Protagonist Therapeutics, Inc. · NASDAQ:PTGX
Protagonist Therapeutics — Investor Presentation (June 2021)
30 slides from Protagonist Therapeutics's investor presentation, Jun 14, 2021
Slide 1: Company Overview
PN - 943 PTG - 200 PN - 232 PN - 235 rusfertide (PTG - 300) PTG - 100 Dinesh V. Patel, PhD President & CEO June 2021 COMPANY OVERVIEW 1
Slide 2: Forward-looking Statements
Forward - looking Statements 2 This presentation and the accompanying oral presentation contain forward - looking statements made pursuant to the safe harbor pro visions of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in thi
Slide 3: Innovative Peptide Technology Platform
Innovative Peptide Technology Platform 3 Multiple Assets from De Novo Discovery to Clinical Development Peptide Chemistry SAR, Leads Oral Stability Peptidomimetics GI Assays Phage Libraries Hits Computational Vectrix, Clusters Vectrix ® Clusters GI - Restricted Targeted GI absorption & delivery Syst
Slide 4: Product Portfolio
Product Portfolio 4 Multiple Development Candidates in Multiple Indications PROGRAMS CANDIDATE DISCOVERY PRE - CLINICAL PHASE 1 PHASE 2 KEY 2021 - 22 MILESTONES HEMATOLOGY & BLOOD DISORDERS Hepcidin Mimetics rusfertide (PTG - 300) s.c. 39 ‡ 3K HQUROOPHQWFRPSOHWH ྃ ‡ 8SGDWHG3K GDWDRUDOS
Slide 5: Polycythemia Vera: Disease, Diagnosis, Prevalence, Treatment Goal
3RO\F\WKHPLD9HUD 5 Disease, Diagnosis, Prevalence, Treatment Goal Myeloproliferative neoplasm characterized by excessive production of red blood cells (RBCs) • Characterized by Janus Kinase 2 (JAK2) mutation Treatment goal is to control hematocrit level <45% • Elevated hematocrit is a hallmark of t
Slide 6: Polycythemia Vera Current Treatment Options
Polycythemia Vera 6 &XUUHQW7UHDWPHQW2SWLRQV 1. MPN Landmark Survey 2017, Trinity Primary Research 2019 • Treatment goal is to maintain HcT ≤ 45% • HcT control may be erratic with up and down excursions from 45% • Can lead to iron deficiency • Recommended when HcT cannot be controlled, or in high -
Slide 7: Understanding PV Patient Journey
Understanding PV Patient Journey 7 Evaluating the Unmet Need of PV Patients in US 1 Symphony Patient Journey Data Large, representative, and longitudinal source of healthcare claims data that captures over 290MM patients with over 78% of a ll prescription claims and 60% of all medical claims. Medica
Slide 8: Real World PV Patient Treatment Data
Real World PV Patient Treatment Data 8 Treatment patterns • Predominant treatment is phlebotomy, regardless of risk • Hydroxyurea is the most commonly used cytoreductive agent • Combination of hydroxyurea and phlebotomy commonly used to control HcT Hematocrit management to NCCN guidelines • Only 22%
Slide 9: Elevated HcT Resulted in Greater Risk of Death
(OHYDWHG +F7 5HVXOWHGLQ*UHDWHU5LVNRI'HDWK 9 Death from CV or Major Thrombotic Events Endpoint 0 6 12 18 24 30 36 42 48 0.85 0.90 0.95 1.00 Months P r o b a b i l i t y o f R e m a i n i n g E v e n t - f r e e Low HCT (<45%) High HCT (45%-50%) Patients with hematocrit between 45 - 50% were
Slide 10: Rusfertide Market Opportunity for PV
10 Rusfertide (PTG – 300) Market Opportunity for PV 7KHUHDUHDSSUR[LPDWHO\ SHRSOHLQWKH8QLWHG6WDWHV OLYLQJZLWK39 ‡ a WUHDWHGSDWLHQWV ‡ *URZLQJPDUNHWZLWKa QHZSDWLHQWVGLDJQRVHGDQQXDOO\ ‡ 0HGLDQVXUYLYDOLQ39DSSURDFKHVRUFDQH[FHHG \HDUV 0DMRULW\RIGL
Slide 11: Phase 2 Trial of Rusfertide in PV Patients
PART 1 (28 wks) PART 2 (up to 12 wks) PART 3 (52 wks) Dose Finding * Efficacy Evaluation * Blinded Withdrawal Open Label Extension * Phase 2 Trial of Rusfertide in PV Patients 11 GOAL: Maintain Hematocrit <45% ELIGIBILITY REQUIREMENTS: • Phlebotomy dependent PV patients diagnosed as per 2016 WHO cri
Slide 12: Therapeutic Phlebotomies Prior to and on Rusfertide
Age/Sex Randomization - 20 - 12 - 8 - 4 0 4 8 12 20 24 28 32 36 48 - 24 52 56 60 64 68 72 76 40 44 84 Therapeutic Phlebotomies Prior to and on Rusfertide Weeks Subject 0.2 0.1 0.0 Pre - Dose Post - Dose Average Rate of phlebotomies 3 Screening Part 1 - Dose finding Part 2
Slide 13: Baseline Characteristics
Baseline Characteristics Characteristics Age (n=62) Range 27 - 76 years (Mean = 56.3 yrs ) Gender (n=62) Females 17 (27.4%) Males 45 (72.6%) Risk (n=62) Low 30 (48.4%) High 32 (51.6%) [Age based – 37.1%, Thrombotic event – 14.5%] Duration since PV Diagnosis ( n=60) <1 yr 11 (18.3%) 1 - <3 yrs 22 (36
Slide 14: Rusfertide Controlled HCT for 1.5 Year while Reducing RBC Count
14 Rusfertide Controlled HCT for 1.5 year while Reducing RBC Count in PV patients 57 45 39 33 N Bas 4 8 16 20 24 28 32 36 40 48 52 56 60 64 68 72 31 27 22 17 17 12 12 7 7 8 5 5 4 3 40 62 HCT (%) Weeks Bas 4 8 16 20 24 28 32 36 40 48 52 56 60 64 68 72 31 25 22 17 16 12 12
Slide 15: Rusfertide Normalized Iron Stores
Rusfertide Normalized Iron Stores Screening Part 2/3 – Blinded withdrawal/OLE Part 1 – Dose finding Part 3 – Open label extension Normalization observed in Ferritin levels Interim data as of May 11, 2021
Slide 16: Improvement in MPN-TSS Scores Following Rusfertide
2.3 1.8 0 0.5 1 1.5 2 2.5 Baseline Week 28 N=55 N=14 2.2 0.7 0 0.5 1 1.5 2 2.5 Baseline Week 28 N=55 1 3.3 2.4 0 1 2 3 4 Baseline Week 28 N=55 N=14 Improvement in MPN - TSS 1 Scores Observed Following Rusfertide Fatigue 3UREOHPVZLWK&RQFHQWUDWLRQ Itching Pruritis 16.9 16 12.8 10.8 0 5 10 15 20 B
Slide 17: Rusfertide Safety Summary
Grade 3 : Not related – Vomiting: 1; continuing drug – Intramural thrombus - lower left buttock 1; continuing drug • No Grade 4 Events • Injection site reaction (ISRs) were most common and associated with 28.1% of injections. All ISRs were transient, and no patient discontinued due to ISR • No drug
Slide 18: Rusfertide Phase 3 Study Design in PV
Rusfertide (PTG - 300) Phase 3 Study Design in PV 18 Based on End - of - Phase - 2 Meeting with FDA and Written Communication with EMA rusfertide Adult Patients with PV* N≈250 Enrollment Criteria: • High risk and low risk patients • Patients requiring frequent phlebotomy • With or without cytoreduct
Slide 19: Rusfertide Market Preparation
5XVIHUWLGH0DUNHW3UHSDUDWLRQ Intend to Demonstrate Clinical & Economic Value Before Approval Confidential – Managed Markets Working Group Meeting 19 Value Proposition Market Access HEOR Competitive Landscape 3RVLWLRQLQJ 6WUDWHJ\ Prescriber Education Distribution Channels • Patients • Prescribers
Slide 20: Hereditary Hemochromatosis (HH) Disease Prevalence and Treatment
Hereditary Hemochromatosis (HH) 20 Disease Prevalence and Treatment If untreated, iron overload can cause hepatomegaly, diabetes mellitus, skin hyperpigmentation, cardiomyopathy, diastolic dysfunction, heart failure, cirrhosis, etc . Iron overload disease Excessive iron accumulation in heart, liver,
Slide 21: Rusfertide Rationale and Phase 2 Clinical PoC Study in HH
Hereditary Hemochromatosis (HH) 21 Rusfertide (PTG - 300) Rationale and Phase 2 Clinical PoC Study Design • 16 patient open - label study • Efficacy measures » Reduction in serum Iron, TSAT, ferritin » Phlebotomy reduction » Liver iron content (LIC) by MRI » Quality of Life (QOL) effects: SF - 36, P
Slide 22: IBD: Paradigm Shift Toward Targeted Oral and Combination Therapy
IBD: Paradigm Shift Toward Targeted Oral and Combination Therapy 22 $*URZLQJ0XOWL %LOOLRQ'ROODU0DUNHW • Injectable TNF mAbs – Blockbusters – Humira ® & Remicade ® • Significant room for improvement – Low response rates / loss of response – Safety concerns - black box warnings TNF mAbs dominate
Slide 23: Oral IL-23R Specific Peptide Antagonists: Janssen Partnership
Oral, IL - 23 Receptor Specific Peptide Antagonists: PTG - 200, PN - 235, PN - 232 23 Janssen Partnership IL - 23R ,/ 5 ȕ IL - 12R β 2 IL - 12R β 1 Stelara ® : Blocks both IL - 23 & IL - 12 pathways PTG - 200 : Designed to block IL - 23 receptor & IL - 23 pathway p19 antibody : Blocks IL - 23
Slide 24: Oral, Gut-Restricted α4β7 Integrin Peptide Antagonist: PN-943
Oral, Gut - Restricted, Investigational a 4 b 7 - Integrin Peptide Antagonist: PN - 943 24 Fully Owned Asset and Novel Approach against Validated Target Vs. %ULVNLQ0HWDO $P-3DWKRO Clinically Validated, IBD Specific Target • T cell homing regulated by α4β7 integrin and MA
Slide 25: PN-943 vs. PTG-100: Blood %RO Based Clinical Proof-of-Concept
PN - 943 vs. PTG - 100: Blood %RO based Clinical Proof - of - Concept* Ph1 NHV Single Ascending Dose Study Ph2A Study UC PTG - 100 900 mg Histologic Remission 44% (7/16) Clinical Remission* 16% (3/19) EDVHGRQEOLQGHGHQGRVFRSLFUH UHDGV Ph1 SAD 100 mg 300 mg 1000 mg 0 20 40 60 80 100 M a x i m u
Slide 26: PN-943 Ph2 IDEAL Study in UC
PN - 943 26 Ph2 IDEAL Study in UC Study Patients • Global enrollment at multiple sites • Subject to COVID - related delays 5 weeks 12 weeks 40 Weeks Week 52 Randomize (n=150) Placebo BID (n=50) Active Drug 450 mg BID (n=50) $FWLYH'UXJ PJ%,' Q PN - 943 Eligibility: Moderate – Severe UC; 3
Slide 27: Protagonist Team
Dinesh Patel, PhD | President & CEO David Liu, PhD | CSO, Head of Discovery & Pre - Clinical Dev 6DPXHO6DNV0'_ &OLQLFDO'HYHORSPHQW$GYLVRU Suneel Gupta, PhD | Chief Development Officer Donald Kalkofen | Chief Financial Officer Tracy Woody | EVP , Commercial Strategy Matthew Gosling | EVP, Gene
Slide 28: Financials
Financials 28 Cash and securities as of March 31, 2021 $279.7M $255M of capital raised during 2020 $20M available on debt facility; zero debt outstanding 43.9M shares of common stock outstanding
Slide 29: Anticipated Timeline
29 Anticipated Timeline Anticipated Key Event June 2021 • Updated data from rusfertide Ph2 PV trial 2021 • Rusfertide Ph2 PV trial update at medical conference • Preliminary rusfertide Ph2 result in HH • Third development program for rusfertide • Complete Ph1 trial of PN - 235 2022 • Initiate rusfer
Slide 30: Thank You
PN - 943 PTG - 200 PN - 232 PN - 235 rusfertide (PTG - 300) PTG - 100 THANK YOU 30
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