Boston Biotech Clinical Research

Leading the SCIO Revolution

SCIO

The new class of service

Strategic Clinical Innovation Organization

What Are the Traditional CROs Missing?

• Strategic planning and projection for decision analysis• Integration with current Regulatory guidance parameters• Customized protocols to maximize your research results• Execution to focus on the right patients, the right indications

What is the Problem ?

• Gaps appear in the development process that may cost success and money• Protocols written for top line visualization that may be lacking scientific rigor• Protocols and clinical studies may become myopic and miss greater options• Increasing Regulatory demand for transparent study results and cross AEs

Bridging the Chasm between Research and FPI

BBCR Service

Safety / Tox Data

Discovery Data

Biomarkers

Trial Design

Objectives/Endpoints

Patients Selection

Patients Safety

RegulationDisease Staging/

KOL

Protocol Synopsis

Patients’ Needs

Current Methods Create Gaps• Breaks in the outsourcing expertise between preclinical research

and CRO• Current Clinical development approach does not allow integration

of product specifications into a human treatment• Unfocused Strategic view leads to a non-cohesive clinical plan• Lack of Innovative approach for strategic study design delays

cost-efficient solutions based on updated regulations and rulings• Failure to identify the proper strategic therapeutic indication and

target patient population fulfilling the study objective/s• Misalignment between Sponsor and CRO on goals and growth

achievements, with conflict of interest on objectives

SCIO fills the gap The SCIO process is crafted to use strategic thinking and

innovation in clinical/medical expertise to improve the sponsor's goals and ability to meet those goals. SCIO success depends on meeting actionable endpoints and sponsor’s goals, not on completing a large number of studies or enrolling a lot of patients.

BBCR methods produce top line benefits that:• Link preclinical data with market opportunity• Move the product into first in man• Create cohesive phase l/ll studies that support POC• Match the study endpoint/s with study objectives• Develop a product specific protocol that connects product

potentials to treatment benefit

BBCR Follows the New Paradigm • No step by step development, requires the creation of a

complete clinical plan up-front

• Moves away from a silos approach towards a translational medicine process during exploratory stage

• Allows to test safety in patients vs. healthy volunteers

• Allows evaluation of efficacy early

• Potentially reduces study time and patient number

• Requires an early dialogue with the regulators

• Requires SCIO expertise for all the above

New Paradigm seeks to Mitigate Trial Implementation Challenges

Clinical Operation • Low interest in study participation• Slow IRB review and patient enrollment• Difficult study management and poor compliance• Laborious data cleanup• Unsatisfactory data results

Finance• Trial high cost• Unpredictable budget• Miss timeline’s milestones

Strategic Clinical Innovation Organization

• Develop Early Roadmap

• Strategic Clinical Plan

• Implement FDA innovation

• Customize trial design

• Optimize patient selection

• Integrate plans and strategies

• Maximize product-value

BBCR’s Approach Advantages

Create clinical roadmap for fundraising Streamline clinical development reducing cost Avoid unneeded clinical trial failure Maximize early stage product-value Optimize management and financial resources Visualize goals and milestones

Pharma Company Approach

•Outsource services •Keep low company’s headcounts•Create “ Medical Affair Strategist” role•Streamline clinical development stages•Implement innovative study designs

BBCR’s team experience

•Pharma and Biotech experience•Proven success in innovative strategies •Up-to-date with innovative regulatory requirements and trial design methodologies

Velaglucerase Alpha (Gene Activated Glucorerebrosidase)

for Gaucher diseases Type 1Case study of Strategic Clinical Innovation

Problem: Develop the product avoiding a “me too” treatment in an orphan population when a very similar product had a market monopoly for 15 years.

Solution: Created a clinical innovative strategy and trial designs that avoided the obvious head to head design, which would had required an unfeasible number of patients, and be at high risk to prove efficacy and safety in this population.

Worked with the Regulatory agency upfront to validate approach, clearly identify endpoints and patient population expectations verses current standards of care.

Velaglucerase Alpha (Gene Activated Glucorerebrosidase)

for Gaucher diseases Type 1Case study of Strategic Clinical Innovation

• The proposed clinical strategy was successful, the product approved

• Shire HGT included acknowledgement for “contribution to the design of the clinical trial” in 3 trial result publications

• Carla Epps, MD, MPH, FAAP, Medical Officer FDA/CDER/OND on May 2011 summarized the clinical strategy:

– Well planned

– Clinically meaningful endpoints

– Each trial had a distinct purpose

BBCR Unique Therapeutic Area focus

Rare Diseases/Orphan Indications Autoimmune Disease/ Inflammation Respiratory Infectious Disease Neurology Drug/Device/Diagnostic Combination Pediatrics and Elderly Device/Pharma Interface

BBCR’s People

• Board of Advisors– Advisory Board members are pharmaceutical and biotech senior management, drug development experts

• Clinical Innovation Team– Approximately 20 team members, flexible virtual organization

• Therapeutic Area Experts (KOLs)– Specialists from : Argentina, Brazil, Canada, Colombia, Check Republic, Israel, Italy, Netherland, Paraguay, Poland, Russia, Serbia, South Africa, Tunisia, UK, US

Your Objectives are Our goals

• We are aligned with the sponsor’s goals• We work seamlessly with our customer• We work with the regulatory agency to gain agreement

Today more than ever

Biotech Company Success

hinges on:

capital efficiency,

lean management,

low risk clinical trials, and

early stage product-value accrual

BBCR helps you to WIN

Thank you for your attention!!

How May We Help You!www.bostonclinicalresearch.com

617 401 2327Cambridge , Ma