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


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


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


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


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


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


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Additional Activities

• Delamanid – Bedaquiline DDI Study

• Public Health Activities • Moldova Pilot Project • Media Advocacy

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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


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


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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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