Paradigm Biopharmaceuticals Ltd. · ASX:PAR
Paradigm Biopharmaceuticals — Investor Presentation (April 2020)
32 slides from Paradigm Biopharmaceuticals's investor presentation, Apr 6, 2020
Slide 1: Release of FDA Minutes and Capital Raising
For personal use only (ASX: PAR) Paul Rennie, CEO & MD • Release of FDA minutes from OA Pre-IND Meeting • Release of TGA Special Access Scheme WOMAC Scores • A$35m Placement 6 March 2020
Slide 2: Paradigm's Mission
Paradigm’s Mission For personal use only Paradigm Biopharmaceuticals LTD (PAR) is a late stage drug development company focusing on repurposing the drug Pentosan Polysulfate Sodium (PPS). Paradigm Aim is to develop and commercialise an ethical, safe and effective Pharmaceutical Agent (PPS) for the t
Slide 3: Executive Summary
Executive Summary For personal use only § Repurposing Pentosan Polysulfate Sodium (“PPS,” “ZILOSUL®”): FDA-approved, 60-yr track record of treating inflammation § Lead program: Osteoarthritis (OA) – 31m sufferers in the US alone § OA Phase 2b trial (n=112) met primary, secondary and exploratory endp
Slide 4: Summary of FDA Pre-IND meeting for Phase 3 OA trial
Summary of FDA Pre-IND meeting for Phase 3 OA trial For personal use only § PAR attended a Pre-IND meeting with FDA on 19 February re its Phase 3 OA trial § The FDA has now provided the minutes of the meeting to PAR § Key outcomes of the FDA meeting include: § Primary endpoint of mean change in pain
Slide 5: Release of first WOMAC scores from TGA special access scheme
Release of first WOMAC scores from TGA special access scheme For personal use only "We need real-world effectiveness data and the analytics to improve access to medicines from real-world data, not just from clinical trials1.” CSL Chief Executive, Paul Perreault. § Paradigm previously reported greate
Slide 6: Mean reduction in WOMAC pain from baseline to Day 81 was 45%
Release of first WOMAC scores from TGA special access scheme For personal use only Mean reduction in WOMAC pain from baseline to Day 81 was 45% Mean Baseline value (95% Mean Post-treatment value Mean Reduction in Pain (percentage) WOMAC Pain Questionnaire Confidence Interval) (95% Confidence Interva
Slide 7: Comparing WOMAC scores from TGA Special Access Scheme to Opiates
Comparing WOMAC scores from TGA Special Access Scheme to Opiates For personal use only In first 34 patients under TGA SAS, PPS has shown a mean reduction in WOMAC pain scores of 45% following the initial 6-week treatment course., Mean reduction in WOMAC pain Additional Comments score at week 12. Pen
Slide 8: Release of first WOMAC scores from TGA special access scheme
Release of first WOMAC scores from TGA special access scheme For personal use only § Note: All subjects with NRS and WOMAC scores at baseline and post-baseline are summarised. % Change from Baseline = ((Result at post-baseline visit - Result at baseline)/Result at baseline)*100 WOMAC scores are grou
Slide 9: Release of PGIC Scores from WOMAC cohort
Release of PGIC Scores from WOMAC cohort TGA special access scheme (Patient Global Impression of Change). For personal use only PGIC Score (Subjects with WOMAC scores) Visit PGIC Scores WOMAC Subjects (N = 34) Post-Baseline No Change (or condition has got worse) 0 Almost the same, hardly any change
Slide 10: Upcoming Milestones/News Flow
Upcoming Milestones/News Flow 2020 For personal use only § Compassionate Use program with NFL ‘Pro Players Elite Network’ ü Early 2020: US Trial initiation (n=10) – First Patient Dosed Feb, All patients dosed March (See ASX ANN 21 Feb, 24 Mar) st th § Q3 CY2020: Results released § Pivotal Phase 3 OA
Slide 11: Capital Raising Details
For personal use only CAPITAL RAISING DETAILS 11 Release of FDA Minutes and Capital Raising – April 2020
Slide 12: Capital Raising Structure
Capital Raising Structure For personal use only § A$35m Placement to Sophisticated and CAPITAL STRUCTURE Professional investors at A$1.30 per share Current Shares on issue 197.8m § A$1.30 per share represents: Placement Shares Issued 26.9m § 23% discount to last closing price of $1.69 Shares on issu
Slide 13: Use Of Funds & Cash Outflows
Use Of Funds & Cash Outflows For personal use only § A$35m will be applied to the additional cost of the 2nd Phase 3 OA trial and put PAR in a fully funded position until the end of 2022 with pivotal trial readouts in OA and MPS. § Updated cost of PH3 OA trials is estimated at A$80m comprising: § 1s
Slide 14: Indicative Timetable
Indicative Timetable For personal use only Trading Halt 6 April 2020 Company resumes trading 8 April 2020 Settlement of New Shares issued under the Placement T+3 trading days 14 Release of FDA Minutes and Capital Raising – April 2020
Slide 15: Overview of PPS / Zilosul
For personal use only OVERVIEW OF PPS / ZILOSUL 15 Release of FDA Minutes and Capital Raising – April 2020
Slide 16: Background - Pentosan Polysulfate Sodium (PPS) / Zilosul
Background - Pentosan Polysulfate Sodium (PPS) / Zilosul For personal use only Pentosan Polysulfate Sodium Excellent Safety Profile § Semi-synthetic drug manufactured from beech-wood hemicellulose § Well established safety profile with no reported serious adverse events § Has been used in humans for
Slide 17: Exclusive Supply & Manufacturing
Exclusive Supply & Manufacturing For personal use only Exclusive Supply PPS Buy per/kg License Exclusivity Royalty § Long term exclusive supply agreement with bene pharmaChem GmbH. § Agreement grants exclusive supply of only FDA approved PPS for § Bene pharmaChem: original developer of PPS and only
Slide 18: Strong Patents & IP Position
Strong Patents & IP Position Multi-faceted IP protection increases barriers to entry for potential competitors For personal use only § Patent protection using PPS for new indications EU patents granted for BMEL, respiratory diseases § Minimum life on patents is 2030 and beyond for more recent patent
Slide 19: Mechanism Of Action (MOA)
Mechanism Of Action (MOA) For personal use only Reduction of NGF as mediator of PAIN. Inhibition of the cartilage degrading enzymes that are known to § PPS downregulates NGF expression in osteocytes knee OA play a key role in the progression of OA. patients § Reduction in serum COMP & ADAMTS-5 in P2
Slide 20: Bone Marrow Lesions: Clinical Implications for Knee OA
BONE MARROW LESIONS (BML): CLINICAL IMPLICATIONS FOR KNEE OA AND DISEASE REGRESSION WITH iPPS THERAPY For personal use only BML appear as increased signal intensity within the bone marrow INCREASING PAIN INCREASED CARTILAGE LOSS HIGH RISK OF JOINT DESTRUCTION Grade 3 medial tibial BML at baseline HI
Slide 21: OA Phase 2b Trial Results
For personal use only OA PHASE 2B TRIAL RESULTS PRIMARY, SECONDARY & EXPLORATORY ENDPOINTS MET 21 Release of FDA Minutes and Capital Raising – April 2020
Slide 22: Summary Phase Data – PPP (NRS 4-6)
SUMMARY PHASE 2 DATA – PPP (NRS 4-6) For personal use only CLINICALLY MEANINGFUL - % of subjects with >50% reduction STRUCTURAL CHANGES - Median % Change from Baseline: in KOOS pain from BL- (PPP NRS 4-6) BMEL Volume in Knee (PPP NRS 4-6) 60 40.0% Percentage of Subjects 30.0% 50 P<0.026 P<0.021 20.0
Slide 23: Phase 2b OA/BML Clinical Trial – Primary And Secondary Endpoints
Phase 2b OA/BML Clinical Trial – Primary And Secondary Endpoints For personal use only iPPS was well tolerated, effective and clinically meaningful in a Phase 2b randomised, double-blind, placebo- controlled, multi-centre clinical trial § Primary endpoint met - change in KOOS pain score from baselin
Slide 24: Recent OA Transactions - Highlights Pharma Interest In OA
Recent OA Transactions - Highlights Pharma Interest In OA For personal use only COMPANIES COMPOUND REGION UPFRONT TOTAL VALUE STATUS Anti-NGF Global US$200m US$1.8bn Phase 3 (Failed) Anti-NGF Global US$250m US$1.25bn Phase 3 Corticosteroid Global Take-over* US$1.0bn* Commercialised Anti-NGF Global (
Slide 25: Market Demand – OA Stages And Treatments
Market Demand – OA Stages And Treatments For personal use only There are no effective treatments for Moderate to Severe OA Effective Non-validated Ineffective Analgesics, NSAIDS, Corticosteroids Opioids *Platelet-rich Plasma, *Stem Cells, *Royal Australian College of General Practitioners hold a ‘st
Slide 26: Successful Re-Purposed Drugs
Successful Re-Purposed Drugs For personal use only BRAND ORIGINAL PHARMA PEAK ANNUAL NEW INDICATION NAME INDICATION COMPANY SALES SPRAVATO Anaesthetic (Ketamine) Treatment Resistant Depression Janssen/J&J Approved March 2019 Structural Analogue of REVLIMID Multiple Myeloma Celgene $9.7B (2018) THALO
Slide 27: Revenue Potential in OA
REVENUE POTENTIAL IN OA For personal use only US MARKET – aiming for approval 2022 Key Assumptions: 10% 20% 30% § 31m OA sufferers in the US Market Penetration Market Penetration Market Penetration § 3m OA sufferers in Aus US$1.5k p.a US$6.2B p.a US$12.0B p.a US$18.6B p.a INDICITATIVE § Annual dosin
Slide 28: Mucopolysaccharidoses (MPS)
For personal use only Mucopolysaccharidoses (MPS) 28 Release of FDA Minutes and Capital Raising – April 2020
Slide 29: Mucopolysaccharidoses (MPS)
Mucopolysaccharidoses (MPS) MPS – An Orphan Indication in need of new treatments For personal use only In November 2018, Paradigm in-licensed the MPS indication from the Icahn School of Medicine at Mount Sinai, New York. The License includes successful Phase 2a safety and efficacy data MPS Market Fa
Slide 30: Mucopolysaccharidoses (MPS) – Patient Centric Program
Mucopolysaccharidoses (MPS) – Patient Centric Program For personal use only § Paradigm presented it’s MPS VI poster at the MPS World Symposium in Orlando, Florida on the 11th Feb. § Poster presented outcomes of patient focus group which aimed to identify and validate clinical development program end
Slide 31: MPS Objective Clinical Data – Phase 2 PPS In MPS Type 1
Mucopolysaccharidosis (MPS) Objective Clinical Data – Phase 2 PPS In Subjects With MPS Type 1. PPS Administered Concomitantly With Aldurazyme (ERT). For personal use only Hennermann J et al “Treatment with pentosan polysulphate in patients with MPS I: results from an open label, randomized, monocent
Slide 32: Disclaimer
Disclaimer For personal use only This document, together with any information communicated by Paradigm Biopharmaceuticals Ltd (known as “Paradigm”, “Paradigm Biopharma” or “the Company”), in any presentation or discussion relating to this document (collectively, “Information”) is confidential, and h
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