This document provides epidemiological data on primary biliary cholangitis (PBC) in China from 2018 to 2030. It estimates that the number of new PBC cases diagnosed annually in China will increase from 20,145 in 2018 to 21,108 in 2030. The total number of prevalent PBC cases in China is estimated to increase from 105,542 cases in 2018 to 110,588 cases in 2030. The document also summarizes the current treatment landscape and pipeline for PBC in China and other major markets.

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Primary biliary cholangitis (PBC)
Market Outlook, Epidemiology, Market Forecast and Compitive
Landscape report 2018 to 2030
Markets- US, Geramany, France, Italy, Spain, UK, Japan and China

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Primary biliary cholangitis (PBC) – Epidemiology
Summary: Incidence, Prevalent & Diagnosed patient pool
Source: 1.The World-bank, 2. Chew M et al., Primary biliary cholangitis: Diagnosis and treatment, 3. Floreani A et al., Different response to UDCA 4. Thelansis PMR Inputs, 5. Lindor K et al., PRACTICE GUIDANCE | Hepatology, VOL. 69, NO. 1,
2019, 6. Liu et al. BMC Gastroenterology 2010, Prevalence of primary biliary cirrhosis
Indicators 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
Population >20 years
(in 000’s)1 1,094,830 - - 1,107,690 - - 1,120,700 - - 1,133,864 - - 1,147,182
PBC diagnosed incidence
[2,4] 20,145 - - 20,381 - - 20,621 - - 20,863 - - 21,108
PBC prevalent cases[2,3,4]
105,542 - - 106,781 - - 108,036 - - 109,304 - - 110,588
Diagnosed prevalent
cases (%)4 77,045 - - 79,125 - - 81,262 - - 83,456 - - 85,709
Diagnosed
prevalent
cases male &
female ratio [3]
Male 16,826 - - 17,280 - - 17,747 - - 18,226 - - 18,718
Female 60,219
- -
61,845
- -
63,515
- -
65,230
- -
66,991
Anti-
mitochondrial
antibodies
(AMA)
screening[4, 5,6]
AMA
+ve
Cases
21,350
- -
21,926
- -
22,518
- -
23,126
- -
23,750
AMA -
ve
Cases
32,800
- -
33,685
- -
34,595
- -
35,529
- -
36,488
• In China 20,145 case of new diagnosed Primary biliary cholangitis (PBC) being report in 2018 the diagnosed incidence cases will reach up to 21,108 by 2030
• In 2018, the estimated prevalent cases of PBC in China were 105,542 and by 2030 the number will reach up to 110,588. In spite of equal patient pool being report as USA, the
diagnosis rate is low in comparison to the other mature market.

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Primary biliary cholangitis (PBC) – Epidemiology
Incidence, Prevalent & Diagnosed patient pool
19,600
19,800
20,000
20,200
20,400
20,600
20,800
21,000
21,200
2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
number
of
patients
Incidence cases of PBC trend
Incidence cases of PBC
1,03,000
1,04,000
1,05,000
1,06,000
1,07,000
1,08,000
1,09,000
1,10,000
1,11,000
2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
number
of
patients
Prevalent cases of PBC trend
Prevalence cases of PBC

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Primary biliary cholangitis (PBC) – Epidemiology
Incidence, Prevalent & Diagnosed patient pool
-
10,000
20,000
30,000
40,000
50,000
60,000
70,000
2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
number
of
patients
Sex specific: Diagnosed prevalent cases of PBC
Male Female
-
10,000
20,000
30,000
40,000
50,000
60,000
70,000
80,000
2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
number
of
patients
Anti-mitochondrial antibodies (AMA) screeningtrend
AMA -ve Cases AMA +ve Cases

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Primary biliary cholangitis (PBC) – Epidemiology
Symptom based patient segmentation
Source: 1. Floreani A et al., Different response to UDCA 2. Thelansis PMR Inputs
-
5,000
10,000
15,000
20,000
25,000
30,000
35,000
40,000
45,000
50,000
2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
number
of
patients
Symptomatic basis patient segmentation
Fatigue Pruritus Jaundice Others

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Primary biliary cholangitis (PBC) – Epidemiology
Stage-wise patient segmentation
Source: 1. Floreani A et al., Different response to UDCA 2. Thelansis PMR Inputs
-
5,000
10,000
15,000
20,000
25,000
30,000
35,000
40,000
2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
number
of
patients
Stage-wise patient segmentation
Stage I Stage II Stage III Stage IV

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Key Products in Development – Primary biliary cholangitis
China vs The World - trial landscape
3, 4%
19, 22%
16, 19%
14, 16%
12, 14%
8, 9%
7, 8%
7, 8%
China has the lowest pipeline activity – 1 Marketed, 1 Phase
I and 1 Preclinical Assets*
China US UK Germany France Spain Italy Japan
• Landscape in China:
• One product (ursodeoxycholic acid) which is widely approved across the world is
also approved in China
• In China the product is available with the brand name – Ursofalk
• Apart from the approved product, the pipeline activity is minimal in China
• No leading Pharma company (Ex-China) is currently pursuing development of an
asset in China
• 2 Chinese companies; Sino Biopharmaceutical and Suzhou Zelgen
Biopharmaceuticals are developing assets for the treatment of PBC
• Sino Biopharmaceutical’s product is in Phase I whereas Suzhou
Zelgen’s product is in preclinical stages
• Landscape Ex-China:
• Among the G-8 countries (US, EU-5, Japan and China), China has the minimum
pipeline activity and hence
• US is the most active market in terms of approved therapies as well as
the pipeline assets
• UK, Germany and France lead in pipeline activity in the EU-5 region
• Primary Insights:
• One promising molecule, Etrasimod (APD334) which was under development for
PBC in Phase II stage has now been terminated (business decision)
• In December 2017, Arena Pharmaceuticals and Everest Medicines entered into a
development and commercialization partnership for ralinepag and etrasimod in
mainland China, Taiwan, Hong Kong, Macau, and South Korea
• Thelansis’ Primary Research suggests that Everest Pharma has also dropped the
plans of developing Etrasimod for PBC
*The molecules are not mutually exclusive across geographies (includes marketed + pipeline) and hence; total count may appear more than the actual number of assets in development
Source: 1. Ursofalk China, 2. TQA-3526 - ChiCTR1800019570 (Phase I) 3. ZG-5266 – Preclinical

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Ursofalk (ursodeoxycholic acid)
Marketed Product profile
Ursofalk (ursodeoxycholic acid) - Aptalis Pharmaceuticals/Dr. Falk Pharma
Product Ursofalk (ursodeoxycholic acid)
Originator Company Aptalis Pharmaceuticals
Partners Sanofi, Dr. Falk Pharma, Asklepion Pharmaceuticals, Retrophin
Target Unspecified
MoA Cholesterol inhibitor
RoA Oral
Delivery Formulations Capsule, Suspension
Origin Chemical
Insights
Aptalis Pharma (formerly Axcan Pharma) has developed ursodeoxycholic acid for the treatment of PBC, and was working on additional indications
such as PSC, hypercholesterolaemia,, prevention of colorectal cancer and as adjunctive therapy for hepatitis-C. It was previously under development
for non-alcoholic steatohepatitis (NASH)
Source: 1. Ursofalk China

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TQA-3526 – Product profile
Phase - Ia clinical study
Source: 1.TQA-3526 - ChiCTR1800019570 (Phase I)
TQA-3526 - Sino Biopharmaceutical
Product/ Company TQA-3526/ Sino Biopharmaceutical
Study ID ChiCTR1800019570
Study title
Evaluation of TQA3526 single-center, randomized, double-blind, placebo-controlled, multi-dose, single-, multiple-dose, and pharmacokinetic Phase
Ia clinical trial in healthy subjects
Study objective
Dose tolerance of TQA3526 (multiple doses, and single doses), pharmacokinetics of TQA3526, Evaluation of TQA3526 drug metabolism
transformation
Study Phase Ia
Partners NA
Target Unspecified
MoA Unspecified
RoA Oral
Recruitment status/ study
Design
Not yet recruiting/ Parallel
Delivery Formulations Tablet
Origin Chemical
Analyst Insights
TQA-3526 is under development by Chia Tai Tianqing Pharmaceutical (Sino Biopharmaceutical) for the treatment of primary biliary cholangitis and
non-alcoholic steatohepatitis. Currently a Phase I in Healthy Volunteers has been registered where PBC and NASH are mentioned as target diseases

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ZG-5266 – Product profile
Phase - Preclinical study/ Early development
Source: 1. . ZG-5266 – Preclinical
ZG-5266 - Suzhou Zelgen Biopharmaceuticals
Product/ Company ZG-5266/ Suzhou Zelgen Biopharmaceuticals
Study ID Unspecified
Study title Unspecified
Study objective Unspecified
Study Phase Preclinical study/ Early development
Partners Unspecified
Target Nuclear receptor subfamily 1 group H member 4
MoA Farnesoid X receptor agonist
RoA Unspecified
Recruitment status/ study
Design
Unspecified
Delivery Formulations Unspecified
Origin Chemical
Analyst Insights
ZG-5266 is a farnesoid x receptor agonist (FXR) under development by Suzhou Zelgen Biopharmaceuticals for the treatment of primary biliary
cholangitis (PBC) and nonalcoholic steatohepatitis (NASH)

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