Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — Technical Presentation (March 2022)
16 slides from Biomarin Pharmaceutical's technical presentation, March 2022
Slide 1: Long-term outcomes of MPS IVA patients treated with elosulfase alfa: MARS after 6 years
Long-term outcomes of MPS IVA patients treated with elosulfase alfa: Findings from the Morquio A Registry Study (MARS) after 6 years John Mitchell, MD McGill University Health Centre, Montreal, Quebec, Canada
Slide 2: MARS Study Information
MARS Study Information • MARS* is an ongoing, multinational, observational study of patients with MPS IVA All patients with a confirmed diagnosis of MPS IVA are eligible to participate Patients are not required to receive elosulfase alfa enzyme replacement therapy (ERT) Key objectives: • Characteriz
Slide 3: Subject Disposition
Subject Disposition As of 12 February 2021, a total of 381 subjects at 65 clinical sites located in 17 countries had enrolled As of 12 Feb 2021 ERT-Naïve ERT-Treated* Subjects enrolled in registry 58 323 (Full Analysis Set) Subjects treated with ERT during registry 314 (97%) (Safety Population) Subj
Slide 4: Subject Demographics & Characteristics
Subject Demographics & Characteristics ERT-Naïve ERT-Treated Sex Female 18 (31.0%) 160 (49.5%) Male 40 (69.0%) 163 (50.5%) Age at MPS IVA diagnosis Median 6.5 years 3.4 years Mean 8.9 years 6.3 years At registry entry: Age, median 21.4 years 13.0 years <5 years 4 (6.9%) 58 (18.0%) ≥19 years 34 (58.6
Slide 5: ERT Exposure
ERT Exposure ERT-Treated (N=323) ERT exposure, years Total mean 5.47 min, max 0.11, 11.98 During MARS mean 3.26 min, max 0.06, 5.97 Age at ERT initiation, years median 9.8 min, max 0.3, 69.4 Age group ERT initiation, n (%) <5 years 83 (25.7%) 5 to <12 years 105 (32.5%) 12 to <19 years 54 (16.7%) ≥19
Slide 6: Normalized uKS: Change from Baseline in ERT-Treated Subjects
Normalized uKS: Change from Baseline in ERT-Treated Subjects • Mean % change from baseline to last follow-up was -52.5% (P<0.0001) – Decrease from baseline was consistent with findings from MOR-004/005 clinical trials1,2 30.9 13.6 (2.84) (1.56) Error bars represent standard errors (SE) CI: confidenc
Slide 7: Normalized uKS: Change from Baseline by Treatment Duration
Normalized uKS: Change from Baseline by Treatment Duration • uKS levels declined rapidly after ERT initiation • After 2 years on treatment, uKS levels were ≈60% lower than at baseline • Reductions were sustained at subsequent time points
Slide 8: 6-Minute Walk Test: Change from Baseline in ERT-Treated Subjects
6-Minute Walk Test: Change from Baseline in ERT-Treated Subjects • Mean change from baseline to last follow-up in the 6MWT was -6.1 m, over a mean treatment duration of 5.5 years 225.6 219.6 (11.05) (13.09) Error bars represent SE
Slide 9: 6-Minute Walk Test: Change from Baseline by Treatment Duration
6-Minute Walk Test: Change from Baseline by Treatment Duration • 6MWT distance remained relatively stable over time – In contrast, untreated subjects in the MorCAP natural history study showed a gradual decline of 4.9-6.8 m/year in endurance1 – Initial mean increase from baseline was comparable in m
Slide 10: Respiratory Function: Change from Baseline in ERT-Treated Subjects
Respiratory Function: Change from Baseline in ERT-Treated Subjects FEV1 FVC • Respiratory outcomes were improved or stabilized over the duration of follow-up • Increases observed after 2 years of treatment were similar in magnitude to those observed in clinical trials1,2 1.0 1.1 1.1 1.4 (0.07) (0.08
Slide 11: Respiratory Function: Change From Baseline by Age at Treatment Initiation
Respiratory Function: Change From Baseline by Age at Treatment Initiation • Respiratory function increased in subjects <18 years and remained stable in older subjects FEV1 FVC 0.9 1.2 1.0 1.0 1.1 1.4 1.3 1.3 (0.06) (0.09) (0.15) (0.15) (0.08) (0.11) (0.21) (0.20) Error bars represent SE
Slide 12: Respiratory Function: Change from Baseline by Treatment Duration
Respiratory Function: Change from Baseline by Treatment Duration FEV1 FVC
Slide 13: Safety Summary: ERT-Treated Subjects
Safety Summary: ERT-Treated Subjects ERT-Treated ERT-Treated (Safety Population) (Safety Population) N=314* N=314* Subjects with ≥1 Reported AE 148 (47.1%) Subjects with ≥1 Reported SAE 110 (35.0%) Subjects with ≥1 Reported 39 (12.4%) Subjects with ≥1 Reported 5 (1.6%) Drug-Related AE** Drug-Related
Slide 14: Safety Summary: ERT-Treated Subjects
Safety Summary: ERT-Treated Subjects • No new safety concerns and no unexpected drug-related AEs were reported over 6 years of follow-up in MARS to date • Temporary treatment interruptions due to an AE were reported for 36 subjects – ERT treatment was subsequently resumed successfully in these subje
Slide 15: Conclusions
Conclusions • MARS is the longest real-world study of elosulfase alfa, with a heterogenous population representative of the MPS IVA population overall • Data collected over the first 6 years of MARS provide real-world evidence for sustained reduction in uKS levels and long-term stabilization of endu
Slide 16: Children and families with MPS IVA; study sponsor and authors
Children and families with MPS IVA Authors: John Mitchell, Barbara K Burton; MARS (NCT02294877) Michael B Bober, Philippe M Campeau; Shelda Cohen; Sara Dosenovic; Carolyn Study sponsor: BioMarin Pharmaceutical, Inc. Ellaway; Kaustuv Bhattacharya; Nathalie Guffon; David Hinds; Alice Lail; Shuan-Pei L
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