Alnylam Pharmaceuticals — Biomedical Watch V2

Traitements9programmes

Essais4liés

Publications3liées

SourceDBlocale

Traitements

9

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
ALN-5288The purpose of this study is to: * Evaluate the safety and tolerability of intrathecal (IT) ALN-5288 in patients with Alzheimer's Disease (AD) * Evaluate the pharmacodynamic (PD) and pharmacokinetic (PK) effects of ALN-5288 after dose administration	Alzheimer	Phase 1	À vérifier	Canada, Netherlands, Spain, United Kingdom	À vérifier
ALN-AGT01 RVRThe purpose of this study is to evaluate the efficacy, pharmacodynamics (PD), and safety of ALN-AGT01 RVR in participants with mild to moderate hypertension pretreated with zilebesiran.	Hypertension	Phase 2	À vérifier	United States	À vérifier
ALN-APPThe purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.	Alzheimer	Phase 1	À vérifier	United States, Canada, Netherlands, United Kingdom	À vérifier
MivelsiranThe purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.	Alzheimer	Phase 2	À vérifier	United States, Spain	À vérifier
ZilebesiranThe purpose of this study is to evaluate the efficacy, pharmacodynamics (PD), and safety of ALN-AGT01 RVR in participants with mild to moderate hypertension pretreated with zilebesiran.	Hypertension	Phase 2	À vérifier	United States	À vérifier
ALN-APPThe purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.	Alzheimer	Phase 1	À vérifier	United States, Canada, Netherlands, United Kingdom	À vérifier
MivelsiranThe purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.	Alzheimer	Phase 2	À vérifier	United States, Spain	À vérifier
ALN-5288The purpose of this study is to: * Evaluate the safety and tolerability of intrathecal (IT) ALN-5288 in patients with Alzheimer's Disease (AD) * Evaluate the pharmacodynamic (PD) and pharmacokinetic (PK) effects of ALN-5288 after dose administration	Alzheimer	Phase 1	À vérifier	Canada, Netherlands, Spain, United Kingdom	À vérifier
ALN-AGT01 RVRThe purpose of this study is to evaluate the efficacy, pharmacodynamics (PD), and safety of ALN-AGT01 RVR in participants with mild to moderate hypertension pretreated with zilebesiran.	Hypertension	Phase 2	À vérifier	United States	À vérifier

Essais cliniques

4

Molécule	Indication / population	Phase	NCT	Titre	Statut
ALN-AGT01 RVR	Hypertension	Phase 2	NCT07553442	A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran	RECRUITING
ALN-5288	Alzheimer	Phase 1	NCT07214727	A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease	RECRUITING
Mivelsiran	Alzheimer	Phase 2	NCT07636811	A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)	RECRUITING
ALN-APP	Alzheimer	Phase 1	NCT05231785	A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD	RECRUITING

Publications

3

Molécule	Indication / population	Titre	Journal	Date
Zilebesiran		Angiotensinogen Reconsidered: Evolving Perspectives in Hypertension Research.	Hypertension (Dallas, Tex. : 1979)
Zilebesiran		Breakthrough drugs in the treatment of hypertension-related disease.	Hypertension research : official journal of the Japanese Society of Hypertension
Zilebesiran		Hypertension at an Inflection Point: Aggressive Targets Meet Novel Mechanisms for Cardiovascular Prevention.	Journal of the American College of Cardiology