Pacira BioSciences, Inc. · NASDAQ:PCRX
Pacira BioSciences — Technical Presentation (2023)
24 slides from Pacira BioSciences's technical presentation, 2023
Slide 1: Stanley Cohen, MD, Metroplex Clinical Research Center
Stanley Cohen, MD Metroplex Clinical Research Center, Dallas, TX I have a financial relationship(s) with: Investigator, Pacira BioSciences, Inc. AND My presentation does include a discussion of investigational use. PCRX201 is utilized in a phase 1, open-label, single ascending dose study
Slide 2: Safety and Preliminary Efficacy of PCRX201, Intra-Articular Gene Therapy for Knee OA
SAFETY AND PRELIMINARY EFFICACY OF PCRX201, AN INTRA-ARTICULAR GENE THERAPY FOR KNEE OSTEOARTHRITIS: A PHASE 1, OPEN-LABEL, SINGLE ASCENDING DOSE STUDY Stanley Cohen,1 Alan Kivitz,2 Lynell Klassen,3 Masato Nakazawa,4 Dennis Parenti,4 Mary DiGiorgi,4 Roy Winston4 1Metroplex Clinical Research Center,
Slide 3: Background
Background IL-1 may be an important driver of inflammation, pain, and potentially disease progression in OA.1 Gene therapy has the potential to offer long-term expression of a therapy at the disease site.2 • Novel helper-dependent adenovirus therapy1 • Devoid of viral elements except for the followi
Slide 4: Open-label Study Design
Open-label Study Design Inclusion criteria Study design • Adults aged 30 to 80 years with moderate-to- severe knee OA • K/L scale grade 2-4 • Prior treatment failure of ≥2 other therapies for OA • Baseline WOMAC pain score ≥4.0 and ≤9.0 Cohort 1: Not pretreated (n=36) Cohort 2: Steroid-pretreated (n
Slide 5: Patient Disposition and Baseline Characteristics
Patient Disposition and Baseline Characteristics Not pretreated Steroid- cohort pretreated cohort 72 patients (n=36) (n=36) enrolled* Median age (IQR), y 62.0 (58.5-67.0) 67.5 (58.5-71.5) Gender, n (%) Not pretreated Steroid-pretreated cohort cohort† Male 15 (41.7) 15 (41.7) (initial) (subsequent) F
Slide 6: Summary of Treatment-Related Adverse Events (TRAEs)
Summary of Treatment-Related Adverse Events (TRAEs) Treatment-Related Index Knee Effusions Other TRAEs Not Steroid- Not Steroid- pretreated pretreated pretreated pretreated cohort cohort cohort cohort (n=36) (n=36) (n=36) (n=36) Knee effusion, n/N (%) Self-limited chills, n/N (%) Low-dose 6/12 (50)
Slide 7: Preliminary Data on Impact of Baseline Systemic NAbs on Safety and WOMAC Scores
Preliminary Data to Assess the Impact of Baseline Systemic NAbs on Safety and WOMAC Scores • Preliminary immunogenicity data include the first 13 patients (5 low dose and 8 moderate dose), assessed at week 26 Systemic NAb positivity at baseline versus knee- Systemic NAb positivity at baseline versus
Slide 8: % Change in WOMAC-A From Baseline by Dose and Treatment
% Change in WOMAC-A From Baseline by Dose and Treatment* Not pretreated Steroid-pretreated Estimated % change from baseline Week, sqrt Patient disposition by visit Cohort Dose Improvements in knee pain were observed across all dose groups and cohorts receiving PCRX201 Pretreatment with steroids did
Slide 9: WOMAC-A Pain Score: Percentage of Patients With ≥50% Decline From Baseline
WOMAC-A Pain Score: Percentage of Patients With ≥50% Decline From Baseline* from baseline in WOMAC-A pain score % of participants with ≥50% decline Not pretreated Steroid-pretreated Week (sqrt) Preliminary efficacy results suggest substantial improvement in pain across all PCRX201 cohorts *Prelimina
Slide 10: Conclusions
PCRX201 was generally well tolerated, with main TRAEs including index 1 knee pain and effusion, which generally resolved within weeks with conservative care or IA steroids There were no treatment-related SAEs, deaths, or late recurrences 2 and no unusual patterns of nonrelated AEs in follow-up Concl
Slide 11: Thank you!
Thank you!
Slide 12: Backup
Backup 12
Slide 13: % Change in WOMAC-A at Week 38 by Dose and Treatment
% Change in WOMAC-A at Week 38 by Dose and Treatment Estimated % change from baseline at week 38 Not pretreated Steroid-pretreated Low Dose Mid Dose High Dose Low Dose Low Dose High Dose n=12 n=14 n=7 n=5 n=9 n=4 WOMAC, Western Ontario and McMaster Universities Osteoarthritis Index. 13
Slide 14: Change in WOMAC-A Pain Scores by K/L Grade
Change in WOMAC-A Pain Scores by K/L Grade* Not pretreated Steroid-pretreated Mean % change from baseline in WOMAC-A K/L Grade 2 Grade 3 Grade 4 Week (sqrt) *Preliminary results of observed data, with no imputations of missing values, through up to 6 months after treatment administration in the last
Slide 15: WOMAC-A Mean Percent Change: Low-Dose Cohorts
WOMAC-A Pain Score: Mean Percent Change From Baseline in the Not Pretreated and Steroid-Pretreated Low-Dose Cohorts* Low-Dose Cohort Not pretreated Steroid-pretreated % change from baseline Week (sqrt) *Preliminary results of observed data, with no imputations of missing values, through up to 6 mont
Slide 16: WOMAC-A Mean Percent Change: Mid-Dose Cohorts
WOMAC-A Pain Score: Mean Percent Change From Baseline in the Not Pretreated and Steroid-Pretreated Mid-Dose Cohorts* Mid-Dose Cohort Not pretreated Steroid-pretreated % change from baseline Week (sqrt) *Preliminary results of observed data, with no imputations of missing values, through up to 6 mont
Slide 17: WOMAC-A Mean Percent Change: High-Dose Cohorts
WOMAC-A Pain Score: Mean Percent Change From Baseline in the Not Pretreated and Steroid-Pretreated High-Dose Cohorts* High-Dose Cohort Not pretreated Steroid-pretreated % change from baseline Week (sqrt) *Preliminary results of observed data, with no imputations of missing values, through up to 6 mo
Slide 18: Change from WOMAC-A Baseline in the Cohorts
Change from WOMAC-A Baseline in the 2 Cohorts* Not pretreated Steroid-pretreated Mean % change from baseline Week Patient disposition by visit Cohort Dose *Preliminary results of observed data, with no imputations of missing values, through up to 6 months after treatment administration in the last p
Slide 19: % Change in WOMAC-A by Dose and Treatment
% Change in WOMAC-A by Dose and Treatment* Not pretreated Steroid-pretreated Estimated % change from baseline Week Patient disposition by visit Cohort Dose *Covariates in the model include cohort, steroid pretreatment, all visits up to week 38, cohort by visit interaction, pretreatment by visit inte
Slide 20: WOMAC-A Pain Score: Percentage of Patients With ≥50% Decline From Baseline
WOMAC-A Pain Score: Percentage of Patients With ≥50% Decline From Baseline* from baseline in WOMAC-A pain score % of participants with ≥50% decline Not pretreated Steroid-pretreated Week Preliminary efficacy results suggest substantial improvement in pain across all PCRX201 cohorts *Preliminary resu
Slide 21: Change in WOMAC-A Pain Scores by K/L Grade
Change in WOMAC-A Pain Scores by K/L Grade* Not pretreated Steroid-pretreated Mean % change from baseline in WOMAC-A K/L Grade 2 Grade 3 Grade 4 Week *Preliminary results of observed data, with no imputations of missing values, through up to 6 months after treatment administration in the last patien
Slide 22: WOMAC-A Mean Percent Change: Low-Dose Cohorts
WOMAC-A Pain Score: Mean Percent Change From Baseline in the Not Pretreated and Steroid-Pretreated Low-Dose Cohorts* Low-Dose Cohort Not pretreated Steroid-pretreated % change from baseline Week *Preliminary results of observed data, with no imputations of missing values, through up to 6 months afte
Slide 23: WOMAC-A Mean Percent Change: Mid-Dose Cohorts
WOMAC-A Pain Score: Mean Percent Change From Baseline in the Not Pretreated and Steroid-Pretreated Mid-Dose Cohorts* Mid-Dose Cohort Not pretreated Steroid-pretreated % change from baseline Week *Preliminary results of observed data, with no imputations of missing values, through up to 6 months afte
Slide 24: WOMAC-A Mean Percent Change: High-Dose Cohorts
WOMAC-A Pain Score: Mean Percent Change From Baseline in the Not Pretreated and Steroid-Pretreated High-Dose Cohorts* High-Dose Cohort Not pretreated Steroid-pretreated % change from baseline Week *Preliminary results of observed data, with no imputations of missing values, through up to 6 months af