a fully integrated biopharmaceutical company

A Fully Integrated Biopharmaceutical Company

元富台股企業日 Feb 11th, 2020

藥華醫藥股份有限公司

2

About PharmaEssentia

P1101 Approved in Feb 2019

P1101 has Far More Potential to be Explored

Commercialization

Summary

Executive Summary

• Fully Integrated Biopharmaceutical Company

Our mission is to discover, develop, manufacture, and bring to market efficacious & cost-effective therapeutics.

• EMA Approval

1st product, Besremi®, approved by EMA for the treatment of Polycythemia vera (PV) in Feb 2019, first country launched in Q3 2019.

• US BLA for PV indication will be submitted soon

• FDA agreed with Phase 3 trial protocol for Essential Thrombocythemia (ET) indication in Sep., 2019

The trial will kick- off by Q1 2020. The global trial includes US, China, Japan, Korea, and Taiwan.

Our Journey to DateSelected highlights since initial founding

2003

2016

2018

2012: Ground breaking for Taichung manufacturing site

2016: Publicly listed on Taipei Exchange

2017: Marketing Authorization Application filed2012

2016: Phase 3 clinical trial (PROUD-PV)completed

2017

2019

2018: Manufacturing plant received EMA GMP Certification

Company Founded

2019: Besremi®Approved by European Commission

2008: P1101 Discovered

2008

Meet our Executive LeadersFounding Members

Ching-Leou Teng, PhD

Chairperson

25+ yr Experience: Ionis Pharmaceuticals,

Food and Drug Administration (FDA)

PhD, University of Michigan

Ko-Chung Lin, PhD

Founder and CEO

25+ yr Experience: Biogen Inc.,

Monsanto - SearlePhD, University of Missouri

Postdoc, University of Michigan

Jack Hwang, PhD

General Manager

25+ yr Experience: Optimer Pharmaceuticals,

Amgen Inc.,Ligand Pharmaceutical,

Array Biopharma.PhD, University of Pennsylvania

Company Highlights

• Establishment: 2003

• Ticker: 6446 TT

• 215 Employees worldwide

• Core technology of PEGylation to tackle unmet

medical needs

• BLA obtained: EMA approved Besremi® in Feb

2019

• Market Cap: Around US$ 1 B (as of Jan 10, 2020)

• Headquarters: Taipei, Taiwan

Taichung site: GMP certified biologics plant

Nankang site: GMP certified PEGylation plant

Nangang Software ParkEMA GMP Certified Plant

National Bio Research Park

Central Taiwan Science ParkEMA GMP Certified Plant

TaipeiHQ

Taichung

Japan

China

MassachusettsUSA

EU Partner

Our Global PresenceInternational Subsidiaries and Partners

Taiwan, HQHong Kong

Korea(Planning)

• Global Employees: 215• M.D. degree : 6• Ph.D. degree : 32• Master degree : 110

8


P1101 Obtained Approval in Feb 2019


Commercialization

Agenda

EU Marketing Authorization Approval

• We discovered and manufactured

Ropeginterferon alfa-2b (P1101);

licensed to AOP Orphan (Austria),

Middle east, and CIS region.

• EMA approved Besremi®

(Ropeginterferon alfa-2b, P1101) on

Feb 15th 2019 for the indication of

Polycythemia Vera (PV).

• Listed in Germany, Austria, Slovenia,

Denmark, and Italy.

Source: https://www.ema.europa.eu/en/medicines/human/EPAR/besremi

Authorization

EU Availability and Pricing

European Union (EU)

Belarus

Ukraine

Germany

France

Italy

SpainPortugal

Ireland

United Kingdom

Greece

SwedenFinland

Denmark

Estonia

Latvia

Lithuania

Poland

Romania

Hungary

Czech Slovakia

Austria

Bulgaria

Netherlands

Belgium

Malta

Luxembourg

Croatia

Slovenia

Cyprus

Iceland

Norway

Liechtenstein

Switzerland

Russia

Source: Denmark Pricing: Danish Medicines Agency (link)Italy: Italian Medicines Agency (AIFA) (link)Slovenia: Ministry of Health (link)

• Denmark € 3,178• Germany € 2,778• Austria € 2,778• Slovenia € 2,430

• Italy

https://www.medicinpriser.dk/Default.aspx?id=15&vnr=495597

https://farmaci.agenziafarmaco.gov.it/bancadatifarmaci/farmaco?farmaco=047624

http://www.cbz.si/zzzs/pao/bazazdr2.nsf/0/86b5697bdbe7dffdc12584620083a5dd!OpenDocument&Click=

Myeloproliferative Neoplasms (MPN)

Chronic Myeloid

LeukemiaMyelofibrosis

Philadelphia chromosome

Essential

thrombocythemia

Polycythemia

Vera

Modified from Cross, Hematology Am Soc Hematol Educ Program;2011:208--‐14.; 2014 EHA and 2013 ASH Education Program

Acute Myeloid Leukemia

Polycythemia Vera

Phase III Pivotal Clinical Study

3 year treatment results presented at ASH 2018

Efficacy at 24 & 36 monthDurability 24 - 36 month

P1101 Dose titration up to optimal dose at 500 mcg for more than 28 weeks vs. HU 8 weeks

PROUD / CONTI

(n= 127)

(n= 127)

(HU)

Results at 36 Months for Polycythemia Vera (PV)

PROUD / CONTI: Phase III Pivotal Clinical Results

Presented at ASH 2018

Primary End PointsRopeginterferonalpha-2b (P1101)

Hydroxyurea p-value

Complete Hemotological Response (CHR)

70.5% 51.4% 0.0122

CHR+ Symptom improvement 52.6% 37.8% 0.0437

Molecular Response (JAK2 mutation)

66.0% 27.0% < 0.0001

Response rate

Better Response Maintenance Profile Compare with HU

107 96 65 56 42 38 32 27 23 19 17 14 12 9 6 4 2 0BAT107 95 84 76 71 68 62 55 49 45 41 35 27 20 10 6 4 0P1101

Number of subjects left

0 3 6 9 12 15 18 21 24 27 30 33 36 39 42 45 48 51

Duration of response, m

0.0

0.1

0.2

0.3

0.4

0.5

0.6

0.7

0.8

0.9

1.0P

rob

ab

ilit

y o

f re

sp

on

se d

ura

tio

n

0 3 6 9 12 15 18 21 24 27 30 33 36 39 42 45 48 51

Duration of response, m

0.0

0.1

0.2

0.3

0.4

0.5

0.6

0.7

0.8

0.9

1.0P

rob

ab

ilit

y o

f re

sp

on

se d

ura

tio

n

(11.9, 21.1)(38.7, --)95% CI

17.97--Median

59 (55.1%)31 (29.0%)Events

107107# of subjects

BATP1101

BAT

P1101

*Long-term study was not designed for comparison. Response curves of BAT are for reference only

Median duration of response* with P1101 exceeds 52 months

Complete Hematologic Response (CHR)

The Lancet HaematologyPublished online January 31, 2020

2020 2021 2022 2023

US JapanChina

Taiwan Korea

Efficacy study in Japanese and Chinese Populations

Timeline for Polycythemia Vera Marketing Approvals

17




Commercialization

Agenda

Essential Thrombocythemia (ET)

Chronic Myeloid

LeukemiaMyelofibrosis

Philadelphia chromosome

Essential

thrombocythemiaPolycythemia

Vera

Acute Myeloid Leukemia

Myeloproliferative Neoplasms (MPN)

Modified from Cross, Hematology Am Soc Hematol Educ Program;2011:208--‐14.; 2014 EHA and 2013 ASH Education Program

FDA agreed with the Essential Thrombocythemia (ET) phase 3 trial protocol in Sep 2019

Ropeginterferon alpha-2b orphan designation was granted by FDA in 2014

This global study is planned to be conducted in US, Taiwan, China, Japan, Korea

• PLT < 400x109/L and WBC <

9.5x109/L

• Improvement or non-

progression in disease-related

signs (splenomegaly)

• Large symptoms improvement

based on the MPN SAF TSS

Fixed dose

Investigational arm: Ropeginterferon alpha-2b (P1101), Q2W, S.C. (N=80)

250 (W0)->350 (W2)->500 (W4)

4W dose escalation Primary Analysis9 to 12M

4W follow up

Primary Analysis9 to 12M

Control arm: Anagrelide, P.O. (N=80)

Maintain optimal blood counts control at acceptable toxicity

Labelled dose

4W follow up

ET Global Phase III Trial

Primary Endpoints

TaiwanUS

Korea ChinaJapan

BLA Filing

Last Patient out

Last Patient in

First Patient in

ApprovalsRegulatory

2020 2021 2022 2023

Clinical Trial

Timeline for ET Trial & Marketing Approvals

Future Growth Strategy

Extend PV approval territories: Taiwan, US, Korea, Japan, China, etc.

Expand P1101 indications and strengthen our collaboration with partners:

- ET- Oncology: solid tumors- Next generations

Enrich pipeline through

1) proprietary science team;

2) collaboration; or

3) acquisition

P1101 is the next generation – Super Interferon

PharmaEssentia (PEG-P-interferon alfa-2b)

MTD = 540 mg

K84 K122

N-terminal proline• Long-acting

• Less frequent dosing

• Less serious adverse events

• Highest maximum tolerated

dose (MTD) comparing to the

marketed products

Roche (PEG interferon alfa-2a)

Merck (PEG interferon alfa-2b)

PEG-Intron(>14 isomers)

MTD = 90 mg

MTD = 180 mg

Clinically Proven ResultsBi-weekly dosing

Weekly dosing

Weekly dosing

P1101 (n=127)n(%)

HU (n=127)

n(%)

HU (n=36)n(%)

Pegasys (n=36)n(%)

Fatigue16

(12.6%)17

(13.4%)10

(28%)18

(50%)

Leukopenia11

(8.7%)27

(21.3%)3

(8%)8

(22%)

Nausea3

(2.4%)15

(11.8%)7

(19%)7

(19%)

Anemia8

(6.3%)31

(24.4%)6

(17%)7

(19%)

Thrombo-cytopenia

19 (15.0%)

36 (28.3%)

7 (19%)

6 (17%)

Depression2

(1.6%)- -

10 (28%)

P1101 Showed Less Depression than Pegasys*

* MPD-RC 112 Study comes from Mount Sinai Tisch Cancer Institute

PROUD-CONTI Study

PROUD/ CONTI Safety Results Presented at ASH Meeting 2016

MPD-RC 112 Study*

*Pegasys is an off-label use medication for treating PV.

More indications for Oncology will be pursued

Solid Tumor• Multiple Myeloma• Bladder Cancer• Malignant Melanoma• Kaposi’s Sarcoma• Squamous cell carcinoma of the conjunctiva• Carcinoid Syndrome

Leukemia• Chronic Myelocytic Leukemia• Hairy Cell Leukemia• Adult T-cell Leukemia (Japan)

Source: WebMD.com

Lymphoma

• Follicular Lymphoma

• Cutaneous T-cell Lymphoma

• Mycosis fungoides

Besremi® (Ropeginterferon alfa-2b/ P1101)

Oleh Zagrijtschuk

Therapeutic Head

Boehringer Ingelheim,AOP Orphan PharmaceuticalsMD, Medical University of Vienna

MBA, WU Executive Academy

Albert Qin

Chief Medical Officer

Biogen,China Health Group,

SymBio Pharmaceuticals, ImmunoGen,

PfizerPhD., Harvard University

Chung-Wei Lee

Director

LifeSpring Immuno, AxcellaHealth, Brigham and Women’s

HospitalMD, National Taiwan UniversityPhD, Massachusetts Institute of

Technology

Joe Tseng

Director

Far Eastarn Polyclinic,NTUH

MD, National Taiwan UniversityPhD., Harvard University

Yi-Wen Huang

Senior Director

Cathay General HospitalMD., China Medical University

Ph.D, National Taiwan University

M.D. Ph.D. (MIT) M.D. Ph.D. (NTU) M.D. Ph.D. (Harvard) M.D. Ph.D. (Harvard)M.D. Ph.D. (U of Vienna)

Meet our Medical TeamExperienced leaders in clinical research

26

Company Profile



Commercialization

Agenda

Senior Marketing Director, Asia - Pacific Jennifer Lin

PROFESSIONAL EXPERIENCE

Takeda Taiwan - Rare Metabolic Business Unit Head

(Fabry disease/Gaucher Disease/Rare immunology/Rare Metabolic Disease)

SHIRE Biotech Taiwan - Business planning & Commercial Operations Lead, Taiwan & HK

Pfizer Taiwan - Commercial Development & Strategic Planning Lead

Janssen Taiwan - Group Product Manager

Bristol-Myers Squibb Taiwan - Regional Sales Manager

EDUCATION and LICENSE

• Durham Business School, University of Durham, UK - MBA

• National Taiwan University - MSc in Pharmacology

• Taipei Medical University – B.S. in Pharmacy

• License of Pharmacist, Taiwan

Marija Sebastian, PhD

VP of Commercialization

Join from Dec, 2017

18yr Experience

Novartis Pharmaceuticals,

Craig Zimmerman, PhD

VP of Regulatory and R&D

Join from April, 2013

25yr Experience

Biogen Inc.

Our Global LeadersExperienced leaders in biotech

US General Manager Qualifications (Recruiting in 2020)

• VP level and C level is preferred

• Small biotech and big pharma

• $Bn sales experience

• Launch 1st Product experience

Samuel Lin, MBA

Sr. Director of Business Operations

Join from May, 2016

Monster Government Solutions

USPTO

Duke University

NCCN Guidelines for PV

High-risk PV

patient

Monitor

Manage CV risk factors

Aspirin

Phlebotomy

Hydroxyurea or interferon

Treatment failure*

Hydroxyurea (if not previously used)

Interferon (if not previously used)

Ruxolitinib

Source: NCCN guidelines for patients 2018

IF

Initial Treatment 2nd line Treatment

* Treatment failure includes inadequate response or loss of response

Marketing EventOnclive Peer Exchange

Source: Onclive: Determining the Potential of Ropeginterferon Alfa-2b in Polycythemia Vera (Link)Youtube (Link)

https://www.onclive.com/peer-exchange/mpn-management/determining-the-potential-of-ropeginterferon-alfa2b-in-polycythemia-vera

https://www.youtube.com/watch?time_continue=1&v=yb8Ikv_DTq4&feature=emb_logo

PharmaEssentia Received Humanitarian Award

Company Auditor

S. Sophia

Chair

Dr. Lin

Representative

K. Yonezu

Director

N. Miyachi

(MA & QA)

Director

Dr. Teng

Director

C. Snow

RA

A. Muko

Finance

Y. Tanaka

HR

D. Doi

Assistant

Y. Amano

Board of Directors

Medical

Director

Clinical Operation.

H. Kawase

RA

T. Adachi

HR

S. Nakagawa IT

H. Song

CRO

A2 healthcare

CRO

EPSI Bio stat

Y. Yamaguchi

PV

M. Takano

Supervisor-general

A. Hasegawa

Non-clinical

M. Iijima

Auditor

Avantia

Lawyer

K. Kitamura

Accountant

K. WangCMC

M.Okamura

H. Takasugi

2020 Japan Organization

藥華生物科技(北京)有限公司組織架構

董事總經理（申維宏）

臨床醫學和市場總監（王煒）

行政兼人事助理（待招聘）

醫院管道主管（劉博漢）

臨床研究副總監（張菁菁）

項目1組-專案經理（鄭滿佳）

項目2組-專案經理（離職，待招聘）

註冊經理（待招聘，

肝病/血液專業）

醫學經理（待招聘，血液專業）

現行組織架構（一年內）

Our Global LeadersPharmaEssentia Korea

General Manager (Recruiting)

• Copy Japan hiring model

• Great relationship with KOLs

• Global pharma experience

• Entrepreneurship

Head of Medical (Recruiting)

• MD, PhD

• Industry experience

• Hematologist

Concentration Technology Indication MarketPre-

ClinicalProof of Concept Pivotal Regulatory Approval

HematologyP1101

PV

EU

US, TW

JP, CN, KR

ET Global

Adult T-Cell Leukemia (ATL) JP, TW, CN

Next Generationsof P1101 MPN Global

Oncology

P1101 + anti PD-1 HCC Global

Oraxol Breast Cancer TW, SG, VN

Oraxol +Ramucirumab Gastric Cancer TW, SG, VN

P1101 Solid Tumors Global

Infectious P1101 HDV US, EU, TW

DermatologyKX01 Psoriasis TW, CN, MY

KX01 Actinic Keratosis TW, VN,MY

Building Our Next Wave of GrowthBy Q1 2020

a fully integrated biopharmaceutical company

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