Raxone (idebenone) and pulmonary care in Duchenne Muscular Dystrophy (DMD)
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1 Raxone (idebenone) and pulmonary care in Duchenne Muscular Dystrophy (DMD) Thomas Meier, PhD February 2018
2 Agenda Medical need for effective treatment of respiratory illness in DMD Understanding respiratory function decline and clinical complications The DELOS study: Efficacy data for idebenone (Raxone ) in slowing respiratory function decline in DMD patients not using glucocorticoids Open for enrolment: the SIDEROS study in patients on glucocorticoid treatment Raising awareness for respiratory function in DMD 2 PPMD Onlus February 2018
3 Medical need for effective treatment of respiratory illness in DMD
4 Diagnosis Medical need for effective treatment of respiratory illness in DMD 5 years 9 years 14 years 20 years Glucocorticoids Loss Ambulation Loss of Respiratory Function Need for Assisted Ventilation Death 4 PPMD Onlus February 2018
5 Increasing muscle weakness leads to a progressive cycle of respiratory function decline in DMD 3 Increased risk of serious infections, including pneumonia Decline in respiratory function 1 Endpoints: Endpoints: Bronchopulmonary AEs Systemic antibiotic treatment Rate of hospitalizations for BAEs PEF, FVC, FEV1 Decreased ability to cough effectively and clear airways Endpoints: PEF, PCF 2 PEF: Peak expiratory flow FVC: Forced vital capacity EV1: Forced expiratory volume in the first secondf PCF: Peak cough flow BAE: Bronchopulmonary adverse events 5 PPMD Onlus February 2018
6 Percentage of deaths Respiratory and cardiac failure remain the leading causes of death in patients with DMD 100 Causes of death in CINRG-Duchenne Natural History Study % 32% 13% Respiratory failure Cardiac failure Other From the total pool of 340 eligible patients, 40 died over the 8 year study period. CINRG-DNHS: Cooperative International Neuromuscular Research Group Duchenne Natural History Study. 6 PPMD Onlus February 2018
7 Understanding respiratory function decline and clinical complications
8 Respiratory muscle dynamics in patients with DMD Collaboration with Prof. Andrea Aliverti DEIB Politecnico di Milano, IT 8 PPMD Onlus February 2018
9 Respiratory function decline in patients with DMD: Spirometry measures PEF: peak expiratory flow FVC: forced vital capacity 9 PPMD Onlus February 2018
10 Respiratory function test Both PEF%p and FVC%p follow a linear and parallel decline in patients over time Change in PEF%p and FVC%p with age 1) FVC%p PEF%p n = Age (years) 1) Data from CINRG-DNHS showing mean ± SEM absolute percentage point decline from baseline. Mayer OH, et al. US Neurology 2017;13: PPMD Onlus February 2018
11 The DELOS study: Efficacy data for idebenone (Raxone ) in slowing respiratory function decline in DMD
12 DELOS: Efficacy and safety of idebenone to delay respiratory function decline in patients not using glucocorticoids (GCs) DMD patients not taking GCs n = 64 1) R 1:1 Assessment schedule Idebenone 300 mg orally 3 times daily (n = 31) 1) Placebo orally 3 times daily (n = 33) # BL Week 13 Week 26 Week 39 Week 52 Study type: Randomized, placebo-controlled trial Key eligibility criteria: years, off chronic glucocorticoids (GCs) and in respiratory function decline (PEF%p 80%) Primary endpoint: Change in PEF%p from baseline to week 52 Other endpoints: Additional analyses: Changes in PEF (L/min and %p), FVC (L and %p) and FEV1 (L and %p) and PCF (L/min) Time to crossing clinically relevant FVC thresholds, frequency / duration of BAEs and associated hospitalization. Respiratory function (PEF and FEV1) were also assessed independently using the home-based device 1) ITT population. %p: percent predicted; BL: baseline; BAEs: bronchopulmonary adverse events; DMD: Duchenne muscular dystrophy; FEV1: forced expiratory volume in 1 second; FVC: forced vital capacity; GC: glucocorticoid; ITT: intention to treat; L: liter; L/min: liter per minute; PCF: peak cough flow; PEF: peak expiratory flow; R: randomized. Buyse GM, et al. Lancet 2015;385: PPMD Onlus February 2018
13 DELOS patients were non-ambulatory with limited upper limb mobility and established respiratory function decline Ambulatory status 92% non-ambulatory Brooke Score ~60% 5; unable to raise hand to mouth Mean age 14 years 13 PPMD Onlus February 2018.
14 Idebenone slows the decline in respiratory function Data from weekly home-based assessment of PEF%p Raxone (idebenone) Placebo 14 PPMD Onlus February 2018
15 Patients in the idebenone group experience fewer respiratory complications and hospitalizations Bronchopulmonary adverse events (AEs) Raxone Placebo Bronchopulmonary Adverse Events Subjects (%) Events Total Days 6 (19.4%) (51.5%) Systemic Antibiotic Use Subjects (%) Events Total Days 7 (22.6%) (39.4%) Hospitalizations (due to respiratory causes) Subjects (%) Events Total Days 1 (3%) (12%) PPMD Onlus February 2018
16 The SIDEROS study: Efficacy of idebenone (Raxone ) in patients on glucocorticoid treatment
17 17 PPMD Onlus February 2018
18 SIDEROS: Efficacy of idebenone to delay respiratory function decline in patients using glucocorticoids (GCs) DMD patients taking GCs n 266 R 1:1 Assessment schedule (Week) Idebenone 300 mg orally 3 times daily Placebo orally 3 times daily BL Study type: Randomized, placebo-controlled trial Key eligibility criteria: 10 years, chronic use of systemic GCs and in respiratory function decline (FVC%p 35%-80% at baseline) Primary endpoint: Change in FVC%p from baseline to week 78 Other endpoints: Changes in PEF%p, time to loss of 10% in FVC and change in inspiratory flow reserve Additional analyses: Change in peak cough flow (PCF), blood oxygen saturation and EtCO 2, Bronchopulmonary illness, Antibiotic use EtCO 2 = End tidal CO2 level, using Capnography 18 PPMD Onlus February 2018
19 SIDEROS trial Currently the largest ongoing clinical trial in DMD Ambulant and non-ambulant patients eligible for enrolment ~60 clinical trial sites in Europe, US and Israel Full reimbursement of travel cost All participants who complete SIDEROS are offered to participate in open-label SIDEROS Extension trial 19 PPMD Onlus February 2018
20 Disease Awareness Campaigns: Raising awareness for respiratory function in DMD
21 Respiratory function awareness in the DMD Patient Community Series of five white board videos Focus groups of patients, caregivers, and physicians Identified topics of importance Available as a community resource (advocacy groups, physicians & companies) Educational webinars for families & physicians planned for late March / early April PPMD Onlus February 2018
22 Santhera s respiratory function awareness campaigns Dedicated website providing information on respiratory function care US website: European website: 22 PPMD Onlus February 2018
23 Santhera s commitment to provide help for patients with DMD Collaborate with international experts to better understand respiratory function decline in patients with DMD; helpful to plan clinical trials Successful phase III DELOS trial demonstrated that idebenone slows decline in respiratory function in patients unable to take glucocorticoids (GCS) Ongoing Phase III SIDEROS trial in ambulant and non-ambulant patients who are using glucocorticoids study open for enrolment! Active contribution to raise awareness of the respiratory diseases component of DMD Santhera aims to make idebenone (Raxone ) available to patients with DMD as soon as possible 23 PPMD Onlus February 2018
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