, particularly the rate ofCHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
Confidential
progression of Alport syndrome, particularly the rate of
decline in renal function in Alport syndrome patients over
time. In the first half of 2015, Regulus expects to initiate a
Phase I clinical study of RG-012 and a Phase II proof-of-
concept study thereafter.
CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
OTHER MOLECULESOTHER MOLECULES

26. OTHER MOLECULES
MOLECULE PHASE MOA COMPANY SUMMARY
CureNephron Discovery
Not
Applicable
Evotec Ag
In January 2012, Evotec AG, Harvard University, Brigham
and Women's Hospital, University of South California
and AstraZeneca collaboratively started the programme
"CureNephron", designed to deliver and exploit novel
therapeutic targets as well as biomarkers that allow
more accurate diagnosis, monitoring and treatment of
chronic and acute kidney disease. As per the company
presentation the next milestone is pharma
collaboration.
Confidential
XX XX XX XX XX
XX XX XX XX XX

27. CHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASECHRONIC KIDNEY DISEASE
MAJOR PLAYERSMAJOR PLAYERS

28. 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

29. COMPANY-ASTELLAS PHARMA
OVERVIEWHEADQUARTERS
Astellas pharma is a U.S. affiliate of Tokyo-based pharmaceutical company
founded in the year 2005 and is headquartered in United States. The
company aims to maintain and strengthen their position as global leader in
transplantation and urology areas. It also focus in other applications like
oncology, urology, cardiology, infectious disease, immunology and
dermatology. Astellas’ sustained commitment to research and development
within the urology scientific community which continues in 2015/2016 as a
collaborative effort with the Canadian Urological Association (CUA). The
CUA Astellas Research Grant Program has been established to support
investigator 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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