Advancing Biotechnology Innovation Ming Zhao, PhD NCI SBIR Development Center June 24, 2014.
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Transcript of Advancing Biotechnology Innovation Ming Zhao, PhD NCI SBIR Development Center June 24, 2014.
Advancing Biotechnology Innovation
http://sbir.cancer.gov
Ming Zhao, PhDNCI SBIR Development Center
June 24, 2014
Discovery and development of therapeutics and diagnostics
2
Target Identification
Chemical Synthesis, HTS
Screening & Lead Selection
Preclinical testing &
Lead Optimization
Clinical Trials I, II & III
FDA Filing, Approval &
Lunch
Therapeutic Agents
Post-Marketing &
Drug Surveillance
$800 Million/14 years $3.4 Billion/Year
Diagnostic Opportunity Identification
Chemical Synthesis, Lead Selection & in vitro Assays
Preclinical testing &
Lead Optimization
Clinical Trials I, II & III
FDA Filing, Approval &
Lunch
Diagnostic Imaging Agents
Post-Marketing &
Drug Surveillance
$150 Million/10 years $400 Million/Year
S-SS
-S
1
2
extracellular
intracellular
Adapted from Gurnett, CA et al, 1996. Neuron 16, 431-440.
Ca++
H2N CO2H
a2d as drug targets
a2 1da2 2d
Gabapentin
Voltage-gated Calcium Channels
Drug Development Process
4
TARGETIDENTIFICATION
The physiological, cellular, and/or
genetic basis of a disease is studied to
identify potential therapeutic targets
• Pregabalin is marketed by Pfizer under the trade name Lyrica. • It was designed as a more potent successor to gabapentin.• Pregabalin received U.S. FDA approval for use in treating epilepsy, diabetic
neuropathic pain, and postherpetic neuralgia in December 2004 and appeared on the U.S. market in fall 2005.
• It is an anticonvulsant drug used for neuropathic pain and as an adjunct therapy for partial seizures
• It is also effective for generalized anxiety disorder and is approved for this use in the European Union and Russia
• Sales reached $3.063 billion in 2010.
• Lyrica is one of four drugs which a subsidiary of Pfizer in 2009 pleaded guilty to misbranding "with the intent to defraud or mislead". Pfizer agreed to pay $2.3 billion in settlement
Pregabalin/Lyrica
5
Discovery and development of therapeutics and diagnostics
7
Target Identification
Chemical Synthesis, HTS
Screening & Lead Selection
Preclinical testing &
Lead Optimization
Clinical Trials I, II & III
FDA Filing, Approval &
Lunch
Therapeutic Agents
Post-Marketing &
Drug Surveillance
$800 Million/14 years $3.4 Billion/Year
Diagnostic Opportunity Identification
Chemical Synthesis, Lead Selection & in vitro Assays
Preclinical testing &
Lead Optimization
Clinical Trials I, II & III
FDA Filing, Approval &
Lunch
Diagnostic Imaging Agents
Post-Marketing &
Drug Surveillance
$150 Million/10 years $400 Million/Year
Molecular Imaging – Platform Technology
• Streptavidin (or avidin) & biotin• Bispecific antibody and hapten• DNA and complementary DNA• PNA and complementary PNA• Morpholino and complementary morpholino• FKBP and rapamycin
The goal is to use PET to image tumor in vivo
PET imaging
The effector molecule is given some time after the targeting
agent. This allows time for the targeting agent to localize in
tumor lesions and, more importantly clear from the
body.
Tumor
Tumortarget
cPNA(effector)
Ab
PNA
NCI SBIR&STTR: Advancing the
Commercialization of New Cancer Innovations
http://sbir.cancer.gov
Congressionally-Mandated Programs
2.8%
0.4%
Set Aside (FY14)
~$700M annually at NIH~$119M annually at NCI
14
Small Business Innovation Research (SBIR)Set-aside program for small business concerns to engage in
Federal R&D with the potential for commercialization Federal agencies with an extramural R&D budget > $100M
Small Business Technology Transfer (STTR)Set-aside program to facilitate cooperative R&D between small
business concerns and U.S. research institutions with the potential for commercialization Federal agencies with an extramural R&D budget > $1B
Phase IIICOMMERCIALIZATION
Phase IIDEVELOPMENT
Phase IFEASIBILITY
• Research & Development• Commercialization plan
required• $1.5 million over 2 years
• Commercialization stage• Use of non-SBIR/STTR
funds
Fast Track ApplicationCombined Phase I & II
SBIR & STTR: Three-Phase Program
• Hard caps on award sizes: $225,000 for Phase I; $1.5 million for Phase II• Certain awards may exceed these caps if covered by topic-specific waivers• Actual funding may vary by topic
• Proof-of-Concept study
• $225,000 over 6 months (SBIR) or 1 year (STTR)
Direct to Phase II• Skip Phase I
15
NCI SBIR Phase IIB Bridge Award
CROSSING THE VALLEY OF DEATH
Phase IIICOMMERCIALIZATION
Phase IIDEVELOPMENT
Phase IFEASIBILITY
NCI SBIR Phase IIB Bridge Award
• Provides up to $1M per year for up to 3 years• Open to any NIH-funded Phase II awardees with projects
relevant to NCI mission • Accelerates commercialization by incentivizing partnerships
with third-party investors & strategic partners earlier in the development process
• Competitive preference and funding priority to applicants that can raise substantial third-party funds (i.e., ≥ 1:1 match)
SBIR & STTR Omnibus Solicitations for Grant Applications
Release: JanuaryReceipt Dates: April 5, August 5, and December 5
See the NIH Guide for other Program Announcements (PA’s) and Requests for Application (RFA’s), i.e. grants
Release: WeeklyReceipt Dates: Various
Solicitation of the NIH & CDC for SBIR Contract Proposals
Release: August – sign up for the email list to get notified!Receipt Date: Early November
Multiple Funding SolicitationsKnow the Application Deadlines
http://grants.nih.gov/grants/guide
Topic 326: Development of Novel Therapeutic Agents That Target Cancer Stem Cells
18
(Fast-Track proposals will not be accepted.)
Budget (total costs): PhI: $225,000 for 9 months; PhII: $1,500,000 for 2 years
Number of anticipated awards: 3-5
Project Goals: Proposals under this topic should be involved the development of novel therapeutic agents that target CSCs. These small molecules or biologics should be designed to target CSCs, CSC-related biomarkers, or CSC pathways that affect fundamental processes associated with carcinogenesis, tumor progression, maintenance, recurrence or metastasis. Particular emphasis is placed on agents that target CSC self-renewal, regeneration, or differentiation processes.
Phase I Activities & Deliverables:• Demonstrate in vitro efficacy for the agent(s) that targets CSCs. • Validate the effect of the agent(s) on CSCs. The offerors are required to provide evidence confirming that the agent(s) specifically targets CSCs (e.g., measurement showing reduced quantity, viability, or frequency of CSCs).• Conduct structure-activity relationship (SAR) studies, medicinal chemistry, and/or lead antibody optimization
(as appropriate).• Perform animal toxicology and pharmacology studies as appropriate for the agent(s) selected for development.• Develop a detailed experimental plan (to be pursued under a future SBIR Phase II award) necessary for filing an IND or an exploratory IND.
Therapeu-tics33%
Devices for Cancer
Therapy7%Imaging
20%
In Vitro Diagnos-
tics21%
Health IT7%
Cancer Control and Epidemiology
11%
TherapeuticsDevices for Cancer TherapyImaging In Vitro DiagnosticsCancer BiologyCancer Control and Epidemiology
SBIR/STTR Portfolio
Health IT
NCI SBIR Development [email protected] Phone: 240.276.5300
http://sbir.cancer.gov
Follow us on Twitter @NCIsbir
Ming Zhao, PhDProgram Director