Kyowa Kirin Co., Ltd. · TSE:4151
Kyowa Kirin — Corporate Update (October 2017)
19 slides from Kyowa Kirin's corporate update, Oct 27, 2017
Slide 1: Clinical/Regulatory Updates of Burosumab and The Current Pre-launch Activities in EU
Clinical/Regulatory Updates of Burosumab and The Current Pre-launch Activities in EU Tomohiro Sudo, EVP Corporate Strategy and Planning Department, Kyowa Kirin, International, plc October 27th, 2017 1 © Kyowa Hakko Kirin Co., Ltd.
Slide 2: Topics to be Updated
Topics to be Updated Clinical Status Update of Burosumab Regulatory Update toward Approval in US and EU Pre-launch Activities in EU October 27th, 2017 2 © Kyowa Hakko Kirin Co., Ltd.
Slide 3: Clinical Status Update
Clinical Status Update October 27th, 2017 3 © Kyowa Hakko Kirin Co., Ltd.
Slide 4: Clinical Status Update of Burosumab
Clinical Status Update of Burosumab What we have achieved in our clinical program Pharmacological effect • Reducing phosphate wasting • Increase in serum P levels Clinical benefits • Increase in 1,25D levels Hurdles in clinical development • Orphan disease – limited number of patients • Limited data
Slide 5: Comprehensive Assessment of Clinical Effects
Comprehensive Assessment of Clinical Effects • Phosphate Phosphate • TmP/GFR metabolism • 1, 25 VD • Bone Parameters; ALP, P1NP, CTx Bone Muscle metabolism function • Pain • Radiographic data; Rickets QOL • Physical function RSS, RGI-C • Growth data improvement improvement • Stiffness • Walking 6MWT
Slide 6: Clinical studies for XLH
Clinical studies for XLH Study Age Study Title status Pediatric Studies as of October 16th UX023-CL201 5-12 Open-label, dose finding, Ph2, n=52 ongoing UX023-CL205 1-4 Open-label, single arm, Ph2, n=13 ongoing UX023-CL002 5-14 Retrospective radiographs from patients on conventional therapy, n=52 com
Slide 7: Clinical Status Updates Summary
Clinical Status Updates Summary We believe we have conducted a comprehensive clinical program to understand the disease and potential benefits and risks of Burosumab We believe that Burosumab addresses the major clinical aspects and important unmet medical needs in XLH We believe that the tota
Slide 8: Regulatory Update
Regulatory Update October 27th, 2017 8 © Kyowa Hakko Kirin Co., Ltd.
Slide 9: EU Regulatory Update (1)
EU Regulatory Update (1) Process overview Around Within 67 end of Days from 2016 Nov 2017 the opinion MAA submission Plan CHMP EU Launch in Review Review process opinion Decision Germany 2016 Dec Validation of MAA We are here. We are currently pursuing a conditional approval of pediatric indicatio
Slide 10: EU Regulatory Update (2)
EU Regulatory Update (2) Around Within 67 end of Days from 2016 Nov 2017 the opinion MAA submission Plan CHMP EU Launch in Review Review process opinion Decision Germany 2016 Dec Validation of MAA We are here. Key Milestones in near future CHMP opinion on peds. indication expected around the end
Slide 11: US Regulatory Update (1)
US Regulatory Update (1) Process overview 2017 Aug. BLA submission PDUFA on Apr. 17, 2018 Plan Review Review process We are here. 2017 Oct. Priority review designation Breakthrough Therapy Designation for pediatric XLH received June 2016 BLA was submitted in August to pursue pediatric and adult
Slide 12: US Regulatory Update (2)
US Regulatory Update (2) Burosumab for the treatment of XLH designated as a drug for a “rare pediatric disease” and eligible for a Priority Review Voucher BLA filed for adults and peds. in August 2017 and includes: 64 week Phase 2 data in 5-12 yr olds and 24 week Phase 2 data in 1-4 yr olds
Slide 13: Pre-launch Activities in EU
Pre-launch Activities in EU October 27th, 2017 13 © Kyowa Hakko Kirin Co., Ltd.
Slide 14: Major Pre-launch Activities in EU
Major Pre-launch Activities in EU Commercial organization: Rare Disease Business Unit in KKI Disease awareness Patient and physician identification Branding and Marketing message preparation (not to be used pre-approval) Market Access (pricing) Supporting an XHL patient community in EU
Slide 15: Disease Awareness Activities
Disease Awareness Activities Disease awareness Physicians Patients Conference/ Visits by MSL Publication XLH link symposium XLH disease Patient status slide organization October 27th, 2017 15 © Kyowa Hakko Kirin Co., Ltd.
Slide 16: Disease Awareness Activities
Disease Awareness Activities 2017) October 27th, 2017 16 © Kyowa Hakko Kirin Co., Ltd.
Slide 17: Patient Identification
Patient Identification MSLs Clinical development program ISTs – database analyses and publication Conferences and KOL discussions Online burden of illness and patient surveys October 27th, 2017 17 © Kyowa Hakko Kirin Co., Ltd.
Slide 18: Registry and EAP
Registry and EAP Registry program • Establish historical database to establish the burden of disease: retrospective analysis of patients with XLH and their clinical history • After approval, registry will include patients treated & untreated with Burosumab - This will allow comparison with concurr
Slide 19: KYOWA KIRIN
KYOWA KIRIN