Proqr Therapeutics N.v. · NASDAQ:PRQR
Proqr Therapeutics — Technical Presentation (July 2020)
22 slides from Proqr Therapeutics's technical presentation, Jul 20, 2020
Slide 1: Forward looking statements
Forward looking statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forwar
Slide 2: >2,000,000 people living with inherited retinal disease
>2,000,000 people living with Inherited retinal disease P r o Q R T h e r a p e u t i c s – E x p e r t P e r s p e c t i v e C a l l 3
Slide 3: Very few have a treatment
Very few have a treatment >2,000,000 people living with Inherited retinal disease P r o Q R T h e r a p e u t i c s – E x p e r t P e r s p e c t i v e C a l l 4
Slide 4: RNA therapies in pipeline
RNA therapies in pipeline >100,000 IRD patients for 50,000 45,000 ~10,000 40,000 35,000 ~7,000 30,000 25,000 ~16,000 20,000 15,000 10,000 5,000 0 Sepofarsen for LCA10 QR-421a for Usher Syndrome QR-1123 for P23H adRP QR-411 for Usher Syndrome QR-1011 for Stargardt’s Disease QRX-461 for Usher Syndrome
Slide 5: ProQR RNA therapy development pipeline
ProQR RNA therapy development pipeline REGISTRATIONAL Ophthalmology P r o Q R T h e r a p e u t i c s – E x p e r t P e r s p e c t i v e C a l l 6 Productive discovery engine leading to steady expansion of the pipeline of molecules targeting Inherited Retinal Diseases Sepofarsen (QR-110) for LCA10
Slide 6: Sepofarsen (QR-110) for LCA10
Sepofarsen (QR-110) for LCA10 LCA10 Lose sight in first years of life No approved therapy currently available p.Cys998X mutation affects ~2,000 patients in the Western world RNA therapy: sepofarsen Goal: Restore vision/ prevent vision loss in patients with LCA10 Locally adminis-tered in the eye. Rou
Slide 7: Phase 1/2 + extension trial design
Phase 1/2 + extension trial design Open label, extension trial, LCA10 patients with 1 or 2 copies of p.Cys998X = DSMC review P r o Q R T h e r a p e u t i c s – E x p e r t P e r s p e c t i v e C a l l 8 001 Study Screening baseline 12 months treatment in worse eye Adult 160/80µg dose Adult 320/160
Slide 8: Phase 1/2 showed rapid and sustained benefit
Phase 1/2 showed rapid and sustained benefit Treated Subjects (n=11) BCVA across 2 cohorts Mobility FST 0,2 0,1 0 -0,1 -0,2 -0,3 -0,4 -0,5 -0,6 -0,7 -0,8 0,2 3,5 Impaired Acuity Less Sensitive Less Impairment 3 0 2,5 -0,2 2 -0,4 * 1,5 -0,6 1 -0,8 0,5 -1 0 -1,2 -0,5 More More Sensitive Improved Acuit
Slide 9: Target registration dose – key outcomes
Target registration dose – key outcomes Onset of effect at month BCVA 3, sustained out to month 12 FST Mobility 0,2 0,2 4,5 4 3,5 3 2,5 2 1,5 1 0,5 0 Impaired Acuity Less Sensitive Less Impairment 0 0 -0,2 -0,2 -0,4 -0,4 -0,6 -0,6 -0,8 -0,8 -1 -1 -1,2 -0,5 More More Sensitive Improved Acuity Impairm
Slide 10: InSight open label extension study update
InSight open label extension study update Preliminary data P r o Q R T h e r a p e u t i c s – E x p e r t P e r s p e c t i v e C a l l P r e l i m i n a r y d a t a 11
Slide 11: InSight study overview
InSight study overview • 9 • 7 subjects from the 11 in Phase 1/2 rolled over into the InSight study 2 subjects elected not to roll over for personal reasons subjects have been dosed so far in the extension study, 4 of which have • been dosed in the second eye • • All subjects have been switched over
Slide 12: Change from baseline to months post dosing
Change from baseline to 3 months post dosing -3 -3 -2,5 -2,5 -2 -2 -1,5 -1,5 -1 -1 -0,5 -0,5 0 0 BCVA FST Red FST Blue BCVA FST Red FST Blue BCVA FST Red FST Blue BCVA FST Red FST Blue 1st eye *= 6 month value of 2nd eye as 3 month visit was missed due to COVID-19 P r o Q R T h e r a p e u t i c s –
Slide 13: Change from baseline: consistent treatment response in both eyes
Change from baseline to 3 months post dosing Consistent treatment response in both eyes -3 -3 -2,5 -2,5 -2 -2 -1,5 -1,5 -1 -1 -0,5 -0,5 0 0 BCVA FST Red FST Blue BCVA FST Red FST Blue BCVA FST Red FST Blue BCVA FST Red FST Blue 1st eye 2nd eye *= 6 month value of 2nd eye as 3 month visit was missed
Slide 14: Subject P2
Subject P2 160 μg 80 μg 80 μg Baseline characteristics 0,5 0 -0,5 -1 -1,5 -2 -2,5 -3 -3,5 2 1,5 1 0,5 0 -0,5 -1 -1,5 1,5 1 0,5 0 -0,5 -1 -1,5 -2 BCVA Change from baseline • • 41yrs, Male, Heterozygous Light Perception, both eyes Treatment response • First eye • Significant and sustained benefit FST
Slide 15: Subject P3
Subject P3 160 μg 80 μg 80 μg 80 μg Baseline characteristics 0,5 0 -0,5 -1 -1,5 -2 -2,5 0,5 BCVA Change from baseline • • 44yrs, Male, Heterozygous 1st eye: 2.4 LogMAR(HM); 2nd eye: 1.66 LogMAR(CF) Treatment response • First eye FST red Change from baseline • Significant and sustained benefit 0 • De
Slide 16: Subject P11
Subject 160 μg P11 Baseline characteristics 0,05 0 -0,05 -0,1 -0,15 -0,2 -0,25 -0,3 -0,35 0,2 0 -0,2 -0,4 -0,6 -0,8 -1 0,2 0 -0,2 -0,4 -0,6 -0,8 -1 BCVA Change from baseline • • 14yrs, Female, Homozygous 1st eye: 0.63 LogMAR(20/80); 2nd eye: 0.48 LogMAR(20/50) Treatment response • First eye FST red
Slide 17: Subject P1
Subject P1 160/80 μg 80 μg 80 μg 80 μg 80 μg 80 μg Baseline characteristics 0,5 0 -0,5 -1 -1,5 -2 -2,5 0,5 0 -0,5 -1 -1,5 -2 -2,5 -3 0,5 0 -0,5 -1 -1,5 -2 -2,5 BCVA Change from baseline • • 19yrs, Male, Heterozygous Light Perception, both eyes Treatment response • First eye • No response on BCVA as
Slide 18: Summary and next steps
Summary and next steps • 4 out of 4 second eyes treated have responded consistently with the confirming the benefit seen in Phase 1/2 Safety profile consistent with that observed in the Phase 1/2 results Data supports selection of 160/80 µg target registration dose Additional longitudinal data suppo
Slide 19: Change from baseline: consistent treatment response in both eyes
Change from baseline to 3 months post dosing Consistent treatment response in both eyes -3 -3 -2,5 -2,5 -2 -2 -1,5 -1,5 -1 -1 -0,5 -0,5 0 0 BCVA FST Red FST Blue FST Red FST Blue BCVA FST Red FST Blue 1st eye 2nd eye *= 6 month value of 2nd eye as 3 month visit was missed due to COVID-19 P r o Q R T
Slide 20: Subject P3: Illustration of BCVA improvement
Subject P3: Illustration of BCVA improvement • Baseline: 2.4 LogMAR (20/1000) Baseline (2.4 LogMAR) M3 (1.12 LogMAR) M9 (0.58 LogMAR) • 3 months: 1.12 LogMAR (20/125) • Improvement: 1.28 LogMAR* *From being worse than legally blind to navigating freely, watching tv and being able to see family faces
Slide 21: Subject P3: Illustration of BCVA improvement
Subject P3: Illustration of BCVA improvement ° Baseline: Baseline (2.4 LogMAR) M3 (1.12 LogMAR) M9 (0.58 LogMAR) 2.4 LogMAR (20/1000) re ee * 3 months: 1.12 LogMAR (20/125) * Improvement: 1.28 LogMAR* *From being worse than legally blind to navigating freely, watching tv and being able to see family
Slide 22: It's in our RNA
\ IT'S IN OUR RNA
ProQR Therapeutics — Technical Presentation (July 2020) on 6ix