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ENZYME REPLACEMENT THERAPY OF THE BRAIN IN MUCOPOLYSACCHARIDOSIS TYPE II

Award Number: R44NS067707
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: 12/01/2011
PERIOD OF PERFORMANCE END DATE: 11/30/2014

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ENZYME REPLACEMENT THERAPY OF THE BRAIN IN MUCOPOLYSACCHARIDOSIS TYPE II - DESCRIPTION (provided by applicant): Type II Mucopolysaccharidosis (MPS), also known as Hunter's syndrome, is a genetic disease that affects the lysosomal enzyme, iduronate-2-sulfatase (IDS). Consequently, patients with MPS-II develop extensive lysosomal storage product accumulation in tissues, including the brain. Children born with MPS-II develop multiple brain disorders including mental retardation and hydrocephalus. The current therapy for MPS-II is Enzyme Replacement Therapy (ERT) with the recombinant human enzyme, IDS. However, ERT does not treat the brain of MPS-II, because IDS 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-II, which is a genetically engineered biopharmaceutical fusion protein. The IDS 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 IDS is fused to the heavy chain of the HIRMAb to create a new chemical entity, called the HIRMAb-IDS fusion protein. Phase I studies show the HIRMAb-IDS 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 IDS enzyme activity. The proposed phase II work will develop a manufacturing plan that can be replicated for future GMP manufacturing. The AGT-182 produced in phase II will be evaluated for safety, toxicology and pharmacokinetics in 8 Rhesus monkeys. The completion of this work will enable entry of the AGT-182 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-II.


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: 2013 ( Subtotal = $646,237 )
2013	2013	ARMAGEN TECHNOLOGIES INC	914 COLORADO BOULEVARD	LOS ANGELES	CA	90401		USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	12/19/2012	NON-COMPETING CONTINUATION	$646,237
														Subtotal = $646,237

Issue Date FY: 2012 ( Subtotal = $406,198 )
2012	2012	ARMAGEN TECHNOLOGIES INC	914 COLORADO BOULEVARD	LOS ANGELES	CA	90401		USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	11/17/2011	COMPETING CONTINUATION	$406,198
														Subtotal = $406,198

Grand Total All Awards = $1,052,435

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ENZYME REPLACEMENT THERAPY OF THE BRAIN IN MUCOPOLYSACCHARIDOSIS TYPE II

Award Number: R44NS067707

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: 12/01/2011

PERIOD OF PERFORMANCE END DATE: 11/30/2014

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