CONFIDENTIAL USE ONLY DO NOT DISTRIBUTE
Transcript of CONFIDENTIAL USE ONLY DO NOT DISTRIBUTE
CONFIDENTIAL USE ONLY – DO NOT DISTRIBUTE
Strictly Confidential: Private Use Only
250+ Employees Globally
( 69 R&D & 42 Commercial)
Committed to Bringing Global Best-in-Class Propriety Therapeutic Products
2 Indications
1stCommercialized Product 2 NDA ongoing
- US PDUFA March 2021
- Korea NDA to be April 2021
2 Marketing Authorizations
- Europe (EMA) in 2019
- Taiwan in 2020
Global Phase 3 Trials
ET studies in US, CN, JP, KR, TW
Besremi®(Ropeginterferon alfa-2b)
- Polycythemia vera (PV)
- Essential thrombocythemia (ET)
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PharmaEssentia
Development
Patients
Discovery
Made In Taiwan
Fully Integrated Global Biopharmaceutical Company with End-to-End Capabilities
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Best-in-Class Management Team with ~75yr+ experience
25+ yr Experience:
Ionis Pharmaceuticals,
Food and Drug
Administration (FDA)
PhD, University of Michigan
25+ yr Experience:
Biogen Inc.,
Monsanto - Searle
PhD, University of Missouri
Postdoc, University of Michigan
2019 Humanitarian Honoree (MPN CR&T Fund)
25+ yr Experience:
Optimer Pharmaceuticals, Amgen Inc.,
Ligand Pharmaceutical,
Array Biopharma.
PhD, University of Pennsylvania
Ko-Chung Lin, PhDFounder & CEO
Ching-Leou Teng, PhDChairwoman
Jack Hwang, PhDGeneral Manager
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Increasing Global Footprint from Taiwan to Worldwide
CONFIDENTIAL – DRAFT – FOR INTERNAL USE ONLY – DO NOT DISTRIBUTE
Taiwan
USA
European Union Partner
Hong Kong
China South Korea
Japan
Taipei - Headquarter
• Nankang site: GMP certified PEGylation plant• Taichung site:
• GMP certified manufacturing/biologics plant
• Filled and finished plant
Two cGMP-compliant Manufacturing Facilities in Taiwan
Senior Field Management – Extensive Experience in Hematology, Oncology and Rare Diseases
27%
17%
56%
R&D Commercial Other
250+employees
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Global Executives with Proven Track of Execution
China General Manager U.S. General Manager Korea General ManagerJapan General Manager
• AGTech, BearingPoint Consulting,
• Biogen Inc.
• Shanghai Institute of Pharmaceutical Ind.
• MBA, Babson College
• MS, Northeastern University
• Shire - Vice President, Global Product Strategy Lead Hemophilia
• Baxalta - Vice President, Global Hemophilia Marketing
• Baxter - VP, Global Hemophilia Marketing
• Vertex - Launch their hepatitis - C drug, Incivik, which reached to $1.0Bn in sales within 8 months
• Novartis Korea
• Executive Advisor for Country President, Korea
• Country President, Korea
• Country President, Taiwan
• General Manager, Oncology Business Unit, Korea
• Shire Japan KK,
• Novartis Pharmaceuticals
• Janssen Pharmaceutical
• Astellas Pharmaceutical
• BA, Kochi University, Japan
Meredith Manning, MBAWarren ShenKatsuya Yonezu Haksun Moon
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Strong Leadership Team with Extensive Biopharma Experience
Tony ChambersVice President,
Sales & Market Access
Danielle HoltschlagSenior Director,
Sales Operations
Kate ReedyVice President,
Marketing and Business Intelligence
Craig ZimmermanVice President,
Clinical Development and Regulatory
Sam LinSenior Director, Business Operations and Strategy
Delphine El MehdiDirector, Medical Affairs
Meredith Manning, MBAU.S. General Manager
Ray Urbanski, M.D., Ph.DHead of Medical Affairs
and Clinical Development
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Our 1st product Besremi®(Ropeginterferon alfa-2b)
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Single form formulation leads to longer half-life and lower toxicity
Notes: HBV – Hepatitis B virus; HCV – Hepatitis C virus
Pegasys*PEG Interferon alfa-2a, for HCV & HBV
Besremi®
Ropeginterferon alfa-2b / P1101
K84K122
P1101 Predominant
form
Clinical Proven Results
• Long-acting (2x duration)
• Less frequent dosing
• once every two weeks (reduced by half)
• Less serious adverse events
• Highest maximum tolerated dose (MTD)
Multiple therapeutic areas
Best-in-Class PEGylated Interferon : Ropeginterferon-Alfa-2b
*Pegasys will be off the US market by end of 2021
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US Marketing Advantage - UnprecedentedInterferon stated as 1st line and 2nd line treatment in NCCN Guidelines before approval
1 Adapted from NCCN Guideline (May 21st, 2020)2 Inadequate response or loss of response
• Monitor
• Manage CV risk factors
• Aspirin
• Phlebotomy
• Hydroxyurea OR
Peginterferon alfa-2a
Treatment failure2
• Clinical Trial
• Ruxolitinib
• Hydroxyurea
• Peginterferon alfa-2a
• Busulphan
IF
2nd line treatmentInitial treatment (1st line)
High-risk PV patient1
High, complete hematologic response
Low toxicity Non-mutagenicLess frequent dosing
schedule
Disease modification to slow or halt disease
progression
Besremi Meeting Key Unmet Needs for PV Treatment
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~100,000Patients diagnosed with
PV in the U.S.3
(~158,000 people livingwith PV in U.S.1,2)
~60 YEARSAverage age of onset4,
diagnosed often by routine blood tests due to vague, non-descript symptoms
Primary symptoms5
Fatigue
Aquagenic pruritis
Headaches
Reddened Face
Weight Loss
Freq
uen
cy o
f O
ccu
rren
ce
2nd lineCytoreductive therapy
Second line: patients currently receiving other cytoreductive agents post failure of HU
1st lineCytoreductive therapy
First line: main treatment goal to focus on short-term CV risk and symptoms relief
Phlebotomy / Aspirin>40% of diagnosed patients are managed with aspirin/phlebotomy
CONVENTIONAL APPROACH TO CARE BASED ON AVAILABLE THERAPEUTIC OPTIONS
Most physicians treat <10 PV
patients
Volume of patients who present with elevated HCT
and are asymptomatic4
~67% ASYMPTOMATIC
Sources: 1 Population - US Census Pop Clock, accessed June 8, 2020, 2 Prevalence rate (2010 marketscan assumption) - Mehta et al 3 Data on File 4 National Organization for Rare Disorders (NORD). Polycythemia Vera. https://rarediseases.org/rare-diseases/polycythemia-vera/. Accessed May 27, 2020. 5 Polycythemia Vera Patient Journey market research by BluePrint Research Group (May 2020)
U.S. market today: Opportunity to advance care in PV
~12,000 Physicians involved in MPN patient care
• MPN specialists• Academic centers• Community oncologists
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Attractive US Market Opportunities in MPNMarket Opportunity
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PDUFA Date - March 13th , 2021
Counting Down for U.S. Marketing Approval
Mar Apr May June July Aug Sep Oct Nov Dec Jan FebOur
ProgressMar
March 13th May 12th
PDUFA March 13th , 2021
month 1st 2nd 3rd 4th 5th 6th 7th 8th 9th 10th 11th 12th
2020 2021
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Available in 16 European Countries ~ € 2,500/250mcg
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)Slovenia: Ministry of Health (link)Italy: Italian Medicines Agency (AIFA) (link) *Ireland: Health Products Regulatory Authority (link)*
1. Germany € 2,708 (250mcg)
2. Austria € 2,708 (250mcg)
3. Slovenia € 2,025 (250mcg)
4. Denmark € 3,178 (250mcg)
5. Italy € 4,450 (500mcg)
6. Ireland
7. United Kingdom
8. The Czech Republic
9. Hungary
10. France
11. Greece
12. Switzerland € 2,557 (250mcg)
13. Sweden € 2,390 (250mcg)
14. Liechtenstein
15. Finland € 2,876 (250mcg)
16. Norway
Kazakhstan
Turkey
Georgia
AzerbaijanArmenia
Belarus
Ukraine
Moldova
European Union (EU)
Besremi listed countries
*Dosing frequency: Bi-weekly
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2nd Indication: Essential Thrombocythemia (ET)
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Phase III Global Pivotal ET study ongoingFiling expected in early 2023
Primary Endpoint
Randomized, open-label, active-controlled study designed to compare the efficacy and safety with ANA (n=160)
• Peripheral blood count remission (months 9 and 12)• Improvement or non-progression in disease-related signs
(months 9 and 12)• Large Symptoms improvement• Absence of hemorrhagic or thrombotic events
StudyType
Second-Line ET Study vs Anagrelide (NCT04285086)
Reference:1. Tefferi (2018) Blood Cancer Journal 8, 2
Analysis(9 mo)
Adult patients with high-risk ET
Eligible PV patients based on WHO 2016 criteria
Patients who were treated with HU or HU resistant/tolerant
Randomization Screen(1 mo)
Ropeginterferon, Q2W, SC
Anagrelide, Daily, oral
Treatment(12 mo)
Analysis(12 mo)
Follow-up(1 mo)
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Global ET Trial Supported by Strong Medical Professionals
Albert Qin, M.D. Ph.D.Chief Medical Officer
SymBio Pharmaceuticals, ImmunoGen, Biogen, Pfizer
PhD., Harvard University
Oleh Zagrijtschuk, M.D. Ph.D.Therapeutic Head, U.S.
Yi-Wen Huang, M.D. Ph.D.Senior Director, Taiwan
Toshiaki Sato, M.D. Ph.D.Chief Medical Director, Japan
Kee-Woo Kim, M.D. Ph. D.Medical Director, Korea
M.D., Medical University of Vienna
MBA, WU Executive Academy
Cathay General Hospital
M.D., China Medical University
Ph.D., NTU
Johns Hopkins University, Post-doc
Oita Medical University, Japan
Ph.D., The Catholic University of Korea
M.D., SoonchunhyangUniversity, Korea
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Besremi® : Key Milestones & Future Growth Strategy
2019 2020 2021 2022 2023
EU EU EU EU EU
TW TW
US
TW
US
KR
TW
US
KR
JP
KR
JP
CN
TW
US
KR
JP
CN
PV
ET
Marketing Authorizations
Extend PV approval territories
Expand Ropeginterferon indications and strengthen our collaboration with partners
Enrich pipeline through
1) proprietary science team;
2) collaboration; or
3) acquisition
Obtained marketing approval of Ropeginterferon in polycythemia vera in EU and Taiwan
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Tremendous Indication Expansion Opportunities
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
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Concentration Technology Indication MarketPre-
ClinicalProof of Concept Pivotal Regulatory Approval
HematologyP1101
PV
EU
TW
US, KR
JP, CN
ET Global
Adult T-Cell Leukemia JP, TW, CN
Next Gen.of 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, SG
KX01Actinic
Keratosis TW
Solid Pipeline Fueling Next Wave of Growth
PDUFA Q1, 2021
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Thank YouPharmaEssentia