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NIH Programs for  Translational Research T l i lR h Stephanie Fertig, MBA (NINDS) T dd Haim, Todd H i PhD (NCI) Kurt Marek, PhD (NHLBI) Jerry Wujek, PhD (NEI) John McKew, PhD (NCATS)

Phase Phase IIB Programs IIB Programs • Competing Renewal Renewal, Phase IIB or Bridges Award • General Omnibus: NIA, NIAAA, NIAID, NICHD, NIDA, NIDCD NIDDK NIDCD, NIDDK, NEI NEI, NIGMS NIGMS, NIMH, NIMH NCATS, NCATS ORIP o Some are focused on projects requiring FDA approval o Specific IC research topics sections (omnibus) have requirements/instructions i t /i t ti

• Specific Announcements: NCI, NHLBI, NINDS o Application includes a fundraising plan o Strongly Encourage Matching Funds

Contact Program Before Applying

SBIR Technology Transfer (SBIR SBIR Technology Transfer (SBIR‐‐TT) • Transfer technology out of the NIH intramural research labs into the private sector • Projects P j t mustt rely l on the th use off NIH iintramural t l ttechnology h l • Investigator Initiated (Grants): NINDS, NHLBI, NEI o Application assignment follows extramural receipt and referral guidelines o NINDS and NEI: Technologies from any NIH intramural lab o 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 awarded o Can apply for an exclusive license agreement

Contract Resources Contract Resources • Offer access to services instead of grant money Examples: o cGMP Manufacturing o Pharmacokinetic/ADME Studies o Pharmacology/Toxicology Studies o Regulatory Support o Clinical Supply Manufacturing o and many others…

• Submission: o May or may not go through o Not a standard grant application

• Review process: o Not a standard review process o Varies from program to program

Contact Program Before Applying

Translational Programs at NIMH g Drug 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) p ((PAR-10-216)) o Novel NeuroAIDS Therapeutics o Innovative Pilot Studies of Novel Mechanism of Action Compounds for Treating Psychiatric Disorders (PAR-11-316)

Resources: • • • •

NIMH Psychoactive Drug Screening Program: Toxicological g Evaluation of Novel Ligands g Program: NIMH Chemical Synthesis and Drug Supply Program NIMH Genetics Repository: https://www nimhgenetics org/

Neurotherapeutics p Discovery Programs y g At NINDS

Target ID



Hit to Lead

Proof of Concept

Lead O ti i ti Optimization

Pre-clinical Safety

Phase I/II Trials

NIH Blueprint Neurotherapeutics Network (BPN) NIH Small Business Program: General SBIR & STTR Translational Cooperative Agreement

SBIR/STTR Clinical Trials

SBIR/STTR Competing Renewal (Phase IIB)

Hit to Lead

Proof of Concept

Lead Optimization

Pre-clinical Safety y

Phase I/II Trials

Blueprint Neurotherapeutics Blueprint  p Neurotherapeutics p Grand Challenge g • “Virtual Pharma” structure

Lead Development Team Principal Investigator Industry-seasoned consultants NIH staff

U01s Bioactivity/ Efficacy Studies ~$125 K direct/year

NIH Contracts Medicinal Chemistry

Data Management


GMP Scale‐Up  GMP S l U Synthesis

Formulation Development

Phase I Clinical Trials Clinical Trials

Up to $10 M value per project, in total

o Industry-standard Industry standard services o Industry-seasoned consultants o Funding for biology

• PI retains control of IP • Entry Requirements: o Strong biological rationale o Robust assay for SAR, biological validation o Well-validated W ll lid t d hit compound d

RFA-NS-13-003 Due Oct 8th

Lead Optimization

Pre-clinical Safety

NINDS NINDS Cooperative Program in  Cooperative Program in Translational Research

• Pre-clinical development and testing of new therapies No mechanistic mechanistic, clinical clinical, or screening projects • Goal: IND/IDE submission by the end of the project • Milestone-driven Milestone driven cooperative agreement program for small business (U44) and academics, etc. (U01) • 3-5 years years, larger budget • Reviewed by therapy development experts • Significant staff involvement • Contact Program Before Applying PAR-11-296/PAR-11-294

Phase I/II Trials

NINDS E l t NINDS Exploratory Clinical Trials Cli i l T i l for Small Business

• NINDS accepts Phase I/II Clinical Trials through specific program announcements • Phase III Clinical Trials are not accepted through the SBIR/STTR program • Evaluation of drugs, biologics, devices, or diagnostics as well g , behavioral or rehabilitation therapies p as surgical, • 3-5 years of funding, larger budget pp includes clinical p protocol,, study y organization g and • Application administration section, data and safety monitoring plan

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

Lead Optimization

Pre-clinical Safety

Phase I/II Trials

NIH S Small ll B Business i P Program: General SBIR & STTR

NINDS SBIR/STTR NINDS SBIR/STTR  Competing Renewal SBIR/STTR Valley of Death Competing Renewal

Private Pri ate Investment In estment Strategic Partner

Specific announcements: •PAR-12-074/PAR-12-075: / Pre-Clinical C Research ffor FDA Approval •PAR-12-076/PAR-12-077: Phase I/II Clinical Trials

Strongly Encourage Matching Funds


NCI Programs for  Translational Research T l i lR h htt // bi Todd Haim, PhD Program Manager NCI SBIR Development Center

NCI SBIR Development Center p Michael Weingarten, MA (Director) Previous • NASA – Program Manager, NASA Technology  Commercialization Program Commercialization Program

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

Patti Weber, DrPH (Program Director) Previous • International Heart Institute of Montana – Tissue Engineering and Surgical Research • Ribi ImmunoChem Research, Inc. – Team Leader, Cardiovascular Pharmacology

Deepa Narayanan, MS (Program Director) Previous • Naviscan PET Systems, Inc., Director, Clinical Data Management (Oncology Imaging & Clinical Trials) • Fox Chase Cancer Center, Scientific Associate (Molecular Imaging Lab)

Catherine Langston, MA (Program Analyst)

Andrew J. Kurtz, PhD (Branch Chief) Previous • NIH – AAAS Science & Technology Policy Fellow • Cedra Corporation – Research Associate, BioAnalytical Assays and Pharmacokinetics Analysis

Jian Lou, PhD (Program Director) Previous • Johnson & Johnson – Research Scientist, Target Validation & Biomarker Development • Lumicyte, Inc. – Director, Molecular Biology  Systems Analysis

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 Cardiac Pathogenesis & Metabolic Disorders i & M b li Di d

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)

Julienne Willis (Program Specialist)

Previous P i • National Academy of Sciences – Christine Mirzayan Science and Technology Policy Fellow • Syapse, Inc. – Biology Associate

NCI SBIR: Translational Research • Targeted Solicitations

• NCI Bridge g Award

• NCI Investor Forum

Targeted Solicitations http://sbir cancer gov/funding/find funding asp

• NCI SBIR Contracts: C t t o o o o o

Released in August: Due around November (one submission date) Approximately 25% of the funding portfolio Topics are ripe for commercialization (e.g. Companion Diagnostics) Tech Transfer: Soliciting proposals for development of specific NCI technologies y at 3PM Conference Session: “The Ins and Outs of NIH SBIR Contracts”– Today

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





• NCI gives competitive preference and funding priority to applicants that raise substantial third-party funds (i.e., (i e ≥ 1:1 match) • Broad Eligibility o Current Phase II awards & and those that ended within the last 2 years o Cancer-related Phase II projects initially funded by other NIH institutes

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

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

• 12 Awards to Date; > 2:1 Leverage

NCI SBIR Investor Forum  Exclusive opportunity for NCI SBIR Awardees to  pitch their technologies to > 100 venture capital or  i h h i h l i 100 i l strategic partner representatives (i.e. big pharma)

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

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

• Each presenting company is mentored by an investment/industry professional • The Th November N b 2010 F Forum h has proven extremely t l productive d ti o 6 out of the 14 presenting companies have closed deals with total value > $230 Million

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

Assists clinical translation of novel therapeutic interventions – anticancer drugs (small molecules, biologics) and imaging agents Provides access to preclinical drug discovery and clinical development resources The next submission deadlines are June 15 and October 15

• NCI PREVENT Preclinical Drug Development Program o o o o

Pipeline to bring new cancer-preventing interventions and biomarkers through preclinical development towards clinical trials Flexible entry-point system to optimize and test an agent with the goal of filing an IND p p g p g p g p For clinical development resources, companies should apply to the Consortia for Early Phase Prevention Trials:

• NCI Clinical Assay Development Program (CADP) o o o o

Provides access to tissue and laboratory resources for analytical and clinical validation of assays Eligible applicants must have a working prototype assay with a clearly defined clinical use The next submission deadlines are June 15 and October 15 http://cadp cancer gov

• For additional resources including biospecimen resources, chemicals repository and cancer imaging archives, please visit htt // bi / / t /