Percheron Therapeutics Limited · ASX:PER
Percheron Therapeutics — Shareholder Meeting (December 2019)
26 slides from Percheron Therapeutics's shareholder meeting presentation, Dec 11, 2019
Slide 1: Annual General Meeting 2019
ASX:ANP | OTC:ATHJY ANNUAL GENERAL MEETING 2019
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS This presentation contains forward-looking statements regarding the Company’s business & the therapeutic & commercial potential of its technologies & products in development. Any statement describing the Company’s goals, expectations, intentions or beliefs is a forward-loo
Slide 3: Antisense Therapeutics Overview
ANTISENSE THERAPEUTICS OVERVIEW Australian, Melbourne- Advanced stage Substantial shareholders Phase II clinical trial Potential for out- based product pipeline with include renowned in Duchenne licensing of ATL1103 for biopharmaceutical positive Phase II Australian institutions in Muscular Dystroph
Slide 4: Antisense – What Is It & How Does It Work?
ANTISENSE – WHAT IS IT & HOW DOES IT WORK? Antisense oligonucleotide drugs are small (12-25 nucleotides) DNA or RNA-like compounds that are chemically modified to create medicines Antisense drugs prevent the production of proteins involved in disease processes by interrupting the translation phase o
Slide 5: Antisense Therapeutics Advanced Stage Clinical Pipeline
ANTISENSE THERAPEUTICS ADVANCED STAGE CLINICAL PIPELINE Targeting diseases where there is a need for improved therapies 1 2 3 ATL1102 IN DMD ATL1103 IN ACROMEGALY ATL1102 IN MS • Conducting Phase II • Phase II clinical trial • Phase II clinical trial clinical trial at Royal completed completed Child
Slide 6: ATL1102: Drug, Target & Activity Overview
ATL1102: DRUG, TARGET & ACTIVITY OVERVIEW 2’-o-methoxyethyl ribose 2’-deoxy “gap” 2’-o-methoxyethyl ribose “2’-MOE” “2’-MOE” 3 9 8 5’ RNaseH active 3’ 20mer phosphorothioate backbone • ATL1102 is designed to inhibit CD49d expression on lymphocytes and thereby reduce their survival, activation and mi
Slide 7: What is DMD?
WHAT IS DMD? o Duchenne Muscular Dystrophy (DMD) is a devastating genetic muscular disease caused by loss of dystrophin with progressive muscle wasting & associated muscle injury leading to inflammation &fibrosis (100% mortality) o Affects boys with an incidence of ~1 in 3,500 & prevalence of ~44,00
Slide 8: Why ATL1102 for DMD?
WHY ATL1102 for DMD? • Improved therapies are needed to ameliorate DMD severity & delay disease progression • DMD is an orphan indication so can benefit from IP & development incentives ATL1102, an antisense drug to CD49d, shown to be a highly Pivotal scientific publication confirming CD49d as a pot
Slide 9: ATL1102 Phase II Study
ATL1102 PHASE II STUDY • Trial being led by RCH Head of Neuromuscular Clinic Professor Monique Ryan & RCH Neuromuscular Fellow Dr Ian Woodcock • Neuromuscular clinic at RCH the largest in the Southern Hemisphere for treating boys with DMD Study Title: A Phase 2 open label study to determine the safe
Slide 10: ATL1102 Phase II Study - Safety Overview
ATL1102 PHASE II STUDY - SAFETY OVERVIEW Preliminary Data Presented by Dr Ian Woodcock at Action Duchenne Conference Nov 2019 on first 7 boys to have completed dosing An Independent Data and Safety Monitoring Board (DSMB) provides safety oversight for the study • The DSMB consists of 3 paediatrician
Slide 11: ATL1102 Phase II Study Overview of Efficacy Parameters - Preliminary Data
ATL1102 PHASE II STUDY Overview of Efficacy Parameters - Preliminary Data The Secondary Objectives of the study include evaluation of the following efficacy parameters: • Lymphocyte-modulation potential to be determined by assessing number and percentages of lymphocytes, CD4+ and CD8+ T cells and, C
Slide 12: ATL1102 Phase II Study Overview of Efficacy Parameters - Preliminary Data
ATL1102 PHASE II STUDY Overview of Efficacy Parameters - Preliminary Data Change from Baseline to Week 24 MyoGrip MyoGrip MyoPinch MyoPinch % Predicted % Predicted Patient No. PUL 2.0 (dom) (dom) (dom) (dom) FVC PEF (Kg) (% Pred) (Kg) (% Pred) 01-001 +2 -0.63 -4.49 0.03 -0.62 -3.20 6.30 01-002 +2 0.
Slide 13: Efficacy Parameters – Comparison to Published Data for Myoset Assessments
ATL1102 Phase II Study Efficacy Parameters – Comparison to Published Data for Myoset Assessments 13 * Ricotti et. al 2016 . PLoS One, 11(9) e0162542 historical results from 8 Non – Ambulant patients on CS for 6month
Slide 14: Summary of Safety and Efficacy Parameters - Preliminary Data
ATL1102 PHASE II STUDY Summary of Safety and Efficacy Parameters - Preliminary Data • ATL1102 appears to be generally safe and well tolerated in non-ambulant boys with DMD • No Serious Adverse Events (SAEs) have been reported • The DSMB, responsible for safety oversight of the study, have no safety
Slide 15: Reporting of data from all subjects post completion of dosing
ATL1102 PHASE II STUDY Reporting of data from all 9 subjects post completion of dosing • All 9 participants have completed their 6 months of dosing • The trial is continuing with patients in the 2 month monitoring phase or follow up period of the trial • The Last Participant Last Visit (in follow up
Slide 16: Phase IIb Clinical Trial
PHASE IIB CLINICAL TRIAL • Scientific Advice (SA) meetings held with three European regulatory authorities • SA meetings focussed on the Phase IIb trial design, dose escalation plans, applicability of the study end-points and the study duration. ANP expects to receive written responses month followi
Slide 17: Treatment Development Focusing Across All Intervention Points
TREATMENT DEVELOPMENT FOCUSING ACROSS ALL INTERVENTION POINTS • Prospect for these therapies to be complementary rather than competitive • Other anti-inflammatory therapies are being tested in ambulant children INTERVENTION POINTS TREATMENT PROVIDERS ANTI-FIBROTICS INFLAMMATION STEROIDS & FIBROSIS A
Slide 18: Market Considerations for ATL1102
MARKET CONSIDERATIONS FOR ATL1102 • ATL1102 - anti-inflammatory and immune modulating agent with potential for multiple clinical applications ANTI-INFLAMATORY CORTICOSTEROIDS DMD THERAPIES Anti-Inflammatory The global steroid market The global DMD drug Therapeutics Market^ is is forecast to attain t
Slide 19: Value Creation Potential of ATL1102 for DMD
VALUE CREATION POTENTIAL OF ATL1102 FOR DMD • Approval based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51- • Cost per patient of Exondys 51 is US$300K/year treated patients • 2nd quarter 2019 total net revenue for Exondys 51 – • Sarepta market capit
Slide 20: DMD & Industry Conferences
DMD & INDUSTRY CONFERENCES Meetings ANP may present at and or attend include: DMD related scientific conferences • SOS ACTT Duchenne Conference in Melbourne, Australia, March 2020 • Muscular Dystrophy Association (MDA) conference in Orlando, Florida US, March 2020 • American Academy of Neurology (AA
Slide 21: ATL1102 for Other Autoimmune – Inflammatory Diseases
ATL1102 for OTHER AUTOIMMUNE – INFLAMMATORY DISEASES • ATL1102 targets CD49d+ immune cells involved in disease processes • CD49d is a clinically validated target in multiple disease indications and a potential superior target in other autoimmune inflammatory disease • Pipeline development focus • CD
Slide 22: ANPOB Listed Options & Capital Management Update
ANPOB LISTED OPTIONS & CAPITAL MANAGEMENT UPDATE • ANPOB options monies received to date (>$650K) plus commitments to exercise from holders including Key Management Personnel will take total to in excess of $1million • Further option exercise anticipated ahead of option expiry on 19 December 2019 •
Slide 23: Board of Directors
BOARD OF DIRECTORS Mr Robert W Moses Mr Mark Diamond Dr Graham Mitchell Dr Gary Pace Mr William Goolsbee Independent Non- Managing Director & Independent Non- Independent Non- Independent Non- Executive Chairman Chief Executive Officer Executive Chairman Executive Director Executive Director Formerl
Slide 24: Corporate Overview
CORPORATE OVERVIEW KEY FINANCIALS Market Capitalisation (at $0.08) A$34M Shares on issue 423.6M 52-week high/low $0.145 - $0.023 Options (ANPOB, $0.08 exp. 19/12/19) 64.3M Cash as at 30 September 2019 $2M OWNERSHIP STRUCTURE Top 40 holders 54.27% Substantial Shareholders • Australian Ethical Investm
Slide 25: Antisense Therapeutics Summary & Value Drivers
ANTISENSE THERAPEUTICS SUMMARY & VALUE DRIVERS Advanced stage product pipeline – two compounds with positive Phase II clinical results published in high quality peer reviewed scientific journals with multiple clinical applications Highly regarded Australian institutional shareholders - Australian Et
Slide 26: For more information
For more information: Investment enquiries Mark Diamond Gennadi Koutchin Managing Director XEC Partners +61 (0) 3 9827 8999 +61 423 500 233 www.antisense.com.au gkoutchin@xecpartners.com.au
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