Background & Aims

The HEAL Pain Therapeutics Development Program (PTDP) (RFA-NS-24-019) is part of the Helping to End Addiction Long-term® Initiative, or NIH HEAL Initiative®, a trans-NIH research effort focused on improving prevention and treatment for opioid misuse and addiction and enhancing pain management. The NIH HEAL Initiative® consists of six research focus areas, of which the PTDP falls into Preclinical and Translational Research in Pain Management. PTDP is part of a suite of funding opportunities that support the development of safe, effective, and non-addictive small molecule, biologic, and combination product therapeutics to treat pain. HEAL Initiative: Studies to Enable Analgesic Discovery (R61/R33) serves as a feeder program to PTDP, and PTDP projects graduate to EPICC-Net or Phase II opportunities once all milestones have been met.

Methods

The HEAL-PTDP focuses on facilitating drug discovery and development in academic and small business settings to allow riskier, high-reward projects an opportunity to test the feasibility of their candidate non-addictive pain therapeutics. Funded investigators work together with the NIH to build a lead development team (LDT) that leverages drug discovery and development expertise to advance their basic research into human testing. The program provides access to senior, ex-biopharma consultants, as well as CRO resources including medicinal chemistry, manufacturing and formulation, DMPK, GLP toxicology, and Phase I clinical testing. Entering applications can be at the Discovery or Development stage. Grants are milestone-driven and progress against milestones is evaluated on an annual basis for a maximum 5-year grant period. End goals include IND submission, completion of a Phase I clinical trial, and formation of partnerships to progress candidate therapeutic through clinical testing.

Results

HEAL-PTDP has awarded a total of 17 cooperative agreements from both academia and small business partners. These awards cover several pain conditions encompassing neuropathic pain, chronic pain, and sickle cell disease pain, as well as others. Modalities of awarded projects including small molecules, biologics, formulations, and combination products. These cooperative drug development efforts have resulted in product licensing to pharmaceutical companies; with two to three Investigational New Drug (IND) application submissions to the United States FDA expected in 2024.

Conclusions

The HEAL-PTDP funds a wide variety of non-addictive pain therapeutic development research efforts by both academia and small business, spanning a range of pain conditions, therapeutic modalities, and treatment targets. The program and its funded investigators work together through NIH cooperative agreement mechanisms to build a team that leverages therapeutic development expertise and CRO resources to advance Discovery and Development stage research projects into human testing.

References

The Helping to End Addiction Long-term® Initiative, NIH HEAL Initiative®, accessed on 26 January 2024, https://heal.nih.gov

Preclinical and Translational Research in Pain Management, NIH HEAL Initiative®, accessed on 26 January 2024, https://heal.nih.gov/research/preclinical-translational

Pain Therapeutics Development Program (PTDP), NIH HEAL Initiative®, accessed on 26 January 2024, https://www.ninds.nih.gov/current-research/trans-agency-activities/ninds-role-heal-initiative/pain-therapeutics-development-program-ptdp

RFA-NS-24-019: HEAL Initiative: Non-addictive Analgesic Therapeutics Development [Small Molecules and Biologics] to Treat Pain (UG3/UH3 Clinical Trial Optional), HHS / NIH, accessed on 26 January 2024, https://grants.nih.gov/grants/guide/rfa-files/RFA-NS-24-019.html

Presenting Author

Matthew Rice

Poster Authors

Matthew Rice

PhD

NIH / NINDS

Lead Author

Carolyn Bondar

PhD

NIH / NINDS

Lead Author

Jim Pomonis

PhD

NIH / NINDS

Lead Author

Mary Ann Pelleymounter

PhD

NIH / NINDS

Lead Author

Topics

  • Treatment/Management: Pharmacology: Non-opioid