Global TB Program Updates - CPTR) · PDF fileGlobal TB Program Updates CPTR Workshop...
Transcript of Global TB Program Updates - CPTR) · PDF fileGlobal TB Program Updates CPTR Workshop...
T B i n n o v a t i o n f o r t o m o r r o w .
Global TB Program Updates CPTR Workshop
Washington D.C., October 2, 2013
INTERNAL - CONFIDENTIAL
Proprietary and Confidential
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Overview
• Progress in clinical development
• Status of phase 3 trials
• Launch of pediatric trials
• Update on regulatory progress
• Additional activities
• DDI studies
• Public health advocacy and education
Proprietary and Confidential
Phase III Study Update (Trial 213)
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• Total duration: 30 mos • Target population:
patients sputum culture+ MDR-TB at enrollment
• Stratification: High risk: HIV+ (S Africa) &/or bilateral cavities Low risk: unilateral or no cavities
• Co-primary endpoints SCC @ 2 months Time to SCC over 6 months
• Study sites: Estonia, Latvia, Lithuania, Peru, Philippines, South Africa, Moldova
• >80% enrolled • 1st patient randomized:
Sept 2011 • Last patient for
randomization: Nov 2013
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Pediatric Investigation Plan (PIP)
1. Development of pediatric formulation
2. 10-week repeat-dose study in juvenile rats of toxicity & toxicokinetics
3. Bioequivalence study: compare delamanid pediatric formulation with tablet formulation in healthy adults
4. Open-label PK trial of delamanid in children (birth to < 18 years) with MDR-TB
5. Open-label extension trial of PK study: evaluate long term safety & tolerability of delamanid in children with MDR-TB
Proprietary and Confidential
Pediatric Studies: Objectives
• Trial 232: Phase 1 PK Age De-escalation study
• Define dose of delamanid in children resulting in AUC
comparable to that observed in MDR-TB trials for adult patients
• Trial 233: Phase 2 Safety Study
• Investigate the safety, tolerability, and PK of delamanid administered for six months in a pediatric population receiving concomitant OBR
Proprietary and Confidential
Study Population
• Current Tablet Formulation • Group 1: Adolescents 12 to 17 years
(100 mg BID, n=6)
• Group 2: Children 6 to 11 years (50 mg BID; n=6)
• Pediatric formulation
• Group 3: Children 3 to 5 years (25 mg BID; n=6) and (50 mg BID; n=6)
• Group 4: Newborns and infants 0 to 2 years (5 mg BID; n=6) and (25 mg BID; n =6)
Overall sample size = 36 children
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232 / 233 Enrollment Status
• Group 1: Currently enrolling patients in Philippines
• Group 2: To start enrollment Jan 2014 in Philippines & South Africa
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232 In Screening 1 Enrolled 5 Completed 5
233 In Screening 3 Enrolled 2
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Regulatory Update
European Medicines Agency (EMA) • Otsuka remains confident in the strength of its filing and has
requested re-examination of the CHMP decision
• New decision expected toward the end of the year
Japan Pharmaceutical & Medical Devices Agency (PMDA) • Otsuka filed with the PMDA in March, decision expected early
next year
U.S. Food & Drug Administration (FDA) • Otsuka is in regular communication with regulators for
preparation of a filing
Proprietary and Confidential
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Additional Activities
• Delamanid – Bedaquiline DDI Study
• Public Health Activities • Moldova Pilot Project • Media Advocacy
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Proprietary and Confidential
Otsuka’s Commitment to Public Health
• Otsuka’s pilot project in Moldova is aimed at exploring innovative models of patient management and support, leading to rational use of new tools and prevention of drug resistance
• Preliminary results show low acceptance of outpatient treatment, resistance to use of injectable in primary care, and need to decentralize decision-making processes
Proprietary and Confidential
Otsuka’s Commitment to TB Advocacy & Education
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• Continuing to promote TB education through “FighTBack” magazine
• Activities during World TB Day including ad (right)
• Committed to working with stakeholders, public/private partners and others
Proprietary and Confidential
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Conclusion
• Otsuka remains committed to its clinical development program
for delamanid and achieving regulatory approval as soon as
possible
• Initiation of clinical trials of pediatric MDR-TB for regulatory
submission
• Continuing public health and advocacy initiatives
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