Official websites use .gov

A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS

A lock ( 🔒 ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

Need Help

Recombinant botulinum toxin with improved safety and effectiveness for treatment of dystonia

Award Number: R43NS122661
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: 03/01/2022
PERIOD OF PERFORMANCE END DATE: 08/31/2023

Group Awards By:

View Award Description

Recombinant botulinum toxin with improved safety and effectiveness for treatment of dystonia - Abstract Botulinum neurotoxin (BoNT) is the most toxic protein known to science. It is also the active ingredient in ® pharmaceutical products like Botox (Allergan), which have proven safe and effective for multiple neuromuscular indications. All current BoNT products are manufactured from Clostridium botulinum cultures and have similar safety characteristics, which include limitations on dosing and a black box warning in labeling. Because of its inherent toxicity and narrow therapeutic window, optimal clinical outcomes are difficult to achieve in indications involving large muscle groups, such as cervical dystonia, post-stroke spasticity and pediatric cerebral palsy. Though improved muscle tone can clearly be achieved, clinicians tend to dose on the side of caution, and overall clinical outcomes leave significant room for improvement. We here propose building on data generated with a previous candidate for a “safer BoNT” pharmaceutical (SBIR Phase 1 1R41NS086115-01A1) which found that specific amino acid substitutions to BoNT/A1 can produce a recombinant BoNT/A1 with an improved safety margin compared to wt BoNT/A1, (US 8,865,186). The initial lead candidate designed and tested, Cyto-012, had an improved safety margin, but its absolute potency was such that doses required for efficacy were also immunogenic. Here, we propose testing a new recombinant BoNT/A1 derivative termed Cyto-014, designed to have potency and immunogenicity similar to wt BoNT/A1, while retaining the desired improvement in safety suggested by our Preliminary Studies. This proposal is intended to compare the dose-response behavior, safety margin and maximum tolerated dose of Cyto-014 relative to wt BoNT/A1 in appropriately powered studies, and to evaluate its immunogenicity and effectiveness for repeat treatment. Successful completion of the proposed studies will support a Phase II study to focus on pre-clinical development activities aimed at filing an IND.


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: 2024 ( Subtotal = $0 )
2024	2023	CYTODEL INC	423 W 127TH ST	NEW YORK	NY	10027	NEW YORK	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	3/28/2024	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2023 ( Subtotal = $138,307 )
2023	2023	CYTODEL INC	423 W 127TH ST	NEW YORK	NY	10027	NEW YORK	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	2/21/2023	NON-COMPETING CONTINUATION	$138,307
														Subtotal = $138,307

Issue Date FY: 2022 ( Subtotal = $276,617 )
2022	2022	CYTODEL, INC.	423 W 127TH ST	NEW YORK	NY	10027	NEW YORK	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	3/1/2022	NEW	$276,617
														Subtotal = $276,617

Grand Total All Awards = $414,924

Top

All Categories

About

Search

Reports

Data Submission

Award Information

Recombinant botulinum toxin with improved safety and effectiveness for treatment of dystonia

Award Number: R43NS122661

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: 03/01/2022

PERIOD OF PERFORMANCE END DATE: 08/31/2023

Federal Websites

Department of Health & Human Services

HHS Operating Divisions

HHS Staff Divisions

Download A Document Viewer