giovanni stropoli, convegno mitocon 2015
TRANSCRIPT
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Santhera Pharmaceuticals
• Founded as a Biotech Merger in 2004 in Liestal Basel Biozentrum Spin-off Myocontract and Heidelberg based Graffinity
• Currently about 25 people, planning idebenone introduction in EU
• Clinical Development in Orphan Diseases(mitochondrial/neuromuscular)
• Direct Distribution of Niche Pharmaceutical Products
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Pipeline with idebenone (Raxone®) in three indications with high unmet medical need
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Primary progressive MS (ppMS):
Phase 2 study in collaboration with NIH
Duchenne Muscular Dystrophy (DMD):
Positive Phase 3 study outcome,
NDA/MAA filing in preparation
Leber’s Hereditary Optic Neuropathy (LHON):
MAA under review in EU
Raxone®: trademark in EUCatena®: alternative trademark
Inherited form of blindness:
Prevalence ~ 2 in 100’000
~ 2’500 patients diagnosed in past 5 years in EU
Genetic disease with clear diagnosis and family pattern
Predominantly young males in all ethnic groups affected
Rapid loss of central vision by functional loss and
degeneration of retinal nerve cells
No treatment available
LHON – an inherited form of blindness
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1 2 3 4 50
RECOVERY
Off - chart
STABILISATION
ONSETYEARS
VA
NATURAL HISTORY
The therapeutic objective:
– stabilize the disease
– promote clinically relevant recovery
Potential window of opportunity for treatment up to ~ 5 years
Therapeutic objectives
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50% of LHON patients treated with Raxone®
experience clinically relevant recovery of vision
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Data from Expanded Access Program
The animation simulates
the average treatment
response in EAP
Raxone® efficacy typically seen within12 months from treatment start
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Data from Expanded Access Program
Majority of patients who show improved vision do so within first 12 months of treatment
Raxone® in Leber’s Hereditary Optic Neuropathy (LHON): pathway to its availability
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Leber’s Hereditary Optic Neuropathy (LHON):
Marketing Authorization Application (MAA)
under review in EU
Temporary Authorization in France,
CHMP Decision for Europe awaited
Authorization for Temporary Use (ATUc) granted in 2014
Use of Raxone® in hospital-treated patients with LHON
Supply of Raxone® fully reimbursed by the government program
Marketing Authorization Application (MAA) for Europe filed in May 2014 (validated in June 2014)
Decision by EMA’s CHMP expected in 1H 2015
Market exclusivity due to Orphan Drug protection
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Raxone® slows the loss of respiratory function in DMD patients not using steroids
First successful Phase 3 study in DMD
Efficacy demonstrated in primary endpoint (Peak Expiratory Flow)
Supportive evidence of efficacy from
other respiratory function endpoints
clinical observations directlyrelated to morbidity and mortality
Treatment was safe and well tolerated
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European introduction plans
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BRITAINIRELAND
PORTUGAL SPAIN
FRANCE
GERMANYGERMANY
AUSTRIAAUSTRIA
HUNGARY
GREECE
ITALY
SWEDEN
FINLAND
SLOVENIA
SLOVAKIA
ROMANIA
BULGARIA
POLAND
CROATIA
CZ
NORWAY
CH
DENMARK
NETH.
BELG.
LUX.
ESTONIA
LATVIA
LITHUANIA
Santhera presence build-up in 4 regional clusters
Potential national /regional distribution agreements for other EU countries
Currently staffing for Europe (and RoW) product introduction