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Sulfamidase Brain Drug Delivery in Sanfilippo Syndrome

Award Number: R44NS088868
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 07/01/2014
PERIOD OF PERFORMANCE END DATE: 03/31/2017

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Sulfamidase Brain Drug Delivery in Sanfilippo Syndrome - ¿ DESCRIPTION (provided by applicant): Type IIIA Mucopolysaccharidosis (MPS), also known as Sanfilippo A syndrome, is a genetic disease that affects the lysosomal enzyme, N-sulfoglucosamine sulfohydrolase (SGSH), also called sulfamidase. Consequently, patients with MPS-IIIA develop extensive lysosomal storage product accumulation in the brain. Children born with MPS- IIIA develop multiple brain disorders including mental retardation and hydrocephalus. The current therapy for lysosomal storage disorders is Enzyme Replacement Therapy (ERT) with the recombinant human enzyme. However, ERT will not treat the brain of MPS- IIIA, because SGSH does not cross the blood-brain barrier (BBB). The present work will continue work on the development of a new treatment of the brain of MPS- IIIA, which is a genetically engineered biopharmaceutical fusion protein named AGT-184. The SGSH is genetically fused to a BBB molecular Trojan horse, which is a genetically engineered peptidomimetic monoclonal antibody (MAb) against an endogenous BBB peptide receptor, the human insulin receptor (HIR). The human SGSH is fused to the heavy chain of the HIRMAb to create a new chemical entity, called the HIRMAb- SGSH fusion protein. Phase I studies show the HIRMAb-SGSH fusion protein could be engineered and expressed by stably transfected host cells. The fusion protein retained high affinity binding to the HIR and retained high SGSH enzyme activity. The proposed phase II work will develop a manufacturing plan that can be replicated for future GMP manufacturing. The AGT-184 produced in phase II will be evaluated for safety, toxicology and pharmacokinetics in a Rhesus monkey dose-finding study. The completion of this work will enable entry of the AGT-184 drug development program into GLP toxicology and GMP manufacturing required for submission of an IND to begin treatment of the brain in patients with MPS- IIIA.


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: 2017 ( Subtotal = $0 )
2017	2016	ARMAGEN INC.	26679 AGOURA RD STE 100	CALABASAS	CA	91302	LOS ANGELES	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	4/18/2017	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2016 ( Subtotal = $612,428 )
2016	2016	ARMAGEN TECHNOLOGIES INC	914 COLORADO BOULEVARD	LOS ANGELES	CA	90401		USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	4/7/2016	NON-COMPETING CONTINUATION	$612,428
														Subtotal = $612,428

Issue Date FY: 2015 ( Subtotal = $421,005 )
2015	2015	ARMAGEN TECHNOLOGIES INC	914 COLORADO BOULEVARD	LOS ANGELES	CA	90401		USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	3/11/2015	COMPETING CONTINUATION	$421,005
														Subtotal = $421,005

Grand Total All Awards = $1,033,433

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Sulfamidase Brain Drug Delivery in Sanfilippo Syndrome

Award Number: R44NS088868

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 07/01/2014

PERIOD OF PERFORMANCE END DATE: 03/31/2017

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