IDSC Expertise Capabilities Services

©2013 IDSC, LLC10/25/2013 WWW.IDSCBIOTECHNETWORK.COM 1

IDSCFrom Hit to the Clinic…Your Drug

Discovery & Development Partner

Capabilities ● Expertise ● Services

Mark CreswellPresident and [email protected]: 734.433.9670Cell: 734.476.4097

©2013 IDSC, LLC

Mission

IDSC's mission is to add significant value to our client's organization by providing drug discovery, development, and outsourcing leadership and expertise to assist our clients in delivering innovative medicines to the clinic and on to the market faster, while minimizing attrition.

IDSC’s scientists have delivered many drugs to the clinic & to the market

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©2013 IDSC, LLC

Stages of Drug Discovery & Development

Hit to LeadLead

Optimization

Preclinical Development to IND Filing

Clinical Development

DevelopmentDiscovery

IDSC’s Services Span:


Identify Clinical

Candidate

Draft & Submit

IND

Draft & Submit

NDA

FDAPre-IND Meeting

FDAPhase 2 Meeting

Evaluate HTS Hits or SBDD

Results

Select lead compounds

©2013 IDSC, LLC

Why Clients Come to IDSC

Interim Leadership

• Filling the gaps

• Most major therapeutic areas

• All line disciplines

• Small molecules, biologics, stem cells, diagnostics, devices, nutraceuticals


Due Diligence

• Investment opportunities

• Program reviews

• SWOT analyses

•Venture capitalists

• Pharma companies

•Academics

Molecular Modeling

• Structure based design

• Pharmacophore modeling

•Homology models

•Docking, etc.

• Small molecules or biologicals

Outsourcing Management

•Medchem, API/ biological, pharmacology, toxicology, ADME, etc.

• Identify CROs & CMOs

•Manage CROs & CMOs

Consulting

•Most major therapeutic areas

•All line disciplines

• Small molecules, biologics, stem cells, diagnostics, devices, nutraceuticals

Fully Integrated Collaborations

• IDSC supplies a full project team to our client.

•All discovery, preclinical development, or clinical development line discipline experts.

©2013 IDSC, LLC

Interim Leadership

Filling gaps in our client’s scientific leadership and project teams

Leadership, program & project management

Med-chem, pharmacology, molecular modeling, chemical development, API management, CMC, formulation, ADME, toxicology, regulatory, clinical study design, clinical operations, clinical pharmacology, outsourcing management (chemistry, API, pharmacology, ADME, toxicology, some pieces of clinical)

Small molecules, biologicals, stem cells, devices, diagnostics, nutraceuticals

• CNS, CV, dermatology, infectious diseases, inflammation, immunology, metabolic diseases, oncology, pain


©2013 IDSC, LLC

Examples of What Our Leaders Do

Medicinal Chemists• Therapeutic area expert

• Evaluates SAR & decides what compounds to make

• Know ADME & design targets to improve ADME, potency, & efficacy

•Designs synthesis and manages outsourcing


API & CMC Managers•Manage CMOs

• Chemistry, biologicals, cell lines…

•Optimize chemistry process

• Troubleshoot biologicals

• Submit regulatory registrations

Clinicians• Support preclinical

development plan

•Draft clinical protocols

• Participate in and/or lead clinical advisory boards

•Oversee clinical trials

•Advise client, preclinical, and clinical project teams

TA Experts and Project Leaders

• Advise client on therapeutic area

• Prepare program reviews

• Lead teams in preparation of gap analyses and development plans

• Serve as project leader for project teams

Regulatory Experts• Guide client and IDSC team on

and assure regulatory guidelines are met

• Assist client with preparation for and accompany client to FDA meetings

• Assist client with preparation of and submission of regulatory documents

ADME, Toxicologists, Pharmacologists

• Design in vitro and in vivo studies

• Identify vendors and manage outsourcing

• Evaluate study data and write INDsubmission study reports

• Make decisions based on study results

©2013 IDSC, LLC

• Small molecules, biologics, nutraceuticals, devices, diagnostics

• Medchem, pharmacology, ADME, toxicology, regulatory, CMC, Process development, clinical, formulation, fill/finish, commercial assessment

• Most major therapeutic areas (slide 10)

• Due diligence - Evaluation of investment and in-licensing opportunities

• SWOT analyses: Strengths, Weaknesses, Opportunities, Threats

• Program reviews, gap analyses, & development plans– Discovery, preclinical development, and clinical development

• Writing & evaluating patents & IP space

• How: Ad hoc consulting, retainers, to partial FTEs


Strategic & Technical Consulting

©2013 IDSC, LLC

Fully Integrated Collaborations

• Drug discovery, pre-clinical development & clinical development collaborations

• For virtual biotechs; universities; foundations; small, medium, & large Pharmas expanding bandwidth…

• IDSC supplies most of or the entire project team (all of the line discipline experts; slides 11 & 12)

• Progress our client’s program from its current state to the next desired milestone: spinout, funding, partnering, licensing…

• IDSC provides the leadership, therapeutic area expertise, line discipline expertise, outsourcing expertise, outsourcing management

• Chemistry, biologics production, cell line production, assays, studies, API manufacturing, and depending on size, pieces of clinical trials are outsourced.


©2013 IDSC, LLC

Rhetorical Question…


Could an interdisciplinary group of 40 pharmaexperts with a combined >1,000 years of drug discovery, preclinical, & clinical development experience make a difference in YOUR drug discovery & development program?

©2013 IDSC, LLC

Therapeutic Area Focus

• Cardiovascular

• CNS

• Dermatology

• Immunology

• Infectious diseases

• Inflammation

• Metabolic diseases

• Oncology

• Pain

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Back to Slide 7

©2013 IDSC, LLC

Discovery Expertise

• Medicinal Chemistry

• Molecular Modeling

• Pharmacology

– in vitro

– in vivo

• ADME

Small

Molecules

&

Biologics


Back to Slide 8

©2013 IDSC, LLC

Development ExpertisePreclinical & Clinical

• ADME / Toxicology / Pathology

• PK/PD/TK Modeling

• Process Chemistry/ API Mgmt / CMC

• Regulatory Compliance

• Clinical Study Design

• FDA Meetings

• BLA/IND/NDA Prep & Submission

• Commercialization Strategy

• Product Assessment & Valuation

• Competitive Intelligence


Small

Molecules

●

Biologics

●

Stem Cells

●

Devices

●

Nutraceuticals

Back to Slide 8

©2013 IDSC, LLC

Broad Outsourcing Expertise:

• Med-Chem, Custom Synthesis• API Supply & Raw Materials– Small Molecules, Biologics

• Pharmacology (In Vitro/In Vivo)• ADME (In Vitro/In Vivo)• Toxicology/Safety• IDSC manages clinical trials internally

while outsourcing non core activities as well as large trials– PhI, II, III Clinical Trials– US & International

Vendor Management

& Project Management

Database of Vendors

…………..


©2013 IDSC, LLC

MEDICAL DEVICES

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©2013 IDSC, LLC

IDSC Medical Device Services

Regulatory Compliance•Determine the correct regulatory strategy

• Ensure actives and goals are aligned with the regulatory strategy

•Write regulatory documentation

•Make regulatory submissions

• Prepare client for meeting with regulatory agencies


Strategic Planning•Guide company strategy

•We help our client:

–Establish a business plan

–Set company goals, timelines, budgets, and milestones

–Generate an exit strategy

• Identify and oversee external vendors

Clinical Development• Establish and run clinical advisory board

• Identify and utilize KOLs

•Determine clinical pathway

•Write clinical protocols

• Ensure nonclinical studies & other development activities align with clinical plan & regulatory requirements

• Clinical operations

Market Assessment•Asset valuation

•Determine market size and potential market penetration

• Competitive intelligence

• Position client to manage negotiations with investors and partners by receiving the highest value for their asset

• Performing product valuations are not just in the domain of “big-pharma”

©2013 IDSC, LLC

DIAGNOSTICS


©2013 IDSC, LLC

IDSC Diagnostics Services

Regulatory Compliance

•Determine the correct regulatory strategy




• Prepare client for meeting with regulatory agencies.








Clinical Development• Establish and run clinical advisory board

• Identify and utilize KOL

•Determine clinical pathway

•Write clinical protocols

• Ensure nonclinical studies & other development activities align with clinical plan & regulatory requirements

• Clinical operations

Market Assessment•Asset valuation•Determine market size and potential market

penetration• Competitive intelligence• Position client to manage negotiations with

investors and partners by receiving the highest value for their asset• Performing product valuations are not just in

the domain of “big-pharma”

©2013 IDSC, LLC

NUTRACEUTICALS


©2013 IDSC, LLC

IDSC Nutraceutical Services

Regulatory Compliance

•Determine the correct regulatory strategy




• Prepare client for meeting with regulatory agencies.








Market Assessment•Asset valuation•Determine market size and potential market

penetration• Competitive intelligence• Position client to manage negotiations with

investors and partners by receiving the highest value for their asset• Performing product valuations are not just in

the domain of “big-pharma”.

©2013 IDSC, LLC

IDSC Nutraceutical Services


Training•Ensure Pharmaceutical companies moving into nutraceuticals gain the needed knowledge to be successful

•Ensure an appropriate understanding of the risks, benefits, and regulations around nutraceuticals

©2013 IDSC, LLC

STEM CELL THERAPIES


©2013 IDSC, LLC

• Somatic (adult), embryonic, pluri-/multi-potent cells

• Non-embryonic sources: Peripheral blood, marrow, adipose tissue, organ tissue, cord blood, amniotic fluid…

• General types: Hematopoietic, mesenchymal, endothelial, neural, cardiac, ….

• Autologous, allogeneic

• Differing regulatory environments worldwide

• At least 17 companies active in heart disease alone

~ Many combinations currently in R&D

and even more to come, with time. ~

Rapidly Evolving Science


©2013 IDSC, LLC

Stem Cells: Clinical Development

Regulatory Compliance•Optimize regulatory strategy

• Ensure activities and goals are aligned with regulatory strategy

• Prepare regulatory documentation

• Create and deliver regulatory submissions

• Prepare client for meeting with regulatory agencies


Strategic Planning•Guide development of company strategy

–Establish a business plan integrated across R&D, manufacturing and clinical lines

–Set and track company goals, timelines, budgets, and milestones



Clinical Development• Establish and manage clinical advisory board

• Identify and utilize KOLs

•Determine clinical pathway to registration

•Design clinical protocols

• Ensure nonclinical studies & other activities align with clinical plan & regulatory requirements

•Manage clinical program and trial execution

Market Assessment• Evaluate client asset

•Determine market size and potential market penetration

•Gather competitive intelligence

• Position client to manage negotiations with investors/ partners in order to receive highest value for their asset

• Performing product valuations are not just in the domain of “big-pharma”

©2013 IDSC, LLC

Summary


IDSC’s clients come to us for Discovery, Preclinical Development, Clinical Development, and Project Management expertise

Expertise

IDSC offers the academic, biotech,

pharma, and VC community a fully

integrated drug discovery and

development partner

Discovery Development

Interim Leadership ● Consulting ● Fully Integrated Collaborations ● Outsourcing Management

Discovery

CommercialAssessment

ClinicalDevelopment

ProjectLeadership

RegulatoryCompliance

PreclinicalDevelopment

CROsClient

Small Molecules ● Biologics ● Stem Cells

Therapeutic Area Expertise• CNS• Cardiovascular• Dermatology• Infectious Diseases• Inflammation• Immunology• Metabolic diseases• Oncology• Pain

Discovery Expertise• Medicinal Chemistry• Molecular Modeling• Pharmacology• Outsourcing Management• ADME

Development Expertise• ADME & PK/PD/TK Modeling• Toxicology & Pathology• API management & CMC• Formulation, Fill & Finish• Regulatory Compliance• FDA meetings, IND/NDA prep &

submission• Clinical Study Design• Commercial Assessment &

Valuation

Hit to LeadLead

Optimization

PreclinicalDevelopmentTo IND Filing

ClinicalDevelopment

Therapeutics . Stem Cells . Nutraceuticals . Devices . Diagnostics

IDSC

©2013 IDSC, LLC

CASE STUDIESIDSC


©2013 IDSC, LLC

• Client was downsizing– Came to IDSC to contract head of med-chem and manage chemistry CROs

• IDSC performed a program & budget review – Performed by IDSC’s experienced oncology med-chemist and pharmacologist

• Based on data and remaining budget - IDSC recommended the client stop med chem activity and focus in vivo efforts on select compounds already in the portfolio.

• The implementation of this recommendation saved the company– They now have a clinical candidate and have scaled material & have begun an IND enabling Tox

Study.

– Had they continued with med-chem, IDSC would have profited, and the client would have burned their budget without getting a candidate.

– IDSC continues in a multi-disciplinary consulting role with this client


To The Rescue (Receptor Antagonist For Oncology)

©2013 IDSC, LLC

From Target to Pre-Candidates

• Client had a biological target based platform• IDSC helped client establish its goals• IDSC trained client in drug discovery strategy & budget• IDSC serves in an overarching discovery role that includes heads of

medicinal chemistry and molecular modeling• Began with mass screen of commercial compounds and analogs of

literature compounds• Lead to multiple novel series & rapid SAR expansion and lead

optimization in best series• IDSC manages 5 CRO chemistry FTEs• IDSC manages in vivo biology, PK/PD, tox, formulation, etc…• Several potential clinical candidates have been identified• Timeline – 9 months


©2013 IDSC, LLC

Relationships Grow: Oncology Development (International Client)

• Web Inquiry led to a gap analysis

• Gap analysis led to ad hoc consulting

• Ad hoc consulting led to CAB (clinical advisory board)

• CAB: Clinical advisory board preparation, attendance, management, summary

• CAB led to a fully integrated 8-member IDSC development team engaged to develop the asset

• Client is virtual10/25/2013 WWW.IDSCBIOTECHNETWORK.COM 28

©2013 IDSC, LLC

Molecular Modeling

• In-House full service molecular modeling supports all appropriate med chem projects (We use the MOE integrated suite)

• IDSC currently serves as the molecular modeling department for several pharmaceutical and biotech companies

• IDSC modeling directors conducted computational fragment-based design studies that led directly to two clinical candidates and three additional chemical leads for a nuclear hormone receptor program


©2013 IDSC, LLC

Program Reviews

• University– Started with 6 programs for relevance of

biological target and quality of lead chemical matter

–Based on results this relationship continues to grow

– Six different IDSC Directors have written program reviews for client

– Trained client on IND process


©2013 IDSC, LLC

40 Consultants – One Roof

• IDSC received a phone call – Client needed a preclinical & phase I development plan & budget to be delivered within 35 days.

• A team of 7 IDSC directors (toxicology, clinical pharmacology, ADME, CMC, API, regulatory, project management) met the ambitious deadline.


©2013 IDSC, LLC

Client

IDSCMed Chemist &

PM, POC

ChemistryCRO

In Vivo Pharmacology

CRO

In Vitro Screening

CRO

IDSCDMPK Director

DMPK /ADMECRO

Keys To Success Team can make key decisions

Good communication

Good project management

Significant TA expertise

Goal oriented team

Manage streamlined logistics process

Management:

Information & DataSample logistics

Decision treeCROs

IDSC Pharmacologist

SampleSample

(Bulk to client)

PM – Project Manager

POC – Point of Contact

DISCOVERY


©2013 IDSC, LLC

PRECLINICALDEVELOPMENT Client

IDSCTA Expert &

PM, POC

In Vivo Pharmacology

CRO

API Scale up and Synthesis

CRO

Toxicology Studies

CRO

DMPK /ADMECRO

Keys To Success Team can make key

decisions Good communication Good project management

Significant TA expertise Goal oriented team Manage streamlined

logistics process

Management:

Information & Data Sample logistics

Decision treeCROs

IDSC Toxicology Director

Sample

(Bulk to client)

PM – Project ManagerPOC – Point of Contact

IDSCClinician

IDSCAPI & CMC

Director

IDSC Regulatory Director

IDSC DMPK/ADME

Director

IDSCClinical

Pharmacologist

FDA

IDSC In-vivo

pharmacology Director

IDSC In-vitro

pharmacology Director

In Vitro Pharmacology

CRO


©2013 IDSC, LLC

Client

IDSCPM, POC

Data mgmt, CRFs,

programming

Supporting Preclinical Studies

CRO

Keys To Success Team can make key

decisions Good communication Good project management Significant TA expertise Goal oriented team Manage streamlined

logistics processIDSC

preclinical Directors PM – Project Manager

POC – Point of Contact

IDSC Clinicians/MDs

IDSCAPI & CMC

Director

IDSCRegulatory Director

IDSCClinical

Pharmacologist

IDSCClinical-Ops

(CRAs/monitors)

FDA

Statistics, IVRS, Med Writing

CRO

CLINICALDEVELOPMENT

Study Sites

API GMP Manufacture

CRO

Clinical PK Analysis CRO


©2013 IDSC, LLC

U of Iowa

U of Michigan

WARF

NVF

Nektar

CRL

Arsenal Cap

U of Louisville

KFSYSCC

Partial Client List

Pfizer

Cubist

Profectus

Lycera

Deciphera

AKI

Altiris

ShaunaRx

Nymirum

Biolytx

Biota

Glenmark

Maxthera

Mazorx

MBI

NAEJA

ReGenX

ApiGenesis


©2013 IDSC, LLC

Leadership

• IDSC’s 40 discovery and development directors have a career averaging 25 years each with high level management expertise.

• Our directors have taken programs from discovery to the clinic and from phase I to market.

• IDSC’s leaders have proven discovery and development track records within big pharma and consultancy having developed their careers and skills at large pharma organizations like Abbott, J&J, Novartis, and Pfizer among others & having led or participated in teams responsible for the development or commercialization of products like Zoloft®, Lyrica®, Nexterone®, Cerebyx®, Linezolid®, and Plazomicin® just to name a few.


©2013 IDSC, LLC

Science & Consulting

• IDSC brings its clients an interdisciplinary leadership team with career expertise in all of the major therapeutic areas (We are especially strong in antibacterials, CNS, CV, oncology, and inflammation).

• The team remains at the cutting edge of their fields by:

– Progressing state of the art programs

– Reviewing current market reports and review articles

– Attending key therapeutic area conferences

– Reading the current literature

• As respected leaders in their field our directors provide insight to the industry by giving conference presentations.


©2013 IDSC, LLC

Outsourcing

• IDSC was founded by leaders who built a $35M global discovery outsourcing program within big Pharma

• We bring the highest value vendors to our fully integrated collaborations

• Fully integrated collaborations bring new insights into our client’s organization in addition to more shots on goal through a very flexible & cost effective business model


©2013 IDSC, LLC10/25/2013 WWW.IDSCBIOTECHNETWORK.COM 41

• Alex Bridges, PhD: 25 years big pharma, 6 years Academics, 5 yrs biotech; oncology, metabolic disease; medicinal chemistry.

• Brent Blumenstein, PhD: 30+ years academia, industry; FDA advisory committees; biostatistics; clinical trials; oncology, urology, epidemiology.

• Mukund Chorghade, PhD: 26 big pharma; regulatory filings; tech transfer; CMC, and API management.

• Lauren Costantini, PhD: 20+ yrs biotech & academia; multiple vice president roles; nutraceuticals, medical foods, devices; neurodegenerative diseases, research, clinical, regulatory, commercialization strategy.

• John Domagala, PhD: 30 years big pharma, global head, infectious diseases, med-chem, 30 clinical candidates, 8 compounds to the clinic.

• Joe Fix, PhD, MBA: 30+ years big pharma, biotech; formulation, fill & finish, product development; CRO management; drug delivery technologies.

• Shelly Glase, PhD: 17 years big pharma; CNS; med-chem; outsourcing management, project management; 5 clinical candidates.

• Richard Kavoussi, MD: 28 years of academia, pharma, consulting expertise. Board certified psychiatrist. Extensive experience in the design, execution, and analysis of clinical psychopharmacology trials.

• Joan Keiser, PhD: 30 years big pharma; cardiovascular, metabolic diseases; pharmacology; global therapeutic area head; portfolio management; idea to POC.

• Francie Kivel: 26 years big pharma & small biotech, clinical development & corporate strategy, program management, clinical operations, stem cell therapies, CV, CNS, oncology, women’s health, inflammation.

• Bill Kenney, PhD: 24 years big pharma; CNS, oncology, antiviral; medicinal chemistry.

• Brad Martin, PhD: 15 years big pharma, CV physiology and regenerative medicine, stem cell therapies, translational research.

• David Moreland, PhD: 24 years big pharma; molecular modeling; CNS, cardiovascular, infectious diseases; 3 clinical candidates.

• Anne Norment, MD, PhD: 20 years biotech; immunology, inflammation; pharmacology; translational research.

Brief BIOs (Partial List)

©2013 IDSC, LLC

• Ron Shebuski, PhD: 20+ year academia, & pharma, cardiovascular, pharmacology, therapeutics, devices, diagnostics.

• Bob Sigler, DVM, PhD: 21 years big pharma; toxicology, pathology; study design; IND support.

• Jeff Simpson, BS: 28 years big pharma, consulting; commercial strategy, product assessment, valuation, lifecycle planning, advisory boards.

• Richard Stead, MD: 24 years experience, clinical and regulatory strategy for development of biotechnology products, including oncology and hematology products; academic experience in hematology & coagulation.

• Ken Tack, MD: 26 years big pharma, Internal medicine, clinical trial design, 3 marketed drugs in infectious disease.

• Charlie Taylor, PhD: 27 years big pharma; CNS; pharmacology; neurological disorders, psychiatric disorders, pain; gabapentin, pregabalin.

• Jim Thomson, PhD: 30 years biotech; biologics; process development, formulation, analytical development, CMC, lyophilization, and stability.

• Howard Welgus, MD: 30 years academia & pharma, global head, dermatology, inflammation, clinician, CMO, medical affairs.

• Lloyd Whitfield, PhD: 30 years big pharma, population PK and PK/PD, dose selection, allometric scaling, and modeling and simulation support.

• Jim Zeller, PhD: 22 years big pharma, 6 years API consultant, chemical development of API processes, API cost analysis, CMO identification and management.

• Bob Zimmerman, PhD: 25 years big-pharma, biotech; oncology pharmacology; large and small species; recombinant proteins, monoclonal antibodies, vaccines, and engineered proteins.

• Mary Zimmerman, MS: 25 years biotech; SVP; regulatory affairs & strategy; IND, CTA, INPD, CTD submissions.


Brief BIOs (Partial List)

©2013 IDSC, LLC

THANK YOUMark Creswell

President & CEO

310 N. Main St.204

Chelsea, MI 48118 USA

Phone: 734.433.9670Fax: 734.433.9671Cell: 734.476.4097

[email protected]

From hit to the clinic:Your drug discovery & development partner


©2013 IDSC, LLC

Gene Family Expertise

• GPCRs

• Kinases

• Cytokines

• Ion channels


• Proteases

• Nuclear receptors

• Transporters

IDSC Expertise Capabilities Services

Health & Medicine

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