Friday, December 5, 2025 12/5/2025

Small molecules combination therapy using polypharmacology approach as a novel treatment paradigm for rare bone disease

Award Number: R43TR004574
ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/10/2023
PERIOD OF PERFORMANCE END DATE: 07/31/2025

Group Awards By:

View Award Description

Small molecules combination therapy using polypharmacology approach as a novel treatment paradigm for rare bone disease - There is a critical need to improve drug development strategies for rare diseases, as more than 7000 rare and neglected diseases currently have no treatments available. At the same time, as new drug therapies remain costly and time-consuming, repositioning existing drugs and drug candidates offers an alternative approach to developing therapeutics for rare diseases. Nostopharma seeks to address this need by developing repurposed drug combinations as a promising approach to achieve a synergistic therapeutic effect, dose, and toxicity reduction. Our Phase I objectives are to demonstrate the feasibility of repurposing a small molecule combination therapy, formulated in a proprietary way, to treat a rare genetic disease- Progressive osseous heteroplasia (POH). POH is a rare disease with no effective drug-based therapy, where bone tissue forms in the extraskeletal soft tissue in response to inactivating mutations in the GNAS (the stimulatory alpha subunit of a guanine nucleotide-binding protein) gene locus. Hedgehog signaling (Hh) has a seminal role in mesenchymal progenitor fate choice and inappropriate differentiation into osteoblasts and ectopic bone formation in soft tissues. Nostopharma will demonstrate the feasibility towards altering the aberrant mesenchymal progenitor cells differentiation utilizing combinations of Hedgehog pathway inhibitors. Our approach simultaneously targets the distinctive components of the Hh pathway to allow synergetic inhibition, which can significantly reduce effective doses and potential side effects and overcome the drug resistance factor that frequently hinders the success of monotherapies. This proof of concept will involve phenotypic screening of drug combinations and in-vivo efficacy in the POH mouse model.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = -$1 )
2026	2023	NOSTOPHARMA LLC	5 GREENLANE CT	POTOMAC	MD	20854	MONTGOMERY	USA	National Center for Advancing Translational Sciences	000	1	11/18/2025	NEW	-$1
														Subtotal = -$1

Issue Date FY: 2024 ( Subtotal = $44,394 )
2024	2024	NOSTOPHARMA LLC	7600 CODDLE HARBOR LN	POTOMAC	MD	20854	MONTGOMERY	USA	National Center for Advancing Translational Sciences	000	1	3/1/2024	SUPPLEMENT FOR EXPANSION	$44,394
														Subtotal = $44,394

Issue Date FY: 2023 ( Subtotal = $318,796 )
2023	2023	NOSTOPHARMA LLC	7600 CODDLE HARBOR LN	POTOMAC	MD	20854	MONTGOMERY	USA	National Center for Advancing Translational Sciences	000	1	8/10/2023	NEW	$318,796
														Subtotal = $318,796

Grand Total All Awards = $363,189

Top

All Categories

About

Search

Reports

Data Submission

Award Information

Small molecules combination therapy using polypharmacology approach as a novel treatment paradigm for rare bone disease

Award Number: R43TR004574

ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 08/10/2023

PERIOD OF PERFORMANCE END DATE: 07/31/2025

Federal Websites

Department of Health & Human Services

HHS Operating Divisions

HHS Staff Divisions

Download A Document Viewer