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Personalized dosing of dichloroacetate for the treatment of rare and common diseases

Award Number: R42HD089804
ORGANIZATION: NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 07/01/2017
PERIOD OF PERFORMANCE END DATE: 12/31/2024

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Personalized dosing of dichloroacetate for the treatment of rare and common diseases - Project Abstract/Summary Pyruvate dehydrogenase complex (PDC) deficiency (PDCD) is a rare disease of mitochondrial energy failure in which the life expectancy of affected children is severely truncated from unrelenting lactic acidosis and/or from progressive neurological and neuromuscular degeneration. Treatment of PDCD remains a serious, unmet, challenge. Dichloroacetate (DCA) represents the first targeted therapy for PDCD by stimulating residual PDC activity in all tissues, including the central nervous system. Based on both controlled trials and open label studies of DCA in mitochondrial diseases, Medosome Biotec, LLC (MBT) and its research partner at the University of Florida (Dr. P. Stacpoole) determined the data were sufficiently compelling to justify a pivotal FDA Phase III (FDA-P3) trial of DCA in this disease. The primary goal of this STTR Phase II B grant proposal is to complete our FDA Phase III trial to support a future New Drug Application (NDA) submission to FDA and marketing approval of DCA for the treatment of PDCD by accomplishing the following Specific Aims: Aim 1: Manufacture, test, and release three additional cGMP batches of DCA. These batches can be used to support the FDA-P3 clinical trial, including the open-label continuation, while also generating the required registration stability program for a future NDA submission to FDA. Aim 2: Complete the pivotal FDA-P3 clinical trial. Milestones include: 1) complete subject recruitment and enrollment within 12 months of receipt of the award; 2) complete the 9 month double blind crossover treatment phase by month 24 of the award; and 3) complete statistical analysis of the double blind data by month 30 of the award. Aim 3: Prepare for NDA submission and commercialization of DCA for PDCD. Milestones include: 1) FDA meeting request in advance of NDA filing (pre- NDA meeting); 2) Secure agreements for cGMP production of DCA; 3) Evaluate commercial production batch size requirements and implement scale up as required; 4) Conduct manufacturing process validation of DCA; 5) Complete pivotal registration stability program; 6) Compile and integrate historical and FDA-P3 safety data. Aim 4: Develop a validated clinical assay for monitoring DCA levels in patients receiving DCA.


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: 2025 ( Subtotal = $0 )
2025	2022	MEDOSOME BIOTEC LLC	3416 S ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	000	6	5/5/2025	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2024 ( Subtotal = $0 )
2024	2022	MEDOSOME BIOTEC LLC	3416 S ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	000	6	8/5/2024	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2022 ( Subtotal = $985,330 )
2022	2022	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	000	6	7/7/2022	NON-COMPETING CONTINUATION	$985,330
														Subtotal = $985,330

Issue Date FY: 2021 ( Subtotal = $992,757 )
2021	2021	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	001	5	8/18/2021	NON-COMPETING CONTINUATION	$992,757
2021	2018	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	000	3	6/14/2021	NON-COMPETING CONTINUATION	$0
														Subtotal = $992,757

Issue Date FY: 2020 ( Subtotal = $1,000,000 )
2020	2020	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	001	4	7/13/2020	COMPETING CONTINUATION	$1,000,000
2020	2018	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	000	3	7/13/2020	NON-COMPETING CONTINUATION	$0
														Subtotal = $1,000,000

Issue Date FY: 2018 ( Subtotal = $695,065 )
2018	2018	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	000	3	9/10/2018	NON-COMPETING CONTINUATION	$695,065
														Subtotal = $695,065

Issue Date FY: 2017 ( Subtotal = $793,897 )
2017	2017	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	001	2	6/28/2017	EXTENSION WITH OR WITHOUT FUNDS	$793,897
2017	2016	MEDOSOME BIOTEC LLC	3416 ASHWOOD DR	BLOOMINGTON	IN	47401	MONROE	USA	Child Health and Human Development Extramural Research	000	1	6/23/2017	NEW	$0
														Subtotal = $793,897

Issue Date FY: 2016 ( Subtotal = $224,978 )
2016	2016	Medosome Biotec, LLC	12085 Research Drive	ALACHUA	FL	32615	ALACHUA	USA	Child Health and Human Development Extramural Research	000	1	9/13/2016	NEW	$224,978
														Subtotal = $224,978

Grand Total All Awards = $4,692,027

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Personalized dosing of dichloroacetate for the treatment of rare and common diseases

Award Number: R42HD089804

ORGANIZATION: NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 07/01/2017

PERIOD OF PERFORMANCE END DATE: 12/31/2024

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