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Commercialization Readiness Pilot (CRP) to maximize vamorolone international labeling and sales

Award Number: SB1NS095423
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: 02/15/2016
PERIOD OF PERFORMANCE END DATE: 08/31/2022

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Commercialization Readiness Pilot (CRP) to maximize vamorolone international labeling and sales - Abstract Vamorolone is a first-in-class dissociative steroidal drug that is being developed for Duchenne muscular dystrophy as a replacement for corticosteroid standard of care (prednisone, deflazacort) by small business ReveraGen BioPharma. ReveraGen completed first-in-patient studies with the aid of a NINDS SBIR Direct-to- Phase II award (48 DMD boys; sequential clinical trials VBP15-002, VBP15-003, VBP15-LTE, Expanded Access Protocol), and these have been published (Conklin et al. 2018; Hoffman et al. 2019). These studies showed dose-responsive improvements in all measures of motor skills, and loss of side effects (no stunting of growth, less Cushingoid and weight gain). Biomarkers have been integrated throughout the vamorolone program, providing objective readouts of drug efficacy and safety that also showed clear dose-response in these initial open label studies. A double-blind pivotal trial of 120 DMD boys (VBP15-004) is currently 40% enrolled and recruiting at 31 sites in 11 countries. This pivotal study is supported in part by a NINDS SBIR Phase IIB award and European Commission Horizons 2020 award, and includes placebo and prednisone arms. It is anticipated that the trial will be fully enrolled in late 2019 or early 2020, and last-patient last visit anticipated in Q3 2020 leading to initiation regulatory drug approval procedures in both US (FDA NDA) and Europe (EMA MAA). ReveraGen has remained a privately held small business, and this has enabled innovations in clinical trial design, use of biomarkers, and regulatory protocols. For example, ReveraGen requested the FDA consider Expanded Access Protocols for patients leaving vamorolone clinical trials, as this would reduce burden on patient families and clinical sites, while maintaining centralized safety information, and this was approved, leading to a FDA press release encouraging others to consider a similar approach. For this Commercialization Readiness Pilot (CRP) application, ReveraGen seeks support to fill key gaps identified through a formal FDA NDA gap analysis carried out in Q3 2019, and to enable worldwide drug approval thus maximizing thus enabling worldwide drug approval and maximizing market access. Technical assistance request is to both develop a global regulatory strategy, and development of a global intellectual property strategy. Support for late stage research and development activities include filling identified gaps is Design and Quality Systems Controls, and conducting a GLP juvenile toxicity study supporting labeling and prescription to the newborn period. Completing the goals of the proposed CRP would lead to worldwide commercialization of vamorolone, and set the stage for expanding vamorolone use in many other clinical disorders were corticosteroids are effective but the severe burden of side effects limits use.


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: 2023 ( Subtotal = $0 )
2023	2021	REVERAGEN BIOPHARMA, INC.	155 GIBBS ST	ROCKVILLE	MD	20850	MONTGOMERY	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	6	8/25/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2020	REVERAGEN BIOPHARMA, INC.	155 GIBBS ST STE 433	ROCKVILLE	MD	20850	MONTGOMERY	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	5	9/7/2022	COMPETING CONTINUATION	-$1
2022	2020	REVERAGEN BIOPHARMA, INC.	155 GIBBS ST STE 433	ROCKVILLE	MD	20850	MONTGOMERY	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	5	8/29/2022	COMPETING CONTINUATION	$1
														Subtotal = $0

Issue Date FY: 2021 ( Subtotal = $1,672,364 )
2021	2021	REVERAGEN BIOPHARMA, INC.	155 GIBBS ST STE 433	ROCKVILLE	MD	20850	MONTGOMERY	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	6	9/1/2021	NON-COMPETING CONTINUATION	$1,672,364
2021	2021	REVERAGEN BIOPHARMA, INC.	155 GIBBS ST STE 433	ROCKVILLE	MD	20850	MONTGOMERY	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	6	9/20/2021	NON-COMPETING CONTINUATION	$0
														Subtotal = $1,672,364

Issue Date FY: 2020 ( Subtotal = $1,680,179 )
2020	2020	REVERAGEN BIOPHARMA, INC.	155 GIBBS ST STE 433	ROCKVILLE	MD	20850	MONTGOMERY	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	5	6/22/2020	COMPETING CONTINUATION	$1,680,179
														Subtotal = $1,680,179

Grand Total All Awards = $3,352,543

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Commercialization Readiness Pilot (CRP) to maximize vamorolone international labeling and sales

Award Number: SB1NS095423

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: 02/15/2016

PERIOD OF PERFORMANCE END DATE: 08/31/2022

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