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/