A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Effect of Inhaled SNSP113 in Adult Subjects with Cystic Fibrosis

Start Date19 Nov 2019

Sponsor / Collaborator

Anagram Therapeutics, Inc.

NCT03309358 / TerminatedPhase 1

A Multiple-Site, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose (SAD) Study to Assess the Safety and Tolerability of Inhaled SNSP113 in Healthy Male Subjects (Part A) and Subjects With Stable Cystic Fibrosis (Part B)

Although Cystic Fibrosis is a complex genetic disease affecting many organs, lung disease is the primary cause of mortality. The objective of this study is to determine the safety and tolerability of SNSP113 in healthy subjects and subjects with stable cystic fibrosis.

Start Date28 Sep 2017

Sponsor / Collaborator

Anagram Therapeutics, Inc.

100 Clinical Results associated with SNSP-113

100 Translational Medicine associated with SNSP-113

100 Patents (Medical) associated with SNSP-113

Literatures (Medical) associated with SNSP-113

01 Nov 2023·Journal of Cystic Fibrosis

SNSP113 (PAAG) improves mucociliary transport and lung pathology in the Scnn1b-Tg murine model of CF lung disease

Article

Author: Baker, Shenda M ; Novak, Lea ; Birket, Susan E ; Rowe, Steven M ; Lindgren, Natalie R ; Harris, Elex S ; Fernandez-Petty, Courtney M

BACKGROUNDMucus stasis, a hallmark of muco-obstructive disease, results from impaired mucociliary transport and leads to lung function decline and chronic infection. Although therapeutics that target mucus stasis in the airway, such as hypertonic saline or rhDNAse, show some therapeutic benefit, they do not address the underlying electrostatic defect apparent in mucins in CF and related conditions. We have previously shown poly (acetyl, arginyl) glucosamine (PAAG, developed as SNSP113), a soluble, cationic polymer, significantly improves mucociliary transport in a rat model of CF by normalizing the charge defects of CF mucin. Here, we report efficacy in the CFTR-sufficient, ENaC hyperactive, Scnn1b-Tg mouse model that develops airway muco-obstruction due to sodium hyperabsorption and airway dehydration.METHODSScnn1b-Tg mice were treated with either 250 µg/mL SNSP113 or vehicle control (1.38% glycerol in PBS) via nebulization once daily for 7 days and then euthanized for analysis. Micro-Optical Coherence Tomography-based evaluation of excised mouse trachea was used to determine the effect on the functional microanatomy. Tissue analysis was performed by routine histopathology.RESULTSNebulized treatment of SNSP113 significantly improved mucociliary transport in the airways of Scnn1b-Tg mice, without altering the airway surface or periciliary liquid layer. In addition, SNSP113 significantly reversed epithelial hypertrophy and goblet cell metaplasia. Finally, SNSP113 significantly ameliorated eosinophilic crystalline pneumonia and lung consolidation in addition to inflammatory macrophage influx in this model.CONCLUSIONOverall, this study extends the efficacy of SNSP113 as a potential therapeutic to alleviate mucus stasis in muco-obstructive diseases in CF and potentially in related conditions.

100 Deals associated with SNSP-113

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	Phase 2	HU	Anagram Therapeutics, Inc.	29 Oct 2019
Staphylococcal Infections	Phase 2	US	Synedgen, Inc.	24 Dec 2017
Antibiotic resistant infection	Phase 2	GB	Anagram Therapeutics, Inc.	28 Sep 2017
Burkholderia Infections	Phase 2	GB	Anagram Therapeutics, Inc.	28 Sep 2017
Infectious Lung Disorder	Phase 2	GB	Anagram Therapeutics, Inc.	28 Sep 2017
Mycobacterium Infections, Nontuberculous	Phase 2	GB	Anagram Therapeutics, Inc.	28 Sep 2017
Pneumonia	Phase 2	GB	Anagram Therapeutics, Inc.	28 Sep 2017
Pulmonary Cystic Fibrosis	Phase 2	GB	Anagram Therapeutics, Inc.	28 Sep 2017

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