The Body of the Application should include the following: (a) Project Description, (b) Budget and Justification, (c) Biographical Information, and (d) Compound Report Card. These four sections should be compiled into a single PDF and uploaded where indicated in the full proposal section of the ADDF Funding Portal.


Project Description

The project description is the central part of the proposal should contain the eight sub-sections listed below (indicate each sub-section by number in the proposal). Sub-sections 1-6 should not exceed 15 pages of written text. Embed figures in the text if possible. Use at least 11pt. font and 1" margins.

1. Background and Rationale

  • Provide evidence that links the biological target or pathway to the disease.
  • Discuss the novelty of the proposed approach and its expected impact on FTD disorders. Discuss related programs in the field; if any are known, please explain the advantages of your program.
  • Discuss potential mechanism-related side effects and off-target activities linked to modulating the target.
  • Discuss the rationale for targeting the proposed study population based on the mechanism of action for the drug candidate.
 

2. Supporting Data

  • Provide relevant supporting data.
  • Complete the Compound Report Card (xls) for the candidate molecule. Insert the form at the end of the PDF.

All information provided is kept under strict confidentiality.

 

3. Project Plan and Objectives

  • Objectives: List specific aims and milestones with clearly defined go/no-go decision points for advancement of the project.
  • Timeline: Provide a schedule for the completion of the proposed milestones/deliverables for each quarter of the year for each year of funding (this can be in table or Gantt chart format).
  • Discuss potential pitfalls of the program with sufficient risk assessment and criteria to substantiate continuation of the program at each milestone.
  • Discuss critical next experiments in order to advance the program to attract additional funding/licensing.
 

4. Experimental Design and Methods

Provide details on the study design and methods. Provide responses to all of the questions below for this section. Indicate your answer to each question by referencing the question number, for example: (Q1) or (Q2).

  1. Clearly define the clinical population and include detailed inclusion/exclusion criteria.
  2. Provide justification for the proposed clinical population based on the mechanism for the therapeutic agent.
  3. Discuss the inclusion of control groups and/or placebo arms.
  4. Provide justification for the treatment duration and dosing.
  5. What are your primary and secondary outcome measures? Have the biomarkers been validated to detect changes in defined patient population within the specified treatment time of the proposed trial?
  6. What is the pharmacodynamic readout of target engagement?
  7. Provide a statistical analysis plan. Include a power analysis to justify the number of subjects per group. Has your power analysis accounted for previously observed variability in your outcome measures?
  8. How will dropouts be reported and managed in the statistical analysis?
  9. Is this a single or multi-site study? If the study includes more than one site, what strategies will be used to reduce variability across different trial sites?
  10. Have you or your collaborators previously recruited the proposed patient population?
  11. What strategies will be used to promote recruitment and complete enrollment in a timely manner? List the channels (i.e. media, public events, community physicians' offices, etc.) that will be used to reach the targeted patient population. Describe the types of materials that will be developed as part of the recruitment toolkit.
  12. How many other academic or industry-sponsored trials in the same patient population as the proposed study are ongoing at your trial site(s) or within the same geographical area?
 

5. Description of Drug Discovery Team and Resources

  • Describe the investigative team, showing that the required expertise and resources are in place to complete the study objectives.
  • Where internal expertise is not available, include a description of external partners (e.g. consultants, contract research organizations (CROs)) that will help to execute the experimental work. Visit ADDF ACCESS to work directly with a concierge who can help you obtain competitive quotes from quality vendors. (Please provide competitive quotes from more than one vendor where possible. Quotes should be uploaded in the Appendix Materials).
  • Discuss the inclusion of any consultants with drug development or clinical expertise that were involved in the design of clinical studies or the development of the commercialization plan.
 

6. Intellectual Property (IP)

  • Provide information on existing IP and stage of prosecution (e.g. use, composition of matter). If no IP currently exists, describe the projected plan to generate IP; note if you expect the project to generate new IP.
  • Indicate any freedom to operate issues.
  • Include a brief discussion on future directions and eventual path towards commercialization.
 

7. Other Support

List other financial support, awarded and pending, and include grant title, principal investigator, percent effort of investigator, granting agency, amount, and projected funding period. Indicate any overlap between the aims or investigator effort from other funding with the proposed work.

 

8. References


Budget and Justification

The budget form (doc) can be downloaded here. Complete the budget template and provide a brief justification for each line item. Please review permissible costs here.


Biographical Information

Include a biosketch for each of the key personnel involved in the project. Existing NIH biosketch forms or other formats are accepted.


Study Population Worksheet

Complete the Compound Report Card (xls) for therapeutic agents.