Simbec-Orion Rare & Orphan Brochure

RARE & ORPHAN EXPERT SUPPORT

FOR CLINICAL DEVELOPMENT WORLDWIDE

MISSION STATEMENT We are Simbec-Orion. An international, full service, boutique CRO; growing by bringing together the best possible people, healthcare professionals and drug developers, from all areas of clinical development.

We focus on a defined series of core therapeutic areas, where we can make best use of our skills elegantly to design, execute and deliver our clients’ clinical development needs.

We are making Simbec-Orion a highly respected and profitable boutique CRO. We do this by working for our clients with excellence, commitment and passion for our trade. We provide an environment in which our colleagues can continue to grow and develop.

We will always remember that our work leads to the improvement of patients’ lives.

OUR THERAPEUTIC EXPERIENCE

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RECENT RARE & ORPHAN EXPERIENCE

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EXPERTISE IN RARE & ORPHAN CLINICAL RESEARCH 23 YEARS PHASE Ib-IV EXPERIENCE

PHASE I – IV

UNIQUE NETWORK OF RARE & ORPHAN INVESTIGATIVE SITES

GLOBAL RARE & ORPHAN ADVISORY GROUP

WORLD LEADING RARE & ORPHAN EXPERTISE IN-HOUSE 6

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SIMBEC-ORION IS ONE OF THE MOST EXPERIENCED RARE & ORPHAN CRO’S IN THE WORLD

FULL CONTINUUM OF PHASE I – IV SERVICES including in-house central labs and IMP management WORLD–CLASS RARE & ORPHAN SITE NETWORK Demonstrable recruitment and study success with reputable sites globally

ACCESS TO LEADING RARE & ORPHAN KOLS Privileged relationships with recognized rare & orphan opinion leaders

INTERNAL MEDICAL AND REGULATORY EXPERTISE With track record of product apporvals in Europe and the US

SIMBEC-ORION CLINICAL DEVELOPMENT THE RARE & ORPHAN CHALLENGE FOR DRUG DEVELOPERS

7,000 RARE DISEASES

With over 7000 rare diseases in the world, only 5% of which have FDA-approved medicines in the US, there is a enormous opportunity for drug developers to significantly change the lives of patients suffering from these conditions. And even though 82% of orphan drugs are approved by the FDA versus 35% of traditional medicines, the nature of rare diseases creates increased complexities for clinical trial design and patient recruitment.

The key to dealing with these challenges is expertise specific to rare & orphan clinical trials. Since 50 % of rare diseases affect children, paediatric expertise and considerations are also of paramount importance.

ONLY 5% TREATED

82% APPROVAL RATE

Simbec-Orion is a leading CRO in Rare & Orphan development.

SIMBEC-ORION CLINICAL DEVELOPMENT – A FULL SERVICE BOUTIQUE CRO WITH UNIQUE RARE & ORPHAN EXPERTISE

50% PAEDIATRIC

Simbec-Orion Clinical Development is the clinical development division of Simbec-Orion, an international full-service boutique CRO covering first in human Phase I clinical studies through to pivotal Phase III studies and post-marketing studies.

Operations in the United Kingdom, France, USA, Germany and Italy. Operational staff across mainland Europe (Western, Eastern & Central) and throughout North America.

One of Simbec-Orion’s significant areas of expertise is the conduct of studies in Rare and Orphan indications. Over the last 23 years we have developed relationships with networks of specialist investigative sites and we have established and nurtured links with patient groups across Europe and North America.

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OUR SYSTEMS AND PROCESSES HAVE BEEN DESIGNED TO ENSURE RAPID AND EFFICIENT START-UP AND CONDUCT OF YOUR RARE & ORPHAN STUDY

RARE & ORPHAN EXPERTISE & GLOBAL NETWORK

TO SUPPORT ALL YOUR CLINICAL DEVELOPMENT NEEDS Rare & Orphan clinical trials are complex and challenging. It is critical that the set-up, site selection and recruitment/retention strategies are developed and implemented based on experience and understanding of the issues involved at a granular level.

After 23 years of developing Rare & Orphan studies we are considered as experts in ensuring efficient delivery within time-lines.

RAPID AND EFFICIENT STUDY START-UP Our network and expertise helps you identify, recruit and retain patients from the very small populations; • Integrated processes regarding site relationships, with access to patient registries • Broad understanding of patient advocacy issues • Rigorous feasibility analysis of potential sites, e.g. site competency in the targeted indication and logistical considerations • Access to noted physicians and paediatricians 8

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Access to intenal and external Rare & Orphan experts

Right strategic direction; faster regulatory approval

Patient recruitment and retention expertise

Rapid start-up; keeping timelines

Rigorous feasibility and study planning

Motivated sites/investigators - ensuring study success

Engagement of KOLs and patient advocacy groups

Innovative and executable development plans

Oracle e-clinical systems

Real time access to reliable data

In-house central labs

Control of logistical challenges

FAST & EFFICIENT PHASE I TO III STUDIES UTILISING IN-HOUSE FULL-SERVICE CAPABILITIES Simbec-Orion’s internal experts in clinical research and development for paediatric and rare diseases therapeutic options, is combined with extensive and successful regulatory interaction and outstanding achievements of product approvals by the EMA and FDA.

SIMBEC-ORION CAN HELP YOU ACHIEVE FASTER APPROVALS THROUGH OUR STRATEGIC DEVELOPMENT AND REGULATORY PLANNING FOR ORPHAN INDICATIONS

SIMBEC-ORION PROVIDES: • Regulatory advice to meet conditions for future marketing, necessitating input from authorities at the onset • Strategic Development and Regulatory Planning for orphan indications – to help you achieve approvals as quickly as possible • Innovative, efficient study designs • Full submission package addressing key focus area per region • Review and gap analysis of customer prepared packages • Full regulatory agency interaction

PROJECT AND SITE MANAGEMENT Our experienced project managers specialise in Rare & Orphan studies. Despite challenging logistics and complex issues, they will ensure that your study is on track by; • Motivating investigators via close site management and motivational visits • Developing country and site-specific monitoring strategy • Working with investigators and patients to manage patients’ adherence to the study protocol • Coordination and prioritisation of all study aspects • Provision of one-stop in-house central labratories

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SIMBEC-ORION LABORATORY SERVICES

ON CAMPUS CENTRAL PATHOLOGY & BIOANALYTICAL LABORATORIES

SIMBEC-ORION LABORATORY SERVICES FULL RANGE OF LC-MS & IMMUNOASSAY EQUIPMENT

Is an integrated element of Simbec-Orion based from the Simbec-Orion Clinical Pharmacology campus. Fast efficient reliable delivery for your complex studies

BIOTEK SYNERGY 96-WELL PLATE READER

THE BIOANALYTICAL LABORATORY DELIVERS • Developing and validating complex assays • Highly selective and sensitive methods • Supporting all types of clinical study

RADIOISOTOPES LABORATORY Our Radioisotopes Laboratory performs radioactivity measurements for human ADME and mass balance studies.

Radio-HPLC and LC-MS/MS techniques are then used to investigate metabolite profiles and to assist the identification of putative metabolites.

API 5500 API 5000’S API 4000 API 365

“37% OF OUR LABORATORY

SERVICES EXPERIENCE HAS BEEN WITH NEW CHEMICAL ENTITIES...

...INCLUDING TRANSFER OF TECHNOLOGY FROM PRECLINICAL TO HUMAN MATRICIES”

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SIMBEC-ORION LABORATORY SERVICES

ON CAMPUS CENTRAL PATHOLOGY & BIOANALYTICAL LABORATORIES

PATHOLOGY EXPERTISE THERAPEUTIC AREA EXPERIENCE

Dedicated pathology support for clinical trials. Designed exclusively for the biopharmaceutical industry with over 30 years of consistent, high quality delivery.

REPRODUCTIVE MALE/FEMALE INFECTIOUS DISEASES 7 day per week analyses

ENDOCRINOLOGY

TECHNOLOGY

RHEUMATOLOGY Clinical Chemistry

Urinalysis DERMATOLOGY

Biochemistry, Immunology, Serology, Routine multi-parameter testing with Drugs of Abuse Screening microscopic analysis

Haematology

Full Blood Count with Differential Cytokine Assays Routine and Specialised Haemostasis Markers e.g. Thrombin Generation Assay

Logistics management

IMMUNOLOGY

Multiplex Assays

Flow Cytometry

Laboratory Computer System

Flow Cytometry - Lymphcyte Subset analysis etc.

Clinical Trial LIMS

Microbiology

Freezer Storage

Bacteria Identification and MIC Susceptibility

Rees Scientific System Monitoring Storage Temperatures i.e. 4C, -20C, -80C and Room Temperature

RESPIRATORY METABOLISM CARDIOLOGY ONCOLOGY UROLOGY

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www.SimbecOrion.com i n fo r m at i o n @ S i m b e c O r i o n .c o m SimbecOrionCRO

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Simbec House Merthyr Tydfil Industrial Park Merthyr Tydfil CF48 4DR United Kingdom

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@SimbecOrion

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