Notice Number: NOT-TR-13-007

Key Dates
Release Date: July 22, 2013
Letter of Intent/Pre-Application Call Due: No later than September 16, 2013
Final Proposal Submission Date: September 30, 2013

Issued by
National Center for Advancing Translational Sciences (NCATS)

Purpose

The National Center for Advancing Translational Sciences (NCATS), Division of Preclinical Innovation (DPI) is announcing an open opportunity to submit proposals to collaborate with the Therapeutics for Rare and Neglected Diseases (TRND) program.

The TRND program performs preclinical and early clinical development of new drugs for rare and neglected tropical diseases, and develops new technologies and paradigms to improve the efficiency of therapeutic development for these diseases. The operational model of TRND is collaboration between intramural NCATS drug development scientists and partners having promising leads and disease/target knowledge, but lacking the expertise or resources to develop these projects into clinical stage programs attractive to biopharmaceutical or other suitable organizations. TRND is establishing collaborations with researchers in the public and private sectors to together de-risk rare and neglected tropical disease drug development projects by accomplishing lead optimization through an Investigational New Drug (IND) application with the Food and Drug Administration (FDA), and in limited circumstances, proof-of-concept human studies.

This Notice is being provided to allow potential applicants sufficient time to contact the Solicitation Coordinator to discuss development of meaningful collaborations and responsive proposals. TRND utilizes a Letter of Intent (LOI)/pre-application teleconference process.

This announcement for collaboration will not use NIH activity codes. Details of the opportunity are below.

Research Initiative Details

TRND collaborative projects will be initiated at various stages of preclinical development, but no earlier than optimization of well-characterized leads, and no later than a new molecular entity (NME), new biologic entity (NBE), or repurposed drug in need of IND-enabling studies. Projects of interest will be at one of the following stages:

• Lead Optimization: Lead Op aims to identify and develop a potent, specific development candidate from among the preliminary series through initial medicinal chemistry. These early-stage candidates must include clear structure-activity relationships (SAR) in at least two structurally distinct chemical series or a well-defined biological lead; reproducible activity in primary and orthogonal assays; efficacy in an accepted animal model (or when not available, cellular model) of the disease; initial indications of favorable Absorption, Distribution, Metabolism, and Excretion (ADME) properties; and favorable head-to-head comparisons versus the prior art.

• New Molecular / Biologic Entity (NME / NBE): This will represent an advanced lead molecule, requiring completion of IND-enabling studies. At minimum, the selected candidate molecule will include clear efficacy data, good drug metabolism and pharmacokinetic (DMPK) properties, and initial non-GLP safety studies demonstrating absence of gross toxicities. Development candidates may require completion of IND-enabling pharmacokinetic / pharmacodynamic / toxicology / formulation studies.

• Repurposing: A repurposing candidate represents a drug previously approved by FDA for another indication, which has been shown to have efficacy in an animal model (or when not available, cellular model) of a rare or neglected tropical disease. Relying heavily on the previous indication data package, a repurposing candidate will be more advanced, in need of formulation, dose-finding, disease-specific toxicology, or other studies to allow clinical testing to commence.

• Platform Technology: These programs will seek development support for a therapeutic candidate directed toward a specific rare or neglected tropical disease indication, but will represent a technology platform with the potential to address a wider range of additional disorders and that can enable more efficient future development of other therapeutics. Both early- and later-stage projects will be considered, as described above.

The TRND program will partner with academic laboratories, not-for-profit organizations, and for-profit companies. Foreign organizations may also apply. TRND uses the online proposal management website, ProposalCentral (https://proposalcentral.altum.com/), to facilitate LOI and Application submission. Applicants must contact the TRND Solicitation Coordinator to complete the LOI and Application submission process. Submitted applications will be reviewed for scientific merit and technical feasibility, as well as program balance and availability of resources. Successful applicants will collaborate with TRND to create and implement a therapeutic development project plan. Note that this is not a grant or contract program. No extramural funding will be provided.

For more information about the TRND program, please visit the TRND website at http://www.ncats.nih.gov/trnd.html.

To ensure sufficient time to develop meaningful proposals, the LOI/discussion process must be completed by no later than September 16, 2013. The application deadline for the current cycle of the program is September 30, 2013.

Inquiries

Please direct all inquiries to:

Andre M. Pilon, PhD
Solicitation Coordinator
Therapeutics for Rare and Neglected Diseases (TRND)
Division of Preclinical Innovation (DPI)
National Center for Advancing Translational Sciences (NCATS)
National Institutes of Health (NIH)
Email: TRND@mail.nih.gov
http://www.ncats.nih.gov/trnd.html