Delving into the Latest Updates on MY-008211A with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Target

CFB

Action

inhibitors

Mechanism

CFB inhibitors(Complement factor B inhibitors)

Therapeutic Areas

Immune System DiseasesHemic and Lymphatic DiseasesUrogenital Diseases+ [3]

Active Indication

Hemoglobinuria, ParoxysmalGlomerulonephritis, IGAC3 glomerulopathy+ [3]

Inactive Indication-

Originator Organization

Shanghai Meiyue Biotech Development Co. Ltd.

Active Organization

Wuhan Createrna Science and Technology Co.,Ltd.

Inactive Organization-

License Organization-

Drug Highest PhasePhase 3

First Approval Date-

RegulationOrphan Drug (United States)

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AbstractThe MYCN gene encodes the transcription factor N-Myc, a driver of neuroblastoma (NB). Targeting G-quadruplexes (G4s) with small molecules is attractive strategy to control the expression of undruggable proteins such as N-Myc. However, selective binders to G4s are challenging to identify due to the structural similarity of many G4s. Here, we report the discovery of a small molecule ligand (4) that targets the noncanonical, hairpin containing G4 structure found in the MYCN gene using small molecule microarrays (SMMs). Unlike many G4 binders, the compound was found to bind to a pocket at the base of the hairpin region of the MYCN G4. This compound stabilizes the G4 and has affinity of 3.5 ± 1.6 μM. Moreover, an improved analog, MY-8, suppressed levels of both MYCN and MYCNOS (a lncRNA embedded within the MYCN gene) in NBEB neuroblastoma cells. This work indicates that the approach of targeting complex, hybrid G4 structures that exist throughout the human genome may be an applicable strategy to achieve selectivity for targeting disease-relevant genes including protein coding (MYCN) as well as non-coding (MYCNOS) gene products.

100 Deals associated with MY-008211A

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemoglobinuria, Paroxysmal	Phase 3	China	Wuhan Createrna Science and Technology Co.,Ltd.	30 Aug 2024
Glomerulonephritis, IGA	Phase 2	China	Wuhan Createrna Science and Technology Co.,Ltd.	30 Nov 2024
C3 glomerulopathy	IND Approval	China	Wuhan Createrna Science and Technology Co.,Ltd.	18 Dec 2023
Diabetic Nephropathies	IND Approval	China	Wuhan Createrna Science and Technology Co.,Ltd.	18 Dec 2023
Idiopathic Membranous Glomerulonephritis	IND Approval	China	Wuhan Createrna Science and Technology Co.,Ltd.	18 Dec 2023
Lupus Nephritis	IND Approval	China	Wuhan Createrna Science and Technology Co.,Ltd.	18 Dec 2023

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06134414 (ASH2024)	Phase 2	Hemoglobinuria, Paroxysmal	-	MY008211A 400 mg bid	oqvyktqfsg(vxanhhnwjh) = n=15 of 34 patients cyfqqqtebg (ayrswpvdef ) View more	-	08 Dec 2024
				MY008211A 600 mg bid