Introduction

The first step to commercialization of an invention is to develop enough information about the invention to determine if a patent application would be successful.  This portion of  The Guide will list a number of scientific steps that could lead to a patentable therapeutic and some of the Scientific Cores/Centers of Excellence that are available on the Northwestern campus to assist at each scientific step.  The cores operate on a fee for service basis.  Also included are topics on how to optimize your invention to meet an unmet medical need, when to disclose your invention to INVO, and the patenting process.   

How to validate a pathological pathway/target

Target Identification/Validation: Use of chemical modification

Target Identification/Validation: Genetic models to demonstrate proof of concept for the critical nature of the target

  • Research objective: By using gene knock in/knock out models or siRNA techniques, establish an unequivocal relationship between the target and disease pathogenesis.
  • Project team:
  • Funding – See “How to create a funding plan”

Assay Development for target or pathway modification

How to identify a hit for my target

Hit identification

How to choose an indication

Research objective: FDA defines the indication for a chemical entity as the defined patient population for which the compound has demonstrated efficacy and safety.  It is very important to determine the proposed patient indication/population as early as possible because the indication will determine the pathway to an IND, NDA and the potential commercial opportunities for the compound.  In many instances, the biological pathway will determine the potential indication but it is possible to identify a number of possible indications for a given compound and prioritize them at a later date.  If the target is thought to be involved in a number of pathological conditions, the indications should be evaluated by

  • probability of scientific success – which indication has the strongest scientific data to support in vivo success
  • regulatory success – which indication has the most FDA approved drugs and thus the most tested regulatory pathway
  • commercial success – which indication has the largest potential market with a potential unmet medical need

Project team: To be determined through discussion with Bill Sargent, Entrepreneur in Residence and/or INVO

Funding:  NA

 

Early stage potential funding

    Funding Opportunities for Academic Drug Developers at NU

 

 

Ideation to patent

Patent to License

Start Up

Types of funding

 

 

 

Non dilutive

 

 

 

NIH

X

 

 

NSF

X

 

 

NCI

X

 

 

DoD

X

 

 

Federal Grants Search Center

X

X

X

BARDA

X

X

X

SBIR/STTR

 

 

X

Foundations

X

X

X

Patient Organizations

X

X

X

NewCures*

 

X

 

Lakeside Discovery*

 

X

 

CBC*

X

X

X

N.XT*

 

X

X

Office of Research Development *

X

X

X

Corporate Relations at Feinberg *

 

 

X

Dilutive

 

 

 

Family and Friends

X

 

 

Angels

X

X

 

Venture Capital

 

X

X

Private Equity

 

X

X

Industry Partnerships

 

X

X

*Northwestern Proprietary Funds

 

 

How to create a Commercial Target Product Profile

Research Objective: An FDA Drug Package Insert defines the universe of data that can be used for promotions and advertising.  By comparing the Package Inserts of the drugs approved in a specific indication, one can create a Commercial Product Profile that defines a market place by the characteristics of existing products. 

  • The Commercial Product Profile                                                                    

Drug characteristics                    

Best

Minimum

Drug x

Boxed Warnings

Indications and Usage

Dosage and Administration

Dosage Forms and Strengths

Contraindications

Warnings and Precautions

Adverse Reactions

   On target

   Off target    

Drug Interactions

Use in Specific Populations    

Drug Abuse and Dependence

Overdosage

Clinical Pharmacology      

Animal efficacy models

 

 

 

 

The Commercial Product Profile should be created early in development to guide optimization of potential lead compounds. During optimization, medicinal chemists can alter chemical configurations of compounds to limit, eliminate or potentiate certain characteristics of the compounds.  Additionally, during the optimization stage, the compounds need to be administered to animal models to confirm their expected actions in vivo.  If the expected activities are not found in vivo, the compounds can again be altered through medicinal chemistry and retested.                        

    Commercial Product Profile  —-> Lead Identification—->  Lead Optimization              

     —->Lead Manufacturing—-> Non-IND Enabling Animal Trials

     —->Lead Optimization

Project team: To be determined through discussion with the Entrepreneur-in -Residence and INVO.

Funding – NA

 

How to identify and optimize a lead compound

Research Objective: Produce more potent and selective compounds.  Once hits are identified thru High Throughput and/or Virtual Screening, they are tested in in vivo and in vitro systems and evaluated for:  on target affinity (efficacy), off target binding (side effects) and appropriate PK/ADME and physiochemical properties.  The most appropriate hits are compared to the characteristics of ideal compounds, described in the Commercial Product Profile, to identify areas for improvement of hit compounds.  Chemical modifications of the hits are made thru medicinal chemistry techniques and retested in in vivo and in vitro systems.  The process is repeated until the optimization yields the most appropriate lead compound(s) for potential patenting and further development.                                                      

 

Project team:

Funding:  See “How to create a funding plan”

Exploratory animal pharmacology and toxicology studies

Pre-Clinical Development

Preclinical studies focused of the pharmacological and toxicological properties of lead compounds can be performed in either a GLP or non-GLP environment.  Good Laboratory Practice (GLP) is a quality system concerned with the organisational process and the conditions under which non-clinical health and environmental safety studies are planned, performed, monitored, recorded, archived and reported.  https://www.fda.gov/ICECI/EnforcementActions/BioresearchMonitoring/ucm135220.htm.

Because GLP studies are more costly and require extensive documentation, most initial, exploratory pre-clinical pharmacology and toxicology studies are non-GLP studies and non-IND enabling.  Once enough information has been collected to move to final pre-clinical pharmacology and toxicology, the studies must be done under GLP conditions.

The choice of animal models efficacy for determining the efficacy of a lead compound(s) can be determined through a review of the literature and a review of drugs already approved for the indication, which can be obtained by accessing their Summary Basis for Approval.

FDA Guidance documents https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM073246.pdf

https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm065014.htm

https://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm362416.htm

 

A.  Pre-clinical pharmacology

1. Primary Pharmacodynamics                                                                          Research objective: Describe the in vivo and in vitro pharmacological effects and the mechanisms of action of the drug related to the desired therapeutic target.  Selection of appropriate animal models, that represent the human disease condition, is critical.  Review the Summary Basis for Approval of licensed drugs and determine the most recent pre-clinical literature to determine the most appropriate animal models.

Project team:
Developmental Therapeutics Core
Pre-Clinical Models Core-NU-Go Kidney
TEST IT Core, SDRC
Proteomics Core (companion diagnostics/biomarkers)
Search Core Websites and Search Core Facilities Database for specific terms of interest

Funding – See “How do I create a funding plan”

2.  Secondary Pharmacodynamics                                                  

Research objective: Describe the in vivo and in vitro pharmacological effects and the mechanisms of action of the drug not related to the desired therapeutic target.  These studies are expected to provide data to calculate a projected safety margin, implications for clinical safety monitoring and mitigation strategies.

Project team:

Developmental Therapeutics Core
Pre-Clinical Models Core-NU-Go Kidney
TEST IT Core, SDRC
Proteomics Core (companion diagnostics/biomarkers Search Core Websites and Search Core Facilities Database for specific terms of interest

Funding – See “How do I create a funding plan”

3.  Pharmacokinetics                                                                                              Research objective: Describe the absorption, distribution, metabolism and excretion of the drug using appropriate in vivo and in vitro

Project team:
Developmental Therapeutics Core
Pre-Clinical Models Core-NU-Go Kidney
TEST IT Core, SDRC
Proteomics Core (companion diagnostics/biomarkers)                                                     Search Core Websites and Search Core Facilities Database for specific terms of interest                                                                                                             

Funding – See “How do I create a funding plan”

4.  Safety Pharmacology                                                                                                       Research objective: Describe the off target pharmacological effects of the drug on the following systems:  neurological, cardiovascular, pulmonary, renal, gastrointestinal, other systems as appropriate and abuse liability.  These studies are expected to provide data to calculate a projected safety margin, implications for clinical safety monitoring and mitigation strategies.

Project team:
Developmental Therapeutics Core
Pre-Clinical Models Core-NU-Go Kidney
TEST IT Core, SDRC
Proteomics Core (companion diagnostics/biomarkers)                                                     Search Core Websites and Search Core Facilities Database for specific terms of interest   

Funding – See “How do I create a funding plan”

 

B.  Pre-Clinical Toxicology

FDA Guidance documents

https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm065014.htmhttps://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM074980.pdf

https://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm362416.htm

Research objective: Describe the toxicological effects of the drug.  Depending on the nature of the drug and the phase of the investigation, the description is expected to include:  the results of acute, subacute, and chronic toxicity tests; the results of tests of the drug’s effects on reproduction and the developing fetus; any special toxicity test related to the drug’s particular mode of administration or conditions of use (e.g., inhalation, dermal, or ocular toxicology); and any in vitro studies intended to evaluate drug toxicity.  Genetic toxicology, carcinogenicity, reproductive and developmental toxicology and additional special toxicology studies may be required.  The pre-clinical toxicology program must be conducted in a GLP laboratory.  This plan is generally discussed with FDA during the pre-IND meeting.

Project team:                                                                                                                     UIC Pre-clinical GLP Toxicology Lab (CBC)                                                                        CRO’s and consultants

Funding– See “How to create a funding plan”

How to patent my invention
How to apply to NewCures for funding

NewCures is a biomedical accelerator that focuses on the development of potential therapeutics from Northwestern University laboratories. Its mission is to strengthen Northwestern’s therapeutic pipeline and to expedite the translational process of early-stage research discoveries into next generation therapeutics to ultimately benefit patients.

Area of Interest

All molecular-based therapeutic projects with an identified biological target with a translational/commercialization trajectory are encouraged to apply. At this time, NewCures does not support projects involving therapeutic delivery/formulation, diagnostics, devices, research tools or healthcare information.

Eligibility

Applicant: Northwestern University principal investigator (PI) with a full-time faculty appointment.

Intellectual property (IP)* related to the application must meet the following criteria:

  • Owned by Northwestern University;
  • If IP is jointly owned, NU must be the lead, and joint owner must be a non-profit organization;
  • Should cover Composition of Matter (issued or pending);
  • Should be eligible for international protection; and
  • Not licensed to a third party.

 

Funding/Benefits

Projects will be supported at a level deemed necessary to achieve carefully evaluated objectives. After project selection and PI agreement, NewCures will:

  • Work with the PI and external experts to formulate a development plan, outlining key pre-clinical experiments for proof-of-concept validation;
  • Collaborate with the PI to execute milestone-driven project plans (i.e., data collection); the experimental work will be outsourced to third party contract research organizations (CROs) – outsourced work will be managed and paid for by NewCures (up to $200K); and
  • Work with INVO and the PI to engage potential private sector partnership and/or investment, contingent on the achievement of desired experimental outcomes/milestones.

*NewCures does not support investigator’s salary, research personnel salaries, project reagents, supplies or equipment, intellectual property services, or travel.

Applications are open year-round, please contact us at

https://newcures.northwestern.edu/contact-us/

newcures.northwestern.edu

https://www.invo.northwestern.edu

 

How to apply to Lakeside Discovery for funding