Alport syndrome (AS) Market outlook Epidemiology Market Forecast and Competitive Landscape Report

2019

Valued Knowledge Partners

Alport syndrome (AS) - Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report - 2020 To 2030 - Sample Report 

Disease overview (Classification, Severity & Stages, Risk factors, Coexisting conditions, pathogenesis, treatment land-

scape) 

Emerging therapies (Pipeline assessment and key clinical trials profile)

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Epidemiology (Incidence, prevalence, mortality and survival)

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Market share and patient share uptake

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KOL’s perspective on current treatment & unmet needs

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Market and product event analysis

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Market forecast 2020- 2030 (10 years of future market projections)

Market outlook & Insight Report

Report overview Report Summary Thelansis “Alport Syndrome (AS) - Market outlook, Epidemiology, Market Forecast and Competitive Landscape - 2020 To 2030 ” this report provides a comprehensive study of the disease area landscape and changing market dynamics with respect to the current & future market events for the 8 MM countries (USA, EU5, Japan & China)

 Key market outlook  11 Year Forecast  08Major Markets  Epidemiology (incidence, prevalence, diagnosed & treatment eligible patient pool)  Therapies (LoT’s & patient share)  Therapy area landscape (approved and emerging)  Pipeline Assessment

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The report provides a comprehensive view of Alport syndrome disease events, classification, severity level, stages, identified and emerging biomarkers, progression, manifestations, symptoms, risk factors, pathogenesis, genetic basis and treatment guidelines at country level

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Alport Syndrome Comprehensive insight on patient segmentation based on Age, Sex, classification (X-linked, Autosomal, and Digenic ), stages (Stage - 0, I, II, III, IV), Renal Manifestation Frequency (hematuria, proteinuria, end-stage renal disease, transplantation), Extra-renal Manifestation (sensorineural hearing loss, Ocular Lesions and others), Inheritance & Mutation (XLAS; COL4A5, ARAS;COL4A3, & COL4A4, ADAS; COL4A3 OR COL4A4) has been provided into the epidemiology (Incidence and Prevalence) section of the Alport Syndrome (AS) and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China

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Drug utilization and analysis on the current clinical practice, KOL perspective on upcoming therapies, estimated TPP for pipeline assets, clinical characteristics upcoming therapies and future treatment paradigm

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Market aggregation on newly diagnosed patient pool, Thelansis estimation on persistence, compliance, adherence and a historic view of changing market dynamics and product-level patient flow of AS in the 8 MM Countries for the period 2020 - 2030

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A comprehensive record of both the present and rising disease burden with respect to the treatments for AS are provided, along with impact of the new treatments and the probable patent expiry considering the current market dynamics. A detailed review of 8MM current and forecasted market including the drug outreach at different country level based on their order of entry

 Future competition Analysis  Current clinical Practices  Future treatment paradigm  Product attribute analysis  Unmet Needs  Market Attractiveness  Emerging Drug Profiles  Market Drivers and Barriers

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Table of content

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MS Excel Based Forecast Model

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Market outlook

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Disease Overview & Epidemiology

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Market Scenario & Competitive Landscape

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KoLs & Payer’s Perspective

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Market Forecast & Pricing Scenario

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Data collection approach (Primary & Secondary research)

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Report Highlights

Note: The table of content is just for ease of users/ reviewer to access different section of the sample page, however the complete table of content has been placed separately in the sample report

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Table of content

1.

Alport Syndrome – Key Findings Summary 1.1. Clinical findings 1.1.1. Disease overview 1.1.2. Therapeutic practices 1.1.3. Future outlook 1.2. Alport Syndrome Commercial findings 1.2.1. Alport Syndrome market scenario 2020 1.2.2. Alport Syndrome market scenario 2025 1.2.3. Alport Syndrome market scenario 2030 2. Alport Syndrome Overview 2.1. Disease Introduction 2.2. Pathophysiology 2.3. Signs and Symptoms 2.4. Risk Factors 2.5. Etiology 2.6. Classification 2.7. Pathogenesis 2.8. Diagnosis 2.9. Complications 2.10. Treatment Algorithm 2.10.1. Treatment in US (guidelines) 2.10.2. Treatment in EU-5 (guidelines) 2.10.3. Treatment in Japan (guidelines) 2.10.4. Treatment in China (guidelines) 2.11. Treatment Goals for Alport Syndrome 2.12. Referral Patterns 2.12.1. Referral Scenario in US 2.12.2. Referral Scenario in EU-5 2.12.3. Referral Scenario in Japan 2.12.4. Referral Scenario in China 2.13.Alport Syndrome Prognosis 2.14.Healthcare burden 2.14.1. Healthcare burden in US

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2.14.2. Healthcare burden in EU-5 2.14.3. Healthcare burden in Japan 2.14.4. Healthcare burden in China 2.15. Unmet Needs in Alport Syndrome management 2.16. Market Opportunity for Alport Syndrome 2.17. KOL Comments on current and upcoming/expected treatment practices in Alport Syndrome 3. Epidemiology 3.1. Epidemiology Overview 3.2. Alport Syndrome Epidemiology by Geography 3.2.1. Alport Syndrome Epidemiology in US (2015-2030) 3.2.1.1.

Incidence of Alport Syndrome 3.2.1.2.

Diagnosed cases 3.2.1.3.

Treatable Patient Pool 3.2.1.4.

Epidemiology Trends 3.2.2. Alport Syndrome Epidemiology in EU-5 (2015-2030) 3.2.2.1.

Incidence of Alport Syndrome 3.2.2.2.

Diagnosed cases 3.2.2.3.

Treatable Patient Pool 3.2.2.4.

Epidemiology Trends 3.2.3. Alport Syndrome Epidemiology in Japan (2015-2030) 3.2.3.1.

Incidence of Alport Syndrome 3.2.3.2.

Diagnosed cases 3.2.3.3.

Treatable Patient Pool 3.2.3.4.

Epidemiology Trends 3.2.4. Alport Syndrome Epidemiology in China (2015-2030) 3.2.4.1.

Incidence of Alport Syndrome 3.2.4.2.

Diagnosed cases 3.2.4.3.

Treatable Patient Pool 3.2.4.4.

Epidemiology Trends 3.3. Epidemiology Trends (World-wide) 4. Alport Syndrome Market Outlook 4.1. US Alport Syndrome Market Forecast 2020-2030 4.1.1. Market Progression (Futuristic)

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4.2.

4.3.

4.4.

4.5.

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4.1.2. Market Trends and Expectations 4.1.2.1.

Worst case scenario 4.1.2.2.

Base Case Scenario 4.1.2.3.

Best Case Scenario 4.1.3. Drivers and Barriers UK Alport Syndrome Market Forecast 2020-2030 4.2.1. Market Progression (Futuristic) 4.2.2. Market Trends and Expectations 4.2.2.1.

Worst case scenario 4.2.2.2.

Base Case Scenario 4.2.2.3.

Best Case Scenario 4.2.3. Drivers and Barriers France Alport Syndrome Market Forecast 2020-2030 4.3.1. Market Progression (Futuristic) 4.3.2. Market Trends and Expectations 4.3.2.1.

Worst case scenario 4.3.2.2.

Base Case Scenario 4.3.2.3.

Best Case Scenario 4.3.3. Drivers and Barriers Germany Alport Syndrome Market Forecast 2020-2030 4.4.1. Market Progression (Futuristic) 4.4.2. Market Trends and Expectations 4.4.2.1.

Worst case scenario 4.4.2.2.

Base Case Scenario 4.4.2.3.

Best Case Scenario 4.4.3. Drivers and Barriers Italy Alport Syndrome Market Forecast 2020-2030 4.5.1. Market Progression (Futuristic) 4.5.2. Market Trends and Expectations 4.5.2.1.

Worst case scenario 4.5.2.2.

Base Case Scenario 4.5.2.3.

Best Case Scenario 4.5.3. Drivers and Barriers

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4.6. Spain Alport Syndrome Market Forecast 2020-2030 4.6.1. Market Progression (Futuristic) 4.6.2. Market Trends and Expectations 4.6.2.1.

Worst case scenario 4.6.2.2.

Base Case Scenario 4.6.2.3.

Best Case Scenario 4.6.3. Drivers and Barriers 4.7. Japan Alport Syndrome Market Forecast 2020-2030 4.7.1. Market Progression (Futuristic) 4.7.2. Market Trends and Expectations 4.7.2.1.

Worst case scenario 4.7.2.2.

Base Case Scenario 4.7.2.3.

Best Case Scenario 4.7.3. Drivers and Barriers 4.8. China Alport Syndrome Market Forecast 2020-2030 4.8.1. Market Progression (Futuristic) 4.8.2. Market Trends and Expectations 4.8.2.1.

Worst case scenario 4.8.2.2.

Base Case Scenario 4.8.2.3.

Best Case Scenario 4.8.3. Drivers and Barriers 4.9. Key Expected Milestones (world-wide) Impacting the Market Alport Syndrome Competitive Landscape 5.1. Pipeline Therapies Overview 5.1.1. Phase II/IIII Therapies 5.1.1.1.

Bardoxolone Methyl (Reata Pharmaceuticals) Profile 5.1.1.1.1.

Current Status 5.1.1.1.2.

Trial details, results 5.1.1.1.3.

Approval Timeline 5.1.1.1.4.

Likelihood of approval 5.1.1.1.5.

Expected Product Positioning 5.1.1.1.6.

Revenue Projections (Till 2030) 5.1.1.2.

Attribute Analysis against rest of the pipeline

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5.1.2. Phase II Therapies 5.1.2.1.

RG-012 (Sanofi) Profile 5.1.2.1.1.

Current Status 5.1.2.1.2.

Trial details, results 5.1.2.1.3.

Approval Timelines 5.1.2.1.4.

Likelihood of approval 5.1.2.1.5.

Expected Product Positioning 5.1.2.1.6.

Revenue Projections (Till 2030) 5.1.2.2.

All other clinical therapies 5.1.3. List of active Pre-clinical Therapies 5.1.3.1.

BMP-7 (Ember Therapeutics) 5.1.3.1.1.

Status in Alport Syndrome 5.1.3.1.2.

Company positioning 5.1.3.2.

VAR 300 (ZyVersa Therapeutics, Inc.) 5.1.3.2.1.

Status in Alport Syndrome 5.1.3.2.2.

Company positioning 5.1.3.3.

All other pre-clinical therapies 5.1.4. List of Inactive/discontinued assets 5.1.4.1.

Business impact of discontinuations on current pipeline 5.1.5. Potential winners/Blockbusters from Alport Syndrome Pipeline Regulatory/Approval Scenario 6.1. Regulatory/Approval Framework in US 6.1.1. Policy Framework 6.1.2. Payer Expectations 6.2. Regulatory/Approval Framework in UK 6.2.1. Policy Framework 6.2.2. Payer Expectations 6.3. Regulatory/Approval Framework in France 6.3.1. Policy Framework 6.3.2. Payer Expectations 6.4. Regulatory/Approval Framework in Germany 6.4.1. Policy Framework 6.4.2. Payer Expectations

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6.5. Regulatory/Approval Framework in Italy 6.5.1. Policy Framework 6.5.2. Payer Expectations 6.6. Regulatory/Approval Framework in Spain 6.6.1. Policy Framework 6.6.2. Payer Expectations 6.7. Regulatory/Approval Framework in Japan 6.7.1. Policy Framework 6.7.2. Payer Expectations 6.8. Regulatory/Approval Framework in China 6.8.1. Policy Framework 6.8.2. Payer Expectations 7. Clinical Trial Assessment – Current and Future Paradigm 7.1. Distribution of Primary Endpoints across trials 7.2. Distribution of Secondary Endpoints across trials 7.3. Evolution and acceptance of surrogate endpoints 7.4. Key Investigator initiated trials 7.5. Attrition analysis 7.5.1. Suspended/Discontinued Assets 7.5.2. Failed Trials, Reasons and Business Impact 7.5.3. Terminated Trials, Reasons and Business Impact 7.5.4. Withdrawn Trials, Reasons and Business Impact 7.6. Trial enrollment scenario and challenges 7.7. Clinical Trial Guidance (across geographies) 8. Thelansis Commentary 8.1. Key Unmet needs in Alport Syndrome 8.2. Possible Best-case Clinical Trial Strategies 8.3. Possible Best Case Targeted Product Profile (TPP) 8.4. Possible Best-case Market positioning strategies 8.5. Possible Best-case Market Access Strategies 8.6. Possible Best-case LCM Strategies 8.7. Overall View on Alport Syndrome Market in Dollar Value 9. Report Methodology

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9.1. Secondary research 9.2. Primary research 9.3. Data collation 9.4. Insight Generation 10. About Thelansis 10.1. Our Capabilities 10.2. Our Services 10.3. Our Contacts 10.4. Disclaimer

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Market Forecasting Model - Snapshot

Patient based forecast model 2020 - 2030 (Snapshot - 1.1)

Patient based forecast model 2020 - 2030 (Snapshot - 1.2)

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Sample Diagrams Market outlook

2020 - 2030 Market size by region/ country; and growth

2020 - 2030 - Market share by region/ country; and growth

Report Summary Current 8 MM Market Incident XX Patients Prevalence XX patients Diagnosed XX Patients Treated XX Patients Marketed XX Drugs Phase II/ III/ XX PR drugs

2025 Market Size

US

$XX MM

US

UK

$XX MM

UK

DE

$XX MM

DE

FR

$XX MM

FR

IT

$XX MM

IT

SP

$XX MM

SP

Filed

XX

JP

$XX MM

JP

Key events

XX

CH

$XX MM

Key players

XX

Total

$XXX MM

CH T o t a l

Sales

Leading Drugs (2020 Market) Drug A

2020 - 2030 Market revenue by country/ region

2020Market Size

Drug B

$XX MM $XX MM

$XX MM $XX MM $XX MM $XX MM $XX MM $XX MM $XX MM $XX MM

DE FR IT SP JP CH

$XXX MM

Leading Drugs (2025 Market)

Sales

Leading Drugs (2025 Market)

Sales

Drug C

$XX MM

Drug C

$XX MM

Drug D

$XX MM

Drug D

$XX MM

Promising Emerging Therapies

Launch Year

Drug A Drug B

XXXX XXXX

Peak Year Sales $XX MM $XX MM

Key Events (2020-2030)

Impact on Market

Probability

High/ Moderate/ Low Source: Thelansis Knowledge partner; Executive summary

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UK

$XX MM $XX MM $XX MM $XX MM $XX MM $XX MM $XX MM $XX MM

Total

+/-

2020 - 2030 Patients by country / region

US

$XXX MM

Prime Unmet Needs

2020 - 2030 market/ epidemiology Country specific scenario

2030 Market Size

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Current Attainment High/Low High/Low Year to peak

Impact treatment paradigm

Opportunity High/Low High/Low Patent expiry

Probability High/ Moderate/Low

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Sample Diagrams Epidemiology

Disease overview: 

Alport syndrome is an inherited progressive form of glomerular disease that is often associated with sensorineural hearing loss and ocular defects. The primary abnormality arises from mutations in genes encoding the type IV collagen protein family. This syndrome is a genetically heterogeneous disease having different mutations and forms of inheritance: X-linked (mutations in COL4A5 gene), autosomal recessive (homozygous or compound heterozygous mutations in COL4A3 or COL4A4 genes) and autosomal dominant (heterozygous mutations in COL4A3 or COL4A4 genes).

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X-linked AS (XLAS) caused by COL4A5 mutations is the most common form, accounting ~80% of AS, however 15 percent of cases, showed mutations in both the COL4A3 or COL4A4 gene and is inherited in an autosomal recessive pattern.

2020 - 2030 Potential patient tower

Number of patients on supportive and off-label therapies

Epidemiology Research: 

The total number of Alport syndrome increased from 128,924 in 2020 to 182,305 by 2030. The USA comprises of ~38 of the total diagnosed cases of AS followed by China, EU5 and Japan. ~80% cases had typical glomerular basement membrane (GBM) changes of AS, ~10% patients initially had pathology features of diffuse thinning of the GBM only, 14% patients showed pathology of focal segmental glomerulosclerosis (FSGS).

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Around ~64% cases shown mutations in COL4A3 and ~32% of cases shown COL4A4. 46% patients had no missense mutation, hoever, ~51% to 39~ patients had one or two missense mutations.

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According to Thelansis, the diagnosed population of Alport syndrome will significantly increase at a CAGR of X% during the period 2020-2030 in 8 MM markets

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Over the course of 11 years till 2030, Thelansis expects the number of prevalent cases to increase at the CAGR of X.XX% in the US. The prevalence may reach X,XXX patients by 2030 in the US. In the EU-5 market(s), the prevalence is expected to reach X,XXX patients by 2030 whereas in Japan the rate of CAGR is relatively less with X.XX%. It is expected that in Japan, the prevalence pool will reach X,XXX in 2030

Treated patients forecast by region/ country 2020 - 2030

Diagnosed incidence Vs. Treated

Key patient segmentation

Market Trends: Key Takeaways

Illustrative sample

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Sample Diagrams Competitive landscape

Alport Syndrome Market Scenario: 

Current clinical practice recommendations for delaying endstage renal disease in patients with Alport syndrome focus on early introduction of RAAS inhibitors to suppress urinary protein excretion

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Angiotensin-converting enzyme (ACE) inhibition lengthens survival in the patients with autosomal recessive Alport syndrome and X-linked Alport syndrome

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chronic kidney failure progresses to end-stage kidney disease, requiring dialysis or transplantation. Kidney transplantation in people with Alport syndrome is usually successful, but some studies have reported that about 10% of transplanted patients develop inflammation of the kidneys

Key targets mutations

Key assets on different phases of development

Competitive Landscape: 

Considering Alport syndrome is an excellent model of CKD with proteinuria and fibrosis, orphan drug designation with premium pricing benefits , number of patients to be treated will be substantial and finally s no approved treatment several drug candidates are in Phase II and II/ III stages of development by various companies. Key assets among these are, Bardoxolone Methyl; Reata Pharmaceuticals, RG-012; Sanofi/ Regulus/ Genzyme, BMP7; Ember Therapeutics, VAR300; ZyVersa Therapeutics. The launch of key assets will positively impact the overall market during 2020 to 2030 in 8 MM countries.

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While there is a lot of competitive activity going on in pipeline for AS, companies like Reata Pharmaceuticals; Bardoxolone Methyl holds Orphan drug designation by US FDA and EMA.

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Patients treated with bardoxolone experienced a significant increase in eGFR of 7.8 mL/min/1.73 m2 (n=18; p=0.003) at Week 12 compared to baseline and to be found significantly improves kidney functions and well tolerated among the different cohort of treatments

Key players in the AS space, with assets under development

Emerging patient share and uptake; 2020 - 20130

Unmet needs

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Sample Diagrams Real-world evidences

KOL Opinion: based on Primary Market Research (PMR) 

“…..No approved therapies, identification of novel biomarkers for clinical use, and progress in understanding the underlying pathophysiology, there are remains a major unmet medical need ….. ….” (Department of Nephrology, Columbia University Medical Center , USA)

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“…..a …. biomarker mutation has……….. systems in the scientific literature…..identification of optimal patient care strategies and interpretation of data are complicated.....…..” (Academic & Researcher, Division Urology & Nephrology, Stanford University School of Medicine, USA)

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“…..among current therapy for AS, abnormal GBM for approximately 50% …... unmet medical need...prioritized if sufficient scientific…...to date there is no pharmacological therapy has been licensed to…....…..” (Department of molecular biology, University of Michigan, USA)

Key stakeholder insight

Market access: Role of HTA bodies in European region

Alport Syndrome Payer’s perspective: 

From the payer’s perspective majorly focused on wide range of treatment options rather than raising concern on cost savings. In coming years the treatment of Alport syndrome could change dramatically as US FDA and EMA has labeled orphan designation some of the key products. The payer’s and reimbursement agencies more concern about strong clinical outcomes data which have great evidence on managing the symptomatic conditions including quality of life, however cost could go up to 135,000 USD to 272,000 USD per year

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The European nations regulatory bodies and payers are having a very conservative mind-set with respect to the approval and access of non potential assets, which will rise the country level disease burden as well as the treatment cost in coming years. Apart from cost, payers have also concern on the quality of life parameters (e.g., therapies or diagnostic tests) that have not demonstrated improvements in the outcomes that matter to patients

Key therapies & cost burden

Regulatory framework & guidelines

Health economics & outcome

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Sample Diagrams Market forecasting

Alport Syndrome Market Forecast: 

Thelansis’ research team expects the Alport syndrome market potential for 2030 at $X.XXB in 8MM countries. Out of this market, 68.5% sales are expected to be contributed from the US market whereas XX.X% is expected to come from EU-5. Japan and China are expected to contribute XX.X% and X.X% respectively.

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Alport syndrome current market stands at $XXXX.XM which is expected to grow from 2019 to 2024 at a CAGR of X.X%. Majorly this is expected because of expected approvals of pharmacotherapies in the space. The market is expected to grow exponentially post 2024 at a CAGR of XX.X% (for the period 2024-2030). The most substantial increase in the market will be witnessed by the US market.

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Major Milestone of market growth is the entry of Orphan designated products like reducing oxidative stress, and inhibiting proinflammatory signaling, which will command a premium pricing and can offer long term cost burden reduction for the patients.

However, targeted therapies do carry a high attrition rate across the orphan indication space and this can be a key catalyst in determining market value for 2030.

Future market - 2020 To 2030

Country-wise revenue estimation

Patient share and market uptake

Alport Syndrome Pricing Scenario: 

Upcoming therapy probably transform the treatment paradigm of Alport syndrome in the near future. Clinical trials shows the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling . Of course, some questions about targeted therapy still remain unsolved.

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Thelansis expects upcoming therapies may raise significant concerns with respect to the treatment cost as much as 135,000 USD to 272,000 USD per patient .

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To control the cost research have developed algorithm for tiered testing guide to enhance the clinical diagnosis of AS, in the other hand it is hard to distinguish genetic variations such as pinpointing the phenotype and exact biomarker.

Disease burden matrix

Market Access

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Methodology Primary Market Research (PMR)

Secondary research

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About Thelansis: - As the name depicts Thelansis is specialized in “Therapy Area Landscape Analysis” along with market intelligence and consultancy that supports pharmaceutical, biotechnology, and diagnostics companies to successfully prepare, launch, and commercialize their products. Our Syndicated research reports include Market Insight, Pipeline Insight, Product Insight, Specialized Reports, Indication Outlook and Market Access Reports. Our focused therapeutic approach provides our clients with clinical and disease area expertise from an integrated team of academic, medical, and industry specialists of our panel, Ask for our report offerings, and Indication specific sample pages by sharing your email ID with us at, p.potdar@thelansis.com

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