Delving into the Latest Updates on AON-D21 with Synapse

Overview

Basic Info

Drug Type

RNA aptamer

Synonyms

Target

C5a

Action

inhibitors

Mechanism

C5a inhibitors(complement C5a inhibitors)

Therapeutic Areas

Infectious DiseasesRespiratory DiseasesNeoplasms+ [2]

Active Indication

Community Acquired PneumoniaLung CancerHemoglobinuria, Paroxysmal

Inactive Indication-

Originator Organization

Aptarion Biotech AgStartup

Active Organization

Aptarion Biotech AgStartup

University of Louisville

Inactive Organization-

Drug Highest PhasePhase 2

First Approval Date-

Regulation-

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Structure/Sequence

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The goal of this clinical trial is to compare the safety and efficacy of AON-D21 versus placebo, both on top of standard of care, in patients with severe community acquired pneumonia admitted to ICU (or similar unit). The main questions to answer are:
* The safety and tolerability of AON-D21 vs placebo.
* The efficacy of AON-D21vs placebo.
* The pharmacokinetics of AON-D21.
* The pharmacodynamics of AON D21.
* To identify biomarkers for patient stratification and analyses in future trials.

Start Date02 Feb 2024

Sponsor / Collaborator

Aptarion Biotech AgStartup

NCT05343819

/ CompletedPhase 1

A Randomized, Single-center, Double-blind, Placebo Controlled Trial With Ascending Multiple Intravenous Doses to Determine Safety, Tolerability and Pharmacokinetics of AON-D21 in Healthy Male Subjects.

The main purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic parameters after multiple ascending intravenous doses of AON-D21 in healthy male subjects.

Start Date12 Apr 2022

Sponsor / Collaborator

Aptarion Biotech AgStartup

NCT05018403

/ CompletedPhase 1

A Randomized, Single-center, Double-blind, Placebo Controlled, First-in-human Trial With Single Ascending Intravenous Doses to Determine Safety, Tolerability and Pharmacokinetics of AON-D21 in Healthy Male Subjects

The main purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic parameters after single ascending intravenous doses of AON-D21 in healthy male subjects.

Start Date03 Aug 2021

Sponsor / Collaborator

Aptarion Biotech AgStartup

100 Clinical Results associated with AON-D21

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100 Translational Medicine associated with AON-D21

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100 Patents (Medical) associated with AON-D21

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1

Literatures (Medical) associated with AON-D21

01 Dec 2017·Stem Cell Reviews and ReportsQ3 · MEDICINE

Signaling of the Complement Cleavage Product Anaphylatoxin C5a Through C5aR (CD88) Contributes to Pharmacological Hematopoietic Stem Cell Mobilization

Q3 · MEDICINE

Article

Author: Ratajczak, Mariusz Z ; Yan, Jun ; Vater, Axel ; Bujko, Kamila ; Hoehlig, Kai ; Rzeszotek, Sylwia

Several mechanisms have been postulated for orchestrating the mobilization of hematopoietic stem/progenitor cells (HSPCs), and we previously proposed that activation of the complement cascade plays a crucial role in the initiation and execution of the egress of HSPCs from bone marrow (BM) into peripheral blood (PB). In support of this notion, we demonstrated that mice deficient in the mannan-binding lectin (MBL) pathway, which activates the proximal part of the complement cascade, as well as mice deficient in the fifth component of the complement cascade (C5), which is part of the distal part of the complement cascade, are poor mobilizers. To further narrow down on the exact mechanisms and the molecules involved, we performed studies in mice that do not express the receptor C5aR, which binds the C5 cleavage fragments, C5a and C5a_desArg. We also employed the plasma stable nucleic acid aptamer AON-D21 that binds and neutralizes C5a and C5a_desArg. We present evidence that mice deficient in C5aR or treated with AON-D21 are poor HSPC mobilizers, thereby establishing a critical role for the C5a/C5a_desArg-C5aR axis in the mobilization process. While enhancing mobilization is of clinical importance for poor mobilizers, inhibition of the complement cascade could be of therapeutic importance in patients suffering from paroxysmal nocturnal hemoglobinuria (PNH) or acquired hemolytic syndrome (aHUS).

100 Deals associated with AON-D21

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Community Acquired Pneumonia	Phase 2	Belgium	Aptarion Biotech AgStartup	02 Feb 2024
Community Acquired Pneumonia	Phase 2	France	Aptarion Biotech AgStartup	02 Feb 2024
Community Acquired Pneumonia	Phase 2	Germany	Aptarion Biotech AgStartup	02 Feb 2024
Community Acquired Pneumonia	Phase 2	United Kingdom	Aptarion Biotech AgStartup	02 Feb 2024
Community Acquired Pneumonia	Phase 2	Spain	Aptarion Biotech AgStartup	02 Feb 2024
Lung Cancer	Phase 1	Germany	Aptarion Biotech AgStartup	25 Aug 2022
Hemoglobinuria, Paroxysmal	Preclinical	Germany	Aptarion Biotech AgStartup	07 Dec 2017
Hemoglobinuria, Paroxysmal	Preclinical	United States	University of Louisville	07 Dec 2017