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Tolerability of SRX246 in Huntington's Disease Patients

Award Number: U44NS090616
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2015
PERIOD OF PERFORMANCE END DATE: 08/31/2019

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Tolerability of SRX246 in Huntington's Disease Patients - DESCRIPTION (provided by applicant): Huntington's Disease (HD) is an inherited disease that results from expansion of a trinucleotide (CAG, cytosine/adenine/guanine) repeat that encodes a polyglutamine tract in the huntingtin protein. Psychiatric symptoms, including irritability and aggression, are common in HD patients. These are among the most distressing aspects of the disease. They have adverse effects on daily life and often result in institutionalization. Despite the frequent occurrence and severe consequences of irritability and aggression in HD, these symptoms have received little attention to date. Effective treatments are lacking and well-validated scales for measuring changes in these symptoms are not available. Faced with a significant unmet need, neurologists cannot currently determine whether new drug therapies might be useful in treating neuropsychiatric symptoms in HD. The Phase II clinical trial we propose in HD patients (n=108)," A randomized, placebo controlled, double blind, multi-center study to assess the tolerability of SRX246 in irritable/aggressive subjects wit Huntington's Disease (HD)," will allow us to rigorously evaluate the tolerability of a potential ne drug for the treatment of irritability and aggression. It will also provide additional safety data n the compound and explore various rating scales for the assessment of changes in these symptoms. Thus, we will obtain critical data that can be used to plan future Phase II or III clinicl trials of drugs that might blunt irritability and aggression in HD. The compound that we propose to test is SRX246, a first-in-class vasopressin 1a (V1a) receptor antagonist. SRX246 crosses the blood-brain barrier following oral administration, exhibits high affinity and selectivity for is target receptor, has a strong safety profile, is well-tolerated in healthy volunteers, and has excellent pharmacokinetics. Extensive preclinical pharmacology studies and an experimental medicine fMRI study in healthy volunteers have shown that SRX246 has CNS effects after oral administration and that it modulates brain circuits involved in responses to stimuli that elicit aggression/fear. These findings strongly suggest that SRX246 might have a beneficial effect on the irritability and aggression seen in a sizable proportion of HD patients. The proposed project will generate data needed to plan a future clinical trial that can rigorously test SRX246 for efficacy as a treatment for irritability and aggression.


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: 2022 ( Subtotal = $1 )
2022	2018	AZEVAN PHARMACEUTICALS INC	116 RESEARCH DR	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	4	9/26/2022	NON-COMPETING CONTINUATION	$1
														Subtotal = $1

Issue Date FY: 2020 ( Subtotal = -$1 )
2020	2018	AZEVAN PHARMACEUTICALS INC	116 RESEARCH DR	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	4	4/1/2020	NON-COMPETING CONTINUATION	-$1
														Subtotal = -$1

Issue Date FY: 2018 ( Subtotal = $764,163 )
2018	2018	AZEVAN PHARMACEUTICALS INC	116 RESEARCH DR	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	4	8/27/2018	NON-COMPETING CONTINUATION	$0
2018	2018	AZEVAN PHARMACEUTICALS INC	116 RESEARCH DR	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	4	2/1/2018	NON-COMPETING CONTINUATION	$764,163
														Subtotal = $764,163

Issue Date FY: 2017 ( Subtotal = $1,541,407 )
2017	2017	AZEVAN PHARMACEUTICALS INC	116 RESEARCH DR	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	002	3	6/22/2017	NON-COMPETING CONTINUATION	$148,018
2017	2017	AZEVAN PHARMACEUTICALS INC	116 RESEARCH DR	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	2/8/2017	NON-COMPETING CONTINUATION	$1,332,155
2017	2017	AZEVAN PHARMACEUTICALS INC	116 RESEARCH DR	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	3	2/27/2017	SUPPLEMENT FOR EXPANSION	$61,234
														Subtotal = $1,541,407

Issue Date FY: 2016 ( Subtotal = $2,214,354 )
2016	2016	AZEVVAN PHARMACEUTICALS INC	111 RESEARCH DRIVE	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	3/21/2016	EXTENSION WITH OR WITHOUT FUNDS	$2,214,354
														Subtotal = $2,214,354

Issue Date FY: 2015 ( Subtotal = $1,115,185 )
2015	2015	AZEVVAN PHARMACEUTICALS INC	111 RESEARCH DRIVE	BETHLEHEM	PA	18015	NORTHAMPTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	8/10/2015	NEW	$1,115,185
														Subtotal = $1,115,185

Grand Total All Awards = $5,635,109

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Tolerability of SRX246 in Huntington's Disease Patients

Award Number: U44NS090616

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

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

PERIOD OF PERFORMANCE START DATE: 09/01/2015

PERIOD OF PERFORMANCE END DATE: 08/31/2019

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