Blueprint Neurotherapeutics Network: Frequently Asked Questions

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 Who should apply?

The Blueprint Neurotherapeutics Network is for neuroscience researchers who have bioactivity assays and small molecule compounds that show activity in these assays but who lack access to the necessary resources and/or expertise to transform these compounds into drug candidates. Applicants must have:

  • At least one small molecule compound with strong evidence of biological activity relevant to the intended indication as a starting point for medicinal chemistry optimization. A single application can include more than one starting compound.
  • A robust, moderate throughput assay of biological activity suitable for testing compounds generated in an iterative medicinal chemistry effort. This assay should be capable of providing robust dose-response data on 20-40 compounds in a 1-2 week cycle.
  • Secondary bioassays and models sufficient to evaluate the potential of a drug candidate for the intended indication.

Further details on the entry requirements are included in the Request for Applications.
This Network is designed for drug development novices as well as for researchers with drug development experience.

 Does the Blueprint Network support drug screening?
No. The Blueprint Network is designed for researchers who have already completed drug screening and have hit compounds with strong evidence of biological activity.
The following NIH programs provide support for compound screening:

 What if I already have access to some drug development services (e.g., medicinal chemistry) or have a project at a more advanced stage? Can I use just a subset of the Blueprint services?
The goal of the Blueprint Network is to ensure that drug development proceed effectively and without impediments. To ensure that this occurs, PIs in the Network are expected to utilize the full range of services provided. PIs may invite their own medicinal chemists and pharmacologists to contribute to the chemical optimization strategy and project design, as participants on the lead development team. However, researchers who would like to use any of their own drug development services or have projects at a more advanced stage should consider using the following NIH drug development programs instead of the Blueprint Network:

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 Are companies eligible for the Blueprint Neurotherapeutics Network?
Yes. Researchers from academic, non-profit, or for-profit organizations are eligible to apply for the Blueprint Network.

 How can my organization compete for contracts to provide Blueprint drug development services?
Organizations that would like to apply to provide Blueprint drug development services should respond to Requests for Proposals published in FedBizOpps.


How will intellectual property be handled?
This Network is structured so that the U01 PI can gain assignment of intellectual property rights (and thereby control the patent prosecution) for drug candidates developed through the Blueprint Network. To this end, the NIH is obtaining a Declaration of Exceptional Circumstances (DEC) to the Federal Acquisition Regulations to ensure that the medicinal chemistry contractor will offer the U01 PI assignment of rights to patent prosecution filings for all compounds generated through the chemistry contract that are directly related to the U01 PI's starting materials. Inventorship will be governed by U.S. law, so that contractor chemists who work under the DEC will be listed on patents. However, the contractor chemists must abide by the royalty-distributing policies of the U01 PI's institution. The medicinal chemistry contractors will retain ownership of inventions on methods of manufacturing or synthesis but must provide broad licenses to the U01 PI to enable subsequent development of drug candidates. Drug development consultants hired by the NIH who have the potential to become inventors must agree to an intellectual property arrangement with the U01 PI's institution before participating in any discussion that could generate intellectual property. The ultimate goal of this Network is to bring new drugs to the clinic, and the NIH will make every effort to facilitate licensing and commercialization by the U01 PI's institution.


What is the mechanism of support?
Successful applicants will receive cooperative agreement awards (U01s) to support bioactivity assays and efficacy studies. Successful applicants will also receive access to research service contracts and drug development consultants funded by the NIH Blueprint.


How will my application be reviewed?
All Blueprint Neurotherapeutics Network applications will be reviewed by a Special Emphasis Panel (SEP) established for this purpose. The SEP will include drug development experts and experts on the relevant disease biology.


What are my chances of getting funded?
The NIH Blueprint currently anticipates funding approximately 10 new drug development projects beginning in FY 2011 and an additional 10 in FY 2012. Each project will be required to meet milestones in order to continue to receive funding and access to resources. Milestones will be established following peer review by a Lead Development Team, which will be co-chaired by the U01 PI and a consultant with senior-level industry experience in drug development and will also include NIH staff and drug development contractors.

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 What would be my role in the drug development process?
The U01 PI will co-chair a Lead Development Team, together with a consultant with senior-level industry experience in drug development. The Lead Development Team will oversee the project and make strategic decisions, including establishment of project milestones and experimental design. In addition, the U01 PI will be responsible for conducting all bioactivity and efficacy assays.

 How should I write Specific Aims for the application, given that much of the work will be done by contractors?
We recommend that your Specific Aims focus on the goals of the work that will be conducted in your laboratory with the funds budgeted in your U01 application. Some examples of activities that could be captured as specific aims are:

  • Validate reproducibility and reliability of primary assay for routine SAR screening.
  • Screen analogs of the parent compound in the primary assay to identify compounds with improved potency and activity.
  • Perform a panel of secondary assays to confirm improvements in potency and activity.
  • Determine the efficacy of the optimized lead compound(s) in an in vivo model of the target disorder.

In addition, we encourage you to state clearly in your application which specific drug properties you hope to improve through a medicinal chemistry effort in addition to activity and potency (e.g., brain penetrance or metabolic stability). If the ADMET properties of your compound have not been characterized at the time of submission, a preliminary study of PK and toxicological properties will likely be conducted under network contracts early in the project. 

 My compounds show activity in models for more than one disorder. Can I include multiple indications in a single application?
Multiple disorders are discouraged. This is because the target patient population and intended use guides the design of the drug and the pre-clinical studies, such as toxicology and formulation. For example, a drug for stroke will have different properties than a drug for Alzheimers, even though the molecular target of drug action may be shared. You may discuss the future potential for your drug to be used in other indications, but you should select a specific disorder for initial development in the Network. In your application, you should include a completed Target Product Profile (TPP) specific to your target disorder. A template for the TPP can be found at: http://www.neuroscienceblueprint.nih.gov/bpdrugs/apply.htm.

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 Do I need to supply information related to human subjects in my application?
No. Clinical trials in the Network will be conducted under contract, outside of the U01.

 The RFA says “it is anticipated that applications will carry direct costs of up to $125,000 per year”—is this a cap? What if my estimated costs for the assays and animal efficacy studies are higher?
The $125K estimate is not a strict budget cap, but the amount of money available to fund U01s within the program is limited. The major benefits from this program are the multi-million dollar value of contract services and an accelerated path to commercialization of your discoveries. To fund as many applications as possible in the first year, we will consider budgets as well as scores while making the project selections. If you believe you have a strong scientific justification for exceeding $125K per year, you may do so, but you should make every effort to streamline your testing scheme and include only critical-path studies. Your budget may be renegotiated if your project is selected for funding.

 If I am a medicinal chemist, how would I participate in the program?
The design of the chemistry strategy will be a close collaboration between the U01 PI and the Lead Development Team consultants and contractors. In cases where an internal chemistry program is ongoing or envisioned by a U01 PI, entering a collaboration with the Blueprint Neurotherapeutics Network can increase the rate at which compounds are generated, provide additional seasoned drug development expertise, and broaden the focus of SAR to include druglike properties. In most cases, potential overlap with existing grant programs is not a concern due to the accelerated, clinically-directed design of this program. Please contact us if you would like to discuss how your particular project might fit into the Network.

 Can I include equipment in my budget?
Equipment requests are allowed, but not encouraged. Equipment requests should be considered only if the equipment is absolutely necessary to the success of the project and cannot be supported by any other means. This is likely to be a subject of negotiation before an award is made.

  Can I budget for travel?
Yes. One to two trips per year is a reasonable estimate. The Steering Committee will discuss each project once or twice per year, which may require your attendance to present and discuss the project progress. Other scientific meetings may be included in your budget, but we encourage you to consider alternative sources of funding for these because of the program’s budget constraints.

 Do I need to include Human Subjects information in my application?
No. Phase I clinical trials in this network will be conducted under a separate contract, so the U01 application does not need to address human subjects concerns.

 Who should I contact for more information?
The primary contact for this Network is Dr. Rebecca Farkas, NINDS Office of Translational Research, (301) 496-1779, farkasr@ninds.nih.gov

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