U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health 

NIH Programs for NIH Programs for T l i l R hT l i l R hTranslational ResearchTranslational Research

Stephanie Fertig, MBA (NINDS)T dd H i PhD (NCI)Todd Haim, PhD (NCI)

Kurt Marek, PhD (NHLBI)Jerry Wujek, PhD (NEI)

John McKew, PhD (NCATS)John McKew, PhD (NCATS)

Phase IIB ProgramsPhase IIB ProgramsPhase IIB ProgramsPhase IIB Programs

• Competing Renewal Phase IIB or Bridges Award• Competing Renewal, Phase IIB or Bridges Award• General Omnibus: NIA, NIAAA, NIAID, NICHD, NIDA,

NIDCD NIDDK NEI NIGMS NIMH NCATS ORIPNIDCD, NIDDK, NEI, NIGMS, NIMH, NCATS, ORIPo Some are focused on projects requiring FDA approvalo Specific IC research topics sections (omnibus) have

i t /i t tirequirements/instructions

• Specific Announcements: NCI, NHLBI, NINDSo Application includes a fundraising plano Application includes a fundraising plano Strongly Encourage Matching Funds

Contact Program Before Applying

SBIR Technology Transfer (SBIRSBIR Technology Transfer (SBIR‐‐TT)TT)• Transfer technology out of the NIH intramural research

labs into the private sectorP j t t l th f NIH i t l t h l• Projects must rely on the use of NIH intramural technology

• Investigator Initiated (Grants): NINDS, NHLBI, NEIApplication assignment follows extramural receipt and referral guidelineso Application assignment follows extramural receipt and referral guidelines

o NINDS and NEI: Technologies from any NIH intramural labo NHLBI: Technologies from any NHLBI intramural lab

• Request for Proposals (Contracts): NCI, NHLBI• Licensing Agreements:

o Granted a royalty-free, non-exclusive internal research-use if awardedo Can apply for an exclusive license agreement

Contract ResourcesContract ResourcesContract ResourcesContract Resources• Offer access to services instead of grant money

Examples:Examples:o cGMP Manufacturingo Pharmacokinetic/ADME Studieso Pharmacology/Toxicology Studieso Pharmacology/Toxicology Studieso Regulatory Supporto Clinical Supply Manufacturingo and many others…o and many others…

• Submission:o May or may not go through grants.gov

Not a standard grant applicationo Not a standard grant application• Review process:

o Not a standard review processo Varies from program to program

Contact Program Before ApplyingContact Program Before Applying

Translational Programs at NIMHTranslational Programs at NIMHggDrug Discovery/Development Programs:• SBIR Program:g

o Complex Technologies and Therapeutics Development for Mental Health (PA-11-133) o Early Stage Clinical Trials (NOT-MH-015)

• Other Programs open to Small Business:o National Cooperative Drug Discovery/ Development Groups (NCDDG) for the

Treatment of Mental Disorders, Drug or Alcohol Addiction (PAR-11-206/PAR-11-207)o Drug Discovery for Nervous System Disorders (PAR-10-001/PAR-10-002)o Novel NeuroAIDS Therapeutics (PAR-10-216)p ( )o Innovative Pilot Studies of Novel Mechanism of Action Compounds for Treating

Psychiatric Disorders (PAR-11-316)

Resources:Resources:• NIMH Psychoactive Drug Screening

Program: http://pdsp.med.unc.edu/indexR.html• Toxicological Evaluation of Novel Ligandsg g

Program: http://www.sri.com/biosciences/nimh/• NIMH Chemical Synthesis and Drug Supply Program http://nimh-

repository.rti.org/• NIMH Genetics Repository: https://www nimhgenetics org/• NIMH Genetics Repository: https://www.nimhgenetics.org/

Neurotherapeutics Discovery Programsp y gAt NINDS

Phase I/IITrials

Target ID Assay Screening Hit to LeadProof

of Pre-clinicalSafety

LeadO ti i ti Trials

ConceptSafetyOptimization

NIH Blueprint Neurotherapeutics Network (BPN)

SBIR/STTR Clinical

Translational Cooperative

NIH Small Business Program: General SBIR & STTR

Clinical Trials

Cooperative Agreement

SBIR/STTR Competing Renewal (Phase IIB)(Phase IIB)

Phase I/IITrials

Hit to LeadProof

ofConcept

Pre-clinicalSafety

LeadOptimization

Blueprint Blueprint NeurotherapeuticsNeurotherapeutics Grand ChallengeGrand Challenge

ConceptyOptimization

pp pp gg• “Virtual Pharma” structure

o Industry-standard servicesLead Development Team

o Industry standard serviceso Industry-seasoned consultantso Funding for biology

Principal InvestigatorIndustry-seasoned consultants

NIH staff

• PI retains control of IP• Entry Requirements:U01s NIH Contracts

o Strong biological rationaleo Robust assay for SAR, biological

validationW ll lid t d hit d

Bioactivity/EfficacyStudies

MedicinalChemistry

DataManagement

GMP S l U o Well-validated hit compound

RFA-NS-13-003~$125 K direct/year

PK/Tox

FormulationDevelopment

GMP Scale‐Up Synthesis

Phase IClinical Trials Due Oct 8th

Up to $10 M value per project, in total

Development Clinical Trials

NINDS Cooperative Program inNINDS Cooperative Program inNINDS Cooperative Program in NINDS Cooperative Program in Translational ResearchTranslational Research

Pre-clinicalSafety

LeadOptimization

• Pre-clinical development and testing of new therapies No mechanistic clinical or screening projectsNo mechanistic, clinical, or screening projects

• Goal: IND/IDE submission by the end of the project• Milestone driven cooperative agreement program for• Milestone-driven cooperative agreement program for

small business (U44) and academics, etc. (U01)• 3-5 years larger budget• 3-5 years, larger budget• Reviewed by therapy development experts• Significant staff involvement• Significant staff involvement • Contact Program Before Applying

PAR-11-296/PAR-11-294

NINDS E l t Cli i l T i lNINDS E l t Cli i l T i lNINDS Exploratory Clinical TrialsNINDS Exploratory Clinical Trialsfor Small Businessfor Small Business

Phase I/IITrials

• NINDS accepts Phase I/II Clinical Trials through specific program announcementsprogram announcements

• Phase III Clinical Trials are not accepted through the SBIR/STTR programSBIR/STTR program

• Evaluation of drugs, biologics, devices, or diagnostics as well as surgical, behavioral or rehabilitation therapiesg , p

• 3-5 years of funding, larger budget• Application includes clinical protocol, study organization and pp p , y g

administration section, data and safety monitoring plan

PAR 12 0 2/PAR 12 0 3PAR-12-072/PAR-12-073

NINDS SBIR/STTRNINDS SBIR/STTRNINDS SBIR/STTR NINDS SBIR/STTR Competing RenewalCompeting Renewal

Phase I/IITrials

Pre-clinicalSafety

LeadOptimization

NIH S ll B i P Pri ate In estmentSBIR/STTRValley of DeathNIH Small Business Program: General SBIR & STTR

Private InvestmentStrategic Partner

SBIR/STTR Competing Renewal

Specific announcements: / C f•PAR-12-074/PAR-12-075: Pre-Clinical Research for FDA Approval

•PAR-12-076/PAR-12-077: Phase I/II Clinical Trials

Strongly Encourage Matching Funds

U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health 

NCI Programs for NCI Programs for T l i l R hT l i l R hTranslational ResearchTranslational Researchhtt // bihttp://sbir.cancer.gov

Todd Haim, PhD Program Manager

NCI SBIR Development CenterNCI SBIR Development Center

NCI SBIR Development CenterNCI SBIR Development CenterppMichael Weingarten, MA (Director)Previous• NASA – Program Manager, NASA Technology Commercialization Program

Andrew J. Kurtz, PhD (Branch Chief)Previous• NIH AAAS Science & Technology Policy FellowCommercialization Program

Greg Evans, PhD (Branch Chief)Previous• NHLBI/NIH – Program Director, Translational and Multicenter Clinical Research in Hemoglobinopathies

• NIH – AAAS Science & Technology Policy Fellow• Cedra Corporation – Research Associate, Bio-

Analytical Assays and Pharmacokinetics Analysis

Jian Lou, PhD (Program Director)PreviousMulticenter Clinical Research in Hemoglobinopathies

• NHGRI/NIH – Senior Staff FellowPrevious• Johnson & Johnson – Research Scientist, Target

Validation & Biomarker Development• Lumicyte, Inc. – Director, Molecular Biology Systems Analysis

Patti Weber, DrPH (Program Director)Previous• International Heart Institute of Montana –

Deepa Narayanan, MS (Program Director)Previous

• International Heart Institute of Montana –Tissue Engineering and Surgical Research

• Ribi ImmunoChem Research, Inc. – Team Leader, Cardiovascular Pharmacology

Todd Haim, PhD (Program Manager)Previous• National Academy of Sciences – Christine Mirzayan

Science and Technology Policy Fellow• Pfizer Research Laboratories – Postdoctoral Fellow, C di P h i & M b li Di dPrevious

• Naviscan PET Systems, Inc., Director, Clinical Data Management (Oncology Imaging & Clinical Trials)

• Fox Chase Cancer Center, Scientific Associate(Molecular Imaging Lab)

Cardiac Pathogenesis & Metabolic Disorders

Amir Rahbar, PhD, MBA (Program Director)Previous• NCI– Program Director, Center for Strategic Scientific Initiatives

• BioInformatics, LLC – Senior Science Market Analyst• Naval Research Laboratory – Research Scientist

Jennifer Shieh, PhD (AAAS Science & Technology Policy Fellow)P i

Catherine Langston, MA (Program Analyst)

Julienne Willis (Program Specialist)Previous• National Academy of Sciences – Christine MirzayanScience and Technology Policy Fellow

• Syapse, Inc. – Biology Associate

NCI SBIR: Translational ResearchNCI SBIR: Translational Research

• Targeted Solicitations

• NCI Bridge Awardg

• NCI Investor Forum

Targeted SolicitationsTargeted Solicitationshttp://sbir cancer gov/funding/find funding asphttp://sbir cancer gov/funding/find funding asphttp://sbir.cancer.gov/funding/find_funding.asphttp://sbir.cancer.gov/funding/find_funding.asp

NCI SBIR C t t• NCI SBIR Contracts: http://sbir.cancer.gov/funding/contracts/o Released in August: Due around November (one submission date)o Approximately 25% of the funding portfolioo Topics are ripe for commercialization (e.g. Companion Diagnostics)o Tech Transfer: Soliciting proposals for development of specific NCI technologieso Conference Session: “The Ins and Outs of NIH SBIR Contracts”– Today at 3PMy

• RFAs and PAs: http://sbir cancer gov/funding/grants/• RFAs and PAs: http://sbir.cancer.gov/funding/grants/o Cancer Diagnostic and Therapeutic Agents Enabled by Nanotechnologyo Image Guided Interventions : Focus is on integration of component technologieso SBIR Innovative Molecular Analysis Technologies (IMAT): Commercial Feasibility

• http://innovation.cancer.gov/o More coming soon

NCI SBIR Bridge AwardNCI SBIR Bridge AwardPhase IIICOMMERCIALIZATION

Phase IIDEVELOPMENT

Phase IFEASIBILITY

NCI SBIR Phase II Bridge AwardCROSSING THE VALLEY OF DEATH

• NCI gives competitive preference and funding priority to applicants that raise substantial third-party funds (i e ≥ 1:1 match)that raise substantial third-party funds (i.e., ≥ 1:1 match)

• Broad Eligibilityo Current Phase II awards & and those that ended within the last 2 yearso Current Phase II awards & and those that ended within the last 2 yearso Cancer-related Phase II projects initially funded by other NIH institutes

• > 75% of NCI SBIR Phase II projects fit the scope of the awardo Cancer Therapeuticso Cancer Imaging Technologies, Interventional Devices & In Vivo Diagnosticso In Vitro and Ex Vivo Cancer Diagnostics and Prognostics

• Special Review to Evaluate Technical and Commercial Meritso Preferred Types of Funds: Cash, liquid assets, convertible debt

• 12 Awards to Date; > 2:1 Leverage

NCI SBIR Investor Forum NCI SBIR Investor Forum 

Exclusive opportunity for NCI SBIR Awardees to i h h i h l i 100 i lpitch their technologies to > 100 venture capital or 

strategic partner representatives (i.e. big pharma)http://sbir.cancer.gov/investorforum/

• The 2012 Forum was the 3rd NCI SBIR Investor Forumo Over 150 meetings occurred between SBIR awardees and Investors

• ~ 15 NCI SBIR Awardees are chosen to present at each evento External Reviewers included representatives from venture capital firms, big pharma, and large

device companies

• Each presenting company is mentored by an investment/industry• Each presenting company is mentored by an investment/industry professional

Th N b 2010 F h t l d ti• The November 2010 Forum has proven extremely productiveo 6 out of the 14 presenting companies have closed deals with total value > $230 Million

NCI Contract ResourcesNCI Contract Resources• NCI Experimental Therapeutics (NExT)

o Assists clinical translation of novel therapeutic interventions – anticancer drugs (small molecules, biologics) and imaging agents

o Provides access to preclinical drug discovery and clinical development resources o The next submission deadlines are June 15 and October 15 o http://next.cancer.gov.

• NCI PREVENT Preclinical Drug Development ProgramNCI PREVENT Preclinical Drug Development Programo Pipeline to bring new cancer-preventing interventions and biomarkers through preclinical

development towards clinical trialso Flexible entry-point system to optimize and test an agent with the goal of filing an INDo http://prevention.cancer.gov/programs-resources/programs/preventp p g p g p g po For clinical development resources, companies should apply to the Consortia for Early Phase

Prevention Trials: http://prevention.cancer.gov/clinicaltrials/management/consortia

• NCI Clinical Assay Development Program (CADP)NCI Clinical Assay Development Program (CADP)o Provides access to tissue and laboratory resources for analytical and clinical validation of assayso Eligible applicants must have a working prototype assay with a clearly defined clinical useo The next submission deadlines are June 15 and October 15o http://cadp cancer govo http://cadp.cancer.gov.

• For additional resources including biospecimen resources, chemicals repository and cancer imaging archives, please visit htt // bi / / t /http://sbir.cancer.gov/resource/grantees/