Delving into the Latest Updates on Acebilustat with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Target

LTA4H

Action

inhibitors

Mechanism

LTA4H inhibitors(Leukotriene A-4 hydrolase inhibitors)

Therapeutic Areas

Congenital DisordersDigestive System DisordersRespiratory Diseases+ [3]

Active Indication-

Inactive Indication

Acne VulgarisCystic FibrosisPneumonia

Originator Organization

Celtaxsys, Inc.

Active Organization-

Inactive Organization

Celtaxsys, Inc.

Drug Highest PhasePendingPhase 2

First Approval Date-

Regulation-

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Structure/Sequence

Molecular FormulaC29H27N3O4

InChIKeyGERJIEKMNDGSCS-DQEYMECFSA-N

CAS Registry943764-99-6

This study is designed to investigate the response of unilateral upper extremity (arm) lymphedema, during pharmacologic treatment of lymphedema with oral placebo and oral acebilustat. Participants will receive "study drug" (Acebilustat or placebo), for 9 months. For 3 of these months, the participant will receive placebo; for 6 of these months, the participant will receive active ingredient, acebilustat. The study is blinded which means that the participant will not be told which study pill they are taking.

Start Date27 Jul 2022

Sponsor / Collaborator

Stanford University

NCT04662060

/ CompletedPhase 2IIT

COVID-19 Outpatient Pragmatic Platform Study (COPPS): A Pragmatic Multi-arm, Adaptive, Phase 2, Blinded, Randomized Placebo-controlled Platform Trial to Assess the Efficacy of Different Investigational Therapeutics in Reducing Time to Disease Resolution or Viral Load Cessation, as Compared to Standard Supportive Care in Outpatients With COVID-19

The overall objective of this study is to efficiently evaluate the clinical efficacy and safety of different investigational therapeutics among adults who have COVID-19 but are not yet sick enough to require hospitalization. The overall hypothesis is that through an adaptive trial design, potential effective therapies (single and combination) may be identified for this group of patients.
COVID-19 Outpatient Pragmatic Platform Study (COPPS) is a pragmatic platform protocol designed to evaluate COVID-19 treatments by assessing their ability to reduce viral shedding (Viral Domain) or improve clinical outcomes (Clinical Domain). To be included into the platform, every investigational product will collect data for both Domain primary endpoints. Individual treatments to be evaluated in the platform will be described in separate sub-protocols.

Start Date23 Apr 2021

Sponsor / Collaborator

Stanford University

NCT04662086

/ CompletedPhase 2IIT

COVID-19 Outpatient Pragmatic Platform Study (COPPS): A Pragmatic Multi-arm, Adaptive, Phase 2, Blinded, Randomized Placebo-controlled Platform Trial to Assess the Efficacy of Different Investigational Therapeutics in Reducing Time to Disease Resolution or Viral Load Cessation, as Compared to Standard Supportive Care in Outpatients With COVID-19

The overall objective of this study is to efficiently evaluate the clinical efficacy and safety of different investigational therapeutics among adults who have COVID-19 but are not yet sick enough to require hospitalization. The overall hypothesis is that through an adaptive trial design, potential effective therapies (single and combination) may be identified for this group of patients.
COVID-19 Outpatient Pragmatic Platform Study (COPPS) is a pragmatic platform protocol designed to evaluate COVID-19 treatments by assessing their ability to reduce viral shedding (Viral Domain) or improve clinical outcomes (Clinical Domain). To be included into the platform, every investigational product will collect data for both Domain primary endpoints. Individual treatments to be evaluated in the platform will be described in separate sub-protocols.

Start Date23 Apr 2021

Sponsor / Collaborator

Stanford University

100 Clinical Results associated with Acebilustat

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100 Translational Medicine associated with Acebilustat

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100 Patents (Medical) associated with Acebilustat

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13

Literatures (Medical) associated with Acebilustat

01 May 2024·Current Opinion in Cardiology

Genetic causes of lymphatic disorders: recent updates on the clinical and molecular aspects of lymphatic disease

Review

Author: Rockson, Stanley G ; Bowman, Catharine

Purpose of reviewThe lymphatic system facilitates several key functions that limit significant morbidity and mortality. Despite the impact and burden of lymphatic disorders, there are many remaining disorders whose genetic substrate remains unknown. The purpose of this review is to provide an update on the genetic causes of lymphatic disorders, while reporting on newly proposed clinical classifications of lymphatic disease.Recent findingsWe reviewed several new mutations in genes that have been identified as potential causes of lymphatic disorders including: MDFIC, EPHB4, and ANGPT2. Furthermore, the traditional St. George's Classification system for primary lymphatic anomalies has been updated to reflect the use of genetic testing, both as a tool for the clinical identification of lymphatic disease and as a method through which new sub-classifications of lymphatic disorders have been established within this framework. Finally, we highlighted recent clinical studies that have explored the impact of therapies such as sirolimus, ketoprofen, and acebilustat on lymphatic disorders.SummaryDespite a growing body of evidence, current literature demonstrates a persistent gap in the number of known genes responsible for lymphatic disease entities. Recent clinical classification tools have been introduced in order to integrate traditional symptom- and time-based diagnostic approaches with modern genetic classifications, as highlighted in the updated St. George's classification system. With the introduction of this novel approach, clinicians may be better equipped to recognize established disease and, potentially, to identify novel causal mutations. Further research is needed to identify additional genetic causes of disease and to optimize current clinical tools for diagnosis and treatment.

01 Apr 2024·Cell Reports

Targeting the GPI transamidase subunit GPAA1 abrogates the CD24 immune checkpoint in ovarian cancer

Article

Author: Kelliher, Michelle A ; Ali, Amjad ; Chowdhury, Shreya Roy ; Thakare, Ritesh P ; Green, Michael R ; Malonia, Sunil K ; Ye, Tianyi ; Zhu, Lihua Julie ; Hu, Kai ; Eisenhaber, Frank ; Kulshreshtha, Ankur ; Su, Chinh Tran-To ; Deibler, Sara K ; Banday, Shahid ; Eisenhaber, Birgit ; Simin, Karl ; Mishra, Alok K ; Pham, Ngoc N H ; Simone, Tessa M ; Thompson, Paul R

CD24 is frequently overexpressed in ovarian cancer and promotes immune evasion by interacting with its receptor Siglec10, present on tumor-associated macrophages, providing a "don't eat me" signal that prevents targeting and phagocytosis by macrophages. Factors promoting CD24 expression could represent novel immunotherapeutic targets for ovarian cancer. Here, using a genome-wide CRISPR knockout screen, we identify GPAA1 (glycosylphosphatidylinositol anchor attachment 1), a factor that catalyzes the attachment of a glycosylphosphatidylinositol (GPI) lipid anchor to substrate proteins, as a positive regulator of CD24 cell surface expression. Genetic ablation of GPAA1 abolishes CD24 cell surface expression, enhances macrophage-mediated phagocytosis, and inhibits ovarian tumor growth in mice. GPAA1 shares structural similarities with aminopeptidases. Consequently, we show that bestatin, a clinically advanced aminopeptidase inhibitor, binds to GPAA1 and blocks GPI attachment, resulting in reduced CD24 cell surface expression, increased macrophage-mediated phagocytosis, and suppressed growth of ovarian tumors. Our study highlights the potential of targeting GPAA1 as an immunotherapeutic approach for CD24⁺ ovarian cancers.

26 Jul 2023·Clinical infectious diseases : an official publication of the Infectious Diseases Society of America

Evaluation of Acebilustat, a Selective Inhibitor of Leukotriene B4 Biosynthesis, for Treatment of Outpatients With Mild-Moderate Coronavirus Disease 2019: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial.

Article

Author: Lee, Justin ; Moss, Richard B ; Lu, Di ; Desai, Manisha ; Langguth, Jamison J ; Duong, Sophie ; Parsonnet, Julie ; Pinsky, Benjamin A ; Hedlin, Haley ; Springman, Eric ; Khosla, Chaitan ; Nicolls, Mark R ; Bollyky, Jenna ; Rogers, Angela J ; Bunning, Bryan ; Bonilla, Hector F ; Levitt, Joseph E ; Elkarra, Nadia ; Zamanian, Roham T ; Heffernan, Eileen

BACKGROUNDThe vast majority of coronavirus disease 2019 (COVID-19) disease occurs in outpatients where treatment is limited to antivirals for high-risk subgroups. Acebilustat, a leukotriene B4 inhibitor, has potential to reduce inflammation and symptom duration.METHODSIn a single-center trial spanning Delta and Omicron variants, outpatients were randomized to 100 mg/d of oral acebilustat or placebo for 28 days. Patients reported daily symptoms via electronic query through day 28 with phone follow-up on day 120 and collected nasal swab samples on days 1-10. The primary outcome was sustained symptom resolution to day 28. Secondary 28-day outcomes included time to first symptom resolution, area under the curve (AUC) for longitudinal daily symptom scores, duration of viral shedding through day 10, and symptoms on day 120.RESULTSSixty participants were randomized to each study arm. At enrollment, the median duration was 4 days (interquartile range, 3-5 days), and the median number of symptoms was 9 (7-11). Most patients (90%) were vaccinated, with 73% having neutralizing antibodies. A minority of participants (44%; 35% in the acebilustat arm and 53% in placebo) had sustained symptom resolution at day 28 (hazard ratio, 0.6 [95% confidence interval, .34-1.04]; P = .07 favoring placebo). There was no difference in the mean AUC for symptom scores over 28 days (difference in mean AUC, 9.4 [95% confidence interval, -42.1 to 60.9]; P = .72). Acebilustat did not affect viral shedding or symptoms at day 120.CONCLUSIONSSustained symptoms through day 28 were common in this low-risk population. Despite this, leukotriene B4 antagonism with acebilustat did not shorten symptom duration in outpatients with COVID-19. Clinical Trials Registration. NCT04662060.

100 Deals associated with Acebilustat

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External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15385

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	Phase 2	Canada	Celtaxsys, Inc.	30 Oct 2015
Cystic Fibrosis	Phase 2	Belgium	Celtaxsys, Inc.	30 Oct 2015
Cystic Fibrosis	Phase 2	Germany	Celtaxsys, Inc.	30 Oct 2015
Cystic Fibrosis	Phase 2	United States	Celtaxsys, Inc.	30 Oct 2015
Cystic Fibrosis	Phase 2	Italy	Celtaxsys, Inc.	30 Oct 2015
Cystic Fibrosis	Phase 2	France	Celtaxsys, Inc.	30 Oct 2015
Cystic Fibrosis	Phase 2	United Kingdom	Celtaxsys, Inc.	30 Oct 2015
Acne Vulgaris	Phase 2	Australia	Celtaxsys, Inc.	01 Apr 2015
Acne Vulgaris	Phase 2	New Zealand	Celtaxsys, Inc.	01 Apr 2015
Pneumonia	Phase 2	Australia	Celtaxsys, Inc.	01 Dec 2012

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04662060 (COPPS, CTgov)	Phase 2	COVID-19	120	Acebilustat	ryinvfysoi(vbtuoqetsy) = rpwdhbqdyf mktukoqiqk (ayyrrqxufl, vqfvjlokqr - tvgalecylq) View more	-	13 Jun 2023
NCT04662086 (CTgov)	Phase 2	COVID-19	122	Acebilustat (Acebilustat)	hvusujojme(moszdhkvwp) = ytarhvljeb djizezfxnu (xylqcpknzh, dqdguauzut - jnckscuyfa) View more	-	30 May 2023
				Camostat (Camostat)	hvusujojme(moszdhkvwp) = tqadkdubza djizezfxnu (xylqcpknzh, xdptilxbjt - dkgkuznwrt) View more
NCT04662086 \| NCT04662060 (Pubmed)	Phase 2	COVID-19	120	Acebilustat 100 mg	jawtsvegjo(dnjqakmqds) = kjwxnbdyrd ugvulfpsaf (gmzmrmbbjq )	Negative	30 Mar 2023
				Placebo	jawtsvegjo(dnjqakmqds) = uxfemxkalh ugvulfpsaf (gmzmrmbbjq )
NCT02443688 (CTgov)	Phase 2	Cystic Fibrosis	200	CTX-4430 (100 mg CTX-4430)	giyggncurk(kztsmrrlcg) = abcakpqwyt grqvtjsjsf (atsghzfefk, qpvrdrsikn - hcpzhfzoyl) View more	-	25 Jul 2019
				CTX-4430 (50 mg CTX-4430)	giyggncurk(kztsmrrlcg) = xukiiagkkr grqvtjsjsf (atsghzfefk, ywntbgygbt - shszmlhmut) View more