Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — Corporate Update (February 2021)
16 slides from Ptc Therapeutics's corporate update, Feb 4, 2021
Slide 1: Translarna Study 045 and Clinical Update
Translarna™ Study 045 and Clinical Update Stuart W. Peltz, Ph.D. Chief Executive Officer
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 . All statements contained in this presentation, other than statements of historic fact, are forward -looking statements, including statements re
Slide 3: Study 045 Design
Study 045 Design* 3 • Ambulatory nmDMD pts aged ≥2 to <8 years Key Eligibility Criteria • Primary • Change from baseline to Week 40 in dystrophin expression, electrochemiluminescence (ECL) assay • Secondary • Change from baseline to Week 40 in dystrophin expression, immunohistochemistry (IHC) assay
Slide 4: Analytical Methods – ECL and IHC Assays
Analytical Methods – ECL and IHC Assays 4 • Quantitative measurement of total dystrophin protein levels • Utilizes electrochemiluminescence technology from Meso Scale Diagnostics ECL Assay • Provides quantifiable measurement of dystrophin localized to the membrane of the muscle cell (consistent with
Slide 5: Study Population Summary
Study Population Summary 5 Text Here Text Here Text Here Enrolled Subjects (ITT Population) (n=20) Evaluable Population (n=18) Subjects with Longer Treatment at Time of 2nd Biopsy (n=8) Text Here Text Here Subjects with Protocol- Specified Treatment Duration at Time of 2nd Biopsy (n=10) • 1 subject
Slide 6: Translarna Treatment Resulted in Increased Dystrophin Expression on both ECL and IHC Assays
Translarna™ Treatment Resulted in Increased Dystrophin Expression on both ECL and IHC Assays *Exploratory; not adjusted for multiplicity 6 Mean % Change from Baseline ITT Population (n=20) Evaluable Population (n=18) ECL Assay IHC Assay ECL Assay* IHC Assay* 6.56% (p=0.24) 4.91% (p=0.11) 9.20% (p=0.
Slide 7: Longer Treatment Duration Resulted in Greater Dystrophin Expression Increase
Longer Treatment Duration Resulted in Greater Dystrophin Expression Increase 7 9.2 23.9 7.0 10.8 ECL IHC Evaluable Population (n=18) Longer Treatment Subgroup (n=8) Mean % Change from Baseline Exploratory; not adjusted for multiplicity
Slide 8: Translarna Treatment Resulted in Reduction in Creatine Kinase (CK) Levels
Translarna Treatment Resulted in Reduction in Creatine Kinase (CK) Levels 8 Mean % Reduction from Baseline (CK) 11.9 19.2 Evaluable Population (n=18) Longer Treatment Subgroup (n=8) Adapted from Goemans N, et al. 20144 Exploratory; not adjusted for multiplicity
Slide 9: Translarna Treatment Resulted in Clinical Improvement
Translarna Treatment Resulted in Clinical Improvement 9 Timed Function Tests (TFTs) and North Star Ambulatory Assessment Mean Change from Baseline (Sec) 0.56 0.74 3.30 1.30 0.30 1.45 1.51 1.17 Descend Stair Stand From Supine Run/Walk 10m Climb Stair Longer Treatment Subgroup (n=8) Evaluable Populati
Slide 10: Study Trends Support Translarna Benefit
Study 045 Trends Support Translarna Benefit 10 Translarna treatment resulted in increased dystrophin expression as assessed by both ECL and IHC assays Increased Dystrophin Expression Longer treatment with Translarna was associated with a greater increase in dystrophin expression Longer Treatment Gre
Slide 11: Study Adds to Existing Totality of Evidence Supporting Clinical Benefit of Translarna
Study 045 Adds to Existing Totality of Evidence Supporting Clinical Benefit of Translarna *Median exposure Study 019 (Long-term Study) ~3.5 years* N=94 Preservation of pulmonary function Production of full-length dystrophin Study 004 (Dystrophin Study) 28 days N=38 Study 007 (RCT) 48 weeks N=174 Stu
Slide 12: Totality of Evidence Shows Consistent Treatment Effect Across Multiple Clinical Endpoints
Totality of Evidence Shows Consistent Treatment Effect for Translarna Across Multiple Clinical Endpoints Phase 2b and 3 studies did not meet their primary endpoints. *In children aged ≥7 to ≤16 years old. † Primary endpoint. ‡ Secondary endpoint. § Patients from the Phase 2b study that met ACT DMD i
Slide 13: STRIDE Registry Provides Long-term Real-world Evidence of Translarna Benefit
STRIDE Registry Provides Long-term Real-world Evidence of Translarna Benefit 13 Multicenter registry study, evaluating the long-term safety and efficacy in a real-world setting Includes nmDMD patients with varied demographics, providing data that is representative of real- world patient experiences
Slide 14: STRIDE Data Demonstrates Translarna Preserves Ambulation and Pulmonary Function
STRIDE Data Demonstrates Translarna Preserves Ambulation and Pulmonary Function Figure adapted from Mercuri E, et al. J Comp Eff Res. 2020; 9(5):341-360. Data cut off 9 July 2018 14 Prevention of loss of pulmonary function Delay of loss of ambulation by 3.5 years P <0.0001 P <0.0001
Slide 15: Next Steps: Discussion with FDA on Potential Approval Pathways
Next Steps: Discussion with FDA on Potential Approval Pathways 15 STRIDE registry of long-term clinical benefit on key aspects of disease progression Real-World Evidence Clinical benefit across multiple endpoints in several placebo-controlled trials Clinical Results Increased full-length dystrophin
Slide 16: Next Steps: Discussion with FDA on Potential Approval Pathways
Next Steps: Discussion with FDA on Potential Approval Pathways 16 Study 041 Results 3Q 2022 Ongoing Clinical Study STRIDE registry of long-term clinical benefit on key aspects of disease progression Real-World Evidence Clinical benefit across multiple endpoints in several placebo-controlled trials C