A Speciality Pharmaceutical Group
GOSH, LONDON, 2 SEPTEMBER 2013 A strategy of growth and geographical expansion
• A specialty pharmaceutical group (€ 828.3 m sales in 2012 and over 3,300 employees of which more than 1,700 field force) • R&D in the cardiovascular and urology fields and in treatments for rare diseases Profile • Marketing operations in the main European markets, in Russia, Poland and other Central and Eastern European countries, in Turkey and in the U.S.A. • Drugs for the treatment of rare diseases marketed worldwide • Proprietary drugs sold worldwide through licensees
• Expand through organic development and through acquisitions • Develop product portfolio by enhancing product pipeline and new Strategy product acquisitions. Prioritize special care. • Increase presence in new markets characterized by high growth • Treatments for rare diseases: develop a global presence
2 Our products are sold in 135 countries
20 Subsidiaries in 20 countries 39 Branches and other forms of territorial presence 76 Countries where Recordati products are sold (under license or exported) A diversified product portfolio . Orphan Europe Orphan drugs 11.7% . Recordati Rare Other corporate products 6.1% Diseases
Livazo 2.4% OTC 17.7% Urorec 4.2% Zanipress 5.8%
Subsidiaries’ local product portfolios 34.8% Zanidip 13.0%
Entact®/Elopram® 5% (escitalopram/citalopram) - Italy
Pharmaceutical chemicals 3.6% Tergynan® - Russia & CIS, France exp. 3% Peptazol® (pantoprazole) - Italy 3%
Other revenue 0.7% Methadone - France 2%
Hexa line - France 2% Data: First quarter 2013 Tora-Dol® (ketorolac) - Italy 2% Total revenue € 244.6 m
4 Drugs for rare diseases
Brand name Indication
ADAGEN® (pegademase bovine) SCID-ADA deficiency CARBAGLU® (carglumic acid) Hyperammonaemia due to NAGS deficiency and to the main organic acidemias COSMEGEN® (dactinomycin) Mainly Wilms’ tumor, childhood rhabdomyosarcoma and choriocarcinoma CYSTADANE® (betaine anhydrous) Homocystinuria CYSTAGON® (cysteamine bitartrate) Nephropathic cystinosis NORMOSANG® (EU-RoW) / Acute attacks of hepatic porphyria PANHEMATIN® (US) (human haemin) PEDEA® (EU-RoW)/ Patent ductus arteriosus NEOPROFEN® (US) (ibuprofen I.V.) VEDROP® (water soluble vitamin E) Vitamin E deficiency in paediatric patients suffering from congenital chronic cholestasis WILZIN® (zinc acetate) Wilson’s disease
5 A well balanced R&D pipeline
Name Originator Indication Phase Approved in EU CARBAGLU® Recordati . Organic acidaemias Phase III in U.S.
ZANIPRESS* Recordati . Essential hypertension Filed in EU
Phase III in US (Nymox) REC 0482 Nymox (NX-1207) . Benign prostatic hyperplasia (BPH) and EU (Recordati) . Opiate dependence In registration in EU Methadone . Treatment of cancer-related pain Phase III-b in EU CYSTADROPS® Recordati . Ocular cystinosis Phase III in EU . Acute lymphoblastic leukaemia Phase II/III GRASPA® Erytech . (ALL) Acute myeloid leukaemia Phase II-b (AML) . Overactive bladder (OAB) and REC 1819 Recordati Phase I in EU Incontinence Recordati/UFPeptid . Overactive bladder (OAB) in REC 0438 Phase I in EU es patients with spinal lesions * New dosage 6 Drugs for rare diseases A worldwide business Huge market potential: > 6,000 rare diseases identified for which treatments exist for only around 300.
Progressive country introduction of rare disease plans and access to diagnostic tests will stimulate the market for orphan drugs. • Sales CAGR 15% since 2008 (acquisition of Orphan Europe) • Carbaglu® (carglumic acid) approved and launched in the U.S. in 2010 to treat NAGS deficiency • New indication (organic acidemias) for Carbaglu® approved in Europe and in development in the U.S. • Graspa®, a new treatment for rare cancers in development, licensed from Erytech in Europe • Present throughout Europe, Middle East and the U.S.. Sales coverage of new territories, either directly or through partnerships, planned. • Sales of drugs for rare diseases in 2012 total € 75,9 million, an increase of 9,5% • Recordati Rare Diseases Inc. operational in the U.S.. Acquisition of a portfolio of products for the treatment of rare diseases from Lundbeck in 2012 (transaction closed in 2013) • Sales to reach around € 140 million in 2015
7 Multistakeholder and complex collaboration to cure rare diseases
Authorities
Interest Industry Patients groups
Payers
8 Expertise
. Pharmaceutical and Preclinical Development . Development of blood-derived products (Viral & Prion validation expertise) . Pharmaceutical formulations development . Non clinical development . Regulatory and PV . 6 centralized EMA approvals . 1 Mutual recognition procedure accomplished . 2 FDA approvals . 10 Orphan Drug Designations (8 in EU and 2 in US) . 1 Paediatric investigational Plan (PIP) approved NPU programs introduced in several countries in Europe & Middle East for all our products . Expertise in RMP and Inspections
9 Investment into the development of registries for post marketing and longitudinal data
In house registries Public/private partnerships . Vedrop European registry . EuroWilson registry . ROCH (Cystadane registry) . EPNET registry . Normosang follow-up survey . European Cystinosis registry . Urea Cycle Disease Consortium Carbaglu protocole surveillance registry in the US . E-IMD Carbaglu protocol surveillance registry The added value of registries: . Characterise the global patient community . Promote improved diagnosis and clinical care
10 . Promote research opportunities Assembling & Distribution
. GMP facility in France . Innovative packaging . 250 batches prepared per year . Reception and storage in deep frozen, cold or room temperature conditions
. Distribution to over 60 countries . Distribution directly to hospitals . >15 000 shipments per year . Traceability and follow-up . Experience in product recalls . Emergency deliveries
11 Marketing and Sales
. Close collaboration with key stakeholders: . Patient organizations . KOLs . Research organizations . Authorities (national and international) . Payers . Patient orientation . Awareness and education . “Find the patients” programs . International networking for medical community . Highly qualified field force
12 Orphan Europe Academy
. Launched in 2000 to mark . Academic scientific OE’s 10th anniversary programme committee . Purpose to provide . Practical hands on tailored solutions to experience « what the text education and training for books don’t teach » healthcare professionals . Limited to small groups of in rare disease 25-35 . Image: . CME accreditation . Promotes OE key values
. Differentiation . Long-term commitment . Customer relationship
13 Adagen @ OE – an example how to help comprehensively SCID ADA patients
Emergency delivery
Trained Logistics sales force
Adagen
Regulatory/ KOLs/ payers guidlines interactions
Newborn diagnostics GRASPA - Erytech
. Graspa addresses unmet medical needs . ALL: Treatment for elderly, relapsed or allergic children and adults . AML: Treatment for fragile and unfit patients . Entry into Oncology/Haematolgy . 50% of Orphan Drugs on the market – 40% of Orphan Drugs in development . Risk mitigated approach . No previous Oncology Drug Development by OE . Oncology is therefore a new market for OE . Synergies with other products . Strengthen R&D activities in this environment 15 List of academic partnerships
• KOLs working at following • Topics Institutions are closely and routinely – Clinical trials working with us: – Publications – NIH, USA – Advisory – Radboud University Nijmegen Medical – Expert opinion Centre, Netherlands – Training – Necker Hospital, France – Registries – ULB Bruxelles, Belgium – Diagnostic – Royal Prince Albert Hospital, Australia – Treatment algorithm – Tubingen University, Germany – Liverpool NHS Hospital, UK – University Hospital Stockton on Tees, UK – CHU Bicêtre, France – Hannover Medical School, Germany – Copenhagen Hospital, Denmark – GOSH, UK – Many more
16 In summary
What we can offer What we are seeking . Track record in developing and . Medicines/technologies/ managing orphan medicines approaches that will make a . Fully integrated and experienced difference for the patient R&D infrastructure (Recordati . Partners that are interested, group) share our values and are . Strong geographical presence committed to work with us . Track record in managing the complex relations between stakeholder . Support and infrastructure in the development of medicines for rare diseases 17