Percheron Therapeutics Limited · ASX:PER
Percheron Therapeutics — Technical Presentation (February 2025)
25 slides from Percheron Therapeutics's technical presentation, Feb 6, 2025
Slide 1: ASX Announcement: Investor Update Webinar Presentation
ASX Announcement INVESTOR UPDATE WEBINAR PRESENTATON Melbourne, Australia – 6 February 2025: Percheron Therapeutics Limited (ASX: PER) (‘the Company’), an international biotechnology company focused on the development of novel therapies for rare diseases, is pleased to provide investors with further
Slide 2: Phase IIb Study of Avicursen (ATL1102) in Duchenne Muscular Dystrophy
For personal use only Phase IIb Study of Avicursen (ATL1102) in Duchenne Muscular Dystrophy Review of Six-Month Data Dr Cathryn Clary Chief Medical Advisor 6 February 2025 www.PercheronTx.com ASX: PER | US OTCQB: PERCF
Slide 3: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements do not relate strictly to historical or current facts and may be accompanied by words such as ‘c
Slide 4: Introduction
Introduction • Antisense Therapeutics previously completed an open-label phase IIa pilot study of ATL1102 in nine non-ambulant boys with Duchenne muscular dystrophy (DMD). For personal use only • The study was conducted at the Royal Childrens’ Hospital in Melbourne, one of the leading DMD treatment
Slide 5: Background
3 For personal use only Background
Slide 6: Duchenne muscular dystrophy is an incurable genetic condition
Duchenne muscular dystrophy is an incurable genetic condition that affects approximately 300,000 children and young adults worldwide For personal use only 1 Inherited genetic Duchenne Incidence is DMD also abnormality renders patient muscular approximately associated with unable to produce normal 2
Slide 7: Phase IIb study designed in three sections with placebo-controlled read-out
The phase IIb study was designed in three sections, with a placebo-controlled primary read-out at six months to meet regulatory requirements For personal use only Patient Population Blinded Treatment Open-Label Treatment Follow-Up Period Placebo Period Period • Genetically (n≈16) confirmed DMD • 6 m
Slide 8: PUL2.0 primary endpoint; MyoGrip and MyoPinch secondary endpoints
PUL2.0 is the primary endpoint of the study; MyoGrip and MyoPinch are included as secondary efficacy endpoints Performance of the Upper Limb Module 2.0 (PUL2.0) MyoGrip and MyoPinch For personal use only MyoGrip dynamometer MyoPinch device • PUL2.0 is a ‘functional endpoint’ – it measures the abilit
Slide 9: Topline Study Data (6-Month Endpoint)
For personal use only Topline Study Data (6-Month Endpoint) 7
Slide 10: Baseline Characteristics
Baseline Characteristics In general, treatment groups were well-balanced at baseline Placebo ATL1102 25mg ATL1102 50mg Commentary For personal use only Sample size, n 17 16 15 • In general, the three arms of the study were well-balanced, with no meaningful differences between them, implying Age, yea
Slide 11: Overall PUL2.0 Measurement at Weeks
Overall PUL2.0 Measurement at 25 Weeks No significant difference in the primary endpoint between the three arms of the study Placebo ATL1102 ATL1102 Placebo ATL1102 ATL1102 Commentary 25mg 50mg 25mg 50mg For personal use only 0.0 • There was no statistically significant ANCOVA Analysis (Primary) dif
Slide 12: PUL2.0 Domains at Weeks
PUL2.0 Domains at 25 Weeks No significant differences between arms in constituent domains of PUL2.0 High-Level Shoulder Domain Mid-Level Elbow Domain Distal-Level Wrist Domain For personal use only Placebo ATL1102 ATL1102 Placebo ATL1102 ATL1102 Placebo ATL1102 ATL1102 25mg 50mg 25mg 50mg 25mg 50mg
Slide 13: MyoGrip and MyoPinch
MyoGrip and MyoPinch No significant differences between treatment groups on MyoGrip and MyoPinch secondary endpoints Commentary MyoGrip MyoPinch For personal use only • MyoGrip and MyoPinch secondary efficacy Placebo ATL1102 ATL1102 Placebo ATL1102 ATL1102 endpoints showed no statistically significa
Slide 14: Safety Outcomes
Safety Outcomes ATL1102 was generally safe and well-tolerated Commentary Placebo ATL1102 25mg ATL1102 50mg For personal use only • ATL1102 was generally safe and well- Number of Patients, n tolerated. No patients discontinued either drug treatment or study participation as a Patients 17 16 15 result
Slide 15: Analysis
13 For personal use only Analysis
Slide 16: Placebo Group Comparison
Placebo Group Comparison In general, our placebo group behaved consistently with prior studies of other drugs in similar populations Fibrogen Capricor Percheron Commentary Placebo Group Behaviour Pamrevlumab CAP-1002 ATL1102 For personal use only LELANTOS-1 HOPE-2 Phase IIb Study • The most directly
Slide 17: Pharmacokinetic – Pharmacodynamic Effects
Pharmacokinetic – Pharmacodynamic Effects ATL1102 appears to engage its target in a dose-dependent fashion, causing intended effects in muscle Pharmacokinetics Pharmacodynamics Biomarker For personal use only (ATL1102 Concentration after 2 hrs) (CD49d+ T-Cells at wk 22) (Serum Creatine Kinase at wk
Slide 18: Treatment Effect Beyond Six Months
Treatment Effect Beyond Six Months No evidence of activity in those patients transitioning from placebo to active after six months Commentary Mean Change in 25mg ATL1102 50mg ATL1102 PUL2.0 (weeks 25 – 37) (weeks 25 – 37) For personal use only • It was intended that all patients would complete twelv
Slide 19: Comparison Against Phase IIa Study
For personal use only Comparison Against Phase IIa Study 17
Slide 20: Comparison Against Phase IIa Study
Comparison Against Phase IIa Study The phase IIa and IIb studies recruited similar populations Commentary Phase IIa Study Phase IIb Study For personal use only • The study population in the phase IIb study ATL1102 25mg Placebo ATL1102 25mg ATL1102 50mg appears broadly similar to that in the phase II
Slide 21: Drugs promising in open label studies sometimes disappoint in randomised studies
Drugs which appear promising in open label exploratory studies sometimes disappoint in confirmatory randomised controlled studies… Peer-Reviewed Publication of Pamrevlumab Single-Arm Fibrogen Press Release of 7 June 2023 For personal use only Open-Label Phase II MISSION Study in Non-Ambulant DMD Des
Slide 22: Positive randomised data not guaranteed of success in confirmatory studies
…and even drugs which showed positive data in randomised studies are not guaranteed of success in confirmatory studies Peer-Reviewed Publication of Puldysa® (Idebanone) Santhera Press Release of 6 October 2020 For personal use only Randomised Phase III DELOS Study in DMD Describing Approval Phase II
Slide 23: Conclusions
21 For personal use only Conclusions
Slide 24: Conclusions
Conclusions • There were no indications of manufacturing, storage, or administration defects with the ATL1102 drug product. The study appears to have been conducted substantially in accordance with the protocol. For personal use only • The drug is measurable in plasma, causes dose-dependent reductio
Slide 25: For personal use only
For personal use only info@PercheronTx.com www.PercheronTx.com
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