PharmaEssentia Corporation

Transcription

1 PharmaEssentia Corporation September 8, 2016

2 Company Information Operation started in May. 2003, in Taipei, Taiwan Market Value = ~US$ 1.3Bn Employees = 150 (2MD, 32 PhD, 72 MS and others) Cash : ~US$ 145M

3 Management Team Ko-Chung Lin, Ph.D Founder and Managing Director 30 years experience of new drug developing Biogen Inc. / Lead Scientist Monsanto Searle Ching-Leou Teng, Ph.D Chairwoman 25 years experience of new drug developing ISIS Pharmaceutical / Assistant Director Food and Drug Administration (FDA) / Reviewer Jack Hwang, Ph.D General Manager 25years experience of new drug developing Optimer Pharma / Director Array Pharma / Group Leader Amgen Inc. / Research Scientist Shu-Fen Li, MBA Director, BD & Strategic planning Siemens / Executive secretary Tradecompass / Product & Business Development Manager

4 Core Technology Single-site PEGylation technology Purest long-acting PEG-IFN P1101 Very mild AE, can be used for various indications. Flexible dosing adjustment. Patent protection until N-terminal Pro >95% K49 A.A. protein K121 K83 K70

5 Comparison with other PEG-IFN PEG-Intron (Merck) (PEG interferon alfa-2b, for HCV) MTD = 90 mg Pegasys (Roche) (PEG interferon alfa-2a, for HCV & HBV) MTD= 180mg a P1101 (PharmaEssentia) (PEG-P-interferon alfa-2b) - Long-acting - Less frequency of dosing - Very mild AE, MTD = 540 mg - More compliance - Can be used for multiple indications. - Flexible dosing adjustment N-terminal proline K84 K122

6 PD study in monkeys Single Dose (30μg/kg) s.c.

7 PK study in healthy male adult Montreal, Anapharm, December 2009 July 2010 MTD = 540 mg

8 P1101 for various indications MPN (Abnormal growth of blood cells) Polycythemia Vera (PV) Essential Thrombocythemia (ET) Chronic myeloid leukemia (CML) Myeloid Fibrosis (MF) Hepatitis (Combo with small molecule drugs) Chronic Hepatitis B (HBV) Chronic Hepatitis C (HCV) Cancer (Combine with PD1/PDL-1) HCC RCC NSCLC

9 Pipeline development Hematology Infectious diseases Oncology

10 Cause of MPN (Myeloproliferative neoplasm) MPN (Abnormal growth of blood cells) Polycythemia Vera (PV) Essential Thrombocythemia (ET) Chronic myeloid leukemia (CML) Myeloid Fibrosis (MF) Abnormal increase of blood progenitor cells. May be related to JAK2 gene mutation( PV ET MF) Without proper treatment, may develop leukemia FDA approved Jakafi for treating MF in 2011 and for treating PV (2 nd line) in The list price of Jakafi exceeds USD $130,000/yr 1 1. Price-Rx

11 Polycythemia Vera (PV) Significant Unmet Needs A disease which the proportion of blood volume that is occupied by red blood cells increases. Treatment : no FDA approved 1 st line treatment (phlebotomy/hydroxyurea/ Pegasys off-label). 1 in 4 patients are refractory to current treatments Jakafi (Incyte) : Jakafi was approved by FDA in Dec, 2014, for 2 nd line treatment for PV. The price tag of Jakafi is >USD $130,000/yr

12 2011 ASH Meeting No DLT, as defined per protocol, were observed in the study. The highest investigated dose of 540 μg every two weeks has been concluded as the Maximum Tolerated Dose (MTD), according to the protocol definition. A response rate of above 70% at six month already observed during the phase I dosefinding part suggests that AOP2014 has efficacy and good tolerability in patients with Polycythemia Vera.

13 2011 ASH Meeting

14 2013 ASH Meeting No DLTs occurred, the highest administered dose 540 μg every two weeks was declared as MTD Observed toxicities are in line with the safety profiles of other interferons alpha and were comparable in low and high dose groups (post hoc analysis) More convenient treatment schedule with observed safety profile in line with expectations from other (pegylated) interferons Durable clinical benefits were achieved in PV patients: overall response rate ~ 90%, cumulative CRs 45-50%; low and high dose groups achieved comparable response rates Freedom of phlebotomy, normalization of hematological parameters and spleen size reduction in majority of patients Significant and sustained decrease of JAK2 allelic burden observed starting from week 28 of treatment

15 2013 ASH Meeting

16 Hematological responses phase I/II Non-responders Partial responders Complete responders Gisslinger et al, 2015

17 2014 ASH Meeting Data demonstrate ability to further reduce the frequency of P1101 administration to once every four weeks, while maintaining response and improving tolerability Continuous reduction of the JAK2 allelic burden indicates that duration of exposure is important to induce molecular responses Safety Decrease of Adverse Event: (all/drug related) 0.17/0.09 (q2w init.) 0.30/0.09 (q2w post 1y) 0.08/0.03 (q4w) (mean count of AEs per patient week exposure)

18 Ropeginterferon alfa-2b(p1101) 在人體臨床實驗治療 PV 之病患已獲得高療效低毒性的成果 23

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20 P1101 PROUD PV Primary Endpoint (4) primary endpoint ClinicalTrials.gov Identifier: NCT Primary Outcome: Disease response rate at 12 months - Hematocrit <45% without phelobotomy - Platelets <400 G/L - Leukocytes <10 G/L - Normal spleen size 2 nd Outcome: JAK2 allelic burden (identification of cure) Jakafi RESPONSE Primary Endpoint (2) ClinicalTrials.gov Identifier: NCT

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22 MPN (Myeloproliferative neoplasm)-p1101 Indications:PV, ET, MF (rare blood diseases) PV ET MF Patient number US ~100,000 ~134,000 ~13,000 EU ~100,000 ~160,000 ~16,000 Orphan Drug Designation (EU/US) Patent till 2032 Unmet medical needs PV CML PV ET MF

23 P1101 for PV - Market Positioning for EU and the USA

24 Orphan Drug Designation of P1101 Advantages of orphan drug Fast review process and small trial size. 7 and 10 years market exclusivity in EU and US, respectively. Price of orphan drug is increasing (price of Jakafi have increased 60% from ) Reduced taxes from the federal government Indication Polycythemia Vera (PV) in EU and US Essential Thrombocythemia (ET) in US Myeloid Fibrosis (MF) in US Year

25 P1101 for PV in the USA and EU Item Jakafi Incyte Corp. Indication Disease Pop. in USA FDA Approval (US$) Line of Therapy MF ~ 12, $130,000 First PV ~150, $130,000 Second (25~35%) EU+US Combined Sales Potential (USD) 2015 Q1:US$ 205M Q2:US$ 240M Q3:US$ 264M Q4:US$ 301M Total: US$ 1.01B P1101 PV ~150,000 Expected 2017 First Line - ET ~135,000 Expected 2019 Second Line Based upon Incyte Financial Filings

26 First Step: in August PEC PK Incyte in DC Two educational (and complimentary) symposia At MPN & MDS Congress in Alexandria, VA August Srdan Verstovsek, MD, PhD The University of Texas M. D. Anderson Cancer Center Houston, Texas

27 Comprehensive and concise education focus on MPN & MDS over lunch Friday, August 28, 2015 The Evolution of Interferon-alpha as Therapy for Patients with Polycythemia Vera and Essential Thrombocythemia 12:40 pm - Welcome and pre-activity polling via ARS 12:45 pm - Indication for cytoreductive therapy in ET and PV: Do we have it right 1:00 pm Long-acting interferon: What is the difference? 1:15 pm Development of long-acting interferon as new therapy for PV and ET 1:30 pm Expert panel discussion: Enhancing outcomes in PV and ET 1:45 pm Concluding remarks, recap of key points, and post-activity polling via ARS 1:50 pm Adjourn Supported by: PharmaEssentia PK Saturday, August 29, 2015 JAK Inhibitors for the Successful Management of Patients with Polycythemia Vera 12:40 pm - Welcome and pre-activity polling via ARS 12:45 pm - Lecture: Evolution of the assessment of clinical benefit in polycythemia vera 1:05 pm - Debate: Should JAK inhibitors be used strictly for hydroxyurea resistant/intolerant polycythemia vera patients or do they have a broader application? 1. Broader application 2. Strictly in HU resistance/intolerance 1:35 pm - Panel discussion: Enhancing the quality of life and optimizing clinical outcomes for all patients with polycythemia vera 1:55 pm - Concluding remarks, recap of key points, and post-activity polling via ARS 2:00 pm - Adjourn Supported by: Incyte Corp.

28 Besremi injection pen for PV

29 Global Trade Name

30 P1101(AOP2014, BESREMI) Milestones Clinical Development Program (ProudPV) Medical Marketing Marking Research Competitive Environment Regulatory Submission Office Opening Hiring of Team(s) Pre-Launch Phase AOP- sales team(s) ready to go Launch - Phase Launch Phase aoporphan.com

31 Timeline for Product Launches Next Three Years Indication P1101 for PV Clinical / Geographic Phase III / EU Phase III / USA PIII Submission/ Review Launch April Last Patient Out P1101 for ET Phase III / USA PIII Subm./ Review P1101 for HCV. GT2 Phase III / Taiwan, Korea PIII Subm./ Review Launch P1101 for HBV Phase III / Global PIII Review Oraxol for Cancer PK+Clinical / Taiwan PIII Review Launch

32 Thank You!