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Christine Densmore, MSNational Institute of Diabetes and Digestive and Kidney
Diseases
Non-Dilutive Funding Conference
January 9, 2019
NIDDK Research: Opportunities for Small Businesses
Twitter: @NIDDKgov
Chronic
Common
Consequential
Costly
Research Mission of NIDDK
NIH Support for Research
NIH supports research in every state and around the world
– Almost 50,000 competitive grants
– More than 300,000 researchers
– More than 2,500 universities, medical schools, and other research institutions
https://www.nih.gov/about-nih/what-we-do/budget
NIH SB FY 2019: $1.145 billion % SBIR/STTR: 3.20% SBIR ; 0.45% STTR
Type 1 Diabetes
Type 2 Diabetes
Childhood Diabetes
Diabetes Complications
Cystic Fibrosis
Inborn Metabolic Errors
Wasting Syndrome
HAART Therapy
Molecular Therapies
Endocrinology of Bone
Obesity
Crohn’s Disease
Ulcerative Colitis
Liver Disease
Hepatitis
Drug Induced Liver Injury
NASH
Hemochromatosis
Obesity
Irritable Bowel Syndrome
Gastroparesis
Barrett’s Esophagus
Chronic Kidney Disease
Polycystic Kidney Disease
Kidney Injury & Failure
Vesicoureteral Reflux
Urinary Incontinence
Urinary Tract Infections
Benign Prostatic Hyperplasia
Chronic Prostatitis
Painful Bladder Syndrome
Anemias & Iron Overload
Sickle Cell Disease
Division of Kidney, Urologic, and Hematologic
Diseases
Division of Diabetes,
Endocrinology, and Metabolic Diseases
Division of Digestive Diseases
and Nutrition
NIDDK’s Extramural Research ProgramsBy Division
*NEW*Office of Nutrition
Rsearch
Leadership of nutrition research at NIDDK & NIH
Strategic planning
Portfolio analysis
Budget and resource allocation
Assessment of research needs /opportunities
NIDDK Budget, Fiscal Years 2014 to 2018
$1,883$1,900
$1,968
$2,010
$2,121
2014 2015 2016 2017 2018
Do
llars
in m
illio
ns
Fiscal Year
Includes $ from Special Statutory Funding Program for Type 1 Diabetes Research
NIH SBIR/STTR Relative IC Contributions FY 2019
NIDDK 2019 Small Business Budget
NIDDK 56.80M
T1D* 2.63M
SBIR
NIDDK 8.10M
T1D* .37M
STTR
What is SBIR & STTR at NIH?
Feasibility
Discovery
Phase I
Full R/D
Phase I + Phase II
Development
Phase IICommercialization
Phase IIICompeting Renewal Award
Phase IIB
Additional R/D
Only Some ICs
Participate
Fast-Track
NIH SBIR & STTR
• America’s Seed Fund -
one of the largest
sources of early-stage
capital for technology
commercialization in the
United States
• Phased funding for US-
owned and operated
small businesses to
engage in federal
research and
development that has a
strong potential for
commercialization
• A critical tool to achieve
NIH’s mission to
improve health and
save lives
NIDDK FY 2018 SBIR/STTR Award Scores
0
2
4
6
8
10
12
14
16
18
10 to 20 21 to 25 26 to 28 29 to 30 31 to 34 35 to 38
# o
f ap
plic
atio
ns
fun
ded
Priority Score
Average NIDDK SBIR/STTR Award by Fiscal Year
$0
$200,000
$400,000
$600,000
$800,000
$1,000,000
$1,200,000
$1,400,000
$1,600,000
$1,800,000
2015 2016 2017 2018
Phase I Phase II
Do
llars
NIDDK SBIR/STTR FY2018 Awards by Sector
Drugs 48%
Devices 18%
Diagnostics 20%
Research Tools 7%
Software and Health IT7%
Technical Assistance Programs NIDDK Supports
Phase I Phase II Phase III
Commercialization Accelerator
Program (CAP)
Niche
Assessment
Program (NAP)
Valley
of
Death
Phase IIB
Market-
Ready
Technology
Early-Stage
Technology
?
Coulter C3i , I-Corps
Commercialization Readiness Pilot
NIDDK-Supported Success Stories!
Division of Diabetes, Endocrinology, & Metabolic Diseases
Mission Areas:Diabetes prevention, diagnosis and management/treatment
Acute and Chronic complications of diabetes
Other endocrine disorders including hypophysis, thyroid, adrenal,
neuroendocrinology and bone metabolism
Cystic Fibrosis and its metabolic impact
Example Technologies:Continuous glucose monitoring and insulin delivery technologies, integration into an
automated artificial pancreas system (APS).
Smart insulin, glucagon and novel drugs for glycemic control
Multiplex antigen panel for early detection and monitoring of Type 1 Diabetes
Technologies to support cell replacement therapies for diabetes
Wound healing and diabetic foot ulcer (DFU) treatments
Visit sbir.nih.gov/niddk/divisions#DEM for contacts in DEM
Integration of Technologies into an Artificial Pancreas System (APS)
- Development of integrated artificial intelligence systems (In Control) to mimic the function of the pancreas for the treatment of diabetes.
- NIDDK supported academic (University of Virginia) and SBIR grants (TypeZero) for its development.
- Being tested in advanced APS international clinical trials
- Fully integrated bionic pancreas (i-Let)
- NIDDK has supported academic (Boston University) and SBIR grants (BetaBionics) for its development.
- Being tested in advanced APS international clinical trials
Glucagon Rescue Pen (G-PenTM)• Treatment of severe hypoglycemia
• Replace existing glucagon emergency kit (GEK)
Glucagon Mini-Dosing Pen • Treatment of moderate hypoglycemia
• Personalized glucagon administration
Artificial Pancreas (Bi-hormonal pump)• For use in dual-chamber pump (insulin and
glucagon)
• Automated glucose control
Novel Glucagon formulations for Hypoglycemia Rescue/Tx and APS use
- NIDDK has supported development and clinical testing with SBIR grants- NDA for the Rescue Pen has been submitted to the FDA
Division of Digestive Diseases & Nutrition
Mission Areas:Digestive diseases, including the alimentary tract, liver, and pancreas
Nutrition and nutrient metabolism
Obesity, including type 2 diabetes, energy balance, neurobiology
Example Technologies:Technologies for visualization and automated analysis of gastrointestinal
motility
Clinical assay for genetic causes of liver disease in children
Immunoassay for studying iron regulation and assessing it clinically
Wearable technologies (e.g., in earbuds or shoe-soles) for monitoring
caloric expenditure
Animal models of metabolic syndrome and obesity that more accurately
recapitulate aspects of these diseases in humans
Visit sbir.nih.gov/niddk/divisions#DDN for contacts in DDN
HemoShear Therapeutics
HUMAN DISEASE MODELS PLATFORM – REVEAL-Tx
• Recreated nearly twenty human disease states
• propionic acidemia
• methylmalonic acidemia
• nonalcoholic steatohepatitis (NASH)
• In combination with advanced proprietary computational biology tools
• reduce risk of failure by enabling deep interrogation of disease pathways
• test hypotheses
• select drug targets under physiologically accurate disease conditions
A bioengineered GI sphincter for Fecal Incontinence
– Uses autologous neuro-progenitor and smooth muscle cells
– Surgically implanted around the patient’s dysfunctional internal anal
sphincter, the root cause of Fecal Incontinence
– The implant is “prewired" so enables new neurological connections
– Status: in Clinical Trials
BioSphincterTM
Intrinsic LifeSciences (ILS)
Developed assays for key iron-regulating hormones for diagnosis of genetic and
acquired iron disorders
• Areas include congenital anemias, hemochromatosis, and inflammatory
diseases in which iron homeostasis is dysregulated
• Products inspired by basic science research conducted by NIDDK R01
grantees (multiple DK divisions)
• Market impact: multiple RUO assays available for purchase
• CAP/CLIA accreditation for clinical diagnostic laboratory (IntrinsicDx)
Visit sbir.nih.gov/niddk/divisions#KUH for contacts in KUH
Division of Kidney, Urologic, & Hematologic Diseases
Mission Areas:Kidney diseases
Urinary tract diseases
Disorders of the blood and blood-forming organs
Example Technologies:Animal models of polycystic kidney disease
Improved glomerular filtration rate (GFR) measurements
Pharmaceutical compounds which promote fistula maturation (for
hemodialysis)
Materials for urinary and dialysis catheters which resist fouling
Injectable thermo-sensitive plugs for control of blood flow during surgery
(e.g., partial nephrectomy)
Chip-based technology for blood diagnostics
Developed a biofouling resistant coating based on the denticles
on sharkskin
• Entered market with technology on urinary catheters
• Multiple licensing deals, including one with Cook Medical
• Successful in raising subsequent follow-on funding
• Received several $MM in R&D grants
• Ultimately acquired by an equity medical device firm
Sharklet Technologies
Feasibility
Discovery
Phase I
Full R/D
Phase I + Phase II
Development
Phase IICommercialization
Phase IIICompeting Renewal Award
Phase IIB
Additional R/D
Only Some ICs
Participate
Fast-Track
A Pipeline to Small Business Opportunities
NIH SBIR & STTR
NIH Common
Fund or Central
Programs
NIDDK Translational
Research for Therapeutic
Discovery and
Development Pipeline
NIDDK Initiatives
with Translational
Implications
http://bit.ly/niddktranslate
Current R01 FOAs for Assay Development, High Throughput Screening, and
Preclinical Therapeutic Validation
Current SBIR/STTR FOAs for Lead Optimization and Preclinical Development
Other Opportunities and Resources for Drug Development
NIDDK Translational Research for Therapeutic Discovery and Development Pipeline
Current Funding Opportunities for Research in NIDDK Mission Areas
• Omnibus/Parent Solicitations
– SBIR Omnibus/Parent Clinical Trial Not AllowedFunding Opportunity Announcement (PA-18-574)
– STTR Omnibus/Parent Clinical Trial Not AllowedFunding Opportunity Announcement (PA-18-575)
• Clinical Trials
– NIDDK Exploratory Clinical Trials for Small Business (R44 Clinical Trial Required) (PAR-18-108) SBIR-only
https://sbir.nih.gov/funding/individual-announcements
https://sbir.nih.gov/funding
NIDDK Funding Opportunities (cont’d)
• Lead Optimization and Preclinical Development of Therapeutic Candidates for Diseases of Interest to the NIDDK
– SBIR/STTR: PA-17-130/131
• Kidney Precision Medicine Project - Technology Development and Validation
– SBIR/STTR: PA-16-452/451
Other Subscribed Program Announcements
• Development of Highly Innovative Tools and Technology for Analysis of Single Cells
– SBIR/STTR: PA-17-147/148
https://sbir.nih.gov/funding/individual-announcements
NIDDK Funding Opportunities Supported by the T1D Program
Examples of Recent* SBIR-only Type 1 Diabetes RFAs:
• Support for Small Business Innovation Research to Develop New Open and Closed-Loop Automated Technologies for Better Type 1 Diabetes Therapy and Monitoring (SBIR) (R43/R44 Clinical Trial Not Allowed)
– SBIR: RFA-DK-17-029
• Development of New Technologies and Bioengineering Solutions for the Advancement of Cell Replacement Therapies for Type 1 Diabetes (R43/R44 Clinical Trial Not Allowed)
– SBIR: RFA-DK-17-030
*no longer active
NIDDK/NIH Support Beyond Grants
Cross-IC technical assistance programs
Showcase opportunities at major
industry and investment events
I- Corps™
Engagement with KOLs and other
stakeholders within the NIDDK-supported
scientific community
NIH Common Fund and Central Programs
Stimulating Peripheral Activity to
Relieve Conditions (SPARC)
Regenerative Medicine
Innovation Project (RMIP)
https://commonfund.nih.gov/
Stimulating Peripheral Activity to Relieve Conditions (SPARC)
• Challenge: Neuromodulation of organ
function holds promise in treating many
diseases. However, the mechanisms of
action for neuromodulation therapies
remain poorly understood. Results among
patients are highly variable. Sham effects
are large.
• Mission: Catalyze rational target
development for peripheral
neuromodulation indications by building
anatomical and functional maps of the
autonomic nervous system in relation to
major organs
https://commonfund.nih.gov/SPARC
SPARC (cont’d)
SPARC 1 Anatomical and Functional Mapping of the Innervation of Major Internal Organs
https://commonfund.nih.gov/sparc/grants
Funding opportunities and collaborative opportunities throughout all four components open to small businesses
SPARC 2 Next Generation Tools and Technologies
SPARC 3 Translational Partnerships for Human Functional Mapping and New Indications
Project Team Leaders from NIDDK
SPARC 4 Data and Resource Center: Datasets that may inform device development by small businesses
Illuminating the Druggable Genome (IDG)
https://druggablegenome.net/
• Druggable Genome - the subset of the ~20,000 genes in the human genome that express proteins potentially able to bind drug-like compounds.
• Goal of the IDG Program –improve our understanding of the properties and functions of proteins that are currently not well studied within commonly drug-targeted protein families.
Hopkins and Groom, NRDD 2002
Rodgers et al NRDD 2018
Human Genome ~20,000
Approved drugs for only ~25% of Targets
Resources and opportunities for small businesses
pharos.nih.gov
Co-Chaired by Dr. Chris Austin (NCATS) and Dr. Griffin Rodgers (NIDDK)
NIDDK Initiatives with Translational Implications
Kidney Precision Medicine Project
The Response: The NIDDK is supporting the Kidney Precision
Medicine Project (KPMP) to improve histologic, anatomic, and molecular
assessment of human kidney biopsies.
• Companion SBIR and STTR funding opportunities
• Opportunity Pool for partnerships
The Problem: Kidney diseases impose a significant public health
burden. There are few therapies to effectively treat acute and
chronic kidney diseases.
The Opportunity: Recent advances in technologies to interrogate
human kidney tissues have set the stage for “precision medicine”
in nephrology, which will enable the right treatment to reach the
right patient at the right time.
Kidney Precision Medicine Project
https://kpmp.org/
Other Opportunities for Businesses at NIDDK
Extramural Clinical Trial Networks- Academic clinical sites with expertise, staff, and access to patients- Partnerships testing drugs, devices, diagnostics- Contact NIDDK Staff: https://www.niddk.nih.gov/research-funding/research-programs
Repositories of Clinical Trial Samples & Data- NIH-funded trials require deposit of samples & data for dissemination- Sample & Data sharing for acceptable study proposals- NIDDK Repository Requests: https://repository.niddk.nih.gov/home/
Intramural Scientists with Specialized Laboratories, Know-How, or IP- Cooperative Research & Co-Development of New Technologies- Contact Intramural Investigators: https://irp.nih.gov/
NIDDK SBIR Contacts
Division of Diabetes,
Endocrinology, &
Metabolic Diseases
Division of Digestive
Diseases & Nutrition
Division of Kidney,
Urologic, & Hematologic
Diseases
Program Contact
Guillermo A. Arreaza-
Rubin, M.D.
Yan Li, Ph.D.
Teresa Jones, M.D.
Christine Densmore, M.S.
Daniel Gossett, Ph.D.
Administrative and
Business Management
Contact
Natasha Loveless
Karin Johnson
Pamela Love
https://sbir.nih.gov/niddk/divisions
